0001558370-25-000953 | 10-K | XBRL Viewer

Schedule of activity within the liability account and proceeds received


	Year Ended December 31,
	2024		2023
Beginning balance at January 1	$	266,670	$	254,328
Interest expense recognized		27,134		28,065
Payments		(39,368)		(15,723)
Carrying value at December 31	$	254,436	$	266,670

Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities

Schedule of activity within the liability account and proceeds received


	Year Ended December 31,
	2024		2023
Beginning balance at January 1	$	238,813	$	139,083
Net proceeds received		146,973		97,933
Interest expense recognized		41,668		23,540
Payments		(40,484)		(21,743)
Carrying value at December 31	$	386,970	$	238,813

Tabular disclosure of long-debt instruments or arrangements, including identification, terms, features, collateral requirements and other information necessary to a fair presentation. These are debt arrangements that originally required repayment more than twelve months after issuance or greater than the normal operating cycle of the entity, if longer.

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Subsequent Events (Details) - Subsequent Event - Forecast - GSK plc - IDRx, Inc. $ in Millions	3 Months Ended
	Mar. 31, 2025 USD ($)
Business Combination, Consideration Transferred
Business combination, consideration transferred	$ 1,000.0
Business Combination, Contingent Consideration Arrangements
Business combination, contingent consideration arrangements, range of outcomes, value, high	$ 150.0

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2024

Schedule of the reconciliation of the effective tax rate


	Year Ended December 31,
	2024		2023		2022
Federal income tax (benefit) at statutory rate	21.00	%	21.00	%	21.00	%
Permanent differences	0.82		(0.42)		(0.38)
U.S. Tax on foreign earnings	—		—		2.52
Federal research and development credits	14.43		2.50		1.39
Federal orphan drug credits	18.41		1.63		(0.42)
State income tax, net of federal benefit	7.91		3.41		2.92
Foreign rate differential	(13.47)		(0.41)		(0.13)
Deferred rate change	(9.21)		2.53		0.52
Foreign tax credit	—		—		0.03
Stock Based Compensation	(5.33)		(0.76)		(1.03)
Other	(0.27)		(0.02)		0.39
Change in valuation allowance	(36.17)		(29.65)		(27.76)
Effective income tax rate	(1.88)	%	(0.19)	%	(0.95)	%

Schedule of the deferred tax assets and liabilities


	As of December 31,
	2024		2023
Deferred tax assets:
Net operating loss carryforwards	$	244,189	$	232,624
Research and development credit carryforwards		67,592		53,329
Orphan drug credit carryforwards		168,799		156,816
Accrued expenses and other		62,187		60,350
Royalty Pharma milestones		—		12,024
R&D capitalization		190,040		153,836
Financing arrangements		63,424		107,693
Deferred rent		19,040		22,204
Other		3,785		1,488
Total gross deferred tax asset		819,056		800,364
Deferred tax liability
Depreciation		(6,123)		(6,107)
Right of use assets		(14,837)		(17,423)
Other		—		(2,068)
Valuation allowance		(798,096)		(774,766)
Net deferred tax asset	$	—	$	—

Schedule of unrecognized tax benefits


	Year ended December 31,
	2024		2023
Beginning balance at January 1	$	211	$	—
Gross Increases - tax positions in prior periods		—		211
Gross Increases - current period tax positions		—		—
Settlements		(211)		—
Lapse of statutes		—		—
Ending balance at December 31	$	—	$	211

Inventory

12 Months Ended

Dec. 31, 2024

Inventory

7. Inventory

Capitalized inventory consists of the following as of December 31, 2024 and 2023 (in thousands):


	As of December 31,
	2024		2023
Raw materials	$	—	$	3,147
Work in process		30,300		29,132
Finished goods		8,975		4,823
Total	$	39,275	$	37,102

Balance sheet classification


	As of December 31,
	2024		2023
Inventory	$	13,611	$	21,223
Other assets		25,664		15,879
Total	$	39,275	$	37,102

Inventory amounts written down as a result of excess, obsolescence, unmarketability or other reasons are charged to cost of sales. For the years ended December 31, 2024, 2023 and 2022, the Company recognized write-downs of $0.4 million, $0.7 million and $2.1 million, respectively. Long-term inventory, which primarily consists of work in process is included in other assets in the consolidated balance sheets.

Income Taxes - Unrecognized Tax Benefits - General Information (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Unrecognized tax benefits
Unrecognized tax benefits	$ 0	$ 211	$ 0

Document and Entity Information - USD ($)	12 Months Ended
Document and Entity Information - USD ($)	Dec. 31, 2024	Feb. 11, 2025	Jun. 30, 2024
Cover [Abstract]
Entity Central Index Key	0001597264
Document Type	10-K
Document Annual Report	true
Document Period End Date	Dec. 31, 2024
Document Transition Report	false
Entity File Number	001-37359
Entity Registrant Name	BLUEPRINT MEDICINES CORPORATION
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	26-3632015
Entity Address, Address Line One	45 Sidney Street
Entity Address, City or Town	Cambridge
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02139
City Area Code	617
Local Phone Number	374-7580
Title of 12(b) Security	Common Stock, par value $0.001 per share
Trading Symbol	BPMC
Security Exchange Name	NASDAQ
Entity Well-known Seasoned Issuer	Yes
Entity Voluntary Filers	No
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Document Financial Statement Error Correction [Flag]	false
Entity Shell Company	false
ICFR Auditor Attestation Flag	true
Entity Listing, Par Value Per Share	$ 0.001
Entity Public Float			$ 6,747,459,120
Entity Common Stock, Shares Outstanding		63,906,011
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2024
Document Fiscal Period Focus	FY
Amendment Flag	false
Auditor Name	Ernst & Young LLP
Auditor Firm ID	42
Auditor Location	Boston, Massachusetts

Restricted Cash - Tabular Disclosure (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021
Reconciliation of cash, cash equivalents, and restricted cash
Cash and cash equivalents	$ 102,014	$ 71,286	$ 119,709
Restricted cash	11,625	10,238	5,195
Total cash, cash equivalents, and restricted cash shown in condensed consolidated statements of cash flows	$ 113,639	$ 81,524	$ 124,904	$ 215,119

Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 761,923	$ 695,885
U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	129,785	169,803
U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	632,138	526,082
Recurring
Fair Value of Financial Instruments
Total	831,652	751,297
Recurring \| U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	129,785	169,803
Recurring \| U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	632,138	526,082
Recurring \| Money market funds
Fair Value of Financial Instruments
Cash equivalents	69,729	55,412
Recurring \| Active Markets (Level 1)
Fair Value of Financial Instruments
Total	701,867	581,494
Recurring \| Active Markets (Level 1) \| U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	632,138	526,082
Recurring \| Active Markets (Level 1) \| Money market funds
Fair Value of Financial Instruments
Cash equivalents	69,729	55,412
Recurring \| Observable Inputs (Level 2)
Fair Value of Financial Instruments
Total	129,785	169,803
Recurring \| Observable Inputs (Level 2) \| U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 129,785	$ 169,803

Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Revenues:
Revenue	$ 508,824	$ 249,380	$ 204,036
Cost and operating expenses:
Cost of sales	20,163	8,540	17,813
Research and development	341,433	427,720	477,419
Selling, general and administrative	359,272	295,141	237,374
Collaboration loss sharing		4,256	8,948
Total cost and operating expenses	720,868	735,657	741,554
Other income (expense):
Interest expense, net	(28,151)	(18,793)	(16,767)
Other income (expense), net	656	(946)	2,004
Debt extinguishment gain	173,676
Total other income (expense)	146,181	(19,739)	(14,763)
Loss before income taxes	(65,863)	(506,016)	(552,281)
Income tax expense	1,226	968	5,236
Net loss	(67,089)	(506,984)	(557,517)
Other comprehensive income (loss):
Unrealized gain (loss) on pension benefit obligations	(761)	(2,781)	666
Unrealized gain (loss) on available-for-sale investments	702	9,759	(7,246)
Currency translation adjustments	3	(30)	270
Comprehensive loss	$ (67,145)	$ (500,036)	$ (563,827)
Net loss per share - basic (in dollars per share)	$ (1.07)	$ (8.37)	$ (9.35)
Net loss per share - diluted (in dollars per share)	$ (1.07)	$ (8.37)	$ (9.35)
Weighted-average number of common shares used in net loss per share - basic (in shares)	62,857	60,558	59,642
Weighted-average number of common shares used in net loss per share - diluted (in shares)	62,857	60,558	59,642
Product revenue, net
Revenues:
Revenue	$ 478,950	$ 204,207	$ 110,993
Product revenue, net \| Nonrelated Party
Revenues:
Revenue	478,950	204,207	110,993
Collaboration, License and Other \| Nonrelated Party
Revenues:
Revenue	$ 29,874	$ 45,173	65,543
License \| Related Party
Revenues:
Revenue			$ 27,500

Marketable Securities - Impairment (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Marketable Securities
Differences between the fair value and amortized cost basis recognized as a loss in its consolidated statements of operations and comprehensive loss	$ 0	$ 0	$ 0
Marketable Securities
Marketable debt securities, charges for credit-related impairments	$ 0	$ 0	$ 0

Nature of Business (Details) - USD ($) $ in Millions	Dec. 31, 2024	Dec. 31, 2023
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities	$ 863.9	$ 767.2

Collaboration, License and Other Agreements - Performance Obligations (Details)	1 Months Ended	12 Months Ended
	Mar. 31, 2016 item	Dec. 31, 2024 item component
Collaborative Arrangement \| IDRx, Inc. \| Director
Performance Obligations
Collaborative arrangement, material promises, number		2
Collaborative arrangement, material promises, combined into distinct performance obligation, number		1
Collaborative Arrangement \| Zai Lab (Shanghai) Co., Ltd.
Performance Obligations
Collaborative arrangement, material components, number \| component		2
Revenue, performance obligation, performance obligations, number		1
Collaborative Arrangement \| Roche, Collaboration (Pralsetnib) Agreement
Performance Obligations
Collaborative arrangement, material promises, number		2
Collaborative Arrangement \| Roche, Collaboration and License (Immunotherapy) Agreement
Performance Obligations
Revenue, performance obligation, performance obligations, number	1
Collaborative Arrangement \| Clementia
Performance Obligations
Collaborative arrangement, material promises, number		4
Revenue, performance obligation, performance obligations, number		3
Collaborative Arrangement \| C Stone
Performance Obligations
Collaborative arrangement, material promises, number		6
Collaborative arrangement, material components, number \| component		2
Revenue, performance obligation, performance obligations, number		3
Arrangement Other than Collaborative, Asset Purchase Agreement \| Rigel Pharmaceuticals, Inc.
Performance Obligations
Revenue, performance obligation, performance obligations, number		3

Stock-based Compensation (Tables)

12 Months Ended

Dec. 31, 2024

Assumptions used in estimating fair value of stock options on grant date


	Year Ended December 31,
	2024		2023		2022
Risk-free interest rate	4.26	%	4.13	%	2.27	%
Expected dividend yield	—		—		—
Expected term (years)	6.0		6.0		6.0
Expected stock price volatility	55.54	%	56.01	%	55.15	%

Summary of stock option activity


		Weighted-		Remaining	Aggregate
		Average		Contractual	Intrinsic
		Exercise		Life	Value(1)
	Shares	Price		(in Years)	(in thousands)
Outstanding at December 31, 2023	6,658,444	$	66.48	6.39	$	178,581
Granted ²	1,075,799		95.77
Exercised	(1,159,269)		61.96
Canceled	(205,250)		75.42
Outstanding at December 31, 2024	6,369,724	$	72.09	6.14	$	115,874
Exercisable at December 31, 2024	4,514,741	$	71.51	5.20	$	82,016

(1)	Intrinsic value represents the amount by which the fair market value as of December 31, 2024 of the underlying common stock exceeds the exercise price of the option.

(2)	Out of stock options granted in 2024, 55,119 stock options were granted under the 2024 Plan.

Summary of restricted stock units activity


		Weighted-Average
		Grant Date
	Shares	Fair Value
Unvested shares at December 31, 2023	2,107,026	$	60.33
Granted ¹	1,130,316		95.24
Vested	(795,219)		64.77
Forfeited	(151,192)		70.34
Unvested shares at December 31, 2024	2,290,931	$	75.36

(1)

Out of restricted stock units granted in 2024, 192,868 restricted stock units were granted under the 2024 Plan.

Summary of performance-based restricted stock units activity


		Weighted-Average
		Grant Date
	Shares	Fair Value
Unvested shares at December 31, 2023	52,500	$	59.32
Granted	90,000		135.84
Vested	—		—
Forfeited	—		—
Unvested shares at December 31, 2024	142,500	$	107.65

Summary of stock-based compensation expense, allocation by type of awards and recognition in statements of operations

The Company recognized stock-based compensation expense totaling $108.9 million, $92.7 million and $99.0 million for the years ended December 31, 2024, 2023 and 2022, respectively. Stock-based compensation expense by award type included within the consolidated statements of operations and comprehensive loss is as follows (in thousands):


	Year Ended December 31,
	2024		2023			2022
Stock options	$	42,031	$	40,560	$	52,931
Restricted stock units		61,722		50,095		45,285
Performance-based restricted stock units		4,445		867		—
Employee stock purchase plan		1,722		1,734		1,418
Subtotal		109,920		93,256		99,634
Capitalized stock-based compensation costs		(1,009)		(584)		(663)
Stock-based compensation expense included in total cost and operating expenses	$	108,911	$	92,672	$	98,971

The following table presents stock-based compensation expense that is included in operating expenses by classification within the consolidated statements of operations and comprehensive loss (in thousands):


	Year Ended December 31,
	2024		2023		2022
Research and development	$	47,455	$	41,534	$	40,302
Selling, general and administrative		61,456		51,138		58,669
Total stock-based compensation expense included in operating expenses	$	108,911	$	92,672	$	98,971

Financing Arrangements - Long-term Debt (Details) - Sale of Future Royalties and Revenues - USD ($) $ in Thousands	Dec. 31, 2024	Feb. 22, 2024	Dec. 31, 2023	Dec. 31, 2022
Royalty Pharma, Royalty Purchase Agreement
Long-term Debt
Long-term debt	$ 0	$ 0
Sixth Street Partners, Future Revenue Purchase Agreement
Long-term Debt
Long-term debt	$ 254,436		$ 266,670	$ 254,328

Amount, after deduction of unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

Collaboration, License and Other Agreements - Contract Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Contract Assets
Accounts receivable, net	$ 75,797	$ 42,827
Unbilled accounts receivable	1,812	351
Contract Liabilities
Contract liabilities, current	2,005	812
Contract liabilities, noncurrent	8,193	4,792
Accrued expenses, current	33,957	49,354
Collaborative Arrangement \| VantAI
Prepaid Assets
Prepaid assets, current	4,971	6,581
Prepaid assets, noncurrent	2,666	6,581
Prepaid assets, total	7,637	13,162
Collaborative Arrangement \| Roche, Collaboration (Pralsetnib) Agreement
Contract Assets
Unbilled accounts receivable		361
Contract Liabilities
Accrued expenses	1,712	7,388
Collaborative Arrangement \| Roche, Collaboration and License (Immunotherapy) Agreement
Contract Liabilities
Contract liabilities	0	0
Collaborative Arrangement \| Clementia
Contract Liabilities
Contract liabilities	0	0
Collaborative Arrangement \| C Stone
Contract Liabilities
Contract liabilities		604
Accrued expenses	2,027	$ 1,863
Arrangement Other than Collaborative, Asset Purchase Agreement \| Rigel Pharmaceuticals, Inc.
Contract Assets
Contract assets, current	2,939
Contract assets, noncurrent	7,380
Contract assets	10,319
Contract Liabilities
Contract liabilities, noncurrent	3,562
Contract liabilities	$ 3,562

Collaboration, License and Other Agreements - Subsequent Event (Details) - GSK plc - IDRx, Inc. - Subsequent Event - Forecast $ in Millions	3 Months Ended
	Mar. 31, 2025 USD ($)
Business Combination, Consideration Transferred
Business combination, consideration transferred	$ 1,000.0
Business Combination, Contingent Consideration Arrangements
Business combination, contingent consideration arrangements, range of outcomes, value, high	$ 150.0

Collaboration, License and Other Agreements - Change in Contract with Customer, Liability (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Collaborative Arrangement \| Roche, Collaboration and License (Immunotherapy) Agreement
Change in Contract with Customer, Liability
Amounts included in the contact liability at the beginning of the period	$ 16.0	$ 4.4

Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2024

Schedule of accrued expenses

Accrued expenses as of December 31, 2024 and 2023 consist of the following (in thousands):


	As of December 31,
	2024		2023
Research, development and commercial contract costs	$	33,957	$	49,354
Employee compensation		48,725		43,434
Accrued professional fees		14,134		12,522
Revenue-related reserves		30,816		17,465
Other		5,456		5,217
Total	$	133,088	$	127,992

Collaboration, License and Other Agreements (Tables)

12 Months Ended

Dec. 31, 2024

Schedule of amounts recognized as reductions to expenses


	Year Ended December 31,
	2024		2023		2022
Reductions to selling, general and administrative expenses	$	980	$	10,982	$	16,600
Increases in research and development expenses	$	1,103	$	24,915	$	19,297

Summary of prepaid assets


	December 31, 2024						December 31, 2023
	Current		Noncurrent		Total		Current		Noncurrent		Total
Prepaid assets	$	4,971	$	2,666	$	7,637	$	6,581	$	6,581	$	13,162

Rigel Pharmaceuticals, Inc. | Collaboration, License and Other

Summary of contract assets and/or contract liabilities


	As of December 31, 2024
	Current		Noncurrent		Total
Contract assets	$	2,939	$	7,380	$	10,319
Contract liabilities	$	—	$	3,562	$	3,562

Roche, Collaboration (Pralsetnib) Agreement

Summary of revenue recognized


	Year Ended December 31,
	2024		2023		2022
Manufacturing and research and development services related to Roche Territory-specific activities	$	324	$	679	$	6,710
Royalty revenue		180		1,464		848
Profit sharing revenue		1,416		—		—
Total Roche pralsetinib collaboration revenue	$	1,920	$	2,143	$	7,558

Summary of contract assets and/or contract liabilities


	December 31,		December 31,
	2024		2023
Unbilled accounts receivable	$	—	$	361
Accrued expenses	$	1,712	$	7,388

C Stone

Summary of revenue recognized


	Year Ended December 31,
	2024		2023		2022
License milestone revenue	$	—	$	9,000	$	6,500
Manufacturing services and royalty revenue related to CStone Territory-specific activities		3,304		8,312		17,794
Total CStone collaboration revenue	$	3,304	$	17,312	$	24,294

C Stone | Collaboration, License and Other

Summary of contract assets and/or contract liabilities


	As of December 31,
	2024		2023
Contract liabilities	$	—	$	604
Accrued expenses	$	2,027	$	1,863

Income Taxes

12 Months Ended

Dec. 31, 2024

Income Taxes

15. Income Taxes

The Company recorded an income tax provision of $1.2 million for the year ended December 31, 2024 due to state income taxes and taxable income from the jurisdictions in which the Company is subject to tax.

A reconciliation of the U.S. statutory income tax rate to the Company’s effective tax rate is as follows for the years ended December 31, 2024, 2023 and 2022:


	Year Ended December 31,
	2024		2023		2022
Federal income tax (benefit) at statutory rate	21.00	%	21.00	%	21.00	%
Permanent differences	0.82		(0.42)		(0.38)
U.S. Tax on foreign earnings	—		—		2.52
Federal research and development credits	14.43		2.50		1.39
Federal orphan drug credits	18.41		1.63		(0.42)
State income tax, net of federal benefit	7.91		3.41		2.92
Foreign rate differential	(13.47)		(0.41)		(0.13)
Deferred rate change	(9.21)		2.53		0.52
Foreign tax credit	—		—		0.03
Stock Based Compensation	(5.33)		(0.76)		(1.03)
Other	(0.27)		(0.02)		0.39
Change in valuation allowance	(36.17)		(29.65)		(27.76)
Effective income tax rate	(1.88)	%	(0.19)	%	(0.95)	%

The Company’s deferred tax assets and liabilities consist of the following (in thousands) as of December 31, 2024 and 2023:


	As of December 31,
	2024		2023
Deferred tax assets:
Net operating loss carryforwards	$	244,189	$	232,624
Research and development credit carryforwards		67,592		53,329
Orphan drug credit carryforwards		168,799		156,816
Accrued expenses and other		62,187		60,350
Royalty Pharma milestones		—		12,024
R&D capitalization		190,040		153,836
Financing arrangements		63,424		107,693
Deferred rent		19,040		22,204
Other		3,785		1,488
Total gross deferred tax asset		819,056		800,364
Deferred tax liability
Depreciation		(6,123)		(6,107)
Right of use assets		(14,837)		(17,423)
Other		—		(2,068)
Valuation allowance		(798,096)		(774,766)
Net deferred tax asset	$	—	$	—

Management has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets and has determined that it is more likely than not that the Company will not recognize the benefits of its net federal, foreign and state deferred tax assets, and as a result, a valuation allowance of $798.1 million and $774.8 million has been established at December 31, 2024 and 2023, respectively. The change in the valuation allowance was $23.3 million and $149.7 million for the years ended December 31, 2024 and 2023, respectively. The increase of deferred tax asset between December 31, 2024 and 2023 is primarily driven by the generation of federal and state net operating losses and the generation of research and development and orphan drug credits, as well as the capitalization and amortization of all Sec. 174 costs associated with research and development costs.

The Company has incurred net operating losses (NOL) since inception with the exception of years 2020 and 2022. As of December 31, 2024, the Company had federal and state NOL carryforwards of $859.2 million and $1,077.3 million, respectively, which begin to expire in 2030, and of which $843.3 million of the Company’s federal NOL is post 2017 NOL that will be carried forward indefinitely. As of December 31, 2024, the Company had federal and state research and development tax credit carryforwards of $39.9 million and $31.0 million, respectively, which begin to expire in 2039 and 2028 respectively. As of December 31, 2024, the Company had federal orphan drug credits of $168.8 million, which begin to expire in 2035 and state investment tax credits of $0.9 million, which have begun to expire in 2024. As of December 31, 2024, the Company has foreign tax credits of $2.5 million which will expire in 2031.

The Company has analyzed and validated its research and development tax credits as well as its orphan drug credits for 2011-2023. The Company generated research credits in 2024 but has not conducted a formal study to document its qualified activities. This study may result in an adjustment to the Company’s research and development credit carryforwards. No amounts are being presented as an uncertain tax position as of December 31, 2024 until such study is completed and the adjustment is known. A valuation allowance has been provided against the Company’s research and development credits and, if an adjustment is required, this adjustment would be offset by an adjustment to the deferred tax asset established for the research and development credit carry-forwards and the valuation allowance.

The Internal Revenue Code of 1986, as amended (the Code), provides for a limitation of the annual use of NOL and other tax attributes (such as research and development tax credit carryforwards) following certain ownership changes (as defined by the Code) that could limit the Company’s ability to utilize these carryforwards. The Company may have experienced such ownership changes in the past, and may experience ownership changes in the future as a result of shifts in its stock ownership, some of which are outside the Company’s control. Approximately $2.0 million of the Company’s NOL carryforwards may not be available for utilization within their applicable carryforward periods based on the Section 382 study in 2021. In addition, U.S. tax laws limit the time during which these carryforwards may be applied against future taxes. Therefore, the Company may not be able to take full advantage of these carryforwards for federal or state income tax purposes.

Interest and penalty charges, if any, related to unrecognized tax benefits would be classified as income tax expense in the accompanying statements of operations and comprehensive income (loss). As of December 31, 2024, the Company did not have any gross unrecognized tax benefit, excluding interest and penalties. As of December 31, 2024, the Company was open to examination in the U.S. federal and certain state jurisdictions for all of the Company’s tax years since the net operating losses may potentially be utilized in future years to reduce taxable income. Since the Company is in a loss carryforward position, it is generally subject to examination by the U.S. federal, state, and local income tax authorities for all tax years in which a loss carryforward is available.

Effective January 1, 2022, a provision of the Tax Cuts and Jobs Act (TCJA) took effect creating a significant change to the treatment of research and experimental (R&E) expenditures under Section 174 of the Code (Sec. 174 expenses). Historically, businesses have had the option of deducting Sec. 174 expenses in the year incurred or capitalizing and amortizing the costs over five years. The TCJA provision, however, eliminates this option and requires Sec. 174 expenses associated with research conducted in the U.S to be capitalized and amortized over a five-year period. For expenses associated with research outside of the United States, Sec. 174 expenses are capitalized and amortized over a 15-year period. The Company has included the tax impact of capitalizing and amortizing these costs over the required periods for the calendar year ended December 31, 2024.

On June 30, 2022, the Company entered into a Royalty Purchase Agreement with Royalty Pharma and a Future Revenue Purchase Agreement with Sixth Street Partners. Pursuant to the agreements, the Company received gross proceeds of $175.0 million from Royalty Pharma in June 2022 and $250.0 million from Sixth Street Partners in July 2022 upon the transactions closing. The total cash consideration of $425.0 million, in its entirety, was considered taxable income for calendar year ended December 31, 2022. In connection with and effective upon the termination of the Roche collaboration agreement, on February 22, 2024, Royalty Pharma and the Company entered into the Royalty Pharma Termination Agreement to terminate the Royalty Purchase Agreement. The Company accounted for the Royalty Pharma Termination Agreement as a debt extinguishment. As a result, during the year ended December 31, 2024 the Company recorded $173.7 million as a debt extinguishment gain in its consolidated financial statements. For additional information, see Note 3, Financing Arrangements. For tax purposes, the debt extinguishment gain was excluded from the December 31, 2024 estimated taxable income calculation since it was previously included in taxable income during the year ended December 31, 2022.

On August 1, 2022, the Company entered into the IDRx License Agreement and IDRx Stock Purchase Agreement. Pursuant to these agreements, the Company licensed its internally discovered KIT exon 13 inhibitor to IDRx in exchange for a 4,509,105 shares of IDRx’s Series A preferred stock and the eligibility to receive future milestones and tiered royalty payments. For book and tax purposes, the value of the equity totaling $27.5 million was included in the taxable income for the year ended December 31, 2022.

As of December 31, 2024, the Company did not have any gross unrecognized tax benefits, excluding interest and penalties due to an audit settlement. The following table provides the reconciliation of the total amounts of the Company’s unrecognized tax benefits for the years ended December 31, 2024 and 2023 (in thousands):


	Year ended December 31,
	2024		2023
Beginning balance at January 1	$	211	$	—
Gross Increases - tax positions in prior periods		—		211
Gross Increases - current period tax positions		—		—
Settlements		(211)		—
Lapse of statutes		—		—
Ending balance at December 31	$	—	$	211

The entire disclosure for income tax.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Accounts Receivable, Net (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Accounts Receivable, net
Credit losses	$ 0	$ 0	$ 0

Collaboration, License and Other Agreements

12 Months Ended

Dec. 31, 2024

Collaboration, License and Other Agreements

11. Collaboration, License and Other Agreements

Rigel

On February 22, 2024, the Company entered into an Asset Purchase Agreement with Rigel Pharmaceuticals, Inc. (“Rigel”) for Rigel to purchase certain assets from the Company comprising the U.S. rights to research, develop, manufacture and commercialize GAVRETO (pralsetinib). Such assets include, among other things, applicable intellectual property related to pralsetinib in the U.S, including patents, copyrights and trademarks, as well as clinical regulatory and commercial data and records. Simultaneously and in connection with entering into the Asset Purchase Agreement, the parties also entered into certain supporting agreements, including a customary transition agreement, (such agreements collectively, the Rigel Agreement), pursuant to which, the Company transitioned certain inventory and regulatory and distribution responsibilities for pralsetinib to Rigel.

Under the terms of the Rigel Agreement, the Company has the right to receive a purchase price of $15.0 million, with $10.0 million paid upon first commercial sale of GAVRETO by Rigel and an additional $5.0 million as a delayed purchase price payable on the later of (i) the first anniversary of the closing date of the transaction, or (ii) the completion of certain transition activities. The Company is also eligible to receive up to $102.5 million in contingent specified regulatory and commercial milestone payments, in addition to tiered percentage royalties ranging from 10 percent to 30 percent on annual net sales of GAVRETO in the U.S. The royalties will be payable until the later of (i) the expiration of the royalty term, as defined in the agreement, which begins on the date of the first commercial sale of

GAVRETO in the U.S., (ii) the date of expiration of the last valid patent claim within the Company’s IP that covers GAVRETO in the U.S., and (iii) the expiration of the last regulatory exclusivity for GAVRETO in the U.S.

The Company determined that the Rigel Agreement is a transaction with a customer and therefore accounted for the transaction in accordance with ASC 606. As of the effective date, the Company determined that the agreement includes three performance obligations: the delivery of (1) the U.S. rights to GAVRETO; (2) additional pralsetinib clinical data; and (3) GAVRETO product to be purchased from Genentech.

The transaction price under ASC 606 was fully constrained at the inception of the Rigel Agreement due to the pending completion of key transition activities stipulated in the agreement, including the transfer of the new drug application (NDA). These key transition activities, including the transfer of the NDA, related patents, and GAVRETO product, were completed in the second quarter of 2024. The performance obligations related to the U.S. rights to GAVRETO and the delivery of GAVRETO product were satisfied at a point in time upon the completion of these key transition activities. The transaction price was determined to be $27.7 million, which consisted of $6.5 million consideration for the GAVRETO product, $10.0 million upfront purchase price payment, and $11.2 million in the milestone and royalty payments that were considered probable of achievement and not subject to significant subsequent reversal of revenue. The transaction price was allocated to the three performance obligations on a relative stand-alone selling price basis.

During the year ended December 31, 2024, the Company recognized total revenue of $24.3 million which is primarily comprised of GAVRETO product consideration of $6.5 million and the transaction price allocated to the U.S. rights to GAVRETO performance obligation of $17.6 million. The additional clinical data performance obligation will be satisfied at a point in time once the transfer of such data to Rigel is completed, and the allocated transaction price of $3.6 million was recorded as deferred revenue on the consolidated financial statements as of December 31, 2024.

The Company reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal.

The following table summarizes the assets and liabilities under the Rigel agreements as of December 31, 2024 (in thousands):


	As of December 31, 2024
	Current		Noncurrent		Total
Contract assets	$	2,939	$	7,380	$	10,319
Contract liabilities	$	—	$	3,562	$	3,562

IDRx

In August 2022, the Company entered into a license agreement with IDRx, Inc. (IDRx), pursuant to which the Company granted IDRx an exclusive, worldwide, royalty-bearing license to exploit the Company’s internally discovered KIT exon 13 inhibitor IDRX-73 (IDRx License Agreement). IDRx is a clinical-stage biopharmaceutical company and among IDRx’s founders are Alexis Borisy, George Demetri, M.D., and Nicholas Lydon, Ph.D., who were each a member of the Company’s board of directors at the time. Due to these relationships, the transaction with IDRx is a related party transaction.

In connection with the IDRx License Agreement, the Company also entered into a stock purchase agreement with IDRx (IDRx Stock Purchase Agreement), pursuant to which the Company received 4,509,105 shares of IDRx’s Series A preferred stock with the right to receive additional shares of IDRx’s Series A preferred stock through an anti-dilution provision subject to a defined financing cap. In July 2023, the Company received 192,282 additional shares under the anti-dilution provision. The shares are restricted from reselling unless IDRx subsequently proposes a resale registered under the Securities Act or if an exemption from registration is otherwise available.

The Company is also eligible to receive up to $217.5 million in contingent cash payments, including specified development, regulatory and sales-based milestone payments. In addition, IDRx is obligated to pay to the Company royalties on aggregate annual worldwide net sales of licensed products at tiered percentage rates up to low-teens, subject to adjustments in specified circumstances under the IDRx License Agreement.

Unless earlier terminated, the IDRx License Agreement will expire on a country-by-country, licensed product-by-licensed product basis upon the latest of: (a) the expiration of the last valid claim within the licensed patents covering such licensed product in a such country, (b) the expiration of the regulatory exclusivity period for such licensed product in such country, or (c) the 10^th anniversary of the first commercial sale of such licensed product in such country. Following the end of the term for any such licensed product and in such region by expiration, the license granted to IDRx will become exclusive, perpetual, irrevocable, fully paid-up and royalty-free. IDRx may terminate the IDRx License Agreement for convenience at any time upon at least twelve months’ prior written notice to the Company. Either party may also terminate the IDRx License Agreement for material breach of the other party or for insolvency, and the Company may terminate the IDRx License Agreement for IDRx’s breach of the anti-dilution provision in the IDRx Stock Purchase Agreement. Upon termination of the license agreement in its entirety, all rights and obligations under the license agreement will terminate and revert back to the Company, and the Company has a license under certain intellectual property of IDRx to continue to exploit the compound and terminated product, subject to a royalty that will be negotiated at the time of termination.

The Company considered the ASC 606 criteria for combining contracts and determined the IDRx License Agreement and the IDRx Stock Purchase Agreement should be combined into a single contract because they were negotiated and entered into in contemplation of one another. Therefore, the Company determined that the 4,509,105 shares of IDRx’s Series A preferred stock and the anti-dilution right to receive additional shares should be attributed to the transaction price of the IDRx License Agreement.

The Company evaluated the IDRx License Agreement under ASC 606. The Company identified the following material promises under the agreement: (1) the exclusive license and (2) the initial know-how transfer. The Company determined that the exclusive license and the initial know-how transfer were not distinct from each other, as the exclusive license has limited value without the corresponding know-how transfer. As such, for the purposes of ASC 606, the Company determined that these two material promises, the exclusive license and the initial know-how transfer, should be combined into one distinct performance obligation. The Company concluded that the license is a functional intellectual property license. The Company determined that IDRx benefited from the license along with the initial know-how transfer at the time of grant, and therefore the related performance obligation is satisfied at a point in time.

For the purposes of ASC 606, the transaction price of the IDRx License Agreement at the contract inception was determined to be $27.5 million and recorded as license revenue-related party on the consolidated statements of operations and comprehensive loss in 2022. The fair value was derived from IDRx’s most recent financing transaction with unrelated investors. All potential milestone payments that the Company is eligible to receive under the IDRx License Agreement have been excluded from the transaction price. The Company reevaluates the transaction price for inclusion of milestone payments and royalties at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. Additionally, the Company is entitled to sales milestones and royalties from the sales of the licensed products, and revenue are recognized when the related sales occur.

The Company concluded the preferred stock investment should be accounted for as an equity investment as it is not mandatorily redeemable nor does the Company have the unilateral right to redeem the preferred stock, and the Company, along with its related parties, do not have a controlling financial interest in IDRx nor have the ability to influence the financial and operating policies through the ownership of preferred stock. IDRx’s preferred stock is not exchange-traded and does not have a readily determinable fair value. Therefore, the preferred stock investment was accounted for under the measurement alternative for equity investments that do not have a readily determinable fair value, at cost of $27.8 million including transaction costs of $0.3 million. If observable price changes in orderly transactions for the identical or similar investment are identified, the Company will adjust the carrying value of the investment to its fair value as of the transaction date. During the year ended December 31, 2024, IDRx completed a Series B preferred stock financing and accordingly, the Company adjusted the carrying value of its investment in IDRx to $28.7 million. The $0.9 million investment gain was recorded as other income on the Company’s consolidated

statements of operations and comprehensive loss. No revenue was recorded under the IDRx License Agreement during the years ended December 31, 2024 and 2023.

In January 2025, IDRx announced that they had entered into an agreement under which GSK plc will acquire IDRx for $1.0 billion upfront with an additional $150.0 million regulatory approval-based milestone payment. The transaction is expected to close in the first quarter 2025.

VantAI

In February 2022, the Company entered into an exclusive collaboration agreement with Oncopia Therapeutics, Inc. d/b/a Proteovant Therapeutics, Inc. (“Proteovant”) (the 2022 Agreement) to jointly research and advance certain protein degrader therapies into development candidates, with VantAI, Inc. (“VantAI”) performing computational chemistry services on behalf of Proteovant under the agreement. In December 2023, the Company entered into an Amended and Restated Collaboration and License Agreement (the “A&R Agreement”) with VantAI and Proteovant, which amended and restated and replaced in its entirety the 2022 Agreement. Under the A&R Agreement, Proteovant ceased its role under the 2022 Agreement and VantAI provides expanded computational support directly to the Company, including computational biology and expanded computational chemistry to advance three novel protein degrader programs, and the Company has the option, at its sole discretion, to expand the collaboration to include a fourth target program.

Under the A&R Agreement, VantAI is eligible to receive up to $1.67 billion in contingent payments including specified research, development, regulatory and commercialization milestones for all the target programs. The Company will be obligated to pay VantAI tiered percentage royalties on a licensed product-by-licensed product basis ranging from the mid-single digits on annual net sales of each licensed product in the applicable territory, subject to adjustment in specified circumstances.

Under the 2022 Agreement, the Company paid Proteovant an upfront payment of $20.0 million in connection with the execution of the 2022 Agreement. This upfront payment was recorded as a prepaid asset on the Company’s consolidated balance sheet and was amortized as research and development expense over the expected research period because the Company concluded that Proteovant was providing the Company with research services throughout such period. The Company determined to continue to amortize the remaining prepaid asset balance as research and development expense over the expected research period of the A&R Agreement as VantAI continued to provide such research and development services. During the year ended December 31, 2024, the Company recorded research and development expense of $7.5 million under the A&R Agreement. During the years ended December 31, 2023 and 2022, research and development expense recorded under the 2022 Agreement was $4.0 million and $4.4 million, respectively. The following table summarizes the prepaid assets associated with the A&R Agreement as of December 31, 2024 and 2023 (in thousands):


	December 31, 2024						December 31, 2023
	Current		Noncurrent		Total		Current		Noncurrent		Total
Prepaid assets	$	4,971	$	2,666	$	7,637	$	6,581	$	6,581	$	13,162

The Company reevaluates the expected research period at the end of each reporting period and prospectively adjusts the amortization of the asset for changes in the expected research period. Each research and development milestone payment is accrued and expensed when probable.

Zai Lab

In November 2021, the Company entered into a collaboration (the Zai Lab agreement) with Zai Lab (Shanghai) Co., Ltd., (Zai Lab) to develop and commercialize certain licensed products for the treatment of EGFR-driven non-small cell lung cancer in Greater China, including Mainland China, Hong Kong, Macau and Taiwan (collectively, the Zai Lab Territory), which currently include BLU-945 and BLU-525. In January 2024, the Company decided to discontinue further investment in the early clinical-stage therapies for EGFR-mutant NSCLC globally. Zai Lab retains its rights to BLU-945 and BLU-525 under the agreement. The Company retains exclusive rights to the licensed products outside the

Zai Lab Territory. The decision to deprioritize the licensed products does not have an impact on the Company’s accounting treatment related to the Zai Lab agreement.

Under the Zai Lab agreement, the Company received an upfront cash payment of $25.0 million and, in addition to the upfront payment received, the Company is eligible to receive up to $590.0 million in contingent payments, including specified development, regulatory and sales-based milestones and tiered percentage royalties on a licensed product-by-licensed product basis ranging from the low-teens to mid-teens on annual net sales of each licensed product in the Zai Lab Territory, subject to adjustment in specified circumstances. Zai Lab is responsible for costs related to clinical trials in the Zai Lab Territory, other than the specified shared services costs as defined in the Zai Lab agreement which are shared by the Company and Zai Lab. Zai Lab is responsible for conducting all development and commercialization activities in the Zai Lab Territory related to the licensed drug candidates.

The Zai Lab agreement will continue on a licensed product-by-product and region-by-region basis until the later of (i) the 12th anniversary of the date of the first commercial sale of a licensed product in the Zai Lab Territory, (ii) the date of expiration of the last valid patent claim related to the Company’s patent rights of the product in the Zai Lab Territory, and (iii) the expiration of the last regulatory exclusivity for that product in a region in the Zai Lab Territory. Zai Lab may terminate the agreement for convenience by giving a written notice (a) at least 12 months after the date of notice, in the event such notice is given after the first commercial sale of a licensed product in the Zai Lab Territory or (b) at least nine months after the date of such notice, in the event such notice is given prior to the first commercial sale of the first licensed product in the Zai Lab Territory. Either party may terminate the Zai Lab agreement for the other party’s uncured material breach or insolvency. Upon termination, all licenses and all other rights granted by the Company to Zai Lab will terminate. Each party will retain its joint ownership interests in any joint collaboration technology.

The Company concluded that the Zai Lab agreement is a collaborative agreement under ASC 808. The Company determined that the Zai Lab agreement contained two material components: (i) licenses granted to Zai Lab to exploit and develop each licensed product in the Zai Lab Territory and related activities in the Zai Lab Territory, including manufacturing, and (ii) global development of the licensed products. The Company concluded that Zai Lab is the Company’s customer for the licenses and related activities in the Zai Lab Territory under ASC 606, whereas payments received by the Company for global development activities, including manufacturing, are accounted for as a reduction of related expenses. The Company did not record significant net reductions of expenses under the Zai Lab agreement during the years ended December 31, 2024, 2023 and 2022.

The Company evaluated the Zai Lab Territory specific licenses and related activities under ASC 606 and identified one performance obligation, which consists of the licenses and their initial know-how transfer at the outset of the arrangement. The manufacturing activities were excluded as performance obligation at the outset of the arrangement because it represented a customer option that was not a material right.

The Company determined that the license is a functional intellectual property license as Zai Lab benefited from the license along with the initial know-how transfer at the time of grant, and therefore the related performance obligation is satisfied at a point in time. The transaction price of the Zai Lab agreement at the outset of the arrangement was determined to be $25.0 million and the Company satisfied the performance obligation upon delivery of the licenses and initial know-how transfer and accordingly, the upfront payment of $25.0 million was recognized as revenue in 2021. All milestone and royalty payments that the Company is eligible to receive were excluded from the transaction price, as all amounts were fully constrained based on the probability of achievement. The Company reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur. Costs that are incurred associated with Zai Lab Territory specific activities are reimbursable from Zai Lab and are recognized as revenue. The Company did not record any revenue under the Zai Lab agreement during the year ended December 31, 2024 and the revenue recorded during the years ended December 31, 2023 and 2022 was not significant.

Roche – Pralsetinib Collaboration

In July 2020, the Company entered into a collaboration agreement (the Roche pralsetinib collaboration agreement) with F. Hoffmann-La Roche Ltd and Genentech, Inc., a member of the Roche Group (collectively, Roche), pursuant to which the Company granted Roche exclusive rights to develop and commercialize the Company’s drug candidate pralsetinib worldwide, excluding the CStone Territory (as defined below), and a co-exclusive license in the U.S. to develop and commercialize pralsetinib. The Roche pralsetinib collaboration agreement was terminated on February 22, 2024 (the collaboration termination effective date), at which time the Company regained commercialization

and development rights to GAVRETO from Roche worldwide excluding the CStone Territory. The Company and Roche continued to perform their respective obligations under the Roche pralsetinib collaboration agreement through the collaboration termination effective date. On February 22, 2024, the Company and Roche entered into a transition agreement (the Roche transition agreement) in connection with the termination of the Roche pralsetinib collaboration agreement.

Under the Roche pralsetinib collaboration agreement, the Company received an upfront cash payment of $675.0 million and Roche Holdings, Inc. (Roche Holdings) purchased 1,035,519 shares of the Company’s common stock at a purchase price of $96.57 per share for consideration of $100.0 million under a stock purchase agreement entered into in July 2020 in connection with the agreement. The fair market value of the common stock issued to Roche Holdings was $79.3 million on the date of issuance and the premium of $20.7 million was attributed to the transaction price of the Roche pralsetinib collaboration agreement.

In the U.S., the Company and Roche co-commercialized pralsetinib and shared profits and losses equally. In addition, the Company received tiered royalties on annual net sales of pralsetinib outside the U.S., excluding the CStone Territory (the Roche Territory). The Company and Roche shared global development costs for pralsetinib at a rate of 45 percent for the Company and 55 percent for Roche.

The Company concluded that the Roche pralsetinib collaboration agreement contained two material promises within the scope of ASC 606, pralsetinib license and the Roche Territory activities. The Company evaluated the Roche pralsetinib license under ASC 606 and concluded that the pralsetinib license was a functional intellectual property license and a distinct performance obligation. The Company determined that Roche benefited from the pralsetinib license at the time of grant, and therefore the related performance obligation was satisfied at a point in time.

The transaction price of the Roche pralsetinib collaboration agreement at the outset of the arrangement was determined to be $695.7 million, which consisted of the upfront cash payment of $675.0 million and the $20.7 million premium on the sale of common stock to Roche Holdings, which was allocated to the performance obligation related to the pralsetinib licenses. Through the collaboration termination effective date, the Company achieved an aggregate of $105.0 million in specified regulatory and commercialization milestones, which were added to the estimated transaction price of the Roche pralsetinib collaboration agreement and recorded as revenue in the respective periods when they were achieved.

For the parties’ participation in global development for pralsetinib and the U.S. commercialization activities for GAVRETO, the Company concluded that those activities and cost-sharing payments related to such activities were within the scope of ASC 808, as both parties were active participants in the development, manufacturing and commercialization activities and are exposed to significant risks and rewards of those activities under the Roche pralsetinib collaboration agreement. Payments to or reimbursements from Roche related to the global development activities were accounted for either as an increase or reduction of research and development expenses.

No operating expenses were recorded under the Roche pralsetinib collaboration since June 30, 2024. The following table summarizes the amounts recognized as reductions to selling, general and administrative expenses related to the commercialization of GAVRETO in the U.S., and reductions to (increases in) research and development expenses related to global development activities for pralsetinib under the Roche pralsetinib collaboration during the years ended December 31, 2024, 2023 and 2022 (in thousands):


	Year Ended December 31,
	2024		2023		2022
Reductions to selling, general and administrative expenses	$	980	$	10,982	$	16,600
Increases in research and development expenses	$	1,103	$	24,915	$	19,297

Roche was the principal for recording product sales to customers in the U.S., and the Company recognized a portion of the profit as revenue and losses as collaboration loss sharing in its consolidated statements of operations and comprehensive loss. During the year ended December 31, 2024, the Company recorded revenue of $1.4 million derived from profit sharing on Roche sales of GAVRETO in the U.S. During the years ended December 31, 2023 and 2022, the Company recorded a collaboration loss sharing expense of $4.3 million and $8.9 million on Roche’s sales of GAVRETO in the U.S., respectively. Costs incurred associated with the Roche Territory activities were reimbursable from Roche and were recognized as revenue

The following table summarizes revenue recognized under the Roche pralsetinib collaboration during the years ended December 31, 2024, 2023 and 2022 (in thousands):


	Year Ended December 31,
	2024		2023		2022
Manufacturing and research and development services related to Roche Territory-specific activities	$	324	$	679	$	6,710
Royalty revenue		180		1,464		848
Profit sharing revenue		1,416		—		—
Total Roche pralsetinib collaboration revenue	$	1,920	$	2,143	$	7,558

Upon termination of the Roche pralsetinib collaboration agreement, the Company chose not to assume responsibility for any ongoing pralsetinib clinical trials, and under the terms of the Roche pralsetinib collaboration agreement, Roche bears sole responsibility for all costs associated with the wind-down of these trials.

Pursuant to the Roche transition agreement, the Company is obligated to reimburse Roche for wind-down costs associated with the marketing and commercialization activities occurred for Roche Territory until December 31, 2026. Additionally, the Company is obligated to reimburse Roche for any U.S. transition related costs that exceeds GAVRETO’s net sales in the U.S., and any remaining net profit are shared equally between the Company and Roche until December 31, 2025. The Company has concluded that such activities and associated payments to Roche are not within the scope of ASC 808 as only the Company is exposed to significant risks and awards associated with those activities. The Company records those wind-down costs and the net amount of U.S. transition costs reimbursable to Roche as selling, general, and administrative expenses when they are incurred. During the year ended December 31, 2024, the Company recorded a total of $5.7 million in such costs.

The following table summarizes the assets and liabilities associated with the Roche pralsetinib agreements as of December 31, 2024 and 2023 (in thousands):


	December 31,		December 31,
	2024		2023
Unbilled accounts receivable	$	—	$	361
Accrued expenses	$	1,712	$	7,388

Clementia

In October 2019, the Company entered into a license agreement (the Clementia agreement) with Clementia Pharmaceuticals, Inc. (Clementia), a wholly-owned subsidiary of Ipsen S.A. Under the Clementia agreement, the Company granted an exclusive, worldwide, royalty-bearing license to Clementia to develop and commercialize BLU-782, the Company’s oral, highly selective investigational ALK2 inhibitor in clinical development for the treatment of fibrodysplasia ossificans progressiva (FOP), now referred to as fidrisertib, as well as specified other compounds related to the BLU-782 program.

Under the Clementia agreement, the Company received an upfront cash payment of $25.0 million and through December 31, 2024, the Company has received an aggregate of $50.0 million in cash milestone payments. Subject to the terms of the Clementia agreement, in addition to the upfront and milestone payments received through December 31, 2024, the Company is eligible to receive up to $460.0 million in contingent payments, including specified development, regulatory and sales-based milestones for licensed products. In addition, Clementia is obligated to pay to the Company royalties on aggregate annual worldwide net sales of licensed products at tiered percentage rates ranging from the low- to mid-teens, subject to adjustment in specified circumstances under the Clementia agreement, and Clementia purchased specified manufacturing inventory from the Company for a total of $1.5 million.

Unless earlier terminated in accordance with the terms of the Clementia agreement, the agreement will expire on a country-by-country, licensed product-by-licensed product basis on the date when no royalty payments are or will become due. Clementia may terminate the agreement at any time upon at least 12 months’ prior written notice to the Company. Either party may terminate the agreement for the other party’s uncured material breach or insolvency and in

certain other circumstances agreed to by the parties. In certain termination circumstances, the Company is entitled to retain specified licenses to be able to continue to exploit the Clementia licensed products.

The Company evaluated the Clementia agreement under ASC 606 as the agreement represented a transaction with a customer. The Company identified the following material promises under the agreement: (1) the exclusive license to develop, manufacture and commercialize BLU-782; (2) the technology transfer of BLU-782 program; (3) the transfer of existing manufacturing inventory; and (4) the transfer of in-process manufacturing inventory. In addition, the Company determined that the exclusive license and technology transfer were not distinct from each other, as the exclusive license has limited value without the corresponding technology transfer. As such, for the purposes of ASC 606, the Company determined that these four material promises, described above, should be combined into three performance obligations: (1) the exclusive license and the technology transfer; (2) the transfer of existing manufacturing inventory; and (3) the transfer of in-process manufacturing inventory.

The Company determined that the transaction price at the outset of the arrangement was $46.5 million, which was allocated to the three performance obligations on a relative stand-alone selling price basis, and was recognized as revenue in prior years.

The Company did not recognize revenue under the Clementia agreement during the years ended December 31, 2024 and 2023. During the year ended December 31, 2022, cash consideration associated with an achieved development milestone of $30.0 million was added to the estimated transaction price for the Clementia agreement and recognized as revenue. All potential milestone payments that the Company is eligible to receive were excluded from the transaction price, as the amounts were fully constrained based on the probability of achievement. The Company reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal.

There was no revenue deferred as a contract liability associated with the Clementia agreement as of December 31, 2024 and 2023.

CStone Pharmaceuticals

In June 2018, the Company entered into a collaboration and license agreement (the CStone agreement) with CStone Pharmaceuticals (CStone) pursuant to which the Company granted CStone exclusive rights to develop and commercialize the Company’s drug candidates avapritinib, pralsetinib and fisogatinib, including back-up and other forms thereof, in Mainland China, Hong Kong, Macau and Taiwan (each, a CStone region and collectively, the CStone Territory), either as a monotherapy or as part of a combination therapy.

The Company received an upfront cash payment of $40.0 million, and through December 31, 2024, the Company has achieved an aggregate of $38.5 million in milestones under this collaboration. Subject to the terms of the CStone agreement, in addition to the upfront payments received and milestones achieved through December 31, 2024, the Company will be eligible to receive up to $307.5 million in contingent payments, including specified development, regulatory and sales-based milestones for licensed products. In addition, CStone is obligated to pay the Company tiered percentage royalties on a licensed product-by-licensed product basis ranging from the mid-teens to low twenties on annual net sales of each licensed product in the CStone Territory, subject to adjustment in specified circumstances. CStone is responsible for costs related to the development of the licensed products in the CStone Territory, other than specified costs related to the development of fisogatinib as a combination therapy in the CStone Territory that are shared by the Company and CStone.

Pursuant to the terms of the CStone agreement, CStone is responsible for conducting all development and commercialization activities in the CStone Territory related to the licensed products. Subject to specified exceptions, during the term of the CStone agreement, each party has agreed that neither it nor its affiliates will conduct specified development and commercialization activities in the CStone Territory related to selective inhibitors of FGFR4, KIT, PDGFRA and RET. In addition, under the CStone agreement, each party has granted the other party specified intellectual property licenses to enable the other party to perform its obligations and exercise its rights under the CStone agreement, including license grants to enable each party to conduct research, development and commercialization activities pursuant to the terms of the CStone agreement.

The CStone agreement will continue on a licensed product-by-licensed product and CStone region-by-CStone region basis until the later of (i) 12 years after the first commercial sale of a licensed product in a CStone region in the CStone Territory and (ii) the date of expiration of the last valid patent claim related to the Company’s patent rights or any joint collaboration patent rights for the licensed product that covers the composition of matter, method of use or method of manufacturing such licensed product in such region. Subject to the terms of the CStone agreement, CStone may terminate the CStone agreement in its entirety or with respect to one or more licensed products for convenience by providing written notice to the Company, and CStone may terminate the CStone agreement with respect to a licensed product for convenience at any time by providing written notice to the Company following the occurrence of specified events. In addition, the Company may terminate the CStone agreement under specified circumstances if CStone or certain other parties challenges the Company’s patent rights or any joint collaboration patent rights or if CStone or its affiliates do not conduct any material development or commercialization activities with respect to one or more licensed products for a specified period of time, subject to specified exceptions. Either party may terminate the CStone agreement for the other party’s uncured material breach or insolvency. In certain termination circumstances, the parties are entitled to retain specified licenses to be able to continue to exploit the licensed products, and in the event of termination by CStone for the Company’s uncured material breach, the Company will be obligated to pay CStone a low single digit percentage royalty on a licensed product-by-licensed product basis on annual net sales of such licensed product in the CStone Territory, subject to a cap and other specified exceptions.

The Company evaluated the CStone agreement to determine whether it is a collaborative arrangement for purposes of ASC 808. The Company determined that there were two material components of the CStone agreement: (i) the CStone Territory-specific license and related activities in the CStone Territory, and (ii) the parties’ participation in global development of the licensed products. The Company concluded that the CStone Territory-specific license and related activities in the CStone Territory are not within the scope of ASC 808 because the Company is not exposed to significant risks and rewards. The Company concluded that CStone is a customer with regard to the component that includes the CStone Territory-specific license and related activities in CStone Territory, which include manufacturing. For the parties’ participation in global development of the licensed products, the Company concluded that the research and development activities and cost-sharing payments related to such activities are within the scope of ASC 808 as both parties are active participants exposed to the risk of the activities under the CStone agreement. The Company concluded that CStone is not a customer with regard to the global development component in the context of the CStone agreement. Therefore, net payments received by the Company for global development activities under the CStone agreement, including manufacturing, are accounted for as a reduction of related expenses.

The Company did not have significant manufacturing and research and development services related to the global development activities during the years ended December 31, 2024 and 2023. During the year ended December 31, 2022, the Company recorded $1.7 million in reduction of related expenses from manufacturing and research and development services related to global development activities, net of expenses payable to CStone.

The Company evaluated the CStone Territory-specific license and related activities in the CStone Territory under ASC 606, as these transactions are considered transactions with a customer. The Company identified the following material promises under the arrangement: (1) the three exclusive licenses granted in the CStone Territory to develop, manufacture and commercialize the three licensed products; (2) the initial know-how transfer for each licensed product; (3) manufacturing activities related to development and commercial supply of the licensed products; (4) participation in the joint steering committee (JSC) and joint project teams (JPT); (5) regulatory responsibilities; and (6) manufacturing technology and continuing know-how transfers. The Company determined that each licensed product is distinct from the other licensed products. In addition, the Company determined that the exclusive licenses and initial know-how transfers for each licensed product were not distinct from each other, as each exclusive license has limited value without the corresponding initial know-how transfer. For purposes of ASC 606, the Company determined that participation on the JSC and JPTs, the regulatory responsibilities and the manufacturing technology and continuing know-how transfers are qualitatively and quantitatively immaterial in the context of the CStone agreement and therefore are excluded from performance obligations. As such, the Company determined that these six material promises, described above, should be combined into one performance obligation for each of the three candidates.

The Company evaluated the provision of manufacturing activities related to development and commercial supply of the licensed products as an option for purposes of ASC 606 to determine whether these manufacturing activities provide CStone with any material rights. The Company concluded that the manufacturing activities were not issued at a significant and incremental discount, and therefore do not provide CStone with any material rights. As such, the manufacturing activities are excluded as performance obligations at the outset of the arrangement.

Based on these assessments, the Company identified three distinct performance obligations at the outset of the CStone agreement, which consists of the following for each licensed product: (1) the exclusive license and (2) the initial know-how transfer.

Under the CStone agreement, in order to evaluate the transaction price for purposes of ASC 606, the Company determined that the upfront amount of $40.0 million constituted the entirety of the consideration to be included in the transaction price at the outset of the arrangement, which was allocated to the three performance obligations. The potential milestone payments that the Company is eligible to receive were excluded from the transaction price, as all milestone amounts were fully constrained based on the probability of achievement. The Company satisfied the performance obligations upon delivery of the licenses, initial know-how transfers and product trademark and recognized the upfront payment of $40.0 million as revenue in 2018.

The Company did not achieve any milestones under the CStone agreement during the year ended December 31, 2024. During the years ended December 31, 2023, and 2022, cash consideration associated with achieved development and sales-based milestones of $9.0 million and $6.5 million, respectively, were added to the estimated transaction price for the CStone agreement and recognized as revenue in such periods. The Company reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal.

Subsequent to the CStone agreement, the Company entered into various commercial supply and manufacturing technology transfer agreements for avapritinib and pralsetinib related to supply of active pharmaceutical ingredient (API), drug substance and commercialization activities conducted specifically for the CStone Territory. The manufacturing activities in these agreements were considered as distinct performance obligations from the CStone collaboration agreement and collaboration revenue is recognized upon delivery of the supply to CStone. Considerations payable to CStone related to the Company’s obligations in connection with commercial supply of pralsetinib for the CStone Territory was recognized as a reduction of collaboration revenue.

A summary of revenue recognized under the CStone agreement during the years ended December 31, 2024, 2023 and 2022 is as follows (in thousands):


	Year Ended December 31,
	2024		2023		2022
License milestone revenue	$	—	$	9,000	$	6,500
Manufacturing services and royalty revenue related to CStone Territory-specific activities		3,304		8,312		17,794
Total CStone collaboration revenue	$	3,304	$	17,312	$	24,294

The Company did not have any contract assets related to the CStone collaboration as of December 31, 2024 and 2023. The following table presents the contract liabilities associated with the CStone collaboration as of December 31, 2024 and 2023 (in thousands):


	As of December 31,
	2024		2023
Contract liabilities	$	—	$	604
Accrued expenses	$	2,027	$	1,863

The Company’s liabilities associated with the CStone collaboration as of December 31, 2024 and 2023 resulted primarily from the Company’s obligations in connection with commercial supply of pralsetinib for the CStone Territory.

Roche – Immunotherapy Collaboration

In March 2016, the Company entered into a collaboration and license agreement (as amended, the Roche immunotherapy agreement) with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (collectively, Roche) for the discovery, development and commercialization of small molecule therapeutics targeting kinases believed to be important in cancer immunotherapy, as single products or possibly in combination with other therapeutics.

On April 30, 2023, the Company and Roche entered into a mutual termination agreement to terminate the Roche immunotherapy agreement. Certain licenses granted by Roche to the Company survived and became exclusive, worldwide, perpetual, royalty-free and irrevocable and the Company retained ownership of all the targets developed under the collaboration.

The Roche immunotherapy agreement represented a vendor-customer relationship under ASC 606 as the Company performed its one performance obligation at the outset of the Roche immunotherapy agreement, which consisted of: (1) the non-exclusive license; (2) the research and development activities through Phase 1; and (3) regulatory responsibilities under Phase 1 clinical trials.

The aggregate net transaction price of the Roche immunotherapy agreement was $64.7 million. The Company recognized revenue associated with the performance obligation using an input method, based on the costs incurred for the research and development activities on each program and the costs expected to be incurred in the future to satisfy the performance obligation for each respective period. The amounts received that had not yet been recognized as revenue were deferred as a contract liability on the Company’s consolidated balance sheet and recognized over the remaining research and development period until the performance obligation was satisfied. The performance obligation was completely satisfied as of June 30, 2023 and no revenue was recognized thereafter for this agreement. During the years ended December 31, 2023 and 2022, the Company recognized research and development services revenue of $25.7 million and $2.3 million, respectively, under the Roche immunotherapy agreement, of which $16.0 million and $4.4 million, respectively, resulted from changes in contract liability balances at the beginning of the period. There was no revenue deferred as a contract liability associated with the Roche immunotherapy agreement as of December 31, 2024 and 2023.

Leases - Total Lease Liability (Details) $ in Thousands	Dec. 31, 2024 USD ($)
Leases
Total future minimum lease payments	$ 97,754
Less imputed interest	(15,618)
Total	$ 82,136

Leases - General Information (Details) $ in Thousands	Dec. 15, 2021 USD ($)	Sep. 19, 2018 USD ($) ft²	Apr. 28, 2017 USD ($) ft² period	Dec. 31, 2024 USD ($)	Dec. 31, 2023 USD ($)	Sep. 18, 2018 USD ($)	Feb. 12, 2015 ft²
Leases
Initial lease commitments				$ 97,754
Restricted cash				11,600	$ 10,200
Corporate Headquarters Lease
Leases
Area leased (in square feet) \| ft²							39,000
Base annual rent, initial	$ 4,500
Base annual rent, maximum	5,500
Allowance for leasehold improvements	$ 800
Security deposit				900
Office and Laboratory Space in Cambridge Massachusetts, 45 Sidney Street
Leases
Area leased (in square feet) \| ft²		139,216	99,833
Lessee, Operating Lease, Existence of Option to Extend			true
Number of consecutive extension periods \| period			2
Extension period of lease term			5 years
Base annual rent, initial			$ 7,700
Base annual rent, maximum			$ 10,600
Allowance for leasehold improvements						$ 17,400
Office and Laboratory Space in Cambridge, Massachusetts, 45 Sidney Street, Expansion Premises
Leases
Base annual rent, initial		$ 3,200
Base annual rent, maximum		$ 4,200
Security deposit				$ 3,300

Accrued Expenses

12 Months Ended

Dec. 31, 2024

Accrued Expenses

10. Accrued Expenses

Accrued expenses as of December 31, 2024 and 2023 consist of the following (in thousands):


	As of December 31,
	2024		2023
Research, development and commercial contract costs	$	33,957	$	49,354
Employee compensation		48,725		43,434
Accrued professional fees		14,134		12,522
Revenue-related reserves		30,816		17,465
Other		5,456		5,217
Total	$	133,088	$	127,992

Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Stock-Based Compensation Expense (Details)	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Employee Stock Option
Stock-based compensation
Expected dividend yield	0.00%	0.00%	0.00%
Award expiration period	10 years	10 years	10 years
Employee Stock
Stock-based compensation
Purchase price on common stock (as a percent)	85.00%	85.00%	85.00%
Discount on fair value, offering date (as a percent)	15.00%	15.00%	15.00%
Discount on fair value, purchase date (as a percent)	15.00%	15.00%	15.00%
Award expiration period	180 days	180 days	180 days

Inventory - Write-down (Details) - USD ($) $ in Millions	12 Months Ended
Inventory - Write-down (Details) - USD ($) $ in Millions	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Inventory
Inventory write-down	$ 0.4	$ 0.7	$ 2.1

Product Revenue - Revenue-related Reserves (Details) - Valuation Allowances and Reserves, Product Revenue - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Product revenue allowance and reserve
Total revenue-related reserves	$ 33,779	$ 19,274	$ 9,788
Accounts Receivable, Net
Product revenue allowance and reserve
Total revenue-related reserves	2,963	1,809
Accrued Expenses
Product revenue allowance and reserve
Total revenue-related reserves	$ 30,816	$ 17,465

Income Taxes - Financing Arrangements (Details) - USD ($) $ in Thousands		1 Months Ended	12 Months Ended
	Jun. 30, 2022	Jul. 31, 2022	Dec. 31, 2024	Dec. 31, 2022
Gain (Loss) on Extinguishment of Debt
Debt extinguishment gain			$ 173,676
Sale of Future Royalties and Revenues
Gross Proceeds
Gross proceeds				$ 425,000
Sale of Future Royalties and Revenues \| Royalty Pharma, Royalty Purchase Agreement
Debt Financing
Debt instrument, issuance date	Jun. 30, 2022
Gross Proceeds
Gross proceeds	$ 175,000
Gain (Loss) on Extinguishment of Debt
Debt extinguishment gain			$ 173,700
Sale of Future Royalties and Revenues \| Sixth Street Partners, Future Revenue Purchase Agreement
Gross Proceeds
Gross proceeds		$ 250,000

Marketable Securities - Realized Gains (Losses) (Details) $ in Thousands	36 Months Ended
	Dec. 31, 2024 USD ($)
Realized gains (losses) from maturities of debt securities
Realized gains (losses) from maturities of debt securities	$ 0

Stock-based Compensation - Stock Options - Weighted Average Assumptions (Details) - Employee Stock Option	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Assumptions used in estimating the fair value of stock options granted
Risk-free interest rate	4.26%	4.13%	2.27%
Expected dividend yield	0.00%	0.00%	0.00%
Expected term (in years)	6 years	6 years	6 years
Expected stock price volatility	55.54%	56.01%	55.15%

Net Loss Per Share (Tables)

12 Months Ended

Dec. 31, 2024

Schedule of common stock equivalents excluded from calculation of diluted net loss per share applicable to common stockholders


	Year Ended December 31,
	2024	2023	2022
Stock options	6,370	6,658	6,233
Restricted stock units	2,291	2,107	1,894
Performance-based restricted stock units	143	53	—
ESPP shares	28	39	55
Total	8,832	8,857	8,182

Employee Benefit Plans - Switzerland Defined Benefit Plan - General Information (Details)	12 Months Ended
	Dec. 31, 2024
General Information
Defined Benefit Plan, Type	us-gaap:PensionPlansDefinedBenefitMember
Defined Benefit Plan, Sponsor Location	country:CH

Indicates location of employer sponsoring defined benefit plan designed to provide retirement benefits.

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Indicates type of defined benefit plan. Includes, but is not limited to, pension plan, other postretirement plan and special and contractual termination benefits payable upon retirement.

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Employee Benefit Plans - Switzerland Defined Benefit Plan - Contributions (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Contributions
Contributions	$ 1.1	$ 1.0	$ 0.8

Leases

12 Months Ended

Dec. 31, 2024

Leases

16. Leases

The Company’s building leases are comprised of office and laboratory spaces under non-cancelable operating leases. The lease agreements contain various clauses for renewal at the Company’s option and only certain exercised renewal options were included in the calculation of the operating lease assets and the operating lease liabilities, as other renewal options were not reasonably certain of being exercised as of December 31, 2024.

38 Sidney Street

In February 2015, the Company entered into a lease for approximately 39,000 rentable square feet of office and laboratory space at 38 Sidney Street in Cambridge, Massachusetts, which was extended in December 2021. The extended lease term will expire on November 30, 2029. The Company agreed to pay an initial annual base rent of approximately $4.5 million, which rises annually until it reaches approximately $5.5 million. The lease extension provided the Company with an allowance for leasehold improvements of $0.8 million improvements to be made to the premises. A security deposit of $0.9 million was recorded as restricted cash on the Company’s consolidated balance sheet as of December 31, 2024. The Company’s sublease agreements for the 38 Sidney Street property expired in 2022.

45 Sidney Street

In April 2017, the Company entered into a lease agreement for approximately 99,833 rentable square feet of office and laboratory space located at 45 Sidney Street in Cambridge, Massachusetts. In September 2018, the Company entered into an amendment to the lease agreement to expand the rentable square footage to approximately 139,216 square feet. The initial term of the lease agreement will expire on November 30, 2029, unless terminated sooner. The lease agreement also provides the Company with an option to extend the lease agreement for two consecutive five-year periods at the then fair market annual rent, as defined in the lease agreement.

The Company has agreed to pay for the 99,833 rentable square feet an initial annual base rent of approximately $7.7 million, which increases annually until it reaches approximately $10.6 million in the last year of the initial term. The Company has also agreed to pay an initial annual base rent of approximately $3.2 million for the expansion premises, which increases annually until it reaches approximately $4.2 million in the last year of the initial term for the expansion premises. The amended lease provided the Company with a total tenant improvement allowance of approximately $17.4 million for improvements to be made to the premises. A security deposit of $3.3 million was recorded as restricted cash on the Company’s consolidated balance sheet as of December 31, 2024.

The lease agreements do not contain residual value guarantees and the components of lease cost for the years ended December 31, 2024, 2023 and 2022 were as follows (in thousands):


	Year Ended December 31,
Operating leases:	2024		2023		2022
Lease cost	$	23,238	$	23,190	$	21,830
Sublease income		-		-		(2,132)
Net lease cost	$	23,238	$	23,190	$	19,698

The Company has not entered into any material short-term leases or financing leases as of December 31, 2024.

Supplemental cash flow information related to leases for the years ended December 31, 2024, 2023 and 2022 was as follows (in thousands):


	Year Ended December 31,
	2024		2023		2022
Cash paid for amounts included in the measurement of lease liabilities	$	18,284	$	17,741	$	15,891
Lease liabilities arising from obtaining right-of-use assets:
Operating leases	$	556	$	915	$	72

The weighted average remaining lease term and weighted average discount rate of the operating leases are as follows:

Future minimum lease payments under non-cancellable leases as of December 31, 2024 were as follows (in thousands):



2025	$	18,757
2026		19,318
2027		19,876
2028		20,451
2029		19,268
Thereafter		84
Total future minimum lease payments		97,754
Less imputed interest		(15,618)
Total	$	82,136

Financing Arrangements - Royalty and Sales Information (Details) - Sixth Street Partners $ in Millions	Jul. 31, 2022 USD ($)
Financing Arrangements
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, percentage rate (as a percent)	9.75%
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, amount, maximum	$ 900.0
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, ratio	1.45
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, amount	$ 362.5
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, percentage increase (as a percent)	15.00%
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, ratio	1.85
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, amount	$ 462.5

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

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Property and Equipment, Net - Property and Equipment and Related Accumulated Depreciation (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Property and Equipment, Net
Total cost	$ 84,348	$ 80,453
Less: accumulated depreciation and amortization	(47,755)	(38,494)
Total	36,593	41,959
Lab equipment
Property and Equipment, Net
Total cost	25,720	21,311
Furniture and fixtures
Property and Equipment, Net
Total cost	4,642	4,644
Computer equipment
Property and Equipment, Net
Total cost	2,062	2,028
Leasehold improvements
Property and Equipment, Net
Total cost	50,787	42,301
Software
Property and Equipment, Net
Total cost	902	657
Construction-in-progress
Property and Equipment, Net
Total cost	$ 235	$ 9,512

Commitments and Contingencies - Indemnification Agreements (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Indemnification Agreement
Indemnification Agreements
Loss contingency accrual	$ 0	$ 0

Stock-based Compensation - Employee Stock Purchase Plan (Details) - shares	1 Months Ended	12 Months Ended
	May 31, 2015	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022	Jan. 01, 2025	Jan. 01, 2024
Stock-based compensation
ESPP shares issued during period (in shares)		65,813	96,290	80,717
Employee Stock
Stock-based compensation
Number of common shares reserved for future issuance (in shares)	243,347	4,196,701
Annual increase for common stock for issuance (as a percent)	1.00%
Increase of common shares reserved for future issuance (in shares)					637,122	611,472

Stock-based Compensation - Stock-based Compensation Expense - By Classification (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Total stock based compensation expense
Stock-based compensation expense	$ 108,911	$ 92,672	$ 98,971
Research and Development
Total stock based compensation expense
Stock-based compensation expense	47,455	41,534	40,302
Selling, General and Administrative
Total stock based compensation expense
Stock-based compensation expense	$ 61,456	$ 51,138	$ 58,669

Income Taxes - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Deferred tax assets:
Net operating loss carryforwards	$ 244,189	$ 232,624
Research and development credit carryforwards	67,592	53,329
Orphan drug credit carryforwards	168,799	156,816
Accrued expenses and other	62,187	60,350
Royalty Pharma milestones		12,024
R&D capitalization	190,040	153,836
Financing arrangements	63,424	107,693
Deferred rent	19,040	22,204
Other	3,785	1,488
Total gross deferred tax asset	819,056	800,364
Deferred tax liability
Depreciation	(6,123)	(6,107)
Right of use assets	(14,837)	(17,423)
Other		(2,068)
Valuation allowance	(798,096)	(774,766)
Net deferred tax asset	$ 0	$ 0

Stock-based Compensation - Stock-based Compensation Expense - By Type (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Total stock based compensation expense
Subtotal	$ 109,920	$ 93,256	$ 99,634
Capitalized stock-based compensation costs	(1,009)	(584)	(663)
Stock-based compensation expense included in total cost and operating expenses	108,911	92,672	98,971
Employee Stock Option
Total stock based compensation expense
Subtotal	42,031	40,560	52,931
Restricted Stock Units
Total stock based compensation expense
Subtotal	61,722	50,095	45,285
Performance-based restricted stock units
Total stock based compensation expense
Subtotal	4,445	867
Employee Stock
Total stock based compensation expense
Subtotal	$ 1,722	$ 1,734	$ 1,418

Restricted Cash (Tables)

12 Months Ended

Dec. 31, 2024

Reconciliation of cash, cash equivalents, and restricted cash


	December 31,
	2024		2023		2022
Cash and cash equivalents	$	102,014	$	71,286	$	119,709
Restricted cash		11,625		10,238		5,195
Total cash, cash equivalents, and restricted cash shown in consolidated statements of cash flows	$	113,639	$	81,524	$	124,904

Stock-based Compensation - Restricted Stock Units - Additional Information (Details) - Restricted Stock Units - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Stock-based Compensation
Total fair value of restricted stock units that vested	$ 75.8	$ 31.6	$ 29.4
Vested (in shares)	795,219

Collaboration, License and Other Agreements - Revenue Recognized (Details) - USD ($) $ in Thousands	1 Months Ended	6 Months Ended	12 Months Ended					24 Months Ended
	Aug. 31, 2022	Dec. 31, 2023	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2018	Dec. 31, 2020
Revenues
Revenue			$ 508,824	$ 249,380	$ 204,036
Collaboration, License and Other \| Rigel Pharmaceuticals, Inc.
Revenues
Revenue			24,300
Collaboration, License and Other \| Zai Lab (Shanghai) Co., Ltd.
Revenues
Revenue			0			$ 25,000
Collaboration, License and Other \| Roche, Collaboration and License (Immunotherapy) Agreement
Revenues
Revenue		$ 0	0
Collaboration, License and Other \| Clementia
Revenues
Revenue			0	0	30,000			$ 46,500
Collaboration, License and Other \| C Stone
Revenues
Revenue							$ 40,000
Collaboration revenue \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue			1,920	2,143	7,558
Collaboration revenue \| C Stone
Revenues
Revenue			3,304	17,312	24,294
License milestone revenue \| C Stone
Revenues
Revenue				9,000	6,500
Territory-specific activities, manufacturing services and royalty \| C Stone
Revenues
Revenue			3,304	8,312	17,794
Territory-specific activities, research and development services \| Roche, Collaboration and License (Immunotherapy) Agreement
Revenues
Revenue				25,700	2,300
Territory-specific activities, manufacturing and research and development services \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue			324	679	6,710
Royalty \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue			180	1,464	848
GAVRETO Product Consideration \| Rigel Pharmaceuticals, Inc.
Revenues
Revenue			6,500
GAVRETO profit share in the U.S. \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue			1,416
GAVRETO Performance Obligation Rights in the U.S. \| Rigel Pharmaceuticals, Inc.
Revenues
Revenue			17,600
License \| Related Party
Revenues
Revenue					27,500
License \| IDRx, Inc. \| Director
Revenues
Revenue	$ 27,500		$ 0	$ 0	$ 27,500

Summary of Significant Accounting Policies and Recent Accounting Pronouncements

12 Months Ended

Dec. 31, 2024

Summary of Significant Accounting Policies and Recent Accounting Pronouncements

2. Summary of Significant Accounting Policies and Recent Accounting Pronouncements

Basis of Presentation

The audited consolidated financial statements of the Company included herein have been prepared in accordance with accounting principles generally accepted in the U.S. (GAAP) as found in the Accounting Standards Codification (ASC) and Accounting Standards Updates (ASU) of the Financial Accounting Standards Board (FASB) and the rules and regulations of the Securities and Exchange Commission (SEC).

The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, Blueprint Medicines Security Corporation, which is a Massachusetts subsidiary created to buy, sell and hold securities, Blueprint Medicines (Switzerland) GmbH, Blueprint Medicines (Netherlands) B.V., Blueprint Medicines (UK) Ltd, Blueprint Medicines (Germany) GmbH, Blueprint Medicines (Spain) S.L., Blueprint Medicines (France) SAS, and Blueprint Medicines (Italy) S.r.L. All intercompany transactions and balances have been eliminated.

Use of Estimates

The preparation of financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: revenue recognition, inventory, operating lease right-of-use assets, operating lease liabilities, stock-based compensation expense, accrued expenses, liabilities related to the sale of future royalties and future revenues, equity investment, debt modification, and income taxes.

Significant Accounting Policies

Revenue Recognition

The Company accounts for contracts with customers in accordance with ASC Topic 606, Revenue from Contracts with Customers (ASC 606). The Company recognizes revenue when its customer obtains control of promised goods or services, in an amount that reflects the consideration which the Company expects to receive in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) the Company satisfies a performance obligation. The Company only applies the five-step model to contracts when it is probable that the Company will collect the consideration it is entitled to in exchange for the goods or services it transfers to the customer. At contract inception, once the contract is determined to be within the scope of ASC 606, the Company assesses the goods or services promised within each contract and determines those that are performance obligations, and assesses whether each promised good or service is distinct. The Company then recognizes as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied.

Consideration received prior to revenue recognition is recorded as deferred revenue in the consolidated balance sheets. Amounts expected to be recognized as revenue within the 12 months following the balance sheet date are classified as current portion of deferred revenue in the accompanying consolidated balance sheets. Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as deferred revenue, net of current portion. Amounts are recorded as accounts receivable if the Company transfer goods or services to a customer before the customer pays consideration and the Company’s right to consideration is unconditional. The Company does not assess whether a contract has a significant financing component if the expectation at contract inception is that the period between payment by the customer and the transfer of the promised goods or services to the customer will be one year or less.

Product revenue

The Company generated product revenue primarily from sales of AYVAKIT in the U.S and sales of AYVAKYT in the European Union to a limited number of specialty distributors and specialty pharmacy providers. These customers subsequently resell the products or dispense the products directly to patients. In addition, the Company entered into arrangements with payors that provide for government mandated rebates, discounts and allowances with respect to the utilization of its products.

Product revenue is recognized when the customer takes control of the product, typically upon delivery to the customer. Product revenue is recorded at the net sales price, or transaction price, which includes estimated reserves for variable consideration resulting from chargebacks, government rebates, trade discounts and allowances, product returns and other incentives that are offered within the contract with customers, healthcare providers, payors and other indirect customers relating to the sales of the Company’s product. Reserves are established based on the amounts earned or to be claimed on the related sales. Where appropriate, the Company utilizes the expected value method to determine the appropriate amount for estimates of variable consideration based on factors such as historical rebate payments for these programs, the Company’s current contractual and statutory requirements, specific known market events and trends, industry data and forecasted customer buying and payment patterns, the percentage of product that are sold via these programs, and the product’s pricing. The amount of variable consideration that is included in the transaction price may be constrained and is included in net product revenues only to the extent that it is probable that a significant reversal in the amount of the cumulative revenue recognized will not occur in a future period. Actual amounts of consideration ultimately received may differ from the Company’s estimates. If actual results vary from the Company’s estimates, the Company adjusts these estimates, which would affect net product revenue and earnings in the period such variances become known.

Chargebacks: Chargebacks for fees and discounts represent the estimated obligations resulting from contractual commitments to sell product to qualified healthcare providers and government agencies at prices lower than the list prices charged to the customers who directly purchase the product from the Company. The customers charge the Company for the difference between what they pay for the product and the ultimate contractually committed or

government required lower selling price to the qualified healthcare providers. These reserves are estimated using the expected value method based upon a range of possible outcomes and are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue.

Government rebates: Government rebates consist of Medicare, Tricare and Medicaid rebates, which were estimated using the expected value method, based upon a range of possible outcomes for the estimated payor mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue. For Medicare, the Company also estimates the number of patients in the prescription drug coverage gap for whom it will owe a rebate under the Medicare Part D program.

Trade discounts and allowances: The Company provides the customers with discounts that are explicitly stated in the contracts and recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, the Company also receives sales order management, inventory management and data services from the customers in exchange for certain fees.

Product returns: The Company estimates the amount of its product sales that may be returned by its customers and records this estimate as a reduction of revenue in the period the related product revenue is recognized. The Company currently estimates product return liabilities using expected value method based on available industry data and its visibility into the inventory remaining in the distribution channel.

Other deductions: Co-pay assistance relates to financial assistance provided to qualified patients, whereby the Company may assist them with prescription drug co-payments required by the patient’s insurance provider. Reserves for co-pay assistance are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue.

Collaboration, license and other revenue

At contract inception, the Company analyzes its collaboration and license arrangements to assess whether such arrangements involve joint operating activities performed by parties that are both active participants in the activities and exposed to significant risks and rewards dependent on the commercial success of such activities and therefore within the scope of ASC Topic 808, Collaborative Arrangements (ASC 808). This assessment is performed throughout the life of the arrangement based on changes in the responsibilities of all parties in the arrangement. For collaboration arrangements within the scope of ASC 808 that contain multiple elements, the Company first determines which elements of the collaboration are deemed to be within the scope of ASC 808 and which elements of the collaboration are more reflective of a vendor-customer relationship and therefore within the scope of ASC 606.

For elements of collaboration arrangements that are accounted for pursuant to ASC 808, an appropriate recognition method is determined and applied consistently, either by analogy to authoritative accounting literature or by applying a reasonable and rational policy election. The Company evaluates the income statement classification for presentation of amounts due from or owed to other participants associated with multiple activities in a collaboration arrangement based on the nature of each separate activity. For the co-commercialization and marketing activities of certain of the Company’s products and product candidates in a collaboration arrangement, where the Company is the principal on sales transactions with third parties, the Company recognizes revenues, cost of sales and operating expenses on a gross basis in their respective lines in its consolidated statements of operations and comprehensive income (loss). Where the Company is not the principal on sales transactions with third parties, the Company records its share of the revenues, cost of sales and operating expenses on a net basis as revenue (expenses) from the collaboration arrangement in its consolidated statements of operations and comprehensive loss.

For elements accounted within scope of ASC 606, to determine the appropriate amount of revenue to be recognized for the arrangements, the Company performs the following steps: (i) identification of the promised goods or services in the contract; (ii) determination of whether the promised goods or services are performance obligations including whether they are distinct in the context of the contract; (iii) measurement of the transaction price, including the constraint on variable consideration; (iv) allocation of the transaction price to the performance obligations; and (v) recognition of revenue when (or as) the Company satisfies each performance obligation. As part of the accounting for these arrangements, the Company must use significant judgment to determine: (a) the performance obligations based on the determination under step (ii) above; (b) the transaction price under step (iii) above; and (c) the stand-alone selling price for each performance obligation identified in the contract for the allocation of transaction price in step (iv) above.

The Company uses judgment to determine whether milestones or other variable consideration, including royalties and sales-based milestones, should be included in the transaction price as described further below. The transaction price is allocated to each performance obligation on a relative stand-alone selling price basis, for which the Company recognizes revenue as or when the performance obligations under the contract are satisfied.

Exclusive Licenses. If the license to the Company’s intellectual property is determined to be distinct from the other promises or performance obligations identified in the arrangement, the Company recognizes revenue from non-refundable, upfront fees allocated to the license when the license is transferred to the customer and the customer is able to use and benefit from the license. In assessing whether a promise or performance obligation is distinct from the other promises, the Company considers factors such as the research, development, manufacturing and commercialization capabilities of the collaboration partner and the availability of the associated expertise in the general marketplace. In addition, the Company considers whether the collaboration partner can benefit from a promise for its intended purpose without the receipt of the remaining promise, whether the value of the promise is dependent on the unsatisfied promise, whether there are other vendors that could provide the remaining promise, and whether it is separately identifiable from the remaining promise. For licenses that are combined with other promises, the Company utilizes judgment to assess the nature of the combined performance obligation to determine whether the combined performance obligation is satisfied over time or at a point in time and, if over time, the appropriate method of measuring progress for purposes of recognizing revenue. The Company evaluates the measure of progress each reporting period and, if necessary, adjusts the measure of performance and related revenue recognition. The measure of progress, and thereby periods over which revenue should be recognized, are subject to estimates by management and may change over the course of the research and development and licensing agreement. Such a change could have a material impact on the amount of revenue the Company records in future periods.

Research and Development Services. The promises under the Company’s collaboration agreements may include research and development services to be performed by the Company on behalf of the partner. Payments or reimbursements resulting from the Company’s research and development efforts are recognized as revenue when the services are performed and presented on a gross basis because the Company is the principal for such efforts. Payments or reimbursements from the partner that are the result of a collaborative relationship with the partner, instead of a customer relationship, such as co-development activities, are recorded as a reduction to research and development expense.

Customer Options. If an arrangement is determined to contain customer options that allow the customer to acquire additional goods or services, the goods and services underlying the customer options that are not determined to be material rights are not considered to be performance obligations at the outset of the arrangement, as they are contingent upon option exercise. The Company evaluates the customer options for material rights, or options to acquire additional goods or services for free or at a discount. If the customer options are determined to represent a material right, the material right is recognized as a separate performance obligation at the outset of the arrangement. The Company allocates the transaction price to material rights based on the relative standalone selling price, which is determined based on the identified discount and the probability that the customer will exercise the option. Amounts allocated to a material right are not recognized as revenue until, at the earliest, the option is exercised.

Milestone Payments. At the inception of each arrangement that includes research or development milestone payments, the Company evaluates whether the milestones are considered probable of being achieved and estimates the amount to be included in the transaction price using the most likely amount method. If it is probable that a significant revenue reversal would not occur, the associated milestone value is included in the transaction price. Milestone payments that are not within the control of the Company or the licensee, such as regulatory approvals, are not considered probable of being achieved until those approvals are received. The Company evaluates factors such as the scientific, clinical, regulatory, commercial, and other risks that must be overcome to achieve the particular milestone in making this assessment. There is considerable judgment involved in determining whether it is probable that a significant revenue reversal would not occur. At the end of each subsequent reporting period, the Company reevaluates the probability of achievement of all milestones subject to constraint and, if necessary, adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment.

Royalties. For license arrangements that include sales-based royalties, including milestone payments upon first commercial sales and milestone payments based on a level of sales, which are the result of a customer-vendor relationship and for which the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some

or all of the royalty has been allocated has been satisfied or partially satisfied. For arrangements involving the sale of intellectual property to customers, the Company estimates and recognizes revenue upon satisfying the related performance obligation, including any royalty payments deemed probable of achievement and not subject to significant subsequent revenue reversal. At the end of each subsequent reporting period, the Company reevaluates the expected royalties and adjusts its estimate of the overall transaction price. Any such adjustments are recorded on a cumulative catch-up basis, which would affect revenues and earnings in the period of adjustment.

For a complete discussion of accounting for collaboration, license and other revenues, see Note 11, Collaboration, License and Other Agreements.

Accounts Receivable, net

Accounts receivable arise from product sales and amounts due from the Company’s collaboration partners. The amount from product sales primarily represents amounts due from specialty distributors and specialty pharmacy providers in the U.S. The Company provides reserves against accounts receivable for current expected credit losses that are estimated based on the composition of its accounts receivable and the financial condition of the counterparty, considering past events, current economic conditions, and reasonable and supportable forecasts about the future economic conditions. The contractual life of our accounts receivable is generally short-term. Amounts determined to be uncollectible are charged or written off against the reserve. For the years ended December 31, 2024, 2023 and 2022, the Company did not record any significant expected credit losses related to outstanding accounts receivable.

Inventory

Inventories are stated at the lower of cost or estimated net realizable value with cost based on the first-in first-out method. Inventory that can be used in either the production of clinical or commercial products is expensed as research and development costs when identified for use in clinical trials. The Company classifies its inventory costs as long-term when it expects to utilize the inventory beyond its normal operating cycle and includes these costs in other assets in the consolidated balance sheets.

Prior to the regulatory approval of its drug candidates, the Company incurs expenses for the manufacture of drug product supplies to support clinical development that could potentially be available to support the commercial launch of those drugs. Until the date at which regulatory approval has been received or is otherwise considered probable, the Company records all such costs as research and development expenses.

The Company performs an assessment of the recoverability of capitalized inventories during each reporting period and writes down any excess and obsolete inventory to its net realizable value in the period in which the impairment is first identified. Such impairment charges, should they occur, are recorded as a component of cost of product sales in the consolidated statements of operations and comprehensive loss. The determination of whether inventory costs will be realizable requires the use of estimates by management. If actual market conditions are less favorable than projected by management, additional write-downs of inventory may be required.

Fair Value Measurements

The Company has certain financial assets and liabilities recorded at fair value which have been classified as Level 1, 2 or 3 within the fair value hierarchy as described in the accounting standards for fair value measurements.

●

Level 1 — Fair values are determined utilizing quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access;

●

Level 2 — Fair values are determined by utilizing quoted prices for identical or similar assets and liabilities in active markets or other market observable inputs such as interest rates, yield curves and foreign currency spot rates; and

●

Level 3 — inputs are unobservable inputs that reflect the Company’s own assumptions about the assumptions market participants would use in pricing the asset or liability. Financial assets and liabilities are classified in their entirety based on the lowest level of input that is significant to the fair value measurement.

The Company’s financial assets, which include cash equivalents and marketable securities, have been initially valued at the transaction price, and subsequently revalued at the end of each reporting period, utilizing third-party pricing services. The pricing services utilize industry standard valuation models, including both income and market based approaches, to determine value.

There have been no changes to the valuation methods during the years ended December 31, 2024, 2023 and 2022.

Cash and cash equivalents

The Company considers all highly liquid investments purchased with original maturities of 90 days or less from the date of purchase to be cash equivalents. As of December 31, 2024 and 2023, the Company’s cash equivalents comprised of money market funds with less than 90 days from the date of purchase. Cash equivalents are reported at fair value.

Marketable securities

The Company classifies its investments in marketable debt securities as available-for-sale when their original maturities, from the date of purchase, exceed three months. Available-for-sale debt securities with a remaining maturity date greater than one year are classified as non-current assets. Available-for-sale marketable debt securities are maintained by an investment manager and mainly consist of U.S. treasury securities and U.S. government agency securities. Available-for-sale marketable debt securities are carried at fair value with the unrealized gains and losses included in other comprehensive income (loss) as a component of stockholders’ equity until realized. Any premium or discount arising at purchase is amortized and/or accreted to interest income and/or expense over the life of the instrument. Realized gains and losses are determined using the specific identification method and are included in other income (expense). Realized losses are recognized only if the Company has the intention to sell the security or it is expecting to be required to sell the security before recovery of its amortized cost basis. The Company also reviews its portfolio of available-for-sale debt securities, using both quantitative and qualitative factors, to determine if declines in fair value below cost have resulted from a credit-related loss. If the decline in fair value is due to credit-related factors, a loss is recognized in other income (expense).

Comprehensive Income (Loss)

Comprehensive income (loss) is defined as the change in equity of a business enterprise during a period from transactions and other events and circumstances from non-owner sources. Other comprehensive income (loss) consisted of foreign currency translation adjustments, unrealized gains and losses on available-for-sale investments and unrealized gains and losses on pension benefit obligations.

Research and Development Expenses

Expenditures relating to research and development are expensed in the period incurred. Research and development expenses consist of both internal and external costs associated with the development of the Company’s selective therapies and building of its discovery platform. As part of the process of preparing the consolidated financial statements, the Company accrues costs for clinical trial activities based upon estimates of the services received and related expenses incurred that have yet to be invoiced by the contract research organizations or other clinical trial vendors that perform the activities.

In certain circumstances, the Company is required to make nonrefundable advance payments to vendors for goods or services that will be received in the future for use in research and development activities. In such circumstances, the nonrefundable advance payments are deferred and capitalized, even when there is no alternative future use for the research and development, until related goods or services are provided. In circumstances where amounts have been paid in excess of costs incurred, the Company records a prepaid expense.

Selling, General and Administrative Expenses

Selling, general and administrative expenses are primarily comprised of compensation and benefits associated with sales and marketing, finance, human resources, legal, information technology and other administrative personnel,

business development, advertising and legal expenses and other general and administrative costs. Advertising costs are expensed as incurred. For years ended December 31, 2024, 2023 and 2022, advertising costs totaled $31.1 million, $16.9 million and $18.1 million, respectively.

Property and Equipment, Net

Property and equipment consists of lab equipment, furniture and fixtures, computer equipment, software, and leasehold improvements, all of which is stated at cost. Expenditures for maintenance and repairs are recorded to expense as incurred, whereas major betterments are capitalized as additions to property and equipment. Depreciation is recognized over the estimated useful lives of the assets using the straight-line method.

Impairment of Long-Lived Assets

The Company continually evaluates whether events or circumstances have occurred that indicate that the carrying amount of any of its long-lived assets might not be recoverable and that impairment might exist. If indicators of impairment are present, the Company performs a test for recoverability. The Company has not recognized any significant impairment charges associated with long-lived assets for the years ended December 31, 2024, 2023 and 2022.

Leases

Leases are accounted for in accordance with ASC Topic 842, Leases (ASC 842). At the inception of a contract, the Company assesses whether the contract is, or contains, a lease. The assessment is based on: (1) whether the contract involves the use of a distinct identified asset, (2) whether the Company obtains the right to substantially all the economic benefit from the use of the asset throughout the period, and (3) whether the Company has the right to direct the use of the asset. At inception of a lease, the Company allocates the consideration in the contract to each lease component based on its relative stand-alone price to determine the lease payments.

Leases are classified as either finance leases or operating leases. A lease is classified as a finance lease if any one of the following criteria are met: the lease transfers ownership of the asset by the end of the lease term, the lease contains an option to purchase the asset that is reasonably certain to be exercised, the lease term is for a major part of the remaining useful life of the asset or the present value of the lease payments equals or exceeds substantially all of the fair value of the asset. A lease is classified as an operating lease if it does not meet any of these criteria.

For all leases at the lease commencement date, a right-of-use asset and a lease liability are recognized. The right-of-use asset represents the right to use the leased asset for the lease term. The lease liability represents the present value of the lease payments under the lease.

The right-of-use asset is initially measured at cost, which primarily comprises the initial amount of the lease liability, plus any initial direct costs incurred if any, less any lease incentives received. All right-of-use assets are reviewed for impairment. The lease liability is initially measured at the present value of the lease payments, discounted using the interest rate implicit in the lease or, if that rate cannot be readily determined, the secured incremental borrowing rate for the same term as the underlying lease.

Lease payments included in the measurement of the lease liability comprise the following: the fixed noncancelable lease payments, payments for optional renewal periods where it is reasonably certain the renewal period will be exercised, and payments for early termination options unless it is reasonably certain the lease will not be terminated early.

Lease cost for operating leases consists of the lease payments plus any initial direct costs, primarily brokerage commissions, and is recognized on a straight-line basis over the lease term. Included in lease cost are any variable lease payments incurred in the period that are not included in the initial lease liability and lease payments incurred in the period for any leases with an initial term of 12 months or less. Lease cost for finance leases consists of the amortization of the right-of-use asset on a straight-line basis over the lease term and interest expense determined on an amortized cost basis. The lease payments are allocated between a reduction of the lease liability and interest expense.

The Company has made an accounting policy election to not recognize leases with an initial term of 12 months or less within its consolidated balance sheets and to recognize those lease payments on a straight-line basis in its consolidated statements of income over the lease term.

Stock-Based Compensation Expense

Stock-based compensation awards are accounted for in accordance with ASC Topic 718, Compensation –Stock Compensation (ASC 718). The Company expenses the fair value of stock awards granted to employees and members of the board of directors over the requisite service period, which is typically the vesting period. Compensation cost for stock-based awards issued to employees is measured using the estimated fair value at the grant date and is adjusted to reflect actual forfeitures.

Fair value of restricted stock awards granted to employees is based upon the quoted closing market price per share on the date of grant. Fair value of options granted to employees at the date of grant are estimated using the Black-Scholes option-pricing model that requires management to apply judgment and make estimates, including:

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expected volatility, which was calculated using the historical volatility of the Company’s publicly traded stock. The Company computed the historical volatility data using the daily closing prices during the equivalent period of the calculated expected term of its stock-based awards;

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risk-free interest rate, which is based on the U.S. Treasury yield curve in effect at the time of grant commensurate with the expected term assumption;

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expected term, which is calculated using the simplified method, as prescribed by the Securities and Exchange Commission Staff Accounting Bulletin No. 107, Share-Based Payment, as the Company has insufficient historical information regarding its stock options to provide a basis for an estimate. Under this approach, the weighted-average expected life is presumed to be the average of the contractual term of ten years and the weighted-average vesting term of the stock options, taking into consideration multiple vesting tranches; and

●

dividend yield, which is zero based on the fact that the Company never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future.

Fair value of performance-based restricted stock unit awards (PSUs) at the grant date are estimated using a lattice model with a Monte Carlo simulation. This valuation methodology utilizes several key assumptions, including defined consecutive trading day average closing stock price on the grant date, valuation date stock price, expected volatilities using historical volatilities, correlation coefficients based on the volatility data, risk-free rates of return and expected dividend yield. The probability of actual shares expected to be earned is considered in the grant date valuation and the expense is not adjusted to reflect the actual units earned. The PSUs cliff vest at the end of the service period and the compensation expense for PSUs is recognized straight-line over the applicable service period.

Stock-based awards issued to non-employees, including directors for non-board-related services, are accounted for based on the fair value of such services received or the fair value of the awards granted on the grant date, whichever is more reliably measured. Stock-based awards subject to service-based vesting conditions are expensed on a straight-line basis over the vesting period.

The purchase price of common stock under the Company’s 2015 employee stock purchase plan (as amended, the 2015 ESPP) is equal to 85% of the lesser of (i) the fair market value per share of the common stock on the first business day of an offering period and (ii) the fair market value per share of the common stock on the purchase date. The fair value of the discounted purchases made under 2015 ESPP is calculated using the Black-Scholes valuation model. The fair value of the look-back provision plus the 15% discount is recognized as compensation expense over the 180-day purchase period.

Equity Investment

Investments in non-marketable equity securities of privately-held companies that do not have readily determinable fair values are carried at cost, less any impairments, plus or minus changes resulting from observable price

changes in orderly transactions for the identical or a similar investment of the same issuer. Each period the Company assesses relevant transactions to identify observable price changes, and the Company regularly monitors these investments to evaluate whether there is an indication of impairment. The Company evaluates whether an investment’s fair value is less than its carrying value using an estimate of fair value, if such an estimate is available. For periods in which there is no estimate of fair value, the Company evaluates whether an event or change in circumstances has occurred that may have a significant adverse effect on the value of the investment.

Liabilities related to the sale of future royalties and revenues

The Company accounts for net proceeds from sales of the Company’s rights to receive future royalty payments and from sales of future revenues as liabilities if the Company has significant continuing involvement in the generation of the related future cash flows. Interest expenses on the liabilities related to the sale of future royalties and revenues are recognized using the effective interest rate method over the life of the related royalty or revenue stream. The liabilities related to the sale of future royalties and revenues and the related interest expenses are based on the Company’s current estimates of future royalties or revenues as well as commercial milestones expected to be achieved and received over the life of the arrangement, which the Company determines by using forecasts of the underlying drug products of the underlying regions. The Company periodically assesses the expected payments and to the extent the amount or timing of the future estimated payments is materially different than previous estimates, the Company accounts for any such change by adjusting the carrying value of the liabilities related to the sale of future royalties and revenues, and prospectively recognizing the related interest expenses.

Modification of debt instruments

When the Company’s debt instrument, where the Company is the debtor, is modified or exchanged, the Company analyzes whether the modification should be accounted for as a modification or an extinguishment. Modified terms are considered substantially different if, after an exchange or modification of debt instruments with the same creditor, the present value of cash flows under the terms of the new debt instrument differs by at least 10% from the present value of the remaining cash flows under the terms of the original debt instrument. If the modified instrument is considered substantially different from the original debt instrument, the modification or exchange is accounted for as an extinguishment. The new instrument is recorded at its fair value and that fair value is used to determine the extinguishment gain or loss.

Income Taxes

The Company recognizes deferred tax assets and liabilities for the expected future tax consequences of events that have been recognized in the Company’s financial statements or tax returns. Under this method, deferred tax assets and liabilities are determined based on differences between the financial statement carrying amounts and the tax bases of the assets and liabilities using the enacted tax rates in effect in the years in which the differences are expected to reverse. A valuation allowance against deferred tax assets is recorded if, based on the weight of the available evidence, it is more likely than not that some or all of the deferred tax assets will not be realized. The Company accounts for uncertain tax positions using a more-likely-than-not threshold for recognizing and resolving uncertain tax positions. The evaluation of uncertain tax positions is based on factors including, but not limited to, changes in the law, the measurement of tax positions taken or expected to be taken in tax returns, the effective settlement of matters subject to audit, new audit activity, and changes in facts or circumstances related to a tax position.

Foreign currency translation

The financial statements of each of the Company’s subsidiaries with a functional currency other than the U.S. dollar are translated into U.S. dollars using period-end exchange rates for assets and liabilities, historical exchange rates for stockholders’ equity and weighted average exchange rates for operating results. Translation gains and losses are included in accumulated other comprehensive income (loss) in stockholders’ equity. Foreign currency transaction gains and losses are included in other (expense) income, net in the results of operations.

Concentrations of Credit Risk and of Significant Suppliers

The Company has no significant off-balance-sheet risk such as foreign exchange contracts, option contracts, or other foreign hedging arrangements. Financial instruments that potentially expose the Company to concentrations of

credit risk primarily consist of cash and cash equivalents, marketable securities, accounts receivable and unbilled account receivables.

The Company maintains its cash, cash equivalents and marketable securities in custodian accounts at high quality financial institutions, and as of December 31, 2024 and 2023, substantially all the Company’s cash, cash equivalents and marketable securities were invested in money market funds and U.S. government agency securities and treasury obligations, and consequently, the Company believes that such funds are subject to minimal credit risk. The Company has adopted an investment policy that limits the amounts the Company may invest in any one type of investment. The Company has not experienced any significant credit losses and does not believe it is exposed to any significant credit risk on these funds.

Accounts receivables and unbilled accounts receivables represent amounts arising from product sales and amounts due from the Company’s collaboration partners. The Company monitors economic conditions to identify facts or circumstances that may indicate that its receivables are at risk of collection. The Company has not experienced any credit losses and does not believe it is exposed to any significant credit risk on these funds.

The Company is dependent on third-party manufacturers to supply products for commercial and research and development activities associated with its drug and drug candidates, as applicable. In particular, the Company relies and expects to continue to rely on a small number of manufacturers to supply it with its requirements for the active pharmaceutical ingredients and formulated drugs related to the Company's drug and drug candidate activities. These activities, including the commercialization of AYVAKIT/AYVAKYT, could be adversely affected by a significant interruption in the supply of active pharmaceutical ingredients and formulated drugs.

Segment and Geographic Information

Operating segments are identified as components of an enterprise about which separate discrete financial information is available for evaluation by the chief operating decision maker in making decisions on how to allocate resources and assess performance. The Company’s chief operating decision maker is the chief executive officer. The chief operating decision maker views the Company’s operations and manage its business as one operating segment. The Company operates in the U.S. and Europe. All material long-lived assets of the Company reside in the U.S. For additional information, see Note 18, Segment Information.

New Accounting Pronouncements

From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies that the Company adopts as of the specified effective date. Unless otherwise discussed below, the Company does not believe that the adoption of recently issued standards have or may have a material impact on its consolidated financial statements and disclosures.

In November 2023, the FASB issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures, which is intended to improve reportable segment disclosure requirements, primarily through additional disclosures about significant segment expenses. The standard is effective for fiscal years beginning after December 15, 2023, and interim periods within fiscal years beginning after December 15, 2024, with early adoption permitted. The Company adopted the new standard in fiscal year 2024 for annual and retrospective reporting periods with all interim disclosures to begin in the first quarter of fiscal year 2025. For additional information, see Note 18, Segment Information.

In December 2023, the FASB issued ASU 2023-09, Improvements to Income Tax Disclosures, which requires entities to disclose disaggregated information about their effective tax rate reconciliation and income taxes paid. The disclosure requirements will be applied on a prospective basis, with the option to apply them retrospectively. The standard is effective for fiscal years beginning after December 15, 2024, with early adoption permitted. The Company is currently evaluating the disclosure requirements related to this new standard.

In November 2024, the FASB issued ASU 2024-03, Income Statement—Reporting Comprehensive Income—Expense Disaggregation Disclosures, which requires disclosure of additional information about specific expense categories in the notes to the financial statements on an interim and annual basis. The standard is effective for fiscal years beginning after December 15, 2026, and for interim periods beginning after December 15, 2027, with early adoption permitted. The Company is currently evaluating the disclosure requirements related to this new standard.

Reclassification

Certain items in the prior year’s consolidated financial statements have been reclassified to conform to the current presentation.

Property and Equipment, Net - Depreciation Expense (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Depreciation expense
Depreciation expense	$ 9.7	$ 7.7	$ 6.5

Fair Value of Financial Instruments

12 Months Ended

Dec. 31, 2024

Fair Value of Financial Instruments

5. Fair Value of Financial Instruments

The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2024 (in thousands):


			Active		Observable		Unobservable
	December 31,		Markets		Inputs		Inputs
Description	2024		(Level 1)		(Level 2)		(Level 3)
Cash equivalents:
Money market funds	$	69,729	$	69,729	$	—	$	—
Marketable securities, available-for-sale:
U.S. government agency securities		129,785		—		129,785		—
U.S. treasury obligations		632,138		632,138		—		—
Total	$	831,652	$	701,867	$	129,785	$	—

The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2023 (in thousands):


			Active		Observable		Unobservable
	December 31,		Markets		Inputs		Inputs
Description	2023		(Level 1)		(Level 2)		(Level 3)
Cash equivalents:
Money market funds	$	55,412	$	55,412	$	—	$	—
Marketable securities, available-for-sale:
U.S. government agency securities		169,803				169,803		—
U.S. treasury obligations		526,082		526,082		—		—
Total	$	751,297	$	581,494	$	169,803	$	—

The entire disclosure for the fair value of financial instruments (as defined), including financial assets and financial liabilities (collectively, as defined), and the measurements of those instruments as well as disclosures related to the fair value of non-financial assets and liabilities. Such disclosures about the financial instruments, assets, and liabilities would include: (1) the fair value of the required items together with their carrying amounts (as appropriate); (2) for items for which it is not practicable to estimate fair value, disclosure would include: (a) information pertinent to estimating fair value (including, carrying amount, effective interest rate, and maturity, and (b) the reasons why it is not practicable to estimate fair value; (3) significant concentrations of credit risk including: (a) information about the activity, region, or economic characteristics identifying a concentration, (b) the maximum amount of loss the entity is exposed to based on the gross fair value of the related item, (c) policy for requiring collateral or other security and information as to accessing such collateral or security, and (d) the nature and brief description of such collateral or security; (4) quantitative information about market risks and how such risks are managed; (5) for items measured on both a recurring and nonrecurring basis information regarding the inputs used to develop the fair value measurement; and (6) for items presented in the financial statement for which fair value measurement is elected: (a) information necessary to understand the reasons for the election, (b) discussion of the effect of fair value changes on earnings, (c) a description of [similar groups] items for which the election is made and the relation thereof to the balance sheet, the aggregate carrying value of items included in the balance sheet that are not eligible for the election; (7) all other required (as defined) and desired information.

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Income Taxes - Expense (Benefit) (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
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Income tax expense	$ 1,226	$ 968	$ 5,236

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Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Recent Accounting Pronouncements (Details)	Dec. 31, 2024
Accounting Standards Update 2023-07
Summary of Significant Accounting Policies and Recent Accounting Pronouncements
Change in Accounting Principle, Accounting Standards Update, Adopted	true
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Summary of Significant Accounting Policies and Recent Accounting Pronouncements
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Stockholders' Equity

12 Months Ended

Dec. 31, 2024

Stockholders' Equity

12. Stockholders’ Equity

In February 2022, the Company entered into an at-the-market (ATM) Facility with Cowen and Company, LLC (Cowen), pursuant to which the Company may offer and sell, from time to time at its sole discretion, shares of its common stock having an aggregate offering price of up to $300.0 million through Cowen as sales agent. During the year ended December 31, 2024, the Company issued and sold 544,719 shares of its common stock under the ATM Facility and received net proceeds of $48.9 million. The Company did not issue any shares under the ATM Facility during the years ended December 31, 2023 and 2022.

The entire disclosure for equity.

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Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Selling, General and Administrative Expenses (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Selling, General and Administrative Expenses
Advertising costs	$ 31.1	$ 16.9	$ 18.1

Employee Benefit Plans - Switzerland Defined Benefit Plan - Tabular Disclosure (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Accumulated Benefit Obligation in Excess of Plan Assets
Fair value of plan assets	$ 9,241	$ 8,416
Projected benefit obligation	(16,065)	(14,632)
Accumulated benefit obligation	12,071	10,984
Unfunded Status
Unfunded status recorded as other long-term liabilities	$ (6,824)	$ (6,216)

Product Revenue - Product Revenue (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Revenues
Revenue	$ 508,824	$ 249,380	$ 204,036
Product revenue, net
Revenues
Revenue	478,950	204,207	110,993
Product revenue, net \| United States
Revenues
Revenue	421,837	181,971	97,226
Product revenue, net \| Rest of World
Revenues
Revenue	$ 57,113	$ 22,236	$ 13,767

Amount, including tax collected from customer, of revenue from satisfaction of performance obligation by transferring promised good or service to customer. Tax collected from customer is tax assessed by governmental authority that is both imposed on and concurrent with specific revenue-producing transaction, including, but not limited to, sales, use, value-added and excise.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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-Topic 606
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Collaboration, License and Other Agreements - Reduction in Expenses (Details) - Collaborative Arrangement - USD ($) $ in Thousands	6 Months Ended	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Roche, Collaboration (Pralsetnib) Agreement
Collaboration, License and Other Agreements
Collaborative arrangement, commercialization, reduction in selling, general and administrative expenses	$ 0	$ 980	$ 10,982	$ 16,600
Collaborative arrangement, global development activities, increase (reduction) in research and development expenses	$ 0	$ (1,103)	$ (24,915)	(19,297)
C Stone
Collaboration, License and Other Agreements
Collaborative arrangement, reduction in manufacturing and research and development expenses				$ 1,700

Product Revenue - Concentration Risk (Details)	12 Months Ended
Product Revenue - Concentration Risk (Details)	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Revenue from Contract with Customer Benchmark \| Customer Concentration Risk \| Customer One
Concentration Risk
Concentration risk (as a percent)	39.00%	43.00%	45.00%
Revenue from Contract with Customer Benchmark \| Customer Concentration Risk \| Customer Two
Concentration Risk
Concentration risk (as a percent)	12.00%
Revenue from Contract with Customer Benchmark \| Customer Concentration Risk \| Customer Three
Concentration Risk
Concentration risk (as a percent)		10.00%	11.00%
Accounts Receivable, Net \| Credit Concentration Risk \| Customer One
Concentration Risk
Concentration risk (as a percent)	31.00%	34.00%
Accounts Receivable, Net \| Credit Concentration Risk \| Customer Two
Concentration Risk
Concentration risk (as a percent)	14.00%
Accounts Receivable, Net \| Credit Concentration Risk \| Customer Three
Concentration Risk
Concentration risk (as a percent)	11.00%	11.00%
Accounts Receivable, Net \| Credit Concentration Risk \| Customer Four
Concentration Risk
Concentration risk (as a percent)		10.00%
Accounts Receivable, Net \| Credit Concentration Risk \| Customer Five
Concentration Risk
Concentration risk (as a percent)		10.00%

Inventory - Balance Sheet Classification (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Inventory
Inventory	$ 13,611	$ 21,223
Other assets	25,664	15,879
Total	$ 39,275	$ 37,102

Property and Equipment, Net - Estimated Useful Lives (Details)	Dec. 31, 2024
Lab equipment
Property and Equipment, Net
Estimated Useful Life (Years)	5 years
Furniture and fixtures
Property and Equipment, Net
Estimated Useful Life (Years)	4 years
Computer equipment
Property and Equipment, Net
Estimated Useful Life (Years)	3 years
Leasehold improvements
Property and Equipment, Net
Property, Plant, and Equipment, Useful Life, Term, Description	us-gaap:UsefulLifeTermOfLeaseMember
Software
Property and Equipment, Net
Estimated Useful Life (Years)	3 years

Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Common Stock	Additional Paid-In Capital	Accumulated Other Comprehensive Loss	Accumulated Deficit	Total
Beginning Balance at Dec. 31, 2021	$ 59	$ 2,250,250	$ (4,133)	$ (1,275,441)	$ 970,735
Beginning Balance (in shares) at Dec. 31, 2021	59,141,086
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan and stock purchase plan	$ 1	8,134			$ 8,135
Issuance of common stock under stock plan and stock purchase plan (in shares)	817,833
At-the-market offerings, net of issuance costs (in shares)					0
Stock-based compensation expense		99,634			$ 99,634
Other comprehensive income (loss)			(6,310)		(6,310)
Net income (loss)				(557,517)	(557,517)
Ending Balance at Dec. 31, 2022	$ 60	2,358,018	(10,443)	(1,832,958)	514,677
Ending Balance (in shares) at Dec. 31, 2022	59,958,919
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan and stock purchase plan	$ 1	22,711			$ 22,712
Issuance of common stock under stock plan and stock purchase plan (in shares)	1,188,317
At-the-market offerings, net of issuance costs (in shares)					0
Stock-based compensation expense		93,256			$ 93,256
Other comprehensive income (loss)			6,948		6,948
Net income (loss)				(506,984)	(506,984)
Ending Balance at Dec. 31, 2023	$ 61	2,473,985	(3,495)	(2,339,942)	$ 130,609
Ending Balance (in shares) at Dec. 31, 2023	61,147,236				61,147,236
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan and stock purchase plan	$ 2	76,365			$ 76,367
Issuance of common stock under stock plan and stock purchase plan (in shares)	2,020,301
At-the-market offerings, net of issuance costs	$ 1	48,935			$ 48,936
At-the-market offerings, net of issuance costs (in shares)	544,719				544,719
Stock-based compensation expense		109,920			$ 109,920
Other comprehensive income (loss)			(56)		(56)
Other		(22)			(22)
Net income (loss)				(67,089)	(67,089)
Ending Balance at Dec. 31, 2024	$ 64	$ 2,709,183	$ (3,551)	$ (2,407,031)	$ 298,665
Ending Balance (in shares) at Dec. 31, 2024	63,712,256				63,712,256

Stock-based Compensation - Stock Options - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Additional disclosures
Weighted-average grant date fair value of options granted	$ 54.15	$ 25.81	$ 32.3
Total intrinsic value of options exercised	$ 45.1	$ 11.6	$ 11.8

Income Taxes - Tax Credit Carryforwards (Details) $ in Millions	Dec. 31, 2024 USD ($)
Foreign Tax Authority
Tax Credit Carryforwards
Tax credit carryforwards	$ 2.5
Research and Development Tax Credit \| Domestic Tax Authority
Tax Credit Carryforwards
Tax credit carryforwards	31.0
Research and Development Tax Credit \| State and Local Jurisdiction
Tax Credit Carryforwards
Tax credit carryforwards	39.9
Orphan Drug Tax Credit \| Domestic Tax Authority
Tax Credit Carryforwards
Tax credit carryforwards	168.8
Investment Tax Credit \| State and Local Jurisdiction
Tax Credit Carryforwards
Tax credit carryforwards	$ 0.9

Commitments and Contingencies - Purchase Commitments (Details) - Manufacturing Agreement, October 2020 $ in Millions	12 Months Ended
	Dec. 31, 2024 USD ($)
Clinical and Commercial Supply Agreements
Unrecorded unconditional purchase obligation, term	5 years
Unrecorded unconditional purchase obligation, future minimum purchase obligations	$ 2.0

Financing Arrangements - General Information (Details) $ in Millions		1 Months Ended	12 Months Ended
	Jun. 30, 2022 USD ($)	Jul. 31, 2022 USD ($) tranche Transaction	Dec. 31, 2022 USD ($)	Dec. 31, 2024 USD ($)
Sixth Street Partners, Financing Arrangements
Debt Financing
Debt instrument, transactions, number \| Transaction		2
Sale of Future Royalties and Revenues
Gross Proceeds
Gross proceeds			$ 425.0
Sale of Future Royalties and Revenues \| Royalty Pharma, Royalty Purchase Agreement
Debt Financing
Debt instrument, issuance date	Jun. 30, 2022
Gross Proceeds
Gross proceeds	$ 175.0
Sale of Future Royalties and Revenues \| Sixth Street Partners, Future Revenue Purchase Agreement
Debt Financing
Debt instrument, face amount		$ 250.0
Transaction costs, gross		5.4
Gross Proceeds
Gross proceeds		250.0
Effective Interest Rate
Debt instrument, interest rate, effective percentage (as a percent)				10.30%
Loans Payable \| Sixth Street Partners, Financing Agreement
Debt Financing
Debt instrument, face amount, potential amount		660.0
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded, each day thereafter		$ 80.0
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities
Debt Financing
Debt instrument, maturity date		Jun. 30, 2028
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities \| Secured Overnight Financing Rate (SOFR)
Debt Financing
Debt instrument, basis spread on variable rate		6.50%
Debt instrument, variable rate floor		1.00%
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities \| Base Rate
Debt Financing
Debt instrument, basis spread on variable rate		5.50%
Debt instrument, variable rate floor		2.00%
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility
Debt Financing
Debt instrument, face amount		$ 150.0
Effective Interest Rate
Debt instrument, interest rate, effective percentage (as a percent)				12.00%
Secured Debt \| Sixth Street Partners, Financing Agreement, 1st Senior Secured Delayed Draw Term Loan Facility
Debt Financing
Line of credit facility, maximum borrowing capacity		$ 250.0
Debt instrument, tranches, potential, number \| tranche		2
Secured Debt \| Sixth Street Partners, Financing Agreement, Incremental Term Loan
Debt Financing
Debt instrument, face amount, potential amount				$ 260.0

Marketable Securities - Maturities (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Amortized cost
Within one year, amortized cost	$ 512,515	$ 639,881
After one through five years, amortized cost	248,896	56,193
Amortized cost	761,411	696,074
Fair value
Within one year, fair value	513,473	639,355
After one through five years, fair value	248,450	56,530
Investments, available-for-sale	$ 761,923	$ 695,885

Amount of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

Reference 1: http://www.xbrl.org/2003/role/exampleRef
-Topic 820
-SubTopic 10
-Name Accounting Standards Codification
-Section 55
-Paragraph 103
-Publisher FASB
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Inventory - Capitalized (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Inventory
Raw materials		$ 3,147
Work in process	$ 30,300	29,132
Finished goods	8,975	4,823
Total	$ 39,275	$ 37,102

Commitments and Contingencies

12 Months Ended

Dec. 31, 2024

Commitments and Contingencies

19. Commitments and Contingencies

Purchase Commitments Associated with Clinical and Commercial Supply Agreements

In connection with the commercialization of AYVAKIT/AYVAKYT, the Company has negotiated manufacturing agreements with certain vendors that require the Company to meet minimum purchase obligations on an annual basis. The aggregate amount of future unconditional purchase obligations under these manufacturing agreements over the period of next five years is approximately $2.0 million as of December 31, 2024.

Legal Proceedings

In the normal course of business, the Company from time to time is named as a party to various legal claims, actions and complaints, which have included and may include matters involving securities, employment, intellectual property, arising from the use of therapeutics utilizing its technology, or others. The Company records a loss contingency reserve for a legal proceeding when it considers the potential loss probable and it can reasonably estimate the amount of the loss or determine a probable range of loss. The Company provides disclosure when it considers a loss reasonably possible or when it determines that a loss in excess of a reserve is reasonably possible. The Company provides an estimate of such reasonably possible losses or an aggregate range of such reasonably possible losses, unless the Company believes that such an estimate cannot be made. The Company expenses the costs related to its legal proceedings as they are incurred. As of December 31, 2024, the Company has not recorded any significant accruals for loss contingencies.

On June 7, 2024, a purported stockholder filed a putative class action lawsuit against the Company in the Court of Chancery of the State of Delaware, with the caption Johnson v. Blueprint Medicines Corporation, Case No. 2024-0625. Plaintiff claims in the complaint that a “Proxy Access” provision in the Company’s Amended and Restated Bylaws, effective November 30, 2022, is invalid under Delaware law because it allegedly usurps the right of stockholders to select the members of the board of directors, and plaintiff seeks declaratory relief invalidating that provision, as well as attorneys’ fees and costs. On October 7, 2024, the lawsuit was consolidated with twelve other lawsuits against companies with similar bylaw provisions under the caption In re Irrevocable Resignation Bylaw Litigation, Consolidated C.A. No. 2024-0538-JTL. On October 11, 2024, the Company, together with the other companies in the consolidated action, filed an opening brief in support of a motion to dismiss the complaint. Plaintiff filed an answering brief on November 25, 2024, and the Company filed a reply brief on December 20, 2024. The Company does not believe the outcome of this matter will have a material effect on its financial position, results of operations, or liquidity.

On November 22, 2024, a purported stockholder filed a putative class action against the Company, the members of the board of directors and certain executive officers of the Company, as well as a derivative action against the members of the board of directors and certain executive officers of the Company, in the Court of Chancery of the State of Delaware in an action captioned Taylor v. Haviland, et al., C.A. No. 2024-1203-JTL (the “Taylor Action”). Plaintiff in the Taylor Action claims that the record date for the Company’s 2024 annual meeting of stockholders, which was the close of business on Friday, April 12, 2024, did not comply with the 60-day maximum under Section 213(a) of the DGCL, because it was 61 days before the date of the 2024 annual meeting. Plaintiff brings direct claims for violation of Section 213(a) of the DGCL and breach of fiduciary duty, and derivative claims for breach of fiduciary duty and unjust enrichment, and seeks a declaration that certain actions taken in connection with the Company’s annual meeting of stockholders are void, as well as attorneys’ fees and costs.

On December 2, 2024, the Company filed a petition pursuant to Section 205 of the DGCL seeking the validation of the certain actions taken in connection with the Company’s 2024 annual meeting of stockholders, retroactive to the date of the 2024 annual meeting, in the Court of Chancery of the State of Delaware in an action captioned In re Blueprint Medicines Corporation, C.A. No. 2024-1234-JTL (the “Section 205 Action”). On December 4, 2024, plaintiff in the Taylor Action agreed to hold the defendants’ answer in abeyance pending resolution of the Section 205 Action. Following the Company’s brief in support of its petition in the Section 205 Action on December 20, 2024, and the lack of any objection, the Court granted the petition on January 23, 2025, such that the stockholder proposals that were presented to and approved by the Company’s stockholders at the 2024 annual meeting, and all actions taken in reliance on the stockholder votes at the annual meeting, were hereby declared valid and effective as of the date of the 2024 annual meeting.

Indemnification Agreements

In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners, and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors and senior management that will require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers of the Company. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company

is not aware of any material claims under indemnification arrangements, and it has not accrued any liabilities related to such obligations in its consolidated financial statements as of December 31, 2024 or 2023.

Stock-Based Compensation - Stock Plans (Details) - shares		1 Months Ended	12 Months Ended
	Jan. 01, 2024	Jun. 30, 2022	Dec. 31, 2024	Jun. 12, 2024	Mar. 31, 2020	Apr. 08, 2015
2015 Stock Option and Incentive Plan
Stock-based compensation
Initial shares of common stock authorized for issuance of stock awards (in shares)						1,460,084
Increase in number of shares available for grant (as a percent)			4.00%
Increase in number of shares available for grant (in shares)	2,445,889
Shares underlying awards outstanding (in shares)			7,832,847
2020 Inducement Plan
Stock-based compensation
Initial shares of common stock authorized for issuance of stock awards (in shares)					1,000,000
Increase in number of shares available for grant (in shares)		1,500,000
Shares underlying awards outstanding (in shares)			724,176
2024 Stock Incentive Plan
Stock-based compensation
Initial shares of common stock authorized for issuance of stock awards (in shares)				9,200,000
Number of shares available for grant (in shares)			9,116,614
Granted, options and equity instruments other than options (in shares)			247,987

Employee Benefit Plans - 401(k) Savings Plan (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
401(k) Savings Plan
401(k) Plan expenses	$ 4.7	$ 4.6	$ 3.7

Net Loss Per Share (Details) - shares shares in Thousands	12 Months Ended
Net Loss Per Share (Details) - shares shares in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	8,832	8,857	8,182
Employee Stock Option
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	6,370	6,658	6,233
Restricted Stock Units
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	2,291	2,107	1,894
Performance-based restricted stock units
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	143	53
Employee Stock
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	28	39	55

Financing Arrangements - Estimated Total Gross Payments (Details) - Secured Debt - Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility $ in Thousands	Dec. 31, 2024 USD ($)
Financing Arrangements
2025	$ 43,917
2026	43,917
2027	43,917
2028	421,897
Total	$ 553,648

Line items represent financial concepts included in a table. These concepts are used to disclose reportable information associated with domain members defined in one or many axes to the table.

Cash and Cash Equivalents

Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Policies)

12 Months Ended

Dec. 31, 2024

Policy Text Blocks

Basis of Presentation

Use of Estimates

Revenue Recognition

Revenue Recognition

Product revenue

Collaboration, license and other revenue

For a complete discussion of accounting for collaboration, license and other revenues, see Note 11, Collaboration, License and Other Agreements.

Accounts Receivable, Net

Accounts Receivable, net

Inventory

Inventory

Fair Value Measurements

Fair Value Measurements

●

Level 1 — Fair values are determined utilizing quoted prices (unadjusted) in active markets for identical assets or liabilities that the Company has the ability to access;

●

There have been no changes to the valuation methods during the years ended December 31, 2024, 2023 and 2022.

Cash and cash equivalents

Marketable Securities

Marketable securities

Comprehensive Income (Loss)

Comprehensive Income (Loss)

Research and Development Expenses

Research and Development Expenses

Selling, General and Administrative Expenses

Selling, General and Administrative Expenses

Property and Equipment, Net

Property and Equipment, Net

Impairment of Long Lived Assets

Impairment of Long-Lived Assets

Leases

Leases

Stock Based Compensation Expense

Stock-Based Compensation Expense

●

risk-free interest rate, which is based on the U.S. Treasury yield curve in effect at the time of grant commensurate with the expected term assumption;

●

dividend yield, which is zero based on the fact that the Company never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future.

Equity Investment

Liabilities related to the sale of future royalties and revenues

Modification of debt instruments

Income Taxes

Income Taxes

Foreign Currency Translation

Foreign currency translation

Concentrations of Credit Risk and of Significant Suppliers

credit risk primarily consist of cash and cash equivalents, marketable securities, accounts receivable and unbilled account receivables.

Segment and Geographic Information

Recent Accounting Pronouncements

New Accounting Pronouncements

Reclassification

Certain items in the prior year’s consolidated financial statements have been reclassified to conform to the current presentation.

Segment Information

12 Months Ended

Dec. 31, 2024

Segment Information

18. Segment Information

The Company operates as one operating segment, focused on discovering, developing and delivering therapies for allergy/immunology and oncology/hematology. The determination of a single business segment is consistent with the consolidated financial information regularly provided to the Company’s chief operating decision maker (“CODM”). The Company’s CEO, as the chief operating decision maker (CODM), uses consolidated, single-segment financial information for purposes of evaluating performance, making operating decisions, allocating resources, and planning and forecasting for future periods.

The CODM assesses performance and decides how to allocate resources based on consolidated net income (loss). This measure is used to monitor budget versus actual results to evaluate the performance of the segment.

The CODM reviews cash, cash equivalents and marketable securities as a measure of segment assets. As of December 31, 2024 and 2023, the Company’s cash, cash equivalents and marketable securities were $863.9 million and $767.2 million, respectively.

The following tables illustrates information about segment revenue, significant segment expenses and segment operating loss for the years ended December 31, 2024, 2023 and 2022 (in thousands):


	Year Ended December 31,
	2024		2023		2022
Revenue	$	508,824	$	249,380	$	204,036
Less¹:
Cost of sales		20,163		8,540		17,813
Research and development expense²:
Compensation and related expenses		114,861		110,740		96,625
Early drug discovery and platform		76,970		70,383		75,542
Clinical and manufacturing related activities		38,889		138,801		198,317
Facilities and IT		35,589		33,783		28,917
Consulting and professional services		24,182		29,189		33,033
Other research and development		3,487		3,290		4,683
Total research and development expense²		293,978		386,186		437,117
Selling, general and administrative expense³:
Compensation and related expenses		127,286		120,756		84,324
Commercial and related expenses		96,604		53,579		33,334
Consulting and professional services		49,575		41,876		36,264
Facilities and IT		12,214		13,126		11,431
Other selling, general and administrative		12,137		14,666		13,352
Total selling, general and administrative expense³		297,816		244,003		178,705
Collaboration loss sharing		—		4,256		8,948
Stock-based compensation		108,911		92,672		98,971
Other segment items⁴		144,955		(20,707)		(19,999)
Net loss	$	(67,089)	$	(506,984)	$	(557,517)

The significant expense categories and amounts align with the segment-level information that is regularly provided to the chief operating decision maker.

Research and development expense for the years ended December 31, 2024, 2023 and 2022 exclude $47.5 million, $41.5 million, and $40.3 million of stock-based compensation expense, respectively.

Selling, general and administrative expense for the years ended December 31, 2024, 2023 and 2022 exclude $61.5 million, $51.1 million, and $58.7 million of stock-based compensation expense, respectively.

Other segment items include interest expense, net, other income (expense), net, debt extinguishment gain, and income tax expense.

The Company operates in the U.S. and Europe. All material long-lived assets of the Company reside in the U.S. For geographic information about the Company’s product revenues, see Note 6, Product Revenue and Related Reserves.

The entire disclosure for reporting segments including data and tables. Reportable segments include those that meet any of the following quantitative thresholds a) it's reported revenue, including sales to external customers and intersegment sales or transfers is 10 percent or more of the combined revenue, internal and external, of all operating segments b) the absolute amount of its reported profit or loss is 10 percent or more of the greater, in absolute amount of 1) the combined reported profit of all operating segments that did not report a loss or 2) the combined reported loss of all operating segments that did report a loss c) its assets are 10 percent or more of the combined assets of all operating segments.

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Nature of Business

12 Months Ended

Dec. 31, 2024

Nature of Business

1. Nature of Business

Blueprint Medicines Corporation (the Company), a Delaware corporation incorporated on October 14, 2008, is a global fully-integrated biopharmaceutical company that invents life-changing medicines in two core focus areas: allergy/inflammation and oncology/hematology. The Company’s approach targets the root causes of disease, using deep scientific knowledge in the Company’s core focus areas and drug discovery expertise across multiple therapeutic modalities.

The Company has a track record of success with two approved medicines, including AYVAKIT®/AYVAKYT® (avapritinib), which the Company is bringing to patients living with systemic mastocytosis (SM) and PDGFRA Exon 18 mutant GIST in the U.S. and Europe. Leveraging the Company’s established research, development, and commercial capability and infrastructure, the Company now aims to significantly scale its impact by advancing a broad pipeline of programs ranging from early science to advanced clinical trials in mast cell diseases including SM and chronic urticaria, breast cancer and other solid tumors.

The Company is subject to a number of risks similar to those of other commercial-stage companies, including but not limited to: successful commercialization of its current and future drugs, either by itself or through collaboration with third parties; establishing safety and efficacy in clinical trials and obtaining regulatory approvals for its drug candidates; competition from other companies; compliance with comprehensive and ongoing regulatory requirements and legislative changes; and the need to obtain adequate additional financing to fund the development of its drug candidates. If the Company is unable to raise capital when needed or on attractive terms, it may be forced to delay, reduce, eliminate or repartner certain of its research and development programs or future commercialization efforts.

As of December 31, 2024, the Company had cash, cash equivalents and marketable securities of $863.9 million. Based on the Company’s current operating plans, the Company anticipates that its existing cash, cash equivalents and marketable securities will be sufficient to enable it to fund its current operations for at least the next twelve months from the issuance of the financial statements.

Employee Benefit Plans - Switzerland Defined Benefit Plan - Expected Benefit Payments (Details) $ in Thousands	Dec. 31, 2024 USD ($)
Benefits expected to be paid
2025	$ 466
2026	599
2027	546
2028	582
2029	623
2030 to 2034	$ 4,028

Income Taxes - Unrecognized Tax Benefits - Roll Forward (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Unrecognized tax benefits
Beginning balance	$ 211	$ 0
Gross Increases - tax positions in prior periods		211
Settlements	(211)
Ending balance	$ 0	$ 211

Marketable Securities

12 Months Ended

Dec. 31, 2024

Marketable Securities

4. Marketable Securities

Marketable securities consisted of the following at December 31, 2024 and 2023 (in thousands):


	Amortized		Unrealized		Unrealized		Fair
December 31, 2024	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	129,897	$	118	$	(230)	$	129,785
U.S. treasury obligations		631,514		1,025		(401)		632,138
Total	$	761,411	$	1,143	$	(631)	$	761,923


	Amortized		Unrealized		Unrealized		Fair
December 31, 2023	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	170,194	$	27	$	(418)	$	169,803
U.S. treasury obligations		525,880		588		(386)		526,082
Total	$	696,074	$	615	$	(804)	$	695,885

The following table summarizes the amortized cost basis and estimated fair value of the Company’s available-for-sale securities by contractual maturity as of December 31, 2024 and 2023 (in thousands):


	As of December 31,
	2024				2023
	Amortized		Fair		Amortized		Fair
	Cost		value		Cost		value
Within one year	$	512,515	$	513,473	$	639,881	$	639,355
After one through five years		248,896		248,450		56,193		56,530
Total	$	761,411	$	761,923	$	696,074	$	695,885

As of December 31, 2024 and 2023, the Company held 32 and 62 debt securities, respectively, that were in an unrealized loss position. The following table summarizes the estimated fair value and the aggregate unrealized loss of the Company’s available-for-sale securities that are in loss position as of December 31, 2024 and 2023 by the length of time the security has been in loss position (in thousands):


	As of December 31,
	2024				2023
	Fair		Unrealized		Fair		Unrealized
	value		losses		value		losses
Debt securities in unrealized loss position for 12 months or less	$	205,910	$	(631)	$	267,917	$	(550)
Debt securities in unrealized loss position for more than 12 months		—		—		64,659		(254)
Total debt securities in unrealized loss position	$	205,910	$	(631)	$	332,576	$	(804)

The Company has the intent and ability to hold its debt securities until recovery of amortized cost basis. As a result, the Company did not recognize any differences between the fair value and amortized cost basis as a loss in its consolidated statements of operations and comprehensive loss for the years ended December 31, 2024, 2023 and 2022. The Company did not record any credit-related impairments for its available-for-sale securities for the years ended December 31, 2024, 2023 and 2022.

The following table summarizes the proceeds from maturities of debt securities during the years ended December 31, 2024, 2023 and 2022 (in thousands):


	Year ended December 31,
	2024		2023		2022
Proceeds from maturities of debt securities	$	923,560	$	1,056,465	$	349,373

The Company did not realize any gains or losses from maturities of debt securities for the years ended December 31, 2024, 2023 and 2022.

Leases (Tables)

12 Months Ended

Dec. 31, 2024

Summary of lease expenses and cash flow and weighted average information


	Year Ended December 31,
Operating leases:	2024		2023		2022
Lease cost	$	23,238	$	23,190	$	21,830
Sublease income		-		-		(2,132)
Net lease cost	$	23,238	$	23,190	$	19,698


	Year Ended December 31,
	2024		2023		2022
Cash paid for amounts included in the measurement of lease liabilities	$	18,284	$	17,741	$	15,891
Lease liabilities arising from obtaining right-of-use assets:
Operating leases	$	556	$	915	$	72

Schedule of future minimum lease payments



2025	$	18,757
2026		19,318
2027		19,876
2028		20,451
2029		19,268
Thereafter		84
Total future minimum lease payments		97,754
Less imputed interest		(15,618)
Total	$	82,136

Financing Arrangements - Roll Forward (Details) - USD ($) $ in Thousands	12 Months Ended		30 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2024
Sale of Future Royalties and Revenues \| Sixth Street Partners, Future Revenue Purchase Agreement
Long-term Debt
Carrying value, beginning balance	$ 266,670	$ 254,328
Interest expense recognized	27,134	28,065
Payments	(39,368)	(15,723)
Carrying value, ending balance	254,436	266,670	$ 254,436
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities
Long-term Debt
Carrying value, beginning balance	238,813	139,083
Net proceeds received	146,973	97,933	382,692
Interest expense recognized	41,668	23,540
Payments	(40,484)	(21,743)
Carrying value, ending balance	$ 386,970	$ 238,813	$ 386,970

Amount, after deduction of unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

Stock-based Compensation - Stock Options - Activity (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Shares
Outstanding at beginning of period (in shares)	6,658,444
Granted (in shares)	1,075,799
Exercised (in shares)	(1,159,269)
Cancelled (in shares)	(205,250)
Outstanding at end of period (in shares)	6,369,724	6,658,444
Weighted-Average Exercise Price
Outstanding at beginning of period (in dollars per share)	$ 66.48
Granted (in dollars per share)	95.77
Exercised (in dollars per share)	61.96
Cancelled (in dollars per share)	75.42
Outstanding at end of period (in dollars per share)	$ 72.09	$ 66.48
Additional disclosures
Shares - Exercisable (in shares)	4,514,741
Weighted-Average Exercise Price - Exercisable (in dollars per share)	$ 71.51
Remaining Contractual Life - Outstanding	6 years 1 month 20 days	6 years 4 months 20 days
Remaining Contractual Life - Exercisable	5 years 2 months 12 days
Aggregate Intrinsic Value - Outstanding	$ 115,874	$ 178,581
Aggregate Intrinsic Value - Exercisable	$ 82,016
2024 Stock Incentive Plan
Shares
Granted (in shares)	55,119

Stock-based Compensation - Stock-based Compensation Expense - General Information (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Total stock based compensation expense
Stock-based compensation expense	$ 108,911	$ 92,672	$ 98,971

Financing Arrangements - Debt Extinguishment (Details) - USD ($) $ in Thousands	1 Months Ended	12 Months Ended
	Feb. 28, 2025	Dec. 31, 2024	Feb. 22, 2024
Gain (Loss) on Extinguishment of Debt
Debt extinguishment gain		$ 173,676
Sale of Future Royalties and Revenues \| Royalty Pharma, Royalty Purchase Agreement
Gain (Loss) on Extinguishment of Debt
Debt extinguishment gain		173,700
Long-term Debt
Long-term debt		0	$ 0
Debt reacquisition liability, current		$ 700
Sale of Future Royalties and Revenues \| Royalty Pharma, Royalty Purchase Agreement \| Subsequent Event
Long-term Debt
Settlement paid	$ 700

Amount, after deduction of unamortized premium (discount) and debt issuance cost, of long-term debt. Excludes lease obligation.

Segment Information - Assessing Segment Performance (Details) - USD ($) $ in Millions	Dec. 31, 2024	Dec. 31, 2023
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities	$ 863.9	$ 767.2

Pay vs Performance Disclosure - USD ($) $ in Thousands	12 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Pay vs Performance Disclosure
Net Income (Loss)	$ (67,089)	$ (506,984)	$ (557,517)

The portion of profit or loss for the period, net of income taxes, which is attributable to the parent.

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Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Impairment of Long-Lived Assets (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Impairment of Long Lived Assets
Long-lived asset impairment	$ 0	$ 0	$ 0

Employee Benefit Plans

12 Months Ended

Dec. 31, 2024

Employee Benefit Plans

17. Employee Benefit Plans

The Company sponsors various retirement and pension plans. The estimates of liabilities and expenses for these plans incorporate a number of assumptions, including expected rates of return on plan assets and interest rates used to discount future benefits.

401(k) Savings Plan

The Company maintains a 401(k) plan for employees (the 401(k) Plan). The 401(k) Plan is intended to qualify under Section 401(k) of the Code, so that contributions to the 401(k) Plan by the Company, and the investment earnings on contributions, are not taxable to the employees until withdrawn from the 401(k) Plan, and so that contributions by the Company, if any, will be deductible by the Company when made. Under the 401(k) Plan, employees may elect to reduce their current compensation by up to the statutorily prescribed annual limit and to have the amount of such reduction

contributed to the 401(k) Plan. The 401(k) Plan permits the Company to make contributions up to the limits allowed by law on behalf of all eligible employees. The expense related to the 401(k) Plan primarily consists of the Company’s matching contributions. The expenses related to the 401(k) Plan for the years ended December 31, 2024, 2023 and 2022 were $4.7 million, $4.6 million and $3.7 million, respectively.

Switzerland Defined Benefit Plan

The Company maintains a pension plan covering employees of its Swiss subsidiary, Blueprint Medicines (Switzerland) GmbH (the “Swiss Plan”). The Swiss Plan is a government-mandated retirement fund that provides employees with a minimum benefit. Employer and employee contributions are made to the Swiss Plan based on various percentages of salary and wages that vary according to employee age and other factors. As is customary with Swiss pension plans, the assets of the Swiss Plan are invested in a collective fund with multiple employers. The Company has no investment authority over the assets of the Swiss Plan, which are held and invested by a Swiss insurance company. The investment strategy of the Swiss Plan is managed by an independent asset manager with the objective of achieving a consistent long-term return which will provide sufficient funding for future pension obligations while limiting risk.

The following table summarizes the plan balances as of December 31, 2024 and 2023 (in thousands):


	December 31,		December 31,
	2024		2023
Fair value of plan assets	$	9,241	$	8,416
Projected benefit obligation		(16,065)		(14,632)
Unfunded status recorded as other long-term liabilities		(6,824)		(6,216)
Accumulated benefit obligation	$	12,071	$	10,984

The Company’s net periodic benefit cost for the years ended December 31, 2024, 2023 and 2022 was $1.4 million, $1.0 million and $1.2 million, respectively. The contributions to the Swiss Plan for the years ended December 31, 2024, 2023 and 2022 were $1.1 million, $1.0 million, and $0.8 million.

The following table summarizes the benefits expected to be paid by the Company in each of the next five years and in aggregate for the five years thereafter as of December 31, 2024 (in thousands):


	As of December 31
	2024
2025	$	466
2026		599
2027		546
2028		582
2029		623
2030 to 2034		4,028

The entire disclosure for retirement benefits.

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-Publisher FASB
-URI https://asc.fasb.org/1943274/2147480266/715-60-50-3

Income Taxes - Reconciliation (Details)	12 Months Ended
Income Taxes - Reconciliation (Details)	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
A reconciliation of the U.S. statutory income tax rate to the Company's effective tax rate
Federal income tax (benefit) at statutory rate	21.00%	21.00%	21.00%
Permanent differences	0.82%	(0.42%)	(0.38%)
U.S. Tax on foreign earnings			2.52%
Federal research and development credits	14.43%	2.50%	1.39%
Federal orphan drug credits	18.41%	1.63%	(0.42%)
State income tax, net of federal benefit	7.91%	3.41%	2.92%
Foreign rate differential	(13.47%)	(0.41%)	(0.13%)
Deferred rate change	(9.21%)	2.53%	0.52%
Foreign tax credit			0.03%
Stock Based Compensation	(5.33%)	(0.76%)	(1.03%)
Other	(0.27%)	(0.02%)	0.39%
Change in valuation allowance	(36.17%)	(29.65%)	(27.76%)
Effective income tax rate	(1.88%)	(0.19%)	(0.95%)

Collaboration, License and Other Agreements - Equity Investment (Details) - USD ($) $ in Thousands		1 Months Ended		12 Months Ended
	Aug. 01, 2022	Jul. 31, 2023	Aug. 31, 2022	Dec. 31, 2024	Dec. 31, 2023
Equity Investment
Equity investment				$ 28,699	$ 27,789
Collaborative Arrangement \| IDRx, Inc. \| Director
Equity Investment
Equity investment, license agreement, shares received (in shares)	4,509,105	192,282	4,509,105
Equity investment			$ 27,800	28,700
Equity investment, transaction costs			$ 300
Equity investment, change in fair value				$ 900

Stock-based Compensation

12 Months Ended

Dec. 31, 2024

Stock-based Compensation

13. Stock-based Compensation

Stock Plans

In 2015, the Company’s board of directors and stockholders approved the 2015 Stock Option and Incentive Plan (the 2015 Plan), which replaced the Company’s 2011 Stock Option and Grant Plan, as amended (the 2011 Plan). The 2015 Plan includes incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock, restricted stock units, performance-based restricted stock units, unrestricted stock, performance-based awards and cash-based awards. The Company initially reserved a total of 1,460,084 shares of common stock for the issuance of awards under the 2015 Plan. The 2015 Plan provided that the number of shares reserved and available for issuance under the 2015 Plan would be cumulatively increased on January 1 of each calendar year by 4% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar year beginning January 1, 2024, the number of shares reserved for issuance under the 2015 Plan was increased by 2,445,889 shares.

In March 2020, the Company’s board of directors adopted the 2020 Inducement Plan (the Inducement Plan), pursuant to which the Company may grant, subject to the terms of the Inducement Plan and Nasdaq rules, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, and other stock-based awards. The

Company initially reserved a total of 1,000,000 shares of common stock for the issuance of awards under the Inducement Plan and in June 2022, the Company’s board of directors approved the reservation of an additional 1,500,000 shares of common stock for the issuance of awards under the Inducement Plan.

At the Company’s annual meeting of stockholders held on June 12, 2024, the Company’s stockholders approved the 2024 Stock Incentive Plan (the 2024 Plan), which replaced the Company’s 2015 Plan and the Inducement Plan. As of December 31, 2024, there were 7,832,847 and 724,176 shares underlying awards outstanding under the 2015 Plan and the Inducement Plan, respectively. No further shares will be granted under the 2015 Plan and the Inducement Plan after the effective date of the 2024 Plan. The 2024 Plan provides for the granting of incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock, restricted stock units, performance-based restricted stock units, unrestricted stock and cash-based awards. The 2024 Plan provides for the issuance of up to 9,200,000 shares. Any shares of common stock underlying any awards that are forfeited, canceled, held back upon exercise or settlement of an award to satisfy the exercise price or tax withholding, repurchased, expire or are otherwise terminated by the Company under the 2024 Plan and the 2015 Plan will be added back to the shares of common stock available for issuance under the 2024 Plan. As of December 31, 2024, there were 9,116,614 shares available for future grant under the 2024 Plan.

On November 22, 2024, the Company learned that the record date for its 2024 annual meeting was 61 days prior to the meeting, exceeding the 60-day limit under Section 213(a) of the Delaware General Corporation Law (DGCL). On December 2, 2024, the Company petitioned the Delaware Court of Chancery under Section 205 of the DGCL to validate the actions taken at the 2024 annual meeting, including the adoption of the 2024 Plan. The Court granted the Company’s petition on January 23, 2025, ratifying these actions as of the meeting date. During the year ended December 31, 2024, the Company granted 247,987 shares under the 2024 Plan. For more information, see “Legal Proceedings” in Note 19, Commitments and Contingencies.

2015 Employee Stock Purchase Plan

In 2015, the Company’s board of directors and stockholders approved the Employee Stock Purchase Plan (the 2015 ESPP), which became effective upon the closing of the Company’s initial public offering in May 2015. The Company initially reserved a total of 243,347 shares of common stock for issuance under the 2015 ESPP. The 2015 ESPP provides that the number of shares reserved and available for issuance under the 2015 ESPP will be cumulatively increased on January 1 of each calendar year by 1% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar years beginning January 1, 2024 and 2025, the number of shares reserved for issuance under the 2015 ESPP was increased by 611,472 and 637,122 shares, respectively. As of December 31, 2024, there were 4,196,701 shares available for issuance under the 2015 ESPP. The Company issued 65,813, 96,290, and 80,717 shares under the ESPP during the years ended December 31, 2024, 2023, and 2022 respectively.

Stock-based Compensation Expense


	Year Ended December 31,
	2024		2023			2022
Stock options	$	42,031	$	40,560	$	52,931
Restricted stock units		61,722		50,095		45,285
Performance-based restricted stock units		4,445		867		—
Employee stock purchase plan		1,722		1,734		1,418
Subtotal		109,920		93,256		99,634
Capitalized stock-based compensation costs		(1,009)		(584)		(663)
Stock-based compensation expense included in total cost and operating expenses	$	108,911	$	92,672	$	98,971


	Year Ended December 31,
	2024		2023		2022
Research and development	$	47,455	$	41,534	$	40,302
Selling, general and administrative		61,456		51,138		58,669
Total stock-based compensation expense included in operating expenses	$	108,911	$	92,672	$	98,971

At December 31, 2024, there was $207.6 million of total unrecognized compensation cost related to non-vested stock awards, which is expected to be recognized over a weighted-average period of 2.4 years.

Stock Options

Stock options granted by the Company generally vest ratably over four years, with a one-year cliff for new employee awards and are exercisable from the date of grant for a period of ten years. The fair value of each option issued to employees was estimated at the date of grant using the Black-Scholes option pricing model with the following weighted-average assumptions:


	Year Ended December 31,
	2024		2023		2022
Risk-free interest rate	4.26	%	4.13	%	2.27	%
Expected dividend yield	—		—		—
Expected term (years)	6.0		6.0		6.0
Expected stock price volatility	55.54	%	56.01	%	55.15	%

The following table summarizes the stock option activity for the year ended December 31, 2024:


		Weighted-		Remaining	Aggregate
		Average		Contractual	Intrinsic
		Exercise		Life	Value(1)
	Shares	Price		(in Years)	(in thousands)
Outstanding at December 31, 2023	6,658,444	$	66.48	6.39	$	178,581
Granted ²	1,075,799		95.77
Exercised	(1,159,269)		61.96
Canceled	(205,250)		75.42
Outstanding at December 31, 2024	6,369,724	$	72.09	6.14	$	115,874
Exercisable at December 31, 2024	4,514,741	$	71.51	5.20	$	82,016

(1)	Intrinsic value represents the amount by which the fair market value as of December 31, 2024 of the underlying common stock exceeds the exercise price of the option.

(2)	Out of stock options granted in 2024, 55,119 stock options were granted under the 2024 Plan.

The weighted average grant date fair value of options granted in the years ended December 31, 2024, 2023 and 2022 was $54.15, $25.81 and $32.30, respectively. The total intrinsic value of options exercised in the years ended December 31, 2024, 2023, and 2022 was $45.1 million, $11.6 million, and $11.8 million, respectively.

At December 31, 2024, the total unrecognized compensation expense related to unvested stock option awards was $71.4 million, which is expected to be recognized over a weighted-average period of approximately 2.4 years.

Restricted stock units

Restricted stock units granted by the Company generally vest ratably over four years. The following table summarizes the restricted stock units activity for the year ended December 31, 2024:


		Weighted-Average
		Grant Date
	Shares	Fair Value
Unvested shares at December 31, 2023	2,107,026	$	60.33
Granted ¹	1,130,316		95.24
Vested	(795,219)		64.77
Forfeited	(151,192)		70.34
Unvested shares at December 31, 2024	2,290,931	$	75.36

(1)

Out of restricted stock units granted in 2024, 192,868 restricted stock units were granted under the 2024 Plan.

The total fair value of restricted stock units vested during the years ended December 31, 2024, 2023 and 2022 was $75.8 million, $31.6 million and $29.4 million, respectively. As of December 31, 2024, the total unrecognized compensation expense related to unvested restricted stock units was $126.2 million, which is expected to be recognized over a weighted-average period of approximately 2.4 years.

Performance-based restricted stock units

In 2023, the Company began granting performance-based restricted stock units (PSUs) that will settle in stock. PSUs awarded to employees have a three-year performance period and vest on the third anniversary of the grant date. The vesting of these awards is subject to the respective employee’s continued employment. The number of PSUs granted represents the target number of units that are eligible to be earned based on the achievement of cumulative three-year performance measures established at the beginning of the performance period, which ends on December 31 of the third year of the performance period. Participants may ultimately earn between zero and 200.0% of the target number of PSUs granted based on the degree of achievement of the performance metric which is measured on a three-year cumulative relative total shareholder return metric. Accordingly, additional PSUs may be issued or currently issued PSUs may be cancelled upon final determination of the number of units earned.

The following table summarizes the PSU activity for year ended December 31, 2024:


		Weighted-Average
		Grant Date
	Shares	Fair Value
Unvested shares at December 31, 2023	52,500	$	59.32
Granted	90,000		135.84
Vested	—		—
Forfeited	—		—
Unvested shares at December 31, 2024	142,500	$	107.65

As of December 31, 2024, the total unrecognized compensation expense related to unvested PSUs was $10.0 million, which is expected to be recognized over a weighted-average period of approximately 1.8 years.

The Company values PSUs on the grant date using a lattice model with a Monte Carlo simulation. This valuation methodology utilizes several key assumptions, including defined consecutive trading day average closing stock price on the grant date, valuation date stock price, expected volatilities using historical volatilities, correlation coefficients based on the volatility data, risk-free rates of return and expected dividend yield. The probability of actual shares expected to be earned is considered in the grant date valuation and the expense is not adjusted to reflect the actual units earned.

Income Taxes - Equity Investment (Details) - USD ($) $ in Thousands		1 Months Ended		12 Months Ended
	Aug. 01, 2022	Jul. 31, 2023	Aug. 31, 2022	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Revenues
Revenue				$ 508,824	$ 249,380	$ 204,036
License \| Related Party
Revenues
Revenue						27,500
License \| IDRx, Inc. \| Director
Revenues
Revenue			$ 27,500	$ 0	$ 0	$ 27,500
Collaborative Arrangement \| IDRx, Inc. \| Director
Equity Investment
Equity investment, license agreement, shares received (in shares)	4,509,105	192,282	4,509,105

Restricted Cash

12 Months Ended

Dec. 31, 2024

Restricted Cash

8. Restricted Cash

The following table provides a reconciliation of cash, cash equivalents, and restricted cash reported within the Company’s consolidated balance sheets that sum to the total of the same such amounts shown in the consolidated statements of cash flows for the years ended December 31, 2024, 2023 and 2022 (in thousands):


	December 31,
	2024		2023		2022
Cash and cash equivalents	$	102,014	$	71,286	$	119,709
Restricted cash		11,625		10,238		5,195
Total cash, cash equivalents, and restricted cash shown in consolidated statements of cash flows	$	113,639	$	81,524	$	124,904

At December 31, 2024 and 2023, $11.6 million and $10.2 million, respectively, of the Company’s cash is restricted by a financial institution primarily related to funds held to satisfy the requirements of certain government agreements and the security deposits for the lease agreements for the Company’s office and laboratory spaces. For additional information, see Note 16, Leases.

Segment Information - General Information (Details) - segment	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Segment Information
Number of operating segments	1	1	1
Segment Reporting, CODM, Individual Title and Position or Group Name	srt:ChiefExecutiveOfficerMember	srt:ChiefExecutiveOfficerMember	srt:ChiefExecutiveOfficerMember

Product Revenue (Tables)

12 Months Ended

Dec. 31, 2024

Schedules of concentration of risk

The Company primarily sells AYVAKIT/AYVAKYT through specialty distributors and specialty pharmacies. The following table summarizes the customers that represent 10% or greater of gross product revenue for the years ended December 31, 2024, 2023, and 2022:


	Year ended December 31,
	2024		2023		2022
Customer 1	39	%	43	%	45	%
Customer 2	12	%	*	%	*	%
Customer 3	*	%	10	%	11	%

* Indicates the customer’s share is under 10%.

The following table summarizes the customers with amounts due that represent 10% or greater of the accounts receivable associated with the Company’s product sales as of December 31, 2024 and 2023:


	As of December 31,
	2024		2023
Customer 1	31	%	34	%
Customer 2	14	%	*	%
Customer 3	11	%	11	%
Customer 4	*	%	10	%
Customer 5	*	%	10	%

* Indicates the customer’s share is under 10%.

Product revenue, net

Summary of revenue recognized


	Year Ended December 31,
	2024		2023		2022
United States	$	421,837	$	181,971	$	97,226
Rest of World		57,113		22,236		13,767
Total product revenue, net	$	478,950	$	204,207	$	110,993

Schedule of product revenue allowance and reserve categories


	Year Ended December 31,
	2024		2023
Beginning balance at January 1	$	19,274	$	9,788
Provision related to sales in the current period		80,847		37,281
Adjustment related to prior periods sales		(566)		(700)
Credits and payments made		(65,776)		(27,095)
Ending balance at December 31	$	33,779	$	19,274


	As of December 31,
	2024		2023
Reduction of accounts receivable, net	$	2,963	$	1,809
Component of accrued expenses		30,816		17,465
Total revenue-related reserves	$	33,779	$	19,274

Stock-based Compensation - Unvested Awards - Activity (Details)	12 Months Ended
	Dec. 31, 2024 $ / shares shares
Restricted Stock Units
Shares
Unvested at beginning of period (in shares)	2,107,026
Granted (in shares)	1,130,316
Vested (in shares)	(795,219)
Forfeited (in shares)	(151,192)
Unvested at end of period (in shares)	2,290,931
Weighted-Average Grant Date Fair Value
Unvested at beginning or period (in dollars per share) \| $ / shares	$ 60.33
Granted (in dollars per share) \| $ / shares	95.24
Vested (in dollars per share) \| $ / shares	64.77
Forfeited (in dollars per share) \| $ / shares	70.34
Unvested at end of period (in dollars per share) \| $ / shares	$ 75.36
Restricted Stock Units \| 2024 Stock Incentive Plan
Shares
Granted (in shares)	192,868
Performance-based restricted stock units
Shares
Unvested at beginning of period (in shares)	52,500
Granted (in shares)	90,000
Unvested at end of period (in shares)	142,500
Weighted-Average Grant Date Fair Value
Unvested at beginning or period (in dollars per share) \| $ / shares	$ 59.32
Granted (in dollars per share) \| $ / shares	135.84
Unvested at end of period (in dollars per share) \| $ / shares	$ 107.65

Stock-based Compensation - Awards - General Information (Details)	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Employee Stock Option
Stock-based compensation
Award expiration period	10 years	10 years	10 years
2015 Stock Option and Incentive Plan \| Employee Stock Option
Stock-based compensation
Award vesting period	4 years	4 years	4 years
Cliff vesting period for new employees	1 year	1 year	1 year
Award expiration period	10 years	10 years	10 years
2015 Stock Option and Incentive Plan \| Restricted Stock Units
Stock-based compensation
Award vesting period	4 years	4 years	4 years

Subsequent Events

12 Months Ended

Dec. 31, 2024

Subsequent Events

20. Subsequent Events

Segment Information (Tables)

12 Months Ended

Dec. 31, 2024

Schedule of segment revenue, significant segment expenses and segment operating loss


	Year Ended December 31,
	2024		2023		2022
Revenue	$	508,824	$	249,380	$	204,036
Less¹:
Cost of sales		20,163		8,540		17,813
Research and development expense²:
Compensation and related expenses		114,861		110,740		96,625
Early drug discovery and platform		76,970		70,383		75,542
Clinical and manufacturing related activities		38,889		138,801		198,317
Facilities and IT		35,589		33,783		28,917
Consulting and professional services		24,182		29,189		33,033
Other research and development		3,487		3,290		4,683
Total research and development expense²		293,978		386,186		437,117
Selling, general and administrative expense³:
Compensation and related expenses		127,286		120,756		84,324
Commercial and related expenses		96,604		53,579		33,334
Consulting and professional services		49,575		41,876		36,264
Facilities and IT		12,214		13,126		11,431
Other selling, general and administrative		12,137		14,666		13,352
Total selling, general and administrative expense³		297,816		244,003		178,705
Collaboration loss sharing		—		4,256		8,948
Stock-based compensation		108,911		92,672		98,971
Other segment items⁴		144,955		(20,707)		(19,999)
Net loss	$	(67,089)	$	(506,984)	$	(557,517)

The significant expense categories and amounts align with the segment-level information that is regularly provided to the chief operating decision maker.

Research and development expense for the years ended December 31, 2024, 2023 and 2022 exclude $47.5 million, $41.5 million, and $40.3 million of stock-based compensation expense, respectively.

Other segment items include interest expense, net, other income (expense), net, debt extinguishment gain, and income tax expense.

Leases - Components of Lease Expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Operating leases:
Lease cost	$ 23,238	$ 23,190	$ 21,830
Sublease income			(2,132)
Net lease cost	$ 23,238	$ 23,190	$ 19,698

Restricted Cash - Additional Information (Details) - USD ($) $ in Millions	Dec. 31, 2024	Dec. 31, 2023
Restricted cash
Restricted cash	$ 11.6	$ 10.2

Product Revenue

12 Months Ended

Dec. 31, 2024

Product Revenue

6. Product Revenue and Related Reserves

The following table summarizes revenue recognized from the sales of AYVAKIT/AYVAKYT for the years ended December 31, 2024, 2023 and 2022 (in thousands):


	Year Ended December 31,
	2024		2023		2022
United States	$	421,837	$	181,971	$	97,226
Rest of World		57,113		22,236		13,767
Total product revenue, net	$	478,950	$	204,207	$	110,993


	Year ended December 31,
	2024		2023		2022
Customer 1	39	%	43	%	45	%
Customer 2	12	%	*	%	*	%
Customer 3	*	%	10	%	11	%

* Indicates the customer’s share is under 10%.

The following table summarizes the customers with amounts due that represent 10% or greater of the accounts receivable associated with the Company’s product sales as of December 31, 2024 and 2023:


	As of December 31,
	2024		2023
Customer 1	31	%	34	%
Customer 2	14	%	*	%
Customer 3	11	%	11	%
Customer 4	*	%	10	%
Customer 5	*	%	10	%

* Indicates the customer’s share is under 10%.

The following table summarizes activity in the product revenue allowance and reserve for the years ended December 31, 2024 and 2023 (in thousands):


	Year Ended December 31,
	2024		2023
Beginning balance at January 1	$	19,274	$	9,788
Provision related to sales in the current period		80,847		37,281
Adjustment related to prior periods sales		(566)		(700)
Credits and payments made		(65,776)		(27,095)
Ending balance at December 31	$	33,779	$	19,274

The total reserves that are included in the Company’s consolidated balance sheets as of December 31, 2024 and 2023, are summarized as follows (in thousands):


	As of December 31,
	2024		2023
Reduction of accounts receivable, net	$	2,963	$	1,809
Component of accrued expenses		30,816		17,465
Total revenue-related reserves	$	33,779	$	19,274

Property and Equipment, Net

12 Months Ended

Dec. 31, 2024

Property and Equipment, Net

9. Property and Equipment, Net

Property and equipment and related accumulated depreciation as of December 31, 2024 and 2023 are as follows (in thousands):


	Estimated
	Useful Life	As of December 31,
	(Years)	2024		2023
Lab equipment	5	$	25,720	$	21,311
Furniture and fixtures	4		4,642		4,644
Computer equipment	3		2,062		2,028
Leasehold improvements	Term of lease		50,787		42,301
Software	3		902		657
Construction-in-progress			235		9,512
Total cost			84,348		80,453
Less: accumulated depreciation and amortization			(47,755)		(38,494)
Total		$	36,593	$	41,959

Property, plant and equipment are recorded at historical cost, net of accumulated depreciation. Depreciation expense for the years ended December 31, 2024, 2023 and 2022 was $9.7 million, $7.7 million and $6.5 million, respectively.

Leases - Cash Flow Information (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Leases
Cash paid for amounts included in the measurement of lease liabilities, operating cash flows from operating leases	$ 18,284	$ 17,741	$ 15,891
Lease liabilities arising from obtaining right-of-use assets, operating leases	$ 556	$ 915	$ 72

Stock-based Compensation - Performance-based Restricted Stock Units (Details) - Performance-based restricted stock units	12 Months Ended
	Dec. 31, 2024
Stock-based compensation
Award service period	3 years
Award vesting period	3 years
Performance metric, cumulative relative total shareholder return, period	3 years
Minimum
Stock-based compensation
Award grants, percentage of target (as a percent)	0.00%
Maximum
Stock-based compensation
Award grants, percentage of target (as a percent)	200.00%

Insider Trading Arrangements - shares

3 Months Ended

Dec. 31, 2024

Sep. 30, 2024

Trading Arrangements, by Individual

Rule 10b5-1 Arrangement Adopted

false

Non-Rule 10b5-1 Arrangement Adopted

false

Rule 10b5-1 Arrangement Terminated

false

Non-Rule 10b5-1 Arrangement Terminated

false

Christina Rossi

Trading Arrangements, by Individual

Material Terms of Trading Arrangement

Name and Title

Action Taken

Type of Trading Arrangement

Nature of Trading Arrangement

Duration of Trading Arrangement

Aggregate Number of Securities

Christina Rossi

(Officer)

Adoption

August 27, 2024

Trading plan intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c)

Sale of the Company’s common stock pursuant to the terms of the trading plan

August 27, 2024 – November 28, 2025

27,291

Name

Christina Rossi

Title

Officer

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

August 27, 2024

Expiration Date

November 28, 2025

Aggregate Available

27,291

Marketable Securities - Unrealized Loss Positions - Tabular Disclosure (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Unrealized loss position, aggregate fair value
Unrealized loss position for less than 12 months, aggregate fair value	$ 205,910	$ 267,917
Unrealized loss position for more than 12 months, aggregate fair value		64,659
Unrealized loss position, aggregate fair value	205,910	332,576
Unrealized loss position
Unrealized loss position for less than 12 months	(631)	(550)
Unrealized loss position for more than 12 months		(254)
Total debt securities in unrealized loss position	$ (631)	$ (804)

Income Taxes - Valuation Allowance (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Valuation allowance
Valuation allowance	$ 798,096	$ 774,766
Change in valuation allowance	$ 23,300	$ 149,700

Property and Equipment, Net (Tables)

12 Months Ended

Dec. 31, 2024

Schedule of property and equipment

Property and equipment and related accumulated depreciation as of December 31, 2024 and 2023 are as follows (in thousands):


	Estimated
	Useful Life	As of December 31,
	(Years)	2024		2023
Lab equipment	5	$	25,720	$	21,311
Furniture and fixtures	4		4,642		4,644
Computer equipment	3		2,062		2,028
Leasehold improvements	Term of lease		50,787		42,301
Software	3		902		657
Construction-in-progress			235		9,512
Total cost			84,348		80,453
Less: accumulated depreciation and amortization			(47,755)		(38,494)
Total		$	36,593	$	41,959

Leases - Future Minimum Lease Payments (Details) $ in Thousands	Dec. 31, 2024 USD ($)
Future Minimum Lease Payments
2025	$ 18,757
2026	19,318
2027	19,876
2028	20,451
2029	19,268
Thereafter	84
Total future minimum lease payments	$ 97,754

Marketable Securities - Proceeds from Maturities (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Proceeds from the maturities of debt securities
Proceeds from the maturities of debt securities	$ 923,560	$ 1,056,465	$ 349,373

Collaboration, License and Other Agreements - General Information (Details) $ in Thousands			1 Months Ended				10 Months Ended	12 Months Ended			51 Months Ended	54 Months Ended
	Oct. 15, 2019 USD ($)	Jun. 01, 2018 USD ($)	Feb. 28, 2022 USD ($)	Nov. 30, 2021 USD ($)	Jul. 31, 2020 USD ($)	Jun. 30, 2018 item	Dec. 31, 2024 USD ($) item	Dec. 31, 2024 USD ($) item	Dec. 31, 2023 USD ($)	Dec. 31, 2022 USD ($)	Dec. 31, 2023 USD ($)	Dec. 31, 2024 USD ($) item	Feb. 22, 2024 USD ($)
Collaboration, License and Other Agreements
Prepaid expenses and other current assets							$ 35,971	$ 35,971	$ 33,351		$ 33,351	$ 35,971
Selling, general, and administrative expenses								359,272	295,141	$ 237,374
Research and development expense								341,433	427,720	477,419
Collaborative Arrangement \| IDRx, Inc. \| Director
Collaboration, License and Other Agreements
Collaborative arrangement, milestones, potential contingent payments							217,500	$ 217,500				217,500
Collaborative arrangement, termination, counterparty written notice period								12 months
Collaborative Arrangement \| VantAI
Collaboration, License and Other Agreements
Collaborative arrangement, milestones, potential contingent payments							$ 1,670,000	$ 1,670,000				$ 1,670,000
Research and development expense								$ 7,500	$ 4,000	$ 4,400
Collaborative arrangement, jointly research and advance novel protein degrader therapies into development candidates, number \| item							3	3				3
Collaborative Arrangement \| Proteovant
Collaboration, License and Other Agreements
Collaborative arrangement, upfront payment			$ 20,000
Collaborative Arrangement \| Zai Lab (Shanghai) Co., Ltd.
Collaboration, License and Other Agreements
Collaborative arrangement, upfront payment, cash payment received				$ 25,000
Collaborative arrangement, milestones, contingent payments eligible to receive				$ 590,000
Collaborative arrangement, termination, counterparty written notice period, period from effective date, given after first commercial sale of licensed product in counterparty territory								12 months
Collaborative arrangement, termination, counterparty written notice period, period from effective date, given prior to first commercial sale of licensed product in counterparty territory								9 months
Collaborative Arrangement \| Roche, Collaboration (Pralsetnib) Agreement
Collaboration, License and Other Agreements
Collaborative arrangement, upfront payment, cash payment received					$ 675,000
Collaborative arrangement, milestones, specified regulatory and commercialization milestones, achieved												$ 105,000
Collaborative arrangement, percentage of global development costs shared, entity (as a percent)					45.00%
Collaborative arrangement, percentage of global development costs shared, counterparty (as a percent)					55.00%
Selling, general, and administrative expenses								$ 5,700
Collaborative Arrangement \| Clementia
Collaboration, License and Other Agreements
Collaborative arrangement, upfront payment, cash payment received	$ 25,000
Collaborative arrangement, milestones, cash payments received											$ 50,000
Collaborative arrangement, milestones, contingent payments eligible to receive	460,000
Collaborative arrangement, inventory purchased by counterparty	$ 1,500
Collaborative arrangement, termination, counterparty written notice period	12 months
Collaborative Arrangement \| C Stone
Collaboration, License and Other Agreements
Collaborative arrangement, upfront payment, cash payment received		$ 40,000
Collaborative arrangement, milestones, cash payments received								38,500
Collaborative arrangement, milestones, contingent payments eligible to receive							$ 307,500	$ 307,500				$ 307,500
Collaborative arrangement, licensed product term from first commercial sale						12 years
Collaborative arrangement, license option rights, number \| item						3
Collaborative arrangement, collaboration programs with exclusive commercialization rights, number \| item						3
Arrangement Other than Collaborative, Asset Purchase Agreement \| Rigel Pharmaceuticals, Inc.
Collaboration, License and Other Agreements
Asset purchase agreement, purchase price, receivable													$ 15,000
Asset purchase agreement, purchase price, receivable, payable upon first commercial sale													10,000
Asset purchase agreement, purchase price, receivable, additional contingent amount payable													5,000
Asset purchase agreement, contingent specified regulatory and commercial milestone payments													$ 102,500
Asset purchase agreement, tiered percentage royalties on annual net sales, low end of range (as a percent)													10.00%
Asset purchase agreement, tiered percentage royalties on annual net sales, high end of range (as a percent)													30.00%
Asset purchase agreement, transaction price							27,700
Asset purchase agreement, transaction price, inventory							6,500
Asset purchase agreement, transaction price, upfront payment							10,000
Asset purchase agreement, transaction price, milestone and royalty payments							$ 11,200

Employee Benefit Plans (Tables)

12 Months Ended

Dec. 31, 2024

Summary of benefits expected to be paid

Summary of plan balances

The following table summarizes the plan balances as of December 31, 2024 and 2023 (in thousands):


	December 31,		December 31,
	2024		2023
Fair value of plan assets	$	9,241	$	8,416
Projected benefit obligation		(16,065)		(14,632)
Unfunded status recorded as other long-term liabilities		(6,824)		(6,216)
Accumulated benefit obligation	$	12,071	$	10,984


	As of December 31
	2024
2025	$	466
2026		599
2027		546
2028		582
2029		623
2030 to 2034		4,028

Employee Benefit Plans - Switzerland Defined Benefit Plan - Net Periodic Benefit Cost (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Net Periodic Benefit Cost
Net periodic benefit cost	$ 1.4	$ 1.0	$ 1.2

Segment Information - Segment Revenue, Significant Segment Expenses and Segment Operating Loss (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Segment Information
Revenue	$ 508,824	$ 249,380	$ 204,036
Cost of sales	20,163	8,540	17,813
Research and development expense	341,433	427,720	477,419
Selling, general, and administrative expenses	359,272	295,141	237,374
Collaboration loss sharing		4,256	8,948
Stock-based compensation	108,911	92,672	98,971
Net Income (Loss)	(67,089)	(506,984)	(557,517)
Single Reportable Segment
Segment Information
Revenue	508,824	249,380	204,036
Cost of sales	20,163	8,540	17,813
Collaboration loss sharing		4,256	8,948
Other segment items	$ 144,955	$ (20,707)	$ (19,999)
Segment Reporting, Other Segment Item, Composition, Description	Other segment items include interest expense, net, other income (expense), net, debt extinguishment gain, and income tax expense.	Other segment items include interest expense, net, other income (expense), net, debt extinguishment gain, and income tax expense.	Other segment items include interest expense, net, other income (expense), net, debt extinguishment gain, and income tax expense.
Operating Segments \| Single Reportable Segment
Segment Information
Compensation and related expenses	$ 114,861	$ 110,740	$ 96,625
Early drug discovery and platform	76,970	70,383	75,542
Clinical and manufacturing related activities	38,889	138,801	198,317
Facilities and IT	35,589	33,783	28,917
Consulting and professional services	24,182	29,189	33,033
Other research and development	3,487	3,290	4,683
Research and development expense	293,978	386,186	437,117
Compensation and related expenses	127,286	120,756	84,324
Commercial and related expenses	96,604	53,579	33,334
Consulting and professional services	49,575	41,876	36,264
Facilities and IT	12,214	13,126	11,431
Other selling, general and administrative	12,137	14,666	13,352
Selling, general, and administrative expenses	297,816	244,003	178,705
Segment Reporting, Reconciling Item, Corporate Nonsegment
Segment Information
Research and development expense	47,500	41,500	40,300
Selling, general, and administrative expenses	$ 61,500	$ 51,100	$ 58,700

Consolidated Balance Sheets - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 102,014	$ 71,286
Marketable securities	513,473	639,355
Accounts receivable	75,797	42,827
Unbilled accounts receivable	1,812	351
Inventory	13,611	21,223
Prepaid expenses and other current assets	35,971	33,351
Total current assets	742,678	808,393
Marketable securities	248,450	56,530
Property and equipment, net	36,593	41,959
Operating lease right-of-use assets, net	64,181	73,691
Restricted cash	11,625	10,238
Equity investment	28,699	27,789
Other assets	47,587	30,650
Total assets	1,179,813	1,049,250
Current liabilities:
Accounts payable	6,790	4,710
Accrued expenses	133,088	127,992
Current portion of operating lease liabilities	13,346	11,933
Current portion of deferred revenue	2,005	812
Current portion of liabilities related to the sale of future royalties and revenues	61,650	39,198
Current portion of term loan	43,917	30,278
Total current liabilities	260,796	214,923
Operating lease liabilities, net of current portion	68,790	81,751
Deferred revenue, net of current portion	8,193	4,792
Liabilities related to the sale of future royalties and revenues, net of current portion	193,524	402,427
Term loan, net of current portion	343,053	208,535
Other long-term liabilities	6,792	6,213
Total liabilities	881,148	918,641
Commitments and Contingencies (Note 19)
Stockholders' equity:
Preferred stock, $0.001 par value; 5,000,000 shares authorized; no shares issued and outstanding
Common stock, $0.001 par value; 120,000,000 shares authorized; 63,712,256 and 61,147,236 shares issued and outstanding at December 31, 2024 and December 31, 2023, respectively	64	61
Additional paid-in capital	2,709,183	2,473,985
Accumulated other comprehensive loss	(3,551)	(3,495)
Accumulated deficit	(2,407,031)	(2,339,942)
Total stockholders' equity	298,665	130,609
Total liabilities and stockholders' equity	$ 1,179,813	$ 1,049,250

Net Loss Per Share

12 Months Ended

Dec. 31, 2024

Net Loss Per Share

14. Net Loss per Share

Basic net loss per share is calculated by dividing net loss by the weighted average shares of common stock outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting weighted average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period. For purposes of the diluted net loss per share calculation, stock options, unvested restricted stock units, PSUs and ESPP shares are considered to be common stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented as a result of the Company’s net loss.

The following common stock equivalents were excluded from the calculation of diluted net loss per share for the years ended December 31, 2024, 2023 and 2022 because including them would have had an anti-dilutive effect (in thousands):


	Year Ended December 31,
	2024	2023	2022
Stock options	6,370	6,658	6,233
Restricted stock units	2,291	2,107	1,894
Performance-based restricted stock units	143	53	—
ESPP shares	28	39	55
Total	8,832	8,857	8,182

Consolidated Balance Sheets (Parenthetical) - $ / shares	Dec. 31, 2024	Dec. 31, 2023
Preferred Stock Disclosures
Preferred stock, par value (in dollars per share)	$ 0.001	$ 0.001
Preferred stock, shares authorized (in shares)	5,000,000	5,000,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0
Common Stock Disclosures
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, shares authorized (in shares)	120,000,000	120,000,000
Common stock, shares issued (in shares)	63,712,256	61,147,236
Common Stock, shares outstanding (in shares)	63,712,256	61,147,236

Product Revenue - Product Revenue Allowance and Reserve (Details) - Valuation Allowances and Reserves, Product Revenue - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023
Product revenue allowance and reserve
Beginning Balance	$ 19,274	$ 9,788
Provision related to sales in the current period	80,847	37,281
Adjustment related to prior periods sales	(566)	(700)
Credits and payments made	(65,776)	(27,095)
Ending Balance	$ 33,779	$ 19,274

Leases - Weighted Average Remaining Lease-term and Weighted Average Discount Rate (Details)	Dec. 31, 2024
Leases
Weighted average remaining lease term in years	4 years 9 months 18 days
Weighted average discount rate	7.30%

Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Segment and Geographic Information (Details) - segment	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Segment and Geographic Information
Number of operating segments	1	1	1

Financing Arrangements - Net Proceeds Received (Details) - Secured Debt - USD ($) $ in Thousands	1 Months Ended			12 Months Ended		30 Months Ended
	May 31, 2024	Aug. 31, 2023	Jul. 31, 2022	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2024
Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities
Proceeds from (Repayments of) Debt
Gross proceeds						$ 400,000
Debt discount/transaction cost						17,308
Net proceeds				$ 146,973	$ 97,933	$ 382,692
Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility
Proceeds from (Repayments of) Debt
Gross proceeds			$ 150,000
Debt discount/transaction cost			12,214
Net proceeds			$ 137,786
Sixth Street Partners, Financing Agreement, 1st Senior Secured Delayed Draw Term Loan Facility
Proceeds from (Repayments of) Debt
Gross proceeds		$ 100,000
Debt discount/transaction cost		2,067
Net proceeds		$ 97,933
Sixth Street Partners, Financing Agreement, 2nd Senior Secured Delayed Draw Term Loan Facility
Proceeds from (Repayments of) Debt
Gross proceeds	$ 150,000
Debt discount/transaction cost	3,027
Net proceeds	$ 146,973

Financing Arrangements

12 Months Ended

Dec. 31, 2024

Financing Arrangements

3. Financing Arrangements

Royalty Pharma Purchase and Sale Agreement

On June 30, 2022, the Company entered into a purchase and sale agreement (Royalty Purchase Agreement) with Royalty Pharma Investments 2019 ICAV (Royalty Pharma). Pursuant to the Royalty Purchase Agreement, the Company received an upfront payment of $175.0 million in consideration for the Company’s rights to receive royalty payments on the net sales of GAVRETO worldwide excluding the CStone Territory (as defined below) and the U.S., under the terms of the Roche pralsetinib collaboration agreement.

Despite selling all rights to receive royalties on GAVRETO net sales of the underlying territories to Royalty Pharma, the Company maintained its involvement in the global co-development of pralsetinib with Roche and was therefore involved in the generation of these future royalties. Due to the Company’s significant continued involvement, any royalties and development and commercialization milestones earned pertaining to the underlying territory under the Roche pralsetinib collaboration agreement were recognized as collaboration revenue on the consolidated statements of operations and comprehensive loss throughout the contract term of the Roche pralsetinib collaboration agreement. The net proceeds received from the transaction were recorded as a liability related to sale of future royalties and revenues on the consolidated balance sheet on June 30, 2022.

The Roche pralsetinib collaboration agreement was terminated in February 2024 and the Company regained commercialization and development rights to GAVRETO from Roche worldwide excluding the CStone Territory. In connection with and effective upon the termination of the Roche pralsetinib collaboration agreement, on February 22, 2024 (the Royalty Pharma Termination Date), Royalty Pharma and the Company agreed to terminate the Royalty Purchase Agreement (Royalty Pharma Termination Agreement). Following the termination of the Royalty Purchase Agreement, the Company has no outstanding obligations under the Royalty Purchase Agreement, other than the remaining royalty payment obligation related to GAVRETO net sales as of the termination effective date. As of December 31, 2024, the Company had no plan to enter into a new arrangement to commercialize GAVRETO outside of the U.S. and the CStone Territory.

The Company has no material outstanding obligations under the Royalty Pharma Termination Agreement and it was accounted for as a debt extinguishment under ASC 470-50 as the terms and conditions of the Royalty Purchase Agreement had undergone a substantial modification and the modified terms are considered substantially different. As a result, during the year ended December 31, 2024, the Company recorded a debt extinguishment gain of $173.7 million as other income in the consolidated statements of operations and comprehensive loss. As of December 31, 2024, the net carrying value of the Company’s liability related to these agreements was $0.7 million, which was determined based on the fair value of the remaining cash flow associated with anticipated future payments outlined in the Royalty Pharma Termination Agreement. The Company subsequently paid the final $0.7 million amount in February 2025.

Financing Arrangements with Sixth Street Partners

In July 2022, the Company closed two transactions pursuant to a purchase and sale agreement (Future Revenue Purchase Agreement) and a debt financing transaction for up to $660.0 million (as amended, Financing Agreement) with Sixth Street Partners. Because two transactions were entered into with the same parties and in contemplation of one another, the Company recorded these transactions based on the relative fair values of each freestanding financial instrument and allocated the proceeds in proportion to those fair value amounts.

Sixth Street Partners Purchase and Sale Agreement

Pursuant to the Future Revenue Purchase Agreement, the Company received gross proceeds of $250.0 million in exchange for future royalty payments at a rate of 9.75% on up to $900 million each year of (i) aggregate worldwide annual net product sales of AYVAKIT/ AYVAKYT (avapritinib) and (ii), if it is approved, aggregate worldwide annual net product sales of BLU-263 (elenestinib), but excluding sales in Greater China, subject to a cumulative cap of 1.45 times the upfront invested capital or a total of $362.5 million. In the event that certain revenue targets are not achieved by specified dates, the royalty rate and cumulative cap shall be increased to 15% and 1.85 times the invested capital (or $462.5 million), respectively.

The Company continues to own the research, development, manufacturing and commercialization of AYVAKIT/ AYVAKYT and if it is approved, elenestinib, and has significant continuing involvement in the generation of the cash flows under the Future Revenue Purchase Agreement. Therefore, the Company continues to account for any revenue earned from worldwide product sales of AYVAKIT/ AYVAKYT and, if it is approved, elenestinib, on its consolidated statements of operations and comprehensive loss. Net proceeds received from the transaction were recorded as a liability related to sale of future royalties and revenues on the consolidated balance sheet. The Company accretes the $250.0 million, net of transaction costs of $5.4 million, to the total of these future payments as interest expense using the effective interest method over the estimated life of the arrangement.

As payments are made to Sixth Street Partners, the balance of the liability is effectively repaid over the life of the Future Revenue Purchase Agreement. In order to determine the amortization of the liability, the Company estimates the total amount of future revenue payments to be paid to Sixth Street Partners over the life of the arrangement. The exact amount of repayment is likely to change each reporting period. A significant increase or decrease in worldwide product sales of AYVAKIT/ AYVAKYT and, if it is approved, elenestinib, will materially impact the liability related to this arrangement, interest expense and the time period for repayment. The Company periodically assesses the expected payments to Sixth Street Partners and prospectively adjusts the amortization of the liability related to this arrangement for material changes in such payments. As of December 31, 2024, the Company’s estimate of this total interest expense resulted in an effective annual interest rate of 10.3%. These estimates contain assumptions that impact the amount recorded and the interest expense that will be recognized in future periods.

As of December 31, 2024, the net carrying value of the liability related to this arrangement was $254.4 million. The following table shows the activity within the liability account during the years ended December 31, 2024 and 2023 (in thousands):


	Year Ended December 31,
	2024		2023
Beginning balance at January 1	$	266,670	$	254,328
Interest expense recognized		27,134		28,065
Payments		(39,368)		(15,723)
Carrying value at December 31	$	254,436	$	266,670

Sixth Street Partners Term Loan

The Financing Agreement entered into by the parties in connection with the transaction provides for (i) a senior secured term loan facility of up to $150.0 million and (ii) a senior secured delayed draw term loan facility of up to $250.0 million to be funded in two tranches at the Company’s choice subject to certain terms and conditions. The term loans will mature on June 30, 2028 and bear interest at a variable rate equal to either the Secured Overnight Financing Rate (SOFR) plus 6.50% or the base rate plus 5.50%, subject to a floor of 1% and 2% with respect to the SOFR and base rate, respectively.

The following table shows the proceeds the Company has received under the Financing Agreement with Sixth Street Partners (in thousands):


				Debt discount/
Term loan draw	Date	Gross proceeds		Transaction cost		Net proceeds
Senior Secured Term Loan Facility	July 2022	$	150,000	$	12,214	$	137,786
1st Senior Secured Delayed Draw Term Loan Facility	August 2023		100,000		2,067		97,933
2nd Senior Secured Delayed Draw Term Loan Facility	May 2024		150,000		3,027		146,973
		$	400,000	$	17,308	$	382,692

Debt discounts and transaction costs have been recorded as a reduction to the carrying amount of the debt on the Company’s consolidated balance sheet and are amortized as additional interest expenses using the effective interest rate method over the period from issuance through maturity. In addition, the Company may at any time request an incremental term loan in an amount not to exceed $260.0 million on terms to be agreed and subject to the consent of Sixth Street Partners providing such incremental term loan. As of December 31, 2024, the Company’s estimate of the total interest expense resulted in an effective annual interest rate of 12.0%. The carrying amount of the debt as of December 31, 2024 is subject to variable interest rates, which are based on current market rates, and as such, approximates fair value.

The following table shows the activity within the liability account during the years ended December 31, 2024 and 2023 (in thousands):


	Year Ended December 31,
	2024		2023
Beginning balance at January 1	$	238,813	$	139,083
Net proceeds received		146,973		97,933
Interest expense recognized		41,668		23,540
Payments		(40,484)		(21,743)
Carrying value at December 31	$	386,970	$	238,813

The total estimated total gross payments due under the term loan are as follows as of December 31, 2024 (in thousands):


	As of December 31, 2024
2025	$	43,917
2026		43,917
2027		43,917
2028		421,897
Total	$	553,648

The Company’s obligations under the Financing Agreement are secured, subject to certain exceptions, by security interests in substantially all assets of the Company and certain of its subsidiaries. The Financing Agreement contains customary negative covenants that, among other things and subject to certain exceptions, could restrict the Company’s ability to incur additional liens, incur additional indebtedness, make investments, including acquisitions, engage in fundamental changes, sell or dispose of assets that constitute collateral, including certain intellectual property, pay dividends or make any distribution or payment on or redeem, retire or purchase any equity interests, amend, modify or waive certain material agreements or organizational documents and make payments of certain subordinated indebtedness. The Financing Agreement also requires the Company to maintain a consolidated liquidity of at least $80.0 million. As of December 31, 2024, the Company was in compliance with the applicable terms and conditions of the covenants under the Financing Agreement.

Inventory (Tables)

12 Months Ended

Dec. 31, 2024

Schedule of capitalized inventory


	As of December 31,
	2024		2023
Raw materials	$	—	$	3,147
Work in process		30,300		29,132
Finished goods		8,975		4,823
Total	$	39,275	$	37,102

Balance sheet classification


	As of December 31,
	2024		2023
Inventory	$	13,611	$	21,223
Other assets		25,664		15,879
Total	$	39,275	$	37,102

Marketable Securities (Tables)

12 Months Ended

Dec. 31, 2024

Schedule of marketable securities


	Amortized		Unrealized		Unrealized		Fair
December 31, 2024	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	129,897	$	118	$	(230)	$	129,785
U.S. treasury obligations		631,514		1,025		(401)		632,138
Total	$	761,411	$	1,143	$	(631)	$	761,923


	Amortized		Unrealized		Unrealized		Fair
December 31, 2023	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	170,194	$	27	$	(418)	$	169,803
U.S. treasury obligations		525,880		588		(386)		526,082
Total	$	696,074	$	615	$	(804)	$	695,885

Summary of the fair value and the number of the available-for-sale securities by their maturities


	As of December 31,
	2024				2023
	Fair		Unrealized		Fair		Unrealized
	value		losses		value		losses
Debt securities in unrealized loss position for 12 months or less	$	205,910	$	(631)	$	267,917	$	(550)
Debt securities in unrealized loss position for more than 12 months		—		—		64,659		(254)
Total debt securities in unrealized loss position	$	205,910	$	(631)	$	332,576	$	(804)

Summary of fair value and number of available-for-sale securities in loss positions


	As of December 31,
	2024				2023
	Amortized		Fair		Amortized		Fair
	Cost		value		Cost		value
Within one year	$	512,515	$	513,473	$	639,881	$	639,355
After one through five years		248,896		248,450		56,193		56,530
Total	$	761,411	$	761,923	$	696,074	$	695,885

Summary of proceeds from maturities of debt securities


	Year ended December 31,
	2024		2023		2022
Proceeds from maturities of debt securities	$	923,560	$	1,056,465	$	349,373

Tabular disclosure of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

Reference 1: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 9
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-9

Reference 2: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 3: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 5: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 6: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aa)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 7: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (aaa)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 8: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 2
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-2

Reference 9: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 10: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 11: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (c)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Reference 12: http://www.xbrl.org/2009/role/commonPracticeRef
-Topic 320
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 3
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481800/320-10-50-3

Collaboration, License and Other Agreements - Transaction Price (Details) - Collaborative Arrangement - USD ($) $ in Millions		1 Months Ended			12 Months Ended
	Oct. 15, 2019	Jul. 31, 2020	Jun. 30, 2018	Mar. 31, 2016	Dec. 31, 2023	Dec. 31, 2022
Roche, Collaboration (Pralsetnib) Agreement
Collaboration, License and Other Agreements
Collaborative arrangement, transaction price		$ 695.7
Collaborative arrangement, transaction price, upfront payment		675.0
Collaborative arrangement, transaction price, stock issued, premium on sale of stock to counterparty		$ 20.7
Roche, Collaboration and License (Immunotherapy) Agreement
Collaboration, License and Other Agreements
Collaborative arrangement, transaction price				$ 64.7
Clementia
Collaboration, License and Other Agreements
Collaborative arrangement, transaction price	$ 46.5
C Stone
Collaboration, License and Other Agreements
Collaborative arrangement, transaction price			$ 40.0
Collaborative arrangement, transaction price, cash consideration received					$ 9.0	$ 6.5

Income Taxes - Net Operating Loss Carryforwards (Details) $ in Millions	Dec. 31, 2024 USD ($)
Net Operating Loss Carryforwards
Net operating loss carryforwards, limitations on use, available for utilization	$ 2.0
Domestic Tax Authority
Net Operating Loss Carryforwards
Net operating loss carryforwards	859.2
Net operating loss carryforwards, unlimited	843.3
State and Local Jurisdiction
Net Operating Loss Carryforwards
Net operating loss carryforwards	$ 1,077.3

Consolidated Statements of Cash Flows - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Cash flows from operating activities
Net loss	$ (67,089)	$ (506,984)	$ (557,517)
Adjustments to reconcile net loss to net cash used in operating activities:
Depreciation and amortization	16,291	11,665	11,735
Non-cash lease expense	9,925	9,109	8,364
Stock-based compensation	108,911	92,672	98,971
Non-cash interest expense	1,184	14,138	15,799
Non-cash customer consideration			(27,500)
Non-cash debt extinguishment gain	(173,676)
Net (accretion of discount) amortization of premium on investments	(22,465)	(17,466)	(982)
Other	(982)	1,147	2,622
Changes in assets and liabilities:
Accounts receivable	(32,845)	(20,715)	1,572
Unbilled accounts receivable	147	13,061	(3,804)
Inventory	(4,026)	(4,366)	(8,922)
Prepaid expenses and other current assets	(2,630)	3,206	(16,938)
Other assets	(11,130)	(4,711)	(16,320)
Accounts payable	2,197	1,880	(5,816)
Accrued expenses	8,207	1,075	9,368
Other long-term liabilities	(12,233)	(7,240)
Deferred revenue	(409)	(12,687)	(4,813)
Operating lease liabilities	(11,963)	(10,631)	(8,096)
Net cash used in operating activities	(192,586)	(436,847)	(502,277)
Cash flows from investing activities
Purchases of property and equipment	(4,630)	(16,062)	(8,919)
Purchases of investments	(966,431)	(766,363)	(489,705)
Maturities of investments	923,560	1,056,465	349,373
Other			(290)
Net cash provided by (used in) investing activities	(47,501)	274,040	(149,541)
Cash flows from financing activities
Proceeds from at-the-market offerings, net of issuance costs	48,936
Net proceeds from stock option exercises and employee stock purchase plan	77,743	21,336	8,196
Net proceeds from term loan facility	146,973	97,933	137,786
Net proceeds from the sale of future royalties and revenues			415,828
Principal payments for financing arrangements	(541)	(44)
Net cash provided by financing activities	273,111	119,225	561,810
Net increase (decrease) in cash, cash equivalents, and restricted cash	33,024	(43,582)	(90,008)
Cash, cash equivalents and restricted cash at beginning of period	81,524	124,904	215,119
Effect of exchange rate changes on cash, cash equivalents and restricted cash	(909)	202	(207)
Cash, cash equivalents and restricted cash at end of period	113,639	81,524	124,904
Supplemental cash flow information
Cash paid for interest	79,852	38,467	9,814
Property and equipment purchases unpaid at period end	425	556	324
Cash paid (refunds received) for taxes, net	$ 1,104	$ (49)	$ 6,505

Marketable Securities - Unrealized Loss Positions - General Information (Details) - security	Dec. 31, 2024	Dec. 31, 2023
Unrealized loss position, number of positions
Number of held securities in an unrealized loss position	32	62

Marketable Securities - Tabular Disclosure (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Marketable securities, available-for-sale:
Amortized cost	$ 761,411	$ 696,074
Unrealized gain	1,143	615
Unrealized losses	(631)	(804)
Fair value	761,923	695,885
U.S. government agency securities
Marketable securities, available-for-sale:
Amortized cost	129,897	170,194
Unrealized gain	118	27
Unrealized losses	(230)	(418)
Fair value	129,785	169,803
U.S. Treasury obligations
Marketable securities, available-for-sale:
Amortized cost	631,514	525,880
Unrealized gain	1,025	588
Unrealized losses	(401)	(386)
Fair value	$ 632,138	$ 526,082

Amount of investment in debt security measured at fair value with change in fair value recognized in other comprehensive income (available-for-sale).

Collaboration, License and Other Agreements - Sale of Stock (Details) - USD ($) $ / shares in Units, $ in Thousands	1 Months Ended	12 Months Ended
	Jul. 31, 2020	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022
Sale of Stock
Stock issued (in shares)		544,719	0	0
Issuance of common stock		$ 48,936
Private Placement
Sale of Stock
Stock issued (in shares)	1,035,519
Share price (in dollars per share)	$ 96.57
Purchase consideration	$ 100,000
Issuance of common stock	79,300
Share purchase consideration, premium	$ 20,700

Equity impact of the value of new stock issued during the period. Includes shares issued in an initial public offering or a secondary public offering.

Collaboration, License and Other Agreements - Collaboration Loss Sharing (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2023	Dec. 31, 2022
Collaboration, License and Other Agreements
Collaboration loss sharing	$ 4,256	$ 8,948
Collaborative Arrangement \| Roche, Collaboration (Pralsetnib) Agreement
Collaboration, License and Other Agreements
Collaboration loss sharing	$ 4,300	$ 8,900

Stock-based Compensation - Unrecognized Compensation Costs (Details) $ in Millions	Dec. 31, 2024 USD ($)
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock awards	$ 207.6
Total unrecognized compensation cost related to non-vested stock option awards	$ 71.4
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 4 months 24 days
Employee Stock Option
Stock-based compensation
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 4 months 24 days
Restricted Stock Units
Stock-based compensation
Total unrecognized compensation cost related to non-vested non-option awards	$ 126.2
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 4 months 24 days
Performance-based restricted stock units
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock awards	$ 10.0
Weighted-average period over which unrecognized compensation cost will be recognized	1 year 9 months 18 days

Accrued Expenses (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023
Accrued Expenses
Research, development and commercial contract costs	$ 33,957	$ 49,354
Employee compensation	48,725	43,434
Accrued professional fees	14,134	12,522
Revenue-related reserves	30,816	17,465
Other	5,456	5,217
Total	$ 133,088	$ 127,992

Stockholders' Equity (Details) - USD ($) $ in Thousands	12 Months Ended
Stockholders' Equity (Details) - USD ($) $ in Thousands	Dec. 31, 2024	Dec. 31, 2023	Dec. 31, 2022	Feb. 28, 2022
Proceeds from Issuance or Sale of Equity [Abstract]
At-the-market facility, aggregate offering price				$ 300,000
At-the-market offerings, net of issuance costs (in shares)	544,719	0	0
Proceeds from at-the-market offerings, net of issuance costs	$ 48,936

Fair Value of Financial Instruments (Tables)

12 Months Ended

Dec. 31, 2024