0001558370-23-001465 | 10-K | XBRL Viewer

Nature of Business

1. Nature of Business

Blueprint Medicines Corporation (the Company), a Delaware corporation incorporated on October 14, 2008, is a precision therapy company focused on genomically defined cancers and blood disorders. The Company’s approach is to leverage its novel research engine to systematically and reproducibly identify drivers of diseases in genomically defined patient populations, and to craft highly selective and potent drug candidates that are intended to provide significant and durable clinical responses to patients.

The Company has two approved precision therapies and is globally advancing multiple programs for systemic mastocytosis (SM), lung cancer, breast cancer and other genomically defined cancers, and cancer immunotherapy. The Company is devoting substantially all of its efforts to research and development for current and future drug candidates and commercialization of AYVAKIT/AYVAKYT, GAVRETO and any current or future drug candidates that obtain marketing approval.

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

As of December 31, 2022, the Company had cash, cash equivalents and marketable securities of $1,078.5 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.

Summary of Significant Accounting Policies and Recent Accounting Pronouncements

12 Months Ended

Dec. 31, 2022

Summary of Significant Accounting Policies and Recent Accounting Pronouncements

2. Summary of Significant Accounting Policies and Recent Accounting Pronouncements

Basis of Presentation

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

The accompanying consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, Blueprint Medicines Security Corporation, which is a Massachusetts subsidiary created to buy, sell and hold securities, Blueprint Medicines (Switzerland) GmbH, Blueprint Medicines (Netherlands) B.V., Blueprint Medicines (UK) Ltd, Blueprint Medicines (Germany) GmbH, Blueprint Medicines (Spain) S.L., Blueprint Medicines (France) SAS, Blueprint Medicines (Italy) S.r.L. Lengo Therapeutics Inc (Lengo), which was acquired on December 30, 2021, was dissolved in June 2022. 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, and income taxes. Actual results may differ from these estimates. The Russian invasion of Ukraine has not had a material impact on the Company’s business, results of operations and financial condition.

Significant Accounting Policies

Revenue Recognition

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

Product revenue

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

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

Chargebacks: Chargebacks for fees and discounts represent the estimated obligations resulting from contractual commitments to sell product to qualified healthcare providers and government agencies at prices lower than the list prices charged to the customers who directly purchase the product from the Company. The customers charge the Company for the difference between what they pay for the product and the ultimate contractually committed or government required lower selling price to the qualified healthcare providers. These reserves are estimated using the expected value method based upon a range of possible outcomes and are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue.

Government rebates: Government rebates consist of Medicare, Tricare and Medicaid rebates, which were estimated using the expected value method, based upon a range of possible outcomes for the estimated payor mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue.

For Medicare, the Company also estimates the number of patients in the prescription drug coverage gap for whom it will owe a rebate under the Medicare Part D program.

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

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

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

Collaboration and license revenue

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

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

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

Exclusive Licenses. If the license to the Company’s intellectual property is determined to be distinct from the other promises or performance obligations identified in the arrangement, the Company recognizes revenue from non-

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

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

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

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

Royalties. For arrangements that include sales-based royalties, including milestone payments upon first commercial sales and milestone payments based on a level of sales, which are the result of a customer-vendor relationship and for which the license is deemed to be the predominant item to which the royalties relate, the Company recognizes revenue at the later of (i) when the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied or partially satisfied. To date, the Company has not recognized any royalty revenue resulting from any of its licensing arrangements.

Consideration received prior to revenue recognition is recorded as deferred revenue in the consolidated balance sheets. Amounts expected to be recognized as revenue within the 12 months following the balance sheet date are classified as current portion of deferred revenue in the accompanying consolidated balance sheets. Amounts not expected to be recognized as revenue within the 12 months following the balance sheet date are classified as deferred

revenue, net of current portion. If the Company transfer goods or services to a customer before the customer pays consideration or before payment is due, the Company records a contract asset as unbilled accounts receivable on the consolidated balance sheets.

For a complete discussion of accounting for collaboration and license revenues, see Note 12, Collaboration and License Agreements.

Accounts Receivable, net

Accounts receivable arise from product sales and amounts due from the Company’s collaboration partners. The amount from product sales represents amounts due from specialty distributors and specialty pharmacy providers in the U.S. and in the European Union. The Company monitors economic conditions and the financial performance and credit worthiness of its counterparties to identify facts or circumstances that may indicate that its receivables are at risk of collection. The Company provides reserves against accounts receivable for estimated losses that may result from a customer’s inability to pay based on the composition of its accounts receivable, considering past events, current economic conditions, and reasonable and supportable forecasts about the future economic conditions. The contractual life of our accounts receivable is generally short-term. Amounts determined to be uncollectible are charged or written-off against the reserve. For the years ended December 31, 2022, 2021 and 2020, the Company did not record any expected credit losses related to outstanding accounts receivable.

Inventory

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

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

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

Fair Value Measurements

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

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

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

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

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

There have been no changes to the valuation methods during the years ended December 31, 2022 and 2021.

Cash and cash equivalents

The Company considers all highly liquid investments purchased with original maturities of 90 days or less from the date of purchase to be cash equivalents. As of December 31, 2022 and 2021, the Company’s cash equivalents comprised of money market funds with less than 90 days from the date of purchase. Cash equivalents are reported at fair value.

Available-for-Sale Investments

The Company classifies marketable debt securities with a remaining maturity when purchased of greater than three months available-for-sale, and marketable debt securities with a remaining maturity date greater than one year as non-current assets. Available-for-sale marketable debt securities are maintained by an investment manager and mainly consist of U.S. treasury securities and U.S. government agency securities. Available-for-sale marketable debt securities are carried at fair value with the unrealized gains and losses included in other comprehensive income (loss) as a component of stockholders’ equity until realized. Any premium or discount arising at purchase is amortized and/or accreted to interest income and/or expense over the life of the instrument. Realized gains and losses are determined using the specific identification method and are included in other income (expense). The Company reviews its portfolio of available-for-sale debt securities, using both quantitative and qualitative factors, to determine if declines in fair value below cost have resulted from a credit-related loss or other factors. If the decline in fair value is due to credit-related factors, a loss is recognized in net income, whereas if the decline in fair value is not due to credit-related factors, the loss is recorded in other comprehensive income (loss).

Comprehensive Income (Loss)

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

Research and Development Expenses

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

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

Selling, General and Administrative Expenses

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

expensed as incurred. For years ended December 31, 2022, 2021 and 2020, advertising costs totaled $18.1 million, $13.5 million and $9.4 million, respectively.

Property and Equipment, Net

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

Impairment of Long-Lived Assets

The Company continually evaluates whether events or circumstances have occurred that indicate that the estimated remaining useful life of its long-lived assets may warrant revision or that the carrying value of these assets may be impaired. The Company has not recognized any impairment charges associated with long-lived assets for the years ended December 31, 2022, 2021 and 2020.

Leases

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

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

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

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

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

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

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

Stock-Based Compensation Expense

Stock-based compensation awards are accounted for in accordance with ASC Topic 718, Compensation –Stock Compensation (ASC 718). The Company expenses the fair value of stock awards granted to employees and members of the board of directors over the requisite service period, which is typically the vesting period. Compensation cost for stock-based awards issued to employees is measured using the estimated fair value at the grant date and is adjusted to reflect actual forfeitures. Fair value of options granted to employees at the date of grant are estimated using the Black-Scholes option-pricing model that requires management to apply judgment and make estimates, including:

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expected volatility, which was calculated using the historical volatility of the Company’s publicly traded stock for awards granted during the year ended December 31, 2022. In prior reporting periods, expected volatility was calculated based on a blend of the Company’s reported volatility data for the length of time that market data is available for the Company’s stock and the historical data for a representative group of publicly traded companies, for which historical information is available. For these analyses, the Company selected companies with comparable characteristics to itself including enterprise value, risk profiles, position within the industry, and with historical share price information sufficient to meet the expected life of the stock-based awards. The Company computed the historical volatility data using the daily closing prices during the equivalent period of the calculated expected term of its stock-based awards;

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

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

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

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

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

Equity Investment

Investments in non-marketable equity securities of privately-held companies that do not have readily determinable fair values are carried at cost, less any impairments, plus or minus changes resulting from observable price changes in orderly transactions for the identical or a similar investment of the same issuer. Each period the Company assesses relevant transactions to identify observable price changes, and the Company regularly monitors these investments to evaluate whether there is an indication of impairment. The Company evaluates whether an investment’s fair value is less than its carrying value using an estimate of fair value, if such an estimate is available. For periods in which there is no estimate of fair value, the Company evaluates whether an event or change in circumstances has occurred that may have a significant adverse effect on the value of the investment.

Liabilities related to the sale of future royalties and revenues

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

Acquisition

When entering an acquisition transaction, the Company first determines whether the transaction is a business combination by applying the definition in ASC Topic 805, Business Combinations (ASC 805). The definition of a “business”, requires considerations in the form of two steps: (1) determination of whether “substantially all” of the fair value of the gross assets acquired is concentrated in a single identifiable asset or a group of similar identifiable assets (i.e., “screen test”); if not, then (2) evaluate whether the set of transferred assets and activities meets the definition of a business which includes, at a minimum, an input and a substantive process that together significantly contribute to the ability to create outputs. If the assets acquired are not a business, the Company accounts for the transaction as an asset acquisition. If the transaction is a business combination, it is accounted for by applying the acquisition method.

In asset acquisitions, the Company allocates the cost of a group of assets acquired to the individual assets acquired or liabilities assumed based on their relative fair values. Goodwill is not recognized in an asset acquisition. Any difference between the cost of an asset acquisition and the fair value of the net assets acquired is allocated to the non-monetary identifiable assets based on their relative fair values. The Company follows the guidance in ASC Topic 730 Research and Development (ASC 730) and determines whether any acquired research and development assets have alternative use. If they have an alternative future use, they are recognized as assets by the Company. If they have no alternative future use, they are charged to research and development expense at the acquisition date.

In business combinations, the acquisition method is applied, where the identifiable assets acquired, the liabilities assumed, and any noncontrolling interest in the acquiree as well as any goodwill is measured and recognized based on its fair value at acquisition date.

Income Taxes

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

Foreign currency translation

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

Concentrations of Credit Risk and of Significant Suppliers

The Company has no significant off-balance-sheet risk such as foreign exchange contracts, option contracts, or other foreign hedging arrangements. Financial instruments that potentially expose the Company to concentrations of credit risk primarily consist of cash and cash equivalents, investments, accounts receivable and unbilled account receivables.

The Company maintains its cash, cash equivalents and marketable securities in custodian accounts at high quality financial institutions, and as of December 31, 2022 and 2021, substantially all the Company’s cash, cash equivalents and marketable securities were invested in money market funds and U.S. government agency securities and treasury obligations, and consequently, the Company believes that such funds are subject to minimal credit risk. The Company has adopted an investment policy that limits the amounts the Company may invest in any one type of investment. The Company has not experienced any credit losses and does not believe it is exposed to any significant credit risk on these funds.

Accounts receivables and unbilled accounts receivables represent amounts arising from product sales and amounts due from the Company’s collaboration partners. The Company monitors economic conditions to identify facts or circumstances that may indicate that its receivables are at risk of collection.

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

Segment and Geographic Information

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

New Accounting Pronouncements

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

Fair value measurements

In June 2022, the FASB issued ASU No. 2022-03, Fair Value Measurement (Topic 820) – Fair Value Measurement of Equity Securities Subject to Contractual Sale Restrictions. This standard clarifies that a contractual restriction on the sale of an equity security is not considered part of the unit of account of the equity security and, therefore, is not considered in measuring fair value. This standard will be effective for the Company on January 1, 2024, and is not expected to have a material impact on the Company’s consolidated financial statements and related disclosures.

Financing Arrangements

12 Months Ended

Dec. 31, 2022

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. 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 and U.S. territory under the terms of the Roche pralsetinib collaboration agreement.

Although the Company sold all of the rights to receive royalties on the net sales of GAVRETO worldwide excluding the CStone territory and U.S. territory to Royalty Pharma, the Company continues to co-develop pralsetinib with Roche globally and is therefore involved in the generation of these future royalties. Due to the Company’s significant continuing involvement, the Company continues to account for any royalties and development and commercialization milestones earned related to the underlying territory under the Roche pralsetinib collaboration agreement as collaboration revenue on its consolidated statements of operations and comprehensive income (loss). Net proceeds from the transaction were recorded as a liability related to sale of future royalties and revenues on the consolidated balance sheet. The Company will accrete the $175.0 million, net of transaction costs of $3.8 million, to the total of these royalties as interest expense using the effective interest method over the estimated life of the arrangement. As of December 31, 2022, the Company’s estimate of this total interest expense resulted in an effective annual interest rate of 6.2%. These estimates contain assumptions that impact the amount recorded and the interest expense that will be recognized in future periods.

As payments are made to Royalty Pharma, the balance of the liability will be effectively repaid over the life of the Royalty Purchase Agreement. In order to determine the amortization of the liability, the Company estimates the total amount of future royalty payments to be paid to Royalty Pharma over the life of the arrangement. The exact amount of repayment is likely to change each reporting period. A significant increase or decrease in GAVRETO’s net revenue of the underlying territory will impact the liability related to this arrangement and interest expense. The Company periodically assesses the expected payments to Royalty Pharma and will prospectively adjust the amortization of the liability related to this arrangement for material changes in such payments, including the prospective impact around the uncertainty related to GAVRETO’s commercial outlook in the underlying territory during the life of the arrangement.

As of December 31, 2022, the carrying value of the liability related to this arrangement was $176.0 million, net of transaction costs of $3.8 million. The following table shows the activity within the liability account (in thousands):


Carrying value as of January 1, 2022	$	—
Sale of future royalties		175,000
Interest expense recognized		5,407
Capitalized closing costs		(3,777)
Interest payments		(628)
Carrying value as of December 31, 2022	$	176,002

Pursuant to the Royalty Purchase Agreement, the Company is eligible to receive certain milestone payments totaling up to $165.0 million, subject to the achievement of specified net sales milestones by Roche. The potential milestone payments will be added to the carrying value of the liability related to this arrangement if and when the milestones are achieved and received.

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 financing transaction for up to $660.0 million (Financing Agreement) with Sixth Street Partners (Sixth Street). Because two transactions were entered into with the same parties and in contemplation of one another, the Company recorded these transactions based on the relative fair values of each freestanding financial instrument and allocated the proceeds in proportion to those fair value amounts.

Sixth Street Partners Purchase and Sale Agreement

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

The Company continues to own the research, development, manufacturing and commercialization of AYVAKIT/ AYVAKYT and if it is approved, elenestinib, and has significant continuing involvement in the generation of the cash flows under the Future Revenue Purchase Agreement. Therefore, the Company continues to account for any revenue earned from worldwide product sales of AYVAKIT/ AYVAKYT and, if it is approved, elenestinib, on its consolidated statements of operations and comprehensive income (loss). Net proceeds from the transaction were recorded as a liability related to sale of future royalties and revenues on the consolidated balance sheet. The Company will accrete 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, the balance of the liability will be 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 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 and will prospectively adjust the amortization of the liability related to this arrangement for material changes in such payments. As of December 31, 2022, the Company’s estimate of this total interest expense resulted in an effective annual interest rate of 11.3%. These estimates contain assumptions that impact the amount recorded and the interest expense that will be recognized in future periods.

As of December 31, 2022, the carrying value of the liability related to this arrangement was $254.3 million, net of transaction costs of $5.4 million. The following table shows the activity within the liability account (in thousands):


Carrying value as of January 1, 2022	$	—
Sale of future revenue		250,000
Interest expense recognized		12,470
Capitalized closing costs		(5,395)
Interest payments		(2,747)
Carrying value as of December 31, 2022	$	254,328

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. 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.

As part of the Financing Agreement with Sixth Street Partners, the Company received gross proceeds of $150.0 million in July 2022 and incurred an aggregate of $12.2 million of debt discounts and transaction costs, which have been recorded as a reduction to the carrying amount of the debt on the Company’s consolidated balance sheet and will be amortized as additional interest expenses using the effective interest rate method over the period from issuance through maturity. As of December 31, 2022, the Company’s estimate of the total interest expense resulted in an effective annual interest rate of 13.0%. The carrying amount of the debt as of December 31, 2022 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 (in thousands):


Carrying value as of January 1, 2022	$	—
Term loan		150,000
Interest expense recognized		7,736
Debt discounts and capitalized closing costs		(12,214)
Interest payments		(6,439)
Carrying value as of December 31, 2022	$	139,083

The total estimated total gross payments due under the term loan are as follows as of December 31, 2022 (in thousands):


	As of December 31, 2022
2023	$	16,851
2024		16,897
2025		16,851
2026		16,851
2027		16,851
Thereafter		158,402
Total	$	242,703

The Company’s obligations under the Financing Agreement is secured, subject to certain exceptions, by security interests in the substantially all of the Company’s assets and the Company’s certain 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 (i) $50.0 million during the period commencing from the date on which the term loans are funded to the date which is the day before the next term loans are funded and (ii) $80.0 million for each day thereafter. As of December 31, 2022, we were in compliance with the applicable terms and conditions of the covenants under the Financing Agreement.

Acquisition

12 Months Ended

Dec. 31, 2022

Acquisition

4. Acquisition

On November 27, 2021, the Company entered into a merger agreement for the acquisition of all the outstanding shares of Lengo Therapeutics Inc. (“Lengo”), a biopharmaceutical company committed to developing novel, best-in-class precision medicines targeting driver mutations in oncology to improve the lives of patients with cancer. The acquisition was completed on December 30, 2021.

Under the terms of the acquisition, the Company agreed to pay Lengo shareholders an upfront consideration of $250.0 million, subject to customary net indebtedness, transaction expenses, and other adjustments, as set forth in the acquisition agreement, and future contingent cash milestone payments of up to $215.0 million, upon achievement of specified regulatory approval and sales milestones. The milestone payments were determined to be contingent consideration which will be recognized when the contingency is resolved, and the consideration is paid or becomes payable.

The total net purchase price was $258.4 million upon closing of the transaction, which consists of the $250.0 million upfront payment, and $8.4 million of adjustments associated with net indebtedness, transaction expenses, and other adjustments per the terms of the agreement.

The acquisition was accounted for as acquisition of assets that did not meet the definition of a business. The asset acquisition did not constitute a business as substantially all of the fair value of the gross assets acquired was concentrated in Lengo’s lead compound LNG-451, now known as BLU-451. The acquired assets and liabilities were recorded at their relative fair values and the Company immediately expensed the acquired intellectual property in the consolidated statement of operations and comprehensive income (loss) in the amount of $260.0 million as the acquired assets represent in-process research and development with no alternative future use.

A summary of the net purchase price and the allocation of the consideration is as follows (in thousands):


Purchase price, net of cash acquired	$	258,377
Identifiable assets and liabilities acquired:
Net current liabilities		(1,580)
In-process research and development		259,957
Total identifiable net assets acquired	$	258,377

Marketable Securities

12 Months Ended

Dec. 31, 2022

Marketable Securities

5. Marketable Securities

Marketable securities consisted of the following at December 31, 2022 and 2021 (in thousands):


	Amortized		Unrealized		Unrealized		Fair
December 31, 2022	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	377,519		—		(4,848)	$	372,671
U.S. treasury obligations		591,193		22		(5,123)		586,092
Total	$	968,712	$	22	$	(9,971)	$	958,763


	Amortized		Unrealized		Unrealized		Fair
December 31, 2021	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	498,582	$	21	$	(1,460)	$	497,143
U.S. treasury obligations		328,801		—		(1,249)		327,552
Total	$	827,383	$	21	$	(2,709)	$	824,695

As of December 31, 2022, the Company held 122 debt securities that were in an unrealized loss position with an aggregate fair value of $922.3 million. Of the 122 debt securities, 56 were in an unrealized loss position for more than twelve months with an aggregate fair value of $550.6 million. As of December 31, 2021, the Company held 74 debt securities that were in an unrealized loss position with an aggregate fair value of $750.5 million, and no security held by the Company was in an unrealized loss position for more than twelve months. The Company has the intent and ability to hold its debt securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable debt securities for the years ended December 31, 2022 and 2021.

The following table summarizes the fair value (in thousands) and the number of the Company’s available-for-sale securities by their maturities at December 31, 2022 and 2021:


	As of December 31,
	2022				2021
	Fair		Number of		Fair		Number of
	value		securities		value		securities
Within one year	$	825,283		104	$	267,166		29
After one through five years		133,480		26		557,529		56
Total	$	958,763		130	$	824,695		85

The Company received proceeds of $349.4 million and $690.8 million from maturities of debt securities for the years ended December 31, 2022 and 2021, respectively. The Company did not realize any gains or losses from maturities of debt securities for the years ended December 31, 2022 and 2021.

Fair Value of Financial Instruments

12 Months Ended

Dec. 31, 2022

Fair Value of Financial Instruments

6. Fair Value of Financial Instruments

The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2022 (in thousands):


			Active		Observable		Unobservable
	December 31,		Markets		Inputs		Inputs
Description	2022		(Level 1)		(Level 2)		(Level 3)
Cash equivalents:
Money market funds	$	95,198	$	95,198	$	—	$	—
U.S. treasury obligations		4,989		4,989		—		—
Marketable securities, available-for-sale:
U.S. government agency securities		372,671		—		372,671		—
U.S. treasury obligations		586,092		586,092		—		—
Total	$	1,058,950	$	686,279	$	372,671	$	—

The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2021 (in thousands):


			Active		Observable		Unobservable
	December 31,		Markets		Inputs		Inputs
Description	2021		(Level 1)		(Level 2)		(Level 3)
Cash equivalents:
Money market funds	$	118,880	$	118,880	$	—	$	—
Marketable securities, available-for-sale:
U.S. government agency securities		497,143		—		497,143		—
U.S. treasury obligations		327,552		327,552		—		—
Total	$	943,575	$	446,432	$	497,143	$	—

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.

+ References + Details

Product Revenue Reserves and Allowances

12 Months Ended

Dec. 31, 2022

Product Revenue Reserves and Allowances

7. Product Revenue Reserves and Allowances

In January 2020, the U.S. Food and Drug Administration (FDA) approved AYVAKIT for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. In September 2020, the European Commission granted conditional marketing authorization to AYVAKYT as a monotherapy for the treatment of adult patients with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. In June 2021, the FDA granted a subsequent approval for AYVAKIT, expanding the labeled indications to include adult patients with advanced systemic mastocytosis (Advanced SM), including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). In March 2022, the European Commission expanded the marketing authorization for AYVAKYT to include the treatment of adult patients with ASM, SM-AHN, or MCL, after at least one systemic therapy.

In September 2020, the FDA granted accelerated approval of GAVRETO for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. In December 2020, the FDA granted a subsequent accelerated approval for GAVRETO, expanding the labeled indications to include adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).

The Company recorded net product revenue from the U.S. product sales of GAVRETO in 2021 until it transferred, on July 1, 2021, certain responsibilities associated with product sales to customers, pricing and distribution matters related to U.S. product sales of GAVRETO to its collaboration partner. The Company did not record any net product revenue from product sales of GAVRETO subsequent to this transition date. Products sales of GAVRETO were

reflected as part of collaboration loss sharing in the consolidated statements of operations and comprehensive income (loss). For additional information, see Note 12, Collaboration and License Agreements.

The following table summarizes revenue recognized from product sales for the years ended December 31, 2022 2021, and 2020 (in thousands):


	Year Ended December 31,
	2022		2021		2020
AYVAKIT/AYVAKYT
United States	$	97,226	$	44,959	$	20,522
Rest of World		13,767		8,022		740
Total		110,993		52,981		21,262
GAVRETO
United States		—		4,706		872
Rest of World		—		—		—
Total		—		4,706		872
Total product revenue, net	$	110,993	$	57,687	$	22,134

The Company primarily sells AYVAKIT/AYVAKYT through specialty distributors and specialty pharmacies. The Company recognized revenues from three customers accounting for 53%, 13%, and 11% of gross product revenues for the year ended December 31, 2022, and 55%, 8%, and 9% of gross product revenues for the year ended December 31, 2021, respectively. As of December 31, 2022, three customers individually accounted for approximately 40%, 18% and 15% of accounts receivable associated with the Company's product sales, as compared to 45%, 13% and 12% as of December 31, 2021, respectively.

The following table summarizes activity in each of the product revenue allowance and reserve categories for the years ended December 31, 2022 and 2021 (in thousands):


	Year Ended December 31,
	2022		2021
Beginning balance at January 1	$	4,345	$	1,192
Provision related to sales in the current period		19,292		8,624
Adjustment related to prior periods sales		(709)		(396)
Credits and payments made		(13,140)		(5,075)
Ending balance at December 31	$	9,788	$	4,345

The total reserves above, which are included in the Company’s consolidated balance sheets as of December 31, 2022 and 2021, are summarized as follows (in thousands):


	As of December 31,
	2022		2021
Reduction of accounts receivable, net	$	742	$	419
Component of accrued expenses		9,046		3,926
Total revenue-related reserves	$	9,788	$	4,345

Inventory

12 Months Ended

Dec. 31, 2022

Inventory

8. Inventory

Capitalized inventory consists of the following at December 31, 2022 and 2021 (in thousands):


	As of December 31,
	2022		2021
Raw materials	$	4,967	$	10,788
Work in process		28,427		17,702
Finished goods		2,791		3,916
Total	$	36,185	$	32,406

Balance sheet classification


	As of December 31,
	2022		2021
Inventory	$	29,697	$	21,817
Other assets		6,488		10,589
Total	$	36,185	$	32,406

Inventory amounts written down as a result of excess, obsolescence, unmarketability or other reasons are charged to cost of sales. For the years ended December 31, 2022 and 2021, the Company recognized write-downs of $2.1 million and $0.6 million, respectively. Long-term inventory, which primarily consists of work in process, is included in other assets in the consolidated balance sheets.

Restricted Cash

12 Months Ended

Dec. 31, 2022

Restricted Cash

9. Restricted Cash

At December 31, 2022 and 2021, respectively, $5.2 million and $5.2 million, of the Company’s cash is restricted by a bank primarily related to security deposits for the lease agreements for the Company’s current and former corporate headquarters. For additional information, see Note 16, Leases.

Property and Equipment, Net

12 Months Ended

Dec. 31, 2022

Property and Equipment, Net

10. Property and Equipment, Net

Property and equipment and related accumulated depreciation are as follows (in thousands):


	Estimated
	Useful Life	As of December 31,
	(Years)	2022		2021
Lab equipment	5	$	15,726	$	13,120
Furniture and fixtures	4		4,106		3,714
Computer equipment	3		1,484		1,714
Leasehold improvements	Term of lease		41,046		36,945
Software	3		657		412
Construction-in-progress			1,607		213
Total cost			64,626		56,118
Less: accumulated depreciation and amortization			(31,282)		(25,418)
Total		$	33,344	$	30,700

Property, plant and equipment are recorded at historical cost, net of accumulated depreciation. Depreciation expense for the years ended December 31, 2022, 2021 and 2020 was $6.5 million, $6.5 million and $6.6 million, respectively.

Accrued Expenses

12 Months Ended

Dec. 31, 2022

Accrued Expenses

11. Accrued Expenses

Accrued expenses consist of the following (in thousands):


	As of December 31,
	2022		2021
External research and development	$	66,041	$	68,164
Employee compensation		30,744		29,166
Accrued professional fees		17,588		12,611
Revenue-related reserves		9,046		3,926
Other		7,704		7,962
Total	$	131,123	$	121,829

Collaboration and License Agreements

12 Months Ended

Dec. 31, 2022

Collaboration and License Agreements

12. Collaboration and License Agreements

IDRx

In August 2022, the Company entered into a license agreement with IDRx, Inc. (IDRx), pursuant to which the Company granted IDRx an exclusive, worldwide, royalty-bearing license to exploit the Company’s internally discovered KIT exon 13 inhibitor IDRX-73 (IDRx License Agreement). IDRx is a clinical-stage biopharmaceutical company and among IDRx’s founders are Alexis Borisy, Nicholas Lydon, Ph.D., and George Demetri, M.D., each of whom are then-current members of our board of directors. 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 and the Company has the right to receive additional shares of IDRx’s Series A preferred stock through an anti-dilution provision subject to a defined financing cap.

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

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

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

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

For the purposes of ASC 606, the transaction price of the IDRx License Agreement at the contract inception was determined to be $27.5 million and recorded as license revenue-related party on the consolidated statements of operations and comprehensive income (loss). The fair value was derived from IDRx’s most recent financing transaction with unrelated investors. All potential milestone payments that the Company is eligible to receive under the IDRx License Agreement have been excluded from the transaction price. The Company reevaluates the transaction price for inclusion of milestone payments and royalties at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and

any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. Additionally, the Company is entitled to sales milestones and royalties from the sales of the licensed products, and revenue are recognized when the related sales occur.

The Company concluded the preferred stock investment should be accounted for as an equity investment as it is not mandatorily redeemable nor does the Company have the unilateral right to redeem the preferred stock, and the Company, along with its related parties, do not have a controlling financial interest in IDRx nor have the ability to influence the financial and operating policies through the ownership of preferred stock. IDRx’s preferred stock is not exchange-traded and does not have a readily determinable fair value. Therefore, the Company accounts for the preferred stock investment under the measurement alternative for equity investments that do not have a readily determinable fair value, which is at cost of $27.8 million, including transaction costs of $0.3 million. As of December 31, 2022, the cost of the investment in IDRx’s preferred stock was $27.8 million and was recorded as equity investment on the consolidated balance sheets. As of December 31, 2022, no adjustments have been recognized related to the preferred stock investment as a result of the application of the measurement.

Proteovant

In February 2022, the Company entered into an exclusive collaboration agreement (the Proteovant collaboration agreement) with Oncopia Therapeutics, Inc., d/b/a Proteovant Therapeutics, Inc. (Proteovant), pursuant to which the parties will jointly research and advance up to two novel protein degrader therapies into development candidates, as well as up to two additional novel protein degrader target programs as may be mutually agreed to by the Company and Proteovant (each program is referred to as a target program). On a target program-by-target program basis, the Company will have an exclusive option to obtain a worldwide, exclusive license to develop and commercialize any licensed compound and licensed product under each target program. Proteovant will have the right to opt into the global development and U.S. commercialization of certain licensed compounds and licensed products under the second target program that the Company options, and if the parties add additional target programs, Proteovant will have the same opt-in right for the fourth target program that the Company options.

The Company paid Proteovant an upfront payment of $20.0 million in connection with the execution of the agreement and Proteovant will be eligible to receive up to an additional $632.0 million in contingent milestone payments including specified research, development, regulatory and commercialization milestones and tiered percentage royalties on a licensed product-by-licensed product basis ranging from the mid- to high-single digits on net sales on the first two target programs, subject to adjustment in specified circumstances. If Proteovant opts in to the second target program, the parties will split profits and losses of that program equally in the U.S. along with development costs and the milestone payments for the program will be reduced accordingly. Proteovant will be eligible to receive milestone payments and royalties on ex-U.S. sales. In addition, the parties may jointly extend the collaboration, with the same structure and financial terms, to two additional program targets through additional funding by the Company.

The Company concluded that Proteovant is providing the Company with research services throughout the period until the Company can exercise its option to obtain a worldwide, exclusive license to develop and commercialize any licensed compound. Therefore, the Company recorded the $20.0 million upfront payment as an asset on the consolidated financial statements and will record it as research and development expense over the expected research period. During the year ended December 31, 2022, the Company recorded research and development expense of $4.4 million under the Proteovant collaboration agreement. The Company reevaluates the research period at the end of each reporting period and as any changes in circumstances occur, and if necessary, the Company adjusts its estimate of expected research period accordingly. Each research and development milestone payment will be 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), pursuant to which the Company granted Zai Lab exclusive rights to develop and commercialize the Company’s drug candidates BLU-701 and BLU-945, including their respective back-up forms and certain other forms thereof, 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), either as a monotherapy or as part of a combination therapy. The collaboration aims to accelerate and expand global development of the licensed products, which currently

include BLU-945 and BLU-525 (which served as a back-up candidate for BLU-701). The Company retains exclusive rights to the licensed products outside the Zai Lab territory.

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 will be shared by the Company and Zai Lab.

Pursuant to the terms of the Zai Lab agreement, Zai Lab is responsible for conducting all development and commercialization activities in the Zai Lab territory related to the licensed drug candidates. In addition, under the Zai Lab 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 Zai Lab agreement, including license grants to enable each party to conduct research, development and commercialization activities pursuant to the terms of the Zai Lab agreement.

The Zai Lab agreement will continue on a licensed product-by-product and region-by-region basis until the later of (i) the 12th anniversary of the date of the first commercial sale of a licensed product in the Zai Lab territory, (ii) the date of expiration of the last valid patent claim related to the Company’s patent rights of the product in the Zai Lab territory, and (iii) the expiration of the last regulatory exclusivity for that product in a region in the Zai Lab territory. Zai Lab may terminate the agreement for convenience by giving a written notice after the second anniversary of the effective date (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 evaluated the Zai Lab agreement to determine whether it is a collaborative arrangement in the scope of ASC 808. The Company concluded that the Zai Lab agreement is a collaborative agreement under ASC 808 as both parties are active participants in the clinical trials and are exposed to significant risks and rewards of those activities under the Zai Lab agreement. 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) the parties’ participation in the global development of the licensed products. The Company used the criteria specified in ASC 606 to determine which of the components of the Zai Lab agreement are performance obligations with a customer and concluded that Zai Lab is the Company’s customer for the licenses and related activities in the Zai Lab territory under ASC 606. The global development activities under the agreement does not present a transaction with a customer and the payments received by the Company for global development activities, including manufacturing, are accounted for as a reduction of related expenses. During the year ended December 31, 2022, the Company did not record material net reduction of expenses under the Zai Lab agreement.

The Company evaluated the Zai Lab territory specific licenses and related activities under ASC 606 as these transactions are considered transactions with a customer and identified three material promises at the outset of the Zai Lab agreement, which consists of the following for each licensed product: (1) the exclusive license, (2) the initial know-how transfer and (3) manufacturing activities related to development and commercial supply of the licensed product in the Zai Lab territory. The Company determined that the exclusive license and the initial know-how transfer were not distinct from each other, as the exclusive license has limited value without the corresponding know-how transfer. As such, for the purposes of ASC 606, the Company determined that these two material promises, the exclusive license and the initial know-how, should be combined into one distinct performance obligation. The Company further evaluated the material promise associated with manufacturing activities related to development and commercial supply of the licensed products in the Zai Lab territory, given Zai Lab is not obligated to purchase any minimum amount or quantities of the development and commercial supply from the Company, the Company concluded that, for the purpose of ASC 606, the provision of manufacturing activities related to development and commercial supply of the licensed product in Zai Lab territory was an option but not a performance obligation of the Company at the inception of the Zai Lab collaboration agreement and will be accounted for if and when exercised. The Company also concluded that there is no separate material right in connection with the development and commercial supply of the licensed product, as the expected

pricing was not issued at a significant and incremental discount. Therefore, the manufacturing activities were excluded as performance obligation at the outset of the arrangement.

The Company evaluated the license under ASC 606 and concluded that the license is a functional intellectual property license. The Company determined that 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. Additionally, the Company is entitled to sales milestones and royalties from Zai Lab upon future sales of the licensed products in the Zai Lab territory, and revenue will be recognized when the related sales occur. Costs that are incurred associated with Zai Lab territory specific activities are reimbursable from Zai Lab and are recognized as revenue. During the year ended December 31, 2022, the Company recorded $0.9 million in revenue related to Zai Lab territory specific activities in the consolidated financial statements.

For the purposes of ASC 606, the transaction price of the Zai Lab agreement at the outset of the arrangement was determined to be $25.0 million, which consisted of the upfront cash payment. The other 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 obligation upon delivery of the licenses and initial know-how transfer and recognized the upfront payment of $25.0 million as revenue in 2021.

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.

Roche – Pralsetinib Collaboration

In July, 2020, the Company entered into a collaboration agreement (the Roche pralsetinib collaboration agreement) with F. Hoffmann-La Roche Ltd and Genentech, Inc., a member of the Roche Group (collectively, Roche), pursuant to which the Company granted Roche exclusive rights to develop and commercialize the Company’s drug candidate pralsetinib worldwide, excluding the CStone territory (as defined below), and a co-exclusive license in the U.S. to develop and commercialize pralsetinib. In addition, Roche has the right to opt in to a next-generation RET compound co-developed by the Company and Roche.

Under the Roche pralsetinib collaboration agreement, the Company received an upfront cash payment of $675.0 million, and through December 31, 2022, the Company has received an aggregate of $105.0 million in specified regulatory and commercialization milestones. In addition to the upfront and milestone payments received through December 31, 2022, the Company is eligible to receive up to $822.0 million in contingent payments, including specified development, regulatory and sales-based milestones for pralsetinib and any licensed product containing a next-generation RET compound.

In the U.S., the Company and Roche agreed to work together to co-commercialize pralsetinib and equally share responsibilities, profits and losses. In addition, the Company is eligible to receive tiered royalties ranging from high-teens to mid-twenties on annual net sales of pralsetinib outside the U.S., excluding Greater China (the Roche territory). The Company and Roche have also agreed to co-develop pralsetinib globally in RET-altered solid tumors, including non-small cell lung cancer, medullary thyroid carcinoma and other thyroid cancers, as well as other solid tumors. The Company and Roche share global development costs for pralsetinib at a rate of 45 percent for the Company and 55 percent for Roche up to a specified amount of aggregate joint development costs, after which the Company’s share of global development costs for pralsetinib will be reduced by a specified percentage. The Company and Roche will also share specified global development costs for any next-generation RET compound co-developed under the collaboration in a similar manner.

Unless earlier terminated in accordance with its terms, the Roche pralsetinib collaboration agreement will expire on a licensed product-by-licensed product basis (i) in the U.S. upon the expiration of the gross profit sharing term for such licensed product and (ii) outside the U.S. on a country-by-country basis at the end of the applicable royalty term for such licensed product. Roche may terminate the agreement in its entirety or on a licensed product-by-licensed product or country-by-country basis subject to certain notice periods. Either party may terminate the Roche pralsetinib collaboration agreement for the other party’s uncured material breach or insolvency. Subject to the terms of the Roche

pralsetinib collaboration agreement, effective upon termination of the agreement, the Company is entitled to retain specified licenses to be able to continue to exploit the licensed products.

In connection with the Roche collaboration agreement, on July 13, 2020, the Company also entered into a stock purchase agreement with Roche Holdings, Inc. (Roche Holdings) pursuant to which the Company issued and sold an aggregate of 1,035,519 of shares of common stock to Roche Holdings at a purchase price of $96.57 per share and received an aggregate of $100.0 million in the third quarter of 2020. The closing for a minority portion of the equity investment occurred following the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and other customary closing conditions.

The Company considered the ASC 606 criteria for combining contracts and determined that the Roche pralsetinib collaboration agreement and stock purchase agreement should be combined into a single contract because they were negotiated and entered into in contemplation of one another. The Company accounted for the common stock issued to Roche Holdings based on the fair market value of the common stock on the dates of issuance. The fair market value of the common stock issued to Roche Holdings was $79.3 million, based on the closing price of the Company’s common stock on the dates of issuance, resulting in a $20.7 million premium. The Company determined that the premium paid by Roche Holdings for the common stock should be attributed to the transaction price of the Roche pralsetinib collaboration agreement.

The Company determined that the Roche pralsetinib collaboration agreement contained four material components: (i) licenses granted to Roche to develop and commercialize pralsetinib worldwide, excluding the CStone territory (pralsetinib license); (ii) the Roche territory-specific commercialization activities for pralsetinib, including manufacturing (Roche territory activities); (iii) the parties’ joint development activities for pralsetinib worldwide, excluding the CStone territory; and (iv) the parties’ joint commercialization activities for pralsetinib in the U.S. The Company considered the guidance in ASC 606 to determine which of the components of the Roche pralsetinib collaboration agreement are performance obligations with a customer and concluded that the pralsetinib license and the Roche territory activities are within the scope of ASC 606 because Roche is the Company’s customer in those transactions.

The Company evaluated the Roche pralsetinib license under ASC 606 and concluded that the pralsetinib license is a functional intellectual property license and is a distinct performance obligation. The Company determined that Roche benefited from the pralsetinib license at the time of grant, and therefore the related performance obligation is satisfied at a point in time.

The Company evaluated the Roche territory activities under ASC 606 and identified one material promise associated with manufacturing activities related to development and commercial supply of pralsetinib in the Roche territory for up to 24 months. Given that Roche is not obligated to purchase any minimum amount or quantities of the development and commercial supply from the Company, the Company concluded that, for the purpose of ASC 606, the provision of manufacturing activities related to development and commercial supply of pralsetinib in Roche territory was an option but not a performance obligation of the Company at the inception of the Roche collaboration agreement and will be accounted for if and when exercised. The Company also concluded that there is no separate material right in connection with the development and commercial supply of pralsetinib, as the expected pricing was not issued at a significant and incremental discount. Therefore, the manufacturing activities were excluded as performance obligations at the outset of the arrangement. Additionally, the Company is entitled to sales milestones and royalties from Roche upon future sales of pralsetinib in the Roche territory, and the revenue is recognized when the related sales occur. Costs that are incurred associated with the Roche territory activities are reimbursable from Roche and are recognized as revenue.

For the purposes of ASC 606, the transaction price of the Roche collaboration agreement at the outset of the arrangement was determined to be $695.7 million, which consisted of the upfront cash payment of $675.0 million and the $20.7 million premium on the sale of common stock to Roche Holdings, which was allocated to the performance obligation related to the pralsetinib licenses. Through December 31, 2022, the Company has achieved an aggregate of $105.0 million in specified regulatory and commercialization milestones and added the $105.0 million to the estimated transaction price of the Roche pralsetinib agreement. The other potential milestone payments that the Company is eligible to receive under the Roche pralsetinib agreement have been excluded from the transaction price, as all the remaining 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, 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 revenue recognized under the Roche pralsetinib collaboration during the years ended December 31, 2022, 2021 and 2020 (in thousands):


	Year Ended December 31,
	2022		2021		2020
Manufacturing and research and development services related to ex-US territory-specific activities	$	6,710	$	6,022	$	2,406
Royalty revenue		848		—		—
License milestone revenue		—		50,000		55,000
Upfront license revenue		—		—		695,694
Total Roche pralsetinib collaboration revenue	$	7,558	$	56,022	$	753,100

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 are within the scope of ASC 808, as both parties are 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 are accounted for as an increase to or reduction of research and development expenses. Prior to July 1, 2021, the Company was the principal for product sales to customers in the U.S. and recognized revenues on sales to third parties in product revenue, net in its consolidated statements of operations and comprehensive income (loss). On July 1, 2021, Roche took over certain responsibilities associated with product sales to customers, pricing and distribution matters for GAVRETO in the U.S. and became the principal for recording product sales to customers in the U.S., and the Company recognized its portion of the commercial losses sharing as collaboration loss sharing in its consolidated statements of operations and comprehensive income (loss).

The following table summarizes the amount from collaboration loss sharing after Roche became the principal for product sales of GAVRETO to customers in the U.S. (in thousands):


	Year Ended December 31,
	2022		2021		2020
The Company's share of loss in the U.S. for GAVRETO	$	8,948	$	7,801	$	—

The following table summarizes the amounts recognized as reductions to selling, general and administrative expenses related to the commercialization of GAVRETO in the U.S., and reductions to (increases in) research and development expenses related to global development activities for pralsetinib under the Roche pralsetinib collaboration during the years ended December 31, 2022, 2021 and 2020 (in thousands):


	Year Ended December 31,
	2022		2021		2020
Reductions to selling, general and administrative expenses	$	16,600	$	18,753	$	10,631
Reductions to (increases in) research and development expenses		(19,297)		11,192		20,459

The following table summarizes the contract assets and liability associated with the Roche pralsetinib collaboration as of December 31, 2022 and 2021 (in thousands):


	December 31,		December 31,
	2022		2021
Accounts receivable, net	$	—	$	2,679
Unbilled accounts receivable		—		6,802
Accrued expenses	$	6,519	$	—

Although the Company sold its right to receive royalty payments from Roche’s sales of GAVRETO in Roche territory to Royalty Pharma in June 2022, given the Company’s significant continuing involvement in the generation of future royalties, the Company continues to account for any royalties and development and commercialization milestones earned related to the Roche territory activities under the Roche pralsetinib collaboration agreement as collaboration revenue on its consolidated statements of operations and comprehensive income (loss). For additional information, see Note 3, Financing Arrangements.

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 Phase 1 clinical development for the treatment of fibrodysplasia ossificans progressiva (FOP), as well as specified other compounds related to the BLU-782 program.

Under the Clementia agreement, the Company received an upfront cash payment of $25.0 million and through December 31, 2022, the Company has received an aggregate of $50.0 million in cash milestone payments. Subject to the terms of the Clementia agreement, in addition to the upfront and milestone payments received through December 31, 2022, 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. The Company recognized revenue in total of $46.2 million related to the license and the technology transfer and inventory transfer in 2019 and $0.3 million related to remaining inventory transfer in 2020.

During the year ended December 31, 2022, cash consideration associated with an achieved development milestone of $30.0 million was added to the estimated transaction price for the Clementia agreement and recognized as revenue. The other 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.

During the years ended December 31, 2021 and 2020, no material revenue was recognized from the Clementia collaboration. There was no revenue deferred as a contract liability associated with the Clementia agreement as of December 31, 2022 and 2021.

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 forms and certain other forms thereof, in Mainland China, Hong Kong, Macau and Taiwan (each, a CStone region and collectively, the CStone territory), either as a monotherapy or as part of a combination therapy.

The Company received an upfront cash payment of $40.0 million, and through December 31, 2022, the Company has achieved an aggregate of $29.5 million in milestones under this collaboration. Subject to the terms of the CStone agreement, in addition to the upfront payments received and milestones achieved through December 31, 2022, the Company will be eligible to receive up to $316.5 million in contingent payments, including specified development, regulatory and sales-based milestones for licensed products. In addition, CStone will be 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.

A summary of manufacturing and research and development services related to the global development activities, net of expenses payable to CStone during the years ended December 31, 2022, 2021 and 2020 is as follows (in thousands):


	Year Ended December 31,
	2022		2021		2020
Manufacturing and research and development services related to global development activities, net of expenses payable to CStone	$	1,749	$	2,358	$	3,060

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.

During the years ended December 31, 2022, 2021, and 2020, cash consideration associated with achieved development and sales-based milestones of $6.5 million, $9.0 million and $2.0 million, respectively, were added to the estimated transaction price for the CStone agreement and recognized as revenue in such periods. The Company reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal.

In 2021, the Company entered into commercial supply agreements and an avapritinib manufacturing technology transfer agreement with CStone related to supply of drug substance of avapritinib and drug product of avapritinib and pralsetinib to assist CStone’s commercialization activities conducted specifically for the CStone territory. In 2022, the Company entered into a pralsetinib manufacturing technology transfer agreement with CStone related to supply of drug substance of pralsetinib. 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 drug substance and drug product to CStone.

A summary of revenue recognized under the CStone agreement during the years ended December 31, 2022, 2021 and 2020 is as follows (in thousands):


	Year Ended December 31,
	2022		2021		2020
License milestone revenue	$	6,500	$	9,000	$	2,000
Manufacturing services and royalty revenue related to CStone territory-specific activities		17,794		24,395		1,630
Total CStone collaboration revenue	$	24,294	$	33,395	$	3,630

The following table presents the contract assets and liability associated with the CStone collaboration as of December 31, 2022 and 2021 (in thousands):


	As of December 31,
	2022		2021
Accounts receivable, net	$	4,991	$	8,164
Unbilled accounts receivable		1,529		5,034
Contract liability	$	2,265	$	4,799

The contract liability associated with the CStone as of December 31, 2022 and 2021 resulted primarily from advance payments made by CStone in connection with commercial supply of pralsetinib for the CStone territory.

Roche – Immunotherapy Collaboration

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

Under the Roche immunotherapy agreement, Roche was originally granted up to five option rights to obtain an exclusive license to exploit products derived from the collaboration programs in the field of cancer immunotherapy. As a result of amendments to the Roche immunotherapy agreement in prior reporting periods and following the ending of the option period for one of the collaboration programs in February 2023, the Company and Roche are currently conducting activities only for the development program for the kinase target MAP4K1 (BLU-852) under the collaboration.

Roche’s option right is triggered upon the achievement of Phase 1 proof-of-concept. If Roche exercises its option, Roche will receive worldwide, exclusive commercialization rights for the licensed product. The Company will retain worldwide rights to any product for which Roche elects not to exercise its option.

Prior to Roche’s exercise of its option, the Company has the lead responsibility for drug discovery and preclinical development of the collaboration program. In addition, the Company has the lead responsibility for the conduct of the Phase 1 clinical trial other than any Phase 1 clinical trial for the product in combination with Roche’s portfolio of therapeutics, for which Roche has the right to lead the conduct of such Phase 1 clinical trial. Pursuant to the Roche immunotherapy agreement, the parties share the costs of Phase 1 development for the collaboration program. In addition, Roche will be responsible for post-Phase 1 development costs for the licensed product for which it retains global commercialization rights.

The Company received an upfront cash payment of $45.0 million, and through December 31, 2022, the Company has achieved an aggregate of $25.0 million in milestone payments under this collaboration. Subject to the terms of the Roche immunotherapy agreement, as amended, in addition to the upfront and milestone payments received through December 31, 2022, the Company is eligible to receive up to approximately $292.5 million in contingent option fee and milestone payments related to specified research, preclinical, clinical, regulatory and sales-based milestones. In addition, the Company will be eligible to receive tiered royalties ranging from low double-digits to high-teens on future net sales of the licensed product. The upfront cash payment and any payments for milestones, option fee and royalties are non-refundable, non-creditable and not subject to set-off.

The Roche immunotherapy agreement will continue until the date when no royalty or other payment obligations are or will become due, unless earlier terminated in accordance with the terms of the Roche immunotherapy agreement. Prior to its exercise of the option, Roche may terminate the Roche immunotherapy agreement at will upon 120 days’ prior written notice to the Company. Following its exercise of the option, Roche may terminate the Roche immunotherapy agreement at will, or, if a licensed product has been commercially sold, on a country-by-country basis, (i) upon 120 days’ prior written notice if the licensed product has not been commercially sold or (ii) upon 180 days’ prior written notice if the licensed product has been commercially sold. Either party may terminate the Roche immunotherapy 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 license to be able to continue to exploit the licensed product.

The Company assessed this arrangement in accordance with ASC 606 upon the adoption of the new standard on January 1, 2018, and concluded that the contract counterparty, Roche, is a customer prior to the exercise, if any, of an option by Roche. The Company identified the following material promises under the arrangement: (1) a non-transferable, sub-licensable and non-exclusive license to use the Company’s intellectual property and collaboration compounds to conduct research activities; (2) research and development activities through Phase 1 clinical trials under the research plan; (3) five option rights for licenses to develop, manufacture, and commercialize the collaboration targets; (4) participation on a joint research committee (JRC) and joint development committee (JDC); and (5) regulatory responsibilities under Phase 1 clinical trials. The Company determined that the license and research and development activities were not distinct from another, as the license has limited value without the performance of the research and development activities. Participation on the JRC and JDC to oversee the research and development activities was determined to be quantitatively and qualitatively immaterial and therefore is excluded from performance obligations. The regulatory responsibilities related to filings and obtaining approvals related to the drugs that may result from each program do not represent separate performance obligations based on their dependence on the research and development efforts. As such, the Company determined that these promises should be combined into a single performance obligation.

The Company evaluated the option rights for licenses to develop, manufacture, and commercialize the collaboration targets to determine whether it provides Roche with any material rights. The Company concluded that the options were not issued at a significant and incremental discount, and therefore do not provide material rights. As such, they are excluded as performance obligations at the outset of the arrangement.

Based on these assessments, the Company identified one performance obligation at the outset of the Roche immunotherapy agreement, which consists of: (1) the non-exclusive license; (2) the research and development activities through Phase 1; and (3) regulatory responsibilities under Phase 1 clinical trials.

Under the Roche immunotherapy agreement, in order to evaluate the appropriate transaction price, the Company determined that as of January 1, 2018, the upfront amount of $45.0 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 single performance obligation. The option exercise payments that may be received are excluded from the transaction price until each customer option is exercised as it was determined that the options are not material rights. The potential milestone payments that the Company is eligible to receive prior to the exercise of the options were initially 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, and, if necessary, adjusts its estimate of the transaction price.

Through December 31, 2022, the Company has achieved an aggregate of $25.0 million in research milestones under this collaboration, and these amounts were added to the estimated transaction price and allocated to the existing performance obligation as it became probable that a significant reversal of cumulative revenue would not occur for each of the research milestones achieved. During the years ended December 31, 2022 and 2021, cash considerations associated with an achieved research milestones of $1.5 million and $4.0 million, respectively, were added to the estimated transaction price. During the year ended December 31, 2022, the Company and Roche endorsed a clinical development plan for IND enabling activities for BLU-852, in combination with atezolizumab, an approved drug product commercialized by Roche, and pursuant to the Roche immunotherapy agreement, the Company is obligated to share with Roche the development cost up to $15.0 million. As a result, during the year ended December 31, 2022, the Company reduced the aggregated transaction price of this collaboration by $15.0 million and recorded its commitment of $15.0 million as a liability on its consolidated financial statements.

The Company recognizes revenue associated with the performance obligation as the research and development services are provided using an input method, according to the costs incurred as related to the research and development activities on each program and the costs expected to be incurred in the future to satisfy the performance obligation. The transfer of control occurs over this time period and, in management’s judgment, is the best measure of progress towards satisfying the performance obligation. The amounts received that have not yet been recognized as revenue are deferred as a contract liability on the Company’s consolidated balance sheet and will be recognized over the remaining research and development period until the performance obligation is satisfied. During the year ended December 31, 2022, a reduction in the costs expected to be incurred in the future to satisfy certain performance obligations under the collaboration became probable as a result of Roche and the Company endorsing the clinical development plan for IND enabling activities for BLU-852 in combination with Roche’s product.

Because of the revision to the cost expected to be incurred to satisfy the performance obligation and the reduction of the $15.0 million payment commitment to Roche from the aggregated transaction price, the Company recorded a cumulative revenue catch-up of $2.9 million during the year ended December 31, 2022. A summary of revenue recognized under the Roche immunotherapy agreement during the years ended December 31, 2022, 2021 and 2020 is as follows (in thousands):


	Year Ended December 31,
	2022		2021		2020
Roche collaboration research and development services revenue	$	2,274	$	7,636	$	14,580

During the years ended December 31, 2022, 2021 and 2020, the Company recognized the following revenue due to the changes in the contract liability balances (in thousands):


	Year Ended December 31,
	2022		2021		2020
Amounts included in the contract liability at the beginning of the period	$	4,377	$	5,080	$	11,546

The following table summarizes the liabilities related to the Roche immunotherapy agreement as of December 31, 2022 and 2021 (in thousands):


	December 31, 2022						December 31, 2021
	Current		Noncurrent		Total		Current		Noncurrent		Total
Deferred revenue	$	2,402	$	13,624	$	16,026	$	6,339	$	25,066	$	31,405
Accrued expenses	$	3,970	$	7,238	$	11,208	$	—	$	—	$	—

As of December 31, 2022, the research and development services related to the performance obligation were expected to be performed over a remaining period of approximately 3 years.

Stock-based Compensation

12 Months Ended

Dec. 31, 2022

Stock-based Compensation

13. Stock-based Compensation

2015 Stock Option and Incentive Plan

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, 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 provides that the number of shares reserved and available for issuance under the 2015 Plan will be cumulatively increased on January 1 of each calendar year by 4% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar years beginning January 1, 2022 and 2023, the number of shares reserved for issuance under the 2015 Plan was increased by 2,365,643 and 2,398,356 shares, respectively. In addition, the total number of shares reserved for issuance is subject to adjustment in the event of a stock split, stock dividend or other change in the Company’s capitalization. As of December 31, 2022, there were 4,116,893 shares available for future grant under the 2015 Plan.

2020 Inducement Plan

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. 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. The number of shares reserved and available for issuance under the Inducement Plan can be increased at any time with the approval of the Company’s board of directors. The Inducement Plan permits the board of directors or a committee thereof to use the stock-based awards available under the Inducement Plan to attract key employees for the growth of the Company. As of December 31, 2022, there were 1,473,235 shares available for future grant under the Inducement Plan.

Stock-based Compensation Expense

The Company recognized stock-based compensation expense totaling $99.0 million, $91.6 million and $75.5 million for the years ended December 31, 2022, 2021 and 2020, respectively.

Stock-based compensation expense by award type included within the consolidated statements of operations and comprehensive income (loss) is as follows (in thousands):


	Year Ended December 31,
	2022		2021			2020
Stock options	$	52,931	$	57,912	$	57,237
Restricted stock units		45,285		33,939		18,407
Employee stock purchase plan		1,418		1,184		958
Subtotal		99,634		93,035		76,602
Capitalized stock-based compensation costs		(663)		(1,405)		(1,076)
Stock-based compensation expense included in total cost and operating expenses	$	98,971	$	91,630	$	75,526

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


	Year Ended December 31,
	2022		2021		2020
Research and development	$	40,302	$	39,670	$	33,642
Selling, general and administrative		58,669		51,960		41,884
Total stock-based compensation expense included in operating expenses	$	98,971	$	91,630	$	75,526

At December 31, 2022, there was $179.7 million of total unrecognized compensation cost related to non-vested stock awards, which is expected to be recognized over a weighted-average period of 2.5 years.

Stock Options

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


	Year Ended December 31,
	2022		2021		2020
Risk-free interest rate	2.27	%	0.96	%	1.02	%
Expected dividend yield	—	%	—	%	—	%
Expected term (years)	6.0		6.0		6.0
Expected stock price volatility	55.15	%	58.03	%	60.48	%

The following table summarizes the stock option activity for the year ended December 31, 2022:


		Weighted-		Remaining	Aggregate
		Average		Contractual	Intrinsic
		Exercise		Life	Value(1)
	Shares	Price		(in Years)	(in thousands)
Outstanding at December 31, 2021	5,682,022	$	69.37	6.99	$	214,675
Granted	1,192,170		60.45
Exercised	(254,933)		18.10
Canceled	(385,808)		79.26
Outstanding at December 31, 2022	6,233,451	$	69.14	6.67	$	14,833
Exercisable at December 31, 2022	4,187,832	$	67.47	5.82	$	14,833

(1)	Intrinsic value represents the amount by which the fair market value as of December 31, 2022 of the underlying common stock exceeds the exercise price of the option.

The weighted average grant date fair value of options granted in the years ended December 31, 2022, 2021 and 2020 was $32.30, $52.93 and $34.77, respectively. The total intrinsic value of options exercised in the years ended December 31, 2022, 2021, and 2020 was $11.8 million, $52.3 million, and $43.3 million, respectively.

At December 31, 2022, the total unrecognized compensation expense related to unvested stock option awards was $75.0 million, which is expected to be recognized over a weighted-average period of approximately 2.4 years.

Restricted stock units

Restricted stock units granted by the Company generally vest ratably over four years. The following table summarizes the restricted stock units activity for the year ended December 31, 2022:


		Weighted-Average
		Grant Date
	Shares	Fair Value
Unvested shares at December 31, 2021	1,590,160	$	83.85
Granted	956,717		60.85
Vested	(482,229)		81.46
Forfeited	(170,654)		78.73
Unvested shares at December 31, 2022	1,893,994	$	73.28

The total fair value of restricted stock units vested during the years ended December 31, 2022, 2021 and 2020 was $29.4 million, $31.6 million and $7.4 million, respectively. As of December 31, 2022, the total unrecognized compensation expense related to unvested restricted stock units was $104.7 million, which is expected to be recognized over a weighted-average period of approximately 2.5 years.

2015 Employee Stock Purchase Plan

In 2015, the Company’s board of directors and stockholders approved the 2015 ESPP, which became effective upon the closing of the IPO in May 2015. The Company initially reserved a total of 243,347 shares of common stock for issuance under the 2015 ESPP. The 2015 ESPP provides that the number of shares reserved and available for issuance under the 2015 ESPP will be cumulatively increased on January 1 of each calendar year by 1% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar years beginning January 1, 2022 and 2023, the number of shares reserved for issuance under the 2015 ESPP was increased by 591,410 and 599,589 shares, respectively. The Company issued 80,717, 43,167, and 38,516 shares under the ESPP during the years ended December 31, 2022, 2021, and 2020 respectively.

Net Loss Per Share

12 Months Ended

Dec. 31, 2022

Net Income (Loss) Per Share

14. Net income (loss) per share

Basic net income (loss) per share (earnings per share, EPS) is calculated by dividing net income (loss) by the weighted average shares of common stock outstanding during the period, without consideration for common stock equivalents. Diluted net income (loss) per share is calculated by adjusting weighted average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period. For purposes of the diluted net income (loss) per share calculation, the effect of stock options, unvested restricted stock units and ESPP shares on 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 (loss) and the number of shares used to compute basic and diluted net income (loss) per share for the years ended December 31, 2022, 2021 and 2020 are as follows (in thousands, except per share data):


	Year Ended December 31,
	2022		2021		2020
Net income (loss) - basic and diluted	$	(557,517)	$	(644,085)	$	313,882
Weighted average shares outstanding - basic		59,642		58,518		54,534
Effect of dilutive securities:
Stock options		—		—		1,303
Restricted stock units		—		—		331
Weighted average shares outstanding - diluted		59,642		58,518		56,168
Net income (loss) per share - basic	$	(9.35)	$	(11.01)	$	5.76
Net income (loss) per share - diluted		(9.35)		(11.01)		5.59

For the years ended December 31, 2022, 2021 and 2020, the following dilutive securities were not included in the computation of net income (loss) per share because the effect would be anti-dilutive (in thousands):


	Year Ended December 31,
	2022	2021	2020
Stock options	6,233	5,682	4,480
Restricted stock units	1,894	1,590	13
ESPP shares	55	24	19
Total	8,182	7,296	4,512

Income Taxes

12 Months Ended

Dec. 31, 2022

Income Taxes

15. Income Taxes

A reconciliation of the U.S. statutory income tax rate to the Company’s effective tax rate is as follows for the years ended December 31, 2022, 2021 and 2020:


	Year Ended December 31,
	2022		2021		2020
Federal income tax (benefit) at statutory rate	21.00	%	21.00	%	21.00	%
Permanent differences	(0.38)		0.07		(0.47)
U.S. Tax on foreign earnings	2.52		—		—
In-process research and development	—		(8.53)		—
Federal research and development credits	1.39		0.76		(0.96)
Federal orphan drug credits	(0.42)		4.02		(10.35)
State income tax, net of federal benefit	2.92		1.26		0.08
Foreign rate differential	(0.13)		(0.10)		0.37
Deferred rate change	0.52		0.79		2.60
Foreign tax credit	0.03		0.39		—
Other	(0.64)		(0.05)		1.50
Change in valuation allowance	(27.76)		(20.08)		(13.42)
Effective income tax rate	(0.95)	%	(0.47)	%	0.35	%

The Company’s deferred tax assets and liabilities consist of the following (in thousands):


	Year Ended December 31,
	2022		2021		2020
Deferred tax assets:
Net operating loss carryforwards	$	193,115	$	243,670	$	140,769
Research and development credit carryforwards		36,874		36,345		23,679
Orphan drug credit carryforwards		148,572		150,859		125,153
Accrued expenses and other		48,896		40,617		32,206
Deferred revenue		4,520		6,598		7,317
Royalty Pharma milestones		11,507		—		—
R&D capitalization		84,077		—		—
Financing arrangements		100,155		—		—
Deferred rent		23,507		24,798		19,308
Jublient license		1,188		448		—
Interest expense		—		29		—
Total gross deferred tax asset		652,411		503,364		348,432
Deferred tax liability
Depreciation		(5,742)		(5,157)		(5,451)
Right of use assets		(18,599)		(20,059)		(14,539)
Debt issuance		(1,970)
UNICAP		(1,003)		(1,966)		—
Prepaid expenses		(2)		(2)		—
Valuation allowance		(625,095)		(476,180)		(328,442)
Net deferred tax asset	$	—	$	—	$	—

Management has evaluated the positive and negative evidence bearing upon the realizability of its deferred tax assets and has determined that it is more likely than not that the Company will not recognize the benefits of its net federal, foreign and state deferred tax assets, and as a result, a valuation allowance of $625.1 million, $476.2 million and $328.4 million has been established at December 31, 2022, 2021 and 2020, respectively. The change in the valuation allowance was $148.9 million, $147.7 million and ($42.0) million for the years ended December 31, 2022, 2021 and 2020, respectively. The increase of deferred tax asset between December 31, 2022 and 2021 is primarily driven by the capital gains generated from the purchase and sale agreements with Royalty Pharma and Sixth Street Partners as well as the Tax Cuts and Jobs Act effective January 1, 2022 requiring capitalization and amortization of all Sec. 174 costs associated with research and development costs.

The Company has incurred net operating losses (NOL) since inception with the exception of years 2020 and 2022. As of December 31, 2022, the Company had federal and state NOL carryforwards of $648.1 million and $894.0 million, respectively, which begin to expire in 2030, and of which $632.3 million of the Company’s federal NOL is post 2017 NOL that will be carried forward indefinitely. As of December 31, 2022, the Company had federal and state research and development tax credit carryforwards of $17.9 million and $20.4 million, respectively, which begin to expire in 2035. As of December 31, 2022, the Company had federal orphan drug credits of $148.6 million, which begin to expire in 2035 and state investment tax credits of $0.4 million, which have begun to expire in 2022. As of December 31, 2022, the Company has foreign tax credits of $2.5 million which will expire in 2031.

The Company has analyzed and validated its research and development tax credits as well as its orphan drug credits for 2011-2021. The Company generated research credits in 2022 but has not conducted a formal study to document its qualified activities. This study may result in an adjustment to the Company’s research and development credit carryforwards. No amounts are being presented as an uncertain tax position as of December 31, 2022 until such study is completed and the adjustment is known. A valuation allowance has been provided against the Company’s research and development credits and, if an adjustment is required, this adjustment would be offset by an adjustment to the deferred tax asset established for the research and development credit carry-forwards and the valuation allowance.

The Internal Revenue Code of 1986, as amended (the Code), provides for a limitation of the annual use of NOL and other tax attributes (such as research and development tax credit carryforwards) following certain ownership changes (as defined by the Code) that could limit the Company’s ability to utilize these carryforwards. The Company

may have experienced such ownership changes in the past, and may experience ownership changes in the future as a result of shifts in its stock ownership, some of which are outside the Company’s control. Approximately $2.0 million of the Company’s NOL carryforwards may not be available for utilization within their applicable carryforward periods based on the Section 382 study in 2021. In addition, U.S. tax laws limit the time during which these carryforwards may be applied against future taxes. Therefore, the Company may not be able to take full advantage of these carryforwards for federal or state income tax purposes. The Company analyzed the ownership changes as of December 31, 2022 and it is more likely than not that an ownership change did not occur. The Company will finalize the analysis as part of the 2022 income tax return filing.

With respect to the net operating losses and research and development tax credit carryforwards acquired from the acquisition of Lengo on December 30, 2021, the Company has completed a study to assess whether an ownership change under Section 382 of the Code has occurred, or whether there have been multiple ownership changes since Lengo’s formation. Based on the study, all of the NOL and research and development credits acquired will be available for utilization within their applicable carryforward periods. An analysis of Lengo Therapeutics, Inc. was not required as it was merged into the Company effective June 24, 2022.

Interest and penalty charges, if any, related to unrecognized tax benefits would be classified as income tax expense in the accompanying statements of operations and comprehensive income (loss). As of December 31, 2022 and 2021, the Company had no unrecognized tax benefits or accrued interest related to unrecognized tax benefits. As of December 31, 2022, the Company was open to examination in the U.S. federal and certain state jurisdictions for all of the Company’s tax years since the net operating losses may potentially be utilized in future years to reduce taxable income. Since the Company is in a loss carryforward position, it is generally subject to examination by the U.S. federal, state, and local income tax authorities for all tax years in which a loss carryforward is available.

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

On June 30, 2022, the Company entered into a royalty purchase agreement with Royalty Pharma and a purchase and sale agreement with Sixth Street Partners. Pursuant to the agreements, the Company received gross proceeds of $175.0 million from Royalty Pharma in June 2022 and $250.0 million from Sixth Street Partners in July 2022 upon the transactions closing. The total cash consideration of $425.0 million, in its entirety, was considered taxable income for calendar year ended December 31, 2022. Therefore, the $425.0 million was included in the taxable income for the year ended December 31, 2022.

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

On August 16, 2022, President Biden signed the Inflation Reduction Act (IRA) of 2022 (H.R. 5376) into law. It includes income tax incentives designed to encourage investment in renewable and alternative energy sources, adoption of electric vehicles, and improvement in the energy efficiency of buildings and communities. To finance these incentives, the law imposes a 15% corporate alternative minimum tax (CAMT) on adjusted financial statement income of corporations with profits over $1 billion. It also introduces a new excise tax on corporate stock buybacks of public US companies. The CAMT is effective for tax years beginning after December 31, 2022 while the excise tax on corporate stock buybacks is effective for repurchases of stock after December 31, 2022. The Company does not anticipate the IRA having a significant impact on the effective tax rate or income tax payable and deferred income tax positions.

The entire disclosure for income taxes. Disclosures may include net deferred tax liability or asset recognized in an enterprise's statement of financial position, net change during the year in the total valuation allowance, approximate tax effect of each type of temporary difference and carryforward that gives rise to a significant portion of deferred tax liabilities and deferred tax assets, utilization of a tax carryback, and tax uncertainties information.

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
-Publisher FASB
-Name Accounting Standards Codification
-Topic 740
-SubTopic 10
-Section 50
-Paragraph 21
-URI https://asc.fasb.org/extlink&oid=121826272&loc=d3e32857-109319

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Leases

12 Months Ended

Dec. 31, 2022

Leases

16. Leases

38 Sidney Street

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

45 Sidney Street

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

The Company has agreed to pay for the 99,833 rentable square feet an initial annual base rent of approximately $7.7 million, which increases annually until it reaches approximately $10.6 million in the last year of the initial term. The Company has also agreed to pay an initial annual base rent of approximately $3.2 million for the expansion premises, which increases annually until it reaches approximately $4.2 million in the last year of the initial term for the expansion premises. The amended lease provided the Company with a total tenant improvement allowance of approximately $17.4 million for improvements to be made to the premises. A security deposit of $3.8 million was recorded as restricted cash on the Company’s consolidated balance sheet as of December 31, 2022.

The lease agreements do not contain residual value guarantees and the components of lease cost for the years ended December 31, 2022, 2021 and 2020 were as follows (in thousands):


	Year Ended December 31,
Operating leases:	2022		2021		2020
Lease cost	$	21,830	$	18,299	$	17,600
Sublease income		(2,132)		(2,174)		(2,919)
Net lease cost	$	19,698	$	16,125	$	14,681

The Company has not entered into any material short-term leases or financing leases as of December 31, 2022.

Supplemental cash flow information related to leases for the years ended December 31, 2022, 2021 and 2020 was as follows (in thousands):


	Year Ended December 31,
	2022		2021		2020
Cash paid for amounts included in the measurement of lease liabilities:	$	15,891	$	14,896	$	14,444
Lease liabilities arising from obtaining right-of-use assets:
Operating leases	$	72	$	28,929	$	479

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


					Operating leases
Weighted average remaining lease term in years					6.8
Weighted average discount rate					7.4%

Future minimum lease payments under non-cancellable leases as of December 31, 2022 were as follows (in thousands):



2023		17,695
2024		18,163
2025		18,559
2026		19,089
2027		19,648
Thereafter		39,264
Total future minimum lease payments		132,418
Less imputed interest		(29,050)
Total	$	103,368

Employee Benefit Plans

12 Months Ended

Dec. 31, 2022

Employee Benefit Plans

17. Employee Benefit Plans

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

401(k) Savings Plan

The Company maintains a 401(k) plan for employees (the 401(k) Plan). The 401(k) Plan is intended to qualify under Section 401(k) of the Code, so that contributions to the 401(k) Plan by employees or by the Company, and the investment earnings on contributions, are not taxable to the employees until withdrawn from the 401(k) Plan, and so that contributions by the Company, if any, will be deductible by the Company when made. Under the 401(k) Plan, employees may elect to reduce their current compensation by up to the statutorily prescribed annual limit and to have the amount of such reduction contributed to the 401(k) Plan. The 401(k) Plan permits the Company to make contributions up to the limits allowed by law on behalf of all eligible employees. The expense related to the 401(k) Plan primarily consists of the Company’s matching contributions. The expenses related to the 401(k) Plan for the years ended December 31, 2022, 2021 and 2020 were $3.7 million, $3.0 million and $1.9 million, respectively.

Switzerland Defined Benefit Plan

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

The following table summarizes the plan balances as of December 31, 2022 and 2021:


	December 31,		December 31,
	2022		2021
Fair value of plan assets	$	5,706	$	4,551
Projected benefit obligation		(8,763)		(7,908)
Unfunded status recorded as other long-term liabilities		(3,057)		(3,357)
Accumulated benefit obligation	$	6,575	$	6,482

The Company’s net periodic benefit cost for the years ended December 31, 2022 and 2021 was $1.2 million and $2.0 million, respectively. The net periodic benefit cost for the year ended December 31, 2020 was not material. The contributions to the Swiss Plan for the years ended December 31, 2022, 2021 and 2020 were not material.

The entire disclosure for retirement benefits.

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Commitments and Contingencies

12 Months Ended

Dec. 31, 2022

Commitments and Contingencies

18. Commitments and Contingencies

Purchase Commitments Associated with Commercial Supply Agreements

In connection with the commercialization of AYVAKIT/AYVAKYT and GAVRETO, 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 minimum purchase obligations under these manufacturing agreements over the period of next five years is approximately $13.3 million as of December 31, 2022.

Legal Proceedings

The Company is not currently a party to any material legal proceedings. At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. The Company expenses the costs related to its legal proceedings as they are incurred.

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. 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 claims under indemnification arrangements, and it has not accrued any liabilities related to such obligations in its consolidated financial statements as of December 31, 2022 or 2021.

Subsequent Events

12 Months Ended

Dec. 31, 2022

Cash and cash equivalents

Subsequent Events

19. Subsequent Events

In February 2023, the option period for one the two collaboration programs under the Roche immunotherapy agreement ended and the Company and Roche are currently conducting activities only for BLU-852 under the collaboration. The Company is in the process of evaluating the revenue impact of this event on its subsequent financial statements.

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

12 Months Ended

Dec. 31, 2022

Policy Text Blocks

Basis of Presentation

Use of Estimates

Revenue Recognition

Revenue Recognition

Product revenue

For Medicare, the Company also estimates the number of patients in the prescription drug coverage gap for whom it will owe a rebate under the Medicare Part D program.

Collaboration and license revenue

For a complete discussion of accounting for collaboration and license revenues, see Note 12, Collaboration and License Agreements.

Accounts Receivable, net

Accounts Receivable, net

Inventory

Inventory

Fair Value Measurements

Fair Value Measurements

●

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

●

There have been no changes to the valuation methods during the years ended December 31, 2022 and 2021.

Cash and cash equivalents

Available-for-sale Investments

Available-for-Sale Investments

Comprehensive Income (Loss)

Comprehensive Income (Loss)

Research and Development Expenses

Research and Development Expenses

Selling, General and Administrative Expenses

Selling, General and Administrative Expenses

expensed as incurred. For years ended December 31, 2022, 2021 and 2020, advertising costs totaled $18.1 million, $13.5 million and $9.4 million, respectively.

Property and Equipment, Net

Property and Equipment, Net

Impairment of Long Lived Assets

Impairment of Long-Lived Assets

Leases

Leases

Stock-Based Compensation Expense

Stock-Based Compensation Expense

●

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

●

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

Equity Investment

Liabilities related to the sale of future royalties and revenues

Acquisition

Acquisition

Income Taxes

Income Taxes

Foreign Currency Translation

Foreign currency translation

Concentrations of Credit Risk and of Significant Suppliers

Segment and Geographic Information

New Accounting Pronouncements

Fair value measurements

Financing Arrangements (Tables)

12 Months Ended

Dec. 31, 2022

Summary of total estimated total gross payments due under the term loan


	As of December 31, 2022
2023	$	16,851
2024		16,897
2025		16,851
2026		16,851
2027		16,851
Thereafter		158,402
Total	$	242,703

Royalty Pharma, Royalty Purchase Agreement

Schedule of activity within the liability account


Carrying value as of January 1, 2022	$	—
Sale of future royalties		175,000
Interest expense recognized		5,407
Capitalized closing costs		(3,777)
Interest payments		(628)
Carrying value as of December 31, 2022	$	176,002

Sixth Street Partners, Future Revenue Purchase Agreement

Schedule of activity within the liability account


Carrying value as of January 1, 2022	$	—
Sale of future revenue		250,000
Interest expense recognized		12,470
Capitalized closing costs		(5,395)
Interest payments		(2,747)
Carrying value as of December 31, 2022	$	254,328

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

Schedule of activity within the liability account


Carrying value as of January 1, 2022	$	—
Term loan		150,000
Interest expense recognized		7,736
Debt discounts and capitalized closing costs		(12,214)
Interest payments		(6,439)
Carrying value as of December 31, 2022	$	139,083

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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Acquisition (Tables)

12 Months Ended

Dec. 31, 2022

Summary of the net purchase price and the allocation of the consideration


Purchase price, net of cash acquired	$	258,377
Identifiable assets and liabilities acquired:
Net current liabilities		(1,580)
In-process research and development		259,957
Total identifiable net assets acquired	$	258,377

Marketable Securities (Tables)

12 Months Ended

Dec. 31, 2022

Schedule of marketable securities

Marketable securities consisted of the following at December 31, 2022 and 2021 (in thousands):


	Amortized		Unrealized		Unrealized		Fair
December 31, 2022	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	377,519		—		(4,848)	$	372,671
U.S. treasury obligations		591,193		22		(5,123)		586,092
Total	$	968,712	$	22	$	(9,971)	$	958,763


	Amortized		Unrealized		Unrealized		Fair
December 31, 2021	Cost		Gain		Losses		Value
Marketable securities, available-for-sale:
U.S. government agency securities	$	498,582	$	21	$	(1,460)	$	497,143
U.S. treasury obligations		328,801		—		(1,249)		327,552
Total	$	827,383	$	21	$	(2,709)	$	824,695

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

The following table summarizes the fair value (in thousands) and the number of the Company’s available-for-sale securities by their maturities at December 31, 2022 and 2021:


	As of December 31,
	2022				2021
	Fair		Number of		Fair		Number of
	value		securities		value		securities
Within one year	$	825,283		104	$	267,166		29
After one through five years		133,480		26		557,529		56
Total	$	958,763		130	$	824,695		85

Fair Value of Financial Instruments (Tables)

12 Months Ended

Dec. 31, 2022

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 December 31, 2022 (in thousands):


			Active		Observable		Unobservable
	December 31,		Markets		Inputs		Inputs
Description	2022		(Level 1)		(Level 2)		(Level 3)
Cash equivalents:
Money market funds	$	95,198	$	95,198	$	—	$	—
U.S. treasury obligations		4,989		4,989		—		—
Marketable securities, available-for-sale:
U.S. government agency securities		372,671		—		372,671		—
U.S. treasury obligations		586,092		586,092		—		—
Total	$	1,058,950	$	686,279	$	372,671	$	—

The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2021 (in thousands):


			Active		Observable		Unobservable
	December 31,		Markets		Inputs		Inputs
Description	2021		(Level 1)		(Level 2)		(Level 3)
Cash equivalents:
Money market funds	$	118,880	$	118,880	$	—	$	—
Marketable securities, available-for-sale:
U.S. government agency securities		497,143		—		497,143		—
U.S. treasury obligations		327,552		327,552		—		—
Total	$	943,575	$	446,432	$	497,143	$	—

Tabular disclosure of financial instruments measured at fair value, including those classified in shareholders' equity measured on a recurring or nonrecurring basis. Disclosures include, but are not limited to, fair value measurements recorded and the reasons for the measurements, level within the fair value hierarchy in which the fair value measurements are categorized and transfers between levels 1 and 2. Nonrecurring fair value measurements are those that are required or permitted in the statement of financial position in particular circumstances.

+ References + Details

Product Revenue Reserves and Allowances (Tables) - Product revenue, net

12 Months Ended

Dec. 31, 2022

Schedule of product revenue allowance and reserve categories


	Year Ended December 31,
	2022		2021		2020
AYVAKIT/AYVAKYT
United States	$	97,226	$	44,959	$	20,522
Rest of World		13,767		8,022		740
Total		110,993		52,981		21,262
GAVRETO
United States		—		4,706		872
Rest of World		—		—		—
Total		—		4,706		872
Total product revenue, net	$	110,993	$	57,687	$	22,134


	Year Ended December 31,
	2022		2021
Beginning balance at January 1	$	4,345	$	1,192
Provision related to sales in the current period		19,292		8,624
Adjustment related to prior periods sales		(709)		(396)
Credits and payments made		(13,140)		(5,075)
Ending balance at December 31	$	9,788	$	4,345


	As of December 31,
	2022		2021
Reduction of accounts receivable, net	$	742	$	419
Component of accrued expenses		9,046		3,926
Total revenue-related reserves	$	9,788	$	4,345

Inventory (Tables)

12 Months Ended

Dec. 31, 2022

Schedule of capitalized inventory


	As of December 31,
	2022		2021
Raw materials	$	4,967	$	10,788
Work in process		28,427		17,702
Finished goods		2,791		3,916
Total	$	36,185	$	32,406

Balance sheet classification


	As of December 31,
	2022		2021
Inventory	$	29,697	$	21,817
Other assets		6,488		10,589
Total	$	36,185	$	32,406

Property and Equipment, Net (Tables)

12 Months Ended

Dec. 31, 2022

Schedule of property and equipment

Property and equipment and related accumulated depreciation are as follows (in thousands):


	Estimated
	Useful Life	As of December 31,
	(Years)	2022		2021
Lab equipment	5	$	15,726	$	13,120
Furniture and fixtures	4		4,106		3,714
Computer equipment	3		1,484		1,714
Leasehold improvements	Term of lease		41,046		36,945
Software	3		657		412
Construction-in-progress			1,607		213
Total cost			64,626		56,118
Less: accumulated depreciation and amortization			(31,282)		(25,418)
Total		$	33,344	$	30,700

Accrued Expenses (Tables)

12 Months Ended

Dec. 31, 2022

Schedule of accrued expenses

Accrued expenses consist of the following (in thousands):


	As of December 31,
	2022		2021
External research and development	$	66,041	$	68,164
Employee compensation		30,744		29,166
Accrued professional fees		17,588		12,611
Revenue-related reserves		9,046		3,926
Other		7,704		7,962
Total	$	131,123	$	121,829

Collaboration and License Agreements (Tables)

12 Months Ended

Dec. 31, 2022

Summary of manufacturing and research and development services related to the global development activities


	Year Ended December 31,
	2022		2021		2020
Manufacturing and research and development services related to global development activities, net of expenses payable to CStone	$	1,749	$	2,358	$	3,060

Schedule of collaboration loss sharing


	Year Ended December 31,
	2022		2021		2020
The Company's share of loss in the U.S. for GAVRETO	$	8,948	$	7,801	$	—

Schedule of amounts recognized as reductions to expenses


	Year Ended December 31,
	2022		2021		2020
Reductions to selling, general and administrative expenses	$	16,600	$	18,753	$	10,631
Reductions to (increases in) research and development expenses		(19,297)		11,192		20,459

Roche, Collaboration (Pralsetnib) Agreement

Summary of contract assets and/or contract liabilities


	Year Ended December 31,
	2022		2021		2020
Manufacturing and research and development services related to ex-US territory-specific activities	$	6,710	$	6,022	$	2,406
Royalty revenue		848		—		—
License milestone revenue		—		50,000		55,000
Upfront license revenue		—		—		695,694
Total Roche pralsetinib collaboration revenue	$	7,558	$	56,022	$	753,100


	December 31,		December 31,
	2022		2021
Accounts receivable, net	$	—	$	2,679
Unbilled accounts receivable		—		6,802
Accrued expenses	$	6,519	$	—

Roche, Collaboration and License (Immunotherapy) Agreement

Roche, Collaboration and License (Immunotherapy) Agreement | Collaboration revenue


	Year Ended December 31,
	2022		2021		2020
Roche collaboration research and development services revenue	$	2,274	$	7,636	$	14,580

Summary of contract assets and/or contract liabilities


	Year Ended December 31,
	2022		2021		2020
Amounts included in the contract liability at the beginning of the period	$	4,377	$	5,080	$	11,546


	December 31, 2022						December 31, 2021
	Current		Noncurrent		Total		Current		Noncurrent		Total
Deferred revenue	$	2,402	$	13,624	$	16,026	$	6,339	$	25,066	$	31,405
Accrued expenses	$	3,970	$	7,238	$	11,208	$	—	$	—	$	—

C Stone

C Stone | Collaboration revenue


	Year Ended December 31,
	2022		2021		2020
License milestone revenue	$	6,500	$	9,000	$	2,000
Manufacturing services and royalty revenue related to CStone territory-specific activities		17,794		24,395		1,630
Total CStone collaboration revenue	$	24,294	$	33,395	$	3,630

Summary of contract assets and/or contract liabilities


	As of December 31,
	2022		2021
Accounts receivable, net	$	4,991	$	8,164
Unbilled accounts receivable		1,529		5,034
Contract liability	$	2,265	$	4,799

Stock-based Compensation (Tables)

12 Months Ended

Dec. 31, 2022

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


	Year Ended December 31,
	2022		2021		2020
Risk-free interest rate	2.27	%	0.96	%	1.02	%
Expected dividend yield	—	%	—	%	—	%
Expected term (years)	6.0		6.0		6.0
Expected stock price volatility	55.15	%	58.03	%	60.48	%

Summary of stock option activity


		Weighted-		Remaining	Aggregate
		Average		Contractual	Intrinsic
		Exercise		Life	Value(1)
	Shares	Price		(in Years)	(in thousands)
Outstanding at December 31, 2021	5,682,022	$	69.37	6.99	$	214,675
Granted	1,192,170		60.45
Exercised	(254,933)		18.10
Canceled	(385,808)		79.26
Outstanding at December 31, 2022	6,233,451	$	69.14	6.67	$	14,833
Exercisable at December 31, 2022	4,187,832	$	67.47	5.82	$	14,833

(1)	Intrinsic value represents the amount by which the fair market value as of December 31, 2022 of the underlying common stock exceeds the exercise price of the option.

Summary of restricted stock units activity


		Weighted-Average
		Grant Date
	Shares	Fair Value
Unvested shares at December 31, 2021	1,590,160	$	83.85
Granted	956,717		60.85
Vested	(482,229)		81.46
Forfeited	(170,654)		78.73
Unvested shares at December 31, 2022	1,893,994	$	73.28

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

Stock-based compensation expense by award type included within the consolidated statements of operations and comprehensive income (loss) is as follows (in thousands):


	Year Ended December 31,
	2022		2021			2020
Stock options	$	52,931	$	57,912	$	57,237
Restricted stock units		45,285		33,939		18,407
Employee stock purchase plan		1,418		1,184		958
Subtotal		99,634		93,035		76,602
Capitalized stock-based compensation costs		(663)		(1,405)		(1,076)
Stock-based compensation expense included in total cost and operating expenses	$	98,971	$	91,630	$	75,526


	Year Ended December 31,
	2022		2021		2020
Research and development	$	40,302	$	39,670	$	33,642
Selling, general and administrative		58,669		51,960		41,884
Total stock-based compensation expense included in operating expenses	$	98,971	$	91,630	$	75,526

Net Loss Per Share (Tables)

12 Months Ended

Dec. 31, 2022

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


	Year Ended December 31,
	2022		2021		2020
Net income (loss) - basic and diluted	$	(557,517)	$	(644,085)	$	313,882
Weighted average shares outstanding - basic		59,642		58,518		54,534
Effect of dilutive securities:
Stock options		—		—		1,303
Restricted stock units		—		—		331
Weighted average shares outstanding - diluted		59,642		58,518		56,168
Net income (loss) per share - basic	$	(9.35)	$	(11.01)	$	5.76
Net income (loss) per share - diluted		(9.35)		(11.01)		5.59

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


	Year Ended December 31,
	2022	2021	2020
Stock options	6,233	5,682	4,480
Restricted stock units	1,894	1,590	13
ESPP shares	55	24	19
Total	8,182	7,296	4,512

Income Taxes (Tables)

12 Months Ended

Dec. 31, 2022

Schedule of the reconciliation of the effective tax rate


	Year Ended December 31,
	2022		2021		2020
Federal income tax (benefit) at statutory rate	21.00	%	21.00	%	21.00	%
Permanent differences	(0.38)		0.07		(0.47)
U.S. Tax on foreign earnings	2.52		—		—
In-process research and development	—		(8.53)		—
Federal research and development credits	1.39		0.76		(0.96)
Federal orphan drug credits	(0.42)		4.02		(10.35)
State income tax, net of federal benefit	2.92		1.26		0.08
Foreign rate differential	(0.13)		(0.10)		0.37
Deferred rate change	0.52		0.79		2.60
Foreign tax credit	0.03		0.39		—
Other	(0.64)		(0.05)		1.50
Change in valuation allowance	(27.76)		(20.08)		(13.42)
Effective income tax rate	(0.95)	%	(0.47)	%	0.35	%

Schedule of the deferred tax assets and liabilities


	Year Ended December 31,
	2022		2021		2020
Deferred tax assets:
Net operating loss carryforwards	$	193,115	$	243,670	$	140,769
Research and development credit carryforwards		36,874		36,345		23,679
Orphan drug credit carryforwards		148,572		150,859		125,153
Accrued expenses and other		48,896		40,617		32,206
Deferred revenue		4,520		6,598		7,317
Royalty Pharma milestones		11,507		—		—
R&D capitalization		84,077		—		—
Financing arrangements		100,155		—		—
Deferred rent		23,507		24,798		19,308
Jublient license		1,188		448		—
Interest expense		—		29		—
Total gross deferred tax asset		652,411		503,364		348,432
Deferred tax liability
Depreciation		(5,742)		(5,157)		(5,451)
Right of use assets		(18,599)		(20,059)		(14,539)
Debt issuance		(1,970)
UNICAP		(1,003)		(1,966)		—
Prepaid expenses		(2)		(2)		—
Valuation allowance		(625,095)		(476,180)		(328,442)
Net deferred tax asset	$	—	$	—	$	—

Leases (Tables)

12 Months Ended

Dec. 31, 2022

Summary of lease expenses and cash flow and weighted average information


	Year Ended December 31,
Operating leases:	2022		2021		2020
Lease cost	$	21,830	$	18,299	$	17,600
Sublease income		(2,132)		(2,174)		(2,919)
Net lease cost	$	19,698	$	16,125	$	14,681


	Year Ended December 31,
	2022		2021		2020
Cash paid for amounts included in the measurement of lease liabilities:	$	15,891	$	14,896	$	14,444
Lease liabilities arising from obtaining right-of-use assets:
Operating leases	$	72	$	28,929	$	479


					Operating leases
Weighted average remaining lease term in years					6.8
Weighted average discount rate					7.4%

Schedule of future minimum lease payments



2023		17,695
2024		18,163
2025		18,559
2026		19,089
2027		19,648
Thereafter		39,264
Total future minimum lease payments		132,418
Less imputed interest		(29,050)
Total	$	103,368

Employee Benefit Plans (Tables)

12 Months Ended

Dec. 31, 2022

Summary of plan balances

The following table summarizes the plan balances as of December 31, 2022 and 2021:


	December 31,		December 31,
	2022		2021
Fair value of plan assets	$	5,706	$	4,551
Projected benefit obligation		(8,763)		(7,908)
Unfunded status recorded as other long-term liabilities		(3,057)		(3,357)
Accumulated benefit obligation	$	6,575	$	6,482

Nature of Business (Details) $ in Millions	Dec. 31, 2022 USD ($)
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities	$ 1,078.5

Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Accounts Receivable, Net (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Allowance for Credit Loss [Abstract]
Credit losses	$ 0	$ 0	$ 0

Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Selling, General and Administrative Expenses (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Selling, General and Administrative Expenses
Advertising costs	$ 18.1	$ 13.5	$ 9.4

Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Impairment of Long-Lived Assets (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Impairment of Long Lived Assets
Long-lived asset impairment	$ 0	$ 0	$ 0

Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Stock-Based Compensation Expense (Details)	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Stock Options
Stock-based compensation
Expected dividend yield	0.00%	0.00%	0.00%
Award expiration period	10 years	10 years	10 years
Employee Stock
Stock-based compensation
Purchase price on common stock (as a percent)	85.00%	85.00%	85.00%
Discount on fair value, offering date (as a percent)	15.00%	15.00%	15.00%
Discount on fair value, purchase date (as a percent)	15.00%	15.00%	15.00%
Award expiration period	180 days	180 days	180 days

Summary of Significant Accounting Policies and Recent Accounting Pronouncements - Segment and Geographic Information (Details) - segment	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Segment and Geographic Information
Number of operating segments	1	1	1

Financing Arrangements - General Information (Details) $ in Thousands		1 Months Ended	12 Months Ended
	Jun. 30, 2022 USD ($)	Jul. 31, 2022 USD ($) tranche Transaction	Dec. 31, 2022 USD ($)
Sixth Street Partners, Financing Arrangements
Debt Financing
Debt instrument, transactions, number \| Transaction		2
Sale of Future Royalties and Revenues
Gross Proceeds
Gross proceeds			$ 425,000
Sale of Future Royalties and Revenues \| Royalty Pharma, Royalty Purchase Agreement
Debt Financing
Debt instrument, issuance date	Jun. 30, 2022
Debt instrument, face amount	$ 175,000
Transaction costs, gross	3,800
Gross Proceeds
Gross proceeds	$ 175,000		$ 175,000
Effective Interest Rate
Debt instrument, interest rate, effective percentage (as a percent)			6.20%
Sale of Future Royalties and Revenues \| Sixth Street Partners, Future Revenue Purchase Agreement
Debt Financing
Debt instrument, face amount		$ 250,000
Transaction costs, gross		5,400
Gross Proceeds
Gross proceeds		250,000	$ 250,000
Effective Interest Rate
Debt instrument, interest rate, effective percentage (as a percent)			11.30%
Loans Payable \| Sixth Street Partners, Financing Agreement
Debt Financing
Debt instrument, face amount, potential amount		660,000
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded		50,000
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded, each day thereafter		$ 80,000
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities
Debt Financing
Debt instrument, maturity date		Jun. 30, 2028
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities \| Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate
Debt Financing
Debt instrument, basis spread on variable rate		6.50%
Debt instrument, variable rate floor		1.00%
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities \| Base Rate
Debt Financing
Debt instrument, basis spread on variable rate		5.50%
Debt instrument, variable rate floor		2.00%
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility
Debt Financing
Debt instrument, face amount		$ 150,000
Debt discounts and transaction costs		12,200
Gross Proceeds
Gross proceeds		150,000	$ 150,000
Effective Interest Rate
Debt instrument, interest rate, effective percentage (as a percent)			13.00%
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Delayed Draw Term Loan Facility
Debt Financing
Line of credit facility, maximum borrowing capacity		$ 250,000
Debt instrument, tranches, potential, number \| tranche		2

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

Financing Arrangements - Long-term Debt (Details) - Sale of Future Royalties and Revenues - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Royalty Pharma, Royalty Purchase Agreement
Long-term Debt
Long-term debt	$ 176,002	$ 0
Debt Issuance Costs, Net
Transaction costs, net	3,800
Sixth Street Partners, Future Revenue Purchase Agreement
Long-term Debt
Long-term debt	254,328	$ 0
Debt Issuance Costs, Net
Transaction costs, net	$ 5,400

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

+ References + Details

Financing Arrangements - Roll Forward (Details) - USD ($) $ in Thousands		1 Months Ended	12 Months Ended
	Jun. 30, 2022	Jul. 31, 2022	Dec. 31, 2022
Sale of Future Royalties and Revenues
Long-term Debt
Sale of future royalties / borrowings			$ 425,000
Sale of Future Royalties and Revenues \| Royalty Pharma, Royalty Purchase Agreement
Long-term Debt
Carrying value, beginning balance			0
Sale of future royalties / borrowings	$ 175,000		175,000
Interest expense recognized			5,407
Debt discounts and capitalized closing costs			(3,777)
Interest payments			(628)
Carrying value, ending balance			176,002
Sale of Future Royalties and Revenues \| Sixth Street Partners, Future Revenue Purchase Agreement
Long-term Debt
Carrying value, beginning balance			0
Sale of future royalties / borrowings		$ 250,000	250,000
Interest expense recognized			12,470
Debt discounts and capitalized closing costs			(5,395)
Interest payments			(2,747)
Carrying value, ending balance			254,328
Secured Debt \| Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility
Long-term Debt
Carrying value, beginning balance			0
Sale of future royalties / borrowings		$ 150,000	150,000
Interest expense recognized			7,736
Debt discounts and capitalized closing costs			(12,214)
Interest payments			(6,439)
Carrying value, ending balance			$ 139,083

Financing Arrangements - Estimated Total Gross Payments (Details) - Secured Debt - Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility $ in Thousands	Dec. 31, 2022 USD ($)
Financing Arrangements
2023	$ 16,851
2024	16,897
2025	16,851
2026	16,851
2027	16,851
Thereafter	158,402
Total	$ 242,703

Financing Arrangements - Additional Information (Details) $ in Millions	Jun. 30, 2022 USD ($)
Debt Financing
Royalty Purchase Agreement, milestone payments, achievement of specified net sales milestones, maximum	$ 165.0

Acquisition - General Information (Details) - Lengo Therapeutics Inc.	Dec. 30, 2021	Nov. 27, 2021
Acquisition
Asset Acquisition, Date of Acquisition Agreement		Nov. 27, 2021
Asset Acquisition, Effective Date of Acquisition	Dec. 30, 2021

Acquisition - Consideration Transferred (Details) - Lengo Therapeutics Inc. $ in Thousands	Dec. 30, 2021 USD ($)
Acquisition
Upfront payment	$ 250,000
Future contingent cash milestone payments	215,000
Total net purchase price	258,377
Adjustments associated with net indebtedness, transaction expenses, and other adjustments	$ 8,400

Acquisition - Acquired Intellectual Property (Details) - USD ($) $ in Thousands		12 Months Ended
	Dec. 30, 2021	Dec. 31, 2021
Acquisition
Acquired in-process research and development		$ 259,957
Lengo Therapeutics Inc.
Acquisition
Acquired in-process research and development	$ 260,000

Acquisition - Purchase Price (Details) $ in Thousands	Dec. 30, 2021 USD ($)
Lengo Therapeutics Inc.
Acquisition
Purchase price, net of cash acquired	$ 258,377

Acquisition - Identifiable Net Assets Acquired (Details) - Lengo Therapeutics Inc. $ in Thousands	Dec. 30, 2021 USD ($)
Acquisition
Net current liabilities	$ (1,580)
In-process research and development	259,957
Total identifiable net assets acquired	$ 258,377

Marketable Securities - Tabular Disclosure (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Marketable securities, available-for-sale:
Amortized cost	$ 968,712	$ 827,383
Unrealized gain	22	21
Unrealized losses	(9,971)	(2,709)
Fair value	958,763	824,695
U.S. government agency securities
Marketable securities, available-for-sale:
Amortized cost	377,519	498,582
Unrealized gain		21
Unrealized losses	(4,848)	(1,460)
Fair value	372,671	497,143
U.S. Treasury obligations
Marketable securities, available-for-sale:
Amortized cost	591,193	328,801
Unrealized gain	22
Unrealized losses	(5,123)	(1,249)
Fair value	$ 586,092	$ 327,552

Marketable Securities - Unrealized Loss Position (Details) $ in Thousands	Dec. 31, 2022 USD ($) security	Dec. 31, 2021 USD ($) security
Unrealized loss position, number of positions
Number of held securities in an unrealized loss position \| security	122	74
Number of held securities in an unrealized loss position for 12 months or longer \| security	56
Unrealized loss position, aggregate fair value
Unrealized loss position, aggregate fair value \| $	$ 922,300	$ 750,500
Unrealized loss position for more than 12 months, aggregate fair value \| $	$ 550,600	$ 0

Marketable Securities - Impairment (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Marketable Securities
Marketable debt securities, charges for credit-related impairments	$ 0	$ 0

Marketable Securities - Maturities (Details) $ in Thousands	Dec. 31, 2022 USD ($) security	Dec. 31, 2021 USD ($) security
Maturities
Within one year, fair value \| $	$ 825,283	$ 267,166
After one through five years, fair value \| $	133,480	557,529
Investments, available-for-sale \| $	$ 958,763	$ 824,695
Within one year, number of securities \| security	104	29
After one through five years, number of securities \| security	26	56
Investments, available-for-sale, number of securities \| security	130	85

Marketable Securities - Proceeds from Maturities (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Proceeds from the maturities of debt securities
Proceeds from the maturities of debt securities	$ 349,373	$ 690,830	$ 538,347

Marketable Securities - Realized Gains (Losses) (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Realized gains (losses) from maturities of debt securities
Realized gains (losses) from maturities of debt securities	$ 0	$ 0

Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 958,763	$ 824,695
U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	372,671	497,143
U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	586,092	327,552
Recurring
Fair Value of Financial Instruments
Total	1,058,950	943,575
Recurring \| U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	372,671	497,143
Recurring \| U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	586,092	327,552
Recurring \| Money market funds
Fair Value of Financial Instruments
Cash equivalents	95,198	118,880
Recurring \| U.S. Treasury obligations
Fair Value of Financial Instruments
Cash equivalents	4,989
Recurring \| Active Markets (Level 1)
Fair Value of Financial Instruments
Total	686,279	446,432
Recurring \| Active Markets (Level 1) \| U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	586,092	327,552
Recurring \| Active Markets (Level 1) \| Money market funds
Fair Value of Financial Instruments
Cash equivalents	95,198	118,880
Recurring \| Active Markets (Level 1) \| U.S. Treasury obligations
Fair Value of Financial Instruments
Cash equivalents	4,989
Recurring \| Observable Inputs (Level 2)
Fair Value of Financial Instruments
Total	372,671	497,143
Recurring \| Observable Inputs (Level 2) \| U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 372,671	$ 497,143

Product Revenue Reserves and Allowances - Product Revenue (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Revenues
Revenue	$ 204,036	$ 180,080	$ 793,735
Product revenue, net
Revenues
Revenue	110,993	57,687	22,134
AYVAKIT and AYVAKYT
Revenues
Revenue	110,993	52,981	21,262
AYVAKIT and AYVAKYT \| United States
Revenues
Revenue	97,226	44,959	20,522
AYVAKIT and AYVAKYT \| Rest of World
Revenues
Revenue	$ 13,767	8,022	740
GAVRETO
Revenues
Revenue		4,706	872
GAVRETO \| United States
Revenues
Revenue		$ 4,706	$ 872

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

Reference 1: http://www.xbrl.org/2003/role/disclosureRef
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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/disclosureRef
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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 7: http://www.xbrl.org/2003/role/disclosureRef
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Reference 8: http://www.xbrl.org/2003/role/disclosureRef
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Reference 9: 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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-Paragraph 1
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-URI https://asc.fasb.org/extlink&oid=6472922&loc=d3e499488-122856

Product Revenue Reserves and Allowances - Concentration Risk (Details)	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Revenue from Contract with Customer Benchmark \| Customer Concentration Risk \| Largest Customers, Largest Customer
Concentration Risk
Concentration risk (as a percent)	53.00%	55.00%
Revenue from Contract with Customer Benchmark \| Customer Concentration Risk \| Largest Customers, Second-largest Customer
Concentration Risk
Concentration risk (as a percent)	13.00%	8.00%
Revenue from Contract with Customer Benchmark \| Customer Concentration Risk \| Largest Customers, Third-largest Customer
Concentration Risk
Concentration risk (as a percent)	11.00%	9.00%
Accounts Receivable, Net \| Credit Concentration Risk \| Largest Customers, Largest Customer
Concentration Risk
Concentration risk (as a percent)	40.00%	45.00%
Accounts Receivable, Net \| Credit Concentration Risk \| Largest Customers, Second-largest Customer
Concentration Risk
Concentration risk (as a percent)	18.00%	13.00%
Accounts Receivable, Net \| Credit Concentration Risk \| Largest Customers, Third-largest Customer
Concentration Risk
Concentration risk (as a percent)	15.00%	12.00%

Product Revenue Reserves and Allowances - Product Revenue Allowance and Reserve (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Product revenue allowance and reserve
Beginning balance	$ 4,345	$ 1,192
Provision related to sales in the current period	19,292	8,624
Adjustment related to prior periods sales	(709)	(396)
Credits and payments made	(13,140)	(5,075)
Ending balance	$ 9,788	$ 4,345

Product Revenue Reserves and Allowances - Revenue-related Reserves (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Revenue-related reserves
Revenue-related reserves	$ 9,788	$ 4,345	$ 1,192
Accounts Receivable, Net
Revenue-related reserves
Revenue-related reserves	742	419
Accrued Expenses
Revenue-related reserves
Revenue-related reserves	$ 9,046	$ 3,926

Inventory - Capitalized (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Inventory, Noncurrent [Abstract]
Raw materials	$ 4,967	$ 10,788
Work in process	28,427	17,702
Finished goods	2,791	3,916
Total	$ 36,185	$ 32,406

Inventory - Balance Sheet Classification (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Inventory
Inventory	$ 29,697	$ 21,817
Other assets	6,488	10,589
Total	$ 36,185	$ 32,406

Inventory - Write-down (Details) - USD ($) $ in Millions	12 Months Ended
Inventory - Write-down (Details) - USD ($) $ in Millions	Dec. 31, 2022	Dec. 31, 2021
Inventory
Inventory write-down	$ 2.1	$ 0.6

Restricted Cash (Details) - USD ($) $ in Millions	Dec. 31, 2022	Dec. 31, 2021
Restricted cash
Restricted cash	$ 5.2	$ 5.2

Property and Equipment, Net - Estimated Useful Lives (Details)	12 Months Ended
	Dec. 31, 2022
Lab equipment
Property and Equipment, Net
Estimated Useful Life (Years)	5 years
Furniture and fixtures
Property and Equipment, Net
Estimated Useful Life (Years)	4 years
Computer equipment
Property and Equipment, Net
Estimated Useful Life (Years)	3 years
Leasehold improvements
Property and Equipment, Net
Estimated Useful Life	Term of lease
Software
Property and Equipment, Net
Estimated Useful Life (Years)	3 years

Property and Equipment, Net - Property and Equipment and Related Accumulated Depreciation (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Property and Equipment, Net
Total cost	$ 64,626	$ 56,118
Less: accumulated depreciation and amortization	(31,282)	(25,418)
Total	33,344	30,700
Lab equipment
Property and Equipment, Net
Total cost	15,726	13,120
Furniture and fixtures
Property and Equipment, Net
Total cost	4,106	3,714
Computer equipment
Property and Equipment, Net
Total cost	1,484	1,714
Leasehold improvements
Property and Equipment, Net
Total cost	41,046	36,945
Software
Property and Equipment, Net
Total cost	657	412
Construction-in-progress
Property and Equipment, Net
Total cost	$ 1,607	$ 213

Property and Equipment, Net - Depreciation Expense (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Depreciation expense
Depreciation expense	$ 6.5	$ 6.5	$ 6.6

Accrued Expenses (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Accrued Expenses
External research and development	$ 66,041	$ 68,164
Employee compensation	30,744	29,166
Accrued professional fees	17,588	12,611
Revenue-related reserves	9,046	3,926
Other	7,704	7,962
Total	$ 131,123	$ 121,829

Collaboration and License Agreements - General Information (Details) $ in Thousands			1 Months Ended			3 Months Ended	12 Months Ended			30 Months Ended	39 Months Ended
	Oct. 15, 2019 USD ($)	Jun. 01, 2018 USD ($) item	Feb. 28, 2022 USD ($)	Nov. 30, 2021 USD ($)	Mar. 31, 2016 USD ($) item	Sep. 30, 2020 USD ($)	Dec. 31, 2022 USD ($) item	Dec. 31, 2021 USD ($)	Dec. 31, 2020 USD ($)	Dec. 31, 2022 USD ($)	Dec. 31, 2022 USD ($)	Jul. 13, 2020
Collaboration Agreements
Prepaid expenses and other current assets							$ 35,024	$ 18,064		$ 35,024	$ 35,024
Research and development expense							477,419	$ 601,033	$ 326,860
Collaborative Arrangement \| IDRx, Inc. \| Director
Collaboration Agreements
Collaborative arrangement, milestones, potential contingent payments							$ 217,500			217,500	217,500
Collaborative arrangement, termination, counterparty written notice period							12 months
Collaborative Arrangement \| Proteovant
Collaboration Agreements
Collaborative arrangement, upfront payment			$ 20,000
Collaborative arrangement, milestones, potential contingent payments			632,000
Prepaid expenses and other current assets			$ 20,000
Research and development expense							$ 4,400
Collaborative Arrangement \| Zai Lab (Shanghai) Co., Ltd.
Collaboration 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							2 years
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 Agreements
Collaborative arrangement, upfront payment, cash payment received						$ 675,000
Collaborative arrangement, milestones, contingent payments eligible to receive							$ 822,000			822,000	822,000
Collaborative arrangement, milestones, specified regulatory and commercialization milestones, achieved							105,000			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 \| Roche, Collaboration and License (Immunotherapy) Agreement
Collaboration Agreements
Collaborative arrangement, upfront payment, cash payment received					$ 45,000
Collaborative arrangement, option fees and milestones, pre-clinical and clinical development events prior to licensing, contingent payments eligible to receive							292,500			292,500	292,500
Collaborative arrangement, milestones, research milestones, achieved							$ 25,000			25,000	25,000
Collaborative arrangement, license option rights, number \| item					5		5
Collaborative arrangement, termination, counterparty written notice period					120 days
Collaborative arrangement, termination, counterparty written notice period, after option exercise, if licensed products have not been commercially sold					120 days
Collaborative arrangement, termination, counterparty written notice period, after option exercise, if licensed products have been commercially sold					180 days
Collaborative arrangement, clinical trials, shared development cost							$ 15,000			15,000	15,000
Collaborative Arrangement \| Clementia
Collaboration Agreements
Collaborative arrangement, upfront payment, cash payment received	$ 25,000
Collaborative arrangement, milestones, cash payments received											20,000
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 Agreements
Collaborative arrangement, upfront payment, cash payment received		$ 40,000
Collaborative arrangement, milestones, cash payments received							29,500
Collaborative arrangement, milestones, contingent payments eligible to receive							$ 316,500			$ 316,500	$ 316,500
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

Collaboration and License Agreements - Transaction Price (Details) - Collaborative Arrangement - USD ($) $ in Millions				1 Months Ended	12 Months Ended
	Jul. 13, 2020	Oct. 15, 2019	Jun. 01, 2018	Nov. 30, 2021	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Zai Lab (Shanghai) Co., Ltd.
Collaboration Agreements
Collaborative arrangement, transaction price				$ 25.0
Roche, Collaboration (Pralsetnib) Agreement
Collaboration 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
Collaborative arrangement, transaction price, milestone payments received					$ 105.0
Roche, Collaboration and License (Immunotherapy) Agreement
Collaboration Agreements
Collaborative arrangement, transaction price, milestone payments received and receivable					25.0
Collaborative arrangement, transaction price, research milestone achieved, cash consideration					1.5	$ 4.0
Collaborative arrangement, transaction price, payment commitment reduction					15.0
Collaborative arrangement, transaction price, reduction					15.0
Clementia
Collaboration Agreements
Collaborative arrangement, transaction price		$ 46.5
Collaborative arrangement, transaction price, inventory purchased by counterparty, in-process manufacturing inventory		$ 0.3
C Stone
Collaboration Agreements
Collaborative arrangement, transaction price			$ 40.0
Collaborative arrangement, transaction price, cash consideration received					$ 6.5	$ 9.0	$ 2.0

Collaboration and License Agreements - Equity Investment (Details) - USD ($) $ in Thousands		1 Months Ended
	Aug. 01, 2022	Aug. 31, 2022	Dec. 31, 2022
Equity Investment
Equity investment			$ 27,789
Collaborative Arrangement \| IDRx, Inc. \| Director
Equity Investment
Equity investment, license agreement, shares received (in shares)	4,509,105	4,509,105
Equity investment		$ 27,800	$ 27,800
Equity investment, transaction costs		$ 300

Collaboration and License Agreements - Sale of Stock (Details) - Private Placement - USD ($) $ / shares in Units, $ in Thousands		3 Months Ended	12 Months Ended
	Jul. 13, 2020	Sep. 30, 2020	Dec. 31, 2020
Sale of Stock
Stock issued (in shares)		1,035,519
Share price (in dollars per share)		$ 96.57
Purchase consideration		$ 100,000
Issuance of common stock	$ 79,300		$ 79,306
Share purchase consideration, premium	$ 20,700

Collaboration and License Agreements - Manufacturing and Research and Development Services (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Collaborative Arrangement \| C Stone
Collaboration Agreements
Manufacturing and research and development services related to global development activities, net of expenses payable	$ 1,749.0	$ 2,358.0	$ 3,060.0

Collaboration and License Agreements - Revenue Recognized (Details) - USD ($) $ in Thousands	1 Months Ended	3 Months Ended	12 Months Ended
	Aug. 31, 2022	Jun. 30, 2018	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020	Dec. 31, 2019
Revenues
Revenue			$ 204,036	$ 180,080	$ 793,735
Collaborative Arrangement \| IDRx, Inc. \| Director
Revenues
Revenue	$ 27,500
Collaborative Arrangement \| Zai Lab (Shanghai) Co., Ltd.
Revenues
Revenue				25,000
Collaborative Arrangement \| Clementia
Revenues
Revenue			30,000
Collaborative Arrangement \| Collaboration revenue \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue			7,558	56,022	753,100
Collaborative Arrangement \| Collaboration revenue \| Roche, Collaboration and License (Immunotherapy) Agreement
Revenues
Revenue			2,274	7,636	14,580
Collaborative Arrangement \| Collaboration revenue \| Clementia
Revenues
Revenue						$ 46,200
Collaborative Arrangement \| Collaboration revenue \| C Stone
Revenues
Revenue		$ 40,000	24,294	33,395	3,630
Collaborative Arrangement \| Upfront license revenue \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue					695,694
Collaborative Arrangement \| License milestone revenue \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue				50,000	55,000
Collaborative Arrangement \| License milestone revenue \| C Stone
Revenues
Revenue			6,500	9,000	2,000
Collaborative Arrangement \| Territory-specific activities \| Zai Lab (Shanghai) Co., Ltd.
Revenues
Revenue			900
Collaborative Arrangement \| Territory-specific activities, manufacturing services and royalty \| C Stone
Revenues
Revenue			17,794	24,395	1,630
Collaborative Arrangement \| Territory-specific activities, manufacturing and research and development services \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue			6,710	$ 6,022	$ 2,406
Collaborative Arrangement \| Territory-specific activities, manufacturing and research and development services \| Roche, Collaboration and License (Immunotherapy) Agreement
Revenues
Cumulative revenue catch-up			(2,900)
Collaborative Arrangement \| Royalty \| Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue			848
Collaborative Arrangement \| License revenue \| IDRx, Inc. \| Director
Revenues
Revenue			$ 27,500

Collaboration and License Agreements - Collaboration Loss Sharing (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Collaboration Agreements
Collaboration loss sharing	$ 8,948	$ 7,801
Collaborative Arrangement \| Roche, Collaboration (Pralsetnib) Agreement
Collaboration Agreements
Collaboration loss sharing	$ (8,948)	$ (7,801)

Collaboration and License Agreements - Reduction in Expenses (Details) - Collaborative Arrangement - Roche, Collaboration (Pralsetnib) Agreement - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Collaboration Agreements
Collaborative arrangement, commercialization, reduction in selling, general and administrative expenses	$ 16,600	$ 18,753	$ 10,631
Collaborative arrangement, global development activities, reductions to (increases in) research and development expenses	$ (19,297)	$ 11,192	$ 20,459

Collaboration and License Agreements - Change in Contract with Customer, Liability (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Collaborative Arrangement \| Roche, Collaboration and License (Immunotherapy) Agreement
Change in Contract with Customer, Liability
Amounts included in the contact liability at the beginning of the period	$ 4,377	$ 5,080	$ 11,546

Collaboration and License Agreements - Contract Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Contract Assets
Accounts receivable, net	$ 23,525	$ 25,155
Unbilled accounts receivable	13,413	11,875
Contract Liabilities
Contract liabilities, current	4,667	11,510
Contract liabilities, noncurrent	13,624	25,066
Accrued expenses, current	66,041	68,164
Collaborative Arrangement \| Roche, Collaboration (Pralsetnib) Agreement
Contract Assets
Accounts receivable, net		2,679
Unbilled accounts receivable		6,802
Contract Liabilities
Accrued expenses	6,519
Collaborative Arrangement \| Roche, Collaboration and License (Immunotherapy) Agreement
Contract Liabilities
Contract liabilities	16,026	31,405
Contract liabilities, current	2,402	6,339
Contract liabilities, noncurrent	13,624	25,066
Accrued expenses	11,208
Accrued expenses, current	3,970
Accrued expenses, noncurrent	7,238
Collaborative Arrangement \| Roche, Collaboration and License (Immunotherapy) Agreement, Phase I Clinical Trials
Contract Liabilities
Accrued expenses	15,000
Collaborative Arrangement \| Clementia
Contract Liabilities
Contract liabilities	0	0
Collaborative Arrangement \| C Stone
Contract Assets
Accounts receivable, net	4,991	8,164
Unbilled accounts receivable	1,529	5,034
Contract Liabilities
Contract liabilities	$ 2,265	$ 4,799

Collaboration and License Agreements - Performance Obligations (Details) - Collaborative Arrangement	12 Months Ended
	Dec. 31, 2022 item
IDRx, Inc. \| Director
Performance Obligations
Collaborative arrangement, material promises, number	2
Collaborative arrangement, material promises, combined into distinct performance obligation, number	1
Zai Lab (Shanghai) Co., Ltd.
Performance Obligations
Collaborative arrangement, material promises, number	3
Collaborative arrangement, material components, number	2
Roche, Collaboration (Pralsetnib) Agreement
Performance Obligations
Collaborative arrangement, material promises, number	1
Collaborative arrangement, material promises, manufacturing activities, term	24 months
Collaborative arrangement, material components, number	4
Roche, Collaboration and License (Immunotherapy) Agreement
Performance Obligations
Revenue, performance obligation, performance obligations, number	1
Roche, Collaboration and License (Immunotherapy) Agreement \| Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date [Axis]: 2023-01-01
Performance Obligations
Revenue, remaining performance obligation, expected timing of satisfaction, period	3 years
Clementia
Performance Obligations
Collaborative arrangement, material promises, number	4
Revenue, performance obligation, performance obligations, number	3
C Stone
Performance Obligations
Collaborative arrangement, material promises, combined into distinct performance obligation, number	6
Collaborative arrangement, material components, number	2
Revenue, performance obligation, performance obligations, number	3

Stock-based Compensation - 2015 Stock Option and Incentive Plan (Details) - 2015 Stock Option and Incentive Plan - shares			12 Months Ended
	Jan. 01, 2023	Jan. 01, 2022	Dec. 31, 2022	Apr. 08, 2015
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)		2365643.00%	4.00%
Increase in number of shares available for grant (in shares)	2,398,356
Number of shares available for grant (in shares)			4,116,893

Stock-based Compensation - 2020 Inducement Plan (Details) - 2020 Inducement Plan - shares	1 Months Ended
	Jun. 30, 2022	Dec. 31, 2022	Mar. 31, 2020
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
Number of shares available for grant (in shares)		1,473,235

Stock-based Compensation - Stock-based Compensation Expense - General Information (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Total stock based compensation expense
Stock-based compensation expense	$ 98,971	$ 91,630	$ 75,526

Stock-based Compensation - Stock-based Compensation Expense - By Type (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Total stock based compensation expense
Subtotal	$ 99,634	$ 93,035	$ 76,602
Capitalized stock-based compensation costs	(663)	(1,405)	(1,076)
Stock-based compensation expense included in total cost and operating expenses	98,971	91,630	75,526
Stock Options
Total stock based compensation expense
Subtotal	52,931	57,912	57,237
Restricted Stock Units
Total stock based compensation expense
Subtotal	45,285	33,939	18,407
Employee Stock
Total stock based compensation expense
Subtotal	$ 1,418	$ 1,184	$ 958

Stock-based Compensation - Stock-based Compensation Expense - By Classification (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Total stock based compensation expense
Stock-based compensation expense	$ 98,971	$ 91,630	$ 75,526
Research and Development
Total stock based compensation expense
Stock-based compensation expense	40,302	39,670	33,642
Selling, General and Administrative
Total stock based compensation expense
Stock-based compensation expense	$ 58,669	$ 51,960	$ 41,884

Stock-based Compensation - Unrecognized Compensation Costs (Details) $ in Millions	Dec. 31, 2022 USD ($)
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock awards	$ 179.7
Total unrecognized compensation cost related to non-vested stock option awards	$ 75.0
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 6 months
Stock Options
Stock-based compensation
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 4 months 24 days
Restricted Stock Units
Stock-based compensation
Total unrecognized compensation cost related to non-vested non-option awards	$ 104.7
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 6 months

Stock-based Compensation - Awards - General Information (Details)	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Stock Options
Stock-based compensation
Award expiration period	10 years	10 years	10 years
2015 Stock Option and Incentive Plan \| Stock Options
Stock-based compensation
Award vesting period	4 years	4 years	4 years
Cliff vesting period for new employees	1 year	1 year	1 year
Award expiration period	10 years	10 years	10 years
2015 Stock Option and Incentive Plan \| Restricted Stock Units
Stock-based compensation
Award vesting period	4 years	4 years	4 years

Stock-based Compensation - Stock Options - Weighted Average Assumptions (Details) - Stock Options	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Assumptions used in estimating the fair value of stock options granted
Risk-free interest rate	2.27%	0.96%	1.02%
Expected dividend yield	0.00%	0.00%	0.00%
Expected term (in years)	6 years	6 years	6 years
Expected stock price volatility	55.15%	58.03%	60.48%

Stock-based Compensation - Stock Options - Activity (Details) - USD ($) $ / shares in Units, $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Shares
Outstanding at beginning of period (in shares)	5,682,022
Granted (in shares)	1,192,170
Exercised (in shares)	(254,933)
Cancelled (in shares)	(385,808)
Outstanding at end of period (in shares)	6,233,451	5,682,022
Weighted-Average Exercise Price
Outstanding at beginning of period (in dollars per share)	$ 69.37
Granted (in dollars per share)	60.45
Exercised (in dollars per share)	18.10
Cancelled (in dollars per share)	79.26
Outstanding at end of period (in dollars per share)	$ 69.14	$ 69.37
Additional disclosures
Shares - Exercisable (in shares)	4,187,832
Weighted-Average Exercise Price - Exercisable (in dollars per share)	$ 67.47
Remaining Contractual Life - Outstanding	6 years 8 months 1 day	6 years 11 months 26 days
Remaining Contractual Life - Exercisable	5 years 9 months 25 days
Aggregate Intrinsic Value - Outstanding	$ 14,833	$ 214,675
Aggregate Intrinsic Value - Exercisable	$ 14,833

Stock-based Compensation - Stock Options - Additional Information (Details) - USD ($) $ / shares in Units, $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Additional disclosures
Weighted-average grant date fair value of options granted	$ 32.30	$ 52.93	$ 34.77
Total intrinsic value of options exercised	$ 11.8	$ 52.3	$ 43.3

Stock-based Compensation - Restricted Stock Units - Activity (Details) - Restricted Stock Units	12 Months Ended
	Dec. 31, 2022 $ / shares shares
Shares
Unvested at beginning of period (in shares) \| shares	1,590,160
Granted (in shares) \| shares	956,717
Vested (in shares) \| shares	(482,229)
Forfeited (in shares) \| shares	(170,654)
Unvested at end of period (in shares) \| shares	1,893,994
Weighted-Average Grant Date Fair Value
Unvested at beginning or period (in dollars per share) \| $ / shares	$ 83.85
Granted (in dollars per share) \| $ / shares	60.85
Vested (in dollars per share) \| $ / shares	81.46
Forfeited (in dollars per share) \| $ / shares	78.73
Unvested at end of period (in dollars per share) \| $ / shares	$ 73.28

Stock-based Compensation - Restricted Stock Units - Additional Information (Details) - Restricted Stock Units - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Stock-based Compensation
Total fair value of restricted stock units that vested	$ 29.4	$ 31.6	$ 7.4
Vested (in shares)	482,229

Stock-based Compensation - Employee Stock Purchase Plan (Details) - Employee Stock - shares	1 Months Ended
	May 31, 2015	Jan. 01, 2023	Jan. 01, 2022
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)		599,589	591,410

Net Income (Loss) Per Share - Net Income (Loss) Per Share (Details) - USD ($) $ / shares in Units, shares in Thousands, $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Net income (loss) - basic and diluted
Net loss	$ (557,517)	$ (644,085)	$ 313,882
Net income (loss) - basic	(557,517)	(644,085)	313,882
Net income (loss) - diluted	$ (557,517)	$ (644,085)	$ 313,882
Weighted average shares outstanding
Weighted average shares outstanding - basic (in shares)	59,642	58,518	54,534
Effect of dilutive securities:
Weighted average shares outstanding - diluted (in shares)	59,642	58,518	56,168
Net income (loss) per share - basic
Net income (loss) per share - basic (in dollars per share)	$ (9.35)	$ (11.01)	$ 5.76
Net income (loss) per share - diluted
Net income (loss) per share - diluted (in dollars per share)	$ (9.35)	$ (11.01)	$ 5.59
Stock Options
Effect of dilutive securities:
Effect of dilutive securities (in shares)			1,303
Restricted Stock Units
Effect of dilutive securities:
Effect of dilutive securities (in shares)			331

Net Income (Loss) Per Share - Anti-dilutive Securities (Details) - shares shares in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	8,182	7,296	4,512
Stock Options
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	6,233	5,682	4,480
Restricted Stock Units
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	1,894	1,590	13
Employee Stock
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	55	24	19

Income Taxes - Reconciliation (Details)	12 Months Ended
Income Taxes - Reconciliation (Details)	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
A reconciliation of the U.S. statutory income tax rate to the Company's effective tax rate
Federal income tax (benefit) at statutory rate	21.00%	21.00%	21.00%
Permanent differences	(0.38%)	0.07%	(0.47%)
U.S. Tax on foreign earnings	2.52%
In-process research and development		(8.53%)
Federal research and development credits	1.39%	0.76%	(0.96%)
Federal orphan drug credits	(0.42%)	4.02%	(10.35%)
State income tax, net of federal benefit	2.92%	1.26%	0.08%
Foreign rate differential	0.13%	0.10%	(0.37%)
Deferred rate change	0.52%	0.79%	2.60%
Foreign tax credit	0.03%	0.39%
Other	(0.64%)	(0.05%)	1.50%
Change in valuation allowance	(27.76%)	(20.08%)	(13.42%)
Effective income tax rate	(0.95%)	(0.47%)	0.35%

Income Taxes - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Deferred tax assets:
Net operating loss carryforwards	$ 193,115	$ 243,670	$ 140,769
Research and development credit carryforwards	36,874	36,345	23,679
Orphan drug credit carryforwards	148,572	150,859	125,153
Accrued expenses and other	48,896	40,617	32,206
Deferred revenue	4,520	6,598	7,317
Royalty Pharma milestones	11,507
R&D capitalization	84,077
Financing arrangements	100,155
Deferred rent	23,507	24,798	19,308
Jublient license	1,188	448
Interest expense		29
Total gross deferred tax asset	652,411	503,364	348,432
Deferred tax liability
Depreciation	(5,742)	(5,157)	(5,451)
Right of use assets	(18,599)	(20,059)	(14,539)
Debt issuance	(1,970)
UNICAP	(1,003)	(1,966)
Prepaid expenses	(2)	(2)
Valuation allowance	(625,095)	(476,180)	(328,442)
Net deferred tax asset	$ 0	$ 0	$ 0

Income Taxes - Valuation Allowance (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Valuation allowance
Valuation allowance	$ 625,095	$ 476,180	$ 328,442
Change in valuation allowance	$ 148,900	$ 147,700	$ (42,000)

Income Taxes - Net Operating Loss Carryforwards (Details) $ in Millions	Dec. 31, 2022 USD ($)
Net Operating Loss Carryforwards
Net operating loss carryforwards, limitations on use, available for utilization	$ 2.0
Federal
Net Operating Loss Carryforwards
Net operating loss carryforwards	648.1
Net operating loss carryforwards, unlimited	632.3
State
Net Operating Loss Carryforwards
Net operating loss carryforwards	$ 894.0

Income Taxes - Tax Credit Carryforwards (Details) $ in Millions	Dec. 31, 2022 USD ($)
Foreign
Tax Credit Carryforwards
Tax credit carryforwards	$ 2.5
Research and Development Tax Credit \| Federal
Tax Credit Carryforwards
Tax credit carryforwards	20.4
Research and Development Tax Credit \| State and Local Jurisdiction [Member]
Tax Credit Carryforwards
Tax credit carryforwards	17.9
Orphan Drug Tax Credit \| Federal
Tax Credit Carryforwards
Tax credit carryforwards	148.6
Investment Tax Credit \| State and Local Jurisdiction [Member]
Tax Credit Carryforwards
Tax credit carryforwards	$ 0.4

Income Taxes - Uncertain Tax Positions (Details) - USD ($) $ in Millions	Dec. 31, 2022	Dec. 31, 2021
Uncertain tax positions
Accrued interest related to uncertain tax positions	$ 0.0	$ 0.0

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

Income Taxes - Equity Investment (Details) - USD ($) $ in Thousands		1 Months Ended	12 Months Ended
	Aug. 01, 2022	Aug. 31, 2022	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Revenues
Revenue			$ 204,036	$ 180,080	$ 793,735
Collaborative Arrangement \| IDRx, Inc. \| Director
Equity Investment
Equity investment, license agreement, shares received (in shares)	4,509,105	4,509,105
Revenues
Revenue		$ 27,500
Collaborative Arrangement \| License revenue \| IDRx, Inc. \| Director
Revenues
Revenue			$ 27,500

Leases - General Information (Details) $ in Thousands	Dec. 15, 2021 USD ($)	Sep. 19, 2018 USD ($) ft²	Apr. 28, 2017 USD ($) ft² period	Dec. 31, 2022 USD ($)	Dec. 31, 2021 USD ($)	Sep. 18, 2018 USD ($)	Feb. 12, 2015 ft²
Leases
Initial lease commitments				$ 132,418
Restricted cash				5,200	$ 5,200
Corporate Headquarters Lease
Leases
Area leased (in square feet) \| ft²							39,000
Base annual rent, initial	$ 4,500
Base annual rent, maximum	5,500
Allowance for leasehold improvements	$ 800
Security deposit				900
Office and Laboratory Space in Cambridge Massachusetts, 45 Sidney Street
Leases
Area leased (in square feet) \| ft²		139,216	99,833
Lessee, Operating Lease, Existence of Option to Extend			true
Number of consecutive extension periods \| period			2
Extension period of lease term			5 years
Base annual rent, initial			$ 7,700
Base annual rent, maximum			$ 10,600
Allowance for leasehold improvements						$ 17,400
Office and Laboratory Space in Cambridge, Massachusetts, 45 Sidney Street, Expansion Premises
Leases
Base annual rent, initial		$ 3,200
Base annual rent, maximum		$ 4,200
Security deposit				$ 3,800

Leases - Components of Lease Expense (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Operating leases:
Lease cost	$ 21,830	$ 18,299	$ 17,600
Sublease income	(2,132)	(2,174)	(2,919)
Net lease cost	$ 19,698	$ 16,125	$ 14,681

Leases - Cash Flow Information (Details) - USD ($) $ in Thousands	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
Leases
Cash paid for amounts included in the measurement of lease liabilities, operating cash flows from operating leases	$ 15,891	$ 14,896	$ 14,444
Lease liabilities arising from obtaining right-of-use assets, operating leases	$ 72	$ 28,929	$ 479

Leases - Weighted Average Remaining Lease-term and Weighted Average Discount Rate (Details)	Dec. 31, 2022
Leases
Weighted average remaining lease term in years	6 years 9 months 18 days
Weighted average discount rate	7.40%

Leases - Future Minimum Lease Payments (Details) $ in Thousands	Dec. 31, 2022 USD ($)
Future Minimum Lease Payments
2023	$ 17,695
2024	18,163
2025	18,559
2026	19,089
2027	19,648
Thereafter	39,264
Total future minimum lease payments	$ 132,418

Leases - Total Lease Liability (Details) $ in Thousands	Dec. 31, 2022 USD ($)
Leases
Total future minimum lease payments	$ 132,418
Less imputed interest	(29,050)
Total	$ 103,368

Employee Benefit Plans - 401(k) Savings Plan (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021	Dec. 31, 2020
401(k) Savings Plan
401(k) Plan expenses	$ 3.7	$ 3.0	$ 1.9

Employee Benefit Plans - Switzerland Defined Benefit Plan - Tabular Disclosure (Details) - USD ($) $ in Thousands	Dec. 31, 2022	Dec. 31, 2021
Accumulated Benefit Obligation in Excess of Plan Assets
Fair value of plan assets	$ 5,706	$ 4,551
Projected benefit obligation	(8,763)	(7,908)
Accumulated benefit obligation	6,575	6,482
Unfunded Status
Unfunded status recorded as other long-term liabilities	$ (3,057)	$ (3,357)

Employee Benefit Plans - Switzerland Defined Benefit Plan - General Information (Details) - USD ($) $ in Millions	12 Months Ended
	Dec. 31, 2022	Dec. 31, 2021
Additional Information
Defined Benefit Plan, Type	us-gaap:PensionPlansDefinedBenefitMember
Defined Benefit Plan, Sponsor Location	country:CH
Net Periodic Benefit Cost
Net periodic benefit cost	$ 1.2	$ 2.0

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

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

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