Blueprint Medicines (BPMC) 10-Q 2 Aug 22 2022 Q2 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - $ / shares	6 Months Ended
	Jun. 30, 2022	Jul. 29, 2022
Cover [Abstract]
Entity Central Index Key	0001597264
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2022
Document Transition Report	false
Entity File Number	001-37359
Entity Registrant Name	BLUEPRINT MEDICINES CORPORATION
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	26-3632015
Entity Address, Address Line One	45 Sidney Street
Entity Address, City or Town	Cambridge
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	02139
City Area Code	617
Local Phone Number	374-7580
Title of 12(b) Security	Common stock, par value $0.001 per share
Trading Symbol	BPMC
Security Exchange Name	NASDAQ
Entity Current Reporting Status	Yes
Entity Interactive Data Current	Yes
Entity Filer Category	Large Accelerated Filer
Entity Small Business	false
Entity Emerging Growth Company	false
Entity Shell Company	false
Entity Listing, Par Value Per Share	$ 0.001
Entity Common Stock, Shares Outstanding		59,719,570
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2022
Document Fiscal Period Focus	Q2
Amendment Flag	false

Condensed Consolidated Balance

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Current assets:
Cash and cash equivalents	$ 173,399	$ 209,948
Marketable securities	679,712	267,166
Accounts receivable	27,123	25,155
Unbilled accounts receivable	2,287	11,875
Inventory	35,454	21,817
Prepaid expenses and other current assets	34,204	18,064
Total current assets	952,179	554,025
Marketable securities	94,048	557,529
Property and equipment, net	32,987	30,700
Operating lease right-of-use assets, net	86,033	90,162
Restricted cash	5,171	5,171
Other assets	22,028	14,638
Total assets	1,192,446	1,252,225
Current liabilities:
Accounts payable	15,053	8,333
Accrued expenses	119,282	121,829
Current portion of operating lease liabilities	9,435	8,093
Current portion of deferred revenue	9,478	11,510
Liability related to the sale of future royalties	5,512
Total current liabilities	158,760	149,765
Operating lease liabilities, net of current portion	98,160	103,315
Deferred revenue, net of current portion	12,661	25,066
Liability related to the sale of future royalties, net of current portion	165,742
Other long-term liabilities	9,373	3,344
Total liabilities	444,696	281,490
Commitments and Contingencies (Note 15)
Stockholders' equity:
Preferred stock, $0.001 par value; 5,000,000 shares authorized; no shares issued and outstanding
Common stock, $0.001 par value; 120,000,000 shares authorized; 59,688,295 and 59,141,086 shares issued and outstanding at June 30, 2022 and December 31, 2021, respectively	60	59
Additional paid-in capital	2,303,087	2,250,250
Accumulated other comprehensive loss	(14,248)	(4,133)
Accumulated deficit	(1,541,149)	(1,275,441)
Total stockholders' equity	747,750	970,735
Total liabilities and stockholders' equity	$ 1,192,446	$ 1,252,225

Condensed Consolidated Balanc_2

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Jun. 30, 2022	Dec. 31, 2021
Preferred Stock Disclosures
Preferred stock, par value (in dollars per share)	$ 0.001	$ 0.001
Preferred stock, shares authorized (in shares)	5,000,000	5,000,000
Preferred stock, shares issued (in shares)	0	0
Preferred stock, shares outstanding (in shares)	0	0
Common Stock Disclosures
Common stock, par value (in dollars per share)	$ 0.001	$ 0.001
Common stock, shares authorized (in shares)	120,000,000	120,000,000
Common stock, shares issued (in shares)	59,688,295	59,141,086
Common Stock, shares outstanding (in shares)	59,688,295	59,141,086

Condensed Consolidated Statemen

Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Revenues:
Revenue	$ 36,547	$ 27,295	$ 99,278	$ 48,871
Cost and operating expenses:
Cost of sales	4,886	6,493	9,964	6,595
Research and development	128,466	80,027	231,599	159,738
Selling, general and administrative	58,688	49,286	115,747	91,288
Collaboration loss sharing	2,145		5,410
Total cost and operating expenses	194,185	135,806	362,720	257,621
Other income (expense):
Interest income, net	427	633	869	1,371
Other income (expense), net	632	(373)	177	(587)
Total other income	1,059	260	1,046	784
Loss before income taxes	(156,579)	(108,251)	(262,396)	(207,966)
Income tax expense	(3,130)	(193)	(3,313)	(193)
Net loss	(159,709)	(108,444)	(265,709)	(208,159)
Other comprehensive loss:
Unrealized losses on available-for-sale investments	(2,308)	(624)	(10,328)	(1,024)
Currency translation adjustments	170	(114)	213	404
Comprehensive loss	$ (161,847)	$ (109,182)	$ (275,824)	$ (208,779)
Net income (loss) per share - basic (in dollars per share)	$ (2.68)	$ (1.86)	$ (4.47)	$ (3.58)
Net income (loss) per share - diluted (in dollars per share)	$ (2.68)	$ (1.86)	$ (4.47)	$ (3.58)
Weighted-average number of common shares used in net loss per share - basic (in shares)	59,617	58,406	59,465	58,216
Weighted-average number of common shares used in net loss per share - diluted (in shares)	59,617	58,406	59,465	58,216
Product revenue, net
Revenues:
Revenue	$ 28,454	$ 11,433	$ 52,295	$ 20,388
Collaboration revenue
Revenues:
Revenue	$ 8,093	$ 15,862	$ 46,983	$ 28,483

Condensed Consolidated Statem_2

Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands	Common Stock	Additional Paid-In Capital	Accumulated Other Comprehensive Loss	Accumulated Deficit	Total
Beginning Balance at Dec. 31, 2020	$ 58	$ 2,106,600	$ (5,214)	$ (631,356)	$ 1,470,088
Beginning Balance (in shares) at Dec. 31, 2020	57,793,533
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan		8,318			8,318
Issuance of common stock under stock plan (in shares)	483,879
Stock-based compensation expense		21,212			21,212
Other comprehensive income (loss)			117		117
Net loss				(99,714)	(99,714)
Ending Balance at Mar. 31, 2021	$ 58	2,136,130	(5,097)	(731,070)	1,400,021
Ending Balance (in shares) at Mar. 31, 2021	58,277,412
Beginning Balance at Dec. 31, 2020	$ 58	2,106,600	(5,214)	(631,356)	1,470,088
Beginning Balance (in shares) at Dec. 31, 2020	57,793,533
Increase (Decrease) in Stockholders' Equity
Net loss					(208,159)
Ending Balance at Jun. 30, 2021	$ 59	2,174,094	(5,835)	(839,514)	1,328,804
Ending Balance (in shares) at Jun. 30, 2021	58,554,559
Beginning Balance at Mar. 31, 2021	$ 58	2,136,130	(5,097)	(731,070)	1,400,021
Beginning Balance (in shares) at Mar. 31, 2021	58,277,412
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan	$ 1	11,709			11,710
Issuance of common stock under stock plan (in shares)	254,823
Purchase of common stock under ESPP		1,733			1,733
Purchase of common stock under ESPP (in shares)	22,324
Stock-based compensation expense		24,522			24,522
Other comprehensive income (loss)			(738)		(738)
Net loss				(108,444)	(108,444)
Ending Balance at Jun. 30, 2021	$ 59	2,174,094	(5,835)	(839,514)	1,328,804
Ending Balance (in shares) at Jun. 30, 2021	58,554,559
Beginning Balance at Dec. 31, 2021	$ 59	2,250,250	(4,133)	(1,275,441)	$ 970,735
Beginning Balance (in shares) at Dec. 31, 2021	59,141,086				59,141,086
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan	$ 1	1,297			$ 1,298
Issuance of common stock under stock plan (in shares)	414,888
Stock-based compensation expense		23,609			23,609
Other comprehensive income (loss)			(7,977)		(7,977)
Net loss				(105,999)	(105,999)
Ending Balance at Mar. 31, 2022	$ 60	2,275,156	(12,110)	(1,381,440)	881,666
Ending Balance (in shares) at Mar. 31, 2022	59,555,974
Beginning Balance at Dec. 31, 2021	$ 59	2,250,250	(4,133)	(1,275,441)	$ 970,735
Beginning Balance (in shares) at Dec. 31, 2021	59,141,086				59,141,086
Increase (Decrease) in Stockholders' Equity
Net loss					$ (265,709)
Ending Balance at Jun. 30, 2022	$ 60	2,303,087	(14,248)	(1,541,149)	$ 747,750
Ending Balance (in shares) at Jun. 30, 2022	59,688,295				59,688,295
Beginning Balance at Mar. 31, 2022	$ 60	2,275,156	(12,110)	(1,381,440)	$ 881,666
Beginning Balance (in shares) at Mar. 31, 2022	59,555,974
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan		535			535
Issuance of common stock under stock plan (in shares)	92,274
Purchase of common stock under ESPP		1,872			1,872
Purchase of common stock under ESPP (in shares)	40,047
Stock-based compensation expense		25,524			25,524
Other comprehensive income (loss)			(2,138)		(2,138)
Net loss				(159,709)	(159,709)
Ending Balance at Jun. 30, 2022	$ 60	$ 2,303,087	$ (14,248)	$ (1,541,149)	$ 747,750
Ending Balance (in shares) at Jun. 30, 2022	59,688,295				59,688,295

Condensed Consolidated Statem_3

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021
Cash flows from operating activities
Net loss	$ (265,709)	$ (208,159)
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities:
Depreciation and amortization	3,098	3,200
Noncash lease expense	4,107	3,073
Stock-based compensation	48,746	44,991
Other	550	1,352
Changes in assets and liabilities:
Accounts receivable	(2,128)	(18,407)
Unbilled accounts receivable	9,591	4,446
Inventory	(14,694)	(4,542)
Prepaid expenses and other current assets	(16,314)	(755)
Other assets	(13,188)	(3,829)
Accounts payable	6,589	(2,544)
Accrued expenses	441	(1,044)
Other long-term liabilities	6,157
Deferred revenue	(14,438)	(2,880)
Operating lease liabilities	(3,790)	(3,845)
Net cash used in operating activities	(250,982)	(188,943)
Cash flows from investing activities
Purchases of property and equipment	(3,709)	(835)
Purchases of investments	(39,937)	(382,644)
Maturities of investments	79,623	400,500
Net cash provided by investing activities	35,977	17,021
Cash flows from financing activities
Gross proceeds from the sale of future royalties	175,000
Net proceeds from stock option exercises and employee stock purchase plan	3,762	21,865
Net cash provided by financing activities	178,762	21,865
Net decrease in cash, cash equivalents, and restricted cash	(36,243)	(150,057)
Cash, cash equivalents and restricted cash at beginning of period	215,119	689,804
Effect of exchange rate changes on cash, cash equivalents and restricted cash	(306)	(259)
Cash, cash equivalents and restricted cash at end of period	178,570	539,488
Supplemental cash flow information
Debt issuance costs incurred but unpaid at period end	5,022
Property and equipment purchases unpaid at period end	1,829	230
Cash paid for taxes, net	$ 771	$ 598

Condensed Consolidated Statem_4

Condensed Consolidated Statements of Cash Flows (Parenthetical) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021	Jun. 30, 2021	Dec. 31, 2020
Reconciliation of cash, cash equivalents, and restricted cash
Cash and cash equivalents	$ 173,399	$ 209,948	$ 534,317
Restricted cash	5,171	5,171	5,171
Total cash, cash equivalents, and restricted cash shown in condensed consolidated statements of cash flows	$ 178,570	$ 215,119	$ 539,488	$ 689,804

Nature of Business

Nature of Business	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
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 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. As of June 30, 2022, the Company had cash, cash equivalents and marketable securities of $947.2 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 Accounti

Summary of Significant Accounting Policies and Recent Accounting Pronouncements	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Summary of Significant Accounting Policies and Recent Accounting Pronouncements	2. Summary of Significant Accounting Policies and Recent Accounting Pronouncements Basis of Presentation The unaudited interim condensed consolidated financial statements of the Company included herein have been prepared in accordance with accounting principles generally accepted in the United States (GAAP) as found in the Accounting Standards Codification (ASC), Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB) and the rules and regulations of the Securities and Exchange Commission (SEC). Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these financial statements should be read in conjunction with the financial statements as of and for the year ended December 31, 2021 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on February 17, 2022 (2021 Annual Report on Form 10-K). The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited financial statements, and updated, as necessary, in this report. In the opinion of the Company’s management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments that are necessary to present fairly the Company’s financial position as of June 30, 2022, the results of its operations for the three and six months ended June 30, 2022 and 2021, stockholder’s equity for the three and six months ended June 30, 2022 and 2021 and cash flows for six months ended June 30, 2022 and 2021. Such adjustments are of a normal and recurring nature. The results for the three and six months ended June 30, 2022 are not necessarily indicative of the results for the year ending December 31, 2022 or for any future period. The accompanying unaudited interim condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, Blueprint Medicines Security Corporation, which is a Massachusetts subsidiary created to buy, sell and hold securities, Blueprint Medicines (Switzerland) GmbH, Blueprint Medicines (Netherlands) B.V., Blueprint Medicines (UK) Ltd, Blueprint Medicines (Germany) GmbH, Blueprint Medicines (Spain) S.L., Blueprint Medicines (France) SAS, and Blueprint Medicines (Italy) S.r.L. 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, liability related to the sale of future royalties, and income taxes. The length of time and full extent to which the ongoing COVID-19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition, including revenues, expenses, reserves and allowances, manufacturing and supply, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain, subject to change and difficult to predict, including as a result of new information that may emerge concerning COVID-19, including the identification and spread of new variants, and the actions taken to contain or treat COVID-19, as well as the economic impact thereof on local, regional, national and international customers and markets. The Company considers the impact of COVID-19 while making the estimates within its consolidated financial statements and there may be changes to those estimates in future periods. 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 The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and six months ended June 30, 2022 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2021 Annual Report on Form 10-K, with the below exception. Liability related to the sale of future royalties ​ The Company accounts for net proceeds from sales of the Company’s rights to receive future royalty payments as a liability related to the sale of future royalties if the Company has significant continuing involvement in the generation of the related future cash flows. Interest on the liability related to the sale of future royalties will be recognized using the effective interest rate method over the life of the related royalty stream. The liability related to the sale of future royalties and the related interest expenses are based on the Company’s current estimates of future royalties and 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 will periodically assess the expected payments and to the extent the amount or timing of the future estimated payments is materially different than previous estimates, the Company will account for any such change by adjusting the liability related to the sale of future royalties and prospectively recognizing the related interest expense. New Accounting Pronouncements

Liability Related to The Sale o

Liability Related to The Sale of Future Royalties	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Liability Related to The Sale of Future Royalties	3. Liability Related to The Sale of Future Royalties ​ 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 are 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 loss. Net proceeds from the transaction were recorded as a liability related to sale of future royalties on the consolidated balance sheet. The Company will amortize the $175.0 million, net of transaction costs of $3.7 million using the effective interest method over the estimated life of the arrangement. 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 received by the Company and paid to Royalty Pharma over the life of the arrangement. The exact amount of repayment is likely to change each reporting period. The Company will periodically assess the expected payments to Royalty Pharma and will prospectively adjust the amortization of the liability related to the sale of future royalties for material changes in such payments. As of June 30, 2022, the carrying value of the liability 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 ​ ​ — Capitalized closing costs ​ ​ (3,746) Payments ​ ​ — Carrying value as of June 30, 2022 ​ $ 171,254 ​ 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 sale of future royalties when the milestones are achieved and received.

Marketable Securities

Marketable Securities	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Marketable Securities	4. Marketable Securities Marketable securities consisted of the following at June 30, 2022 and December 31, 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair June 30, 2022 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 451,134 ​ $ — ​ ​ (6,717) ​ $ 444,417 U.S. treasury obligations ​ ​ 335,643 ​ ​ — ​ ​ (6,300) ​ ​ 329,343 Total ​ $ 786,777 ​ $ — ​ $ (13,017) ​ $ 773,760 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 June 30, 2022, the Company held 77 debt securities that were in an unrealized loss position with an aggregate fair value of $773.8 million. Of the 77 debt securities, 3 were in an unrealized loss position for more than twelve months with an aggregate fair value of $39.2 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. As of December 31, 2021, there were no securities held by the Company in an unrealized loss position for more than twelve months. The Company has the intent and ability to hold such securities until recovery. As a result, the Company did not impairments marketable securities ​ As of June 30, 2022, 14 securities with an aggregate fair value of $94.0 million had remaining maturities between one year and five years. As of December 31, 2021, 56 securities with an aggregate fair value of $557.5 million had remaining maturities between one year and five years. ​ The Company received proceeds of $79.6 million from maturities of debt securities for the three and six months ended June 30, 2022, and $185.1 million and $400.5 million for the three and six months ended June 30, 2021, respectively. The Company did not realize any gains or losses from maturities of debt securities for the three and six months ended June 30, 2022 and 2021.

Fair Value of Financial Instrum

Fair Value of Financial Instruments	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Fair Value of Financial Instruments	5. Fair Value of Financial Instruments The following table summarizes cash equivalents and marketable securities measured at fair value on a recurring basis as of June 30, 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ June 30, ​ Markets ​ Inputs ​ Inputs Description ​ 2022 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 8,940 ​ $ 8,940 ​ $ — ​ $ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 444,417 ​ ​ — ​ ​ 444,417 ​ ​ — U.S. treasury obligations ​ ​ 329,343 ​ ​ 329,343 ​ ​ — ​ ​ — Total ​ $ 782,700 ​ $ 338,283 ​ $ 444,417 ​ $ — ​ The following table summarizes 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 ​ $ — ​

Product Revenue Reserves and Al

Product Revenue Reserves and Allowances	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Product Revenue Reserves and Allowances	6. 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. For additional information, see Note 10, Collaboration and License Agreements The following table summarizes revenue recognized from product sales for the three and six months ended June 30, 2022 and 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2022 2021 2022 2021 AYVAKIT/AYVAKYT ​ $ 28,454 ​ $ 8,555 ​ $ 52,295 ​ $ 15,682 GAVRETO ​ ​ — ​ ​ 2,878 ​ ​ — ​ ​ 4,706 Total product revenue ​ $ 28,454 ​ $ 11,433 ​ $ 52,295 ​ $ 20,388 ​ ​ The following table summarizes activity in each of the product revenue allowance and reserve categories for the six months ended June 30, 2022 and 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended June 30, ​ 2022 ​ 2021 Beginning balance at January 1 $ 4,345 ​ $ 1,192 Provision related to product sales 9,408 ​ ​ 3,199 Adjustment related to prior periods sales (570) ​ ​ — Credits and payments made (6,120) ​ ​ (1,837) Ending balance at June 30 $ 7,063 ​ $ 2,554 ​ ​ The total reserves that are included in the Company’s unaudited condensed consolidated balance sheets as of June 30, 2022 and December 31, 2021, are summarized as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2022 ​ 2021 Reduction of accounts receivable, net $ 912 ​ $ 419 Component of accrued expenses ​ 6,151 ​ ​ 3,926 Total revenue-related reserves $ 7,063 ​ $ 4,345 ​ ​ ​

Inventory

Inventory	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Inventory	7. Inventory Capitalized inventory consists of the following at June 30, 2022 and December 31, 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2022 2021 Raw materials ​ $ 14,443 ​ $ 10,788 Work in process ​ ​ 21,986 ​ ​ 17,702 Finished goods ​ 2,523 ​ 3,916 Total ​ $ 38,952 ​ $ 32,406 ​ Balance sheet classification ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2022 2021 Inventory ​ $ 35,454 ​ $ 21,817 Other assets ​ 3,498 ​ 10,589 Total ​ $ 38,952 ​ $ 32,406 ​ ​ Inventory amounts written down as a result of excess, obsolescence, unmarketability or other reasons are charged to cost of sales. The Company did not recognize material write-down

Restricted Cash

Restricted Cash	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Restricted Cash	8. Restricted Cash At June 30, 2022 and December 31, 2021, $5.2 million and $5.2 million, respectively, of the Company’s cash is restricted by a bank primarily related to security deposits for the Company’s building lease agreements.

Accrued Expenses

Accrued Expenses	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Accrued Expenses	9. Accrued Expenses Accrued expenses consist of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2022 2021 Research, development and commercial contract costs ​ $ 60,166 ​ $ 68,164 Employee compensation ​ ​ 19,959 ​ ​ 29,166 Accrued professional fees ​ 22,519 ​ 12,611 Revenue-related reserves ​ ​ 6,151 ​ ​ 3,926 Other ​ ​ 10,487 ​ ​ 7,962 Total ​ $ 119,282 ​ $ 121,829 ​ ​ ​

Collaboration and License Agree

Collaboration and License Agreements	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Collaboration and License Agreements	10 . Collaboration and License Agreements Proteovant an exclusive as well as up to two additional novel protein degrader target programs as may be mutually agreed to by the Company and Proteovant (each 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 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 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 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 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 will be 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 12 th second 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 three and six months ended June 30, 2022, no material reduction of expenses was recorded from 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 and are recognized as revenue. During the three and six months ended June 30, 2022, the Company recorded $0.5 million and $0.6 million, respectively, in revenue related to Zai Lab territory specific activities in the unaudited condensed consolidated financial statements. For the purposes of ASC 606, the transaction price of the Zai Lab agreement as of 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 during the year ended December 31, 2021. The Company will reevaluate 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 will adjust 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 Under the Roche pralsetinib collaboration agreement, the Company received an upfront cash payment of $675.0 million, and through June 30, 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 June 30, 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 will 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 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 revenue are 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 June 30, 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 will reevaluate 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 will adjust 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 three and six months ended June 30, 2022 and 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2022 2021 2022 2021 Manufacturing and research and development services related to $ 35 $ 1,912 $ 691 $ 3,684 Royalty revenue ​ 313 ​ — ​ 600 — Total Roche pralsetinib collaboration revenue $ 348 $ 1,912 $ 1,291 $ 3,684 ​ ​ 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 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 loss. The following table summarizes the amount recognized from collaboration loss sharing after Roche became the principal for product sales of GAVRETO to customers in the U.S. (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 The Company's share of loss in the U.S. for pralsetinib ​ $ 2,145 ​ $ — ​ $ 5,410 ​ $ — ​ ​ 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 or increases in research and development expenses related to global development activities for pralsetinib under the Roche pralsetinib collaboration during the three and six months ended June 30, 2022 and 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ 2022 2021 2022 2021 Reductions to selling, general and administrative expenses $ 4,416 ​ $ 2,157 $ 9,248 ​ $ 5,155 Reductions to (increases in) research and development expenses $ (3,714) ​ $ 2,072 $ (3,704) ​ $ 6,154 ​ ​ The following table summarizes the contract assets associated with the Roche pralsetinib collaboration as of June 30, 2022 and December 31, 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2022 ​ 2021 Accounts receivable, net ​ $ 980 $ 2,679 Unbilled accounts receivable ​ $ — $ 6,802 ​ ​ In June 2022, although the Company sold its right to receive royalty payments from Roche’s sales of GAVRETO in Roche territory to Royalty Pharma, 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 loss. For additional information, see Note 3 – Liability related to the sale of future royalties 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 June 30, 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 June 30, 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 to purchase 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 on or after the second anniversary of the effective date of the agreement upon at least 12 months’ prior written notice to the Company, which cannot be delivered before the first anniversary of the effective date. 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 as of the outset of the arrangement was $46.5 million, which consisted of the upfront amount of $25.0 million, the $20.0 million cash milestone payment received in the third quarter of 2020 During the three months ended June 30, 2022, no revenue was recognized from the Clementia collaboration. During the six months ended June 30, 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 will reevaluate 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 will adjust 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 three and six months ended June 30, 2021, 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 June 30, 2022 and December 31, 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 June 30, 2022, the Company has achieved an aggregate of $27.0 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 June 30, 2022, the Company will be eligible to receive up to $319.0 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 will be 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 will be 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, 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 three and six months ended June 30, 2022 and 2021 is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 Manufacturing and research and development services related to global development activities, net of expenses payable to CStone ​ $ 662 $ 269 $ 1,150 $ 1,008 ​ ​ 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 agree

Stock-based Compensation

Stock-based Compensation	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Stock-based Compensation	11. 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 share 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 year beginning January 1, 2022, the number of shares reserved for issuance under the 2015 Plan was increased by 2,365,643 shares. 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 June 30, 2022, there were 4,096,926 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 further 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 June 30, 2022, there were 1,606,017 shares available for future grant under the Inducement Plan. Stock options The following table summarizes the stock option activity for the six months ended June 30, 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ ​ Shares ​ ​ Exercise Price Outstanding at December 31, 2021 5,682,022 ​ $ 69.37 Granted 1,013,605 ​ ​ 61.67 Exercised (119,121) ​ ​ 15.39 Canceled (189,289) ​ ​ 79.11 Outstanding at June 30, 2022 6,387,217 ​ $ 68.86 Exercisable at June 30, 2022 3,881,205 ​ $ 64.81 ​ As of June 30, 2022, the total unrecognized compensation expense related to unvested stock option awards was $97.4 million, which is expected to be recognized over a weighted-average period of approximately 2.65 years. Restricted stock units The following table summarizes the restricted stock units activity for the six months ended June 30, 2022: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ Shares Grant Date Fair Value Unvested shares at December 31, 2021 1,590,160 ​ $ 83.85 Granted 743,828 ​ 61.43 Vested (388,087) ​ 79.36 Forfeited (88,534) ​ 78.98 Unvested shares at June 30, 2022 1,857,367 ​ $ 76.02 ​ As of June 30, 2022, the total unrecognized compensation expense related to unvested restricted stock units was $122.4 million, which is expected to be recognize over a weighted-average period of approximately 2.84 years. 2015 Employee Stock Purchase Plan In 2015, the Company’s board of directors and stockholders approved the 2015 Employee Stock Purchase Plan (the 2015 ESPP), which became effective upon the closing of the Company’s initial public offering in May 2015. The Company initially reserved a total of 243,347 shares of common stock for issuance under the 2015 ESPP. The 2015 ESPP provides that the number of shares reserved and available for issuance under the 2015 ESPP will be cumulatively increased on January 1 of each calendar year by 1% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar year beginning January 1, 2022, the number of shares reserved for issuance under the 2015 ESPP was increased by 591,410 shares. Stock-based compensation expense The Company recognized stock-based compensation expense totaling $25.3 million and $48.7 million for the three and six months ended June 30, 2022, respectively, and $24.3 million and $45.0 million for the three and six months ended June 30, 2021, respectively. Stock-based compensation expense by award type included within the unaudited condensed consolidated statements of operations and comprehensive loss was as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ ​ June 30, ​ ​ 2022 ​ 2021 ​ ​ 2022 ​ 2021 Stock options $ 12,811 ​ $ 14,965 ​ $ 25,755 ​ $ 29,107 Restricted stock units ​ 12,396 ​ ​ 9,270 ​ ​ 22,782 ​ ​ 16,064 Employee stock purchase plan ​ 317 ​ ​ 287 ​ ​ 596 ​ ​ 563 Subtotal ​ 25,524 ​ ​ 24,522 ​ ​ 49,133 ​ ​ 45,734 Capitalized stock-based compensation costs ​ (177) ​ ​ (215) ​ ​ (387) ​ ​ (743) Stock-based compensation expense included in total cost and operating expenses $ 25,347 ​ $ 24,307 ​ $ 48,746 ​ $ 44,991 ​ Stock-based compensation expense, that is included in operating expenses, ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 Research and development ​ $ 10,497 $ 10,485 $ 20,538 $ 19,432 Selling, general and administrative ​ 14,850 ​ 13,822 ​ 28,208 ​ 25,559 Total ​ $ 25,347 ​ $ 24,307 ​ $ 48,746 ​ $ 44,991 ​

Net Loss Per Share

Net Loss Per Share	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Net Income (Loss) Per Share	12. Net Loss per Share Basic net loss per share is calculated by dividing net loss by the weighted average shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting weighted average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period. For purposes of the dilutive net loss per share calculation, stock options, unvested restricted stock units and ESPP shares are considered to be common stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented as a result of the Company’s net loss. The following common stock equivalents were excluded from the calculation of diluted net loss per share for the three and six months ended June 30, 2022 and 2021 because including them would have had an anti-dilutive effect (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ 2022 2021 Stock options 6,387 ​ 6,199 Restricted stock units 1,857 ​ 1,556 ESPP shares ​ 46 ​ 25 Total 8,290 7,780 ​ ​ ​

Income Taxes

Income Taxes	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Income Taxes	13. Income Taxes The Company utilizes the asset and liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities using enacted tax rates in effect for years in which the temporary differences are expected to reverse. The Company provides a valuation allowance when it is more likely than not that deferred tax assets will not be realized. The realization of deferred income tax assets is dependent on the generation of sufficient taxable income during future periods in which temporary differences are expected to reverse. Where the realization of such assets does not meet the more likely than not criterion, the Company applies a valuation allowance against the deferred income tax asset under consideration. The valuation allowance is reviewed periodically and if the assessment of the more likely than not criterion changes, the valuation allowance is adjusted accordingly. As of June 30, 2022, the Company has a full valuation allowance applied against its U.S. and foreign deferred tax assets. On March 11, 2021, President Joe Biden signed into law a relief and stimulus package known as the American Rescue Plan Act of 2021 (ARPA) stimulus package. While this Act provides various tax provisions including for example, extending the employee retention credit through the end of 2021, modifying the paid sick and family leave credits, repealing the worldwide interest allocation rules that were scheduled to take effect in 2021, and expanding the number of employees subject to the limit on the deduction for executive compensation under Section 162(m) beginning in 2027, among other things, based on the Company’s initial review of the various business tax provisions offered in the ARPA along with having a valuation allowance on its U.S. deferred tax assets, it does not believe that there is an impact to the Company and as such the recording of a discrete item was not required during the three and six months ended June 30, 2022. 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 prepared an analysis of 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, is considered taxable income for calendar year ended December 31, 2022. Therefore, the $425.0 million is included in the estimated taxable income calculation when estimating the Company’s forecasted 2022 annual effective tax rate. As of June 30, 2022, the Company expects to be in a taxable income position for the calendar year ended December 31, 2022, and recorded an income tax expense of $3.1 million and $3.3 million for the three and six months ended June 30, 2022, respectively.

Leases

Leases	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Leases	14. Leases The Company’s building leases are comprised of office and laboratory spaces under non-cancelable operating leases. The lease agreements contain various clauses for renewal at the Company’s option, and the renewal options were not included in the calculation of the operating lease assets and the operating lease liabilities as the renewal options were not reasonably certain of being exercised as of June 30, 2022. The lease agreements do not contain residual value guarantees and t ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ June 30, ​ June 30, Operating leases: 2022 ​ 2021 ​ 2022 ​ 2021 Lease cost $ 4,942 ​ $ 4,451 ​ $ 10,911 ​ $ 9,177 Sublease income ​ (600) ​ ​ (268) ​ ​ (1,381) ​ ​ (595) Net lease cost $ 4,342 ​ $ 4,183 ​ $ 9,530 ​ $ 8,582 ​ ​ The Company has not entered into any material short-term leases or financing leases as of June 30, 2022. Supplemental cash flow information related to leases for the six months ended June 30, 2022 and 2021 is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ June 30, ​ 2022 ​ 2021 Cash paid for amounts included in the measurement of lease liabilities: $ 7,763 ​ $ 7,394 Lease liabilities arising from obtaining right-of-use assets: ​ ​ ​ ​ ​ Operating leases $ — ​ $ — ​ ​ The weighted average remaining lease term and weighted average discount rate of the operating leases are as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Operating leases Weighted average remaining lease term in years ​ ​ ​ ​ 7.3 Weighted average discount rate ​ ​ ​ ​ 7.4% ​

Commitments and Contingencies

Commitments and Contingencies	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Commitments and Contingencies	15. 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. During the six months ended June 30, 2022, there were no material changes to the Company’s contractual obligations . 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 from their status or service as directors or officers of the Company. The maximum potential amount of future payments that the Company could be required to make, or otherwise be liable for, 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 June 30, 2022 or December 31, 2021.

Subsequent Events

Subsequent Events	6 Months Ended
	Jun. 30, 2022
Disclosure Text Block
Subsequent Events	​ ​ 16. Subsequent events Synthetic Royalty Facility ​ In July 2022, the Company closed a transaction pursuant to a purchase and sale agreement with Sixth Street Partners and 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, if it is approved, BLU-263, excluding in Greater China, and (ii) aggregate worldwide annual net product sales of BLU-263, 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. Net proceeds from the transaction will be recorded as a liability on the consolidated balance sheet. Debt Facility In July 2022, the Company closed on a financing transaction for up to $660.0 million with Sixth Street Partners (financing agreement). 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 loans will mature on June 30, 2028 and bear interest at a variable rate equal to either the Secured Overnight Financing Rate (SOFR) plus six and one half percent ( 6.50% ) or the base rate plus five and one half percent ( 5.50% ), subject to a floor of one percent ( 1% ) and two percent ( 2% ) with respect to the SOFR and base rate, respectively. The initial gross proceeds of $150.0 million was funded in July 2022. In addition, the Company may at any time request an incremental term loan in an amount not to exceed $260.0 million on terms to be agreed and subject to the consent of the lenders providing such incremental term loan. The Company’s obligations under the financing agreement will be 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 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 have 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. License Agreement and Stock Purchase and Rights Agreement with IDRx ​ In August 2022, the Company entered into a license agreement and a stock purchase and rights agreement with IDRx, Inc., a recently launched clinical-stage biopharmaceutical company. Pursuant to the agreements, the Company licensed its internally discovered KIT exon 13 inhibitor to IDRx in exchange for a 15% Series A preferred equity investment in IDRx and the eligibility to receive future milestone and tiered royalty payments. Among IDRx’s founders are Alexis Borisy, George Demetri, M.D., and Nicholas Lydon, Ph.D., who each currently serve as members of the board of directors of the Company .

Summary of Significant Accoun_2

Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Policies)	6 Months Ended
	Jun. 30, 2022
Policy Text Blocks
Basis of Presentation	Basis of Presentation The unaudited interim condensed consolidated financial statements of the Company included herein have been prepared in accordance with accounting principles generally accepted in the United States (GAAP) as found in the Accounting Standards Codification (ASC), Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB) and the rules and regulations of the Securities and Exchange Commission (SEC). Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these financial statements should be read in conjunction with the financial statements as of and for the year ended December 31, 2021 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, filed with the SEC on February 17, 2022 (2021 Annual Report on Form 10-K). The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited financial statements, and updated, as necessary, in this report. In the opinion of the Company’s management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments that are necessary to present fairly the Company’s financial position as of June 30, 2022, the results of its operations for the three and six months ended June 30, 2022 and 2021, stockholder’s equity for the three and six months ended June 30, 2022 and 2021 and cash flows for six months ended June 30, 2022 and 2021. Such adjustments are of a normal and recurring nature. The results for the three and six months ended June 30, 2022 are not necessarily indicative of the results for the year ending December 31, 2022 or for any future period. The accompanying unaudited interim condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, Blueprint Medicines Security Corporation, which is a Massachusetts subsidiary created to buy, sell and hold securities, Blueprint Medicines (Switzerland) GmbH, Blueprint Medicines (Netherlands) B.V., Blueprint Medicines (UK) Ltd, Blueprint Medicines (Germany) GmbH, Blueprint Medicines (Spain) S.L., Blueprint Medicines (France) SAS, and Blueprint Medicines (Italy) S.r.L. 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	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, liability related to the sale of future royalties, and income taxes. The length of time and full extent to which the ongoing COVID-19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition, including revenues, expenses, reserves and allowances, manufacturing and supply, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain, subject to change and difficult to predict, including as a result of new information that may emerge concerning COVID-19, including the identification and spread of new variants, and the actions taken to contain or treat COVID-19, as well as the economic impact thereof on local, regional, national and international customers and markets. The Company considers the impact of COVID-19 while making the estimates within its consolidated financial statements and there may be changes to those estimates in future periods. 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	Significant Accounting Policies The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and six months ended June 30, 2022 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2021 Annual Report on Form 10-K, with the below exception.
Liability related to the sale of future royalties	Liability related to the sale of future royalties ​ The Company accounts for net proceeds from sales of the Company’s rights to receive future royalty payments as a liability related to the sale of future royalties if the Company has significant continuing involvement in the generation of the related future cash flows. Interest on the liability related to the sale of future royalties will be recognized using the effective interest rate method over the life of the related royalty stream. The liability related to the sale of future royalties and the related interest expenses are based on the Company’s current estimates of future royalties and 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 will periodically assess the expected payments and to the extent the amount or timing of the future estimated payments is materially different than previous estimates, the Company will account for any such change by adjusting the liability related to the sale of future royalties and prospectively recognizing the related interest expense.
New Accounting Pronouncements	New Accounting Pronouncements

Liability Related to The Sale_2

Liability Related to The Sale of Future Royalties (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Schedule of activity within the liability account	​ ​ ​ ​ ​ Carrying value as of January 1, 2022 ​ $ — Sale of future royalties ​ ​ 175,000 Interest expense recognized ​ ​ — Capitalized closing costs ​ ​ (3,746) Payments ​ ​ — Carrying value as of June 30, 2022 ​ $ 171,254 ​

Marketable Securities (Tables)

Marketable Securities (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Schedule of marketable securities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair June 30, 2022 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 451,134 ​ $ — ​ ​ (6,717) ​ $ 444,417 U.S. treasury obligations ​ ​ 335,643 ​ ​ — ​ ​ (6,300) ​ ​ 329,343 Total ​ $ 786,777 ​ $ — ​ $ (13,017) ​ $ 773,760 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ 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 ​

Fair Value of Financial Instr_2

Fair Value of Financial Instruments (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Schedule of financial instruments measured at fair value	The following table summarizes cash equivalents and marketable securities measured at fair value on a recurring basis as of June 30, 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ June 30, ​ Markets ​ Inputs ​ Inputs Description ​ 2022 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 8,940 ​ $ 8,940 ​ $ — ​ $ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 444,417 ​ ​ — ​ ​ 444,417 ​ ​ — U.S. treasury obligations ​ ​ 329,343 ​ ​ 329,343 ​ ​ — ​ ​ — Total ​ $ 782,700 ​ $ 338,283 ​ $ 444,417 ​ $ — ​ The following table summarizes 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 ​ $ —

Product Revenue Reserves and _2

Product Revenue Reserves and Allowances (Tables) - Product revenue, net	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Summary of revenue recognized	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2022 2021 2022 2021 AYVAKIT/AYVAKYT ​ $ 28,454 ​ $ 8,555 ​ $ 52,295 ​ $ 15,682 GAVRETO ​ ​ — ​ ​ 2,878 ​ ​ — ​ ​ 4,706 Total product revenue ​ $ 28,454 ​ $ 11,433 ​ $ 52,295 ​ $ 20,388 ​
Schedule of product revenue allowance and reserve categories	​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended June 30, ​ 2022 ​ 2021 Beginning balance at January 1 $ 4,345 ​ $ 1,192 Provision related to product sales 9,408 ​ ​ 3,199 Adjustment related to prior periods sales (570) ​ ​ — Credits and payments made (6,120) ​ ​ (1,837) Ending balance at June 30 $ 7,063 ​ $ 2,554 ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2022 ​ 2021 Reduction of accounts receivable, net $ 912 ​ $ 419 Component of accrued expenses ​ 6,151 ​ ​ 3,926 Total revenue-related reserves $ 7,063 ​ $ 4,345 ​

Inventory (Tables)

Inventory (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Schedule of capitalized inventory	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2022 2021 Raw materials ​ $ 14,443 ​ $ 10,788 Work in process ​ ​ 21,986 ​ ​ 17,702 Finished goods ​ 2,523 ​ 3,916 Total ​ $ 38,952 ​ $ 32,406 ​ Balance sheet classification ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2022 2021 Inventory ​ $ 35,454 ​ $ 21,817 Other assets ​ 3,498 ​ 10,589 Total ​ $ 38,952 ​ $ 32,406 ​

Accrued Expenses (Tables)

Accrued Expenses (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Schedule of accrued expenses	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2022 2021 Research, development and commercial contract costs ​ $ 60,166 ​ $ 68,164 Employee compensation ​ ​ 19,959 ​ ​ 29,166 Accrued professional fees ​ 22,519 ​ 12,611 Revenue-related reserves ​ ​ 6,151 ​ ​ 3,926 Other ​ ​ 10,487 ​ ​ 7,962 Total ​ $ 119,282 ​ $ 121,829 ​

Collaboration and License Agr_2

Collaboration and License Agreements (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Summary of manufacturing and research and development services related to the global development activities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 Manufacturing and research and development services related to global development activities, net of expenses payable to CStone ​ $ 662 $ 269 $ 1,150 $ 1,008 ​
Schedule of collaboration loss sharing	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 The Company's share of loss in the U.S. for pralsetinib ​ $ 2,145 ​ $ — ​ $ 5,410 ​ $ — ​
Schedule of amounts recognized as reductions to expenses	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ 2022 2021 2022 2021 Reductions to selling, general and administrative expenses $ 4,416 ​ $ 2,157 $ 9,248 ​ $ 5,155 Reductions to (increases in) research and development expenses $ (3,714) ​ $ 2,072 $ (3,704) ​ $ 6,154 ​
Roche, Collaboration (Pralsetnib) Agreement
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Summary of revenue recognized	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2022 2021 2022 2021 Manufacturing and research and development services related to $ 35 $ 1,912 $ 691 $ 3,684 Royalty revenue ​ 313 ​ — ​ 600 — Total Roche pralsetinib collaboration revenue $ 348 $ 1,912 $ 1,291 $ 3,684 ​
Roche, Collaboration (Pralsetnib) Agreement | Collaboration revenue
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Summary of contract assets and/or contract liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2022 ​ 2021 Accounts receivable, net ​ $ 980 $ 2,679 Unbilled accounts receivable ​ $ — $ 6,802 ​
Roche, Collaboration and License (Immunotherapy) Agreement
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Summary of revenue recognized	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 Roche collaboration research and development services revenue ​ $ 1,183 $ 1,349 $ (765) $ 2,885 ​
Roche, Collaboration and License (Immunotherapy) Agreement | Collaboration revenue
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Summary of contract assets and/or contract liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 Amounts included in the contract liability at the beginning of the period ​ $ 1,395 $ 1,064 $ 2,405 $ 2,302 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, 2022 December 31, 2021 ​ Current Noncurrent Total Current Noncurrent Total Deferred revenue $ 4,903 $ ​ 12,661 $ ​ 17,564 $ 6,339 $ ​ 25,066 $ ​ 31,405 Accrued expenses 7,780 ​ 6,157 ​ 13,937 — ​ — ​ — ​
C Stone
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Summary of revenue recognized	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 License milestone revenue ​ $ — $ — $ 4,000 $ 9,000 Manufacturing services and royalty revenue related to CStone territory-specific activities ​ ​ 6,082 ​ ​ 12,302 ​ ​ 11,294 ​ ​ 12,573 Total CStone collaboration revenue ​ $ 6,082 ​ $ 12,302 ​ $ 15,294 ​ $ 21,573 ​
C Stone | Collaboration revenue
Table Text Blocks
Summary of contract assets and/or contract liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2022 ​ 2021 Accounts receivable, net ​ $ 11,858 $ 8,164 Unbilled accounts receivable ​ $ 1,873 $ 5,034

Stock-based Compensation (Table

Stock-based Compensation (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Summary of stock option activity	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ ​ Shares ​ ​ Exercise Price Outstanding at December 31, 2021 5,682,022 ​ $ 69.37 Granted 1,013,605 ​ ​ 61.67 Exercised (119,121) ​ ​ 15.39 Canceled (189,289) ​ ​ 79.11 Outstanding at June 30, 2022 6,387,217 ​ $ 68.86 Exercisable at June 30, 2022 3,881,205 ​ $ 64.81
Summary of restricted stock units activity	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ Shares Grant Date Fair Value Unvested shares at December 31, 2021 1,590,160 ​ $ 83.85 Granted 743,828 ​ 61.43 Vested (388,087) ​ 79.36 Forfeited (88,534) ​ 78.98 Unvested shares at June 30, 2022 1,857,367 ​ $ 76.02
Summary of stock-based compensation expense, allocation by type of awards and recognition in statements of operations	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ ​ June 30, ​ ​ 2022 ​ 2021 ​ ​ 2022 ​ 2021 Stock options $ 12,811 ​ $ 14,965 ​ $ 25,755 ​ $ 29,107 Restricted stock units ​ 12,396 ​ ​ 9,270 ​ ​ 22,782 ​ ​ 16,064 Employee stock purchase plan ​ 317 ​ ​ 287 ​ ​ 596 ​ ​ 563 Subtotal ​ 25,524 ​ ​ 24,522 ​ ​ 49,133 ​ ​ 45,734 Capitalized stock-based compensation costs ​ (177) ​ ​ (215) ​ ​ (387) ​ ​ (743) Stock-based compensation expense included in total cost and operating expenses $ 25,347 ​ $ 24,307 ​ $ 48,746 ​ $ 44,991 Stock-based compensation expense, that is included in operating expenses, ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2022 2021 ​ 2022 2021 Research and development ​ $ 10,497 $ 10,485 $ 20,538 $ 19,432 Selling, general and administrative ​ 14,850 ​ 13,822 ​ 28,208 ​ 25,559 Total ​ $ 25,347 ​ $ 24,307 ​ $ 48,746 ​ $ 44,991

Net Loss Per Share (Tables)

Net Loss Per Share (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Schedule of common stock equivalents excluded from calculation of diluted net loss per share applicable to common stockholders	​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ 2022 2021 Stock options 6,387 ​ 6,199 Restricted stock units 1,857 ​ 1,556 ESPP shares ​ 46 ​ 25 Total 8,290 7,780 ​

Leases (Tables)

Leases (Tables)	6 Months Ended
	Jun. 30, 2022
Table Text Blocks
Summary of lease expenses and cash flow and weighted average information	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ June 30, ​ June 30, Operating leases: 2022 ​ 2021 ​ 2022 ​ 2021 Lease cost $ 4,942 ​ $ 4,451 ​ $ 10,911 ​ $ 9,177 Sublease income ​ (600) ​ ​ (268) ​ ​ (1,381) ​ ​ (595) Net lease cost $ 4,342 ​ $ 4,183 ​ $ 9,530 ​ $ 8,582 ​ ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ June 30, ​ 2022 ​ 2021 Cash paid for amounts included in the measurement of lease liabilities: $ 7,763 ​ $ 7,394 Lease liabilities arising from obtaining right-of-use assets: ​ ​ ​ ​ ​ Operating leases $ — ​ $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Operating leases Weighted average remaining lease term in years ​ ​ ​ ​ 7.3 Weighted average discount rate ​ ​ ​ ​ 7.4%

Nature of Business (Details)

Nature of Business (Details) $ in Millions	Jun. 30, 2022 USD ($)
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities	$ 947.2

Liability Related to The Sale_3

Liability Related to The Sale of Future Royalties - General Information (Details) - USD ($) $ in Thousands		1 Months Ended	2 Months Ended	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2022	Jul. 31, 2022	Jun. 30, 2022
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties				$ 175,000
Sale of Future Royalties
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties			$ 425,000
Sale of Future Royalties | Royalty Pharma, Royalty Purchase Agreement
Debt Financing
Debt instrument, issuance date	Jun. 30, 2022
Debt instrument, face amount	$ 175,000	$ 175,000		175,000
Transaction costs	3,700	3,700		3,700
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties	$ 175,000	$ 175,000		$ 175,000

Liability Related to The Sale_4

Liability Related to The Sale of Future Royalties - Long-term Debt (Details) - Sale of Future Royalties - Royalty Pharma, Royalty Purchase Agreement - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Long-term debt
Long-term debt, gross	$ 175,000
Transaction costs	3,700
Long-term debt	$ 171,254	$ 0

Liability Related to The Sale_5

Liability Related to The Sale of Future Royalties - Roll Forward (Details) - USD ($) $ in Thousands		1 Months Ended	2 Months Ended	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2022	Jul. 31, 2022	Jun. 30, 2022
Long-term debt
Sale of future royalties				$ 175,000
Sale of Future Royalties
Long-term debt
Sale of future royalties			$ 425,000
Sale of Future Royalties | Royalty Pharma, Royalty Purchase Agreement
Long-term debt
Carrying value, beginning balance				0
Sale of future royalties	$ 175,000	$ 175,000		175,000
Capitalized closing costs				(3,746)
Carrying value, ending balance	$ 171,254	$ 171,254		$ 171,254

Liability Related to The Sale_6

Liability Related to The Sale of Future Royalties - Additional Information (Details) $ in Millions	Jun. 30, 2022 USD ($)
Debt Financing
Royalty Purchase Agreement, milestone payments, achievement of specified net sales milestones, maximum	$ 165

Marketable Securities - Tabular

Marketable Securities - Tabular Disclosure (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Marketable securities, available-for-sale:
Amortized cost	$ 786,777	$ 827,383
Unrealized gain		21
Unrealized losses	(13,017)	(2,709)
Fair value	773,760	824,695
U.S. government agency securities
Marketable securities, available-for-sale:
Amortized cost	451,134	498,582
Unrealized gain		21
Unrealized losses	(6,717)	(1,460)
Fair value	444,417	497,143
U.S. Treasury obligations
Marketable securities, available-for-sale:
Amortized cost	335,643	328,801
Unrealized losses	(6,300)	(1,249)
Fair value	$ 329,343	$ 327,552

Marketable Securities - Unreali

Marketable Securities - Unrealized Loss Position (Details) $ in Millions	Jun. 30, 2022 USD ($) security	Dec. 31, 2021 USD ($) security
Unrealized loss position, number of positions
Number of held securities in an unrealized loss position | security	77	74
Number of held securities in an unrealized loss position for 12 months or longer | security	3	0
Unrealized loss position, aggregate fair value
Unrealized loss position | $	$ 773.8	$ 750.5
Unrealized loss position for more than 12 months | $	$ 39.2

Marketable Securities - Impairm

Marketable Securities - Impairment (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Marketable Securities
Marketable debt securities, charges for credit-related impairments	$ 0	$ 0	$ 0	$ 0

Marketable Securities - Remaini

Marketable Securities - Remaining Maturities (Details) $ in Millions	Jun. 30, 2022 USD ($) security	Dec. 31, 2021 USD ($) security
Investments, available-for-sale
Securities with remaining maturities greater than one year | security	14	56
Aggregate fair value with remaining maturities greater than one year | $	$ 94	$ 557.5

Marketable Securities - Proceed

Marketable Securities - Proceeds from Maturities (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Proceeds from the maturities of debt securities
Proceeds from the maturities of debt securities	$ 79,600	$ 185,100	$ 79,623	$ 400,500

Marketable Securities - Realize

Marketable Securities - Realized Gains (Losses) (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Realized gains (losses) from maturities of debt securities
Realized gains (losses) from maturities of debt securities	$ 0	$ 0	$ 0	$ 0

Fair Value of Financial Instr_3

Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 773,760	$ 824,695
U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	444,417	497,143
U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	329,343	327,552
Recurring
Fair Value of Financial Instruments
Total	782,700	943,575
Recurring | U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	444,417	497,143
Recurring | U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	329,343	327,552
Recurring | Money market funds
Fair Value of Financial Instruments
Cash equivalents	8,940	118,880
Recurring | Active Markets (Level 1)
Fair Value of Financial Instruments
Total	338,283	446,432
Recurring | Active Markets (Level 1) | U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	329,343	327,552
Recurring | Active Markets (Level 1) | Money market funds
Fair Value of Financial Instruments
Cash equivalents	8,940	118,880
Recurring | Observable Inputs (Level 2)
Fair Value of Financial Instruments
Total	444,417	497,143
Recurring | Observable Inputs (Level 2) | U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 444,417	$ 497,143

Product Revenue Reserves and _3

Product Revenue Reserves and Allowances - Product Revenue (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Revenues
Revenue	$ 36,547	$ 27,295	$ 99,278	$ 48,871
Product revenue, net
Revenues
Revenue	28,454	11,433	52,295	20,388
AYVAKIT and AYVAKYT
Revenues
Revenue	$ 28,454	8,555	$ 52,295	15,682
GAVRETO
Revenues
Revenue		$ 2,878		$ 4,706

Product Revenue Reserves and _4

Product Revenue Reserves and Allowances - Product Revenue Allowance and Reserve (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021
Product revenue allowance and reserve
Beginning balance	$ 4,345	$ 1,192
Provision related to sales in the current period	9,408	3,199
Adjustment related to prior periods sales	(570)
Credits and payments made	(6,120)	(1,837)
Ending balance	$ 7,063	$ 2,554

Product Revenue Reserves and _5

Product Revenue Reserves and Allowances - Revenue-related Reserves (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021	Jun. 30, 2021	Dec. 31, 2020
Revenue-related reserves
Revenue-related reserves	$ 7,063	$ 4,345	$ 2,554	$ 1,192
Accounts Receivable, Net
Revenue-related reserves
Revenue-related reserves	912	419
Accrued Expenses
Revenue-related reserves
Revenue-related reserves	$ 6,151	$ 3,926

Inventory - Capitalized (Detail

Inventory - Capitalized (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Inventory, Noncurrent [Abstract]
Raw materials	$ 14,443	$ 10,788
Work in process	21,986	17,702
Finished goods	2,523	3,916
Total	$ 38,952	$ 32,406

Inventory - Balance Sheet Class

Inventory - Balance Sheet Classification (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Inventory
Inventory	$ 35,454	$ 21,817
Other assets	3,498	10,589
Total	$ 38,952	$ 32,406

Inventory - Write-down (Details

Inventory - Write-down (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Inventory
Inventory write-down	$ 0	$ 0	$ 0	$ 0

Restricted Cash (Details)

Restricted Cash (Details) - USD ($) $ in Millions	Jun. 30, 2022	Dec. 31, 2021
Restricted cash
Restricted cash	$ 5.2	$ 5.2

Accrued Expenses (Details)

Accrued Expenses (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Accrued Expenses
External research and development	$ 60,166	$ 68,164
Employee compensation	19,959	29,166
Accrued professional fees	22,519	12,611
Revenue-related reserves	6,151	3,926
Other	10,487	7,962
Total	$ 119,282	$ 121,829

Collaboration and License Agr_3

Collaboration and License Agreements - General Information (Details) $ in Thousands

1 Months Ended

2 Months Ended

3 Months Ended

6 Months Ended

Feb. 26, 2022 USD ($)

Oct. 15, 2019 USD ($)

Jun. 01, 2018 USD ($) item

Jul. 31, 2020 USD ($)

Oct. 31, 2019 USD ($)

Mar. 31, 2016 USD ($) item

Dec. 31, 2021 USD ($)

Jun. 30, 2022 USD ($)

Jun. 30, 2021 USD ($)

Sep. 30, 2020 USD ($)

Dec. 31, 2019 item

Jun. 30, 2022 USD ($) item

Jun. 30, 2021 USD ($)

Mar. 31, 2022 USD ($)

Nov. 08, 2021 USD ($)

Jul. 13, 2020 USD ($)

Collaboration Agreements

Prepaid expenses and other current assets

$ 18,064

$ 34,204

$ 34,204

Research and development expense

128,466

$ 80,027

231,599

$ 159,738

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

1,000

$ 1,500

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

Collaborative arrangement, milestones, specified regulatory and commercialization milestones, achieved

$ 105,000

Collaborative arrangement, percentage of global development costs shared, entity (as a percent)

45%

Collaborative arrangement, percentage of global development costs shared, counterparty (as a percent)

55%

Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement

Collaboration Agreements

Collaborative arrangement, upfront payment, cash payment received

$ 45,000

Collaborative arrangement, milestones, cash payments received, aggregate

23,500

23,500

Collaborative arrangement, option fees and milestones, contingent payments eligible to receive

$ 319,300

Collaborative arrangement, milestones, research milestones, achieved

23,500

$ 23,500

Collaborative arrangement, license option rights, number | item

5

Collaborative arrangement, collaboration programs, number | item

2

Collaborative arrangement, collaboration programs, licensed products, commercialization rights, counterparty exercises option, counterparty receives worldwide exclusivity, number | item

2

Collaborative arrangement, collaboration programs, licensed products, commercialization rights, counterparty exercises option, entity retains domestic and counterparty receives international, number | item

1

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

Collaborative Arrangement | Clementia

Collaboration Agreements

Collaborative arrangement, upfront payment, cash payment received

$ 25,000

Collaborative arrangement, milestones, cash payments received

$ 20,000

$ 50,000

Collaborative arrangement, milestones, contingent payments eligible to receive

460,000

Collaborative arrangement, inventory purchased by counterparty

$ 1,500

Collaborative arrangement, termination, counterparty written notice period

12 months

Collaborative Arrangement | C Stone

Collaboration Agreements

Collaborative arrangement, upfront payment, cash payment received

$ 40,000

Collaborative arrangement, milestones, contingent payments eligible to receive

$ 319,000

Collaborative arrangement, milestones, achieved

$ 27,000

$ 27,000

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 Agr_4

Collaboration and License Agreements - Transaction Price (Details) - Collaborative Arrangement - USD ($) $ in Millions				1 Months Ended	2 Months Ended	3 Months Ended	6 Months Ended	12 Months Ended
	Jul. 13, 2020	Oct. 15, 2019	Jun. 01, 2018	Mar. 31, 2016	Dec. 31, 2021	Mar. 31, 2022	Jun. 30, 2022	Dec. 31, 2021
Zai Lab (Shanghai) Co., Ltd.
Collaboration Agreements
Collaborative arrangement, transaction price					$ 25
Roche, Collaboration (Pralsetnib) Agreement
Collaboration Agreements
Collaborative arrangement, transaction price	$ 695.7
Collaborative arrangement, transaction price, upfront payment	675
Collaborative arrangement, transaction price, stock issued, premium on sale of stock to counterparty	$ 20.7
Collaborative arrangement, transaction price, milestone payments receivable							$ 105
Roche, Collaboration and License (Immunotherapy) Agreement
Collaboration Agreements
Collaborative arrangement, transaction price				$ 45			$ 53.5	$ 68.5
Collaborative arrangement, transaction price, payment commitment reduction						$ 15
Clementia
Collaboration Agreements
Collaborative arrangement, transaction price		$ 46.5
Collaborative arrangement, transaction price, upfront payment		25
Collaborative arrangement, transaction price, milestone payments receivable		20
Collaborative arrangement, transaction price, inventory purchased by counterparty, existing manufacturing inventory		1.2
Collaborative arrangement, transaction price, inventory purchased by counterparty, in-process manufacturing inventory		$ 0.3
C Stone
Collaboration Agreements
Collaborative arrangement, transaction price			$ 40

Collaboration and License Agr_5

Collaboration and License Agreements - Sale of Stock (Details) - Private Placement $ / shares in Units, $ in Millions	Jul. 13, 2020 USD ($) $ / shares shares
Sale of Stock
Stock issued (in shares) | shares	1,035,519
Share price (in dollars per share) | $ / shares	$ 96.57
Purchase consideration	$ 100
Issuance of common stock	79.3
Share purchase consideration, premium	$ 20.7

Collaboration and License Agr_6

Collaboration and License Agreements - Manufacturing and Research and Development Services (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Collaborative Arrangement | C Stone
Collaboration Agreements
Manufacturing and research and development services related to global development activities, net of expenses payable	$ 662	$ 269	$ 1,150	$ 1,008

Collaboration and License Agr_7

Collaboration and License Agreements - Revenue Recognized (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended		12 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021	Dec. 31, 2021	Dec. 31, 2019	Dec. 31, 2018
Revenues
Revenue	$ 36,547	$ 27,295	$ 99,278	$ 48,871
Collaboration revenue
Revenues
Revenue	8,093	15,862	46,983	28,483
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	348	1,912	1,291	3,684
Collaborative Arrangement | Collaboration revenue | Roche, Collaboration and License (Immunotherapy) Agreement
Revenues
Revenue, net	1,183	1,349	(765)	2,885
Collaborative Arrangement | Collaboration revenue | Clementia
Revenues
Revenue						$ 46,200
Collaborative Arrangement | Collaboration revenue | C Stone
Revenues
Revenue	6,082	12,302	15,294	21,573			$ 40,000
Collaborative Arrangement | License milestone revenue | C Stone
Revenues
Revenue	0	0	4,000	9,000
Collaborative Arrangement | Territory-specific activities | Zai Lab (Shanghai) Co., Ltd.
Revenues
Revenue	500		600
Collaborative Arrangement | Territory-specific activities, manufacturing services and royalty | C Stone
Revenues
Revenue	6,082	12,302	11,294	12,573
Collaborative Arrangement | Territory-specific activities, manufacturing and research and development services | Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue	35	$ 1,912	691	$ 3,684
Collaborative Arrangement | Royalty | Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue	$ 313		$ 600

Collaboration and License Agr_8

Collaboration and License Agreements - Collaboration Loss Sharing (Details) - USD ($) $ in Thousands	3 Months Ended	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2022
Collaboration Agreements
Collaboration loss sharing	$ 2,145	$ 5,410
Collaborative Arrangement | Roche, Collaboration (Pralsetnib) Agreement
Collaboration Agreements
Collaboration loss sharing	$ 2,145	$ 5,410

Collaboration and License Agr_9

Collaboration and License Agreements - Reduction in Expenses (Details) - Collaborative Arrangement - Roche, Collaboration (Pralsetnib) Agreement - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Collaboration Agreements
Collaborative arrangement, commercialization, reduction in selling, general and administrative expenses	$ 4,416	$ 2,157	$ 9,248	$ 5,155
Collaborative arrangement, global development activities, reduction in research and development expenses	$ (3,714)	$ 2,072	$ (3,704)	$ 6,154

Collaboration and License Ag_10

Collaboration and License Agreements - Change in Contract with Customer, Liability (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
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	$ 1,395	$ 1,064	$ 2,405	$ 2,302

Collaboration and License Ag_11

Collaboration and License Agreements - Contract Assets (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Collaboration Agreements
Accounts receivable	$ 27,123	$ 25,155
Unbilled accounts receivable	2,287	11,875
Collaborative Arrangement | Roche, Collaboration (Pralsetnib) Agreement
Collaboration Agreements
Accounts receivable	980	2,679
Unbilled accounts receivable		6,802
Collaborative Arrangement | C Stone
Collaboration Agreements
Accounts receivable	11,858	8,164
Unbilled accounts receivable	$ 1,873	$ 5,034

Collaboration and License Ag_12

Collaboration and License Agreements - Liabilities (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Contract Liabilities
Contract liabilities, current	$ 9,478	$ 11,510
Contract liabilities, noncurrent	12,661	25,066
Accrued expenses, current	60,166	68,164
Collaborative Arrangement | Clementia
Contract Liabilities
Contract liabilities	0	0
Collaborative Arrangement | C Stone
Contract Liabilities
Contract liabilities	4,600	4,800
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement
Contract Liabilities
Contract liabilities	17,564	31,405
Contract liabilities, current	4,903	6,339
Contract liabilities, noncurrent	12,661	$ 25,066
Accrued expenses	13,937
Accrued expenses, current	7,780
Accrued expenses, noncurrent	6,157
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement, Phase I Clinical Trials
Contract Liabilities
Accrued expenses	$ 15,000

Collaboration and License Ag_13

Collaboration and License Agreements - Performance Obligations (Details) - Collaborative Arrangement	6 Months Ended
	Jun. 30, 2022 component item
Zai Lab (Shanghai) Co., Ltd.
Performance Obligations
Collaborative arrangement, material promises, number	3
Collaborative arrangement, material promises, combined into distinct performance obligation, number	2
Collaborative arrangement, material components, number | component	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 | component	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]: 2022-07-01
Performance Obligations
Revenue, remaining performance obligation, expected timing of satisfaction, period	3 years 6 months
Clementia
Performance Obligations
Collaborative arrangement, material promises, number	4
Revenue, performance obligation, performance obligations, number	3
C Stone
Performance Obligations
Collaborative arrangement, material promises, number	6
Collaborative arrangement, material components, number | component	2
Revenue, performance obligation, performance obligations, number	3

Stock-based Compensation - 2015

Stock-based Compensation - 2015 Stock Option and Incentive Plan (Details) - 2015 Stock Option and Incentive Plan - shares		6 Months Ended
	Jan. 01, 2022	Jun. 30, 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)		4%
Increase in number of shares available for grant (in shares)	2,365,643
Number of shares available for grant (in shares)		4,096,926

Stock-based Compensation - 2020

Stock-based Compensation - 2020 Inducement Plan (Details) - 2020 Inducement Plan - shares	1 Months Ended
	Jun. 30, 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)	1,606,017

Stock-based Compensation - Stoc

Stock-based Compensation - Stock Options - Activity (Details)	6 Months Ended
	Jun. 30, 2022 $ / shares shares
Shares
Outstanding at beginning of period (in shares) | shares	5,682,022
Granted (in shares) | shares	1,013,605
Exercised (in shares) | shares	(119,121)
Cancelled (in shares) | shares	(189,289)
Outstanding at end of period (in shares) | shares	6,387,217
Weighted-Average Exercise Price
Outstanding at beginning of period (in dollars per share) | $ / shares	$ 69.37
Granted (in dollars per share) | $ / shares	61.67
Exercised (in dollars per share) | $ / shares	15.39
Cancelled (in dollars per share) | $ / shares	79.11
Outstanding at end of period (in dollars per share) | $ / shares	$ 68.86
Additional disclosures
Shares - Exercisable (in shares) | shares	3,881,205
Weighted-Average Exercise Price - Exercisable (in dollars per share) | $ / shares	$ 64.81

Stock-based Compensation - Unre

Stock-based Compensation - Unrecognized Compensation Costs (Details) $ in Millions	Jun. 30, 2022 USD ($)
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock option awards	$ 97.4
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 7 months 24 days
Restricted Stock Units
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock awards	$ 122.4
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 10 months 2 days

Stock-based Compensation - Rest

Stock-based Compensation - Restricted Stock Units - Activity (Details) - Restricted Stock Units	6 Months Ended
	Jun. 30, 2022 $ / shares shares
Shares
Unvested at beginning of period (in shares) | shares	1,590,160
Granted (in shares) | shares	743,828
Vested (in shares) | shares	(388,087)
Forfeited (in shares) | shares	(88,534)
Unvested at end of period (in shares) | shares	1,857,367
Weighted-Average Grant Date Fair Value
Unvested at beginning or period (in dollars per share) | $ / shares	$ 83.85
Granted (in dollars per share) | $ / shares	61.43
Vested (in dollars per share) | $ / shares	79.36
Forfeited (in dollars per share) | $ / shares	78.98
Unvested at end of period (in dollars per share) | $ / shares	$ 76.02

Stock-based Compensation - Empl

Stock-based Compensation - Employee Stock Purchase Plan (Details) - Employee Stock - shares	1 Months Ended
	May 31, 2015	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%
Increase of common shares reserved for future issuance (in shares)		591,410

Stock-based Compensation - St_2

Stock-based Compensation - Stock-based Compensation Expense - General Information (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Total stock based compensation expense
Stock-based compensation expense	$ 25,347	$ 24,307	$ 48,746	$ 44,991

Stock-based Compensation - St_3

Stock-based Compensation - Stock-based Compensation Expense - By Type (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Total stock based compensation expense
Subtotal	$ 25,524	$ 24,522	$ 49,133	$ 45,734
Capitalized stock-based compensation costs	(177)	(215)	(387)	(743)
Stock-based compensation expense included in total cost and operating expenses	25,347	24,307	48,746	44,991
Stock Options
Total stock based compensation expense
Subtotal	12,811	14,965	25,755	29,107
Restricted Stock Units
Total stock based compensation expense
Subtotal	12,396	9,270	22,782	16,064
Employee Stock
Total stock based compensation expense
Subtotal	$ 317	$ 287	$ 596	$ 563

Stock-based Compensation - St_4

Stock-based Compensation - Stock-based Compensation Expense - By Classification (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Total stock based compensation expense
Stock-based compensation expense	$ 25,347	$ 24,307	$ 48,746	$ 44,991
Research and Development
Total stock based compensation expense
Stock-based compensation expense	10,497	10,485	20,538	19,432
General and Administrative Expense
Total stock based compensation expense
Stock-based compensation expense	$ 14,850	$ 13,822	$ 28,208	$ 25,559

Net Loss Per Share (Details)

Net Loss Per Share (Details) - shares shares in Thousands	3 Months Ended
	Jun. 30, 2022	Jun. 30, 2021
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	8,290	7,780
Stock Options
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	6,387	6,199
Restricted Stock Units
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	1,857	1,556
Employee Stock
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	46	25

Income Taxes - Purchase and Sal

Income Taxes - Purchase and Sale Agreements (Details) - USD ($) $ in Thousands		1 Months Ended		2 Months Ended	6 Months Ended
	Jun. 30, 2022	Jul. 31, 2022	Jun. 30, 2022	Jul. 31, 2022	Jun. 30, 2022
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties					$ 175,000
Sale of Future Royalties
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties				$ 425,000
Sale of Future Royalties | Royalty Pharma, Royalty Purchase Agreement
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties	$ 175,000		$ 175,000		$ 175,000
Sale of Future Royalties | Sixth Street Partners, Purchase and Sale Agreement | Subsequent Event
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties		$ 250,000

Income Taxes - Income Tax Expen

Income Taxes - Income Tax Expense (Benefit) (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Income Taxes
Income tax expense (benefit)	$ 3,130	$ 193	$ 3,313	$ 193

Leases - Components of Lease Ex

Leases - Components of Lease Expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021	Jun. 30, 2022	Jun. 30, 2021
Operating leases cost:
Lease cost	$ 4,942	$ 4,451	$ 10,911	$ 9,177
Sublease income	(600)	(268)	(1,381)	(595)
Net lease cost	$ 4,342	$ 4,183	$ 9,530	$ 8,582

Leases - Cash Flow Information

Leases - Cash Flow Information (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2022	Jun. 30, 2021
Leases
Cash paid for amounts included in the measurement of lease liabilities, operating cash flows from operating leases	$ 7,763	$ 7,394

Leases - Weighted Average Remai

Leases - Weighted Average Remaining Lease-term and Weighted Average Discount Rate (Details)	Jun. 30, 2022
Leases
Weighted average remaining lease term in years	7 years 3 months 18 days
Weighted average discount rate	7.40%

Commitments and Contingencies (

Commitments and Contingencies (Details) - USD ($) $ in Thousands	Jun. 30, 2022	Dec. 31, 2021
Indemnification Agreement
Indemnification Agreements
Loss contingency accrual	$ 0	$ 0

Subsequent Events - Synthetic R

Subsequent Events - Synthetic Royalty Facility (Details) - USD ($) $ in Thousands	1 Months Ended	2 Months Ended	6 Months Ended
	Jul. 31, 2022	Jul. 31, 2022	Jun. 30, 2022
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties			$ 175,000
Sale of Future Royalties
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties		$ 425,000
Subsequent Event | Sixth Street Partners
Gross proceeds from the sale of future royalties
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, percentage rate (as a percent)	9.75%	9.75%
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, amount, maximum	$ 900,000	$ 900,000
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, ratio	1.45	1.45
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, amount	$ 362,500	$ 362,500
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%	15%
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	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	$ 462,500	$ 462,500
Subsequent Event | Sale of Future Royalties | Sixth Street Partners, Purchase and Sale Agreement
Gross proceeds from the sale of future royalties
Gross proceeds from the sale of future royalties	$ 250,000

Subsequent Events - Debt Facili

Subsequent Events - Debt Facility (Details) - Subsequent Event $ in Millions	1 Months Ended
	Jul. 31, 2022 USD ($) tranche
Loans Payable | Sixth Street Partners, Financing Agreement
Debt Financing
Debt instrument, face amount, potential amount	$ 660
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded	50
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded, each day thereafter	80
Loans Payable | Sixth Street Partners, Financing Agreement, Incremental Term Loan
Debt Financing
Debt instrument, face amount, potential amount	$ 260
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%
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%
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility
Debt Financing
Debt instrument, face amount	$ 150
Gross proceeds from issuance of secured debt	150
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Delayed Draw Term Loan Facility
Debt Financing
Debt instrument, face amount, potential amount	$ 250
Debt instrument, tranches, number | tranche	2

Subsequent Events - License Agr

Subsequent Events - License Agreement (Details)	Aug. 02, 2022
Subsequent Event | IDRx
Subsequent Events
License agreement, share in preferred equity received (as a percent)	15%