Blueprint Medicines (BPMC) 10-Q 2 Aug 23 2023 Q2 Quarterly report Financial data

Document and Entity Information

Document and Entity Information - $ / shares	6 Months Ended
	Jun. 30, 2023	Jul. 28, 2023
Cover [Abstract]
Entity Central Index Key	0001597264
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Jun. 30, 2023
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		60,664,749
Current Fiscal Year End Date	--12-31
Document Fiscal Year Focus	2023
Document Fiscal Period Focus	Q2
Amendment Flag	false

Condensed Consolidated Balance

Condensed Consolidated Balance Sheets - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Current assets:
Cash and cash equivalents	$ 75,527	$ 119,709
Marketable securities	647,107	825,283
Accounts receivable	35,147	23,525
Unbilled accounts receivable	3,120	13,413
Inventory	17,016	29,697
Prepaid expenses and other current assets	33,418	35,024
Total current assets	811,335	1,046,651
Marketable securities	113,932	133,480
Property and equipment, net	36,957	33,344
Operating lease right-of-use assets, net	77,529	81,854
Restricted cash	6,352	5,195
Equity investment	27,789	27,789
Other assets	32,551	21,589
Total assets	1,106,445	1,349,902
Current liabilities:
Accounts payable	6,979	2,729
Accrued expenses	101,162	131,123
Current portion of operating lease liabilities	11,292	10,579
Current portion of deferred revenue	3,328	4,667
Current portion of liabilities related to the sale of future royalties and revenues	28,410	17,285
Current portion of term loan	17,857	16,851
Total current liabilities	169,028	183,234
Operating lease liabilities, net of current portion	87,049	92,789
Deferred revenue, net of current portion	5,000	13,624
Liabilities related to the sale of future royalties and revenues, net of current portion	408,931	413,045
Term loan, net of current portion	122,146	122,232
Other long-term liabilities	3,161	10,301
Total liabilities	795,315	835,225
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; 60,638,275 and 59,958,919 shares issued and outstanding at June 30, 2023 and December 31, 2022, respectively	61	60
Additional paid-in capital	2,410,601	2,358,018
Accumulated other comprehensive loss	(4,221)	(10,443)
Accumulated deficit	(2,095,311)	(1,832,958)
Total stockholders' equity	311,130	514,677
Total liabilities and stockholders' equity	$ 1,106,445	$ 1,349,902

Condensed Consolidated Balanc_2

Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares	Jun. 30, 2023	Dec. 31, 2022
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)	60,638,275	59,958,919
Common Stock, shares outstanding (in shares)	60,638,275	59,958,919

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, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Revenues:
Revenue	$ 57,570	$ 36,547	$ 120,857	$ 99,278
Cost and operating expenses:
Cost of sales	2,323	4,886	5,498	9,964
Research and development	110,063	128,466	222,135	231,599
Selling, general and administrative	71,931	58,688	142,882	115,747
Collaboration loss sharing	1,234	2,145	2,530	5,410
Total cost and operating expenses	185,551	194,185	373,045	362,720
Other income (expense):
Interest income (expense), net	(3,996)	427	(9,815)	869
Other income (expense), net	(626)	632	359	177
Total other income (expense), net	(4,622)	1,059	(9,456)	1,046
Loss before income taxes	(132,603)	(156,579)	(261,644)	(262,396)
Income tax expense	190	3,130	710	3,313
Net loss	(132,793)	(159,709)	(262,354)	(265,709)
Other comprehensive loss:
Unrealized gains (losses) on available-for-sale investments	361	(2,308)	6,181	(10,328)
Currency translation adjustments	20	170	41	213
Comprehensive loss	$ (132,412)	$ (161,847)	$ (256,132)	$ (275,824)
Net loss per share - basic (in dollars per share)	$ (2.19)	$ (2.68)
Net loss per share - diluted (in dollars per share)	$ (2.19)	$ (2.68)	$ (4.35)	$ (4.47)
Weighted-average number of common shares used in net loss per share - basic (in shares)	60,516	59,617
Weighted-average number of common shares used in net loss per share - diluted (in shares)	60,516	59,617	60,322	59,465
Product revenue, net
Revenues:
Revenue	$ 39,876	$ 28,454	$ 78,945	$ 52,295
Collaboration and license revenue
Revenues:
Revenue	$ 17,694	$ 8,093	$ 41,912	$ 46,983

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, 2021	$ 59	$ 2,250,250	$ (4,133)	$ (1,275,441)	$ 970,735
Beginning Balance (in shares) at Dec. 31, 2021	59,141,086
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan	$ 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
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
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 and stock purchase plan		2,407			2,407
Issuance of common stock under stock plan and stock purchase plan (in shares)	132,321
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
Beginning Balance at Dec. 31, 2022	$ 60	2,358,018	(10,443)	(1,832,958)	$ 514,677
Beginning Balance (in shares) at Dec. 31, 2022	59,958,919				59,958,919
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan (in shares)	457,416
Stock-based compensation expense		23,340			$ 23,340
Other comprehensive income (loss)			5,841		5,841
Net loss				(129,560)	(129,560)
Ending Balance at Mar. 31, 2023	$ 60	2,381,358	(4,602)	(1,962,518)	414,298
Ending Balance (in shares) at Mar. 31, 2023	60,416,335
Beginning Balance at Dec. 31, 2022	$ 60	2,358,018	(10,443)	(1,832,958)	$ 514,677
Beginning Balance (in shares) at Dec. 31, 2022	59,958,919				59,958,919
Increase (Decrease) in Stockholders' Equity
Net loss					$ (262,354)
Ending Balance at Jun. 30, 2023	$ 61	2,410,601	(4,221)	(2,095,311)	$ 311,130
Ending Balance (in shares) at Jun. 30, 2023	60,638,275				60,638,275
Beginning Balance at Mar. 31, 2023	$ 60	2,381,358	(4,602)	(1,962,518)	$ 414,298
Beginning Balance (in shares) at Mar. 31, 2023	60,416,335
Increase (Decrease) in Stockholders' Equity
Issuance of common stock under stock plan and stock purchase plan	$ 1	5,298			5,299
Issuance of common stock under stock plan and stock purchase plan (in shares)	221,940
Stock-based compensation expense		23,945			23,945
Other comprehensive income (loss)			381		381
Net loss				(132,793)	(132,793)
Ending Balance at Jun. 30, 2023	$ 61	$ 2,410,601	$ (4,221)	$ (2,095,311)	$ 311,130
Ending Balance (in shares) at Jun. 30, 2023	60,638,275				60,638,275

Condensed Consolidated Statem_3

Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Cash flows from operating activities
Net loss	$ (262,354)	$ (265,709)
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities:
Depreciation and amortization	7,262	3,098
Non-cash lease expense	4,457	4,107
Stock-based compensation	47,017	48,746
Non-cash interest expense	8,297
Other	(3,611)	550
Changes in assets and liabilities:
Accounts receivable	(13,105)	(2,128)
Unbilled accounts receivable	10,291	9,591
Inventory	(8,112)	(14,694)
Prepaid expenses and other current assets	1,678	(16,314)
Other assets	(1,419)	(13,188)
Accounts payable	4,218	6,589
Accrued expenses	(25,694)	441
Other long-term liabilities	(7,238)	6,157
Deferred revenue	(9,963)	(14,438)
Operating lease liabilities	(5,160)	(3,790)
Net cash used in operating activities	(253,436)	(250,982)
Cash flows from investing activities
Purchases of property and equipment	(5,138)	(3,709)
Purchases of investments	(407,770)	(39,937)
Maturities of investments	617,765	79,623
Net cash provided by investing activities	204,857	35,977
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	5,298	3,762
Net cash provided by financing activities	5,298	178,762
Net decrease in cash, cash equivalents, and restricted cash	(43,281)	(36,243)
Cash, cash equivalents and restricted cash at beginning of period	124,904	215,119
Effect of exchange rate changes on cash, cash equivalents and restricted cash	256	(306)
Cash, cash equivalents and restricted cash at end of period	81,879	178,570
Supplemental cash flow information
Cash paid for interest	15,167
Debt issuance costs incurred but unpaid at period end		5,022
Property and equipment purchases unpaid at period end	2,603	1,829
Cash paid for taxes, net	$ 676	$ 771

Condensed Consolidated Statem_4

Condensed Consolidated Statements of Cash Flows (Parenthetical) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022	Jun. 30, 2022	Dec. 31, 2021
Reconciliation of cash, cash equivalents, and restricted cash
Cash and cash equivalents	$ 75,527	$ 119,709	$ 173,399
Restricted cash	6,352	5,195	5,171
Total cash, cash equivalents, and restricted cash shown in condensed consolidated statements of cash flows	$ 81,879	$ 124,904	$ 178,570	$ 215,119

Nature of Business

Nature of Business	6 Months Ended
	Jun. 30, 2023
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, breast cancer and other genomically defined cancers, and cancer immunotherapy. The Company is devoting substantially all of its efforts to research and development for current and future drug candidates and commercialization of AYVAKIT/AYVAKYT, GAVRETO and any current or future drug candidates that obtain marketing approval. As of June 30, 2023, the Company had cash, cash equivalents and marketable securities of $836.6 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, 2023
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, 2022 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 16, 2023 (2022 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, 2023, the results of its operations for the three and six months ended June 30, 2023 and 2022, stockholder’s equity for the three and six months ended June 30, 2023 and 2022 and cash flows for six months ended June 30, 2023 and 2022. Such adjustments are of a normal and recurring nature. The results for the three and six months ended June 30, 2023 are not necessarily indicative of the results for the year ending December 31, 2023 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. All intercompany transactions and balances have been eliminated. ​ Use of Estimates The preparation of financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: revenue recognition, inventory, operating lease right-of-use assets, operating lease liabilities, stock-based compensation expense, accrued expenses, liabilities related to the sale of future royalties and future revenues, equity investment, and income taxes. Actual results may differ from these estimates. The Russian invasion of Ukraine has not had a material impact on the Company’s business, results of operations and financial condition. Significant Accounting Policies The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and six months ended June 30, 2023 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2022 Annual Report on Form 10-K, with the exception of the below new policy. Performance-Based Restricted Stock Unit Awards New Accounting Pronouncements

Financing Arrangements

Financing Arrangements	6 Months Ended
	Jun. 30, 2023
Disclosure Text Block
Financing Arrangements	3. Financing Arrangements ​ Royalty Pharma Purchase and Sale Agreement On June 30, 2022, the Company entered into a purchase and sale agreement (Royalty Purchase Agreement) with Royalty Pharma. Pursuant to the Royalty Purchase Agreement, the Company received an upfront payment of $175.0 million in consideration for the Company’s rights to receive royalty payments on the net sales of GAVRETO worldwide excluding the CStone Territory (as defined below) and U.S. territory under the terms of the Roche pralsetinib collaboration agreement. Although the Company sold all of the rights to receive royalties on the net sales of GAVRETO worldwide excluding the CStone Territory and U.S. territory to Royalty Pharma, the Company continues to co-develop pralsetinib with Roche globally and is therefore involved in the generation of these future royalties. Due to the Company’s significant continuing involvement, the Company continues to account for any royalties and development and commercialization milestones earned related to the underlying territory under the Roche pralsetinib collaboration agreement as collaboration revenue on its consolidated statements of operations and comprehensive loss. Net proceeds from the transaction were recorded as a liability related to sale of future royalties and revenues on the consolidated balance sheet. The Company accretes the $175.0 million, net of transaction costs of $3.8 million, to the total of these royalties as interest expense using the effective interest method over the estimated life of the arrangement. These estimates contain assumptions that impact the amount recorded and the interest expense that will be recognized in future periods. As payments are made to Royalty Pharma, the balance of the liability will be effectively repaid over the life of the Royalty Purchase Agreement. In order to determine the amortization of the liability, the Company estimates the total amount of future royalty payments to be paid to Royalty Pharma over the life of the arrangement. The exact amount of repayment is likely to change each reporting period. A significant increase or decrease in GAVRETO’s net revenue of the underlying territory will impact interest expense and the liability related to this arrangement. The Company periodically assesses the expected payments to Royalty Pharma and prospectively adjusts the amortization of the liability related to this arrangement for material changes in such payments including the prospective impact around the uncertainty related to GAVRETO’s commercial outlook in the underlying territory during the life of the arrangement. In February 2023, the Company received written notice from Roche of their election to terminate for convenience the Roche pralsetinib collaboration agreement. The termination will become effective February 2024, at which time the Company will regain commercialization and development rights to GAVRETO from Roche worldwide excluding the CStone Territory. As of June 30, 2023, none of the derecognition conditions under ASC 405 Liabilities 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. However, following the termination of the Roche pralsetinib collaboration agreement, if the specified net sales milestone thresholds under the Royalty Purchase Agreement with Royalty Pharma are not otherwise met, the Company may no longer be eligible to receive any of the contingent milestone payments under the Royalty Purchase Agreement. The potential milestone payments will be added to the carrying value of the liability related to this arrangement when and if the milestones are achieved and received. Financing Arrangements with Sixth Street Partners ​ ​ Sixth Street Partners Purchase and Sale Agreement ​ Pursuant to the Future Revenue Purchase Agreement, the Company received gross proceeds of $250.0 million in exchange for future royalty payments at a rate of 9.75% on up to $900 million each year of (i) aggregate worldwide annual net product sales of AYVAKIT/ AYVAKYT (avapritinib) and (ii), if it is approved, aggregate worldwide annual net product sales of BLU-263 (elenestinib), but excluding sales in Greater China, subject to a cumulative cap of 1.45 times the upfront invested capital or a total of $362.5 million. In the event that certain revenue targets are not achieved by specified dates, the royalty rate and cumulative cap shall be increased to 15% and 1.85 times the invested capital (or $462.5 million), respectively. ​ The Company continues to own the research, development, manufacturing and commercialization of AYVAKIT/ AYVAKYT and if it is approved, elenestinib, and has significant continuing involvement in the generation of the cash flows under the Future Revenue Purchase Agreement. Therefore, the Company continues to account for any revenue earned from worldwide product sales of AYVAKIT/ AYVAKYT and, if it is approved, elenestinib, on its consolidated statements of operations and comprehensive loss. Net proceeds received from the transaction were recorded as a liability related to sale of future royalties and revenues on the consolidated balance sheet. The Company accretes the $250.0 million, net of transaction costs of $5.4 million, to the total of these future payments as interest expense using the effective interest method over the estimated life of the arrangement. ​ As payments are made to Sixth Street Partners, the balance of the liability is effectively repaid over the life of the Future Revenue Purchase Agreement. In order to determine the amortization of the liability, the Company estimates the total amount of future revenue payments to be paid to Sixth Street Partners over the life of the arrangement. The exact amount of repayment is likely to change each reporting period. A significant increase or decrease in worldwide product sales of AYVAKIT/ AYVAKYT and, if it is approved, elenestinib, will materially impact the liability related to this arrangement, interest expense and the time period for repayment. The Company periodically assesses the expected payments to Sixth Street Partners and prospectively adjusts the amortization of the liability related to this arrangement for material changes in such payments. As of June 30, 2023, the Company’s estimate of this total interest expense resulted in an effective annual interest rate of 10.8%. These estimates contain assumptions that impact the amount recorded and the interest expense that will be recognized in future periods. ​ As of June 30, 2023, the net carrying value of the liability related to this arrangement was $261.7 million. The following table shows the activity within the liability account (in thousands): ​ ​ ​ ​ ​ Carrying value as of January 1, 2023 ​ $ 254,328 Interest expense recognized ​ ​ 14,021 Payments ​ ​ (6,644) Carrying value as of June 30, 2023 ​ $ 261,705 ​ Sixth Street Partners Term Loan ​ ​ As part of the Financing Agreement with Sixth Street Partners, the Company received gross proceeds of $150.0 million in July 2022 and incurred an aggregate of $12.2 million of debt discounts and transaction costs, which have been recorded as a reduction to the carrying amount of the debt on the Company’s consolidated balance sheet and are amortized as additional interest expenses using the effective interest rate method over the period from issuance through maturity. As of June 30, 2023, the Company’s estimate of the total interest expense resulted in an effective annual interest rate of 13.6%. The carrying amount of the debt as of June 30, 2023 is subject to variable interest rates, which are based on current market rates, and as such, approximates fair value ​ The following table shows the activity within the liability account (in thousands): ​ ​ ​ ​ ​ Carrying value as of January 1, 2023 ​ $ 139,083 Interest expense recognized ​ ​ 9,443 Payments ​ ​ (8,523) Carrying value as of June 30, 2023 ​ $ 140,003 ​ June 30, 2023, the Company was in compliance with the applicable terms and conditions of the covenants under the Financing Arrangement. ​

Marketable Securities

Marketable Securities	6 Months Ended
	Jun. 30, 2023
Disclosure Text Block
Marketable Securities	4. Marketable Securities Marketable securities consisted of the following at June 30, 2023 and December 31, 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair June 30, 2023 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 241,322 ​ ​ — ​ ​ (1,795) ​ $ 239,527 U.S. treasury obligations ​ ​ 523,485 ​ ​ 7 ​ ​ (1,980) ​ ​ 521,512 Total ​ $ 764,807 ​ $ 7 ​ $ (3,775) ​ $ 761,039 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair December 31, 2022 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 377,519 ​ ​ — ​ ​ (4,848) ​ $ 372,671 U.S. treasury obligations ​ ​ 591,193 ​ ​ 22 ​ ​ (5,123) ​ ​ 586,092 Total ​ $ 968,712 ​ $ 22 ​ $ (9,971) ​ $ 958,763 ​ The following table summarizes the estimated fair value (in thousands) and number of the Company’s available-for-sale securities that are in loss position as of June 30, 2023 and December 31, 2022 by the length of time the security has been in loss position: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, 2023 December 31, 2022 ​ ​ Fair Number of Fair Number of ​ ​ value securities value securities Debt securities in unrealized loss position for 12 months or less ​ $ 625,248 ​ ​ 110 ​ $ 371,746 ​ 66 Debt securities in unrealized loss position for more than 12 months ​ ​ 96,291 ​ ​ 14 ​ ​ 550,561 ​ 56 Total debt securities in unrealized loss position ​ $ 721,539 ​ ​ 124 ​ $ 922,307 ​ 122 ​ The Company has the intent and ability to hold its debt securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable debt securities for the three and six months ended June 30, 2023 and 2022. ​ The following table summarizes the amortized cost basis and estimated fair value of the Company’s available-for-sale securities by contractual maturity as of June 30, 2023 and December 31, 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, 2023 December 31, 2022 ​ Amortized Fair Amortized Fair ​ Cost value Cost value Within one year $ 649,622 $ 647,107 $ 834,440 $ 825,283 After one through five years ​ 115,185 ​ 113,932 ​ 134,272 ​ 133,480 Total $ 764,807 $ 761,039 $ 968,712 $ 958,763 ​ ​ The following table summarizes the proceeds from maturities of debt securities during the three and six months ended June 30, 2023 and 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2023 2022 2023 2022 Proceeds from maturities of debt securities ​ $ 309,515 ​ $ 79,623 ​ $ 617,765 ​ $ 79,623 ​ ​ The Company did no t realize any gains or losses from maturities of debt securities for the three and six months ended June 30, 2023 and 2022.

Fair Value of Financial Instrum

Fair Value of Financial Instruments	6 Months Ended
	Jun. 30, 2023
Disclosure Text Block
Fair Value of Financial Instruments	5. Fair Value of Financial Instruments The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of June 30, 2023 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ June 30, ​ Markets ​ Inputs ​ Inputs Description ​ 2023 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 62,861 ​ ​ 62,861 ​ ​ — ​ $ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 239,527 ​ ​ — ​ ​ 239,527 ​ ​ — U.S. treasury obligations ​ ​ 521,512 ​ ​ 521,512 ​ ​ — ​ ​ — Total ​ $ 823,900 ​ $ 584,373 ​ $ 239,527 ​ $ — ​ The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ December 31, ​ Markets ​ Inputs ​ Inputs Description ​ 2022 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 95,198 ​ $ 95,198 ​ $ — ​ $ — U.S. treasury obligations ​ ​ 4,989 ​ ​ 4,989 ​ ​ — ​ ​ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 372,671 ​ ​ — ​ ​ 372,671 ​ ​ — U.S. treasury obligations ​ ​ 586,092 ​ ​ 586,092 ​ ​ — ​ ​ — Total ​ $ 1,058,950 ​ $ 686,279 ​ $ 372,671 ​ $ — ​

Product Revenue Reserves and Al

Product Revenue Reserves and Allowances	6 Months Ended
	Jun. 30, 2023
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 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 May 2023, the FDA approved AYVAKIT for the treatment of adult patients with indolent SM. The following table summarizes revenue recognized from product sales for the three and six months ended June 30, 2023 and 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2023 2022 2023 2022 United States ​ $ 34,307 ​ $ 24,682 ​ $ 69,234 ​ $ 45,834 Rest of World ​ ​ 5,569 ​ ​ 3,772 ​ ​ 9,711 ​ ​ 6,461 Total product revenue ​ $ 39,876 ​ $ 28,454 ​ $ 78,945 ​ $ 52,295 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2023 2022 2023 2022 Customer 1 ​ 41 % ​ 44 % ​ 42 % ​ 46 % Customer 2 ​ 11 % ​ 11 % ​ 11 % ​ 11 % ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2023 ​ 2022 Customer 1 ​ 34 % ​ 33 % Customer 2 ​ 11 % ​ 15 % Customer 3 ​ * ​ ​ 12 % Customer 4 ​ 10 % ​ * ​ * Indicates the customer’s share is under 10% . The following table summarizes activity in each of the product revenue allowance and reserve categories for the six months ended June 30, 2023 and 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended June 30, ​ 2023 ​ 2022 Beginning balance at January 1 $ 9,788 ​ $ 4,345 Provision related to sales in the current period 16,108 ​ ​ 9,408 Adjustment related to prior periods sales (531) ​ ​ (570) Credits and payments made (10,553) ​ ​ (6,120) Ending balance at June 30 $ 14,812 ​ $ 7,063 ​ The total reserves that are included in the Company’s unaudited condensed consolidated balance sheets as of June 30, 2023 and December 31, 2022, are summarized as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2023 ​ 2022 Reduction of accounts receivable, net $ 1,257 ​ $ 742 Component of accrued expenses ​ 13,555 ​ ​ 9,046 Total revenue-related reserves $ 14,812 ​ $ 9,788

Inventory

Inventory	6 Months Ended
	Jun. 30, 2023
Disclosure Text Block
Inventory	7. Inventory Capitalized inventory consists of the following at June 30, 2023 and December 31, 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2023 2022 Raw materials ​ $ 1,599 ​ $ 4,967 Work in process ​ ​ 32,750 ​ ​ 28,427 Finished goods ​ 2,589 ​ 2,791 Total ​ $ 36,938 ​ $ 36,185 ​ Balance sheet classification ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2023 2022 Inventory ​ $ 17,016 ​ $ 29,697 Other assets ​ 19,922 ​ 6,488 Total ​ $ 36,938 ​ $ 36,185 ​ Inventory amounts written down as a result of excess, obsolescence, unmarketability or other reasons are charged to cost of sales. The Company recognized a write-down of $0.5 million and $2.0 million for the three and six months ended June 30, 2023, respectively. The Company did not recognize any write-down for the three and six months ended June 30, 2022. Long-term inventory, which primarily consists of work in process and raw materials, is included in other assets in the unaudited condensed consolidated balance sheets.

Restricted Cash

Restricted Cash	6 Months Ended
	Jun. 30, 2023
Disclosure Text Block
Restricted Cash	8. Restricted Cash At June 30, 2023 and December 31, 2022, $6.4 million and $5.2 million, respectively, of the Company’s cash is restricted by a financial institution primarily related to security deposits for the lease agreements for the Company’s office and laboratory spaces.

Accrued Expenses

Accrued Expenses	6 Months Ended
	Jun. 30, 2023
Disclosure Text Block
Accrued Expenses	9. Accrued Expenses Accrued expenses consist of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2023 2022 Research, development and commercial contract costs ​ $ 40,401 ​ $ 66,041 Employee compensation ​ ​ 26,179 ​ ​ 30,744 Accrued professional fees ​ 13,516 ​ 17,588 Revenue-related reserves ​ ​ 13,555 ​ ​ 9,046 Other ​ ​ 7,511 ​ ​ 7,704 Total ​ $ 101,162 ​ $ 131,123

Collaboration and License Agree

Collaboration and License Agreements	6 Months Ended
	Jun. 30, 2023
Disclosure Text Block
Collaboration and License Agreements	10 . Collaboration and License Agreements IDRx In August 2022, the Company entered into a license agreement with IDRx, Inc. (IDRx), pursuant to which the Company granted IDRx an exclusive, worldwide, royalty-bearing license to exploit the Company’s internally discovered KIT exon 13 inhibitor IDRX-73 (IDRx License Agreement). IDRx is a clinical-stage biopharmaceutical company and a mong IDRx’s founders are Alexis Borisy, George Demetri, M.D., and Nicholas Lydon, Ph.D., who were each members of the Company’s board of directors at the time. Due to these relationships, the transaction with IDRx is a related party transaction. In connection with the IDRx License Agreement, the Company also entered into a stock purchase agreement with IDRx (IDRx Stock Purchase Agreement), pursuant to which the Company received 4,509,105 shares of IDRx’s Series A preferred stock and the Company has the right to receive additional shares of IDRx’s Series A preferred stock through an anti-dilution provision subject to a defined financing cap. The Company is also eligible to receive up to $217.5 million in contingent cash payments, including specified development, regulatory and sales-based milestone payments. In addition, IDRx is obligated to pay to the Company royalties on aggregate annual worldwide net sales of licensed products at tiered percentage rates up to low-teens, subject to adjustments in specified circumstances under the IDRx License Agreement. Unless earlier terminated, the IDRx License Agreement will expire on a country-by-country, licensed product-by-licensed product basis upon the latest of: (a) the expiration of the last valid claim within the licensed patents covering such licensed product in a such country, (b) the expiration of the regulatory exclusivity period for such licensed product in such country, or (c) the 10th anniversary of the first commercial sale of such licensed product in such country. Following the end of the term for any such licensed product and in such region by expiration, the license granted to IDRx will become exclusive, perpetual, irrevocable, fully paid-up and royalty-free. IDRx may terminate the IDRx License Agreement for convenience at any time upon at least twelve months ’ prior written notice to the Company. Either party may also terminate the IDRx License Agreement for material breach of the other party or for insolvency, and the Company may terminate the IDRx License Agreement for IDRx’s breach of the anti-dilution provision in the IDRx Stock Purchase Agreement. Upon termination of the license agreement in its entirety, all rights and obligations under the license agreement will terminate and revert back to the Company, and the Company has a license under certain intellectual property of IDRx to continue to exploit the compound and terminated product, subject to a royalty that will be negotiated at the time of termination. The Company considered the ASC 606 criteria for combining contracts and determined the IDRx License Agreement and the IDRx Stock Purchase Agreement should be combined into a single contract because they were negotiated and entered into in contemplation of one another. Therefore, the Company determined that the 4,509,105 shares of IDRx’s Series A preferred stock and the anti-dilution right to receive additional shares should be attributed to the transaction price of the IDRx License Agreement. The Company evaluated the IDRx License Agreement under ASC 606. The Company identified the following material promises under the agreement: (1) the exclusive license and (2) the initial know-how transfer. The Company determined that the exclusive license and the initial know-how transfer were not distinct from each other, as the exclusive license has limited value without the corresponding know-how transfer. As such, for the purposes of ASC 606, the Company determined that these two material promises, the exclusive license and the initial know-how transfer, should be combined into one distinct performance obligation. The Company concluded that the license is a functional intellectual property license. The Company determined that IDRx benefited from the license along with the initial know-how transfer at the time of grant, and therefore the related performance obligation is satisfied at a point in time. For the purposes of ASC 606, the transaction price of the IDRx License Agreement at the contract inception was determined to be $27.5 million and recorded as license revenue-related party on the consolidated statements of operations and comprehensive loss during the year ended December 31, 2022. The fair value was derived from IDRx’s most recent financing transaction with unrelated investors. All potential milestone payments that the Company is eligible to receive under the IDRx License Agreement have been excluded from the transaction price. The Company reevaluates the transaction price for inclusion of milestone payments and royalties at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. Additionally, the Company is entitled to sales milestones and royalties from the sales of the licensed products, and revenue are recognized when the related sales occur. The Company concluded the preferred stock investment should be accounted for as an equity investment as it is not mandatorily redeemable nor does the Company have the unilateral right to redeem the preferred stock, and the Company, along with its related parties, do not have a controlling financial interest in IDRx nor have the ability to influence the financial and operating policies through the ownership of preferred stock. IDRx’s preferred stock is not exchange-traded and does not have a readily determinable fair value. Therefore, the Company accounts for the preferred stock investment under the measurement alternative for equity investments that do not have a readily determinable fair value, which is at cost of $27.8 million including transaction costs of $0.3 million. As of June 30, 2023, the cost of the investment in IDRx’s preferred stock was $27.8 million and was recorded as equity investment on the condensed consolidated balance sheets. As of June 30, 2023, no adjustments have been recognized related to the preferred stock investment as a result of the application of the measurement alternative. 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 program is referred to as a target program) On a target program-by-target program basis, the Company has an exclusive option to obtain a worldwide, exclusive license to develop and commercialize any licensed compound and licensed product under each target program. Proteovant has 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) to develop and commercialize certain licensed products for the treatment of EGFR-driven non-small cell lung cancer in Greater China, including Mainland China, Hong Kong, Macau and Taiwan (collectively, the Zai Lab Territory). The collaboration aims to accelerate and expand global development of the licensed products, which currently include BLU-945 and BLU-525 (which served as a back-up candidate for BLU-701). The Company retains exclusive rights to the licensed products outside the Zai Lab Territory. Under the Zai Lab agreement, the Company received an upfront cash payment of $25.0 million and, in addition to the upfront payment received, the Company is eligible to receive up to $590.0 million in contingent payments, including specified development, regulatory and sales-based milestones and tiered percentage royalties on a licensed product-by-licensed product basis ranging from the low-teens to mid-teens on annual net sales of each licensed product in the Zai Lab Territory, subject to adjustment in specified circumstances. Zai Lab is responsible for costs related to clinical trials in the Zai Lab Territory, other than the specified shared services costs as defined in the Zai Lab agreement which are 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, 2023, the Company did not record such reductions of expenses under the Zai Lab agreement, and such reductions were insignificant during the three and six months ended June 30, 2022. The Company evaluated the Zai Lab Territory specific licenses and related activities under ASC 606 as these transactions are considered transactions with a customer and identified three material promises at the outset of the Zai Lab agreement, which consists of the following for each licensed product: (1) the exclusive license, (2) the initial know-how transfer and (3) manufacturing activities related to development and commercial supply of the licensed product in the Zai Lab Territory. The Company determined that the exclusive license and the initial know-how transfer were not distinct from each other, as the exclusive license has limited value without the corresponding know-how transfer. As such, for the purposes of ASC 606, the Company determined that these two material promises, the exclusive license and the initial know-how, should be combined into one distinct performance obligation. The Company further evaluated the material promise associated with manufacturing activities related to development and commercial supply of the licensed products in the Zai Lab Territory, given Zai Lab is not obligated to purchase any minimum amount or quantities of the development and commercial supply from the Company, the Company concluded that, for the purpose of ASC 606, the provision of manufacturing activities related to development and commercial supply of the licensed product in Zai Lab Territory was an option but not a performance obligation of the Company at the inception of the Zai Lab collaboration agreement and will be accounted for if and when exercised. The Company also concluded that there is no separate material right in connection with the development and commercial supply of the licensed product, as the expected pricing was not issued at a significant and incremental discount. Therefore, the manufacturing activities were excluded as performance obligation at the outset of the arrangement. The Company evaluated the license under ASC 606 and concluded that the license is a functional intellectual property license. The Company determined that Zai Lab benefited from the license along with the initial know-how transfer at the time of grant, and therefore the related performance obligation is satisfied at a point in time. Additionally, the Company is entitled to sales milestones and royalties from Zai Lab upon future sales of the licensed products in the Zai Lab Territory, and revenue will be recognized when the related sales occur. Costs that are incurred associated with Zai Lab Territory specific activities are reimbursable from Zai Lab and are recognized as revenue. During the three months ended June 30, 2023, the Company did not record revenue under the Zai Lab agreement and revenue recorded during the six months ended June 30, 2023 was negligible. 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. For the purposes of ASC 606, the transaction price of the Zai Lab agreement at the outset of the arrangement was determined to be $25.0 million, which consisted of the upfront cash payment. The other potential milestone payments that the Company is eligible to receive were excluded from the transaction price, as all milestone amounts were fully constrained based on the probability of achievement. The Company satisfied the performance obligation upon delivery of the licenses and initial know-how transfer, and recognized the upfront payment of $25.0 million as revenue in 2021. The Company reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. Roche – Pralsetinib Collaboration Under the Roche pralsetinib collaboration agreement, the Company received an upfront cash payment of $675.0 million, and through June 30, 2023, the Company received an aggregate of $105.0 million in specified regulatory and commercialization milestones. In February 2023, the Company received written notice from Roche of their election to terminate for convenience the Roche pralsetinib collaboration agreement. The termination will become effective in February 2024, at which time the Company will regain commercialization and development rights to GAVRETO from Roche worldwide excluding the CStone Territory. Until the termination of the collaboration agreement is effective, the parties continue to perform their respective obligations under the collaboration agreement, including with respect to the development and commercialization of GAVRETO. The Company will not be entitled to receive payment for milestones, if any, achieved during the period between the receipt of the notice of termination and the effective date of termination. The termination of the collaboration agreement does not have an impact on the Company’s current accounting treatment related to the Roche pralsetinib collaboration. In the U.S., the Company and Roche agreed to work together to co-commercialize pralsetinib and equally share responsibilities, profits and losses. In addition, the Company is eligible to receive tiered royalties ranging from high-teens to mid-twenties on annual net sales of pralsetinib outside the U.S., excluding Greater China (the Roche Territory). The Company and Roche have also agreed to co-develop pralsetinib globally in RET-altered solid tumors, including non-small cell lung cancer, medullary thyroid carcinoma and other thyroid cancers, as well as other solid tumors. The Company and Roche share global development costs for pralsetinib at a rate of 45 percent for the Company and 55 percent for Roche up to a specified amount of aggregate joint development costs, after which the Company’s share of global development costs for pralsetinib will be reduced by a specified percentage. The Company and Roche will also share specified global development costs for any next-generation RET compound co-developed under the collaboration in a similar manner. 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 is accounted for if and when exercised. The Company also concluded that there is no separate material right in connection with the development and commercial supply of pralsetinib, as the expected pricing was not issued at a significant and incremental discount. Therefore, the manufacturing activities were excluded as performance obligations at the outset of the arrangement. Additionally, the Company is entitled to sales milestones and royalties from Roche upon future sales of pralsetinib in the Roche Territory, and the revenue is recognized when the related sales occur. Costs that are incurred associated with the Roche Territory activities are reimbursable from Roche and are recognized as revenue. For the purposes of ASC 606, the transaction price of the Roche collaboration agreement at the outset of the arrangement was determined to be $695.7 million, which consisted of the upfront cash payment of $675.0 million and the $20.7 million premium on the sale of common stock to Roche Holdings, which was allocated to the performance obligation related to the pralsetinib licenses. Through June 30, 2023, the Company has achieved an aggregate of $105.0 million in specified regulatory and commercialization milestones which were added to the estimated transaction price of the Roche pralsetinib agreement. The following table summarizes revenue recognized under the Roche pralsetinib collaboration during the three and six months ended June 30, 2023 and 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2023 2022 2023 2022 Manufacturing and research and development services related to $ 229 $ 35 $ 356 $ 691 Royalty revenue ​ 366 ​ 313 ​ 803 600 Total Roche pralsetinib collaboration revenue $ 595 $ 348 $ 1,159 $ 1,291 ​ ​ 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. 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 (which indication was subsequently voluntarily withdrawn by Roche in June 2023, followed by the official FDA withdrawal on July 20, 2023), 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). 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 loss sharing from product sales of GAVRETO to customers in the U.S. during the three and six months ended June 30, 2023 and 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ June 30, ​ June 30, ​ 2023 2022 ​ 2023 2022 The Company's share of loss in the U.S. for pralsetinib $ 1,234 $ 2,145 ​ $ 2,530 ​ $ 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 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, 2023 and 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ 2023 2022 2023 2022 Reductions to selling, general and administrative expenses $ 2,774 $ 4,416 $ 5,555 ​ $ 9,248 Increases in research and development expenses $ 5,347 $ 3,714 $ 13,623 ​ $ 3,704 ​ The following table summarizes the contract assets and liability associated with the Roche pralsetinib collaboration as of June 30, 2023 and December 31, 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2023 ​ 2022 Unbilled accounts receivable $ 380 $ — Accrued expenses $ 4,573 $ 6,519 ​ Although the Company sold its right to receive royalty payments from Roche’s sales of GAVRETO in Roche Territory to Royalty Pharma in June 2022, given the Company’s significant continuing involvement in the generation of future royalties, the Company continues to account for any royalties 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 – Financing Arrangements Clementia In October 2019, the Company entered into a license agreement (the Clementia agreement) with Clementia Pharmaceuticals, Inc. (Clementia), a wholly-owned subsidiary of Ipsen S.A. Under the Clementia agreement, the Company granted an exclusive, worldwide, royalty-bearing license to Clementia to develop and commercialize BLU-782, the Company’s oral, highly selective investigational ALK2 inhibitor in Phase 1 clinical development for the treatment of fibrodysplasia ossificans progressiva (FOP), as well as specified other compounds related to the BLU-782 program. Under the Clementia agreement, the Company received an upfront cash payment of $25.0 million and through June 30, 2023, 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, 2023, the Company is eligible to receive up to $460.0 million in contingent payments, including specified development, regulatory and sales-based milestones for licensed products. In addition, Clementia is obligated to pay to the Company royalties on aggregate annual worldwide net sales of licensed products at tiered percentage rates ranging from the low- to mid-teens, subject to adjustment in specified circumstances under the Clementia agreement, and Clementia purchased specified manufacturing inventory from the Company for a total of $1.5 million. Unless earlier terminated in accordance with the terms of the Clementia agreement, the agreement will expire on a country-by-country, licensed product-by-licensed product basis on the date when no royalty payments are or will become due. Clementia may terminate the agreement at any time upon at least 12 months’ prior written notice to the Company. Either party may terminate the agreement for the other party’s uncured material breach or insolvency and in certain other circumstances agreed to by the parties. In certain termination circumstances, the Company is entitled to retain specified licenses to be able to continue to exploit the Clementia licensed products. The Company evaluated the Clementia agreement under ASC 606, as the agreement represented a transaction with a customer. The Company identified the following material promises under the agreement: (1) the exclusive license to develop, manufacture and commercialize BLU-782; (2) the technology transfer of BLU-782 program; (3) the transfer of existing manufacturing inventory; and (4) the transfer of in-process manufacturing inventory. In addition, the Company determined that the exclusive license and technology transfer were not distinct from each other, as the exclusive license has limited value without the corresponding technology transfer. As such, for the purposes of ASC 606, the Company determined that these four material promises, described above, should be combined into three performance obligations: (1) the exclusive license and the technology transfer; (2) the transfer of existing manufacturing inventory; and (3) the transfer of in-process manufacturing inventory. The Company determined that the transaction price at the outset of the arrangement was $46.5 million, which was allocated to the three performance obligations on a relative stand-alone selling price basis, and was recognized as revenue During the three and six months ended June 30, 2023, and 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 reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. There was no revenue deferred as a contract liability associated with the Clementia agreement as of June 30, 2023 and December 31, 2022. 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, 2023, the Company has achieved an aggregate of $38.5 million in milestones under this collaboration. Subject to the terms of the CStone agreement, in addition to the upfront payments received and milestones achieved through June 30, 2023, the Company will be eligible to receive up to $307.5 million in contingent payments, including specified development, regulatory and sales-based milestones for licensed products. In addition, CStone will be obligated to pay the Company tiered percentage royalties on a licensed product-by-licensed product basis ranging from the mid-teens to low twenties on annual net sales of each licensed product in the CStone Territory, subject to adjustment in specified circumstances. CStone is responsible for costs related to the development of the licensed products in the CStone Territory, other than specified costs r

Stock-based Compensation

Stock-based Compensation	6 Months Ended
	Jun. 30, 2023
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, performance-based restricted stock units, unrestricted stock, performance-based awards and cash-based awards. The Company initially reserved a total of 1,460,084 shares of common stock for the issuance of awards under the 2015 Plan. The 2015 Plan 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, 2023, the number of shares reserved for issuance under the 2015 Plan was increased by 2,398,356 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, 2023, there were 4,839,806 shares available for future grant under the 2015 Plan. 2020 Inducement Plan In March 2020, the Company’s board of directors adopted the 2020 Inducement Plan (the Inducement Plan), pursuant to which the Company may grant, subject to the terms of the Inducement Plan and Nasdaq rules, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, and other stock-based awards. The Company initially reserved a total of 1,000,000 shares of common stock for the issuance of awards under the Inducement Plan. In June 2022, the Company’s board of directors approved the reservation of an additional 1,500,000 shares of common stock for the issuance of awards under the Inducement Plan. The number of shares reserved and available for issuance under the Inducement Plan can be increased at any time with the approval of the Company’s board of directors. The Inducement Plan permits the board of directors or a committee thereof to use the stock-based awards available under the Inducement Plan to attract key employees for the growth of the Company. As of June 30, 2023, there were 1,382,793 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, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ ​ Shares ​ ​ Exercise Price Outstanding at December 31, 2022 6,233,451 ​ $ 69.14 Granted 1,126,487 ​ ​ 44.54 Exercised (97,004) ​ ​ 30.48 Canceled (192,527) ​ ​ 72.40 Outstanding at June 30, 2023 7,070,407 ​ $ 65.67 Exercisable at June 30, 2023 4,594,568 ​ $ 68.34 ​ As of June 30, 2023, the total unrecognized compensation expense related to unvested stock option awards was $78.2 million, which is expected to be recognized over a weighted-average period of approximately 2.75 years. Restricted stock units The following table summarizes the restricted stock units activity for the six months ended June 30, 2023: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ Shares Grant Date Fair Value Unvested shares at December 31, 2022 1,893,944 ​ $ 73.28 Granted 851,876 ​ 44.07 Vested (524,345) ​ 72.51 Forfeited (72,401) ​ 65.62 Unvested shares at June 30, 2023 2,149,074 ​ $ 62.16 ​ As of June 30, 2023, the total unrecognized compensation expense related to unvested restricted stock units was $112.5 million, which is expected to be recognized over a weighted-average period of approximately 2.75 years. Performance-based restricted stock units During the first quarter of 2023, the Company began granting performance-based restricted stock units (PSUs) that will settle in stock. PSUs awarded to employees have a third ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ ​ ​ ​ Grant Date ​ Shares Grant Date Fair Value Unvested shares at December 31, 2022 — ​ $ — Granted 52,500 ​ 59.32 Vested — ​ — Forfeited — ​ — Unvested shares at June 30, 2023 52,500 ​ $ 59.32 ​ 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, 2023, the number of shares reserved for issuance under the 2015 ESPP was increased by 599,589 shares. Stock-based compensation expense The Company recognized stock-based compensation expense of $23.8 million and $47.0 million for the three and six months ended June 30, 2023, respectively, and $25.3 million and $48.7 million for the three and six months ended June 30, 2022, 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, ​ ​ 2023 ​ 2022 ​ ​ 2023 ​ 2022 Stock options $ 10,337 ​ $ 12,811 ​ $ 21,197 ​ $ 25,755 Restricted stock units ​ 12,913 ​ ​ 12,396 ​ ​ 24,853 ​ ​ 22,782 Performance-based restricted stock units ​ 257 ​ ​ — ​ ​ 344 ​ ​ ​ Employee stock purchase plan ​ 438 ​ ​ 317 ​ ​ 891 ​ ​ 596 Subtotal ​ 23,945 ​ ​ 25,524 ​ ​ 47,285 ​ ​ 49,133 Capitalized stock-based compensation costs ​ (124) ​ ​ (177) ​ ​ (268) ​ ​ (387) Stock-based compensation expense included in total cost and operating expenses $ 23,821 ​ $ 25,347 ​ $ 47,017 ​ $ 48,746 ​ Stock-based compensation expense, that is included in operating expenses, ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2023 2022 ​ 2023 2022 Research and development ​ $ 10,227 $ 10,497 $ 20,344 $ 20,538 Selling, general and administrative ​ 13,594 ​ 14,850 ​ 26,673 ​ 28,208 Total ​ $ 23,821 ​ $ 25,347 ​ $ 47,017 ​ $ 48,746

Net Loss Per Share

Net Loss Per Share	6 Months Ended
	Jun. 30, 2023
Disclosure Text Block
Net 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, PSUs and ESPP shares are considered to be common stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented as a result of the Company’s net loss. ​ The following common stock equivalents were excluded from the calculation of diluted net loss per share for the six months ended June 30, 2023 and 2022 because including them would have had an anti-dilutive effect (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ 2023 2022 Stock options 7,070 ​ 6,387 Restricted stock units 2,149 ​ 1,857 Performance-based restricted stock units ​ 53 ​ — ESPP shares ​ 47 ​ 46 Total 9,319 8,290

Income Taxes

Income Taxes	6 Months Ended
	Jun. 30, 2023
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, 2023, the Company has a full valuation allowance applied against its U.S. and foreign deferred tax assets. Effective January 1, 2022, a provision of the Tax Cuts and Jobs Act (TCJA) has taken effect creating a significant change to the treatment of research and experimental (R&E) expenditures under Section 174 of the IRC (Sec. 174 expenses). Historically, businesses have had the option of deducting Sec. 174 expenses in the year incurred or capitalizing and amortizing the costs over five years. The new TCJA provision, however, eliminates this option and will require Sec. 174 expenses associated with research conducted in the U.S to be capitalized and amortized over a five-year period. For expenses associated with research outside of the United States, Sec. 174 expenses will be capitalized and amortized over a 15-year period. The Company has included the tax impact of capitalizing and amortizing these costs over the required periods for the six months ended June 30, 2023. On June 30, 2022, the Company entered into a Royalty Purchase Agreement with Royalty Pharma and a Future Revenue Purchase Agreement with Sixth Street Partners. Pursuant to the agreements, the Company received gross proceeds of $175.0 million from Royalty Pharma in June 2022 and $250.0 million from Sixth Street Partners in July 2022 upon the transactions closing. The total cash consideration of $425.0 million, in its entirety, was considered taxable income for calendar year ended December 31, 2022. Therefore, the $425.0 million was 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 expected 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. On August 16, 2022, President Biden signed the Inflation Reduction Act (IRA) of 2022 (H.R. 5376) into law. It includes income tax incentives designed to encourage investment in renewable and alternative energy sources, adoption of electric vehicles, and improvement in the energy efficiency of buildings and communities. To finance these incentives, the law imposes a 15% corporate alternative minimum tax (CAMT) on adjusted financial statement income of corporations with profits over $1 billion. It also introduces a new excise tax on corporate stock buybacks of public US companies. The CAMT is effective for tax years beginning after December 31, 2022 while the excise tax on corporate stock buybacks is effective for repurchases of stock after December 31, 2022. The Company does not anticipate the IRA having a significant impact on the effective tax rate or income tax payable and deferred income tax positions. As of June 30, 2023, the Company expects to be in a taxable loss position for the calendar year ended December 31, 2023, and has recorded an income tax expense of $0.2 million and $0.7 million for the three and six months ended June 30, 2023. The Company recorded an income tax provision despite forecasting a taxable loss due to the impact of recording unfavorable discrete items in the period, and taxable income from the jurisdictions in which the Company is subject to tax. As of June 30, 2023, the Company had gross unrecognized tax benefit, excluding interest and penalties of approximately $0.2 million. As of December 31, 2022, the Company did not have any gross unrecognized tax benefit. The company recognizes interest and penalties related to unrecognized tax benefits in the provision for income taxes. As of June 30, 2023, the Company has accrued an insignificant amount of interest and penalties related to uncertain tax positions.

Leases

Leases	6 Months Ended
	Jun. 30, 2023
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 only certain exercised renewal options were included in the calculation of the operating lease assets and the operating lease liabilities, as other renewal options were not reasonably certain of being exercised as of June 30, 2023. The lease agreements do not contain residual value guarantees and t ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ June 30, ​ June 30, Operating leases: 2023 ​ 2022 ​ 2023 ​ 2022 Lease cost $ 5,662 ​ $ 4,942 ​ $ 11,543 ​ $ 10,911 Sublease income ​ — ​ ​ (600) ​ ​ — ​ ​ (1,381) Net lease cost $ 5,662 ​ $ 4,342 ​ $ 11,543 ​ $ 9,530 ​ The Company’s sublease agreements for the 38 Sidney Street property expired in 2022. The Company has not entered into any material short-term leases or financing leases as of June 30, 2023. Supplemental cash flow information related to leases for the six months ended June 30, 2023 and 2022 is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ June 30, ​ 2023 ​ 2022 Cash paid for amounts included in the measurement of lease liabilities: $ 8,812 ​ $ 7,763 Lease liabilities arising from obtaining right-of-use assets: ​ ​ ​ ​ ​ Operating leases $ 112 ​ $ — ​ The weighted average remaining lease term and weighted average discount rate of the operating leases are as follows: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Operating leases Weighted average remaining lease term in years ​ ​ ​ ​ 6.3 Weighted average discount rate ​ ​ ​ ​ 7.4%

Commitments and Contingencies

Commitments and Contingencies	6 Months Ended
	Jun. 30, 2023
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, 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, 2023, there was a decrease of $7.3 million in the Company’s contractual obligations described . Legal Proceedings The Company is not currently a party to any material legal proceedings. At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. The Company expenses the costs related to its legal proceedings as they are incurred. Indemnification Agreements In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners, and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors and senior management that will require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers of the Company. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company is not aware of any claims under indemnification arrangements, and it has no t accrued any liabilities related to such obligations in its condensed consolidated financial statements as of June 30, 2023 or December 31, 2022.

Summary of Significant Accoun_2

Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Policies)	6 Months Ended
	Jun. 30, 2023
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, 2022 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 16, 2023 (2022 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, 2023, the results of its operations for the three and six months ended June 30, 2023 and 2022, stockholder’s equity for the three and six months ended June 30, 2023 and 2022 and cash flows for six months ended June 30, 2023 and 2022. Such adjustments are of a normal and recurring nature. The results for the three and six months ended June 30, 2023 are not necessarily indicative of the results for the year ending December 31, 2023 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. All intercompany transactions and balances have been eliminated.
Use of Estimates	Use of Estimates The preparation of financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: revenue recognition, inventory, operating lease right-of-use assets, operating lease liabilities, stock-based compensation expense, accrued expenses, liabilities related to the sale of future royalties and future revenues, equity investment, and income taxes. Actual results may differ from these estimates. The Russian invasion of Ukraine has not had a material impact on the Company’s business, results of operations and financial condition.
Significant Accounting Policies	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, 2023 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2022 Annual Report on Form 10-K, with the exception of the below new policy.
Performance-Based Restricted Stock Unit Awards	Performance-Based Restricted Stock Unit Awards
New Accounting Pronouncements	New Accounting Pronouncements

Financing Arrangements (Tables)

Financing Arrangements (Tables)	6 Months Ended
	Jun. 30, 2023
Sixth Street Partners, Future Revenue Purchase Agreement
Table Text Blocks
Schedule of activity within the liability account	​ ​ ​ ​ ​ Carrying value as of January 1, 2023 ​ $ 254,328 Interest expense recognized ​ ​ 14,021 Payments ​ ​ (6,644) Carrying value as of June 30, 2023 ​ $ 261,705 ​
Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility
Table Text Blocks
Schedule of activity within the liability account	​ ​ ​ ​ ​ Carrying value as of January 1, 2023 ​ $ 139,083 Interest expense recognized ​ ​ 9,443 Payments ​ ​ (8,523) Carrying value as of June 30, 2023 ​ $ 140,003 ​

Marketable Securities (Tables)

Marketable Securities (Tables)	6 Months Ended
	Jun. 30, 2023
Table Text Blocks
Schedule of marketable securities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair June 30, 2023 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 241,322 ​ ​ — ​ ​ (1,795) ​ $ 239,527 U.S. treasury obligations ​ ​ 523,485 ​ ​ 7 ​ ​ (1,980) ​ ​ 521,512 Total ​ $ 764,807 ​ $ 7 ​ $ (3,775) ​ $ 761,039 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair December 31, 2022 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 377,519 ​ ​ — ​ ​ (4,848) ​ $ 372,671 U.S. treasury obligations ​ ​ 591,193 ​ ​ 22 ​ ​ (5,123) ​ ​ 586,092 Total ​ $ 968,712 ​ $ 22 ​ $ (9,971) ​ $ 958,763
Summary of fair value and number of available-for-sale securities in loss positions	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, 2023 December 31, 2022 ​ ​ Fair Number of Fair Number of ​ ​ value securities value securities Debt securities in unrealized loss position for 12 months or less ​ $ 625,248 ​ ​ 110 ​ $ 371,746 ​ 66 Debt securities in unrealized loss position for more than 12 months ​ ​ 96,291 ​ ​ 14 ​ ​ 550,561 ​ 56 Total debt securities in unrealized loss position ​ $ 721,539 ​ ​ 124 ​ $ 922,307 ​ 122
Summary of the fair value and the number of the available-for-sale securities by their maturities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, 2023 December 31, 2022 ​ Amortized Fair Amortized Fair ​ Cost value Cost value Within one year $ 649,622 $ 647,107 $ 834,440 $ 825,283 After one through five years ​ 115,185 ​ 113,932 ​ 134,272 ​ 133,480 Total $ 764,807 $ 761,039 $ 968,712 $ 958,763
Summary of proceeds from maturities of debt securities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2023 2022 2023 2022 Proceeds from maturities of debt securities ​ $ 309,515 ​ $ 79,623 ​ $ 617,765 ​ $ 79,623

Fair Value of Financial Instr_2

Fair Value of Financial Instruments (Tables)	6 Months Ended
	Jun. 30, 2023
Table Text Blocks
Schedule of financial instruments measured at fair value	The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of June 30, 2023 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ June 30, ​ Markets ​ Inputs ​ Inputs Description ​ 2023 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 62,861 ​ ​ 62,861 ​ ​ — ​ $ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 239,527 ​ ​ — ​ ​ 239,527 ​ ​ — U.S. treasury obligations ​ ​ 521,512 ​ ​ 521,512 ​ ​ — ​ ​ — Total ​ $ 823,900 ​ $ 584,373 ​ $ 239,527 ​ $ — ​ The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2022 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ December 31, ​ Markets ​ Inputs ​ Inputs Description ​ 2022 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 95,198 ​ $ 95,198 ​ $ — ​ $ — U.S. treasury obligations ​ ​ 4,989 ​ ​ 4,989 ​ ​ — ​ ​ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 372,671 ​ ​ — ​ ​ 372,671 ​ ​ — U.S. treasury obligations ​ ​ 586,092 ​ ​ 586,092 ​ ​ — ​ ​ — Total ​ $ 1,058,950 ​ $ 686,279 ​ $ 372,671 ​ $ —

Product Revenue Reserves and _2

Product Revenue Reserves and Allowances (Tables)	6 Months Ended
	Jun. 30, 2023
Table Text Blocks
Schedules of concentration of risk	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2023 2022 2023 2022 Customer 1 ​ 41 % ​ 44 % ​ 42 % ​ 46 % Customer 2 ​ 11 % ​ 11 % ​ 11 % ​ 11 % ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2023 ​ 2022 Customer 1 ​ 34 % ​ 33 % Customer 2 ​ 11 % ​ 15 % Customer 3 ​ * ​ ​ 12 % Customer 4 ​ 10 % ​ * ​
Product revenue, net
Table Text Blocks
Summary of revenue recognized	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2023 2022 2023 2022 United States ​ $ 34,307 ​ $ 24,682 ​ $ 69,234 ​ $ 45,834 Rest of World ​ ​ 5,569 ​ ​ 3,772 ​ ​ 9,711 ​ ​ 6,461 Total product revenue ​ $ 39,876 ​ $ 28,454 ​ $ 78,945 ​ $ 52,295
Schedule of product revenue allowance and reserve categories	​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended June 30, ​ 2023 ​ 2022 Beginning balance at January 1 $ 9,788 ​ $ 4,345 Provision related to sales in the current period 16,108 ​ ​ 9,408 Adjustment related to prior periods sales (531) ​ ​ (570) Credits and payments made (10,553) ​ ​ (6,120) Ending balance at June 30 $ 14,812 ​ $ 7,063 ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2023 ​ 2022 Reduction of accounts receivable, net $ 1,257 ​ $ 742 Component of accrued expenses ​ 13,555 ​ ​ 9,046 Total revenue-related reserves $ 14,812 ​ $ 9,788

Inventory (Tables)

Inventory (Tables)	6 Months Ended
	Jun. 30, 2023
Table Text Blocks
Schedule of capitalized inventory	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2023 2022 Raw materials ​ $ 1,599 ​ $ 4,967 Work in process ​ ​ 32,750 ​ ​ 28,427 Finished goods ​ 2,589 ​ 2,791 Total ​ $ 36,938 ​ $ 36,185 ​ Balance sheet classification ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2023 2022 Inventory ​ $ 17,016 ​ $ 29,697 Other assets ​ 19,922 ​ 6,488 Total ​ $ 36,938 ​ $ 36,185

Accrued Expenses (Tables)

Accrued Expenses (Tables)	6 Months Ended
	Jun. 30, 2023
Table Text Blocks
Schedule of accrued expenses	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2023 2022 Research, development and commercial contract costs ​ $ 40,401 ​ $ 66,041 Employee compensation ​ ​ 26,179 ​ ​ 30,744 Accrued professional fees ​ 13,516 ​ 17,588 Revenue-related reserves ​ ​ 13,555 ​ ​ 9,046 Other ​ ​ 7,511 ​ ​ 7,704 Total ​ $ 101,162 ​ $ 131,123

Collaboration and License Agr_2

Collaboration and License Agreements (Tables)	6 Months Ended
	Jun. 30, 2023
Table Text Blocks
Schedule of collaboration loss sharing	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ June 30, ​ June 30, ​ 2023 2022 ​ 2023 2022 The Company's share of loss in the U.S. for pralsetinib $ 1,234 $ 2,145 ​ $ 2,530 ​ $ 5,410 ​
Schedule of amounts recognized as reductions to expenses	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ 2023 2022 2023 2022 Reductions to selling, general and administrative expenses $ 2,774 $ 4,416 $ 5,555 ​ $ 9,248 Increases in research and development expenses $ 5,347 $ 3,714 $ 13,623 ​ $ 3,704
Roche, Collaboration (Pralsetnib) Agreement
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Summary of revenue recognized	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2023 2022 2023 2022 Manufacturing and research and development services related to $ 229 $ 35 $ 356 $ 691 Royalty revenue ​ 366 ​ 313 ​ 803 600 Total Roche pralsetinib collaboration revenue $ 595 $ 348 $ 1,159 $ 1,291 ​
Summary of contract assets and/or contract liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2023 ​ 2022 Unbilled accounts receivable $ 380 $ — Accrued expenses $ 4,573 $ 6,519
Roche, Collaboration and License (Immunotherapy) Agreement
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Summary of revenue recognized	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2023 2022 ​ 2023 2022 Roche collaboration research and development services revenue $ 3,709 $ 1,183 $ 25,705 $ (765) ​
Roche, Collaboration and License (Immunotherapy) Agreement | Collaboration revenue
Table Text Blocks
Summary of contract assets and/or contract liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2023 2022 ​ 2023 2022 Amounts included in the contract liability at the beginning of the period $ 221 $ 1,395 $ 16,025 $ 2,405 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, 2023 December 31, 2022 ​ Current Noncurrent Total Current Noncurrent Total Deferred revenue $ — $ — $ — $ 2,402 $ 13,624 $ 16,026 Accrued expenses $ 55 $ — $ 55 $ 3,970 $ 7,238 $ 11,208
C Stone
Table Text Blocks
Summary of revenue recognized	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended Six Months Ended ​ ​ June 30, June 30, ​ ​ 2023 2022 2023 2022 License milestone revenue ​ $ 9,000 $ — $ 9,000 $ 4,000 Manufacturing services and royalty revenue related to CStone Territory-specific activities ​ ​ 4,390 ​ ​ 6,082 ​ 6,042 ​ ​ 11,294 Total CStone collaboration revenue ​ $ 13,390 ​ $ 6,082 $ 15,042 ​ $ 15,294 ​
C Stone | Collaboration revenue
Table Text Blocks
Summary of contract assets and/or contract liabilities	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2023 2022 Accounts receivable, net $ 11,219 $ 4,991 Unbilled accounts receivable ​ 2,850 ​ 1,529 Contract liability ​ ​ 3,328 ​ ​ 2,265

Stock-based Compensation (Table

Stock-based Compensation (Tables)	6 Months Ended
	Jun. 30, 2023
Table Text Blocks
Summary of stock option activity	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ ​ Shares ​ ​ Exercise Price Outstanding at December 31, 2022 6,233,451 ​ $ 69.14 Granted 1,126,487 ​ ​ 44.54 Exercised (97,004) ​ ​ 30.48 Canceled (192,527) ​ ​ 72.40 Outstanding at June 30, 2023 7,070,407 ​ $ 65.67 Exercisable at June 30, 2023 4,594,568 ​ $ 68.34
Summary of restricted stock units activity	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ Shares Grant Date Fair Value Unvested shares at December 31, 2022 1,893,944 ​ $ 73.28 Granted 851,876 ​ 44.07 Vested (524,345) ​ 72.51 Forfeited (72,401) ​ 65.62 Unvested shares at June 30, 2023 2,149,074 ​ $ 62.16
Summary of performance-based restricted stock units activity	​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ ​ ​ ​ Grant Date ​ Shares Grant Date Fair Value Unvested shares at December 31, 2022 — ​ $ — Granted 52,500 ​ 59.32 Vested — ​ — Forfeited — ​ — Unvested shares at June 30, 2023 52,500 ​ $ 59.32
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, ​ ​ 2023 ​ 2022 ​ ​ 2023 ​ 2022 Stock options $ 10,337 ​ $ 12,811 ​ $ 21,197 ​ $ 25,755 Restricted stock units ​ 12,913 ​ ​ 12,396 ​ ​ 24,853 ​ ​ 22,782 Performance-based restricted stock units ​ 257 ​ ​ — ​ ​ 344 ​ ​ ​ Employee stock purchase plan ​ 438 ​ ​ 317 ​ ​ 891 ​ ​ 596 Subtotal ​ 23,945 ​ ​ 25,524 ​ ​ 47,285 ​ ​ 49,133 Capitalized stock-based compensation costs ​ (124) ​ ​ (177) ​ ​ (268) ​ ​ (387) Stock-based compensation expense included in total cost and operating expenses $ 23,821 ​ $ 25,347 ​ $ 47,017 ​ $ 48,746 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2023 2022 ​ 2023 2022 Research and development ​ $ 10,227 $ 10,497 $ 20,344 $ 20,538 Selling, general and administrative ​ 13,594 ​ 14,850 ​ 26,673 ​ 28,208 Total ​ $ 23,821 ​ $ 25,347 ​ $ 47,017 ​ $ 48,746

Net Loss Per Share (Tables)

Net Loss Per Share (Tables)	6 Months Ended
	Jun. 30, 2023
Table Text Blocks
Schedule of common stock equivalents excluded from calculation of diluted net loss per share applicable to common stockholders	​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ 2023 2022 Stock options 7,070 ​ 6,387 Restricted stock units 2,149 ​ 1,857 Performance-based restricted stock units ​ 53 ​ — ESPP shares ​ 47 ​ 46 Total 9,319 8,290

Leases (Tables)

Leases (Tables)	6 Months Ended
	Jun. 30, 2023
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: 2023 ​ 2022 ​ 2023 ​ 2022 Lease cost $ 5,662 ​ $ 4,942 ​ $ 11,543 ​ $ 10,911 Sublease income ​ — ​ ​ (600) ​ ​ — ​ ​ (1,381) Net lease cost $ 5,662 ​ $ 4,342 ​ $ 11,543 ​ $ 9,530 ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ June 30, ​ 2023 ​ 2022 Cash paid for amounts included in the measurement of lease liabilities: $ 8,812 ​ $ 7,763 Lease liabilities arising from obtaining right-of-use assets: ​ ​ ​ ​ ​ Operating leases $ 112 ​ $ — ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Operating leases Weighted average remaining lease term in years ​ ​ ​ ​ 6.3 Weighted average discount rate ​ ​ ​ ​ 7.4%

Nature of Business - General In

Nature of Business - General Information (Details)	Jun. 30, 2023 item
Approved precision therapies
Approved precision therapies, number	2

Nature of Business - Cash and C

Nature of Business - Cash and Cash Equivalents and Marketable Securities (Details) $ in Millions	Jun. 30, 2023 USD ($)
Cash, cash equivalents and marketable securities
Cash, cash equivalents and marketable securities	$ 836.6

Financing Arrangements - Genera

Financing Arrangements - General Information (Details) $ in Thousands		1 Months Ended	3 Months Ended	12 Months Ended
	Jun. 30, 2022 USD ($)	Jul. 31, 2022 USD ($) Transaction tranche	Jun. 30, 2023 USD ($)	Dec. 31, 2022 USD ($)
Sixth Street Partners, Financing Arrangements
Debt Financing
Debt instrument, transactions, number | Transaction		2
Sale of Future Royalties and Revenues
Gross Proceeds
Gross proceeds				$ 425,000
Sale of Future Royalties and Revenues | Royalty Pharma, Royalty Purchase Agreement
Debt Financing
Debt instrument, issuance date	Jun. 30, 2022
Debt instrument, face amount	$ 175,000
Transaction costs, gross	3,800
Debt instrument, interest expense accretion			$ 0
Gross Proceeds
Gross proceeds	$ 175,000
Sale of Future Royalties and Revenues | Sixth Street Partners, Future Revenue Purchase Agreement
Debt Financing
Debt instrument, face amount		$ 250,000
Transaction costs, gross		5,400
Gross Proceeds
Gross proceeds		250,000
Effective Interest Rate
Debt instrument, interest rate, effective percentage (as a percent)			10.80%
Loans Payable | Sixth Street Partners, Financing Agreement
Debt Financing
Debt instrument, face amount, potential amount		660,000
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded		50,000
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded, each day thereafter		$ 80,000
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities
Debt Financing
Debt instrument, maturity date		Jun. 30, 2028
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities | Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate
Debt Financing
Debt instrument, basis spread on variable rate		6.50%
Debt instrument, variable rate floor		1%
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		$ 150,000
Debt discounts and transaction costs		12,200
Gross Proceeds
Gross proceeds		150,000
Effective Interest Rate
Debt instrument, interest rate, effective percentage (as a percent)			13.60%
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Delayed Draw Term Loan Facility
Debt Financing
Debt instrument, face amount, potential amount		$ 250,000
Debt instrument, tranches, number | tranche		2

Financing Arrangements - Royalt

Financing Arrangements - Royalty and Sales Information (Details) - Sixth Street Partners $ in Millions	Jul. 31, 2022 USD ($)
Financing Arrangements
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, percentage rate (as a percent)	9.75%
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, amount, maximum	$ 900
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, ratio	1.45
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, amount	$ 362.5
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, percentage increase (as a percent)	15%
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, ratio	1.85
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, amount	$ 462.5

Financing Arrangements - Long-t

Financing Arrangements - Long-term Debt (Details) - Sale of Future Royalties and Revenues - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Royalty Pharma, Royalty Purchase Agreement
Long-term Debt
Long-term debt	$ 175,600
Sixth Street Partners, Future Revenue Purchase Agreement
Long-term Debt
Long-term debt	$ 261,705	$ 254,328

Financing Arrangements - Roll F

Financing Arrangements - Roll Forward (Details) - USD ($) $ in Thousands		1 Months Ended	6 Months Ended	12 Months Ended
	Jun. 30, 2022	Jul. 31, 2022	Jun. 30, 2023	Dec. 31, 2022
Sale of Future Royalties and Revenues
Long-term Debt
Sale of future royalties / borrowings				$ 425,000
Sale of Future Royalties and Revenues | Royalty Pharma, Royalty Purchase Agreement
Long-term Debt
Sale of future royalties / borrowings	$ 175,000
Carrying value, ending balance			$ 175,600
Sale of Future Royalties and Revenues | Sixth Street Partners, Future Revenue Purchase Agreement
Long-term Debt
Carrying value, beginning balance			254,328
Sale of future royalties / borrowings		$ 250,000
Interest expense recognized			14,021
Payments			(6,644)
Carrying value, ending balance			261,705	254,328
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility
Long-term Debt
Carrying value, beginning balance			139,083
Sale of future royalties / borrowings		$ 150,000
Interest expense recognized			9,443
Payments			(8,523)
Carrying value, ending balance			$ 140,003	$ 139,083

Financing Arrangements - Additi

Financing Arrangements - Additional Information (Details) $ in Millions	Jun. 30, 2022 USD ($)
Debt Financing
Royalty Purchase Agreement, milestone payments, achievement of specified net sales milestones, maximum	$ 165

Marketable Securities - Tabular

Marketable Securities - Tabular Disclosure (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Marketable securities, available-for-sale:
Amortized cost	$ 764,807	$ 968,712
Unrealized gain	7	22
Unrealized losses	(3,775)	(9,971)
Fair value	761,039	958,763
U.S. government agency securities
Marketable securities, available-for-sale:
Amortized cost	241,322	377,519
Unrealized losses	(1,795)	(4,848)
Fair value	239,527	372,671
U.S. Treasury obligations
Marketable securities, available-for-sale:
Amortized cost	523,485	591,193
Unrealized gain	7	22
Unrealized losses	(1,980)	(5,123)
Fair value	$ 521,512	$ 586,092

Marketable Securities - Unreali

Marketable Securities - Unrealized Loss Positions (Details) $ in Thousands	Jun. 30, 2023 USD ($) security	Dec. 31, 2022 USD ($) security
Unrealized loss position, aggregate fair value
Unrealized loss position for less than 12 months, aggregate fair value | $	$ 625,248	$ 371,746
Unrealized loss position for more than 12 months, aggregate fair value | $	96,291	550,561
Unrealized loss position, aggregate fair value | $	$ 721,539	$ 922,307
Unrealized loss position, number of positions
Number of held securities in an unrealized loss position for less than 12 months | security	110	66
Number of held securities in an unrealized loss position for 12 months or longer | security	14	56
Number of held securities in an unrealized loss position | security	124	122

Marketable Securities - Impairm

Marketable Securities - Impairment (Details) - USD ($) $ in Thousands	3 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Marketable Securities
Marketable debt securities, charges for credit-related impairments	$ 0	$ 0

Marketable Securities - Maturit

Marketable Securities - Maturities (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Amortized cost
Within one year, amortized cost	$ 649,622	$ 834,440
After one through five years, amortized cost	115,185	134,272
Amortized cost	764,807	968,712
Fair value
Within one year, fair value	647,107	825,283
After one through five years, fair value	113,932	133,480
Investments, available-for-sale	$ 761,039	$ 958,763

Marketable Securities - Proceed

Marketable Securities - Proceeds from Maturities (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Proceeds from the maturities of debt securities
Proceeds from the maturities of debt securities	$ 309,515	$ 79,623	$ 617,765	$ 79,623

Marketable Securities - Realize

Marketable Securities - Realized Gains (Losses) (Details) - USD ($) $ in Thousands	3 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Realized gains (losses) from maturities of debt securities
Realized gains (losses) from maturities of debt securities	$ 0	$ 0

Fair Value of Financial Instr_3

Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 761,039	$ 958,763
U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	239,527	372,671
U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	521,512	586,092
Recurring
Fair Value of Financial Instruments
Total	823,900	1,058,950
Recurring | U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	239,527	372,671
Recurring | U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	521,512	586,092
Recurring | Money market funds
Fair Value of Financial Instruments
Cash equivalents	62,861	95,198
Recurring | U.S. Treasury obligations
Fair Value of Financial Instruments
Cash equivalents		4,989
Recurring | Active Markets (Level 1)
Fair Value of Financial Instruments
Total	584,373	686,279
Recurring | Active Markets (Level 1) | U.S. Treasury obligations
Fair Value of Financial Instruments
Marketable securities, available-for-sale	521,512	586,092
Recurring | Active Markets (Level 1) | Money market funds
Fair Value of Financial Instruments
Cash equivalents	62,861	95,198
Recurring | Active Markets (Level 1) | U.S. Treasury obligations
Fair Value of Financial Instruments
Cash equivalents		4,989
Recurring | Observable Inputs (Level 2)
Fair Value of Financial Instruments
Total	239,527	372,671
Recurring | Observable Inputs (Level 2) | U.S. government agency securities
Fair Value of Financial Instruments
Marketable securities, available-for-sale	$ 239,527	$ 372,671

Product Revenue Reserves and _3

Product Revenue Reserves and Allowances - Product Revenue (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Revenues
Revenue	$ 57,570	$ 36,547	$ 120,857	$ 99,278
Product revenue, net
Revenues
Revenue	39,876	28,454	78,945	52,295
AYVAKIT and AYVAKYT
Revenues
Revenue	34,307	24,682	69,234	45,834
GAVRETO
Revenues
Revenue	$ 5,569	$ 3,772	$ 9,711	$ 6,461

Product Revenue Reserves and _4

Product Revenue Reserves and Allowances - Concentration Risk (Details)	3 Months Ended		6 Months Ended		12 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022	Dec. 31, 2022
Revenue from Contract with Customer Benchmark | Customer Concentration Risk | Customer One
Concentration Risk
Concentration risk (as a percent)	41%	44%	42%	46%
Revenue from Contract with Customer Benchmark | Customer Concentration Risk | Customer Two
Concentration Risk
Concentration risk (as a percent)	11%	11%	11%	11%
Accounts Receivable, Net | Credit Concentration Risk | Customer One
Concentration Risk
Concentration risk (as a percent)			34%		33%
Accounts Receivable, Net | Credit Concentration Risk | Customer Two
Concentration Risk
Concentration risk (as a percent)			11%		15%
Accounts Receivable, Net | Credit Concentration Risk | Customer Three
Concentration Risk
Concentration risk (as a percent)					12%
Accounts Receivable, Net | Credit Concentration Risk | Customer Three | Maximum
Concentration Risk
Concentration risk (as a percent)			10%
Accounts Receivable, Net | Credit Concentration Risk | Customer Four
Concentration Risk
Concentration risk (as a percent)			10%
Accounts Receivable, Net | Credit Concentration Risk | Customer Four | Maximum
Concentration Risk
Concentration risk (as a percent)					10%

Product Revenue Reserves and _5

Product Revenue Reserves and Allowances - Product Revenue Allowance and Reserve (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Product revenue allowance and reserve
Beginning balance	$ 9,788	$ 4,345
Provision related to sales in the current period	16,108	9,408
Adjustment related to prior periods sales	(531)	(570)
Credits and payments made	(10,553)	(6,120)
Ending balance	$ 14,812	$ 7,063

Product Revenue Reserves and _6

Product Revenue Reserves and Allowances - Revenue-related Reserves (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022	Jun. 30, 2022	Dec. 31, 2021
Revenue-related reserves
Revenue-related reserves	$ 14,812	$ 9,788	$ 7,063	$ 4,345
Accounts Receivable, Net
Revenue-related reserves
Revenue-related reserves	1,257	742
Accrued Expenses
Revenue-related reserves
Revenue-related reserves	$ 13,555	$ 9,046

Inventory - Capitalized (Detail

Inventory - Capitalized (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Inventory
Raw materials	$ 1,599	$ 4,967
Work in process	32,750	28,427
Finished goods	2,589	2,791
Total	$ 36,938	$ 36,185

Inventory - Balance Sheet Class

Inventory - Balance Sheet Classification (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Inventory
Inventory	$ 17,016	$ 29,697
Other assets	19,922	6,488
Total	$ 36,938	$ 36,185

Inventory - Write-down (Details

Inventory - Write-down (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Inventory
Inventory write-down	$ 500	$ 0	$ 2,000	$ 0

Restricted Cash (Details)

Restricted Cash (Details) - USD ($) $ in Millions	Jun. 30, 2023	Dec. 31, 2022
Restricted cash
Restricted cash	$ 6.4	$ 5.2

Accrued Expenses (Details)

Accrued Expenses (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Accrued Expenses
External research and development	$ 40,401	$ 66,041
Employee compensation	26,179	30,744
Accrued professional fees	13,516	17,588
Revenue-related reserves	13,555	9,046
Other	7,511	7,704
Total	$ 101,162	$ 131,123

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

36 Months Ended

86 Months Ended

Feb. 26, 2022 USD ($) item Program

Oct. 15, 2019 USD ($)

Jun. 01, 2018 USD ($) item

Oct. 31, 2019 USD ($)

Mar. 31, 2016 USD ($) Program Right

Dec. 31, 2021 USD ($)

Jun. 30, 2023 USD ($)

Jun. 30, 2022 USD ($)

Sep. 30, 2020 USD ($)

Jun. 30, 2023 USD ($)

Jun. 30, 2022 USD ($)

Jun. 30, 2023 USD ($)

Apr. 30, 2023 Program

Dec. 31, 2022 USD ($)

Nov. 08, 2021 USD ($)

Jul. 13, 2020

Collaboration Agreements

Prepaid expenses and other current assets

$ 33,418

$ 33,418

$ 33,418

$ 35,024

Research and development expense

110,063

$ 128,466

222,135

$ 231,599

Collaborative Arrangement | IDRx, Inc. | Director

Collaboration Agreements

Collaborative arrangement, milestones, potential contingent payments

217,500

$ 217,500

217,500

Collaborative arrangement, termination, counterparty written notice period

12 months

Collaborative Arrangement | Proteovant

Collaboration Agreements

Collaborative arrangement, upfront payment

$ 20,000

Collaborative arrangement, milestones, potential contingent payments

632,000

Prepaid expenses and other current assets

$ 20,000

Research and development expense

4,300

$ 1,000

$ 5,700

$ 1,500

Collaborative arrangement, jointly research and advance novel protein degrader therapies into development candidates, number | item

2

Collaborative arrangement, jointly research and advance additional novel protein degrader target programs, number | Program

2

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, specified regulatory and commercialization milestones, achieved

$ 105,000

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

25,000

25,000

25,000

Collaborative arrangement, milestones, research milestones, achieved

25,000

25,000

25,000

Collaborative arrangement, option rights, maximum | Right

5

Collaborative arrangement, development programs, terminated | Program

4

Collaborative arrangement, development programs | Program

5

Collaborative arrangement, agreement termination, clinical development costs payment commitment reduction

5,300

Collaborative Arrangement | Clementia

Collaboration Agreements

Collaborative arrangement, upfront payment, cash payment received

$ 25,000

Collaborative arrangement, milestones, cash payments received

$ 50,000

Collaborative arrangement, milestones, contingent payments eligible to receive

460,000

Collaborative arrangement, inventory purchased by counterparty

$ 1,500

Collaborative arrangement, termination, counterparty written notice period

12 months

Collaborative Arrangement | C Stone

Collaboration Agreements

Collaborative arrangement, upfront payment, cash payment received

$ 40,000

Collaborative arrangement, milestones, contingent payments eligible to receive

$ 307,500

Collaborative arrangement, milestones, achieved

$ 38,500

$ 38,500

$ 38,500

Collaborative arrangement, licensed product term from first commercial sale

12 years

Collaborative arrangement, license option rights, number | item

3

Collaborative arrangement, collaboration programs with exclusive commercialization rights, number | item

3

Collaboration and License Agr_4

Collaboration and License Agreements - Transaction Price (Details) - Collaborative Arrangement - USD ($) $ in Millions				1 Months Ended	2 Months Ended	6 Months Ended
	Jul. 13, 2020	Oct. 15, 2019	Jun. 01, 2018	Mar. 31, 2016	Dec. 31, 2021	Jun. 30, 2023	Jun. 30, 2022
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
Roche, Collaboration and License (Immunotherapy) Agreement
Collaboration Agreements
Collaborative arrangement, transaction price				$ 45
Collaborative arrangement, transaction price, payment commitment reduction							$ 15
Collaborative arrangement, transaction price, increase						$ 9.7
Clementia
Collaboration Agreements
Collaborative arrangement, transaction price		$ 46.5
C Stone
Collaboration Agreements
Collaborative arrangement, transaction price			$ 40

Collaboration and License Agr_5

Collaboration and License Agreements - Equity Investment (Details) - USD ($) $ in Thousands	Aug. 01, 2022	Jun. 30, 2023	Dec. 31, 2022
Equity Investment
Equity investment		$ 27,789	$ 27,789
Collaborative Arrangement | IDRx, Inc. | Director
Equity Investment
Equity investment, license agreement, shares received (in shares)	4,509,105
Equity investment	$ 27,800	$ 27,800
Equity investment, transaction costs	$ 300

Collaboration and License Agr_6

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_7

Collaboration and License Agreements - Revenue Recognized (Details) - USD ($) $ in Thousands		3 Months Ended		6 Months Ended		12 Months Ended		24 Months Ended
	Aug. 01, 2022	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022	Dec. 31, 2021	Dec. 31, 2018	Dec. 31, 2020
Revenues
Revenue		$ 57,570	$ 36,547	$ 120,857	$ 99,278
License revenue | IDRx, Inc. | Director
Revenues
Revenue	$ 27,500
Collaborative Arrangement | Zai Lab (Shanghai) Co., Ltd.
Revenues
Revenue						$ 25,000
Collaborative Arrangement | Clementia
Revenues
Revenue		0			30,000			$ 46,500
Collaborative Arrangement | Collaboration revenue | Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue		595	348	1,159	1,291
Collaborative Arrangement | Collaboration revenue | Roche, Collaboration and License (Immunotherapy) Agreement
Revenues
Revenue		3,709		25,705	(765)
Revenue, net			1,183
Cumulative revenue catch-up		3,500		25,100
Collaborative Arrangement | Collaboration revenue | C Stone
Revenues
Revenue		13,390	6,082	15,042	15,294		$ 40,000
Collaborative Arrangement | License milestone revenue | C Stone
Revenues
Revenue		9,000	0	9,000	4,000
Collaborative Arrangement | Territory-specific activities | Zai Lab (Shanghai) Co., Ltd.
Revenues
Revenue		0	500	0	600
Collaborative Arrangement | Territory-specific activities, manufacturing services and royalty | C Stone
Revenues
Revenue		4,390	6,082	6,042	11,294
Collaborative Arrangement | Territory-specific activities, manufacturing and research and development services | Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue		229	35	356	691
Collaborative Arrangement | Royalty | Roche, Collaboration (Pralsetnib) Agreement
Revenues
Revenue		$ 366	$ 313	$ 803	$ 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, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Collaboration Agreements
Collaboration loss sharing	$ 1,234	$ 2,145	$ 2,530	$ 5,410
Collaborative Arrangement | Roche, Collaboration (Pralsetnib) Agreement
Collaboration Agreements
Collaboration loss sharing	$ 1,234	$ 2,145	$ 2,530	$ 5,410

Collaboration and License Agr_9

Collaboration and License Agreements - Reduction in Expenses (Details) - Collaborative Arrangement - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Roche, Collaboration (Pralsetnib) Agreement
Collaboration Agreements
Collaborative arrangement, commercialization, reduction in selling, general and administrative expenses	$ 2,774	$ 4,416	$ 5,555	$ 9,248
Collaborative arrangement, global development activities, increases in research and development expenses	$ 5,347	3,714	$ 13,623	3,704
C Stone
Collaboration Agreements
Collaborative arrangement, reduction in manufacturing and research and development expenses		$ 600		$ 1,200

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, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement
Change in Contract with Customer, Liability
Amounts included in the contact liability at the beginning of the period	$ 221	$ 1,395	$ 16,025	$ 2,405

Collaboration and License Ag_11

Collaboration and License Agreements - Contract Assets and Liabilities (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Contract Assets
Accounts receivable, net	$ 35,147	$ 23,525
Unbilled accounts receivable	3,120	13,413
Contract Liabilities
Contract liabilities, current	3,328	4,667
Contract liabilities, noncurrent	5,000	13,624
Accrued expenses, current	40,401	66,041
Collaborative Arrangement | Roche, Collaboration (Pralsetnib) Agreement
Contract Assets
Unbilled accounts receivable	380
Contract Liabilities
Accrued expenses	4,573	6,519
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement
Contract Liabilities
Contract liabilities	0	16,026
Contract liabilities, current	0	2,402
Contract liabilities, noncurrent	0	13,624
Accrued expenses	55	11,208
Accrued expenses, current	55	3,970
Accrued expenses, noncurrent	0	7,238
Collaborative Arrangement | Clementia
Contract Liabilities
Contract liabilities	0	0
Collaborative Arrangement | C Stone
Contract Assets
Accounts receivable, net	11,219	4,991
Unbilled accounts receivable	2,850	1,529
Contract Liabilities
Contract liabilities	$ 3,328	$ 2,265

Collaboration and License Ag_12

Collaboration and License Agreements - Performance Obligations (Details) - Collaborative Arrangement	6 Months Ended
	Jun. 30, 2023 item component
IDRx, Inc. | Director
Performance Obligations
Collaborative arrangement, material promises, number	2
Collaborative arrangement, material promises, combined into distinct performance obligation, number	1
Zai Lab (Shanghai) Co., Ltd.
Performance Obligations
Collaborative arrangement, material promises, number	3
Collaborative arrangement, material 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
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, 2023	Jun. 30, 2023	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)	2,398,356
Number of shares available for grant (in shares)		4,839,806

Stock-based Compensation - 2020

Stock-based Compensation - 2020 Inducement Plan (Details) - 2020 Inducement Plan - shares	1 Months Ended
	Jun. 30, 2022	Jun. 30, 2023	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)		1,382,793

Stock-based Compensation - Stoc

Stock-based Compensation - Stock Options - Activity (Details)	6 Months Ended
	Jun. 30, 2023 $ / shares shares
Shares
Outstanding at beginning of period (in shares) | shares	6,233,451
Granted (in shares) | shares	1,126,487
Exercised (in shares) | shares	(97,004)
Cancelled (in shares) | shares	(192,527)
Outstanding at end of period (in shares) | shares	7,070,407
Weighted-Average Exercise Price
Outstanding at beginning of period (in dollars per share) | $ / shares	$ 69.14
Granted (in dollars per share) | $ / shares	44.54
Exercised (in dollars per share) | $ / shares	30.48
Cancelled (in dollars per share) | $ / shares	72.40
Outstanding at end of period (in dollars per share) | $ / shares	$ 65.67
Additional disclosures
Shares - Exercisable (in shares) | shares	4,594,568
Weighted-Average Exercise Price - Exercisable (in dollars per share) | $ / shares	$ 68.34

Stock-based Compensation - Perf

Stock-based Compensation - Performance-based Restricted Stock Units (Details) - Performance-based restricted stock units	6 Months Ended
	Jun. 30, 2023
Stock-based compensation
Award service period	3 years
Award vesting period	3 years
Performance metric, cumulative relative total shareholder return, period	3 years
Minimum
Stock-based compensation
Award grants, percentage of target (as a percent)	0%
Maximum
Stock-based compensation
Award grants, percentage of target (as a percent)	200%

Stock-based Compensation - Unve

Stock-based Compensation - Unvested Awards - Activity (Details)	6 Months Ended
	Jun. 30, 2023 $ / shares shares
Restricted Stock Units
Shares
Unvested at beginning of period (in shares) | shares	1,893,944
Granted (in shares) | shares	851,876
Vested (in shares) | shares	(524,345)
Forfeited (in shares) | shares	(72,401)
Unvested at end of period (in shares) | shares	2,149,074
Weighted-Average Grant Date Fair Value
Unvested at beginning or period (in dollars per share) | $ / shares	$ 73.28
Granted (in dollars per share) | $ / shares	44.07
Vested (in dollars per share) | $ / shares	72.51
Forfeited (in dollars per share) | $ / shares	65.62
Unvested at end of period (in dollars per share) | $ / shares	$ 62.16
Performance-based restricted stock units
Shares
Unvested at beginning of period (in shares) | shares	0
Granted (in shares) | shares	52,500
Unvested at end of period (in shares) | shares	52,500
Weighted-Average Grant Date Fair Value
Unvested at beginning or period (in dollars per share) | $ / shares	$ 0
Granted (in dollars per share) | $ / shares	59.32
Unvested at end of period (in dollars per share) | $ / shares	$ 59.32

Stock-based Compensation - Unre

Stock-based Compensation - Unrecognized Compensation Costs (Details) $ in Millions	Jun. 30, 2023 USD ($)
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock option awards	$ 78.2
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 9 months
Restricted Stock Units
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock awards	$ 112.5
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 9 months
Performance-based restricted stock units
Stock-based compensation
Total unrecognized compensation cost related to non-vested stock awards	$ 2.8
Weighted-average period over which unrecognized compensation cost will be recognized	2 years 8 months 1 day

Stock-based Compensation - Empl

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

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, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Total stock based compensation expense
Stock-based compensation expense	$ 23,821	$ 25,347	$ 47,017	$ 48,746

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, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Total stock based compensation expense
Subtotal	$ 23,945	$ 25,524	$ 47,285	$ 49,133
Capitalized stock-based compensation costs	(124)	(177)	(268)	(387)
Stock-based compensation expense included in total cost and operating expenses	23,821	25,347	47,017	48,746
Employee Stock Option [Member]
Total stock based compensation expense
Subtotal	10,337	12,811	21,197	25,755
Restricted Stock Units
Total stock based compensation expense
Subtotal	12,913	12,396	24,853	22,782
Performance-based restricted stock units
Total stock based compensation expense
Subtotal	257		344
Employee Stock
Total stock based compensation expense
Subtotal	$ 438	$ 317	$ 891	$ 596

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, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Total stock based compensation expense
Stock-based compensation expense	$ 23,821	$ 25,347	$ 47,017	$ 48,746
Research and Development
Total stock based compensation expense
Stock-based compensation expense	10,227	10,497	20,344	20,538
General and Administrative Expense
Total stock based compensation expense
Stock-based compensation expense	$ 13,594	$ 14,850	$ 26,673	$ 28,208

Net Loss Per Share (Details)

Net Loss Per Share (Details) - shares shares in Thousands	3 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	9,319	8,290
Employee Stock Option [Member]
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	7,070	6,387
Restricted Stock Units
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	2,149	1,857
Performance-based restricted stock units
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	53
Employee Stock
Antidilutive securities excluded from computation of earnings per share
Antidilutive securities excluded from computation of earnings per share	47	46

Income Taxes - Financing Arrang

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

Income Taxes - Expense (Benefit

Income Taxes - Expense (Benefit) (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Income tax expense (benefit)
Income tax expense	$ 190	$ 3,130	$ 710	$ 3,313

Income Taxes - Unrecognized Tax

Income Taxes - Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Unrecognized tax benefits
Unrecognized tax benefits	$ 200	$ 0

Leases - Components of Lease Ex

Leases - Components of Lease Expense (Details) - USD ($) $ in Thousands	3 Months Ended		6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022	Jun. 30, 2023	Jun. 30, 2022
Operating leases:
Lease cost	$ 5,662	$ 4,942	$ 11,543	$ 10,911
Sublease income		(600)		(1,381)
Net lease cost	$ 5,662	$ 4,342	$ 11,543	$ 9,530

Leases - Cash Flow Information

Leases - Cash Flow Information (Details) - USD ($) $ in Thousands	6 Months Ended
	Jun. 30, 2023	Jun. 30, 2022
Leases
Cash paid for amounts included in the measurement of lease liabilities, operating cash flows from operating leases	$ 8,812	$ 7,763
Lease liabilities arising from obtaining right-of-use assets, operating leases	$ 112

Leases - Weighted Average Remai

Leases - Weighted Average Remaining Lease-term and Weighted Average Discount Rate (Details)	Jun. 30, 2023
Leases
Weighted average remaining lease term in years	6 years 3 months 18 days
Weighted average discount rate	7.40%

Commitments and Contingencies -

Commitments and Contingencies - Purchase Commitments (Details) $ in Millions	6 Months Ended
	Jun. 30, 2023 USD ($)
Manufacturing Agreement, October 2020
Manufacturing Agreements
Unrecorded unconditional purchase obligation, period increase (decrease)	$ (7.3)

Commitments and Contingencies_2

Commitments and Contingencies - Indemnification Agreements (Details) - USD ($) $ in Thousands	Jun. 30, 2023	Dec. 31, 2022
Indemnification Agreement
Indemnification Agreements
Loss contingency accrual	$ 0	$ 0

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended				6 Months Ended
	Jun. 30, 2023	Mar. 31, 2023	Jun. 30, 2022	Mar. 31, 2022	Jun. 30, 2023	Jun. 30, 2022
Pay vs Performance Disclosure
Net Income (Loss)	$ (132,793)	$ (129,560)	$ (159,709)	$ (105,999)	$ (262,354)	$ (265,709)

Insider Trading Arrangements

Insider Trading Arrangements	6 Months Ended
	Jun. 30, 2023 shares
Trading Arrangements, by Individual
Material Terms of Trading Arrangement	​ ​ ​ ​ ​ ​ ​ Name and Title Action Taken Type of Trading Arrangement Nature of Trading Arrangement Duration of Trading Arrangement Aggregate Number of Securities Jefferey W. Albers ( Chairman of the Board of Directors ) Adoption ( 5/26/23 ) Trading plan intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c) Sale of the Company’s common stock pursuant to the terms of the plan 5/26/23 – 5/31/24 135,073 ​ Christopher Murray ( Chief Technical Operations and Quality Officer ) Adoption ( 6/5/23 ) Trading plan intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c) Sale of the Company’s common stock pursuant to the terms of the plan 6/5/23 – 6/5/24 11,821 ​
Jefferey W. Albers
Trading Arrangements, by Individual
Name	Jefferey W. Albers
Title	Chairman of the Board of Directors
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	5/26/23
Termination Date	5/31/24
Aggregate Available	135,073
Christopher Murray
Trading Arrangements, by Individual
Name	Christopher Murray
Title	Chief Technical Operations and Quality Officer
Rule 10b5-1 Arrangement Adopted	true
Adoption Date	6/5/23
Termination Date	6/5/24
Aggregate Available	11,821