Document and Entity Information
Document and Entity Information - $ / shares | 6 Months Ended | |
Jun. 30, 2021 | Jul. 26, 2021 | |
Cover [Abstract] | ||
Entity Central Index Key | 0001597264 | |
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Jun. 30, 2021 | |
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 | 58,574,031 | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2021 | |
Document Fiscal Period Focus | Q2 | |
Amendment Flag | false |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 534,317 | $ 684,636 |
Marketable securities | 94,032 | 187,213 |
Accounts receivable, net | 25,473 | 7,096 |
Unbilled accounts receivable | 13,767 | 18,213 |
Inventory | 15,211 | 8,581 |
Prepaid expenses and other current assets | 21,270 | 22,020 |
Total current assets | 704,070 | 927,759 |
Marketable securities | 751,702 | 677,873 |
Property and equipment, net | 31,848 | 34,129 |
Operating lease right-of-use assets, net | 64,446 | 67,539 |
Restricted cash | 5,171 | 5,168 |
Other assets | 11,558 | 5,925 |
Total assets | 1,568,795 | 1,718,393 |
Current liabilities: | ||
Accounts payable | 1,847 | 4,370 |
Accrued expenses | 107,173 | 105,938 |
Current portion of operating lease liabilities | 8,465 | 7,935 |
Deferred revenue, current | 7,912 | 12,559 |
Total current liabilities | 125,397 | 130,802 |
Operating lease liabilities, net of current portion | 77,272 | 81,669 |
Deferred revenue, net of current portion | 30,365 | 28,599 |
Other long-term liabilities | 6,957 | 7,235 |
Total liabilities | 239,991 | 248,305 |
Commitments (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; 58,277,412 and 57,793,533 shares issued and outstanding at March 31, 2021 and December 31, 2020, respectively | 59 | 58 |
Additional paid-in capital | 2,174,094 | 2,106,600 |
Accumulated other comprehensive loss | (5,835) | (5,214) |
Accumulated deficit | (839,514) | (631,356) |
Total stockholders' equity | 1,328,804 | 1,470,088 |
Total liabilities and stockholders' equity | $ 1,568,795 | $ 1,718,393 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Jun. 30, 2021 | Dec. 31, 2020 |
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) | 58,554,559 | 57,793,533 |
Common Stock, shares outstanding (in shares) | 58,554,559 | 57,793,533 |
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, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Revenues: | ||||
Revenue | $ 27,295 | $ 8,343 | $ 48,871 | $ 14,510 |
Cost and operating expenses: | ||||
Cost of sales | 6,493 | 127 | 6,595 | 150 |
Research and development | 80,027 | 91,079 | 159,738 | 175,225 |
Selling, general and administrative | 49,286 | 42,174 | 91,288 | 77,829 |
Total cost and operating expenses | 135,806 | 133,380 | 257,621 | 253,204 |
Other income (expense): | ||||
Interest income, net | 633 | 1,586 | 1,371 | 4,490 |
Other expense, net | (373) | (23) | (587) | (224) |
Total other income | 260 | 1,563 | 784 | 4,266 |
Loss before income taxes | (108,251) | (123,474) | (207,966) | (234,428) |
Income tax expense | (193) | (193) | ||
Net loss | (108,444) | (123,474) | (208,159) | (234,428) |
Other comprehensive loss: | ||||
Unrealized gain (losses) on available-for-sale investments | (624) | (1,057) | (1,024) | 1,435 |
Currency translation adjustments | (114) | 6 | 404 | (21) |
Comprehensive loss | $ (109,182) | $ (124,525) | $ (208,779) | $ (233,014) |
Net loss per share - basic (in dollars per share) | $ (1.86) | $ (2.28) | $ (3.58) | $ (4.39) |
Net loss per share - diluted (in dollars per share) | $ (1.86) | $ (2.28) | $ (3.58) | $ (4.39) |
Weighted-average number of common shares used in net loss per share - basic (in shares) | 58,406 | 54,217 | 58,216 | 53,436 |
Weighted-average number of common shares used in net loss per share - diluted (in shares) | 58,406 | 54,217 | 58,216 | 53,436 |
Product revenue, net | ||||
Revenues: | ||||
Revenue | $ 11,433 | $ 5,680 | $ 20,388 | $ 9,138 |
Collaboration revenue | ||||
Revenues: | ||||
Revenue | $ 15,862 | $ 2,663 | $ 28,483 | $ 5,372 |
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, 2019 | $ 49 | $ 1,412,083 | $ (2,534) | $ (945,239) | $ 464,359 |
Beginning Balance (in shares) at Dec. 31, 2019 | 49,272,223 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan | $ 1 | 1,612 | 1,613 | ||
Issuance of common stock under stock plan (in shares) | 186,166 | ||||
Stock-based compensation expense | 17,026 | 17,026 | |||
Issuance of common stock | $ 4 | 308,419 | 308,423 | ||
Issuance of common stock (in shares) | 4,710,144 | ||||
Other comprehensive income | 2,465 | 2,465 | |||
Net loss | (110,953) | (110,953) | |||
Ending Balance at Mar. 31, 2020 | $ 54 | 1,739,140 | (69) | (1,056,192) | 682,933 |
Ending Balance (in shares) at Mar. 31, 2020 | 54,168,533 | ||||
Beginning Balance at Dec. 31, 2019 | $ 49 | 1,412,083 | (2,534) | (945,239) | 464,359 |
Beginning Balance (in shares) at Dec. 31, 2019 | 49,272,223 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Net loss | (234,428) | ||||
Ending Balance at Jun. 30, 2020 | $ 54 | 1,762,099 | (1,120) | (1,179,666) | 581,367 |
Ending Balance (in shares) at Jun. 30, 2020 | 54,284,501 | ||||
Beginning Balance at Mar. 31, 2020 | $ 54 | 1,739,140 | (69) | (1,056,192) | 682,933 |
Beginning Balance (in shares) at Mar. 31, 2020 | 54,168,533 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan | 2,335 | 2,335 | |||
Issuance of common stock under stock plan (in shares) | 98,950 | ||||
Purchase of common stock under ESPP | 942 | 942 | |||
Purchase of common stock under ESPP (in shares) | 17,018 | ||||
Stock-based compensation expense | 19,675 | 19,675 | |||
Other comprehensive income | (1,051) | (1,051) | |||
Net loss | (123,474) | (123,474) | |||
Other | 7 | 7 | |||
Ending Balance at Jun. 30, 2020 | $ 54 | 1,762,099 | (1,120) | (1,179,666) | 581,367 |
Ending Balance (in shares) at Jun. 30, 2020 | 54,284,501 | ||||
Beginning Balance at Dec. 31, 2020 | $ 58 | 2,106,600 | (5,214) | (631,356) | $ 1,470,088 |
Beginning Balance (in shares) at Dec. 31, 2020 | 57,793,533 | 57,793,533 | |||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan | 8,318 | $ 8,318 | |||
Issuance of common stock under stock plan (in shares) | 483,879 | ||||
Stock-based compensation expense | 21,212 | 21,212 | |||
Other comprehensive income | 117 | 117 | |||
Net loss | (99,714) | (99,714) | |||
Ending Balance at Mar. 31, 2021 | $ 58 | 2,136,130 | (5,097) | (731,070) | 1,400,021 |
Ending Balance (in shares) at Mar. 31, 2021 | 58,277,412 | ||||
Beginning Balance at Dec. 31, 2020 | $ 58 | 2,106,600 | (5,214) | (631,356) | $ 1,470,088 |
Beginning Balance (in shares) at Dec. 31, 2020 | 57,793,533 | 57,793,533 | |||
Increase (Decrease) in Stockholders' Equity | |||||
Net loss | $ (208,159) | ||||
Ending Balance at Jun. 30, 2021 | $ 59 | 2,174,094 | (5,835) | (839,514) | $ 1,328,804 |
Ending Balance (in shares) at Jun. 30, 2021 | 58,554,559 | 58,554,559 | |||
Beginning Balance at Mar. 31, 2021 | $ 58 | 2,136,130 | (5,097) | (731,070) | $ 1,400,021 |
Beginning Balance (in shares) at Mar. 31, 2021 | 58,277,412 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan | $ 1 | 11,709 | 11,710 | ||
Issuance of common stock under stock plan (in shares) | 254,823 | ||||
Purchase of common stock under ESPP | 1,733 | 1,733 | |||
Purchase of common stock under ESPP (in shares) | 22,324 | ||||
Stock-based compensation expense | 24,522 | 24,522 | |||
Other comprehensive income | (738) | (738) | |||
Net loss | (108,444) | (108,444) | |||
Ending Balance at Jun. 30, 2021 | $ 59 | $ 2,174,094 | $ (5,835) | $ (839,514) | $ 1,328,804 |
Ending Balance (in shares) at Jun. 30, 2021 | 58,554,559 | 58,554,559 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Cash flows from operating activities | ||
Net loss | $ (208,159) | $ (234,428) |
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: | ||
Depreciation and amortization | 3,200 | 3,239 |
Noncash lease expense | 3,073 | 2,823 |
Stock-based compensation | 44,991 | 36,387 |
Other | 1,352 | 243 |
Changes in assets and liabilities: | ||
Accounts receivable | (18,407) | (2,740) |
Unbilled accounts receivable | 4,446 | 1,872 |
Inventory | (4,542) | (2,748) |
Prepaid expenses and other current assets | (755) | (9,159) |
Other assets | (3,829) | 2,018 |
Accounts payable | (2,544) | 802 |
Accrued expenses | (1,044) | (4,573) |
Deferred revenue | (2,880) | (1,368) |
Operating lease liabilities | (3,845) | (2,860) |
Net cash used in operating activities | (188,943) | (210,492) |
Cash flows from investing activities | ||
Purchases of property and equipment | (835) | (2,466) |
Purchases of investments | (382,644) | (232,148) |
Maturities of investments | 400,500 | 306,035 |
Net cash provided by investing activities | 17,021 | 71,421 |
Cash flows from financing activities | ||
Proceeds from public offerings of common stock, net of issuance cost | 308,750 | |
Net proceeds from stock option exercises and employee stock purchase plan | 21,865 | 4,886 |
Payment of offering costs | (311) | |
Net cash provided by financing activities | 21,865 | 313,325 |
Net increase in cash, cash equivalents, and restricted cash | (150,057) | 174,254 |
Cash, cash equivalents and restricted cash at beginning of period | 689,804 | 119,604 |
Effect of exchange rate changes on cash, cash equivalents and restricted cash | (259) | (12) |
Cash, cash equivalents and restricted cash at end of period | 539,488 | 293,846 |
Supplemental cash flow information | ||
Property and equipment purchases unpaid at period end | 230 | 116 |
Cash paid for taxes, net | $ 598 | $ 47 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Cash Flows (Parenthetical) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 | Jun. 30, 2020 | Dec. 31, 2019 |
Reconciliation of cash, cash equivalents, and restricted cash | ||||
Cash and cash equivalents | $ 534,317 | $ 684,636 | $ 288,678 | |
Restricted cash | 5,171 | 5,168 | 5,168 | |
Total cash, cash equivalents, and restricted cash shown in condensed consolidated statements of cash flows | $ 539,488 | $ 689,804 | $ 293,846 | $ 119,604 |
Nature of Business
Nature of Business | 6 Months Ended |
Jun. 30, 2021 | |
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 hematologic disorders. The Company’s approach is to leverage its novel target discovery engine to systematically and reproducibly identify kinases that are drivers of diseases and to craft highly selective and potent drug candidates that may provide significant and durable clinical responses for patients without adequate treatment options. The Company has two approved precision therapies and is globally advancing multiple programs for genomically defined cancers, systemic mastocytosis, 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, 2021, the Company had cash, cash equivalents and marketable securities of $1,380.1 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, 2021 | |
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, 2020 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on February 17, 2021 (the 2020 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, 2021, the results of its operations for the three and six months ended June 30, 2021 and 2020, stockholder’s equity for the three and six months ended June 30, 2021 and 2020 and cash flows for six months ended June 30, 2021 and 2020. Such adjustments are of a normal and recurring nature. The results for the three and six months ended June 30, 2021 are not necessarily indicative of the results for the year ending December 31, 2021 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, and income taxes. The length of time and full extent to which the ongoing COVID-19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition, including revenues, expenses, reserves and allowances, manufacturing, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain, subject to change and difficult to predict, including as a result of new information that may emerge concerning COVID-19, including the identification and spread of new variants, and the actions taken to contain or treat COVID-19, as well as the economic impact thereof on local, regional, national and international customers and markets. The Company considers the impact of COVID-19 while making the estimates within its consolidated financial statements and there may be changes to those estimates in future periods. Actual results may differ from these estimates. 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, 2021 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2020 Annual Report on Form 10-K. New Accounting Pronouncements Reclassification ​ Certain items in the prior year’s condensed consolidated financial statements have been reclassified to conform to the current presentation. ​ |
Marketable Securities
Marketable Securities | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Marketable Securities | 3. Marketable securities Marketable securities consisted of the following at June 30, 2021 and December 31, 2020 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair June 30, 2021 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 652,235 ​ $ 176 ​ $ (300) ​ $ 652,111 U.S. treasury obligations ​ ​ 193,563 ​ ​ 140 ​ ​ (80) ​ ​ 193,623 Total ​ $ 845,798 ​ $ 316 ​ $ (380) ​ $ 845,734 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair December 31, 2020 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 746,770 ​ $ 513 ​ $ (14) ​ $ 747,269 U.S. treasury obligations ​ ​ 117,368 ​ ​ 449 ​ ​ — ​ ​ 117,817 Total ​ $ 864,138 ​ $ 962 ​ $ (14) ​ $ 865,086 ​ ​ As of June 30, 2021, the Company held 35 debt securities that were in an unrealized loss position with an aggregate fair value of $422.8 million. As of December 31, 2020, the Company held 8 debt securities that were in an unrealized loss position with an aggregate fair value of $125.7 million. As of June 30, 2021 and December 31, 2020, there were no securities held by the Company in an unrealized loss position for more than twelve months. The Company has the intent and ability to hold such securities until recovery. As a result, the Company did not ​ As of June 30, 2021, 69 securities with an aggregate fair value of $751.7 million had remaining maturities between one year and five years. As of December 31, 2020, 65 securities with an aggregate fair value of $677.9 million had remaining maturities between one year and five years. ​ The Company received proceeds of $185.1 million and $400.5 million from maturities of debt securities for the three and six months ended June 30, 2021, respectively, and proceeds of $105.4 million and $306.0 million from maturities of debt securities for the three and six months ended June 30, 2020, respectively. The Company did not realize any gains or losses from maturities of debt securities for the three and six months ended June 30, 2021 and 2020. |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Fair Value of Financial Instruments | 4. Fair Value of Financial Instruments The following table summarizes cash equivalents and marketable securities measured at fair value on a recurring basis as of June 30, 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ June 30, ​ Markets ​ Inputs ​ Inputs Description ​ 2021 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 505,142 ​ $ 505,142 ​ $ — ​ $ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 652,111 ​ ​ — ​ ​ 652,111 ​ ​ — U.S. treasury obligations ​ ​ 193,623 ​ ​ 193,623 ​ ​ — ​ ​ — Total ​ $ 1,350,876 ​ $ 698,765 ​ $ 652,111 ​ $ — ​ The following table summarizes cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2020 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ December 31, ​ Markets ​ Inputs ​ Inputs Description ​ 2020 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 420,567 ​ $ 420,567 ​ $ — ​ $ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 747,269 ​ ​ — ​ ​ 747,269 ​ ​ — U.S. treasury obligations ​ ​ 117,817 ​ ​ 117,817 ​ ​ — ​ ​ — Total ​ $ 1,285,653 ​ $ 538,384 ​ $ 747,269 ​ $ — ​ |
Product Revenue Reserves and Al
Product Revenue Reserves and Allowances | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Product Revenue Reserves and Allowances | 5. Product Revenue Reserves and Allowances In January 2020, the U.S. Food and Drug Administration (FDA) approved AYVAKIT PDGFRA exon 18 mutation, including PDGFRA D842V mutations who require systemic therapy, or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). For the three and six months ended June 30, 2021, the Company generated aggregate net product revenue of $11.4 million and $20.4 million from product sales including AYVAKIT/AYVAKYT and GAVRETO, respectively. For the three and six months ended June 30, 2020, the Company generated aggregate net product revenue of $5.7 million and $9.1 million from sales of AYVAKIT, respectively. The following table summarizes activity in each of the product revenue allowance and reserve categories for the six months ended June 30, 2021 (in thousands): ​ ​ ​ ​ ​ Amount Beginning balance at January 1, 2021 $ 1,192 Provision related to product sales 3,199 Credits and payments made (1,837) Ending balance at June 30, 2021 $ 2,554 ​ ​ The total reserves that are included in the Company’s unaudited condensed consolidated balance sheets, are summarized as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2021 ​ 2020 Reduction of accounts receivable, net $ 391 ​ $ 227 Component of accrued expenses ​ 2,163 ​ ​ 966 Total revenue-related reserves $ 2,554 ​ $ 1,192 ​ ​ |
Inventory
Inventory | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Inventory | 6. Inventory Capitalized inventory consists of the following at June 30, 2021 and December 31, 2020 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2021 2020 Raw materials ​ ​ 3,507 ​ ​ — Work in process ​ ​ 13,475 ​ ​ 9,488 Finished goods ​ 1,868 ​ 914 Total ​ $ 18,850 ​ $ 10,402 ​ Balance sheet classification ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2021 2020 Inventory ​ $ 15,211 ​ $ 8,581 Other assets ​ 3,639 ​ 1,821 Total ​ $ 18,850 ​ $ 10,402 ​ ​ Inventory amounts written down as a result of excess, obsolescence, unmarketability or other reasons are charged to cost of sales. For the three and six months ended June 30, 2021, the Company recognized a write-down of $0.4 million and $0.4 million, respectively. There was no such write-down for the three and six months ended June 30, 2020. Long-term inventory, which primarily consists of work in process, is included in other assets in the unaudited condensed consolidated balance sheets. |
Restricted Cash
Restricted Cash | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Restricted Cash | 7. Restricted Cash At June 30, 2021 and December 31, 2020, $5.2 million and $5.2 million, respectively, of the Company’s cash is restricted by a bank primarily related to security deposits for the Company’s building lease agreements. |
Property and Equipment, Net
Property and Equipment, Net | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Property and Equipment, Net | 8. Property and Equipment, Net Property and equipment and related accumulated depreciation are as follows (in thousands, except year data): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Estimated ​ ​ ​ ​ ​ ​ ​ ​ Useful Life ​ June 30, ​ December 31, ​ ​ (Years) ​ 2021 ​ 2020 Lab equipment 5 $ 12,246 $ 11,418 Furniture and fixtures 4 ​ 3,417 ​ 3,420 Computer equipment 3 ​ 1,555 ​ 1,513 Leasehold improvements Term of lease ​ 36,945 ​ 36,946 Software 3 ​ 412 ​ 412 Construction-in-progress ​ ​ ​ ​ 162 ​ ​ 151 Total cost ​ ​ ​ 54,737 ​ 53,860 Less: accumulated depreciation and amortization ​ ​ ​ (22,889) ​ (19,731) Total ​ ​ ​ $ 31,848 ​ $ 34,129 ​ Property, plant and equipment are recorded at historical cost, net of accumulated depreciation. For the three and six months ended June 30, 2021, depreciation expense totaled $1.6 million and $3.2 million, respectively, compared to $1.7 million and $3.2 million, respectively, for the three and six months ended June 30, 2020. |
Accrued Expenses
Accrued Expenses | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Accrued Expenses | 9. Accrued Expenses Accrued expenses consist of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2021 2020 Research, development and commercial contract costs ​ $ 68,793 ​ $ 60,255 ​ Employee compensation ​ ​ 16,667 ​ ​ 27,622 ​ Accrued professional fees ​ 14,064 ​ 10,986 ​ Revenue-related reserves ​ ​ 2,163 ​ ​ 966 ​ Other ​ ​ 5,486 ​ ​ 6,109 ​ Total ​ $ 107,173 ​ $ 105,938 ​ ​ |
Collaboration and License Agree
Collaboration and License Agreements | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Collaboration and License Agreements | 10 . Collaboration and License Agreements Roche – Pralsetinib Collaboration Under the Roche pralsetinib collaboration agreement, the Company received an upfront cash payment of $675.0 million, and through June 30, 2021, the Company has received a total of $55.0 million in specified regulatory and commercialization milestones. In addition to the upfront and milestone payments received through June 30, 2021, the Company is eligible to receive up to $872.0 million in contingent payments, including specified development, regulatory and sales-based milestones for pralsetinib and any licensed product containing a next-generation RET compound. In the U.S., the Company and Roche agreed to work together to co-commercialize pralsetinib and equally share responsibilities, profits and losses. In addition, the Company is eligible to receive tiered royalties ranging from high-teens to mid-twenties on annual net sales of pralsetinib outside the U.S., excluding Greater China (the Roche territory). The Company and Roche have also agreed to co-develop pralsetinib globally in RET-altered solid tumors, including non-small cell lung cancer, medullary thyroid carcinoma and other thyroid cancers, as well as other solid tumors. The Company and Roche will share global development costs for pralsetinib at a rate of 45 percent for the Company and 55 percent for Roche up to a specified amount of aggregate joint development costs, after which the Company’s share of global development costs for pralsetinib will be reduced by a specified percentage. The Company and Roche will also share specified global development costs for any next-generation RET compound co-developed under the collaboration in a similar manner. Unless earlier terminated in accordance with its terms, the Roche pralsetinib collaboration agreement will expire on a licensed product-by-licensed product basis (i) in the U.S. upon the expiration of the gross profit sharing term for such licensed product and (ii) outside the U.S. on a country-by-country basis at the end of the applicable royalty term for such licensed product. Roche may terminate the agreement in its entirety or on a licensed product-by-licensed product or country-by-country basis subject to certain notice periods. Either party may terminate the Roche pralsetinib collaboration agreement for the other party’s uncured material breach or insolvency. Subject to the terms of the Roche pralsetinib collaboration agreement, effective upon termination of the agreement, the Company is entitled to retain specified licenses to be able to continue to exploit the licensed products. In connection with the Roche collaboration agreement, on July 13, 2020, the Company also entered into a stock purchase agreement with Roche Holdings, Inc. (Roche Holdings) pursuant to which the Company issued and sold an aggregate of 1,035,519 of shares of common stock to Roche Holdings at a purchase price of $96.57 per share and received $100.0 million in the third quarter of 2020. The closing for a minority portion of the equity investment occurred following the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and other customary closing conditions. The Company considered the ASC 606 criteria for combining contracts and determined that the Roche pralsetinib collaboration agreement and stock purchase agreement should be combined into a single contract because they were negotiated and entered into in contemplation of one another. The Company accounted for the common stock issued to Roche Holdings based on the fair market value of the common stock on the dates of issuance. The fair market value of the common stock issued to Roche Holdings was $79.3 million, based on the closing price of the Company’s common stock on the dates of issuance, resulting in a $20.7 million premium. The Company determined that the premium paid by Roche Holdings for the common stock should be attributed to the transaction price of the Roche pralsetinib collaboration agreement. The Company determined that the Roche pralsetinib collaboration agreement contained four material components: (i) licenses granted to Roche to develop and commercialize pralsetinib worldwide, excluding the CStone territory (pralsetinib license); (ii) the Roche territory-specific commercialization activities for pralsetinib, including manufacturing Roche territory activities); (iii) the parties’ joint development activities for pralsetinib worldwide, excluding the CStone territory; and (iv) the parties’ joint commercialization activities for pralsetinib in the U.S. The Company considered the guidance in ASC 606 to determine which of the components of the Roche pralsetinib collaboration agreement are performance obligations with a customer and concluded that the pralsetinib license and the Roche territory activities are within the scope of ASC 606 because Roche is the Company’s customer in those transactions. The Company evaluated the Roche pralsetinib license under ASC 606 and concluded that the pralsetinib license is a functional intellectual property license and is a distinct performance obligation. The Company determined that Roche benefited from the pralsetinib license at the time of grant, and therefore the related performance obligation is satisfied at a point in time. The Company evaluated the Roche territory activities under ASC 606 and identified one material promise associated with manufacturing activities related to development and commercial supply of pralsetinib in the Roche territory for up to 24 months. Given that Roche is not obligated to purchase any minimum amount or quantities of the development and commercial supply from the Company, the Company concluded that, for the purpose of ASC 606, the provision of manufacturing activities related to development and commercial supply of pralsetinib in Roche territory was an option but not a performance obligation of the Company at the inception of the Roche collaboration agreement and will be accounted for if and when exercised. The Company also concluded that there is no separate material right in connection with the development and commercial supply of pralsetinib, as the expected pricing was not issued at a significant and incremental discount. Therefore, the manufacturing activities were excluded as performance obligations at the outset of the arrangement. Additionally, the Company is entitled to sales milestones and royalties from Roche upon future sales of pralsetinib in the Roche territory, and revenue will be 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, 2021, the Company has achieved The following table summarizes revenue recognized under the Roche pralsetinib collaboration during the three and six months ended June 30, 2021 and 2020 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 Manufacturing and other services related to Roche territory-specific activities ​ $ 1,912 ​ $ — ​ $ 3,684 ​ $ — Total Roche pralsetinib collaboration revenue ​ $ 1,912 $ — $ 3,684 $ — ​ For the parties’ participation in global development for pralsetinib and the U.S. commercialization activities for GAVRETO, the Company concluded that those activities and cost-sharing payments related to such activities are within the scope of ASC 808, as both parties are active participants in the development, manufacturing and commercialization activities and are exposed to significant risks and rewards of those activities under the Roche pralsetinib collaboration agreement. Payments to or reimbursements from Roche related to the global development activities are accounted for as an increase to or reduction of research and development expenses. Prior to July 1, 2021, the Company was the principal for product sales to customers in the U.S. and recognized revenues on sales to third parties in product revenue, net in its unaudited condensed consolidated statements of operations and comprehensive loss. After July 1, 2021, Roche has taken over the responsibilities associated with product sales to customers, pricing and distribution matters for GAVRETO in the U.S. and has become the principal for product sales to customers in the U.S., and the Company will recognize its portion of the commercial profits and losses sharing as revenue (expenses) from the collaboration arrangement in its condensed consolidated statements of operations and comprehensive loss. During the three and six months ended June 30, 2021, the Company recorded $2.2 million and $5.2 million, respectively, as reductions in selling, general and administrative expenses in connection with the commercialization of GAVRETO in the U.S. and $2.1 million and $6.2 million, respectively, as reductions in research and development expenses in connection with global development activities for pralsetinib. The following table summarizes the contract assets associated with the Roche pralsetinib collaboration as of June 30, 2021 and December 31, 2020 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2021 ​ 2020 Accounts receivable, net ​ $ 11,865 $ — Unbilled accounts receivable ​ $ 7,224 $ 17,600 ​ ​ Clementia On October 15, 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, 2021, the Company has received $20.0 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, 2021, the Company is eligible to receive up to $490.0 million in potential development, regulatory and sales-based milestone payments 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 on or after the second anniversary of the effective date of the agreement upon at least 12 months’ prior written notice to the Company, which cannot be delivered before the first anniversary of the effective date. Either party may terminate the agreement for the other party’s uncured material breach or insolvency and in certain other circumstances agreed to by the parties. In certain termination circumstances, the Company is entitled to retain specified licenses to be able to continue to exploit the Clementia licensed products. The Company evaluated the Clementia agreement under ASC 606, as the agreement represented a transaction with a customer. The Company identified the following material promises under the agreement: (1) the exclusive license to develop, manufacture and commercialize BLU-782; (2) the technology transfer of BLU-782 program; (3) the transfer of existing manufacturing inventory; and (4) the transfer of in-process manufacturing inventory. In addition, the Company determined that the exclusive license and technology transfer were not distinct from each other, as the exclusive license has limited value without the corresponding technology transfer. As such, for the purposes of ASC 606, the Company determined that these four material promises, described above, should be combined into three performance obligations: (1) the exclusive license and the technology transfer; (2) the transfer of existing manufacturing inventory; and (3) the transfer of in-process manufacturing inventory. The Company determined that the transaction price as of the outset of the arrangement was $46.5 million, which consisted of the upfront amount of $25.0 million, the $20.0 million cash milestone payment received in the third quarter of 2020 , the purchase of existing manufacturing inventory of $1.2 million and the purchase of in-process manufacturing inventory of $0.3 million. 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 transaction price was allocated to the three performance obligations on a relative stand-alone selling price basis. The Company satisfies the performance obligations upon delivery of the license and completion of the technology transfer and inventory transfers. During 2019, the Company completed the delivery of the license, the technology transfer and the transfer of existing manufacturing inventory and recognized a total of $46.2 million as revenue. During the three and six months ended June 30, 2021 and 2020, no material revenue was recognized from the Clementia collaboration. There was no revenue deferred as a contract liability associated with the Clementia agreement as of June 30, 2021 and December 31, 2020. CStone Pharmaceuticals On June 1, 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, 2021, the Company has achieved $23.0 million in milestones under this collaboration. Subject to the terms of the CStone agreement, in addition to the upfront payments received and milestones achieved through June 30, 2021, the Company will be eligible to receive up to approximately $323.0 million in additional milestone payments, including $95.5 million related to development and regulatory milestones and $227.5 million related to sales-based milestones. In addition, CStone will be obligated to pay the Company tiered percentage royalties on a licensed product-by-licensed product basis ranging from the mid-teens to low twenties on annual net sales of each licensed product in the CStone territory, subject to adjustment in specified circumstances. CStone will be responsible for costs related to the development of the licensed products in the CStone territory, other than specified costs related to the development of fisogatinib as a combination therapy in the CStone territory that will be shared by the Company and CStone. Pursuant to the terms of the CStone agreement, CStone will be responsible for conducting all development and commercialization activities in the CStone territory related to the licensed products. Subject to specified exceptions, during the term of the CStone agreement, each party has agreed that neither it nor its affiliates will conduct specified development and commercialization activities in the CStone territory related to selective inhibitors of FGFR4, KIT, PDGFRA and RET. In addition, under the CStone agreement, each party has granted the other party specified intellectual property licenses to enable the other party to perform its obligations and exercise its rights under the CStone agreement, including license grants to enable each party to conduct research, development and commercialization activities pursuant to the terms of the CStone agreement. The CStone agreement will continue on a licensed product-by-licensed product and CStone region-by-CStone region basis until the later of (i) 12 years after the first commercial sale of a licensed product in a CStone region in the CStone territory and (ii) the date of expiration of the last valid patent claim related to the Company’s patent rights or any joint collaboration patent rights for the licensed product that covers the composition of matter, method of use or method of manufacturing such licensed product in such region. Subject to the terms of the CStone agreement, CStone may terminate the CStone agreement in its entirety or with respect to one or more licensed products for convenience by providing written notice to the Company after June 1, 2019, and CStone may terminate the CStone agreement with respect to a licensed product for convenience at any time by providing written notice to the Company following the occurrence of specified events. In addition, the Company may terminate the CStone agreement under specified circumstances if CStone or certain other parties challenges the Company’s patent rights or any joint collaboration patent rights or if CStone or its affiliates do not conduct any material development or commercialization activities with respect to one or more licensed products for a specified period of time, subject to specified exceptions. Either party may terminate the CStone agreement for the other party’s uncured material breach or insolvency. In certain termination circumstances, the parties are entitled to retain specified licenses to be able to continue to exploit the licensed products, and in the event of termination by CStone for the Company’s uncured material breach, the Company will be obligated to pay CStone a low single digit percentage royalty on a licensed product-by-licensed product basis on annual net sales of such licensed product in the CStone territory, subject to a cap and other specified exceptions. The Company evaluated the CStone agreement to determine whether it is a collaborative arrangement for purposes of ASC 808. The Company determined that there were two material components of the CStone agreement: (i) the CStone territory-specific license and related activities in the CStone territory, and (ii) the parties’ participation in global development of the licensed products. The Company concluded that the CStone territory-specific license and related activities in the CStone territory are not within the scope of ASC 808 because the Company is not exposed to significant risks and rewards. The Company concluded that CStone is a customer with regard to the component that includes the CStone territory-specific license and related activities in CStone territory, which include manufacturing. For the parties’ participation in global development of the licensed products, the Company concluded that the research and development activities and cost-sharing payments related to such activities are within the scope of ASC 808 as both parties are active participants exposed to the risk of the activities under the CStone agreement. The Company concluded that CStone is not a customer with regard to the global development component in the context of the CStone agreement. Therefore, payments received by the Company for global development activities under the CStone agreement, including manufacturing, will be accounted for as a reduction of related expenses. A summary of manufacturing and research and development services related to the global development activities, net of expenses payable to CStone during the three and six months ended June 30, 2021 and 2020 is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 Manufacturing and research and development services related to global development activities, net of expenses payable to CStone ​ $ 269 $ 920 $ 1,008 $ 2,450 ​ ​ The Company evaluated the CStone territory-specific license and related activities in the CStone territory under ASC 606, as these transactions are considered transactions with a customer. The Company identified the following material promises under the arrangement: (1) the three exclusive licenses granted in the CStone territory to develop, manufacture and commercialize the three licensed products; (2) the initial know-how transfer for each licensed product; (3) manufacturing activities related to development and commercial supply of the licensed products; (4) participation in the joint steering committee (JSC) and joint project teams (JPT); (5) regulatory responsibilities; and (6) manufacturing technology and continuing know-how transfers. The Company determined that each licensed product is distinct from the other licensed products. In addition, the Company determined that the exclusive licenses and initial know-how transfers for each licensed product were not distinct from each other, as each exclusive license has limited value without the corresponding initial know-how transfer. For purposes of ASC 606, the Company determined that that participation on the JSC and JPTs, the regulatory responsibilities and the manufacturing technology and continuing know-how transfers are qualitatively and quantitatively immaterial in the context of the CStone agreement and therefore are excluded from performance obligations. As such, the Company determined that these six material promises, described above, should be combined into one performance obligation for each of the three candidates. The Company evaluated the provision of manufacturing activities related to development and commercial supply of the licensed products as an option for purposes of ASC 606 to determine whether these manufacturing activities provide CStone with any material rights. The Company concluded that the manufacturing activities were not issued at a significant and incremental discount, and therefore do not provide CStone with any material rights. As such, the manufacturing activities are excluded as performance obligations at the outset of the arrangement. Based on these assessments, the Company identified three distinct performance obligations at the outset of the CStone agreement, which consists of the following for each licensed product: (1) the exclusive license and (2) the initial know-how transfer. Under the CStone agreement, in order to evaluate the transaction price for purposes of ASC 606, the Company determined that the upfront amount of $40.0 million constituted the entirety of the consideration to be included in the transaction price as of the outset of the arrangement, which was allocated to the three performance obligations. The potential milestone payments that the Company is eligible to receive were excluded from the transaction price, as all milestone amounts were fully constrained based on the probability of achievement. The Company satisfied the performance obligations upon delivery of the licenses, initial know-how transfers and product trademark and recognized the upfront payment of $40.0 million as revenue during the second quarter of 2018. During the three months ended June 30, 2021 and 2020, no milestones were achieved under the CStone agreement. During the six months ended June 30, 2021 and 2020, cash consideration associated with achieved regulatory and development milestones of $9.0 million and $2.0 million, respectively, were added to the estimated transaction price and recognized as revenue in such periods. The Company will reevaluate the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company will adjust its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. During the three months ended June 30, 2021, the Company entered into commercial supply agreements and avapritinib manufacturing technology transfer agreement with CStone for supply drug substance of avapritinib and drug product of avapritinib and pralsetinib to assist CStone’s commercialization activities conducted specifically for the CStone territory. The manufacturing activities were considered as distinct performance obligations from the CStone agreement and collaboration revenue was recognized upon delivery of the drug substance and drug product to CStone. A summary of revenue recognized under the CStone agreement during the three and six months ended June 30, 2021 and 2020 is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 License milestone revenue ​ $ — $ — $ 9,000 $ 2,000 Manufacturing services related to CStone territory-specific activities ​ ​ 10,355 ​ ​ 1,263 ​ ​ 10,538 ​ ​ 1,397 Royalty revenue ​ ​ 1,947 ​ ​ — ​ ​ 2,035 ​ ​ — Total CStone collaboration revenue ​ $ 12,302 ​ $ 1,263 ​ $ 21,573 ​ $ 3,397 ​ ​ The following table presents the contract assets associated with the CStone collaboration as of June 30, 2021 and December 31, 2020 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ ​ December 31, ​ ​ 2021 ​ 2020 Accounts receivable, net ​ $ 5,486 $ 563 Unbilled accounts receivable ​ $ 6,512 $ — ​ As of June 30, 2021, the Company had $6.2 million of deferred revenue as a contract liability associated with the CStone collaboration, of which $1.7 million was included in current liabilities. This contract liability resulted from advance payments made by CStone in connection with commercial supply of avapritinib and pralsetinib for the CStone territory. The contract liability associated with the CStone collaboration was $6.5 million at December 31, 2020. Roche – Immunotherapy Collaboration In March 2016, the Company entered into a collaboration and license agreement (as amended, the Roche immunotherapy agreement) with F. Hoffmann-La Roche Ltd and Hoffmann-La Roche Inc. (collectively, Roche) for the discovery, development and commercialization of small molecule therapeutics targeting kinases believed to be important in cancer immunotherapy (including BLU-852, a development candidate for the kinase target MAP4K1, which is believed to play a role in T cell regulation), as single products or possibly in combination with other therapeutics. Under the Roche immunotherapy agreement, Roche was originally granted up to five option rights to obtain an exclusive license to exploit products derived from the collaboration programs in the field of cancer immunotherapy. Such option rights are triggered upon the achievement of Phase 1 proof-of-concept. As a result of amendments to the Roche immunotherapy agreement in the fourth quarter of 2019 and in the first quarter of 2021, the Company and Roche are currently conducting activities for up to two programs under the collaboration. Prior to Roche’s exercise of an option, the Company will have the lead responsibility for drug discovery and pre-clinical development of all collaboration programs. In addition, the Company will have the lead responsibility for the conduct of all Phase 1 clinical trials other than those Phase 1 clinical trials for any product in combination with Roche’s portfolio of therapeutics, for which Roche will have the right to lead the conduct of such Phase 1 clinical trials. Pursuant to the Roche immunotherapy agreement, the parties will share the costs of Phase 1 development for each collaboration program. In addition, Roche will be responsible for post-Phase 1 development costs for each licensed product for which it retains global commercialization rights, and the Company and Roche will share post-Phase 1 development costs for each licensed product for which the Company retains commercialization rights in the U.S. The Company received an upfront cash payment of $45.0 million, and through June 30, 2021, the Company has received $19.5 million in milestone payments under this collaboration. Subject to the terms of the Roche immunotherapy agreement, as amended, in addition to the upfront and milestone payments received through June 30, 2021, the Company is eligible to receive up to approximately $323.3 million in contingent option fees and milestone payments related to specified research, pre-clinical, clinical, regulatory and sales-based milestones. In addition, for any licensed product for which Roche retains worldwide commercialization rights, the Company will be eligible to receive tiered royalties ranging from low double-digits to high-teens on future net sales of the licensed product. For any licensed product for which the Company retains commercialization rights in the U.S., the Company and Roche will be eligible to receive tiered royalties ranging from mid-single-digits to low double-digits on future net sales in the other party’s respective territories in which it commercializes the licensed product. The upfront cash payment and any payments for milestones, option fees and royalties are non-refundable, non-creditable and not subject to set-off. The Roche immunotherapy agreement will continue until the date when no royalty or other payment obligations are or will become due, unless earlier terminated in accordance with the terms of the Roche immunotherapy agreement. Prior to its exercise of its first option, Roche may terminate the Roche immunotherapy agreement at will, in whole or on a collaboration target-by-collaboration target basis, upon 120 days’ prior written notice to the Company. Following its exercise of an option, Roche may terminate the Roche immunotherapy agreement at will, in whole, on a collaboration target-by-collaboration target basis, on a collaboration program-by-collaboration program basis or, if a licensed product has been commercially sold, on a country-by-country basis, (i) upon 120 days’ prior written notice if a licensed product has not been commercially sold or (ii) upon 180 days’ prior written notice if a licensed product has been commercially sold. Either party may terminate the Roche immunotherapy agreement for the other party’s uncured material breach or insolvency and in certain other circumstances agreed to by the parties. In certain termination circumstances, the Company is entitled to retain specified licenses to be able to continue to exploit the licensed products. The Company assessed this arrangement in accordance with ASC 606 upon the adoption of the new standard on January 1, 2018, and concluded that the contract counterparty, Roche, is a customer prior to the exercise, if any, of an option by Roche. The Company identified the following material promises under the arrangement: (1) a non-transferable, sub-licensable and non-exclusive license to use the Company’s intellectual property and collaboration compounds to conduct research activities; (2) research and development activities through Phase 1 clinical trials under the research plan; (3) five option rights for licenses to develop, manufacture, and commercialize the collaboration targets; (4) participation on a joint research committee (JRC) and joint development committee (JDC); and (5) regulatory responsibilities under Phase 1 clinical trials. The Company determined that the license and research and development activities were not distinct from another, as the license has limited value without the performance of the research and development activities. Participation on the JRC and JDC to oversee the research and development activities was determined to be quantitatively and qualitatively immaterial and therefore is excluded from performance obligations. The regulatory responsibilities related to filings and obtaining approvals related to the drugs that may result from each program do not represent separate performance obligations based on their dependence on the research and development efforts |
Stock-based Compensation
Stock-based Compensation | 6 Months Ended |
Jun. 30, 2021 | |
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Stock-based Compensation | 11. Stock-based compensation 2015 Stock Option and Incentive Plan In 2015, the Company’s board of directors and stockholders approved the 2015 Stock Option and Incentive Plan (the 2015 Plan), which replaced the Company’s 2011 Stock Option and Grant Plan, as amended (the 2011 Plan). The 2015 Plan includes incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock, restricted stock units, unrestricted stock, performance share awards and cash-based awards. The Company initially reserved a total of 1,460,084 shares of common stock for the issuance of awards under the 2015 Plan. The 2015 Plan provides that the number of shares reserved and available for issuance under the 2015 Plan will be cumulatively increased on January 1 of each calendar year by 4% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar year beginning January 1, 2021, the number of shares reserved for issuance under the 2015 Plan was increased by 2,311,741 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, 2021, there were 2,913,775 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. 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, 2021, there were 485,774 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, 2021: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ ​ Shares ​ ​ Exercise Price Outstanding at December 31, 2020 6,030,641 ​ $ 61.28 Granted 908,661 ​ ​ 98.95 Exercised (468,534) ​ ​ 42.73 Canceled (271,982) ​ ​ 77.49 Outstanding at June 30, 2021 6,198,786 ​ $ 67.49 Exercisable at June 30, 2021 3,428,017 ​ $ 57.20 ​ As of June 30, 2021, the total unrecognized compensation expense related to unvested stock option awards was $120.2 million, which is expected to be recognized over a weighted-average period of approximately 2.61 years. Restricted stock units The following table summarizes the restricted stock units activity for the six months ended June 30, 2021: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ Shares Grant Date Fair Value Unvested shares at December 31, 2020 1,171,686 ​ $ 65.37 Granted 778,521 ​ 99.41 Vested (270,168) ​ 63.67 Forfeited (123,644) ​ 73.44 Unvested shares at June 30, 2021 1,556,395 ​ $ 82.05 ​ As of June 30, 2021, the total unrecognized compensation expense related to unvested restricted stock units was $113.7 million, which is expected to be recognize over a weighted-average period of approximately 3.07 years. 2015 Employee Stock Purchase Plan In 2015, the Company’s board of directors and stockholders approved the 2015 Employee Stock Purchase Plan (the 2015 ESPP), which became effective upon the closing of the Company’s initial public offering in May 2015. The Company initially reserved a total of 243,347 shares of common stock for issuance under the 2015 ESPP. The 2015 ESPP provides that the number of shares reserved and available for issuance under the 2015 ESPP will be cumulatively increased on January 1 of each calendar year by 1% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar year beginning January 1, 2021, the number of shares reserved for issuance under the 2015 ESPP was increased by 577,935 shares. Stock-based compensation expense The Company recognized stock-based compensation expense totaling $24.3 million and $45.0 million for the three and six months ended June 30, 2021, respectively, and $19.5 million and $36.4 million for the three and six months ended June 30, 2020, 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, ​ ​ 2021 ​ 2020 ​ ​ 2021 ​ 2020 Stock options $ 14,965 ​ $ 14,629 ​ $ 29,107 ​ $ 28,344 Restricted stock units ​ 9,270 ​ ​ 4,844 ​ ​ 16,064 ​ ​ 7,998 Employee stock purchase plan ​ 287 ​ ​ 202 ​ ​ 563 ​ ​ 359 Subtotal ​ 24,522 ​ ​ 19,675 ​ ​ 45,734 ​ ​ 36,701 Capitalized stock-based compensation costs ​ (215) ​ ​ (147) ​ ​ (743) ​ ​ (314) Stock-based compensation expense included in total cost and operating expenses $ 24,307 ​ $ 19,528 ​ $ 44,991 ​ $ 36,387 ​ Stock-based compensation expense by classification within the unaudited condensed consolidated statements of operations and comprehensive loss is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 Research and development ​ $ 10,485 $ 8,709 $ 19,432 $ 16,507 Selling, general and administrative ​ 13,822 ​ 10,819 ​ 25,559 ​ 19,880 Total ​ $ 24,307 ​ $ 19,528 ​ ​ 44,991 ​ ​ 36,387 ​ |
Net Loss Per Share
Net Loss Per Share | 6 Months Ended |
Jun. 30, 2021 | |
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 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 periods indicated because including them would have had an anti-dilutive effect (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ ​ June 30, ​ 2021 2020 Stock options 6,199 ​ 6,661 Restricted stock units 1,556 ​ 1,151 ESPP shares ​ 25 ​ 26 Total 7,780 7,838 ​ |
Income Taxes
Income Taxes | 6 Months Ended |
Jun. 30, 2021 | |
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, 2021, the Company has a full valuation allowance applied against its U.S. and foreign deferred tax assets. On March 11, 2021, President Joe Biden signed into law a relief and stimulus package known as the American Rescue Plan Act of 2021 (ARPA) stimulus package. While this Act provides various tax provisions including for example, extending the employee retention credit through the end of 2021, modifying the paid sick and family leave credits, repealing the worldwide interest allocation rules that were scheduled to take effect in 2021, and expanding the number of employees subject to the limit on the deduction for executive compensation under Section 162(m) beginning in 2027, among other things, based on the Company’s initial review of the various business tax provisions offered in the ARPA along with having a valuation allowance on its U.S. deferred tax assets, it does not believe that there is an impact to the Company and as such the recording of a discrete item was not required during the three and six months ended June 30, 2021. |
Leases
Leases | 6 Months Ended |
Jun. 30, 2021 | |
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 have remaining lease terms of one to eight years and contain various clauses for renewal at the Company’s option. The renewal options were not included in the calculation of the operating lease assets and the operating lease as the renewal option is not reasonably certain of being exercised. The lease agreements do not contain residual value guarantees and t ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ June 30, ​ June 30, Operating leases: 2021 ​ 2020 ​ 2021 ​ 2020 Lease cost $ 4,451 ​ $ 4,328 ​ $ 9,177 ​ $ 8,709 Sublease income ​ (268) ​ ​ (730) ​ ​ (595) ​ ​ (1,451) Net lease cost $ 4,183 ​ $ 3,598 ​ $ 8,582 ​ $ 7,258 ​ ​ The Company has not entered into any material short-term leases or financing leases as of June 30, 2021. Supplemental cash flow information related to leases for the six months ended June 30, 2021 and 2020 is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ June 30, ​ 2021 ​ 2020 Cash paid for amounts included in the measurement of lease liabilities: $ 7,394 ​ $ 7,172 Lease liabilities arising from obtaining right-of-use assets: ​ ​ ​ ​ ​ Operating leases $ - ​ $ 497 ​ ​ 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 ​ ​ ​ ​ 8.0 Weighted average discount rate ​ ​ ​ ​ 8.2% ​ |
Commitments
Commitments | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Commitments | 15. Commitments Manufacturing Agreements In connection with the commercialization of AYVAKIT/AYVAKYT and GAVRETO, the Company has negotiated manufacturing agreements with certain vendors that require the Company to meet minimum purchase obligations on an annual basis. During the three months ended June 30, 2021, there were no material changes to the Company’s contractual obligations . |
Subsequent Events
Subsequent Events | 6 Months Ended |
Jun. 30, 2021 | |
Disclosure Text Block | |
Subsequent Events | 16. Subsequent Event ​ |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Policies) | 6 Months Ended |
Jun. 30, 2021 | |
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, 2020 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020, filed with the SEC on February 17, 2021 (the 2020 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, 2021, the results of its operations for the three and six months ended June 30, 2021 and 2020, stockholder’s equity for the three and six months ended June 30, 2021 and 2020 and cash flows for six months ended June 30, 2021 and 2020. Such adjustments are of a normal and recurring nature. The results for the three and six months ended June 30, 2021 are not necessarily indicative of the results for the year ending December 31, 2021 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, and income taxes. The length of time and full extent to which the ongoing COVID-19 pandemic will directly or indirectly impact the Company’s business, results of operations and financial condition, including revenues, expenses, reserves and allowances, manufacturing, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain, subject to change and difficult to predict, including as a result of new information that may emerge concerning COVID-19, including the identification and spread of new variants, and the actions taken to contain or treat COVID-19, as well as the economic impact thereof on local, regional, national and international customers and markets. The Company considers the impact of COVID-19 while making the estimates within its consolidated financial statements and there may be changes to those estimates in future periods. Actual results may differ from these estimates. |
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, 2021 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2020 Annual Report on Form 10-K. |
Reclassification | Reclassification ​ Certain items in the prior year’s condensed consolidated financial statements have been reclassified to conform to the current presentation. |
New Accounting Pronouncements | New Accounting Pronouncements |
Marketable Securities (Tables)
Marketable Securities (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Table Text Blocks | |
Schedule of marketable securities | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair June 30, 2021 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 652,235 ​ $ 176 ​ $ (300) ​ $ 652,111 U.S. treasury obligations ​ ​ 193,563 ​ ​ 140 ​ ​ (80) ​ ​ 193,623 Total ​ $ 845,798 ​ $ 316 ​ $ (380) ​ $ 845,734 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Amortized ​ Unrealized ​ Unrealized ​ Fair December 31, 2020 Cost Gain ​ Losses ​ Value Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ $ 746,770 ​ $ 513 ​ $ (14) ​ $ 747,269 U.S. treasury obligations ​ ​ 117,368 ​ ​ 449 ​ ​ — ​ ​ 117,817 Total ​ $ 864,138 ​ $ 962 ​ $ (14) ​ $ 865,086 ​ |
Fair Value of Financial Instr_2
Fair Value of Financial Instruments (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Table Text Blocks | |
Schedule of financial instruments measured at fair value | The following table summarizes cash equivalents and marketable securities measured at fair value on a recurring basis as of June 30, 2021 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ June 30, ​ Markets ​ Inputs ​ Inputs Description ​ 2021 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 505,142 ​ $ 505,142 ​ $ — ​ $ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 652,111 ​ ​ — ​ ​ 652,111 ​ ​ — U.S. treasury obligations ​ ​ 193,623 ​ ​ 193,623 ​ ​ — ​ ​ — Total ​ $ 1,350,876 ​ $ 698,765 ​ $ 652,111 ​ $ — ​ The following table summarizes cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2020 (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Active Observable Unobservable ​ ​ December 31, ​ Markets ​ Inputs ​ Inputs Description ​ 2020 ​ (Level 1) ​ (Level 2) ​ (Level 3) Cash equivalents: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 420,567 ​ $ 420,567 ​ $ — ​ $ — Marketable securities, available-for-sale: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ U.S. government agency securities ​ ​ 747,269 ​ ​ — ​ ​ 747,269 ​ ​ — U.S. treasury obligations ​ ​ 117,817 ​ ​ 117,817 ​ ​ — ​ ​ — Total ​ $ 1,285,653 ​ $ 538,384 ​ $ 747,269 ​ $ — |
Product Revenue Reserves and _2
Product Revenue Reserves and Allowances (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Product revenue, net | |
Table Text Blocks | |
Schedule of product revenue allowance and reserve categories | ​ ​ ​ ​ ​ Amount Beginning balance at January 1, 2021 $ 1,192 Provision related to product sales 3,199 Credits and payments made (1,837) Ending balance at June 30, 2021 $ 2,554 ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2021 ​ 2020 Reduction of accounts receivable, net $ 391 ​ $ 227 Component of accrued expenses ​ 2,163 ​ ​ 966 Total revenue-related reserves $ 2,554 ​ $ 1,192 ​ |
Inventory (Tables)
Inventory (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Table Text Blocks | |
Schedule of capitalized inventory | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2021 2020 Raw materials ​ ​ 3,507 ​ ​ — Work in process ​ ​ 13,475 ​ ​ 9,488 Finished goods ​ 1,868 ​ 914 Total ​ $ 18,850 ​ $ 10,402 ​ Balance sheet classification ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2021 2020 Inventory ​ $ 15,211 ​ $ 8,581 Other assets ​ 3,639 ​ 1,821 Total ​ $ 18,850 ​ $ 10,402 ​ |
Property and Equipment, Net (Ta
Property and Equipment, Net (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Table Text Blocks | |
Schedule of property and equipment | Property and equipment and related accumulated depreciation are as follows (in thousands, except year data): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Estimated ​ ​ ​ ​ ​ ​ ​ ​ Useful Life ​ June 30, ​ December 31, ​ ​ (Years) ​ 2021 ​ 2020 Lab equipment 5 $ 12,246 $ 11,418 Furniture and fixtures 4 ​ 3,417 ​ 3,420 Computer equipment 3 ​ 1,555 ​ 1,513 Leasehold improvements Term of lease ​ 36,945 ​ 36,946 Software 3 ​ 412 ​ 412 Construction-in-progress ​ ​ ​ ​ 162 ​ ​ 151 Total cost ​ ​ ​ 54,737 ​ 53,860 Less: accumulated depreciation and amortization ​ ​ ​ (22,889) ​ (19,731) Total ​ ​ ​ $ 31,848 ​ $ 34,129 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Table Text Blocks | |
Schedule of accrued expenses | Accrued expenses consist of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2021 2020 Research, development and commercial contract costs ​ $ 68,793 ​ $ 60,255 ​ Employee compensation ​ ​ 16,667 ​ ​ 27,622 ​ Accrued professional fees ​ 14,064 ​ 10,986 ​ Revenue-related reserves ​ ​ 2,163 ​ ​ 966 ​ Other ​ ​ 5,486 ​ ​ 6,109 ​ Total ​ $ 107,173 ​ $ 105,938 ​ |
Collaboration and License Agr_2
Collaboration and License Agreements (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Table Text Blocks | |
Summary of manufacturing and research and development services related to the global development activities | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 Manufacturing and research and development services related to global development activities, net of expenses payable to CStone ​ $ 269 $ 920 $ 1,008 $ 2,450 ​ |
Roche, Collaboration (Pralsetnib) Agreement | |
Table Text Blocks | |
Summary of revenue recognized | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 Manufacturing and other services related to Roche territory-specific activities ​ $ 1,912 ​ $ — ​ $ 3,684 ​ $ — Total Roche pralsetinib collaboration revenue ​ $ 1,912 $ — $ 3,684 $ — ​ |
Roche, Collaboration (Pralsetnib) Agreement | Collaboration revenue | |
Table Text Blocks | |
Summary of contract assets and/or contract liabilities | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2021 ​ 2020 Accounts receivable, net ​ $ 11,865 $ — Unbilled accounts receivable ​ $ 7,224 $ 17,600 ​ ​ |
Roche, Collaboration and License (Immunotherapy) Agreement | |
Table Text Blocks | |
Summary of revenue recognized | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 Roche immunotherapy collaboration research and development services revenue ​ $ 1,349 $ 1,109 $ 2,885 $ 1,684 ​ |
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, ​ ​ 2021 2020 ​ 2021 2020 Amounts included in the contract liability at the beginning of the period ​ $ 1,064 $ 1,061 $ 2,302 $ 2,050 ​ |
C Stone | |
Table Text Blocks | |
Summary of revenue recognized | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 License milestone revenue ​ $ — $ — $ 9,000 $ 2,000 Manufacturing services related to CStone territory-specific activities ​ ​ 10,355 ​ ​ 1,263 ​ ​ 10,538 ​ ​ 1,397 Royalty revenue ​ ​ 1,947 ​ ​ — ​ ​ 2,035 ​ ​ — Total CStone collaboration revenue ​ $ 12,302 ​ $ 1,263 ​ $ 21,573 ​ $ 3,397 ​ |
C Stone | Collaboration revenue | |
Table Text Blocks | |
Summary of contract assets and/or contract liabilities | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ ​ December 31, ​ ​ 2021 ​ 2020 Accounts receivable, net ​ $ 5,486 $ 563 Unbilled accounts receivable ​ $ 6,512 $ — |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Table Text Blocks | |
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, ​ ​ 2021 ​ 2020 ​ ​ 2021 ​ 2020 Stock options $ 14,965 ​ $ 14,629 ​ $ 29,107 ​ $ 28,344 Restricted stock units ​ 9,270 ​ ​ 4,844 ​ ​ 16,064 ​ ​ 7,998 Employee stock purchase plan ​ 287 ​ ​ 202 ​ ​ 563 ​ ​ 359 Subtotal ​ 24,522 ​ ​ 19,675 ​ ​ 45,734 ​ ​ 36,701 Capitalized stock-based compensation costs ​ (215) ​ ​ (147) ​ ​ (743) ​ ​ (314) Stock-based compensation expense included in total cost and operating expenses $ 24,307 ​ $ 19,528 ​ $ 44,991 ​ $ 36,387 Stock-based compensation expense by classification within the unaudited condensed consolidated statements of operations and comprehensive loss is as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ ​ 2021 2020 ​ 2021 2020 Research and development ​ $ 10,485 $ 8,709 $ 19,432 $ 16,507 Selling, general and administrative ​ 13,822 ​ 10,819 ​ 25,559 ​ 19,880 Total ​ $ 24,307 ​ $ 19,528 ​ ​ 44,991 ​ ​ 36,387 |
Summary of stock option activity | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ ​ Shares ​ ​ Exercise Price Outstanding at December 31, 2020 6,030,641 ​ $ 61.28 Granted 908,661 ​ ​ 98.95 Exercised (468,534) ​ ​ 42.73 Canceled (271,982) ​ ​ 77.49 Outstanding at June 30, 2021 6,198,786 ​ $ 67.49 Exercisable at June 30, 2021 3,428,017 ​ $ 57.20 |
Summary of restricted stock units activity | ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-Average ​ Shares Grant Date Fair Value Unvested shares at December 31, 2020 1,171,686 ​ $ 65.37 Granted 778,521 ​ 99.41 Vested (270,168) ​ 63.67 Forfeited (123,644) ​ 73.44 Unvested shares at June 30, 2021 1,556,395 ​ $ 82.05 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
Table Text Blocks | |
Schedule of common stock equivalents excluded from calculation of diluted net loss per share applicable to common stockholders | ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ ​ June 30, ​ 2021 2020 Stock options 6,199 ​ 6,661 Restricted stock units 1,556 ​ 1,151 ESPP shares ​ 25 ​ 26 Total 7,780 7,838 ​ |
Leases (Tables)
Leases (Tables) | 6 Months Ended |
Jun. 30, 2021 | |
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: 2021 ​ 2020 ​ 2021 ​ 2020 Lease cost $ 4,451 ​ $ 4,328 ​ $ 9,177 ​ $ 8,709 Sublease income ​ (268) ​ ​ (730) ​ ​ (595) ​ ​ (1,451) Net lease cost $ 4,183 ​ $ 3,598 ​ $ 8,582 ​ $ 7,258 ​ ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ June 30, ​ 2021 ​ 2020 Cash paid for amounts included in the measurement of lease liabilities: $ 7,394 ​ $ 7,172 Lease liabilities arising from obtaining right-of-use assets: ​ ​ ​ ​ ​ Operating leases $ - ​ $ 497 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Operating leases Weighted average remaining lease term in years ​ ​ ​ ​ 8.0 Weighted average discount rate ​ ​ ​ ​ 8.2% |
Nature of Business (Details)
Nature of Business (Details) $ in Millions | Jun. 30, 2021USD ($) |
Cash, cash equivalents and marketable securities | |
Cash, cash equivalents and marketable securities | $ 1,380.1 |
Marketable Securities - Tabular
Marketable Securities - Tabular Disclosure (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Marketable securities, available-for-sale: | ||
Amortized cost | $ 845,798 | $ 864,138 |
Unrealized gain | 316 | 962 |
Unrealized losses | (380) | (14) |
Fair value | 845,734 | 865,086 |
U.S. government agency securities | ||
Marketable securities, available-for-sale: | ||
Amortized cost | 652,235 | 746,770 |
Unrealized gain | 176 | 513 |
Unrealized losses | (300) | (14) |
Fair value | 652,111 | 747,269 |
U.S. Treasury obligations | ||
Marketable securities, available-for-sale: | ||
Amortized cost | 193,563 | 117,368 |
Unrealized gain | 140 | 449 |
Unrealized losses | (80) | |
Fair value | $ 193,623 | $ 117,817 |
Marketable Securities - Unreali
Marketable Securities - Unrealized Loss Position (Details) $ in Millions | Jun. 30, 2021USD ($)security | Dec. 31, 2020USD ($)security |
Unrealized loss position, number of positions | ||
Number of held securities in an unrealized loss position | 35 | 8 |
Number of held securities in an unrealized loss position for 12 months or longer | 0 | 0 |
Unrealized loss position, aggregate fair value | ||
Unrealized loss position | $ | $ 422.8 | $ 125.7 |
Marketable Securities - Other-t
Marketable Securities - Other-than-temporary Impairment (Details) $ in Thousands | 3 Months Ended |
Jun. 30, 2021USD ($) | |
Other-than-temporary impairment loss, debt securities, available-for-sale securities | |
Other-than-temporary impairment loss, debt securities, available-for-sale | $ 0 |
Marketable Securities - Remaini
Marketable Securities - Remaining Maturities (Details) $ in Millions | Jun. 30, 2021USD ($)security | Dec. 31, 2020USD ($)security |
Investments, available-for-sale | ||
Securities with remaining maturities greater than one year | security | 69 | 65 |
Aggregate fair value with remaining maturities greater than one year | $ | $ 751.7 | $ 677.9 |
Marketable Securities - Proceed
Marketable Securities - Proceeds from Maturities (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Proceeds from the maturities of debt securities | ||||
Proceeds from the maturities of debt securities | $ 185,100 | $ 105,400 | $ 400,500 | $ 306,035 |
Marketable Securities - Realize
Marketable Securities - Realized Gains (Losses) (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Realized gains (losses) from maturities of debt securities | ||||
Realized gains (losses) from maturities of debt securities | $ 0 | $ 0 | $ 0 | $ 0 |
Fair Value of Financial Instr_3
Fair Value of Financial Instruments (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | $ 845,734 | $ 865,086 |
U.S. government agency securities | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 652,111 | 747,269 |
U.S. Treasury obligations | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 193,623 | 117,817 |
Recurring | ||
Fair Value of Financial Instruments | ||
Total | 1,350,876 | 1,285,653 |
Recurring | U.S. government agency securities | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 652,111 | 747,269 |
Recurring | U.S. Treasury obligations | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 193,623 | 117,817 |
Recurring | Money market funds | ||
Fair Value of Financial Instruments | ||
Cash equivalents | 505,142 | 420,567 |
Recurring | Active Markets (Level 1) | ||
Fair Value of Financial Instruments | ||
Total | 698,765 | 538,384 |
Recurring | Active Markets (Level 1) | U.S. Treasury obligations | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 193,623 | 117,817 |
Recurring | Active Markets (Level 1) | Money market funds | ||
Fair Value of Financial Instruments | ||
Cash equivalents | 505,142 | 420,567 |
Recurring | Observable Inputs (Level 2) | ||
Fair Value of Financial Instruments | ||
Total | 652,111 | 747,269 |
Recurring | Observable Inputs (Level 2) | U.S. government agency securities | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | $ 652,111 | $ 747,269 |
Product Revenue Reserves and _3
Product Revenue Reserves and Allowances - Product Revenue (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Revenues | ||||
Revenue | $ 27,295 | $ 8,343 | $ 48,871 | $ 14,510 |
Product revenue, net | ||||
Revenues | ||||
Revenue | $ 11,433 | $ 5,680 | $ 20,388 | $ 9,138 |
Product Revenue Reserves and _4
Product Revenue Reserves and Allowances - Product Revenue Allowance and Reserve (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2021USD ($) | |
Product revenue allowance and reserve | |
Beginning balance | $ 1,192 |
Provision related to sales in the current period | 3,199 |
Credits and payments made | (1,837) |
Ending balance | $ 2,554 |
Product Revenue Reserves and _5
Product Revenue Reserves and Allowances - Revenue-related Reserves (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Revenue-related reserves | ||
Revenue-related reserves | $ 2,554 | $ 1,192 |
Accounts Receivable, Net | ||
Revenue-related reserves | ||
Revenue-related reserves | 391 | 227 |
Accrued Expenses | ||
Revenue-related reserves | ||
Revenue-related reserves | $ 2,163 | $ 966 |
Inventory - Capitalized (Detail
Inventory - Capitalized (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Inventory, Noncurrent [Abstract] | ||
Raw materials | $ 3,507 | |
Work in process | 13,475 | $ 9,488 |
Finished goods | 1,868 | 914 |
Total | $ 18,850 | $ 10,402 |
Inventory - Balance Sheet Class
Inventory - Balance Sheet Classification (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Inventory | ||
Inventory | $ 15,211 | $ 8,581 |
Other assets | 3,639 | 1,821 |
Total | $ 18,850 | $ 10,402 |
Inventory - Write-down (Details
Inventory - Write-down (Details) - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Inventory | ||||
Inventory write-down | $ 0.4 | $ 0 | $ 0.4 | $ 0 |
Restricted Cash (Details)
Restricted Cash (Details) - USD ($) $ in Millions | Jun. 30, 2021 | Dec. 31, 2020 |
Restricted cash | ||
Restricted cash | $ 5.2 | $ 5.2 |
Property and Equipment, Net - E
Property and Equipment, Net - Estimated Useful Lives (Details) | 6 Months Ended |
Jun. 30, 2021 | |
Lab equipment | |
Property and Equipment, Net | |
Estimated Useful Life (Years) | 5 years |
Furniture and fixtures | |
Property and Equipment, Net | |
Estimated Useful Life (Years) | 4 years |
Computer equipment | |
Property and Equipment, Net | |
Estimated Useful Life (Years) | 3 years |
Leasehold improvements | |
Property and Equipment, Net | |
Estimated Useful Life | Term of lease |
Software | |
Property and Equipment, Net | |
Estimated Useful Life (Years) | 3 years |
Property and Equipment, Net - P
Property and Equipment, Net - Property and Equipment and Related Accumulated Depreciation (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Property and Equipment, Net | ||
Total cost | $ 54,737 | $ 53,860 |
Less: accumulated depreciation and amortization | (22,889) | (19,731) |
Total | 31,848 | 34,129 |
Lab equipment | ||
Property and Equipment, Net | ||
Total cost | 12,246 | 11,418 |
Furniture and fixtures | ||
Property and Equipment, Net | ||
Total cost | 3,417 | 3,420 |
Computer equipment | ||
Property and Equipment, Net | ||
Total cost | 1,555 | 1,513 |
Leasehold improvements | ||
Property and Equipment, Net | ||
Total cost | 36,945 | 36,946 |
Software | ||
Property and Equipment, Net | ||
Total cost | 412 | 412 |
Construction-in-progress | ||
Property and Equipment, Net | ||
Total cost | $ 162 | $ 151 |
Property and Equipment, Net - D
Property and Equipment, Net - Depreciation Expense (Details) - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Depreciation expense | ||||
Depreciation expense | $ 1.6 | $ 1.7 | $ 3.2 | $ 3.2 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Accrued Expenses | ||
Research, development and commercial contract costs | $ 68,793 | $ 60,255 |
Employee compensation | 16,667 | 27,622 |
Accrued professional fees | 14,064 | 10,986 |
Revenue-related reserves | 2,163 | 966 |
Other | 5,486 | 6,109 |
Total | $ 107,173 | $ 105,938 |
Collaboration and License Agr_3
Collaboration and License Agreements - General Information (Details) - Collaborative Arrangement $ in Millions | Oct. 15, 2019USD ($) | Jun. 01, 2018USD ($)item | Jul. 31, 2020USD ($) | Oct. 31, 2019USD ($) | Jun. 30, 2018USD ($) | Mar. 31, 2016USD ($)item | Sep. 30, 2020USD ($) | Dec. 31, 2019item | Jun. 30, 2021USD ($) | Jul. 13, 2020USD ($) |
Roche, Collaboration (Pralsetnib) Agreement | ||||||||||
Collaboration Agreements | ||||||||||
Collaborative arrangement, upfront payment, cash payment received | $ 675 | |||||||||
Collaborative arrangement, milestones, cash payments received | $ 55 | |||||||||
Collaborative arrangement, milestones, contingent payments eligible to receive | $ 872 | |||||||||
Collaborative arrangement, milestones, specified regulatory and commercialization milestones, achieved | 55 | |||||||||
Collaborative arrangement, percentage of global development costs shared, entity (as a percent) | 45.00% | |||||||||
Collaborative arrangement, percentage of global development costs shared, counterparty (as a percent) | 55.00% | |||||||||
Roche, Collaboration and License (Immunotherapy) Agreement | ||||||||||
Collaboration Agreements | ||||||||||
Collaborative arrangement, upfront payment, cash payment received | $ 45 | |||||||||
Collaborative arrangement, milestones, cash payments received | $ 19.5 | |||||||||
Collaborative arrangement, option fees and milestones, contingent payments eligible to receive | $ 323.3 | |||||||||
Collaborative arrangement, milestones, research milestones, achieved | 19.5 | |||||||||
Collaborative arrangement, license option rights, number | item | 5 | |||||||||
Collaborative arrangement, collaboration programs, number | item | 2 | |||||||||
Collaborative arrangement, collaboration programs, licensed products, commercialization rights, counterparty exercises option, counterparty receives worldwide exclusivity, number | item | 2 | |||||||||
Collaborative arrangement, termination, counterparty written notice period | 120 days | |||||||||
Collaborative arrangement, termination, counterparty written notice period, after option exercise, if licensed products have not been commercially sold | 120 days | |||||||||
Collaborative arrangement, termination, counterparty written notice period, after option exercise, if licensed products have been commercially sold | 180 days | |||||||||
Clementia | ||||||||||
Collaboration Agreements | ||||||||||
Collaborative arrangement, upfront payment, cash payment received | $ 25 | |||||||||
Collaborative arrangement, milestones, cash payments received | $ 20 | $ 20 | ||||||||
Collaborative arrangement, milestones, contingent payments eligible to receive | 490 | |||||||||
Collaborative arrangement, inventory purchased by counterparty | $ 1.5 | |||||||||
Collaborative arrangement, termination, counterparty written notice period | 12 months | |||||||||
C Stone | ||||||||||
Collaboration Agreements | ||||||||||
Collaborative arrangement, upfront payment, cash payment received | $ 40 | |||||||||
Collaborative arrangement, milestones, contingent payments eligible to receive | 323 | |||||||||
Collaborative arrangement, milestones, development and regulatory milestones, contingent payments eligible to receive | 95.5 | |||||||||
Collaborative arrangement, milestones, sales-based milestones, contingent payments eligible to receive | $ 227.5 | |||||||||
Collaborative arrangement, milestones, achieved | $ 23 | |||||||||
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 | Jul. 13, 2020 | Oct. 15, 2019 | Jun. 01, 2018 | Mar. 31, 2016 | Jun. 30, 2021 |
Roche, Collaboration (Pralsetnib) Agreement | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 695.7 | ||||
Collaborative arrangement, transaction price, upfront payment | 675 | ||||
Collaborative arrangement, transaction price, stock issued, premium on sale of stock to counterparty | $ 20.7 | ||||
Collaborative arrangement, transaction price, milestone payments receivable | $ 55 | ||||
Roche, Collaboration and License (Immunotherapy) Agreement | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 45 | ||||
Clementia | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 46.5 | ||||
Collaborative arrangement, transaction price, upfront payment | 25 | ||||
Collaborative arrangement, transaction price, milestone payments receivable | 20 | ||||
Collaborative arrangement, transaction price, inventory purchased by counterparty, existing manufacturing inventory | 1.2 | ||||
Collaborative arrangement, transaction price, inventory purchased by counterparty, in-process manufacturing inventory | $ 0.3 | ||||
C Stone | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 40 |
Collaboration and License Agr_5
Collaboration and License Agreements - Sale of Stock (Details) - USD ($) $ / shares in Units, $ in Thousands | Jul. 13, 2020 | Mar. 31, 2020 |
Sale of Stock | ||
Issuance of common stock | $ 308,423 | |
Private Placement | ||
Sale of Stock | ||
Stock issued (in shares) | 1,035,519 | |
Share price (in dollars per share) | $ 96.57 | |
Purchase consideration | $ 100,000 | |
Issuance of common stock | 79,300 | |
Share purchase consideration, premium | $ 20,700 |
Collaboration and License Agr_6
Collaboration and License Agreements - Manufacturing and Research and Development Services (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Collaborative Arrangement | C Stone | ||||
Collaboration Agreements | ||||
Manufacturing and research and development services related to global development activities, net of expenses payable | $ 269 | $ 920 | $ 1,008 | $ 2,450 |
Collaboration and License Agr_7
Collaboration and License Agreements - Revenue Recognized (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | 12 Months Ended | |||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2018 | Jun. 30, 2021 | Jun. 30, 2020 | Dec. 31, 2019 | |
Revenues | ||||||
Revenue | $ 27,295 | $ 8,343 | $ 48,871 | $ 14,510 | ||
Collaboration revenue | ||||||
Revenues | ||||||
Revenue | 15,862 | 2,663 | 28,483 | 5,372 | ||
Collaborative Arrangement | Collaboration revenue | Roche, Collaboration (Pralsetnib) Agreement | ||||||
Revenues | ||||||
Revenue | 1,912 | 3,684 | ||||
Collaborative Arrangement | Collaboration revenue | Roche, Collaboration and License (Immunotherapy) Agreement | ||||||
Revenues | ||||||
Revenue | 1,349 | 1,109 | 2,885 | 1,684 | ||
Collaborative Arrangement | Collaboration revenue | Clementia | ||||||
Revenues | ||||||
Revenue | 0 | 0 | 0 | 0 | $ 46,200 | |
Collaborative Arrangement | Collaboration revenue | C Stone | ||||||
Revenues | ||||||
Revenue | 12,302 | 1,263 | $ 40,000 | 21,573 | 3,397 | |
Collaborative Arrangement | License milestone revenue | C Stone | ||||||
Revenues | ||||||
Revenue | 0 | 0 | 9,000 | 2,000 | ||
Collaborative Arrangement | Territory-specific activities, manufacturing services | C Stone | ||||||
Revenues | ||||||
Revenue | 10,355 | $ 1,263 | 10,538 | $ 1,397 | ||
Collaborative Arrangement | Territory-specific activities, manufacturing and other services | Roche, Collaboration (Pralsetnib) Agreement | ||||||
Revenues | ||||||
Revenue | 1,912 | 3,684 | ||||
Collaborative Arrangement | Royalty | C Stone | ||||||
Revenues | ||||||
Revenue | $ 1,947 | $ 2,035 |
Collaboration and License Agr_8
Collaboration and License Agreements - Reduction in Expenses (Details) - Collaborative Arrangement - Roche, Collaboration (Pralsetnib) Agreement - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended |
Jun. 30, 2021 | Jun. 30, 2021 | |
Selling, General and Administrative Expenses | ||
Collaboration Agreements | ||
Collaborative arrangement, commercialization, reduction in expenses | $ 2.2 | $ 5.2 |
Research and Development Expense | ||
Collaboration Agreements | ||
Collaborative arrangement, commercialization, reduction in expenses | $ 2.1 | $ 6.2 |
Collaboration and License Agr_9
Collaboration and License Agreements - Change in Contract with Customer, Liability (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement | ||||
Change in Contract with Customer, Liability | ||||
Amounts included in the contact liability at the beginning of the period | $ 1,064 | $ 1,061 | $ 2,302 | $ 2,050 |
Collaboration and License Ag_10
Collaboration and License Agreements - Contract Assets (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Collaboration Agreements | ||
Accounts receivable, net | $ 25,473 | $ 7,096 |
Unbilled accounts receivable | 13,767 | 18,213 |
Collaborative Arrangement | Roche, Collaboration (Pralsetnib) Agreement | ||
Collaboration Agreements | ||
Accounts receivable, net | 11,865 | |
Unbilled accounts receivable | 7,224 | 17,600 |
Collaborative Arrangement | C Stone | ||
Collaboration Agreements | ||
Accounts receivable, net | 5,486 | $ 563 |
Unbilled accounts receivable | $ 6,512 |
Collaboration and License Ag_11
Collaboration and License Agreements - Contract Liabilities (Details) - USD ($) $ in Thousands | Jun. 30, 2021 | Dec. 31, 2020 |
Contract Liability | ||
Deferred revenue, current | $ 7,912 | $ 12,559 |
Collaborative Arrangement | Clementia | ||
Contract Liability | ||
Deferred revenue | 0 | 0 |
Collaborative Arrangement | C Stone | ||
Contract Liability | ||
Deferred revenue | 6,200 | $ 6,500 |
Deferred revenue, current | 1,700 | |
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement | ||
Contract Liability | ||
Deferred revenue | 32,000 | |
Deferred revenue, current | $ 6,200 |
Collaboration and License Ag_12
Collaboration and License Agreements - Performance Obligations (Details) - Collaborative Arrangement | 6 Months Ended |
Jun. 30, 2021item | |
Roche, Collaboration (Pralsetnib) Agreement | |
Performance Obligations | |
Collaborative arrangement, material promises, number | 1 |
Collaborative arrangement, material promises, manufacturing activities, term | 24 months |
Roche, Collaboration and License (Immunotherapy) Agreement | Revenue, Remaining Performance Obligation, Expected Timing of Satisfaction, Start Date [Axis]: 2021-07-01 | |
Performance Obligations | |
Revenue, remaining performance obligation, expected timing of satisfaction, period | 3 years 9 months 18 days |
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 | 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 | Jan. 01, 2021 | Jun. 30, 2021 | 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) | 4.00% | ||
Increase in number of shares available for grant (in shares) | 2,311,741 | ||
Number of shares available for grant (in shares) | 2,913,775 |
Stock-based Compensation - 2020
Stock-based Compensation - 2020 Inducement Plan (Details) - 2020 Inducement Plan - shares | Jun. 30, 2021 | Mar. 31, 2020 |
Stock-based compensation | ||
Initial shares of common stock authorized for issuance of stock awards (in shares) | 1,000,000 | |
Number of shares available for grant (in shares) | 485,774 |
Stock-based Compensation - Stoc
Stock-based Compensation - Stock Options (Details) | 6 Months Ended |
Jun. 30, 2021$ / sharesshares | |
Shares | |
Outstanding at beginning of period (in shares) | shares | 6,030,641 |
Granted (in shares) | shares | 908,661 |
Exercised (in shares) | shares | (468,534) |
Cancelled (in shares) | shares | (271,982) |
Outstanding at end of period (in shares) | shares | 6,198,786 |
Weighted-Average Exercise Price | |
Outstanding at beginning of period (in dollars per share) | $ / shares | $ 61.28 |
Granted (in dollars per share) | $ / shares | 98.95 |
Exercised (in dollars per share) | $ / shares | 42.73 |
Cancelled (in dollars per share) | $ / shares | 77.49 |
Outstanding at end of period (in dollars per share) | $ / shares | $ 67.49 |
Additional disclosures | |
Shares - Exercisable (in shares) | shares | 3,428,017 |
Weighted-Average Exercise Price - Exercisable (in dollars per share) | $ / shares | $ 57.20 |
Stock-based Compensation - Unre
Stock-based Compensation - Unrecognized Compensation Costs (Details) $ in Millions | Jun. 30, 2021USD ($) |
Stock-based compensation | |
Total unrecognized compensation cost related to non-vested stock option awards | $ 120.2 |
Weighted-average period over which unrecognized compensation cost will be recognized | 2 years 7 months 9 days |
Restricted Stock Units | |
Stock-based compensation | |
Total unrecognized compensation cost related to non-vested stock awards | $ 113.7 |
Weighted-average period over which unrecognized compensation cost will be recognized | 3 years 25 days |
Stock-based Compensation - Rest
Stock-based Compensation - Restricted Stock Units (Details) - Restricted Stock Units | 6 Months Ended |
Jun. 30, 2021$ / sharesshares | |
Shares | |
Unvested at beginning of period (in shares) | shares | 1,171,686 |
Granted (in shares) | shares | 778,521 |
Vested (in shares) | shares | (270,168) |
Forfeited (in shares) | shares | (123,644) |
Unvested at end of period (in shares) | shares | 1,556,395 |
Weighted-Average Grant Date Fair Value | |
Unvested at beginning or period (in dollars per share) | $ / shares | $ 65.37 |
Granted (in dollars per share) | $ / shares | 99.41 |
Vested (in dollars per share) | $ / shares | 63.67 |
Forfeited (in dollars per share) | $ / shares | 73.44 |
Unvested at end of period (in dollars per share) | $ / shares | $ 82.05 |
Stock-based Compensation - Empl
Stock-based Compensation - Employee Stock Purchase Plan (Details) - Employee Stock - shares | 1 Months Ended | |
May 31, 2015 | Jan. 01, 2021 | |
Stock-based compensation | ||
Number of common shares reserved for future issuance (in shares) | 243,347 | |
Annual increase for common stock for issuance (as a percent) | 1.00% | |
Increase of common shares reserved for future issuance (in shares) | 577,935 |
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, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Total stock based compensation expense | ||||
Stock-based compensation expense | $ 24,307 | $ 19,528 | $ 44,991 | $ 36,387 |
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, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Total stock based compensation expense | ||||
Subtotal | $ 24,522 | $ 19,675 | $ 45,734 | $ 36,701 |
Capitalized stock-based compensation costs | (215) | (147) | (743) | (314) |
Stock-based compensation expense included in total cost and operating expenses | 24,307 | 19,528 | 44,991 | 36,387 |
Stock Options | ||||
Total stock based compensation expense | ||||
Subtotal | 14,965 | 14,629 | 29,107 | 28,344 |
Restricted Stock Units | ||||
Total stock based compensation expense | ||||
Subtotal | 9,270 | 4,844 | 16,064 | 7,998 |
Employee Stock | ||||
Total stock based compensation expense | ||||
Subtotal | $ 287 | $ 202 | $ 563 | $ 359 |
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, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Total stock based compensation expense | ||||
Stock-based compensation expense | $ 24,307 | $ 19,528 | $ 44,991 | $ 36,387 |
Research and Development | ||||
Total stock based compensation expense | ||||
Stock-based compensation expense | 10,485 | 8,709 | 19,432 | 16,507 |
General and Administrative Expense | ||||
Total stock based compensation expense | ||||
Stock-based compensation expense | $ 13,822 | $ 10,819 | $ 25,559 | $ 19,880 |
Net Income Per Share (Details)
Net Income Per Share (Details) - shares shares in Thousands | 6 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 7,780 | 7,838 |
Stock Options | ||
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 6,199 | 6,661 |
Restricted Stock Units | ||
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 1,556 | 1,151 |
Employee Stock | ||
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 25 | 26 |
Leases - Remaining Lease Terms
Leases - Remaining Lease Terms (Details) | Jun. 30, 2021 |
Minimum | |
Leases | |
Operating lease remaining lease term | 1 year |
Maximum | |
Leases | |
Operating lease remaining lease term | 8 years |
Leases - Components of Lease Ex
Leases - Components of Lease Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2021 | Jun. 30, 2020 | Jun. 30, 2021 | Jun. 30, 2020 | |
Operating leases cost: | ||||
Lease cost | $ 4,451 | $ 4,328 | $ 9,177 | $ 8,709 |
Sublease income | (268) | (730) | (595) | (1,451) |
Net lease cost | $ 4,183 | $ 3,598 | $ 8,582 | $ 7,258 |
Leases - Cash Flow Information
Leases - Cash Flow Information (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2021 | Jun. 30, 2020 | |
Leases | ||
Cash paid for amounts included in the measurement of lease liabilities, operating cash flows from operating leases | $ 7,394 | $ 7,172 |
Lease liabilities arising from obtaining right-of-use assets, operating leases | $ 497 |
Leases - Weighted Average Remai
Leases - Weighted Average Remaining Lease-term and Weighted Average Discount Rate (Details) | Jun. 30, 2021 |
Leases | |
Weighted average remaining lease term in years | 8 years |
Weighted average discount rate | 8.20% |
Subsequent Events (Details)
Subsequent Events (Details) | 1 Months Ended |
Jul. 29, 2021 | |
Collaborative Arrangement | Subsequent Event | University of Texas MD Anderson Cancer Center | |
Subsequent Events | |
Collaborative arrangement, strategic research collaboration, term | 3 years |