0001558370-25-006108 | 10-Q | XBRL Viewer

Net Earnings Per Share

13. Net Earnings per Share

Basic earnings per share (EPS) is calculated by dividing net income by the weighted average number of shares of common stock outstanding during the period, without consideration of common stock equivalents. Diluted EPS 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 EPS calculation, the effect of stock options, unvested restricted stock units, PSUs and ESPP shares on the weighted average number of shares is calculated using the treasury stock method. In periods with reported net operating losses, all common stock equivalents are deemed anti-dilutive such that basic net loss per share and diluted net loss per share are equal.

The calculation of net income and the number of shares used to compute basic and diluted EPS are as follows (in thousands, except for per share data):


	Three Months Ended
	March 31,
	2025		2024
Net income	$	496	$	89,136
Weighted average shares outstanding - basic		64,096		61,580
Effect of dilutive securities:
Stock options		1,381		1,165
Restricted stock units		942		985
Performance-based restricted stock units		107		72
Weighted average shares outstanding - diluted		66,526		63,802
Net income per share - basic	$	0.01	$	1.45
Net income per share - diluted	$	0.01	$	1.40

The following table sets forth the potential shares excluded from the calculation of EPS for the three months ended March 31, 2025 and 2024 because their inclusion would be anti-dilutive (in thousands):


	March 31,
	2025	2024
Stock options	2,026	2,126
Restricted stock units	18	372
Performance-based restricted stock units	53	31
ESPP shares	28	39
Total	2,125	2,568

Collaboration, License and Other Agreements - Revenue Recognized (Details) - USD ($) $ in Thousands		1 Months Ended	3 Months Ended			9 Months Ended	12 Months Ended		24 Months Ended
	Aug. 01, 2022	Aug. 31, 2022	Mar. 31, 2025	Jun. 30, 2024	Mar. 31, 2024	Mar. 31, 2025	Dec. 31, 2021	Dec. 31, 2018	Dec. 31, 2020
Revenues
Revenue			$ 149,413		$ 96,116
Collaboration, License and Other
Revenues
Revenue					3,591
Collaboration, License and Other \| Rigel Pharmaceuticals, Inc.
Revenues
Revenue				$ 24,300
Collaboration, License and Other \| Zai Lab (Shanghai) Co., Ltd.
Revenues
Revenue			0		0		$ 25,000
Collaboration, License and Other \| Clementia
Revenues
Revenue			0		0				$ 46,500
Collaboration, License and Other \| C Stone
Revenues
Revenue								$ 40,000
Collaboration revenue \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue						$ 0
Territory-specific activities, manufacturing services and royalty \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue					500
Territory-specific activities, manufacturing services and royalty \| C Stone
Revenues
Revenue, net			(393)		1,653
GAVRETO profit share in the U.S. \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue					1,400
License \| IDRx, Inc. \| Director
Revenues
Revenue	$ 27,500	$ 27,500	$ 0		$ 0

Accrued Expenses

3 Months Ended

Mar. 31, 2025

Accrued Expenses

9. Accrued Expenses

Accrued expenses as of March 31, 2025 and December 31, 2024 consist of the following (in thousands):


	March 31,		December 31,
	2025		2024
Research, development and commercial contract costs	$	32,637	$	33,957
Employee compensation		21,771		48,725
Accrued professional fees		17,999		14,134
Revenue-related reserves		40,348		30,816
Other		9,300		5,456
Total	$	122,055	$	133,088

Income Taxes

3 Months Ended

Mar. 31, 2025

Income Taxes

14. Income Taxes

The Company utilizes the asset and liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities using enacted tax rates in effect for years in which the temporary differences are expected to reverse. The Company provides a valuation allowance when it is more likely than not that deferred tax assets will not be realized.

The realization of deferred income tax assets is dependent on the generation of sufficient taxable income during future periods in which temporary differences are expected to reverse. Where the realization of such assets does not meet the more likely than not criterion, the Company applies a valuation allowance against the deferred income tax asset under consideration. The valuation allowance is reviewed periodically and if the assessment of the more likely than not criterion changes, the valuation allowance is adjusted accordingly. As of March 31, 2025, the Company has a full valuation allowance applied against its U.S. and foreign deferred tax assets.

During the three months ended March 31, 2025, GSK acquired IDRx for an upfront consideration of $1.0 billion and an additional $150.0 million in regulatory approval-based milestone payments. The Company received consideration in the amount of $78.7 million and the Company recorded a gain on sale of investment in the amount of

$50.0 million during the three months ended March 31, 2025. For tax purposes, the gain will also be included in the taxable income calculation for the year ended December 31, 2025.

As of March 31, 2025, the Company expects to be in a taxable income position for the calendar year ended December 31, 2025, and has recorded an income tax expense of $0.8 million for the three months ended March 31, 2025.

The Company recognizes interest and penalties related to unrecognized tax benefits in the provision for income taxes. As of March 31, 2025, the Company did not have any gross unrecognized tax benefit.

The entire disclosure for income tax.

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Marketable Securities - Proceeds from Maturities (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Proceeds from the maturities of debt securities
Proceeds from the maturities of debt securities	$ 271,500	$ 278,400

Collaboration, License and Other Agreements - Equity Investment (Details) - USD ($) $ in Thousands		1 Months Ended	3 Months Ended
	Aug. 01, 2022	Jul. 31, 2023	Mar. 31, 2025	Dec. 31, 2024
Equity Investment
Equity investment				$ 28,699
Equity investment gain			$ 50,039
Proceeds from sale of equity investment			78,737
GSK Plc \| IDRx, Inc.
Business Combination, Consideration Transferred
Business combination, consideration transferred			1,000,000
Business Combination, Contingent Consideration Arrangements
Business combination, contingent consideration arrangements, range of outcomes, value, high			150,000
Collaborative Arrangement \| IDRx, Inc. \| Director
Equity Investment
Equity investment, license agreement, shares received (in shares)	4,509,105	192,282
Equity investment	$ 27,800			$ 28,700
Equity investment (in shares)				4,701,387
Equity investment, transaction costs	$ 300
Equity investment gain			50,000
Proceeds from sale of equity investment			$ 78,700

Accrued Expenses (Tables)

3 Months Ended

Mar. 31, 2025

Schedule of accrued expenses


	March 31,		December 31,
	2025		2024
Research, development and commercial contract costs	$	32,637	$	33,957
Employee compensation		21,771		48,725
Accrued professional fees		17,999		14,134
Revenue-related reserves		40,348		30,816
Other		9,300		5,456
Total	$	122,055	$	133,088

Restricted Cash - Additional Information (Details) - USD ($) $ in Millions	Mar. 31, 2025	Dec. 31, 2024
Restricted cash
Restricted cash	$ 11.8	$ 11.6

Financing Arrangements - Debt Extinguishment (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2024	Mar. 31, 2025	Feb. 22, 2022
Gain (Loss) on Extinguishment of Debt
Debt extinguishment gain	$ 173,658
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

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

Net Earnings Per Share - Anti-dilutive Securities (Details) - shares shares in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	2,125	2,568
Employee Stock Option
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	2,026	2,126
Restricted Stock Units (RSUs)
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	18	372
Performance-based restricted stock units
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	53	31
Employee Stock
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	28	39

Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Policies)	3 Months Ended
	Mar. 31, 2025
Policy Text Blocks
Basis of Presentation	Basis of Presentation The unaudited interim condensed 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), Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB) and the rules and regulations of the Securities and Exchange Commission (SEC). Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these financial statements should be read in conjunction with the financial statements as of and for the year ended December 31, 2024 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on February 13, 2025 (2024 Annual Report on Form 10-K). The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited financial statements, and updated, as necessary, in this report. In the opinion of the Company’s management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments that are necessary to present fairly the Company’s financial position as of March 31, 2025, the results of its operations for the three months ended March 31, 2025 and 2024, stockholders’ equity for the three months ended March 31, 2025 and 2024 and cash flows for the three months ended March 31, 2025 and 2024. Such adjustments are of a normal and recurring nature. The results for the three months ended March 31, 2025 are not necessarily indicative of the results for the year ending December 31, 2025 or for any other future period. The accompanying unaudited interim condensed 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	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	Significant Accounting Policies The significant accounting policies used in preparation of these condensed consolidated financial statements for the three months ended March 31, 2025 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2024 Annual Report on Form 10-K.
Recent Accounting Pronouncements	Recent 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 condensed consolidated financial statements and disclosures. 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 annual reporting periods beginning after December 15, 2024, with early adoption permitted. The Company will adopt the new disclosure requirements in its 2025 Annual Report on Form 10-K. In November 2024, the FASB issued ASU 2024-03, Disaggregation of Income Statement Expenses, which is intended to improve disclosures by requiring additional information about specific expense categories in the notes to the financial statements on an annual and interim basis. The standard will be effective for annual reporting periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027, with early adoption permitted. The standard updates may be applied on either a prospective or retrospective basis. The Company is currently evaluating the disclosure requirements related to this new standard.
Reclassification	Reclassification Certain items in the prior year’s condensed consolidated financial statements have been reclassified to conform to the current presentation.

Fair Value of Financial Instruments

3 Months Ended

Mar. 31, 2025

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 March 31, 2025 (in thousands):


			Active		Observable		Unobservable
	March 31,		Markets		Inputs		Inputs
Description	2025		(Level 1)		(Level 2)		(Level 3)
Cash equivalents:
Money market funds	$	67,898	$	67,898	$	—	$	—
U.S. treasury obligations		22,402		22,402		—		—
Marketable securities, available-for-sale:
U.S. government agency securities		190,651		—		190,651		—
U.S. treasury obligations		586,888		586,888		—		—
Total	$	867,839	$	677,188	$	190,651	$	—

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 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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Restricted Cash - Tabular Disclosure (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024	Mar. 31, 2024	Dec. 31, 2023
Reconciliation of cash, cash equivalents, and restricted cash
Cash and cash equivalents	$ 122,245	$ 102,014	$ 113,326
Restricted cash	11,847	11,625	10,634
Total cash, cash equivalents, and restricted cash shown in condensed consolidated statements of cash flows	$ 134,092	$ 113,639	$ 123,960	$ 81,524

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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Collaboration, License and Other Agreements (Tables)

3 Months Ended

Mar. 31, 2025

Summary of prepaid assets


	March 31, 2025						December 31, 2024
	Current		Noncurrent		Total		Current		Noncurrent		Total
Prepaid and other assets	$	5,030	$	5,816	$	10,846	$	4,971	$	2,666	$	7,637

Rigel Pharmaceuticals, Inc. | Collaboration, License and Other

Summary of contract assets and/or contract liabilities


	March 31, 2025						December 31, 2024
	Current		Noncurrent		Total		Current		Noncurrent		Total
Contract assets	$	2,915	$	6,602	$	9,517	$	2,939	$	7,380	$	10,319
Contract liabilities	$	—	$	3,562	$	3,562	$	—	$	3,562	$	3,562

Roche, Collaboration (Pralsetnib) Agreement | Collaboration, License and Other

Summary of contract assets and/or contract liabilities


	March 31,		December 31,
	2025		2024
Accrued expenses	$	365	$	1,712

C Stone | Collaboration, License and Other

Summary of revenue recognized

Summary of contract assets and/or contract liabilities


	March 31,		December 31,
	2025		2024
Accrued expenses	$	548	$	2,027

Inventory - Write-down (Details) - USD ($) $ in Thousands	3 Months Ended
Inventory - Write-down (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Mar. 31, 2024
Inventory
Inventory write-down	$ 0	$ 0

Leases - Components of Lease Expense (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Lease, Cost [Abstract]
Lease cost	$ 6,252	$ 5,572
Net lease cost	$ 6,252	$ 5,572

Stock-based Compensation - Stock-based Compensation Expense - By Type (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Total stock based compensation expense
Subtotal	$ 29,299	$ 24,457
Capitalized stock-based compensation costs	(243)	(232)
Stock-based compensation expense included in total cost and operating expenses	29,056	24,225
Employee Stock Option
Total stock based compensation expense
Subtotal	10,268	9,573
Restricted Stock Units (RSUs)
Total stock based compensation expense
Subtotal	16,827	13,836
Performance-based restricted stock units
Total stock based compensation expense
Subtotal	1,844	594
Employee Stock
Total stock based compensation expense
Subtotal	$ 360	$ 454

Stock-based Compensation - Performance-based Restricted Stock Units (Details) - Performance-based restricted stock units	3 Months Ended
	Mar. 31, 2025
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%

Inventory (Tables)

3 Months Ended

Mar. 31, 2025

Schedule of capitalized inventory


	March 31,		December 31,
	2025		2024
Work in process	$	35,517	$	30,300
Finished goods		10,020		8,975
Total	$	45,537	$	39,275

Balance sheet classification


	March 31,		December 31,
	2025		2024
Inventory	$	12,953	$	13,611
Other assets		32,584		25,664
Total	$	45,537	$	39,275

Document and Entity Information - $ / shares	3 Months Ended
Document and Entity Information - $ / shares	Mar. 31, 2025	Apr. 29, 2025
Cover [Abstract]
Entity Central Index Key	0001597264
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Mar. 31, 2025
Document Transition Report	false
Securities Act 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 Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Listing, Par Value Per Share	$ 0.001
Entity Common Stock, Shares Outstanding		64,582,163
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2025
Document Fiscal Period Focus	Q1
Amendment Flag	false

Income Taxes - Equity Investment (Details) $ in Thousands	3 Months Ended
Income Taxes - Equity Investment (Details) $ in Thousands	Mar. 31, 2025 USD ($)
Equity Investment
Proceeds from sale of equity investment	$ 78,737
Equity investment gain	50,039
GSK Plc \| IDRx, Inc.
Business Combination, Consideration Transferred
Business combination, consideration transferred	1,000,000
Business Combination, Contingent Consideration Arrangements
Business combination, contingent consideration arrangements, range of outcomes, value, high	150,000
Collaborative Arrangement \| IDRx, Inc. \| Director
Equity Investment
Proceeds from sale of equity investment	78,700
Equity investment gain	$ 50,000

Net Earnings Per Share (Tables)

3 Months Ended

Mar. 31, 2025

Schedule of net income (loss) and the number of shares used to compute basic and diluted net income (loss) per share


	Three Months Ended
	March 31,
	2025		2024
Net income	$	496	$	89,136
Weighted average shares outstanding - basic		64,096		61,580
Effect of dilutive securities:
Stock options		1,381		1,165
Restricted stock units		942		985
Performance-based restricted stock units		107		72
Weighted average shares outstanding - diluted		66,526		63,802
Net income per share - basic	$	0.01	$	1.45
Net income per share - diluted	$	0.01	$	1.40

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


	March 31,
	2025	2024
Stock options	2,026	2,126
Restricted stock units	18	372
Performance-based restricted stock units	53	31
ESPP shares	28	39
Total	2,125	2,568

Financing Arrangements

3 Months Ended

Mar. 31, 2025

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.

Due to the Company's significant involvement in the global co-development of pralsetinib with Roche, which directly contributed to the generation of future GAVRETO royalties, 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 income (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 March 31, 2025, 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. This agreement was accounted for as a debt extinguishment under ASC 470-50 because 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, the Company recorded a debt extinguishment gain of $173.7 million as other income in the unaudited condensed consolidated statements of operations and comprehensive income during the three months ended March 31, 2024. As of March 31, 2025, the Company paid off the final royalty amount related to the Royalty Pharma Termination Agreement.

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, the 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 unaudited condensed consolidated statements of operations and comprehensive income. 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 March 31, 2025, 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 March 31, 2025, the net carrying value of the liability related to this arrangement was $246.6 million. The following table shows the activity within the liability account during the three months ended March 31, 2025 and 2024 (in thousands):


	Three Months Ended March 31,
	2025		2024
Carrying value as of January 1	$	254,436	$	266,670
Interest expense recognized		6,199		7,096
Payments		(14,003)		(6,875)
Carrying value as of March 31	$	246,632	$	266,891

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 March 31, 2025, 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 March 31, 2025 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 three months ended March 31, 2025 and 2024 (in thousands):


	Three Months Ended March 31,
	2025		2024
Carrying value as of January 1	$	386,970	$	238,813
Interest expense recognized		11,605		7,977
Payments		(10,829)		(7,405)
Carrying value as of March 31	$	387,746	$	239,385

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 March 31, 2025, the Company was in compliance with the applicable terms and conditions of the covenants under the Financing Agreement.

Stock-based Compensation - Stock-based Compensation Expense - General Information (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Total stock based compensation expense
Stock-based compensation expense	$ 29,056	$ 24,225

Financing Arrangements - Long-term Debt (Details) - Sale of Future Royalties and Revenues - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024	Mar. 31, 2024	Dec. 31, 2023	Feb. 22, 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	$ 246,632	$ 254,436	$ 266,891	$ 266,670

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

Stock-based Compensation - Unrecognized Compensation Costs (Details) $ in Millions	Mar. 31, 2025 USD ($)
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock option awards	$ 102.6
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 9 months 18 days
Restricted Stock Units (RSUs)
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock awards	$ 197.4
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 10 months 24 days
Performance-based restricted stock units
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock awards	$ 29.1
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 3 months 3 days

Product Revenue and Related Reserves - Product Revenue Allowance and Reserve - Roll Forward (Details) - Valuation Allowances and Reserves, Product Revenue - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Product Revenue Allowance and Reserve
Beginning Balance	$ 33,779	$ 19,274
Provision related to sales in the current period	28,964	16,926
Adjustment related to prior periods sales	(2,061)	(309)
Credits and payments made	(17,422)	(12,488)
Ending Balance	$ 43,260	$ 23,403

Inventory - Balance Sheet Classification (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Inventory
Inventory	$ 12,953	$ 13,611
Other assets	32,584	25,664
Total	$ 45,537	$ 39,275

Fair Value of Financial Instruments (Tables)

3 Months Ended

Mar. 31, 2025

Schedule of financial instruments measured at fair value

The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of March 31, 2025 (in thousands):


			Active		Observable		Unobservable
	March 31,		Markets		Inputs		Inputs
Description	2025		(Level 1)		(Level 2)		(Level 3)
Cash equivalents:
Money market funds	$	67,898	$	67,898	$	—	$	—
U.S. treasury obligations		22,402		22,402		—		—
Marketable securities, available-for-sale:
U.S. government agency securities		190,651		—		190,651		—
U.S. treasury obligations		586,888		586,888		—		—
Total	$	867,839	$	677,188	$	190,651	$	—

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	$	—

Stockholders' Equity (Details) - USD ($) $ in Thousands	3 Months Ended
Stockholders' Equity (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Mar. 31, 2024	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)	0	544,719
Proceeds from at-the-market offerings, net of issuance costs		$ 48,936

Stock-based Compensation (Tables)

3 Months Ended

Mar. 31, 2025

Summary of stock option activity


		Weighted-Average
	Shares	Exercise Price
Outstanding at December 31, 2024	6,369,724	$	72.09
Granted	795,360		97.50
Exercised	(213,210)		62.65
Canceled	(26,578)		74.98
Outstanding at March 31, 2025	6,925,296	$	75.28
Exercisable at March 31, 2025	4,538,663	$	72.02

Summary of restricted stock units activity


		Weighted-Average
	Shares	Grant Date Fair Value
Unvested shares at December 31, 2024	2,290,931	$	75.36
Granted	932,355		97.06
Vested	(638,445)		75.03
Forfeited	(34,936)		75.91
Unvested shares at March 31, 2025	2,549,905	$	83.37

Summary of performance-based restricted stock units activity


		Weighted-Average
		Grant Date
	Shares	Grant Date Fair Value
Unvested shares at December 31, 2024	142,500	$	107.65
Granted	152,825		136.93
Vested	—		—
Forfeited	—		—
Unvested shares at March 31, 2025	295,325	$	122.80

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


	Three Months Ended
		March 31,
		2025		2024
Stock options	$	10,268	$	9,573
Restricted stock units		16,827		13,836
Performance-based restricted stock units		1,844		594
Employee stock purchase plan		360		454
Subtotal		29,299		24,457
Capitalized stock-based compensation costs		(243)		(232)
Stock-based compensation expense included in total cost and operating expenses	$	29,056	$	24,225


	Three Months Ended
	March 31,
	2025		2024
Research and development	$	12,127	$	10,875
Selling, general and administrative		16,929		13,350
Total	$	29,056	$	24,225

Segment Information (Tables)

3 Months Ended

Mar. 31, 2025

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


	Three Months Ended
	March 31,
	2025		2024
Revenue	$	149,413	$	96,116
Less¹:
Cost of sales		2,802		3,191
Research and development expense²:
Compensation and related expenses		31,790		29,592
Early drug discovery and platform⁵		18,170		14,565
Facilities and IT⁵		12,977		10,580
Clinical and manufacturing related activities⁵		8,251		17,857
Consulting and professional services		7,446		4,294
Other		1,129		428
Total research and development expense²		79,763		77,316
Selling, general and administrative expense³:
Compensation and related expenses		33,852		33,116
Commercial and related expenses⁵		27,053		17,552
Consulting and professional services		9,869		14,500
Facilities and IT		4,834		2,779
Other⁵		3,270		2,260
Total selling, general and administrative expense³		78,878		70,207
Stock-based compensation		29,056		24,225
Other segment items⁴		41,582		167,959
Net income	$	496	$	89,136

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 three months ended March 31, 2025 and 2024 exclude $12.1 million and $10.9 million of stock-based compensation expense, respectively.

Selling, general and administrative expense for the three months ended March 31, 2025 and 2024 exclude $16.9 million and $13.4 million of stock-based compensation expense, respectively.

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

Certain prior period items are recast to conform to the current period presentation.

Product Revenue and Related Reserves (Tables)

3 Months Ended

Mar. 31, 2025

Schedules of concentration of risk

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


	March 31,		December 31,
	2025		2024
Customer 1	28	%	31	%
Customer 2	13	%	11	%
Customer 3	11	%	14	%

Product

Summary of revenue recognized


	Three Months Ended
	March 31,
	2025		2024
United States	$	129,446	$	83,136
Rest of World		19,967		9,389
Total product revenue	$	149,413	$	92,525

Schedule of product revenue allowance and reserve categories


	Three Months Ended March 31,
	2025		2024
Beginning balance at January 1	$	33,779	$	19,274
Provision related to sales in the current period		28,964		16,926
Adjustment related to prior periods sales		(2,061)		(309)
Credits and payments made		(17,422)		(12,488)
Ending balance at March 31	$	43,260	$	23,403


	March 31,		December 31,
	2025		2024
Reduction of accounts receivable, net	$	2,912	$	2,963
Component of accrued expenses		40,348		30,816
Total revenue-related reserves	$	43,260	$	33,779

Net Earnings Per Share - Computation (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Net income - basic and diluted
Net Income (Loss)	$ 496	$ 89,136
Net income - basic	496	89,136
Net income - diluted	$ 496	$ 89,136
Weighted average shares outstanding
Weighted average shares outstanding - basic (in shares)	64,096	61,580
Effect of dilutive securities:
Weighted average shares outstanding - diluted (in shares)	66,526	63,802
Net income per share - basic
Net income per share - basic (in dollars per share)	$ 0.01	$ 1.45
Net income per share - diluted
Net income per share - diluted (in dollars per share)	$ 0.01	$ 1.4
Employee Stock Option
Effect of dilutive securities:
Effect of dilutive securities (in shares)	1,381	1,165
Restricted Stock Units (RSUs)
Effect of dilutive securities:
Effect of dilutive securities (in shares)	942	985
Performance-based restricted stock units
Effect of dilutive securities:
Effect of dilutive securities (in shares)	107	72

Stock-based Compensation - Employee Stock Purchase Plan (Details) - Employee Stock - shares	1 Months Ended
	May 31, 2015	Jan. 01, 2025
Stock-based compensation
Number of common shares reserved for future issuance (in shares)	243,347
Annual increase for common stock for issuance (as a percent)	1.00%
Increase of common shares reserved for future issuance (in shares)		637,122

Segment Information - General Information (Details) - segment	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Segment Information
Number of operating segments	1	1
Segment Reporting, CODM, Individual Title and Position or Group Name	srt:ChiefExecutiveOfficerMember	srt:ChiefExecutiveOfficerMember

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Mar. 31, 2025	Dec. 31, 2024
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)	64,563,911	63,712,256
Common Stock, shares outstanding (in shares)	64,563,911	63,712,256

Marketable Securities - Realized Gains (Losses) (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Realized gains (losses) from maturities of debt securities
Realized gains (losses) from maturities of debt securities	$ 0	$ 0

Commitments and Contingencies - Indemnification Agreements (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Indemnification Agreement
Indemnification Agreements
Loss contingency accrual	$ 0	$ 0

Product Revenue and Related Reserves

3 Months Ended

Mar. 31, 2025

Product Revenue and Related Reserves

6. Product Revenue and Related Reserves

The Company generates product revenue from the sales of AYVAKIT/AYVAKYT. The following table summarizes net revenue recognized from product sales for the three months ended March 31, 2025 and 2024 (in thousands):


	Three Months Ended
	March 31,
	2025		2024
United States	$	129,446	$	83,136
Rest of World		19,967		9,389
Total product revenue	$	149,413	$	92,525

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 three months ended March 31, 2025 and 2024 (in thousands):

* 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 March 31, 2025 and December 31, 2024 (in thousands):


	March 31,		December 31,
	2025		2024
Customer 1	28	%	31	%
Customer 2	13	%	11	%
Customer 3	11	%	14	%

The following table summarizes activity in the product revenue allowance and reserve categories for the three months ended March 31, 2025 and 2024 (in thousands):


	Three Months Ended March 31,
	2025		2024
Beginning balance at January 1	$	33,779	$	19,274
Provision related to sales in the current period		28,964		16,926
Adjustment related to prior periods sales		(2,061)		(309)
Credits and payments made		(17,422)		(12,488)
Ending balance at March 31	$	43,260	$	23,403

The total reserves that are included in the Company’s unaudited condensed consolidated balance sheets as of March 31, 2025 and December 31, 2024, are summarized as follows (in thousands):


	March 31,		December 31,
	2025		2024
Reduction of accounts receivable, net	$	2,912	$	2,963
Component of accrued expenses		40,348		30,816
Total revenue-related reserves	$	43,260	$	33,779

Financing Arrangements - Roll Forward (Details) - USD ($) $ in Thousands	3 Months Ended		33 Months Ended
	Mar. 31, 2025	Mar. 31, 2024	Mar. 31, 2025
Sale of Future Royalties and Revenues \| Sixth Street Partners, Future Revenue Purchase Agreement
Long-term Debt
Carrying value, beginning balance	$ 254,436	$ 266,670
Interest expense recognized	6,199	7,096
Payments	(14,003)	(6,875)
Carrying value, ending balance	246,632	266,891	$ 246,632
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities
Long-term Debt
Carrying value, beginning balance	386,970	238,813
Net proceeds received			382,692
Interest expense recognized	11,605	7,977
Payments	(10,829)	(7,405)
Carrying value, ending balance	$ 387,746	$ 239,385	$ 387,746

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

Stock-based Compensation - Unvested Awards (Details)	3 Months Ended
Stock-based Compensation - Unvested Awards (Details)	Mar. 31, 2025 $ / shares shares
Restricted Stock Units (RSUs)
Shares
Unvested at beginning of period (in shares) \| shares	2,290,931
Granted (in shares) \| shares	932,355
Vested (in shares) \| shares	(638,445)
Forfeited (in shares) \| shares	(34,936)
Unvested at end of period (in shares) \| shares	2,549,905
Weighted-Average Grant Date Fair Value
Unvested at beginning or period (in dollars per share) \| $ / shares	$ 75.36
Granted (in dollars per share) \| $ / shares	97.06
Vested (in dollars per share) \| $ / shares	75.03
Forfeited (in dollars per share) \| $ / shares	75.91
Unvested at end of period (in dollars per share) \| $ / shares	$ 83.37
Performance-based restricted stock units
Shares
Unvested at beginning of period (in shares) \| shares	142,500
Granted (in shares) \| shares	152,825
Unvested at end of period (in shares) \| shares	295,325
Weighted-Average Grant Date Fair Value
Unvested at beginning or period (in dollars per share) \| $ / shares	$ 107.65
Granted (in dollars per share) \| $ / shares	136.93
Unvested at end of period (in dollars per share) \| $ / shares	$ 122.8

Marketable Securities - Impairment (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Marketable Securities
Marketable debt securities, charges for credit-related impairments	$ 0	$ 0

Stock-Based Compensation - Stock Plans (Details) - shares	1 Months Ended	3 Months Ended
	Jun. 30, 2022	Mar. 31, 2025	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%
Shares underlying awards outstanding (in shares)		6,983,233
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)		668,819
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)		7,281,467

Segment Information

3 Months Ended

Mar. 31, 2025

Segment Information

16. 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 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 March 31, 2025 and December 31, 2024, the Company’s cash, cash equivalents and marketable securities were $899.8 million and $863.9 million, respectively.

The following tables illustrates information about segment revenue, significant segment expenses and segment operating loss for the three months ended March 31, 2025 and 2024 (in thousands):


	Three Months Ended
	March 31,
	2025		2024
Revenue	$	149,413	$	96,116
Less¹:
Cost of sales		2,802		3,191
Research and development expense²:
Compensation and related expenses		31,790		29,592
Early drug discovery and platform⁵		18,170		14,565
Facilities and IT⁵		12,977		10,580
Clinical and manufacturing related activities⁵		8,251		17,857
Consulting and professional services		7,446		4,294
Other		1,129		428
Total research and development expense²		79,763		77,316
Selling, general and administrative expense³:
Compensation and related expenses		33,852		33,116
Commercial and related expenses⁵		27,053		17,552
Consulting and professional services		9,869		14,500
Facilities and IT		4,834		2,779
Other⁵		3,270		2,260
Total selling, general and administrative expense³		78,878		70,207
Stock-based compensation		29,056		24,225
Other segment items⁴		41,582		167,959
Net income	$	496	$	89,136

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 three months ended March 31, 2025 and 2024 exclude $12.1 million and $10.9 million of stock-based compensation expense, respectively.

Selling, general and administrative expense for the three months ended March 31, 2025 and 2024 exclude $16.9 million and $13.4 million of stock-based compensation expense, respectively.

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

Certain prior period items are recast to conform to the current period presentation.

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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Accrued Expenses (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Accrued Expenses
Research, development and commercial contract costs	$ 32,637	$ 33,957
Employee compensation	21,771	48,725
Accrued professional fees	17,999	14,134
Revenue-related reserves	40,348	30,816
Other	9,300	5,456
Total	$ 122,055	$ 133,088

Condensed 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, 2023	$ 61	$ 2,473,985	$ (3,495)	$ (2,339,942)	$ 130,609
Beginning Balance (in shares) at Dec. 31, 2023	61,147,236
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan	$ 1	17,900			17,901
Issuance of common stock under stock plan (in shares)	902,292
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		24,457			$ 24,457
Other comprehensive income (loss)			(352)		(352)
Net income				89,136	89,136
Ending Balance at Mar. 31, 2024	$ 63	2,565,277	(3,847)	(2,250,806)	310,687
Ending Balance (in shares) at Mar. 31, 2024	62,594,247
Beginning Balance at Dec. 31, 2024	$ 64	2,709,183	(3,551)	(2,407,031)	$ 298,665
Beginning Balance (in shares) at Dec. 31, 2024	63,712,256				63,712,256
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan	$ 1	13,356			$ 13,357
Issuance of common stock under stock plan (in shares)	851,655
At-the-market offerings, net of issuance costs (in shares)					0
Stock-based compensation expense		29,299			$ 29,299
Other comprehensive income (loss)			314		314
Net income				496	496
Ending Balance at Mar. 31, 2025	$ 65	$ 2,751,838	$ (3,237)	$ (2,406,535)	$ 342,131
Ending Balance (in shares) at Mar. 31, 2025	64,563,911				64,563,911

Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Details)	Mar. 31, 2025
Accounting Standards Update 2023-09
Summary of Significant Accounting Policies and Recent Accounting Pronouncements
Change in Accounting Principle, Accounting Standards Update, Adopted	false

Indicates (true false) whether accounting standards update was adopted.

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Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended
Pay vs Performance Disclosure - USD ($) $ in Thousands	Mar. 31, 2025	Mar. 31, 2024
Pay vs Performance Disclosure
Net Income (Loss)	$ 496	$ 89,136

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

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-URI https://asc.fasb.org/1943274/2147482790/220-10-45-1A

Reference 36: http://www.xbrl.org/2003/role/disclosureRef
-Topic 220
-SubTopic 10
-Name Accounting Standards Codification
-Section 45
-Paragraph 1B
-Subparagraph (a)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147482790/220-10-45-1B

Reference 37: http://fasb.org/us-gaap/role/ref/legacyRef
-Topic 942
-SubTopic 220
-Name Accounting Standards Codification
-Section S99
-Paragraph 1
-Subparagraph (SX 210.9-04(22))
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147478524/942-220-S99-1

Product Revenue and Related Reserves - Product Revenue Allowance and Reserve - Balance Sheet Location (Details) - Valuation Allowances and Reserves, Product Revenue - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024	Mar. 31, 2024	Dec. 31, 2023
Product Revenue Allowance and Reserve
Reduction of accounts receivable, net	$ 2,912	$ 2,963
Component of accrued expenses	40,348	30,816
Total revenue-related reserves	$ 43,260	$ 33,779	$ 23,403	$ 19,274

Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 777,539	$ 761,923
U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	190,651	129,785
U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	586,888	632,138
Fair Value, Recurring
Fair Value of Financial Instruments
Total	867,839	831,652
Fair Value, Recurring \| U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	190,651	129,785
Fair Value, Recurring \| U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	586,888	632,138
Fair Value, Recurring \| Money Market Funds
Fair Value of Financial Instruments
Cash equivalents	67,898	69,729
Fair Value, Recurring \| U.S. Treasury obligations
Fair Value of Financial Instruments
Cash equivalents	22,402
Fair Value, Recurring \| Fair Value, Inputs, Level 1
Fair Value of Financial Instruments
Total	677,188	701,867
Fair Value, Recurring \| Fair Value, Inputs, Level 1 \| U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	586,888	632,138
Fair Value, Recurring \| Fair Value, Inputs, Level 1 \| Money Market Funds
Fair Value of Financial Instruments
Cash equivalents	67,898	69,729
Fair Value, Recurring \| Fair Value, Inputs, Level 1 \| U.S. Treasury obligations
Fair Value of Financial Instruments
Cash equivalents	22,402
Fair Value, Recurring \| Fair Value, Inputs, Level 2
Fair Value of Financial Instruments
Total	190,651	129,785
Fair Value, Recurring \| Fair Value, Inputs, Level 2 \| U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 190,651	$ 129,785

Commitments and Contingencies - Purchase Commitments (Details) - Clinical Manufacturing Agreements $ in Millions	3 Months Ended
	Mar. 31, 2025 USD ($)
Clinical and Commercial Supply Agreements
Unrecorded unconditional purchase obligation, term	5 years
Unrecorded unconditional purchase obligation, future minimum purchase obligations	$ 2.0

Marketable Securities

3 Months Ended

Mar. 31, 2025

Sixth Street Partners, Future Revenue Purchase Agreement

Marketable Securities

4. Marketable Securities

Marketable securities consisted of the following at March 31, 2025 and December 31, 2024 (in thousands):


	Amortized		Unrealized		Unrealized		Fair
March 31, 2025	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	190,673	$	91	$	(113)	$	190,651
U.S. treasury obligations		585,897		1,144		(153)		586,888
Total	$	776,570	$	1,235	$	(266)	$	777,539


	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

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 March 31, 2025 and December 31, 2024 (in thousands):


	March 31, 2025				December 31, 2024
	Amortized		Fair		Amortized		Fair
	Cost		value		Cost		value
Within one year	$	453,575	$	453,992	$	512,515	$	513,473
After one through five years		322,995		323,547		248,896		248,450
Total	$	776,570	$	777,539	$	761,411	$	761,923

As of March 31, 2025 and December 31, 2024, the Company held 33 and 32 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 March 31, 2025 and December 31, 2024 by the length of time the security has been in loss position (in thousands):


	March 31, 2025				December 31, 2024
	Fair		Unrealized		Fair		Unrealized
	value		losses		value		losses
Debt securities in unrealized loss position for 12 months or less	$	222,844	$	(266)	$	205,910	$	(631)
Debt securities in unrealized loss position for more than 12 months		—		—		—		—
Total debt securities in unrealized loss position	$	222,844	$	(266)	$	205,910	$	(631)

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 condensed consolidated statements of operations and comprehensive income for the three months ended March 31, 2025 and 2024. The Company did not record any credit-related impairments for its available-for-sale securities for the three months ended March 31, 2025 and 2024.

The following table summarizes the proceeds from maturities of debt securities during the three months ended March 31, 2025 and 2024 (in thousands):

The Company did not realize any gains or losses from maturities of debt securities for the three months ended March 31, 2025 and 2024.

Collaboration, License and Other Agreements - Transaction Price (Details) - Collaborative Arrangement - USD ($) $ in Millions	Jul. 13, 2020	Oct. 15, 2019	Jun. 01, 2018
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
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

Leases - Weighted Average Remaining Lease-term and Weighted Average Discount Rate (Details)	Mar. 31, 2025
Leases
Weighted average remaining lease term in years	4 years 7 months 6 days
Weighted average discount rate	7.30%

Leases - Cash Flow Information (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Leases
Cash paid for amounts included in the measurement of lease liabilities, operating cash flows from operating leases	$ 4,663	$ 4,546

Financing Arrangements (Tables)

3 Months Ended

Mar. 31, 2025

Schedule of activity within the liability account and proceeds received


	Three Months Ended March 31,
	2025		2024
Carrying value as of January 1	$	254,436	$	266,670
Interest expense recognized		6,199		7,096
Payments		(14,003)		(6,875)
Carrying value as of March 31	$	246,632	$	266,891

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

Schedule of activity within the liability account and proceeds received


				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


	Three Months Ended March 31,
	2025		2024
Carrying value as of January 1	$	386,970	$	238,813
Interest expense recognized		11,605		7,977
Payments		(10,829)		(7,405)
Carrying value as of March 31	$	387,746	$	239,385

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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Reference 3: http://www.xbrl.org/2003/role/exampleRef
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Reference 4: http://www.xbrl.org/2009/role/commonPracticeRef
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Material Terms of Trading Arrangement

Insider Trading Arrangements - Fouad Namouni

3 Months Ended

Mar. 31, 2025

Trading Arrangements, by Individual

Name and Title

Action Taken

Type of Trading Arrangement

Nature of Trading Arrangement

Duration of Trading Arrangement

Aggregate Number of Securities

Fouad Namouni

(President, Research and Development)

Adoption

March 11, 2025

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

March 11, 2025 – March 2, 2026

13,944

Name

Fouad Namouni

Title

President, Research and Development

Rule 10b5-1 Arrangement Adopted

true

Adoption Date

March 11, 2025

Expiration Date

March 2, 2026

Aggregate Available

13,944

Collaboration, License and Other Agreements - Sale of Stock (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended
	Jul. 31, 2020	Mar. 31, 2025	Mar. 31, 2024
Sale of Stock
Stock issued (in shares)		0	544,719
Issuance of common stock			$ 48,936
Private Placement
Sale of Stock
Purchase consideration	$ 100,000
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.

Inventory

3 Months Ended

Mar. 31, 2025

Inventory

7. Inventory

Capitalized inventory consists of the following at March 31, 2025 and December 31, 2024 (in thousands):


	March 31,		December 31,
	2025		2024
Work in process	$	35,517	$	30,300
Finished goods		10,020		8,975
Total	$	45,537	$	39,275

Balance sheet classification


	March 31,		December 31,
	2025		2024
Inventory	$	12,953	$	13,611
Other assets		32,584		25,664
Total	$	45,537	$	39,275

Inventory amounts written down as a result of excess, obsolescence, unmarketability or other reasons are charged to cost of sales. The Company did not recognize significant write-downs of inventory during the three months ended March 31, 2025 and 2024. Long-term inventory, which consists of work in process and finished goods, is included in other assets in the unaudited condensed consolidated balance sheets.

Stock-based Compensation - Stock Options (Details)	3 Months Ended
Stock-based Compensation - Stock Options (Details)	Mar. 31, 2025 $ / shares shares
Shares
Outstanding at beginning of period (in shares) \| shares	6,369,724
Granted (in shares) \| shares	795,360
Exercised (in shares) \| shares	(213,210)
Cancelled (in shares) \| shares	(26,578)
Outstanding at end of period (in shares) \| shares	6,925,296
Weighted-Average Exercise Price
Outstanding at beginning of period (in dollars per share) \| $ / shares	$ 72.09
Granted (in dollars per share) \| $ / shares	97.5
Exercised (in dollars per share) \| $ / shares	62.65
Cancelled (in dollars per share) \| $ / shares	74.98
Outstanding at end of period (in dollars per share) \| $ / shares	$ 75.28
Additional disclosures
Shares - Exercisable (in shares) \| shares	4,538,663
Weighted-Average Exercise Price - Exercisable (in dollars per share) \| $ / shares	$ 72.02

Nature of Business (Details) - USD ($) $ in Millions	Mar. 31, 2025	Dec. 31, 2024
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities	$ 899.8	$ 863.9

Marketable Securities - Unrealized Loss Positions - Tabular Disclosure (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Unrealized loss position, aggregate fair value
Unrealized loss position for less than 12 months, aggregate fair value	$ 222,844	$ 205,910
Unrealized loss position, aggregate fair value	222,844	205,910
Unrealized loss position
Unrealized loss position for less than 12 months	(266)	(631)
Total debt securities in unrealized loss position	$ (266)	$ (631)

Income Taxes - Expense (Benefit) (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Income tax expense (benefit)
Income tax expense	$ 789	$ 180

Amount of current income tax expense (benefit) and deferred income tax expense (benefit) pertaining to continuing operations.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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Reference 2: http://www.xbrl.org/2003/role/disclosureRef
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Reference 3: http://www.xbrl.org/2003/role/disclosureRef
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Reference 4: http://www.xbrl.org/2003/role/exampleRef
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Reference 5: http://www.xbrl.org/2003/role/disclosureRef
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Reference 6: http://www.xbrl.org/2003/role/disclosureRef
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Reference 10: http://www.xbrl.org/2003/role/disclosureRef
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Reference 11: http://www.xbrl.org/2003/role/disclosureRef
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Reference 12: http://www.xbrl.org/2003/role/disclosureRef
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Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Cash flows from operating activities
Net income	$ 496	$ 89,136
Adjustments to reconcile net income to net cash used in operating activities:
Depreciation and amortization	4,459	4,810
Non-cash lease expense	2,592	2,404
Stock-based compensation	29,056	24,225
Non-cash interest expense	776	793
Net (accretion of discount) amortization of premium on investments	(3,332)	(5,193)
Non-cash debt extinguishment gain		(173,658)
Equity investment gain	(50,039)
Other	(141)	(827)
Changes in assets and liabilities:
Accounts receivable	(1,397)	(17,339)
Inventory	187	2,696
Prepaid expenses and other current assets	(961)	1,016
Other assets	(8,876)	(4,332)
Accounts payable	(6,044)	313
Accrued expenses	(13,552)	(25,826)
Deferred revenue and other long-term liabilities	(4,500)	3,158
Operating lease liabilities	(3,203)	(2,889)
Net cash used in operating activities	(54,479)	(101,513)
Cash flows from investing activities
Purchases of property and equipment	(1,831)	(1,104)
Purchases of investments	(283,327)	(200,165)
Maturities of investments	271,500	278,400
Proceeds from sale of equity investment	78,737
Net cash provided by investing activities	65,079	77,131
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	13,298	18,396
Principal payments for financing arrangements	(4,106)	(369)
Net cash provided by financing activities	9,192	66,963
Net increase in cash, cash equivalents, and restricted cash	19,792	42,581
Cash, cash equivalents and restricted cash at beginning of period	113,639	81,524
Effect of exchange rate changes on cash, cash equivalents and restricted cash	661	(145)
Cash, cash equivalents and restricted cash at end of period	134,092	123,960
Supplemental cash flow information
Cash paid for interest	21,465	14,280
Property and equipment purchases unpaid at period end	698	1,834
Cash paid for taxes, net	$ 703	$ 520

Stock-based Compensation

3 Months Ended

Mar. 31, 2025

Stock-based Compensation

12. 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.

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 March 31, 2025, there were 6,983,233 and 668,819 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 March 31, 2025, there were 7,281,467 shares available for future grant under the 2024 Plan.

2015 Employee Stock Purchase Plan

In 2015, the Company’s board of directors and stockholders approved the 2015 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 year beginning January 1, 2025, the number of shares reserved for issuance under the 2015 ESPP was increased by 637,122 shares.

Stock options

The following table summarizes the stock option activity for the three months ended March 31, 2025:


		Weighted-Average
	Shares	Exercise Price
Outstanding at December 31, 2024	6,369,724	$	72.09
Granted	795,360		97.50
Exercised	(213,210)		62.65
Canceled	(26,578)		74.98
Outstanding at March 31, 2025	6,925,296	$	75.28
Exercisable at March 31, 2025	4,538,663	$	72.02

As of March 31, 2025, the total unrecognized compensation expense related to unvested stock option awards was $102.6 million, which is expected to be recognized over a weighted-average period of approximately 2.8 years.

Restricted stock units

The following table summarizes the restricted stock units activity for the three months ended March 31, 2025:


		Weighted-Average
	Shares	Grant Date Fair Value
Unvested shares at December 31, 2024	2,290,931	$	75.36
Granted	932,355		97.06
Vested	(638,445)		75.03
Forfeited	(34,936)		75.91
Unvested shares at March 31, 2025	2,549,905	$	83.37

As of March 31, 2025, the total unrecognized compensation expense related to unvested restricted stock units was $197.4 million, which is expected to be recognized over a weighted-average period of approximately 2.9 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 the three months ended March 31, 2025:


		Weighted-Average
		Grant Date
	Shares	Grant Date Fair Value
Unvested shares at December 31, 2024	142,500	$	107.65
Granted	152,825		136.93
Vested	—		—
Forfeited	—		—
Unvested shares at March 31, 2025	295,325	$	122.80

As of March 31, 2025, the total unrecognized compensation expense related to unvested PSUs was $29.1 million, which is expected to be recognized over a weighted-average period of approximately 2.26 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.

Stock-based compensation expense

The Company recognized stock-based compensation expense of $29.1 million and $24.2 million for the three months ended March 31, 2025 and 2024, respectively. Stock-based compensation expense by award type included within the unaudited condensed consolidated statements of operations and comprehensive income was as follows (in thousands):


	Three Months Ended
		March 31,
		2025		2024
Stock options	$	10,268	$	9,573
Restricted stock units		16,827		13,836
Performance-based restricted stock units		1,844		594
Employee stock purchase plan		360		454
Subtotal		29,299		24,457
Capitalized stock-based compensation costs		(243)		(232)
Stock-based compensation expense included in total cost and operating expenses	$	29,056	$	24,225

Stock-based compensation expense, that is included in operating expenses, by classification within the unaudited condensed consolidated statements of operations and comprehensive income was as follows (in thousands):


	Three Months Ended
	March 31,
	2025		2024
Research and development	$	12,127	$	10,875
Selling, general and administrative		16,929		13,350
Total	$	29,056	$	24,225

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Current assets:
Cash and cash equivalents	$ 122,245	$ 102,014
Marketable securities	453,992	513,473
Accounts receivable	78,083	75,797
Unbilled accounts receivable	1,718	1,812
Inventory	12,953	13,611
Prepaid expenses and other current assets	33,078	35,971
Total current assets	702,069	742,678
Marketable securities	323,547	248,450
Property and equipment, net	36,050	36,593
Operating lease right-of-use assets, net	61,607	64,181
Restricted cash	11,847	11,625
Equity investment		28,699
Other assets	60,484	47,587
Total assets	1,195,604	1,179,813
Current liabilities:
Accounts payable	800	6,790
Accrued expenses	122,055	133,088
Current portion of operating lease liabilities	13,736	13,346
Current portion of deferred revenue	2,279	2,005
Current portion of liabilities related to the sale of future royalties and revenues	67,917	61,650
Current portion of term loan	43,797	43,917
Total current liabilities	250,584	260,796
Operating lease liabilities, net of current portion	65,216	68,790
Deferred revenue, net of current portion	8,037	8,193
Liabilities related to the sale of future royalties and revenues, net of current portion	178,715	193,524
Term loan, net of current portion	343,949	343,053
Other long-term liabilities	6,972	6,792
Total liabilities	853,473	881,148
Commitments and Contingencies (Note 17)
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; 64,563,911 and 63,712,256 shares issued and outstanding at March 31, 2025 and December 31, 2024, respectively	65	64
Additional paid-in capital	2,751,838	2,709,183
Accumulated other comprehensive loss	(3,237)	(3,551)
Accumulated deficit	(2,406,535)	(2,407,031)
Total stockholders' equity	342,131	298,665
Total liabilities and stockholders' equity	$ 1,195,604	$ 1,179,813

Restricted Cash (Tables)

3 Months Ended

Mar. 31, 2025

Reconciliation of cash, cash equivalents, and restricted cash


	March 31,		March 31,
	2025		2024
Cash and cash equivalents	$	122,245	$	113,326
Restricted cash		11,847		10,634
Total cash, cash equivalents, and restricted cash shown in condensed consolidated statements of cash flows	$	134,092	$	123,960

Collaboration, License and Other Agreements - Contract Assets and Liabilities (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Contract Assets
Accounts receivable, net	$ 78,083	$ 75,797
Unbilled accounts receivable	1,718	1,812
Prepaid and Other Assets
Prepaid and other assets, current	33,078	35,971
Contract Liabilities
Contract liabilities, current	2,279	2,005
Contract liabilities, noncurrent	8,037	8,193
Accrued expenses, current	32,637	33,957
Collaborative Arrangement \| VantAI
Prepaid and Other Assets
Prepaid and other assets, current	5,030	4,971
Prepaid and other assets, noncurrent	5,816	2,666
Prepaid and other assets	10,846	7,637
Collaborative Arrangement \| Roche, Collaboration (Pralsetnib) Agreement
Contract Liabilities
Accrued expenses	365	1,712
Collaborative Arrangement \| C Stone
Contract Liabilities
Accrued expenses	548	2,027
Arrangement Other than Collaborative, Asset Purchase Agreement \| Rigel Pharmaceuticals, Inc.
Contract Assets
Contract assets, current	2,915	2,939
Contract assets, noncurrent	6,602	7,380
Contract assets	9,517	10,319
Contract Liabilities
Contract liabilities, noncurrent	3,562	3,562
Contract liabilities	$ 3,562	$ 3,562

Segment Information - Segment Revenue, Significant Segment Expenses and Segment Operating Loss (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Segment Information
Revenue	$ 149,413	$ 96,116
Cost of sales	2,802	3,191
Research and development expense, consulting and professional services	7,446	4,294
Research and development expense	91,890	88,191
Selling, general and administrative expense, facilities and IT	4,834	2,779
Selling, general, and administrative expenses	95,807	83,557
Stock-based compensation	29,056	24,225
Net Income (Loss)	496	89,136
Single Reportable Segment
Segment Information
Revenue	149,413	96,116
Cost of sales	2,802	3,191
Other segment items	$ 41,582	$ 167,959
Segment Reporting, Other Segment Item, Composition, Description	Other segment items include interest expense, net, other income, net, equity investment gain, debt extinguishment gain, and income tax expense.	Other segment items include interest expense, net, other income, net, equity investment gain, debt extinguishment gain, and income tax expense.
Operating Segments \| Single Reportable Segment
Segment Information
Research and development expense, compensation and related expenses	$ 31,790	$ 29,592
Research and development expense, early drug discovery and platform	18,170	14,565
Research and development expense, facilities and IT	12,977	10,580
Research and development expense, clinical and manufacturing related activities	8,251	17,857
Research and development expense, other	1,129	428
Research and development expense	79,763	77,316
Selling, general and administrative expense, compensation and related expenses	33,852	33,116
Selling, general and administrative expense, commercial and related expenses	27,053	17,552
Selling, general and administrative expense, consulting and professional services	9,869	14,500
Selling, general and administrative expense, other	3,270	2,260
Selling, general, and administrative expenses	78,878	70,207
Segment Reporting, Reconciling Item, Corporate Nonsegment
Segment Information
Research and development expense	12,100	10,900
Selling, general, and administrative expenses	$ 16,900	$ 13,400

Marketable Securities - Tabular Disclosure (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Marketable securities, available-for-sale:
Amortized cost	$ 776,570	$ 761,411
Unrealized gain	1,235	1,143
Unrealized losses	(266)	(631)
Fair value	777,539	761,923
U.S. government agency securities
Marketable securities, available-for-sale:
Amortized cost	190,673	129,897
Unrealized gain	91	118
Unrealized losses	(113)	(230)
Fair value	190,651	129,785
U.S. Treasury obligations
Marketable securities, available-for-sale:
Amortized cost	585,897	631,514
Unrealized gain	1,144	1,025
Unrealized losses	(153)	(401)
Fair value	$ 586,888	$ 632,138

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

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Nature of Business

3 Months Ended

Mar. 31, 2025

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.

As of March 31, 2025, the Company had cash, cash equivalents and marketable securities of $899.8 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.

Marketable Securities - Unrealized Loss Positions - General Information (Details) - security	Mar. 31, 2025	Dec. 31, 2024
Unrealized loss position, number of positions
Number of held securities in an unrealized loss position	33	32

Segment Information - Assessing Segment Performance (Details) - USD ($) $ in Millions	Mar. 31, 2025	Dec. 31, 2024
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities	$ 899.8	$ 863.9

Leases (Tables)

3 Months Ended

Mar. 31, 2025

Summary of lease expenses and cash flow and weighted average information


	Three Months Ended
	March 31,
	2025		2024
Cash paid for amounts included in the measurement of lease liabilities:	$	4,663	$	4,546
Lease liabilities arising from obtaining right-of-use assets:
Operating leases	$	—	$	—

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

Marketable Securities (Tables)

3 Months Ended

Mar. 31, 2025

Schedule of marketable securities


	Amortized		Unrealized		Unrealized		Fair
March 31, 2025	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	190,673	$	91	$	(113)	$	190,651
U.S. treasury obligations		585,897		1,144		(153)		586,888
Total	$	776,570	$	1,235	$	(266)	$	777,539


	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

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


	March 31, 2025				December 31, 2024
	Amortized		Fair		Amortized		Fair
	Cost		value		Cost		value
Within one year	$	453,575	$	453,992	$	512,515	$	513,473
After one through five years		322,995		323,547		248,896		248,450
Total	$	776,570	$	777,539	$	761,411	$	761,923

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


	March 31, 2025				December 31, 2024
	Fair		Unrealized		Fair		Unrealized
	value		losses		value		losses
Debt securities in unrealized loss position for 12 months or less	$	222,844	$	(266)	$	205,910	$	(631)
Debt securities in unrealized loss position for more than 12 months		—		—		—		—
Total debt securities in unrealized loss position	$	222,844	$	(266)	$	205,910	$	(631)

Summary of proceeds from maturities of debt securities

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
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Stockholders' Equity

3 Months Ended

Mar. 31, 2025

Stockholders' Equity

11. Stockholders’ Equity

In February 2022, the Company entered into an at-the-market (ATM) facility (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. The Company did not issue any shares under the ATM Facility during the three months ended March 31, 2025. During the three months ended March 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 entire disclosure for equity.

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-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

Reference 18: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 18
-Subparagraph (b)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

Reference 19: http://www.xbrl.org/2003/role/disclosureRef
-Topic 505
-SubTopic 10
-Name Accounting Standards Codification
-Section 50
-Paragraph 18
-Subparagraph (d)
-Publisher FASB
-URI https://asc.fasb.org/1943274/2147481112/505-10-50-18

Collaboration, License and Other Agreements

3 Months Ended

Mar. 31, 2025

Collaboration, License and Other Agreements

10. 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. The transaction price allocated to the U.S. rights to GAVRETO and GAVRETO product was recognized as revenue of $24.3 million in the second quarter of 2024. 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 condensed consolidated financial statements as of March 31, 2025. During the three months ended March 31, 2025, insignificant revenue was recognized under the Rigel agreements.

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 March 31, 2025 and December 31, 2024 (in thousands):


	March 31, 2025						December 31, 2024
	Current		Noncurrent		Total		Current		Noncurrent		Total
Contract assets	$	2,915	$	6,602	$	9,517	$	2,939	$	7,380	$	10,319
Contract liabilities	$	—	$	3,562	$	3,562	$	—	$	3,562	$	3,562

IDRx

In August 2022, the Company entered into a license agreement with IDRx, Inc. (IDRx), granting 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. In July 2023, the Company received 192,282 additional shares under an anti-dilution provision under the IDRx Stock Purchase Agreement and as of December 31, 2024, the Company owned a total of 4,701,387 shares of IDRx’s Series A preferred stock.

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, the Company is eligible to receive 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 10th 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 combined the IDRx License Agreement and the IDRx Stock Purchase Agreement into a single contract under ASC 606. Therefore, the Company determined that the shares of IDRx’s Series A preferred stock should be attributed to the transaction price of the IDRx License Agreement. The transaction price of the IDRx License Agreement at the contract inception was determined to be $27.5 million, which was based on the fair value derived from IDRx’s then-most recent financing transaction with unrelated investors. The Company identified two material promises in the IDRx License Agreement, which were the exclusive license and the initial know-how transfer, which were combined into one distinct performance obligation. The Company concluded that the license is a functional intellectual property license and that IDRx benefited from the license along with the initial know-how transfer at the time of grant, and therefore the related performance obligation was satisfied at a point in time and the revenue for the transaction price of $27.5 million was recorded in 2022.

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, revenue from any sales milestones and royalties shall be recognized when the related sales occur.

The Company accounted for the preferred stock investment as an equity investment under the measurement alternative for equity investments without a readily determinable fair value, at cost of $27.8 million including transaction costs of $0.3 million in 2022. Subsequently, in August 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.

During the three months ended March 31, 2025, GSK plc (GSK) acquired IDRx for an upfront cash consideration of $1.0 billion with an additional $150.0 million contingent consideration. The Company received $78.7 million in cash from this transaction, which resulted in a net investment gain of $50.0 million. Consequently, the Company derecognized its investment in IDRx on its unaudited condensed consolidated balance sheets. Any additional contingent consideration received related to this acquisition will be recognized as an investment gain once it is realized.

The acquisition does not affect the Company's rights related to the license agreement. The Company will continue to be eligible to receive contingent cash payments under the IDRx License Agreement as outlined above. No revenue was recorded under the IDRx License Agreement during the three months ended March 31, 2025 and 2024.

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 a total of $1.67 billion in contingent payments including specified research, development, regulatory and commercialization milestones for all the target programs. As of March 31, 2025, VantAI has achieved $8.5 million of such contingent payments. 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 three months ended March 31, 2025 and 2024, the Company recorded research and development expense of $1.8 million and $2.1 million, respectively, under the A&R Agreement.

The following table summarizes the assets associated with the A&R Agreement as of March 31, 2025 and December 31, 2024 (in thousands):


	March 31, 2025						December 31, 2024
	Current		Noncurrent		Total		Current		Noncurrent		Total
Prepaid and other assets	$	5,030	$	5,816	$	10,846	$	4,971	$	2,666	$	7,637

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 12^th 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. No reduction of expenses was recorded under the Zai Lab agreement during the three months ended March 31, 2025 and 2024.

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 milestone 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. No revenue was recorded under the Zai Lab agreement during the three months ended March 31, 2025 and 2024.

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), granting 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 worldwide commercialization and development rights to GAVRETO 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, when the Company and Roche entered into a transition agreement (the Roche transition 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 shares of the Company’s common stock at a purchase price of $100.0 million, with a premium of $20.7 million 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 pralsetinib license was deemed a functional intellectual property license and a distinct performance obligation, satisfied at the time of grant. The initial transaction price of the Roche pralsetinib collaboration agreement at the outset of the arrangement was determined to be $695.7 million, including the $675.0 million upfront cash payment 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 have been recorded under the Roche pralsetinib collaboration since June 30, 2024. During the three months ended March 31, 2024, the Company recorded a $1.0 million reduction to selling, general and administrative expenses in connection with the commercialization of GAVRETO in the U.S and a $1.4 million increase in research and development expenses related to global development activities for pralsetinib under the Roche pralsetinib collaboration.

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 income. No collaboration revenue has been recorded under the Roche pralsetinib collaboration since June 30, 2024. During the three months ended March 31, 2024, the Company recorded revenue of $1.4 million derived from profit sharing on Roche sales of GAVRETO in the U.S. and $0.5 million revenue related to Roche Territory specific activities and royalties earned during the three months ended March 31, 2024.

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 three months ended March 31, 2025, the Company recorded $0.4 million in such costs. Such costs were insignificant during the three months ended March 31, 2024.

The following table summarizes the Company’s liabilities associated with the Roche transition agreement as of March 31, 2025 and December 31, 2024 (in thousands):


	March 31,		December 31,
	2025		2024
Accrued expenses	$	365	$	1,712

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 March 31, 2025, 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 March 31, 2025, 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.

No revenue was recorded under the Clementia Agreement during the three months ended March 31, 2025 and 2024. 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.

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 March 31, 2025, 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 March 31, 2025, 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 three months ended March 31, 2025 and 2024.

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 three months ended March 31, 2025 and 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.

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 three months ended March 31, 2025 and 2024 is as follows (in thousands):

The following table presents the contract liabilities associated with the CStone collaboration as of March 31, 2025 and December 31, 2024 (in thousands):


	March 31,		December 31,
	2025		2024
Accrued expenses	$	548	$	2,027

The Company’s liabilities associated with the CStone collaboration as of March 31, 2025 primarily related to payment associated with the Company’s manufacturing services and royalty revenue related to the CStone territory-specific activities. The Company’s liabilities as of December 31, 2024 resulted from the Company’s obligations in connection with commercial supply of pralsetinib for the CStone Territory.

Condensed Consolidated Statements of Operations and Comprehensive Income - USD ($) shares in Thousands, $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Revenues:
Revenue	$ 149,413	$ 96,116
Cost and operating expenses:
Cost of sales	2,802	3,191
Research and development	91,890	88,191
Selling, general and administrative	95,807	83,557
Total cost and operating expenses	190,499	174,939
Other income (expense):
Interest expense, net	(8,129)	(5,895)
Other income, net	461	376
Equity investment gain	50,039
Debt extinguishment gain		173,658
Total other income, net	42,371	168,139
Income before income taxes	1,285	89,316
Income tax expense	789	180
Net income	496	89,136
Other comprehensive income (loss):
Unrealized gain (loss) on available-for-sale investments	457	(564)
Currency translation adjustments	(143)	212
Comprehensive income	$ 810	$ 88,784
Net income per share - basic (in dollars per share)	$ 0.01	$ 1.45
Net income per share - diluted (in dollars per share)	$ 0.01	$ 1.4
Weighted-average number of common shares used in net income per share - basic (in shares)	64,096	61,580
Weighted-average number of common shares used in net income per share - diluted (in shares)	66,526	63,802
Product
Revenues:
Revenue	$ 149,413	$ 92,525
Collaboration, License and Other
Revenues:
Revenue		$ 3,591

Financing Arrangements - Net Proceeds Received (Details) - Secured Debt - USD ($) $ in Thousands	1 Months Ended			33 Months Ended
	May 31, 2024	Aug. 31, 2023	Jul. 31, 2022	Mar. 31, 2025
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				$ 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

Collaboration, License and Other Agreements - Reduction in Expenses (Details) - Collaborative Arrangement - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Mar. 31, 2025	Mar. 31, 2024	Mar. 31, 2025
Zai Lab (Shanghai) Co., Ltd.
Collaboration, License and Other Agreements
Collaborative arrangement, global development activities, increase (reduction) in research and development expenses	$ 0	$ 0
Roche, Collaboration (Pralsetnib) Agreement
Collaboration, License and Other Agreements
Collaborative arrangement, commercialization, reduction in selling, general and administrative expenses		1,000	$ 0
Collaborative arrangement, global development activities, increase (reduction) in research and development expenses		$ 1,400	$ 0

Product Revenue and Related Reserves - Product Revenue (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Revenues
Revenue	$ 149,413	$ 96,116
Product
Revenues
Revenue	149,413	92,525
Product \| United States
Revenues
Revenue	129,446	83,136
Product \| Rest of World
Revenues
Revenue	$ 19,967	$ 9,389

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.

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Inventory - Capitalized (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Inventory
Work in process	$ 35,517	$ 30,300
Finished goods	10,020	8,975
Total	$ 45,537	$ 39,275

Product Revenue and Related Reserves - Concentration Risk (Details)	3 Months Ended		12 Months Ended
	Mar. 31, 2025	Mar. 31, 2024	Dec. 31, 2024
Revenue from Contract with Customer Benchmark \| Customer Concentration Risk \| Customer One
Concentration Risk
Concentration risk (as a percent)	38.00%	39.00%
Revenue from Contract with Customer Benchmark \| Customer Concentration Risk \| Customer Two
Concentration Risk
Concentration risk (as a percent)	15.00%
Accounts Receivable \| Credit Concentration Risk \| Customer One
Concentration Risk
Concentration risk (as a percent)	28.00%		31.00%
Accounts Receivable \| Credit Concentration Risk \| Customer Two
Concentration Risk
Concentration risk (as a percent)	13.00%		11.00%
Accounts Receivable \| Credit Concentration Risk \| Customer Three
Concentration Risk
Concentration risk (as a percent)	11.00%		14.00%

Collaboration, License and Other Agreements - General Information (Details) $ in Thousands			1 Months Ended		2 Months Ended	3 Months Ended			13 Months Ended	63 Months Ended
	Oct. 15, 2019 USD ($)	Jun. 01, 2018 USD ($) item	Feb. 28, 2022 USD ($)	Oct. 31, 2019 USD ($)	Dec. 31, 2021 USD ($)	Mar. 31, 2025 USD ($) item	Mar. 31, 2024 USD ($)	Sep. 30, 2020 USD ($)	Mar. 31, 2025 USD ($) item	Mar. 31, 2025 USD ($) item	Dec. 31, 2024 USD ($)	Feb. 22, 2024 USD ($)	Nov. 08, 2021 USD ($)	Jul. 13, 2020
Collaboration, License and Other Agreements
Prepaid expenses and other current assets						$ 33,078			$ 33,078	$ 33,078	$ 35,971
Selling, general, and administrative expenses						95,807	$ 83,557
Research and development expense						91,890	88,191
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, upfront payment			$ 20,000
Collaborative arrangement, milestones, potential contingent payments						$ 1,670,000			1,670,000	1,670,000
Collaborative arrangement, milestones, contingent payments achieved						8,500			8,500	8,500
Prepaid expenses and other current assets						5,030			$ 5,030	$ 5,030	$ 4,971
Research and development expense						$ 1,800	$ 2,100
Collaborative arrangement, jointly research and advance novel protein degrader therapies into development candidates, number \| item						3			3	3
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%
Collaborative arrangement, selling, general, and administrative expenses, wind-down costs and net transition costs reimbursable						$ 400
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, contingent payments eligible to receive		$ 307,500
Collaborative arrangement, milestones, achieved						$ 38,500			$ 38,500	$ 38,500
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

Summary of Significant Accounting Policies and Recent Accounting Pronouncements

3 Months Ended

Mar. 31, 2025

Summary of Significant Accounting Policies and Recent Accounting Pronouncements

2. Summary of Significant Accounting Policies and Recent Accounting Pronouncements

Basis of Presentation

The unaudited interim condensed 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), Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB) and the rules and regulations of the Securities and Exchange Commission (SEC). Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these financial statements should be read in conjunction with the financial statements as of and for the year ended December 31, 2024 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024, filed with the SEC on February 13, 2025 (2024 Annual Report on Form 10-K).

The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited financial statements, and updated, as necessary, in this report. In the opinion of the Company’s management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments that are necessary to present fairly the Company’s financial position as of March 31, 2025, the results of its operations for the three months ended March 31, 2025 and 2024, stockholders’ equity for the three months ended March 31, 2025 and 2024 and cash flows for the three months ended March 31, 2025 and 2024. Such adjustments are of a normal and recurring nature. The results for the three months ended March 31, 2025 are not necessarily indicative of the results for the year ending December 31, 2025 or for any other future period.

The accompanying unaudited interim condensed 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

The significant accounting policies used in preparation of these condensed consolidated financial statements for the three months ended March 31, 2025 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2024 Annual Report on Form 10-K.

Recent 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 condensed consolidated financial statements and disclosures.

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 annual reporting periods beginning after December 15, 2024, with early adoption permitted. The Company will adopt the new disclosure requirements in its 2025 Annual Report on Form 10-K.

In November 2024, the FASB issued ASU 2024-03, Disaggregation of Income Statement Expenses, which is intended to improve disclosures by requiring additional information about specific expense categories in the notes to the financial statements on an annual and interim basis. The standard will be effective for annual reporting periods beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027, with early adoption permitted. The standard updates may be applied on either a prospective or retrospective basis. The Company is currently evaluating the disclosure requirements related to this new standard.

Reclassification

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

Commitments and Contingencies

3 Months Ended

Mar. 31, 2025

Commitments and Contingencies

17. 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 March 31, 2025.

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 March 31, 2025, 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, and a fourteenth lawsuit was consolidated on April 7, 2025, all 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 lawsuit 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 claimed 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 brought 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 sought a declaration that certain actions taken in connection with the Company’s annual meeting of stockholders were 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 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 declared valid and effective as of the date of the 2024 annual meeting. As a result, on March 17, 2025, the Taylor Action was dismissed as moot, with the court retaining jurisdiction to determine plaintiff’s counsel’s application for an award of attorneys’ fees and reimbursement of expenses.

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 condensed consolidated financial statements as of March 31, 2025 or December 31, 2024.

Stock-based Compensation - Stock-based Compensation Expense - By Classification (Details) - USD ($) $ in Thousands	3 Months Ended
	Mar. 31, 2025	Mar. 31, 2024
Total stock based compensation expense
Stock-based compensation expense	$ 29,056	$ 24,225
Research and Development Expense
Total stock based compensation expense
Stock-based compensation expense	12,127	10,875
General and Administrative Expense
Total stock based compensation expense
Stock-based compensation expense	$ 16,929	$ 13,350

Restricted Cash

3 Months Ended

Mar. 31, 2025

Restricted Cash

8. Restricted Cash

The following table provides a reconciliation of cash, cash equivalents, and restricted cash as reported within the Company's condensed consolidated balance sheets to the total of such amounts shown in the condensed consolidated statements of cash flows for the three months ended March 31, 2025 and 2024 (in thousands):


	March 31,		March 31,
	2025		2024
Cash and cash equivalents	$	122,245	$	113,326
Restricted cash		11,847		10,634
Total cash, cash equivalents, and restricted cash shown in condensed consolidated statements of cash flows	$	134,092	$	123,960

As of March 31, 2025 and December 31, 2024, $11.8 million and $11.6 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 15, Leases.

Leases

3 Months Ended

Mar. 31, 2025

Leases

15. 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 March 31, 2025. The lease agreements do not contain residual value guarantees.

Lease costs for the three months ended March 31, 2025 and 2024 were as follows (in thousands):

The Company has not entered into any material short-term leases or financing leases as of March 31, 2025.

Supplemental cash flow information related to leases for the three months ended March 31, 2025 and 2024 was as follows (in thousands):


	Three Months Ended
	March 31,
	2025		2024
Cash paid for amounts included in the measurement of lease liabilities:	$	4,663	$	4,546
Lease liabilities arising from obtaining right-of-use assets:
Operating leases	$	—	$	—

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

Collaboration, License and Other Agreements - Performance Obligations (Details)	1 Months Ended	3 Months Ended
	Jul. 31, 2020 item	Mar. 31, 2025 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 \| 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

Marketable Securities - Maturities (Details) - USD ($) $ in Thousands	Mar. 31, 2025	Dec. 31, 2024
Amortized cost
Within one year, amortized cost	$ 453,575	$ 512,515
After one through five years, amortized cost	322,995	248,896
Amortized cost	776,570	761,411
Fair value
Within one year, fair value	453,992	513,473
After one through five years, fair value	323,547	248,450
Investments, available-for-sale	$ 777,539	$ 761,923

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