Document and Entity Information
Document and Entity Information - $ / shares | 9 Months Ended | |
Sep. 30, 2023 | Oct. 23, 2023 | |
Cover [Abstract] | ||
Entity Central Index Key | 0001597264 | |
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Sep. 30, 2023 | |
Document Transition Report | false | |
Entity File Number | 001-37359 | |
Entity Registrant Name | BLUEPRINT MEDICINES CORPORATION | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 26-3632015 | |
Entity Address, Address Line One | 45 Sidney Street | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02139 | |
City Area Code | 617 | |
Local Phone Number | 374-7580 | |
Title of 12(b) Security | Common stock, par value $0.001 per share | |
Trading Symbol | BPMC | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Listing, Par Value Per Share | $ 0.001 | |
Entity Common Stock, Shares Outstanding | 60,789,712 | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q3 | |
Amendment Flag | false |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 55,972 | $ 119,709 |
Marketable securities | 657,012 | 825,283 |
Accounts receivable | 40,861 | 23,525 |
Unbilled accounts receivable | 393 | 13,413 |
Inventory | 16,457 | 29,697 |
Prepaid expenses and other current assets | 37,477 | 35,024 |
Total current assets | 808,172 | 1,046,651 |
Marketable securities | 114,242 | 133,480 |
Property and equipment, net | 40,619 | 33,344 |
Operating lease right-of-use assets, net | 75,221 | 81,854 |
Restricted cash | 9,696 | 5,195 |
Equity investment | 27,789 | 27,789 |
Other assets | 29,560 | 21,589 |
Total assets | 1,105,299 | 1,349,902 |
Current liabilities: | ||
Accounts payable | 6,963 | 2,729 |
Accrued expenses | 110,436 | 131,123 |
Current portion of operating lease liabilities | 11,617 | 10,579 |
Current portion of deferred revenue | 3,010 | 4,667 |
Current portion of liabilities related to the sale of future royalties and revenues | 35,137 | 17,285 |
Current portion of term loan | 30,221 | 16,851 |
Total current liabilities | 197,384 | 183,234 |
Operating lease liabilities, net of current portion | 84,036 | 92,789 |
Deferred revenue, net of current portion | 5,000 | 13,624 |
Liabilities related to the sale of future royalties and revenues, net of current portion | 405,010 | 413,045 |
Term loan, net of current portion | 208,157 | 122,232 |
Other long-term liabilities | 3,101 | 10,301 |
Total liabilities | 902,688 | 835,225 |
Commitments and Contingencies (Note 15) | ||
Stockholders' equity: | ||
Preferred stock, $0.001 par value; 5,000,000 shares authorized; no shares issued and outstanding | ||
Common stock, $0.001 par value; 120,000,000 shares authorized; 60,740,744 and 59,958,919 shares issued and outstanding at September 30, 2023 and December 31, 2022, respectively | 61 | 60 |
Additional paid-in capital | 2,434,258 | 2,358,018 |
Accumulated other comprehensive loss | (2,683) | (10,443) |
Accumulated deficit | (2,229,025) | (1,832,958) |
Total stockholders' equity | 202,611 | 514,677 |
Total liabilities and stockholders' equity | $ 1,105,299 | $ 1,349,902 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2023 | Dec. 31, 2022 |
Preferred Stock Disclosures | ||
Preferred stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common Stock Disclosures | ||
Common stock, par value (in dollars per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 120,000,000 | 120,000,000 |
Common stock, shares issued (in shares) | 60,740,744 | 59,958,919 |
Common Stock, shares outstanding (in shares) | 60,740,744 | 59,958,919 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations and Comprehensive Loss - USD ($) shares in Thousands, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Revenues: | ||||
Revenue | $ 56,566 | $ 65,977 | $ 177,423 | $ 165,255 |
Cost and operating expenses: | ||||
Cost of sales | 2,782 | 3,000 | 8,280 | 12,965 |
Research and development | 110,252 | 127,981 | 330,184 | 359,579 |
Selling, general and administrative | 70,741 | 57,608 | 215,826 | 173,354 |
Collaboration loss sharing | 1,771 | 1,665 | 4,301 | 7,076 |
Total cost and operating expenses | 185,546 | 190,254 | 558,591 | 552,974 |
Other income (expense): | ||||
Interest expense, net | (3,808) | (8,396) | (13,624) | (7,527) |
Other income (expense), net | (728) | 396 | (369) | 575 |
Total other expense, net | (4,536) | (8,000) | (13,993) | (6,952) |
Loss before income taxes | (133,516) | (132,277) | (395,161) | (394,671) |
Income tax expense | 197 | 886 | 907 | 4,200 |
Net loss | (133,713) | (133,163) | (396,068) | (398,871) |
Other comprehensive loss: | ||||
Unrealized gains (losses) on available-for-sale investments | 1,443 | (843) | 7,624 | (11,171) |
Currency translation adjustments | 95 | 265 | 136 | 478 |
Comprehensive loss | $ (132,175) | $ (133,741) | $ (388,308) | $ (409,564) |
Net loss per share - basic (in dollars per share) | $ (2.20) | $ (2.23) | ||
Net loss per share - diluted (in dollars per share) | $ (2.20) | $ (2.23) | $ (6.55) | $ (6.70) |
Weighted-average number of common shares used in net loss per share - basic (in shares) | 60,688 | 59,758 | ||
Weighted-average number of common shares used in net loss per share - diluted (in shares) | 60,688 | 59,758 | 60,445 | 59,564 |
Product revenue, net | ||||
Revenues: | ||||
Revenue | $ 54,228 | $ 28,634 | $ 133,173 | $ 80,929 |
Product revenue, net | Nonrelated Party | ||||
Revenues: | ||||
Revenue | 54,228 | 28,634 | 133,173 | 80,929 |
Collaboration and License | Nonrelated Party | ||||
Revenues: | ||||
Revenue | $ 2,338 | 9,843 | $ 44,250 | 56,826 |
License | Related Party | ||||
Revenues: | ||||
Revenue | $ 27,500 | $ 27,500 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Common Stock | Additional Paid-In Capital | Accumulated Other Comprehensive Loss | Accumulated Deficit | Total |
Beginning Balance at Dec. 31, 2021 | $ 59 | $ 2,250,250 | $ (4,133) | $ (1,275,441) | $ 970,735 |
Beginning Balance (in shares) at Dec. 31, 2021 | 59,141,086 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan | $ 1 | 1,297 | 1,298 | ||
Issuance of common stock under stock plan (in shares) | 414,888 | ||||
Stock-based compensation expense | 23,609 | 23,609 | |||
Other comprehensive income (loss) | (7,977) | (7,977) | |||
Net loss | (105,999) | (105,999) | |||
Ending Balance at Mar. 31, 2022 | $ 60 | 2,275,156 | (12,110) | (1,381,440) | 881,666 |
Ending Balance (in shares) at Mar. 31, 2022 | 59,555,974 | ||||
Beginning Balance at Dec. 31, 2021 | $ 59 | 2,250,250 | (4,133) | (1,275,441) | 970,735 |
Beginning Balance (in shares) at Dec. 31, 2021 | 59,141,086 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Net loss | (398,871) | ||||
Ending Balance at Sep. 30, 2022 | $ 60 | 2,329,385 | (14,826) | (1,674,312) | 640,307 |
Ending Balance (in shares) at Sep. 30, 2022 | 59,808,492 | ||||
Beginning Balance at Mar. 31, 2022 | $ 60 | 2,275,156 | (12,110) | (1,381,440) | 881,666 |
Beginning Balance (in shares) at Mar. 31, 2022 | 59,555,974 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan and stock purchase plan | 2,407 | 2,407 | |||
Issuance of common stock under stock plan and stock purchase plan (in shares) | 132,321 | ||||
Stock-based compensation expense | 25,524 | 25,524 | |||
Other comprehensive income (loss) | (2,138) | (2,138) | |||
Net loss | (159,709) | (159,709) | |||
Ending Balance at Jun. 30, 2022 | $ 60 | 2,303,087 | (14,248) | (1,541,149) | 747,750 |
Ending Balance (in shares) at Jun. 30, 2022 | 59,688,295 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan | 2,030 | 2,030 | |||
Issuance of common stock under stock plan (in shares) | 120,197 | ||||
Stock-based compensation expense | 24,268 | 24,268 | |||
Other comprehensive income (loss) | (578) | (578) | |||
Net loss | (133,163) | (133,163) | |||
Ending Balance at Sep. 30, 2022 | $ 60 | 2,329,385 | (14,826) | (1,674,312) | 640,307 |
Ending Balance (in shares) at Sep. 30, 2022 | 59,808,492 | ||||
Beginning Balance at Dec. 31, 2022 | $ 60 | 2,358,018 | (10,443) | (1,832,958) | $ 514,677 |
Beginning Balance (in shares) at Dec. 31, 2022 | 59,958,919 | 59,958,919 | |||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan (in shares) | 457,416 | ||||
Stock-based compensation expense | 23,340 | $ 23,340 | |||
Other comprehensive income (loss) | 5,841 | 5,841 | |||
Net loss | (129,560) | (129,560) | |||
Ending Balance at Mar. 31, 2023 | $ 60 | 2,381,358 | (4,602) | (1,962,518) | 414,298 |
Ending Balance (in shares) at Mar. 31, 2023 | 60,416,335 | ||||
Beginning Balance at Dec. 31, 2022 | $ 60 | 2,358,018 | (10,443) | (1,832,958) | $ 514,677 |
Beginning Balance (in shares) at Dec. 31, 2022 | 59,958,919 | 59,958,919 | |||
Increase (Decrease) in Stockholders' Equity | |||||
Net loss | $ (396,068) | ||||
Ending Balance at Sep. 30, 2023 | $ 61 | 2,434,258 | (2,683) | (2,229,025) | $ 202,611 |
Ending Balance (in shares) at Sep. 30, 2023 | 60,740,744 | 60,740,744 | |||
Beginning Balance at Mar. 31, 2023 | $ 60 | 2,381,358 | (4,602) | (1,962,518) | $ 414,298 |
Beginning Balance (in shares) at Mar. 31, 2023 | 60,416,335 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan and stock purchase plan | $ 1 | 5,298 | 5,299 | ||
Issuance of common stock under stock plan and stock purchase plan (in shares) | 221,940 | ||||
Stock-based compensation expense | 23,945 | 23,945 | |||
Other comprehensive income (loss) | 381 | 381 | |||
Net loss | (132,794) | (132,794) | |||
Ending Balance at Jun. 30, 2023 | $ 61 | 2,410,601 | (4,221) | (2,095,312) | 311,129 |
Ending Balance (in shares) at Jun. 30, 2023 | 60,638,275 | ||||
Increase (Decrease) in Stockholders' Equity | |||||
Issuance of common stock under stock plan | 445 | 445 | |||
Issuance of common stock under stock plan (in shares) | 102,469 | ||||
Stock-based compensation expense | 23,212 | 23,212 | |||
Other comprehensive income (loss) | 1,538 | 1,538 | |||
Net loss | (133,713) | (133,713) | |||
Ending Balance at Sep. 30, 2023 | $ 61 | $ 2,434,258 | $ (2,683) | $ (2,229,025) | $ 202,611 |
Ending Balance (in shares) at Sep. 30, 2023 | 60,740,744 | 60,740,744 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Cash flows from operating activities | ||
Net loss | $ (396,068) | $ (398,871) |
Adjustments to reconcile net income (loss) to net cash provided by (used in) operating activities: | ||
Depreciation and amortization | 10,134 | 4,719 |
Non-cash lease expense | 6,759 | 6,212 |
Stock-based compensation | 70,087 | 72,855 |
Non-cash interest expense | 11,886 | 8,389 |
Non-cash customer consideration | (27,500) | |
Net (accretion of discount) amortization of premium on marketable securities | (11,527) | 844 |
Other | 2,342 | (57) |
Changes in assets and liabilities: | ||
Accounts receivable | (18,970) | 5,553 |
Unbilled accounts receivable | 13,019 | 9,282 |
Inventory | (5,195) | (20,653) |
Prepaid expenses and other current assets | (2,449) | (15,575) |
Other assets | (1,724) | (11,886) |
Accounts payable | 4,244 | (2,031) |
Accrued expenses | (15,100) | 7,833 |
Other long-term liabilities | (7,235) | 5,730 |
Deferred revenue | (10,280) | (19,952) |
Operating lease liabilities | (7,842) | (5,764) |
Net cash used in operating activities | (357,919) | (380,872) |
Cash flows from investing activities | ||
Purchases of property and equipment | (11,389) | (7,438) |
Purchases of investments | (648,705) | (258,654) |
Maturities of investments | 855,365 | 170,123 |
Other | (289) | |
Net cash provided by (used in) investing activities | 195,271 | (96,258) |
Cash flows from financing activities | ||
Net proceeds from the sale of future royalties and revenues | 415,836 | |
Net proceeds from term loan facility | 97,968 | 137,797 |
Net proceeds from stock option exercises and employee stock purchase plan | 5,729 | 5,686 |
Net cash provided by financing activities | 103,697 | 559,319 |
Net increase (decrease) in cash, cash equivalents, and restricted cash | (58,951) | 82,189 |
Cash, cash equivalents and restricted cash at beginning of period | 124,904 | 215,119 |
Effect of exchange rate changes on cash, cash equivalents and restricted cash | (285) | (685) |
Cash, cash equivalents and restricted cash at end of period | 65,668 | 296,623 |
Supplemental cash flow information | ||
Cash paid for interest | 25,455 | 2,956 |
Property and equipment purchases unpaid at period end | 1,921 | 195 |
Cash paid for taxes, net | $ 762 | $ 4,018 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Cash Flows (Parenthetical) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | Dec. 31, 2021 |
Reconciliation of cash, cash equivalents, and restricted cash | ||||
Cash and cash equivalents | $ 55,972 | $ 119,709 | $ 291,430 | |
Restricted cash | 9,696 | 5,195 | 5,193 | |
Total cash, cash equivalents, and restricted cash shown in condensed consolidated statements of cash flows | $ 65,668 | $ 124,904 | $ 296,623 | $ 215,119 |
Nature of Business
Nature of Business | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Nature of Business | 1. Nature of Business Blueprint Medicines Corporation (the Company), a Delaware corporation incorporated on October 14, 2008, is a precision therapy company focused on genomically defined cancers and blood disorders. The Company’s approach is to leverage its novel research engine to systematically and reproducibly identify drivers of diseases in genomically defined patient populations, and to craft highly selective and potent drug candidates that are intended to provide significant and durable clinical responses to patients. The Company has brought its approved medicines, AYVAKIT®/AYVAKYT® (avapritinib) and GAVRETO® (pralsetinib), to patients in the U.S. and Europe, and the Company is globally advancing multiple programs for mast cell disorders, including SM and chronic urticaria, breast cancer and other cancers vulnerable to CDK2 inhibition, as well as EGFR-mutant lung cancer. 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 September 30, 2023, the Company had cash, cash equivalents and marketable securities of $827.2 million. Based on the Company’s current operating plans, the Company anticipates that its existing cash, cash equivalents and marketable securities will be sufficient to enable it to fund its current operations for at least the next twelve months from the issuance of the financial statements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies and Recent Accounting Pronouncements | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Summary of Significant Accounting Policies and Recent Accounting Pronouncements | 2. Summary of Significant Accounting Policies and Recent Accounting Pronouncements Basis of Presentation The unaudited interim condensed consolidated financial statements of the Company included herein have been prepared in accordance with accounting principles generally accepted in the United States (GAAP) as found in the Accounting Standards Codification (ASC), Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB) and the rules and regulations of the Securities and Exchange Commission (SEC). Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these financial statements should be read in conjunction with the financial statements as of and for the year ended December 31, 2022 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 16, 2023 (2022 Annual Report on Form 10-K). The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited financial statements, and updated, as necessary, in this report. In the opinion of the Company’s management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments that are necessary to present fairly the Company’s financial position as of September 30, 2023, the results of its operations for the three and nine months ended September 30, 2023 and 2022, stockholder’s equity for the three and nine months ended September 30, 2023 and 2022 and cash flows for nine months ended September 30, 2023 and 2022. Such adjustments are of a normal and recurring nature. The results for the three and nine months ended September 30, 2023 are not necessarily indicative of the results for the year ending December 31, 2023 or for any future period. The accompanying unaudited interim condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, Blueprint Medicines Security Corporation, which is a Massachusetts subsidiary created to buy, sell and hold securities, Blueprint Medicines (Switzerland) GmbH, Blueprint Medicines (Netherlands) B.V., Blueprint Medicines (UK) Ltd., Blueprint Medicines (Germany) GmbH, Blueprint Medicines (Spain) S.L., Blueprint Medicines (France) SAS, and Blueprint Medicines (Italy) S.r.L. All intercompany transactions and balances have been eliminated. Use of Estimates The preparation of financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: revenue recognition, inventory, operating lease right-of-use assets, operating lease liabilities, stock-based compensation expense, accrued expenses, liabilities related to the sale of future royalties and future revenues, equity investment, and income taxes. Actual results may differ from these estimates. Significant Accounting Policies The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and nine months ended September 30, 2023 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2022 Annual Report on Form 10-K, with the exception of the below new policy. Performance-Based Restricted Stock Unit Awards New Accounting Pronouncements |
Financing Arrangements
Financing Arrangements | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Financing Arrangements | 3. Financing Arrangements Royalty Pharma Purchase and Sale Agreement On June 30, 2022, the Company entered into a purchase and sale agreement (Royalty Purchase Agreement) with Royalty Pharma. Pursuant to the Royalty Purchase Agreement, the Company received an upfront payment of $175.0 million in consideration for the Company’s rights to receive royalty payments on the net sales of GAVRETO worldwide excluding the CStone Territory (as defined below) and U.S. territory under the terms of the Roche pralsetinib collaboration agreement. Although the Company sold all of the rights to receive royalties on the net sales of GAVRETO worldwide excluding the CStone Territory and U.S. territory to Royalty Pharma, the Company continues to co-develop pralsetinib with Roche globally and is therefore involved in the generation of these future royalties. Due to the Company’s significant continuing involvement, the Company continues to account for any royalties and development and commercialization milestones earned related to the underlying territory under the Roche pralsetinib collaboration agreement as collaboration revenue on its consolidated statements of operations and comprehensive loss. Net proceeds from the transaction were recorded as a liability related to sale of future royalties and revenues on the consolidated balance sheet. The Company accretes the $175.0 million, net of transaction costs of $3.8 million, to the total of these royalties as interest expense using the effective interest method over the estimated life of the arrangement. These estimates contain assumptions that impact the amount recorded and the interest expense that will be recognized in future periods. As payments are made to Royalty Pharma, the balance of the liability will be effectively repaid over the life of the Royalty Purchase Agreement. In order to determine the amortization of the liability, the Company estimates the total amount of future royalty payments to be paid to Royalty Pharma over the life of the arrangement. The exact amount of repayment is likely to change each reporting period. A significant increase or decrease in GAVRETO’s net revenue of the underlying territory will impact interest expense and the liability related to this arrangement. The Company periodically assesses the expected payments to Royalty Pharma and prospectively adjusts the amortization of the liability related to this arrangement for material changes in such payments including the prospective impact around the uncertainty related to GAVRETO’s commercial outlook in the underlying territory during the life of the arrangement. In February 2023, the Company received written notice from Roche of their election to terminate for convenience the Roche pralsetinib collaboration agreement. The termination will become effective February 2024, at which time the Company will regain commercialization and development rights to GAVRETO from Roche worldwide excluding the CStone Territory. As of September 30, 2023, none of the derecognition conditions under ASC 405 Liabilities Pursuant to the Royalty Purchase Agreement, the Company is eligible to receive certain milestone payments totaling up to $165.0 million, subject to the achievement of specified net sales milestones by Roche. However, following the termination of the Roche pralsetinib collaboration agreement, if the specified net sales milestone thresholds under the Royalty Purchase Agreement with Royalty Pharma are not otherwise met, the Company may no longer be eligible to receive any of the contingent milestone payments under the Royalty Purchase Agreement. The potential milestone payments will be added to the carrying value of the liability related to this arrangement when and if the milestones are achieved and received. Financing Arrangements with Sixth Street Partners Sixth Street Partners Purchase and Sale Agreement Pursuant to the Future Revenue Purchase Agreement, the Company received gross proceeds of $250.0 million in exchange for future royalty payments at a rate of 9.75% on up to $900 million each year of (i) aggregate worldwide annual net product sales of AYVAKIT/AYVAKYT (avapritinib) and (ii), if it is approved, aggregate worldwide annual net product sales of BLU-263 (elenestinib), but excluding sales in Greater China, subject to a cumulative cap of 1.45 times the upfront invested capital or a total of $362.5 million. In the event that certain revenue targets are not achieved by specified dates, the royalty rate and cumulative cap shall be increased to 15% and 1.85 times the invested capital (or $462.5 million), respectively. The Company continues to own the research, development, manufacturing and commercialization of AYVAKIT/AYVAKYT and if it is approved, elenestinib, and has significant continuing involvement in the generation of the cash flows under the Future Revenue Purchase Agreement. Therefore, the Company continues to account for any revenue earned from worldwide product sales of AYVAKIT/AYVAKYT and, if it is approved, elenestinib, on its consolidated statements of operations and comprehensive loss. Net proceeds received from the transaction were recorded as a liability related to sale of future royalties and revenues on the consolidated balance sheet. The Company accretes the $250.0 million, net of transaction costs of $5.4 million, to the total of these future payments as interest expense using the effective interest method over the estimated life of the arrangement. As payments are made to Sixth Street Partners, the balance of the liability is effectively repaid over the life of the Future Revenue Purchase Agreement. In order to determine the amortization of the liability, the Company estimates the total amount of future revenue payments to be paid to Sixth Street Partners over the life of the arrangement. The exact amount of repayment is likely to change each reporting period. A significant increase or decrease in worldwide product sales of AYVAKIT/AYVAKYT and, if it is approved, elenestinib, will materially impact the liability related to this arrangement, interest expense and the time period for repayment. The Company periodically assesses the expected payments to Sixth Street Partners and prospectively adjusts the amortization of the liability related to this arrangement for material changes in such payments. As of September 30, 2023, the Company’s estimate of this total interest expense resulted in an effective annual interest rate of 10.8%. These estimates contain assumptions that impact the amount recorded and the interest expense that will be recognized in future periods. As of September 30, 2023, the net carrying value of the liability related to this arrangement was $264.9 million. The following table shows the activity within the liability account (in thousands): Carrying value as of January 1, 2023 $ 254,328 Interest expense recognized 21,043 Payments (10,484) Carrying value as of September 30, 2023 $ 264,887 Sixth Street Partners Term Loan As part of the Financing Agreement with Sixth Street Partners, the Company received gross proceeds of $150.0 million in July 2022 and incurred an aggregate of $12.2 million of debt discounts and transaction costs. In August 2023, the Company received the first tranche of the senior secured delayed draw term loan facility in the amount of $100.0 million in gross proceeds and incurred $2.0 million of transaction costs. Debt discounts and transaction costs have been recorded as a reduction to the carrying amount of the debt on the Company’s consolidated balance sheet and are amortized as additional interest expenses using the effective interest rate method over the period from issuance through maturity. In addition, the Company may at any time request an incremental term loan in an amount not to exceed $260.0 million on terms to be agreed and subject to the consent of Sixth Street Partners providing such incremental term loan. As of September 30, 2023, the Company’s estimate of the total interest expense resulted in an effective annual interest rate of 13.3%. The carrying amount of the debt as of September 30, 2023 is subject to variable interest rates, which are based on current market rates, and as such, approximates fair value The following table shows the activity within the liability account (in thousands): Carrying value as of January 1, 2023 $ 139,083 Net proceeds received from the delayed draw term loan 97,968 Interest expense recognized 15,557 Payments (14,230) Carrying value as of September 30, 2023 $ 238,378 September 30, 2023, the Company was in compliance with the applicable terms and conditions of the covenants under the Financing Arrangement. |
Marketable Securities
Marketable Securities | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Marketable Securities | 4. Marketable Securities Marketable securities consisted of the following at September 30, 2023 and December 31, 2022 (in thousands): Amortized Unrealized Unrealized Fair September 30, 2023 Cost Gain Losses Value Marketable securities, available-for-sale: U.S. government agency securities $ 201,990 — (1,127) $ 200,863 U.S. treasury obligations 571,588 7 (1,204) 570,391 Total $ 773,578 $ 7 $ (2,331) $ 771,254 Amortized Unrealized Unrealized Fair December 31, 2022 Cost Gain Losses Value Marketable securities, available-for-sale: U.S. government agency securities $ 377,519 — (4,848) $ 372,671 U.S. treasury obligations 591,193 22 (5,123) 586,092 Total $ 968,712 $ 22 $ (9,971) $ 958,763 The following table summarizes the estimated fair value (in thousands) and number of the Company’s available-for-sale securities that are in loss position as of September 30, 2023 and December 31, 2022 by the length of time the security has been in loss position: September 30, 2023 December 31, 2022 Fair Number of Fair Number of value securities value securities Debt securities in unrealized loss position for 12 months or less $ 624,319 116 $ 371,746 66 Debt securities in unrealized loss position for more than 12 months 56,975 10 550,561 56 Total debt securities in unrealized loss position $ 681,294 126 $ 922,307 122 The Company has the intent and ability to hold its debt securities until recovery. As a result, the Company did not record any charges for credit-related impairments for its marketable debt securities for the three and nine months ended September 30, 2023 and 2022. The following table summarizes the amortized cost basis and estimated fair value of the Company’s available-for-sale securities by contractual maturity as of September 30, 2023 and December 31, 2022 (in thousands): September 30, 2023 December 31, 2022 Amortized Fair Amortized Fair Cost value Cost value Within one year $ 658,760 $ 657,012 $ 834,440 $ 825,283 After one through five years 114,818 114,242 134,272 133,480 Total $ 773,578 $ 771,254 $ 968,712 $ 958,763 The following table summarizes the proceeds from maturities of debt securities during the three and nine months ended September 30, 2023 and 2022 (in thousands): Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Proceeds from maturities of debt securities $ 237,600 $ 90,500 $ 855,365 $ 170,123 The Company did no t realize any gains or losses from maturities of debt securities for the three and nine months ended September 30, 2023 and 2022. |
Fair Value of Financial Instrum
Fair Value of Financial Instruments | 9 Months Ended |
Sep. 30, 2023 | |
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Fair Value of Financial Instruments | 5. Fair Value of Financial Instruments The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of September 30, 2023 (in thousands): Active Observable Unobservable September 30, Markets Inputs Inputs Description 2023 (Level 1) (Level 2) (Level 3) Cash equivalents: Money market funds $ 47,510 47,510 — $ — Marketable securities, available-for-sale: U.S. government agency securities 200,863 — 200,863 — U.S. treasury obligations 570,391 570,391 — — Total $ 818,764 $ 617,901 $ 200,863 $ — The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2022 (in thousands): Active Observable Unobservable December 31, Markets Inputs Inputs Description 2022 (Level 1) (Level 2) (Level 3) Cash equivalents: Money market funds $ 95,198 $ 95,198 $ — $ — U.S. treasury obligations 4,989 4,989 — — Marketable securities, available-for-sale: U.S. government agency securities 372,671 — 372,671 — U.S. treasury obligations 586,092 586,092 — — Total $ 1,058,950 $ 686,279 $ 372,671 $ — |
Product Revenue Reserves and Al
Product Revenue Reserves and Allowances | 9 Months Ended |
Sep. 30, 2023 | |
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Product Revenue Reserves and Allowances | 6. Product Revenue Reserves and Allowances In January 2020, the U.S. Food and Drug Administration (FDA) approved AYVAKIT for the treatment of adults with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring a PDGFRA exon 18 mutation, including PDGFRA D842V mutations. In September 2020, the European Commission granted conditional marketing authorization to AYVAKYT as a monotherapy for the treatment of adult patients with unresectable or metastatic GIST harboring the PDGFRA D842V mutation. In June 2021, the FDA granted a subsequent approval for AYVAKIT, expanding the labeled indications to include adult patients with advanced SM, including aggressive SM (ASM), SM with an associated hematological neoplasm (SM-AHN) and mast cell leukemia (MCL). In March 2022, the European Commission expanded the marketing authorization for AYVAKYT to include the treatment of adult patients with ASM, SM-AHN, or MCL, after at least one systemic therapy. In May 2023, the FDA approved AYVAKIT for the treatment of adult patients with indolent SM. The following table summarizes revenue recognized from product sales for the three and nine months ended September 30, 2023 and 2022 (in thousands): Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 United States $ 49,121 $ 25,054 $ 118,355 $ 70,888 Rest of World 5,107 3,580 14,818 10,041 Total product revenue $ 54,228 $ 28,634 $ 133,173 $ 80,929 Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Customer 1 42 % 45 % 42 % 46 % Customer 2 11 % 10 % 11 % 11 % September 30, December 31, 2023 2022 Customer 1 31 % 33 % Customer 2 13 % 15 % Customer 3 11 % * Customer 4 * 12 % * Indicates the customer’s share is under 10% . The following table summarizes activity in each of the product revenue allowance and reserve categories for the nine months ended September 30, 2023 and 2022 (in thousands): Nine Months Ended September 30, 2023 2022 Beginning balance at January 1 $ 9,788 $ 4,345 Provision related to sales in the current period 25,874 13,606 Adjustment related to prior periods sales (545) (645) Credits and payments made (17,469) (9,980) Ending balance at September 30 $ 17,648 $ 7,326 The total reserves that are included in the Company’s unaudited condensed consolidated balance sheets as of September 30, 2023 and December 31, 2022, are summarized as follows (in thousands): September 30, December 31, 2023 2022 Reduction of accounts receivable, net $ 1,547 $ 742 Component of accrued expenses 16,101 9,046 Total revenue-related reserves $ 17,648 $ 9,788 |
Inventory
Inventory | 9 Months Ended |
Sep. 30, 2023 | |
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Inventory | 7. Inventory Capitalized inventory consists of the following at September 30, 2023 and December 31, 2022 (in thousands): September 30, December 31, 2023 2022 Raw materials $ 1,599 $ 4,967 Work in process 29,335 28,427 Finished goods 3,475 2,791 Total $ 34,409 $ 36,185 Balance sheet classification September 30, December 31, 2023 2022 Inventory $ 16,457 $ 29,697 Other assets 17,952 6,488 Total $ 34,409 $ 36,185 Inventory amounts written down as a result of excess, obsolescence, unmarketability or other reasons are charged to cost of sales. The Company recognized a write-down of $0.1 million and $2.2 million for the three and nine months ended September 30, 2023, respectively. The Company did not recognize any write-down for the three and nine months ended September 30, 2022. Long-term inventory, which primarily consists of work in process and raw materials, is included in other assets in the unaudited condensed consolidated balance sheets. |
Restricted Cash
Restricted Cash | 9 Months Ended |
Sep. 30, 2023 | |
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Restricted Cash | 8. Restricted Cash At September 30, 2023 and December 31, 2022, $9.7 million and $5.2 million, respectively, of the Company’s cash is restricted by a financial institution primarily related to funds held to satisfy the requirements of certain government agreements and the security deposits for the lease agreements for the Company’s office and laboratory spaces. |
Accrued Expenses
Accrued Expenses | 9 Months Ended |
Sep. 30, 2023 | |
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Accrued Expenses | 9. Accrued Expenses Accrued expenses consist of the following (in thousands): September 30, December 31, 2023 2022 Research, development and commercial contract costs $ 38,699 $ 66,041 Employee compensation 34,517 30,744 Accrued professional fees 14,592 17,588 Revenue-related reserves 16,101 9,046 Other 6,527 7,704 Total $ 110,436 $ 131,123 |
Collaboration and License Agree
Collaboration and License Agreements | 9 Months Ended |
Sep. 30, 2023 | |
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Collaboration and License Agreements | 10 . Collaboration and License Agreements IDRx In August 2022, the Company entered into a license agreement with IDRx, Inc. (IDRx), pursuant to which the Company granted IDRx an exclusive, worldwide, royalty-bearing license to exploit the Company’s internally discovered KIT exon 13 inhibitor IDRX-73 (IDRx License Agreement). IDRx is a clinical-stage biopharmaceutical company and a mong IDRx’s founders are Alexis Borisy, George Demetri, M.D., and Nicholas Lydon, Ph.D., who were each members of the Company’s board of directors at the time. Due to these relationships, the transaction with IDRx is a related party transaction. In connection with the IDRx License Agreement, the Company also entered into a stock purchase agreement with IDRx (IDRx Stock Purchase Agreement), pursuant to which the Company received 4,509,105 shares of IDRx’s Series A preferred stock with the right to receive additional shares of IDRx’s Series A preferred stock through an anti-dilution provision subject to a defined financing cap. In July 2023, the Company received 192,282 additional shares under the anti-dilution provision. The Company is also eligible to receive up to $217.5 million in contingent cash payments, including specified development, regulatory and sales-based milestone payments. In addition, IDRx is obligated to pay to the Company royalties on aggregate annual worldwide net sales of licensed products at tiered percentage rates up to low-teens, subject to adjustments in specified circumstances under the IDRx License Agreement. Unless earlier terminated, the IDRx License Agreement will expire on a country-by-country, licensed product-by-licensed product basis upon the latest of: (a) the expiration of the last valid claim within the licensed patents covering such licensed product in a such country, (b) the expiration of the regulatory exclusivity period for such licensed product in such country, or (c) the 10th anniversary of the first commercial sale of such licensed product in such country. Following the end of the term for any such licensed product and in such region by expiration, the license granted to IDRx will become exclusive, perpetual, irrevocable, fully paid-up and royalty-free. IDRx may terminate the IDRx License Agreement for convenience at any time upon at least twelve months ’ prior written notice to the Company. Either party may also terminate the IDRx License Agreement for material breach of the other party or for insolvency, and the Company may terminate the IDRx License Agreement for IDRx’s breach of the anti-dilution provision in the IDRx Stock Purchase Agreement. Upon termination of the license agreement in its entirety, all rights and obligations under the license agreement will terminate and revert back to the Company, and the Company has a license under certain intellectual property of IDRx to continue to exploit the compound and terminated product, subject to a royalty that will be negotiated at the time of termination. The Company considered the ASC 606 criteria for combining contracts and determined the IDRx License Agreement and the IDRx Stock Purchase Agreement should be combined into a single contract because they were negotiated and entered into in contemplation of one another. Therefore, the Company determined that the 4,509,105 shares of IDRx’s Series A preferred stock and the anti-dilution right to receive additional shares should be attributed to the transaction price of the IDRx License Agreement. The Company evaluated the IDRx License Agreement under ASC 606. The Company identified the following material promises under the agreement: (1) the exclusive license and (2) the initial know-how transfer. The Company determined that the exclusive license and the initial know-how transfer were not distinct from each other, as the exclusive license has limited value without the corresponding know-how transfer. As such, for the purposes of ASC 606, the Company determined that these two material promises, the exclusive license and the initial know-how transfer, should be combined into one distinct performance obligation. The Company concluded that the license is a functional intellectual property license. The Company determined that IDRx benefited from the license along with the initial know-how transfer at the time of grant, and therefore the related performance obligation is satisfied at a point in time. For the purposes of ASC 606, the transaction price of the IDRx License Agreement at the contract inception was determined to be $27.5 million and recorded as license revenue-related party on the consolidated statements of operations and comprehensive loss during the year ended December 31, 2022. The fair value was derived from IDRx’s most recent financing transaction with unrelated investors. All potential milestone payments that the Company is eligible to receive under the IDRx License Agreement have been excluded from the transaction price. The Company reevaluates the transaction price for inclusion of milestone payments and royalties at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. Additionally, the Company is entitled to sales milestones and royalties from the sales of the licensed products, and revenue are recognized when the related sales occur. The Company concluded the preferred stock investment should be accounted for as an equity investment as it is not mandatorily redeemable nor does the Company have the unilateral right to redeem the preferred stock, and the Company, along with its related parties, do not have a controlling financial interest in IDRx nor have the ability to influence the financial and operating policies through the ownership of preferred stock. IDRx’s preferred stock is not exchange-traded and does not have a readily determinable fair value. Therefore, the Company accounts for the preferred stock investment under the measurement alternative for equity investments that do not have a readily determinable fair value, which is at cost of $27.8 million including transaction costs of $0.3 million. As of September 30, 2023, the cost of the investment in IDRx’s preferred stock was $27.8 million and was recorded as equity investment on the condensed consolidated balance sheets. As of September 30, 2023, no adjustments have been recognized related to the preferred stock investment as a result of the application of the measurement alternative. Proteovant an exclusive as well as up to two additional novel protein degrader target programs as may be mutually agreed to by the Company and Proteovant (each program is referred to as a target program) On a target program-by-target program basis, the Company has an exclusive option to obtain a worldwide, exclusive license to develop and commercialize any licensed compound and licensed product under each target program. Proteovant has the right to opt into the global development and U.S. commercialization of certain licensed compounds and licensed products under the second target program that the Company options, and if the parties add additional target programs, Proteovant will have the same opt-in right for the fourth target program that the Company options. The Company concluded that Proteovant is providing the Company with research services throughout the period until the Company can exercise its option to obtain a worldwide, exclusive license to develop and commercialize any licensed compound Zai Lab In November 2021, the Company entered into a collaboration (the Zai Lab agreement) with Zai Lab (Shanghai) Co., Ltd., (Zai Lab) to develop and commercialize certain licensed products for the treatment of EGFR-driven non-small cell lung cancer in Greater China, including Mainland China, Hong Kong, Macau and Taiwan (collectively, the Zai Lab Territory). The collaboration aims to accelerate and expand global development of the licensed products, which currently include BLU-945 and BLU-525 (which served as a back-up candidate for BLU-701). The Company retains exclusive rights to the licensed products outside the Zai Lab Territory. Under the Zai Lab agreement, the Company received an upfront cash payment of $25.0 million and, in addition to the upfront payment received, the Company is eligible to receive up to $590.0 million in contingent payments, including specified development, regulatory and sales-based milestones and tiered percentage royalties on a licensed product-by-licensed product basis ranging from the low-teens to mid-teens on annual net sales of each licensed product in the Zai Lab Territory, subject to adjustment in specified circumstances. Zai Lab is responsible for costs related to clinical trials in the Zai Lab Territory, other than the specified shared services costs as defined in the Zai Lab agreement which are shared by the Company and Zai Lab. Pursuant to the terms of the Zai Lab agreement, Zai Lab is responsible for conducting all development and commercialization activities in the Zai Lab Territory related to the licensed drug candidates. In addition, under the Zai Lab agreement, each party has granted the other party specified intellectual property licenses to enable the other party to perform its obligations and exercise its rights under the Zai Lab agreement, including license grants to enable each party to conduct research, development and commercialization activities pursuant to the terms of the Zai Lab agreement. The Zai Lab agreement will continue on a licensed product-by-product and region-by-region basis until the later of (i) the 12 th second The Company evaluated the Zai Lab agreement to determine whether it is a collaborative arrangement in the scope of ASC 808. The Company concluded that the Zai Lab agreement is a collaborative agreement under ASC 808 as both parties are active participants in the clinical trials and are exposed to significant risks and rewards of those activities under the Zai Lab agreement. The Company determined that the Zai Lab agreement contained two material components: (i) licenses granted to Zai Lab to exploit and develop each licensed product in the Zai Lab Territory and related activities in the Zai Lab Territory, including manufacturing, and (ii) the parties’ participation in the global development of the licensed products. The Company used the criteria specified in ASC 606 to determine which of the components of the Zai Lab agreement are performance obligations with a customer and concluded that Zai Lab is the Company’s customer for the licenses and related activities in the Zai Lab Territory under ASC 606. The global development activities under the agreement does not present a transaction with a customer and the payments received by the Company for global development activities, including manufacturing, are accounted for as a reduction of related expenses. During the three and nine months ended September 30, 2023, the Company did not record such reductions of expenses under the Zai Lab agreement. During the three and nine months ended September 30, 2022, the Company recorded $0.2 million and $0.5 million, respectively, in reductions of expenses under the Zai Lab agreement. The Company evaluated the Zai Lab Territory specific licenses and related activities under ASC 606 as these transactions are considered transactions with a customer and identified three material promises at the outset of the Zai Lab agreement, which consists of the following for each licensed product: (1) the exclusive license, (2) the initial know-how transfer and (3) manufacturing activities related to development and commercial supply of the licensed product in the Zai Lab Territory. The Company determined that the exclusive license and the initial know-how transfer were not distinct from each other, as the exclusive license has limited value without the corresponding know-how transfer. As such, for the purposes of ASC 606, the Company determined that these two material promises, the exclusive license and the initial know-how, should be combined into one distinct performance obligation. The Company further evaluated the material promise associated with manufacturing activities related to development and commercial supply of the licensed products in the Zai Lab Territory, given Zai Lab is not obligated to purchase any minimum amount or quantities of the development and commercial supply from the Company, the Company concluded that, for the purpose of ASC 606, the provision of manufacturing activities related to development and commercial supply of the licensed product in Zai Lab Territory was an option but not a performance obligation of the Company at the inception of the Zai Lab collaboration agreement and will be accounted for if and when exercised. The Company also concluded that there is no separate material right in connection with the development and commercial supply of the licensed product, as the expected pricing was not issued at a significant and incremental discount. Therefore, the manufacturing activities were excluded as performance obligation at the outset of the arrangement. The Company evaluated the license under ASC 606 and concluded that the license is a functional intellectual property license. The Company determined that Zai Lab benefited from the license along with the initial know-how transfer at the time of grant, and therefore the related performance obligation is satisfied at a point in time. Additionally, the Company is entitled to sales milestones and royalties from Zai Lab upon future sales of the licensed products in the Zai Lab Territory, and revenue will be recognized when the related sales occur. Costs that are incurred associated with Zai Lab Territory specific activities are reimbursable from Zai Lab and are recognized as revenue. The revenue recognized under the Zai Lab agreement during the three and nine months ended September 30, 2023 was negligible. During the three and nine months ended September 30, 2022, the Company recorded $0.2 million and $0.8 million, respectively, in revenue related to Zai Lab Territory specific activities. For the purposes of ASC 606, the transaction price of the Zai Lab agreement at the outset of the arrangement was determined to be $25.0 million, which consisted of the upfront cash payment. The other potential milestone payments that the Company is eligible to receive were excluded from the transaction price, as all milestone amounts were fully constrained based on the probability of achievement. The Company satisfied the performance obligation upon delivery of the licenses and initial know-how transfer, and recognized the upfront payment of $25.0 million as revenue in 2021. The Company reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. Roche – Pralsetinib Collaboration Under the Roche pralsetinib collaboration agreement, the Company received an upfront cash payment of $675.0 million, and through September 30, 2023, the Company received an aggregate of $105.0 million in specified regulatory and commercialization milestones. In February 2023, the Company received written notice from Roche of their election to terminate for convenience the Roche pralsetinib collaboration agreement. The termination will become effective in February 2024, at which time the Company will regain commercialization and development rights to GAVRETO from Roche worldwide excluding the CStone Territory. Until the termination of the collaboration agreement is effective, the parties continue to perform their respective obligations under the collaboration agreement, including with respect to the development and commercialization of GAVRETO. The Company will not be entitled to receive payment for milestones, if any, achieved during the period between the receipt of the notice of termination and the effective date of termination. The termination of the collaboration agreement does not have an impact on the Company’s current accounting treatment related to the Roche pralsetinib collaboration. In the U.S., the Company and Roche agreed to work together to co-commercialize pralsetinib and equally share responsibilities, profits and losses. In addition, the Company is eligible to receive tiered royalties ranging from high-teens to mid-twenties on annual net sales of pralsetinib outside the U.S., excluding Greater China (the Roche Territory). The Company and Roche also agreed to co-develop pralsetinib globally in RET-altered solid tumors, including non-small cell lung cancer, medullary thyroid carcinoma and other thyroid cancers, as well as other solid tumors. The Company and Roche share global development costs for pralsetinib at a rate of 45 percent for the Company and 55 percent for Roche up to a specified amount of aggregate joint development costs, after which the Company’s share of global development costs for pralsetinib will be reduced by a specified percentage. The Company and Roche will also share specified global development costs for any next-generation RET compound co-developed under the collaboration in a similar manner. In connection with the Roche collaboration agreement, on July 13, 2020, the Company also entered into a stock purchase agreement with Roche Holdings, Inc. (Roche Holdings) pursuant to which the Company issued and sold an aggregate of 1,035,519 shares of common stock to Roche Holdings at a purchase price of $96.57 per share and received an aggregate of $100.0 million in the third quarter of 2020. The closing for a minority portion of the equity investment occurred following the expiration of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended, and other customary closing conditions. The Company considered the ASC 606 criteria for combining contracts and determined that the Roche pralsetinib collaboration agreement and stock purchase agreement should be combined into a single contract because they were negotiated and entered into in contemplation of one another. The Company accounted for the common stock issued to Roche Holdings based on the fair market value of the common stock on the dates of issuance. The fair market value of the common stock issued to Roche Holdings was $79.3 million, based on the closing price of the Company’s common stock on the dates of issuance, resulting in a $20.7 million premium. The Company determined that the premium paid by Roche Holdings for the common stock should be attributed to the transaction price of the Roche pralsetinib collaboration agreement. The Company determined that the Roche pralsetinib collaboration agreement contained four material components: (i) licenses granted to Roche to develop and commercialize pralsetinib worldwide, excluding the CStone Territory (pralsetinib license); (ii) the Roche Territory-specific commercialization activities for pralsetinib, including manufacturing (Roche Territory activities); (iii) the parties’ joint development activities for pralsetinib worldwide, excluding the CStone Territory; and (iv) the parties’ joint commercialization activities for pralsetinib in the U.S. The Company considered the guidance in ASC 606 to determine which of the components of the Roche pralsetinib collaboration agreement are performance obligations with a customer and concluded that the pralsetinib license and the Roche Territory activities are within the scope of ASC 606 because Roche is the Company’s customer in those transactions. The Company evaluated the Roche pralsetinib license under ASC 606 and concluded that the pralsetinib license is a functional intellectual property license and is a distinct performance obligation. The Company determined that Roche benefited from the pralsetinib license at the time of grant, and therefore the related performance obligation is satisfied at a point in time. The Company evaluated the Roche Territory activities under ASC 606 and identified one material promise associated with manufacturing activities related to development and commercial supply of pralsetinib in the Roche Territory for up to 24 months. Given that Roche is not obligated to purchase any minimum amount or quantities of the development and commercial supply from the Company, the Company concluded that, for the purpose of ASC 606, the provision of manufacturing activities related to development and commercial supply of pralsetinib in Roche Territory was an option but not a performance obligation of the Company at the inception of the Roche collaboration agreement and is accounted for if and when exercised. The Company also concluded that there is no separate material right in connection with the development and commercial supply of pralsetinib, as the expected pricing was not issued at a significant and incremental discount. Therefore, the manufacturing activities were excluded as performance obligations at the outset of the arrangement. Additionally, the Company is entitled to sales milestones and royalties from Roche upon future sales of pralsetinib in the Roche Territory, and the revenue is recognized when the related sales occur. Costs that are incurred associated with the Roche Territory activities are reimbursable from Roche and are recognized as revenue. For the purposes of ASC 606, the transaction price of the Roche collaboration agreement at the outset of the arrangement was determined to be $695.7 million, which consisted of the upfront cash payment of $675.0 million and the $20.7 million premium on the sale of common stock to Roche Holdings, which was allocated to the performance obligation related to the pralsetinib licenses. Through September 30, 2023, the Company has achieved an aggregate of $105.0 million in specified regulatory and commercialization milestones which were added to the estimated transaction price of the Roche pralsetinib agreement. The following table summarizes revenue recognized under the Roche pralsetinib collaboration during the three and nine months ended September 30, 2023 and 2022 (in thousands): Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Manufacturing and research and development services related to $ 213 $ 3,971 $ 570 $ 4,663 Royalty revenue 300 311 1,102 910 Total Roche pralsetinib collaboration revenue $ 513 $ 4,282 $ 1,672 $ 5,573 For the parties’ participation in global development for pralsetinib and the U.S. commercialization activities for GAVRETO, the Company concluded that those activities and cost-sharing payments related to such activities are within the scope of ASC 808, as both parties are active participants in the development, manufacturing and commercialization activities and are exposed to significant risks and rewards of those activities under the Roche pralsetinib collaboration agreement. Payments to or reimbursements from Roche related to the global development activities are accounted for as an increase to or reduction of research and development expenses. In September 2020, the FDA granted accelerated approval of GAVRETO for the treatment of adult patients with metastatic RET fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA approved test. In December 2020, the FDA granted a subsequent accelerated approval for GAVRETO, expanding the labeled indications to include adult and pediatric patients 12 years of age and older with advanced or metastatic RET-mutant medullary thyroid cancer (MTC) who require systemic therapy (which indication was subsequently voluntarily withdrawn by Roche in June 2023, followed by the official FDA withdrawal on July 20, 2023), or with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate). In August 2023, the FDA converted the accelerated approval of GAVRETO to full regular approval for the treatment of adult patients with metastatic RET fusion-positive NSCLC. Prior to July 1, 2021, the Company was the principal for product sales to customers in the U.S. and recognized revenues on sales to third parties in product revenue, net in its consolidated statements of operations and comprehensive loss. On July 1, 2021, Roche took over certain responsibilities associated with product sales to customers, pricing and distribution matters for GAVRETO in the U.S. and became the principal for recording product sales to customers in the U.S., and the Company recognized its portion of the commercial losses sharing as collaboration loss sharing in its consolidated statements of operations and comprehensive loss. The following table summarizes the amount recognized from loss sharing from product sales of GAVRETO to customers in the U.S. during the three and nine months ended September 30, 2023 and 2022 (in thousands): Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 The Company's share of loss in the U.S. for pralsetinib $ 1,771 $ 1,665 $ 4,301 $ 7,076 The following table summarizes the amounts recognized as reductions to selling, general and administrative expenses related to the commercialization of GAVRETO in the U.S. and increases in research and development expenses related to global development activities for pralsetinib under the Roche pralsetinib collaboration during the three and nine months ended September 30, 2023 and 2022 (in thousands): Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Reductions to selling, general and administrative expenses $ 3,063 $ 3,560 $ 8,618 $ 12,808 Increases in research and development expenses $ 7,603 $ 6,111 $ 21,226 $ 9,815 The following table summarizes the contract assets and liability associated with the Roche pralsetinib collaboration as of September 30, 2023 and December 31, 2022 (in thousands): September 30, December 31, 2023 2022 Unbilled accounts receivable $ 304 $ — Accrued expenses $ 6,221 $ 6,519 Although the Company sold its right to receive royalty payments from Roche’s sales of GAVRETO in Roche Territory to Royalty Pharma in June 2022, given the Company’s significant continuing involvement in the generation of future royalties, the Company continues to account for any royalties earned related to the Roche Territory activities under the Roche pralsetinib collaboration agreement as collaboration revenue on its consolidated statements of operations and comprehensive loss. For additional information, see Note 3 – Financing Arrangements Clementia In October 2019, the Company entered into a license agreement (the Clementia agreement) with Clementia Pharmaceuticals, Inc. (Clementia), a wholly-owned subsidiary of Ipsen S.A. Under the Clementia agreement, the Company granted an exclusive, worldwide, royalty-bearing license to Clementia to develop and commercialize BLU-782, the Company’s oral, highly selective investigational ALK2 inhibitor in Phase 1 clinical development for the treatment of fibrodysplasia ossificans progressiva (FOP), as well as specified other compounds related to the BLU-782 program. Under the Clementia agreement, the Company received an upfront cash payment of $25.0 million and through September 30, 2023, the Company has received an aggregate of $50.0 million in cash milestone payments. Subject to the terms of the Clementia agreement, in addition to the upfront and milestone payments received through September 30, 2023, the Company is eligible to receive up to $460.0 million in contingent payments, including specified development, regulatory and sales-based milestones for licensed products. In addition, Clementia is obligated to pay to the Company royalties on aggregate annual worldwide net sales of licensed products at tiered percentage rates ranging from the low- to mid-teens, subject to adjustment in specified circumstances under the Clementia agreement, and Clementia purchased specified manufacturing inventory from the Company for a total of $1.5 million. Unless earlier terminated in accordance with the terms of the Clementia agreement, the agreement will expire on a country-by-country, licensed product-by-licensed product basis on the date when no royalty payments are or will become due. Clementia may terminate the agreement at any time upon at least 12 months’ prior written notice to the Company. Either party may terminate the agreement for the other party’s uncured material breach or insolvency and in certain other circumstances agreed to by the parties. In certain termination circumstances, the Company is entitled to retain specified licenses to be able to continue to exploit the Clementia licensed products. The Company evaluated the Clementia agreement under ASC 606, as the agreement represented a transaction with a customer. The Company identified the following material promises under the agreement: (1) the exclusive license to develop, manufacture and commercialize BLU-782; (2) the technology transfer of BLU-782 program; (3) the transfer of existing manufacturing inventory; and (4) the transfer of in-process manufacturing inventory. In addition, the Company determined that the exclusive license and technology transfer were not distinct from each other, as the exclusive license has limited value without the corresponding technology transfer. As such, for the purposes of ASC 606, the Company determined that these four material promises, described above, should be combined into three performance obligations: (1) the exclusive license and the technology transfer; (2) the transfer of existing manufacturing inventory; and (3) the transfer of in-process manufacturing inventory. The Company determined that the transaction price at the outset of the arrangement was $46.5 million, which was allocated to the three performance obligations on a relative stand-alone selling price basis, and was recognized as revenue During the three and nine months ended September 30, 2023, and during the three months ended September 30, 2022, no revenue was recognized from the Clementia collaboration. During the nine months ended September 30, 2022, cash consideration associated with an achieved development milestone of $30.0 million was added to the estimated transaction price for the Clementia agreement and recognized as revenue. The other potential milestone payments that the Company is eligible to receive were excluded from the transaction price, as the amounts were fully constrained based on the probability of achievement. The Company reevaluates the transaction price at the end of each reporting period and as uncertain events are resolved or other changes in circumstances occur, and if necessary, the Company adjusts its estimate of the transaction price, and any addition to the transaction price would be recognized as revenue when it becomes probable that inclusion would not lead to a significant revenue reversal. There was no revenue deferred as a contract liability associated with the Clementia agreement as of September 30, 2023 and December 31, 2022. CStone Pharmaceuticals In June 2018, the Company entered into a collaboration and license agreement (the CStone agreement) with CStone Pharmaceuticals (CStone) pursuant to which the Company granted CStone exclusive rights to develop and commercialize the Company’s drug candidates avapritinib, pralsetinib and fisogatinib, including back-up forms and certain other forms thereof, in Mainland China, Hong Kong, Macau and Taiwan (each, a CStone region and collectively, the CStone Territory), either as a monotherapy or as part of a combination therapy. The Company received an upfront cash payment of $40.0 million, and through September 30, 2023, the Company has achieved an aggregate of $38.5 million in milestones under this collaboration. Subject to the terms of the CStone agreement, in addition to the upfront payments received and milestones achieved through September 30, 2023, the Company will be eligible to receive up to $307.5 million in contingent payments, including specified development, regulatory and sales-based milestones for licensed products. In additio |
Stock-based Compensation
Stock-based Compensation | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Stock-based Compensation | 11. Stock-based Compensation 2015 Stock Option and Incentive Plan In 2015, the Company’s board of directors and stockholders approved the 2015 Stock Option and Incentive Plan (the 2015 Plan), which replaced the Company’s 2011 Stock Option and Grant Plan, as amended (the 2011 Plan). The 2015 Plan includes incentive stock options, nonstatutory stock options, stock appreciation rights, restricted stock, restricted stock units, performance-based restricted stock units, unrestricted stock, performance-based awards and cash-based awards. The Company initially reserved a total of 1,460,084 shares of common stock for the issuance of awards under the 2015 Plan. The 2015 Plan provides that the number of shares reserved and available for issuance under the 2015 Plan will be cumulatively increased on January 1 of each calendar year by 4% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar year beginning January 1, 2023, the number of shares reserved for issuance under the 2015 Plan was increased by 2,398,356 shares. In addition, the total number of shares reserved for issuance is subject to adjustment in the event of a stock split, stock dividend or other change in the Company’s capitalization. As of September 30, 2023, there were 4,896,334 shares available for future grant under the 2015 Plan. 2020 Inducement Plan In March 2020, the Company’s board of directors adopted the 2020 Inducement Plan (the Inducement Plan), pursuant to which the Company may grant, subject to the terms of the Inducement Plan and Nasdaq rules, nonstatutory stock options, stock appreciation rights, restricted stock awards, restricted stock units, and other stock-based awards. The Company initially reserved a total of 1,000,000 shares of common stock for the issuance of awards under the Inducement Plan. In June 2022, the Company’s board of directors approved the reservation of an additional 1,500,000 shares of common stock for the issuance of awards under the Inducement Plan. The number of shares reserved and available for issuance under the Inducement Plan can be increased at any time with the approval of the Company’s board of directors. The Inducement Plan permits the board of directors or a committee thereof to use the stock-based awards available under the Inducement Plan to attract key employees for the growth of the Company. As of September 30, 2023, there were 1,366,923 shares available for future grant under the Inducement Plan. Stock options The following table summarizes the stock option activity for the nine months ended September 30, 2023: Weighted-Average Shares Exercise Price Outstanding at December 31, 2022 6,233,451 $ 69.14 Granted 1,182,245 45.31 Exercised (154,433) 22.02 Canceled (277,649) 70.90 Outstanding at September 30, 2023 6,983,614 $ 66.08 Exercisable at September 30, 2023 4,756,302 $ 68.88 As of September 30, 2023, the total unrecognized compensation expense related to unvested stock option awards was $69.2 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 nine months ended September 30, 2023: Weighted-Average Shares Grant Date Fair Value Unvested shares at December 31, 2022 1,893,944 $ 73.28 Granted 888,010 44.78 Vested (569,385) 72.64 Forfeited (119,829) 64.97 Unvested shares at September 30, 2023 2,092,740 $ 61.85 As of September 30, 2023, the total unrecognized compensation expense related to unvested restricted stock units was $99.0 million, which is expected to be recognized over a weighted-average period of approximately 2.55 years. Performance-based restricted stock units During the first quarter of 2023, the Company began granting performance-based restricted stock units (PSUs) that will settle in stock. PSUs awarded to employees have a third Weighted-Average Grant Date Shares Grant Date Fair Value Unvested shares at December 31, 2022 — $ — Granted 52,500 59.32 Vested — — Forfeited — — Unvested shares at September 30, 2023 52,500 $ 59.32 2015 Employee Stock Purchase Plan In 2015, the Company’s board of directors and stockholders approved the 2015 Employee Stock Purchase Plan (the 2015 ESPP), which became effective upon the closing of the Company’s initial public offering in May 2015. The Company initially reserved a total of 243,347 shares of common stock for issuance under the 2015 ESPP. The 2015 ESPP provides that the number of shares reserved and available for issuance under the 2015 ESPP will be cumulatively increased on January 1 of each calendar year by 1% of the number of shares of common stock issued and outstanding on the immediately preceding December 31 or such lesser amount as specified by the compensation committee of the board of directors. For the calendar year beginning January 1, 2023, the number of shares reserved for issuance under the 2015 ESPP was increased by 599,589 shares. Stock-based compensation expense The Company recognized stock-based compensation expense of $23.1 million and $70.1 million for the three and nine months ended September 30, 2023, respectively, and $24.1 million and $72.9 million for the three and nine months ended September 30, 2022, respectively. Stock-based compensation expense by award type included within the unaudited condensed consolidated statements of operations and comprehensive loss was as follows (in thousands): Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Stock options $ 9,998 $ 12,062 $ 31,195 $ 37,819 Restricted stock units 12,536 11,802 37,389 34,583 Performance-based restricted stock units 261 — 605 — Employee stock purchase plan 417 404 1,308 1,000 Subtotal 23,212 24,268 70,497 73,402 Capitalized stock-based compensation costs (141) (159) (410) (547) Stock-based compensation expense included in total cost and operating expenses $ 23,071 $ 24,109 $ 70,087 $ 72,855 Stock-based compensation expense, that is included in operating expenses, Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Research and development $ 11,169 $ 10,010 $ 31,512 $ 30,548 Selling, general and administrative 11,902 14,099 38,575 42,307 Total $ 23,071 $ 24,109 $ 70,087 $ 72,855 |
Net Loss Per Share
Net Loss Per Share | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Net Loss Per Share | 12. Net Loss per Share Basic net loss per share is calculated by dividing net loss by the weighted average shares outstanding during the period, without consideration for common stock equivalents. Diluted net loss per share is calculated by adjusting weighted average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period. For purposes of the dilutive net loss per share calculation, stock options, unvested restricted stock units, PSUs and ESPP shares are considered to be common stock equivalents but are excluded from the calculation of diluted net loss per share, as their effect would be anti-dilutive; therefore, basic and diluted net loss per share were the same for all periods presented as a result of the Company’s net loss. The following common stock equivalents were excluded from the calculation of diluted net loss per share for the three and nine months ended September 30, 2023 and 2022 because including them would have had an anti-dilutive effect (in thousands): September 30, 2023 2022 Stock options 6,984 6,316 Restricted stock units 2,093 1,838 Performance-based restricted stock units 53 — ESPP shares 47 46 Total 9,177 8,200 |
Income Taxes
Income Taxes | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Income Taxes | 13. Income Taxes The Company utilizes the asset and liability method of accounting for income taxes. Under this method, deferred tax assets and liabilities are determined based on the difference between the financial statement carrying amounts and tax basis of assets and liabilities using enacted tax rates in effect for years in which the temporary differences are expected to reverse. The Company provides a valuation allowance when it is more likely than not that deferred tax assets will not be realized. The realization of deferred income tax assets is dependent on the generation of sufficient taxable income during future periods in which temporary differences are expected to reverse. Where the realization of such assets does not meet the more likely than not criterion, the Company applies a valuation allowance against the deferred income tax asset under consideration. The valuation allowance is reviewed periodically and if the assessment of the more likely than not criterion changes, the valuation allowance is adjusted accordingly. As of September 30, 2023, the Company has a full valuation allowance applied against its U.S. and foreign deferred tax assets. Effective January 1, 2022, a provision of the Tax Cuts and Jobs Act (TCJA) has taken effect creating a significant change to the treatment of research and experimental (R&E) expenditures under Section 174 of the IRC (Sec. 174 expenses). Historically, businesses have had the option of deducting Sec. 174 expenses in the year incurred or capitalizing and amortizing the costs over five years. The new TCJA provision, however, eliminates this option and will require Sec. 174 expenses associated with research conducted in the U.S to be capitalized and amortized over a five-year period. For expenses associated with research outside of the United States, Sec. 174 expenses will be capitalized and amortized over a 15-year period. The Company has included the tax impact of capitalizing and amortizing these costs over the required periods for the nine months ended September 30, 2023. On June 30, 2022, the Company entered into a Royalty Purchase Agreement with Royalty Pharma and a Future Revenue Purchase Agreement with Sixth Street Partners. Pursuant to the agreements, the Company received gross proceeds of $175.0 million from Royalty Pharma in June 2022 and $250.0 million from Sixth Street Partners in July 2022 upon the transactions closing. The total cash consideration of $425.0 million, in its entirety, was considered taxable income for calendar year ended December 31, 2022. Therefore, the $425.0 million was included in the estimated taxable income calculation when estimating the Company’s forecasted 2022 annual effective tax rate. As of September 30, 2022, the Company expected to be in a taxable income position for the calendar year ended December 31, 2022, and recorded an income tax expense of $0.9 million and $4.2 million for the three and nine months ended September 30, 2022, respectively. On August 16, 2022, President Biden signed the Inflation Reduction Act (IRA) of 2022 (H.R. 5376) into law. It includes income tax incentives designed to encourage investment in renewable and alternative energy sources, adoption of electric vehicles, and improvement in the energy efficiency of buildings and communities. To finance these incentives, the law imposes a 15% corporate alternative minimum tax (CAMT) on adjusted financial statement income of corporations with profits over $1 billion. It also introduces a new excise tax on corporate stock buybacks of public US companies. The CAMT is effective for tax years beginning after December 31, 2022 while the excise tax on corporate stock buybacks is effective for repurchases of stock after December 31, 2022. The Company does not anticipate the IRA having a significant impact on the Company’s effective tax rate or income tax payable and deferred income tax positions. As of September 30, 2023, the Company expects to be in a taxable loss position for the calendar year ended December 31, 2023, and has recorded an income tax expense of $0.2 million and $0.9 million for the three and nine months ended September 30, 2023. The Company recorded an income tax provision despite forecasting a taxable loss due to the impact of recording unfavorable discrete items in the period, and taxable income from the jurisdictions in which the Company is subject to tax. As of September 30, 2023, the Company had gross unrecognized tax benefit, excluding interest and penalties of approximately $0.2 million. As of December 31, 2022, the Company did not have any gross unrecognized tax benefit. The company recognizes interest and penalties related to unrecognized tax benefits in the provision for income taxes. As of September 30, 2023, the Company recorded an insignificant amount of interest and penalties related to uncertain tax positions. |
Leases
Leases | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Leases | 14. Leases The Company’s building leases are comprised of office and laboratory spaces under non-cancelable operating leases. The lease agreements contain various clauses for renewal at the Company’s option and only certain exercised renewal options were included in the calculation of the operating lease assets and the operating lease liabilities, as other renewal options were not reasonably certain of being exercised as of September 30, 2023. The lease agreements do not contain residual value guarantees and t Three Months Ended Nine Months Ended September 30, September 30, Operating leases: 2023 2022 2023 2022 Lease cost $ 5,758 $ 5,322 $ 17,301 $ 16,233 Sublease income — (600) — (2,132) Net lease cost $ 5,758 $ 4,722 $ 17,301 $ 14,101 The Company’s sublease agreements for the 38 Sidney Street property expired in 2022. The Company has not entered into any material short-term leases or financing leases as of September 30, 2023. Supplemental cash flow information related to leases for the nine months ended September 30, 2023 and 2022 is as follows (in thousands): Nine Months Ended September 30, 2023 2022 Cash paid for amounts included in the measurement of lease liabilities: $ 13,249 $ 11,668 Lease liabilities arising from obtaining right-of-use assets: Operating leases $ 109 $ — The weighted average remaining lease term and weighted average discount rate of the operating leases are as follows: Operating leases Weighted average remaining lease term in years 6.1 Weighted average discount rate 7.4% |
Commitments and Contingencies
Commitments and Contingencies | 9 Months Ended |
Sep. 30, 2023 | |
Disclosure Text Block | |
Commitments and Contingencies | 15. Commitments and Contingencies Purchase Commitments Associated with Commercial Supply Agreements In connection with the commercialization of AYVAKIT/AYVAKYT, the Company has negotiated manufacturing agreements with certain vendors that require the Company to meet minimum purchase obligations on an annual basis. During the nine months ended September 30, 2023, there was a decrease of $7.3 million in the Company’s contractual obligations described . Legal Proceedings The Company is not currently a party to any material legal proceedings. At each reporting date, the Company evaluates whether or not a potential loss amount or a potential range of loss is probable and reasonably estimable under the provisions of the authoritative guidance that addresses accounting for contingencies. The Company expenses the costs related to its legal proceedings as they are incurred. Indemnification Agreements In the ordinary course of business, the Company may provide indemnification of varying scope and terms to vendors, lessors, business partners, and other parties with respect to certain matters including, but not limited to, losses arising out of breach of such agreements or from intellectual property infringement claims made by third parties. In addition, the Company has entered into indemnification agreements with members of its board of directors and senior management that will require the Company, among other things, to indemnify them against certain liabilities that may arise by reason of their status or service as directors or officers of the Company. The maximum potential amount of future payments the Company could be required to make under these indemnification agreements is, in many cases, unlimited. To date, the Company has not incurred any material costs as a result of such indemnifications. The Company is not aware of any claims under indemnification arrangements, and it has no t accrued any liabilities related to such obligations in its condensed consolidated financial statements as of September 30, 2023 or December 31, 2022. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies and Recent Accounting Pronouncements (Policies) | 9 Months Ended |
Sep. 30, 2023 | |
Policy Text Blocks | |
Basis of Presentation | Basis of Presentation The unaudited interim condensed consolidated financial statements of the Company included herein have been prepared in accordance with accounting principles generally accepted in the United States (GAAP) as found in the Accounting Standards Codification (ASC), Accounting Standards Update (ASU) of the Financial Accounting Standards Board (FASB) and the rules and regulations of the Securities and Exchange Commission (SEC). Certain information and footnote disclosures normally included in financial statements prepared in accordance with GAAP have been condensed or omitted from this report, as is permitted by such rules and regulations. Accordingly, these financial statements should be read in conjunction with the financial statements as of and for the year ended December 31, 2022 and notes thereto included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, filed with the SEC on February 16, 2023 (2022 Annual Report on Form 10-K). The unaudited interim condensed consolidated financial statements have been prepared on the same basis as the audited financial statements, and updated, as necessary, in this report. In the opinion of the Company’s management, the accompanying unaudited interim condensed consolidated financial statements contain all adjustments that are necessary to present fairly the Company’s financial position as of September 30, 2023, the results of its operations for the three and nine months ended September 30, 2023 and 2022, stockholder’s equity for the three and nine months ended September 30, 2023 and 2022 and cash flows for nine months ended September 30, 2023 and 2022. Such adjustments are of a normal and recurring nature. The results for the three and nine months ended September 30, 2023 are not necessarily indicative of the results for the year ending December 31, 2023 or for any future period. The accompanying unaudited interim condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries, Blueprint Medicines Security Corporation, which is a Massachusetts subsidiary created to buy, sell and hold securities, Blueprint Medicines (Switzerland) GmbH, Blueprint Medicines (Netherlands) B.V., Blueprint Medicines (UK) Ltd., Blueprint Medicines (Germany) GmbH, Blueprint Medicines (Spain) S.L., Blueprint Medicines (France) SAS, and Blueprint Medicines (Italy) S.r.L. All intercompany transactions and balances have been eliminated. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies and in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates. Estimates are used in the following areas, among others: revenue recognition, inventory, operating lease right-of-use assets, operating lease liabilities, stock-based compensation expense, accrued expenses, liabilities related to the sale of future royalties and future revenues, equity investment, and income taxes. Actual results may differ from these estimates. |
Significant Accounting Policies | Significant Accounting Policies The significant accounting policies used in preparation of these condensed consolidated financial statements for the three and nine months ended September 30, 2023 are consistent with those discussed in Note 2 to the consolidated financial statements in the 2022 Annual Report on Form 10-K, with the exception of the below new policy. |
Performance-Based Restricted Stock Unit Awards | Performance-Based Restricted Stock Unit Awards |
New Accounting Pronouncements | New Accounting Pronouncements |
Financing Arrangements (Tables)
Financing Arrangements (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Sixth Street Partners, Future Revenue Purchase Agreement | |
Table Text Blocks | |
Schedule of activity within the liability account | Carrying value as of January 1, 2023 $ 254,328 Interest expense recognized 21,043 Payments (10,484) Carrying value as of September 30, 2023 $ 264,887 |
Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities | |
Table Text Blocks | |
Schedule of activity within the liability account | Carrying value as of January 1, 2023 $ 139,083 Net proceeds received from the delayed draw term loan 97,968 Interest expense recognized 15,557 Payments (14,230) Carrying value as of September 30, 2023 $ 238,378 |
Marketable Securities (Tables)
Marketable Securities (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Schedule of marketable securities | Amortized Unrealized Unrealized Fair September 30, 2023 Cost Gain Losses Value Marketable securities, available-for-sale: U.S. government agency securities $ 201,990 — (1,127) $ 200,863 U.S. treasury obligations 571,588 7 (1,204) 570,391 Total $ 773,578 $ 7 $ (2,331) $ 771,254 Amortized Unrealized Unrealized Fair December 31, 2022 Cost Gain Losses Value Marketable securities, available-for-sale: U.S. government agency securities $ 377,519 — (4,848) $ 372,671 U.S. treasury obligations 591,193 22 (5,123) 586,092 Total $ 968,712 $ 22 $ (9,971) $ 958,763 |
Summary of fair value and number of available-for-sale securities in loss positions | September 30, 2023 December 31, 2022 Fair Number of Fair Number of value securities value securities Debt securities in unrealized loss position for 12 months or less $ 624,319 116 $ 371,746 66 Debt securities in unrealized loss position for more than 12 months 56,975 10 550,561 56 Total debt securities in unrealized loss position $ 681,294 126 $ 922,307 122 |
Summary of the fair value and the number of the available-for-sale securities by their maturities | September 30, 2023 December 31, 2022 Amortized Fair Amortized Fair Cost value Cost value Within one year $ 658,760 $ 657,012 $ 834,440 $ 825,283 After one through five years 114,818 114,242 134,272 133,480 Total $ 773,578 $ 771,254 $ 968,712 $ 958,763 |
Summary of proceeds from maturities of debt securities | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Proceeds from maturities of debt securities $ 237,600 $ 90,500 $ 855,365 $ 170,123 |
Fair Value of Financial Instr_2
Fair Value of Financial Instruments (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Schedule of financial instruments measured at fair value | The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of September 30, 2023 (in thousands): Active Observable Unobservable September 30, Markets Inputs Inputs Description 2023 (Level 1) (Level 2) (Level 3) Cash equivalents: Money market funds $ 47,510 47,510 — $ — Marketable securities, available-for-sale: U.S. government agency securities 200,863 — 200,863 — U.S. treasury obligations 570,391 570,391 — — Total $ 818,764 $ 617,901 $ 200,863 $ — The following table summarizes the Company’s cash equivalents and marketable securities measured at fair value on a recurring basis as of December 31, 2022 (in thousands): Active Observable Unobservable December 31, Markets Inputs Inputs Description 2022 (Level 1) (Level 2) (Level 3) Cash equivalents: Money market funds $ 95,198 $ 95,198 $ — $ — U.S. treasury obligations 4,989 4,989 — — Marketable securities, available-for-sale: U.S. government agency securities 372,671 — 372,671 — U.S. treasury obligations 586,092 586,092 — — Total $ 1,058,950 $ 686,279 $ 372,671 $ — |
Product Revenue Reserves and _2
Product Revenue Reserves and Allowances (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Schedules of concentration of risk | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Customer 1 42 % 45 % 42 % 46 % Customer 2 11 % 10 % 11 % 11 % September 30, December 31, 2023 2022 Customer 1 31 % 33 % Customer 2 13 % 15 % Customer 3 11 % * Customer 4 * 12 % |
Product revenue, net | |
Table Text Blocks | |
Summary of revenue recognized | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 United States $ 49,121 $ 25,054 $ 118,355 $ 70,888 Rest of World 5,107 3,580 14,818 10,041 Total product revenue $ 54,228 $ 28,634 $ 133,173 $ 80,929 |
Schedule of product revenue allowance and reserve categories | Nine Months Ended September 30, 2023 2022 Beginning balance at January 1 $ 9,788 $ 4,345 Provision related to sales in the current period 25,874 13,606 Adjustment related to prior periods sales (545) (645) Credits and payments made (17,469) (9,980) Ending balance at September 30 $ 17,648 $ 7,326 September 30, December 31, 2023 2022 Reduction of accounts receivable, net $ 1,547 $ 742 Component of accrued expenses 16,101 9,046 Total revenue-related reserves $ 17,648 $ 9,788 |
Inventory (Tables)
Inventory (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Schedule of capitalized inventory | September 30, December 31, 2023 2022 Raw materials $ 1,599 $ 4,967 Work in process 29,335 28,427 Finished goods 3,475 2,791 Total $ 34,409 $ 36,185 Balance sheet classification September 30, December 31, 2023 2022 Inventory $ 16,457 $ 29,697 Other assets 17,952 6,488 Total $ 34,409 $ 36,185 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Schedule of accrued expenses | September 30, December 31, 2023 2022 Research, development and commercial contract costs $ 38,699 $ 66,041 Employee compensation 34,517 30,744 Accrued professional fees 14,592 17,588 Revenue-related reserves 16,101 9,046 Other 6,527 7,704 Total $ 110,436 $ 131,123 |
Collaboration and License Agr_2
Collaboration and License Agreements (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Schedule of collaboration loss sharing | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 The Company's share of loss in the U.S. for pralsetinib $ 1,771 $ 1,665 $ 4,301 $ 7,076 |
Schedule of amounts recognized as reductions to expenses | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Reductions to selling, general and administrative expenses $ 3,063 $ 3,560 $ 8,618 $ 12,808 Increases in research and development expenses $ 7,603 $ 6,111 $ 21,226 $ 9,815 |
Roche, Collaboration (Pralsetnib) Agreement | |
Table Text Blocks | |
Summary of revenue recognized | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Manufacturing and research and development services related to $ 213 $ 3,971 $ 570 $ 4,663 Royalty revenue 300 311 1,102 910 Total Roche pralsetinib collaboration revenue $ 513 $ 4,282 $ 1,672 $ 5,573 |
Summary of contract assets and/or contract liabilities | September 30, December 31, 2023 2022 Unbilled accounts receivable $ 304 $ — Accrued expenses $ 6,221 $ 6,519 |
C Stone | |
Table Text Blocks | |
Summary of revenue recognized | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 License milestone revenue $ — $ — $ 9,000 $ 4,000 Manufacturing services and royalty revenue related to CStone Territory-specific activities 1,820 2,915 7,861 14,209 Total CStone collaboration revenue $ 1,820 $ 2,915 $ 16,861 $ 18,209 |
C Stone | Collaboration revenue | |
Table Text Blocks | |
Summary of contract assets and/or contract liabilities | September 30, December 31, 2023 2022 Accounts receivable, net $ 5,338 $ 4,991 Unbilled accounts receivable 83 1,529 Contract liability 3,010 2,265 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Summary of stock option activity | Weighted-Average Shares Exercise Price Outstanding at December 31, 2022 6,233,451 $ 69.14 Granted 1,182,245 45.31 Exercised (154,433) 22.02 Canceled (277,649) 70.90 Outstanding at September 30, 2023 6,983,614 $ 66.08 Exercisable at September 30, 2023 4,756,302 $ 68.88 |
Summary of restricted stock units activity | Weighted-Average Shares Grant Date Fair Value Unvested shares at December 31, 2022 1,893,944 $ 73.28 Granted 888,010 44.78 Vested (569,385) 72.64 Forfeited (119,829) 64.97 Unvested shares at September 30, 2023 2,092,740 $ 61.85 |
Summary of performance-based restricted stock units activity | Weighted-Average Grant Date Shares Grant Date Fair Value Unvested shares at December 31, 2022 — $ — Granted 52,500 59.32 Vested — — Forfeited — — Unvested shares at September 30, 2023 52,500 $ 59.32 |
Summary of stock-based compensation expense, allocation by type of awards and recognition in statements of operations | Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Stock options $ 9,998 $ 12,062 $ 31,195 $ 37,819 Restricted stock units 12,536 11,802 37,389 34,583 Performance-based restricted stock units 261 — 605 — Employee stock purchase plan 417 404 1,308 1,000 Subtotal 23,212 24,268 70,497 73,402 Capitalized stock-based compensation costs (141) (159) (410) (547) Stock-based compensation expense included in total cost and operating expenses $ 23,071 $ 24,109 $ 70,087 $ 72,855 Three Months Ended Nine Months Ended September 30, September 30, 2023 2022 2023 2022 Research and development $ 11,169 $ 10,010 $ 31,512 $ 30,548 Selling, general and administrative 11,902 14,099 38,575 42,307 Total $ 23,071 $ 24,109 $ 70,087 $ 72,855 |
Net Loss Per Share (Tables)
Net Loss Per Share (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Schedule of common stock equivalents excluded from calculation of diluted net loss per share applicable to common stockholders | September 30, 2023 2022 Stock options 6,984 6,316 Restricted stock units 2,093 1,838 Performance-based restricted stock units 53 — ESPP shares 47 46 Total 9,177 8,200 |
Leases (Tables)
Leases (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Table Text Blocks | |
Summary of lease expenses and cash flow and weighted average information | Three Months Ended Nine Months Ended September 30, September 30, Operating leases: 2023 2022 2023 2022 Lease cost $ 5,758 $ 5,322 $ 17,301 $ 16,233 Sublease income — (600) — (2,132) Net lease cost $ 5,758 $ 4,722 $ 17,301 $ 14,101 Nine Months Ended September 30, 2023 2022 Cash paid for amounts included in the measurement of lease liabilities: $ 13,249 $ 11,668 Lease liabilities arising from obtaining right-of-use assets: Operating leases $ 109 $ — Operating leases Weighted average remaining lease term in years 6.1 Weighted average discount rate 7.4% |
Nature of Business (Details)
Nature of Business (Details) $ in Millions | Sep. 30, 2023 USD ($) |
Cash, cash equivalents and marketable securities | |
Cash, cash equivalents and marketable securities | $ 827.2 |
Financing Arrangements - Genera
Financing Arrangements - General Information (Details) $ in Thousands | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||
Jun. 30, 2022 USD ($) | Aug. 31, 2023 USD ($) | Jul. 31, 2022 USD ($) tranche Transaction | Sep. 30, 2023 USD ($) | Dec. 31, 2022 USD ($) | |
Sixth Street Partners, Financing Arrangements | |||||
Debt Financing | |||||
Debt instrument, transactions, number | Transaction | 2 | ||||
Sale of Future Royalties and Revenues | |||||
Gross Proceeds | |||||
Gross proceeds | $ 425,000 | ||||
Sale of Future Royalties and Revenues | Royalty Pharma, Royalty Purchase Agreement | |||||
Debt Financing | |||||
Debt instrument, issuance date | Jun. 30, 2022 | ||||
Debt instrument, face amount | $ 175,000 | ||||
Transaction costs, gross | 3,800 | ||||
Debt instrument, interest expense accretion | $ 0 | ||||
Gross Proceeds | |||||
Gross proceeds | $ 175,000 | ||||
Sale of Future Royalties and Revenues | Sixth Street Partners, Future Revenue Purchase Agreement | |||||
Debt Financing | |||||
Debt instrument, face amount | $ 250,000 | ||||
Transaction costs, gross | 5,400 | ||||
Gross Proceeds | |||||
Gross proceeds | 250,000 | ||||
Effective Interest Rate | |||||
Debt instrument, interest rate, effective percentage (as a percent) | 10.80% | ||||
Loans Payable | Sixth Street Partners, Financing Agreement | |||||
Debt Financing | |||||
Debt instrument, face amount, potential amount | 660,000 | ||||
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded | 50,000 | ||||
Debt instrument, covenants, consolidated liquidity, period commencing from date term loan funded to date day before next term loans funded, each day thereafter | $ 80,000 | ||||
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities | |||||
Debt Financing | |||||
Debt instrument, maturity date | Jun. 30, 2028 | ||||
Effective Interest Rate | |||||
Debt instrument, interest rate, effective percentage (as a percent) | 13.30% | ||||
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities | Secured Overnight Financing Rate (SOFR) Overnight Index Swap Rate | |||||
Debt Financing | |||||
Debt instrument, basis spread on variable rate | 6.50% | ||||
Debt instrument, variable rate floor | 1% | ||||
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities | Base Rate | |||||
Debt Financing | |||||
Debt instrument, basis spread on variable rate | 5.50% | ||||
Debt instrument, variable rate floor | 2% | ||||
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facility | |||||
Debt Financing | |||||
Debt instrument, face amount | $ 150,000 | ||||
Debt discounts and transaction costs | 12,200 | ||||
Gross Proceeds | |||||
Gross proceeds | 150,000 | ||||
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Delayed Draw Term Loan Facility | |||||
Debt Financing | |||||
Debt instrument, face amount, potential amount | $ 250,000 | ||||
Debt instrument, tranches, number | tranche | 2 | ||||
Transaction costs, gross | $ 2,000 | ||||
Gross Proceeds | |||||
Gross proceeds | $ 100,000 | ||||
Secured Debt | Sixth Street Partners, Financing Agreement, Incremental Term Loan | |||||
Debt Financing | |||||
Debt instrument, face amount, potential amount | $ 260,000 |
Financing Arrangements - Royalt
Financing Arrangements - Royalty and Sales Information (Details) - Sixth Street Partners $ in Millions | Jul. 31, 2022 USD ($) |
Financing Arrangements | |
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, percentage rate (as a percent) | 9.75% |
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, amount, maximum | $ 900 |
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, ratio | 1.45 |
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, amount | $ 362.5 |
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, percentage increase (as a percent) | 15% |
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, ratio | 1.85 |
Purchase and sale agreement, royalties, future royalty payments, annual worldwide net product sales, cumulative cap of upfront invested capital, revenue targets not achieved, amount | $ 462.5 |
Financing Arrangements - Long-t
Financing Arrangements - Long-term Debt (Details) - Sale of Future Royalties and Revenues - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Royalty Pharma, Royalty Purchase Agreement | ||
Long-term Debt | ||
Long-term debt | $ 175,300 | |
Sixth Street Partners, Future Revenue Purchase Agreement | ||
Long-term Debt | ||
Long-term debt | $ 264,887 | $ 254,328 |
Financing Arrangements - Roll F
Financing Arrangements - Roll Forward (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
Sale of Future Royalties and Revenues | Royalty Pharma, Royalty Purchase Agreement | |
Long-term Debt | |
Carrying value, ending balance | $ 175,300 |
Sale of Future Royalties and Revenues | Sixth Street Partners, Future Revenue Purchase Agreement | |
Long-term Debt | |
Carrying value, beginning balance | 254,328 |
Interest expense recognized | 21,043 |
Payments | (10,484) |
Carrying value, ending balance | 264,887 |
Secured Debt | Sixth Street Partners, Financing Agreement, Senior Secured Term Loan Facilities | |
Long-term Debt | |
Carrying value, beginning balance | 139,083 |
Net proceeds received | 97,968 |
Interest expense recognized | 15,557 |
Payments | (14,230) |
Carrying value, ending balance | $ 238,378 |
Financing Arrangements - Additi
Financing Arrangements - Additional Information (Details) $ in Millions | Jun. 30, 2022 USD ($) |
Debt Financing | |
Royalty Purchase Agreement, milestone payments, achievement of specified net sales milestones, maximum | $ 165 |
Marketable Securities - Tabular
Marketable Securities - Tabular Disclosure (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Marketable securities, available-for-sale: | ||
Amortized cost | $ 773,578 | $ 968,712 |
Unrealized gain | 7 | 22 |
Unrealized losses | (2,331) | (9,971) |
Fair value | 771,254 | 958,763 |
U.S. government agency securities | ||
Marketable securities, available-for-sale: | ||
Amortized cost | 201,990 | 377,519 |
Unrealized losses | (1,127) | (4,848) |
Fair value | 200,863 | 372,671 |
U.S. Treasury obligations | ||
Marketable securities, available-for-sale: | ||
Amortized cost | 571,588 | 591,193 |
Unrealized gain | 7 | 22 |
Unrealized losses | (1,204) | (5,123) |
Fair value | $ 570,391 | $ 586,092 |
Marketable Securities - Unreali
Marketable Securities - Unrealized Loss Positions (Details) $ in Thousands | Sep. 30, 2023 USD ($) security | Dec. 31, 2022 USD ($) security |
Unrealized loss position, aggregate fair value | ||
Unrealized loss position for less than 12 months, aggregate fair value | $ | $ 624,319 | $ 371,746 |
Unrealized loss position for more than 12 months, aggregate fair value | $ | 56,975 | 550,561 |
Unrealized loss position, aggregate fair value | $ | $ 681,294 | $ 922,307 |
Unrealized loss position, number of positions | ||
Number of held securities in an unrealized loss position for less than 12 months | security | 116 | 66 |
Number of held securities in an unrealized loss position for 12 months or longer | security | 10 | 56 |
Number of held securities in an unrealized loss position | security | 126 | 122 |
Marketable Securities - Impairm
Marketable Securities - Impairment (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Marketable Securities | ||||
Marketable debt securities, charges for credit-related impairments | $ 0 | $ 0 | $ 0 | $ 0 |
Marketable Securities - Maturit
Marketable Securities - Maturities (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Amortized cost | ||
Within one year, amortized cost | $ 658,760 | $ 834,440 |
After one through five years, amortized cost | 114,818 | 134,272 |
Amortized cost | 773,578 | 968,712 |
Fair value | ||
Within one year, fair value | 657,012 | 825,283 |
After one through five years, fair value | 114,242 | 133,480 |
Investments, available-for-sale | $ 771,254 | $ 958,763 |
Marketable Securities - Proceed
Marketable Securities - Proceeds from Maturities (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Proceeds from the maturities of debt securities | ||||
Proceeds from the maturities of debt securities | $ 237,600 | $ 90,500 | $ 855,365 | $ 170,123 |
Marketable Securities - Realize
Marketable Securities - Realized Gains (Losses) (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
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 | Sep. 30, 2023 | Dec. 31, 2022 |
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | $ 771,254 | $ 958,763 |
U.S. government agency securities | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 200,863 | 372,671 |
U.S. Treasury obligations | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 570,391 | 586,092 |
Recurring | ||
Fair Value of Financial Instruments | ||
Total | 818,764 | 1,058,950 |
Recurring | U.S. government agency securities | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 200,863 | 372,671 |
Recurring | U.S. Treasury obligations | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 570,391 | 586,092 |
Recurring | Money market funds | ||
Fair Value of Financial Instruments | ||
Cash equivalents | 47,510 | 95,198 |
Recurring | U.S. Treasury obligations | ||
Fair Value of Financial Instruments | ||
Cash equivalents | 4,989 | |
Recurring | Active Markets (Level 1) | ||
Fair Value of Financial Instruments | ||
Total | 617,901 | 686,279 |
Recurring | Active Markets (Level 1) | U.S. Treasury obligations | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | 570,391 | 586,092 |
Recurring | Active Markets (Level 1) | Money market funds | ||
Fair Value of Financial Instruments | ||
Cash equivalents | 47,510 | 95,198 |
Recurring | Active Markets (Level 1) | U.S. Treasury obligations | ||
Fair Value of Financial Instruments | ||
Cash equivalents | 4,989 | |
Recurring | Observable Inputs (Level 2) | ||
Fair Value of Financial Instruments | ||
Total | 200,863 | 372,671 |
Recurring | Observable Inputs (Level 2) | U.S. government agency securities | ||
Fair Value of Financial Instruments | ||
Marketable securities, available-for-sale | $ 200,863 | $ 372,671 |
Product Revenue Reserves and _3
Product Revenue Reserves and Allowances - Product Revenue (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Revenues | ||||
Revenue | $ 56,566 | $ 65,977 | $ 177,423 | $ 165,255 |
Product revenue, net | ||||
Revenues | ||||
Revenue | 54,228 | 28,634 | 133,173 | 80,929 |
AYVAKIT and AYVAKYT | ||||
Revenues | ||||
Revenue | 49,121 | 25,054 | 118,355 | 70,888 |
GAVRETO | ||||
Revenues | ||||
Revenue | $ 5,107 | $ 3,580 | $ 14,818 | $ 10,041 |
Product Revenue Reserves and _4
Product Revenue Reserves and Allowances - Concentration Risk (Details) | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
Revenue from Contract with Customer Benchmark | Customer Concentration Risk | Customer One | |||||
Concentration Risk | |||||
Concentration risk (as a percent) | 42% | 45% | 42% | 46% | |
Revenue from Contract with Customer Benchmark | Customer Concentration Risk | Customer Two | |||||
Concentration Risk | |||||
Concentration risk (as a percent) | 11% | 10% | 11% | 11% | |
Accounts Receivable, Net | Credit Concentration Risk | Customer One | |||||
Concentration Risk | |||||
Concentration risk (as a percent) | 31% | 33% | |||
Accounts Receivable, Net | Credit Concentration Risk | Customer Two | |||||
Concentration Risk | |||||
Concentration risk (as a percent) | 13% | 15% | |||
Accounts Receivable, Net | Credit Concentration Risk | Customer Three | |||||
Concentration Risk | |||||
Concentration risk (as a percent) | 11% | ||||
Accounts Receivable, Net | Credit Concentration Risk | Customer Three | Maximum | |||||
Concentration Risk | |||||
Concentration risk (as a percent) | 10% | ||||
Accounts Receivable, Net | Credit Concentration Risk | Customer Four | |||||
Concentration Risk | |||||
Concentration risk (as a percent) | 12% | ||||
Accounts Receivable, Net | Credit Concentration Risk | Customer Four | Maximum | |||||
Concentration Risk | |||||
Concentration risk (as a percent) | 10% |
Product Revenue Reserves and _5
Product Revenue Reserves and Allowances - Product Revenue Allowance and Reserve (Details) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Product revenue allowance and reserve | ||
Beginning balance | $ 9,788 | $ 4,345 |
Provision related to sales in the current period | 25,874 | 13,606 |
Adjustment related to prior periods sales | (545) | (645) |
Credits and payments made | (17,469) | (9,980) |
Ending balance | $ 17,648 | $ 7,326 |
Product Revenue Reserves and _6
Product Revenue Reserves and Allowances - Revenue-related Reserves (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | Dec. 31, 2021 |
Revenue-related reserves | ||||
Revenue-related reserves | $ 17,648 | $ 9,788 | $ 7,326 | $ 4,345 |
Accounts Receivable, Net | ||||
Revenue-related reserves | ||||
Revenue-related reserves | 1,547 | 742 | ||
Accrued Expenses | ||||
Revenue-related reserves | ||||
Revenue-related reserves | $ 16,101 | $ 9,046 |
Inventory - Capitalized (Detail
Inventory - Capitalized (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Inventory | ||
Raw materials | $ 1,599 | $ 4,967 |
Work in process | 29,335 | 28,427 |
Finished goods | 3,475 | 2,791 |
Total | $ 34,409 | $ 36,185 |
Inventory - Balance Sheet Class
Inventory - Balance Sheet Classification (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Inventory | ||
Inventory | $ 16,457 | $ 29,697 |
Other assets | 17,952 | 6,488 |
Total | $ 34,409 | $ 36,185 |
Inventory - Write-down (Details
Inventory - Write-down (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Inventory | ||||
Inventory write-down | $ 100 | $ 0 | $ 2,200 | $ 0 |
Restricted Cash (Details)
Restricted Cash (Details) - USD ($) $ in Millions | Sep. 30, 2023 | Dec. 31, 2022 |
Restricted cash | ||
Restricted cash | $ 9.7 | $ 5.2 |
Accrued Expenses (Details)
Accrued Expenses (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Accrued Expenses | ||
External research and development | $ 38,699 | $ 66,041 |
Employee compensation | 34,517 | 30,744 |
Accrued professional fees | 14,592 | 17,588 |
Revenue-related reserves | 16,101 | 9,046 |
Other | 6,527 | 7,704 |
Total | $ 110,436 | $ 131,123 |
Collaboration and License Agr_3
Collaboration and License Agreements - General Information (Details) $ in Thousands | 1 Months Ended | 2 Months Ended | 3 Months Ended | 9 Months Ended | 45 Months Ended | 86 Months Ended | ||||||||||
Feb. 26, 2022 USD ($) item Program | Oct. 15, 2019 USD ($) | Jun. 01, 2018 USD ($) item | Oct. 31, 2019 USD ($) | Dec. 31, 2021 USD ($) | Sep. 30, 2023 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2020 USD ($) | Sep. 30, 2023 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2023 USD ($) | Apr. 30, 2023 Program | Dec. 31, 2022 USD ($) | Nov. 08, 2021 USD ($) | Jul. 13, 2020 | Mar. 31, 2016 Program Right | |
Collaboration Agreements | ||||||||||||||||
Prepaid expenses and other current assets | $ 37,477 | $ 37,477 | $ 37,477 | $ 35,024 | ||||||||||||
Research and development expense | 110,252 | $ 127,981 | 330,184 | $ 359,579 | ||||||||||||
Collaborative Arrangement | IDRx, Inc. | Director | ||||||||||||||||
Collaboration Agreements | ||||||||||||||||
Collaborative arrangement, milestones, potential contingent payments | 217,500 | $ 217,500 | 217,500 | |||||||||||||
Collaborative arrangement, termination, counterparty written notice period | 12 months | |||||||||||||||
Collaborative Arrangement | Proteovant | ||||||||||||||||
Collaboration Agreements | ||||||||||||||||
Collaborative arrangement, upfront payment | $ 20,000 | |||||||||||||||
Collaborative arrangement, milestones, potential contingent payments | 632,000 | |||||||||||||||
Prepaid expenses and other current assets | $ 20,000 | |||||||||||||||
Research and development expense | 600 | $ 1,400 | $ 6,200 | $ 2,900 | ||||||||||||
Collaborative arrangement, jointly research and advance novel protein degrader therapies into development candidates, number | item | 2 | |||||||||||||||
Collaborative arrangement, jointly research and advance additional novel protein degrader target programs, number | Program | 2 | |||||||||||||||
Collaborative Arrangement | Zai Lab (Shanghai) Co., Ltd. | ||||||||||||||||
Collaboration Agreements | ||||||||||||||||
Collaborative arrangement, upfront payment, cash payment received | $ 25,000 | |||||||||||||||
Collaborative arrangement, milestones, contingent payments eligible to receive | $ 590,000 | |||||||||||||||
Collaborative arrangement, termination, counterparty written notice period, period from effective date | 2 years | |||||||||||||||
Collaborative arrangement, termination, counterparty written notice period, period from effective date, given after first commercial sale of licensed product in counterparty territory | 12 months | |||||||||||||||
Collaborative arrangement, termination, counterparty written notice period, period from effective date, given prior to first commercial sale of licensed product in counterparty territory | 9 months | |||||||||||||||
Collaborative Arrangement | Roche, Collaboration (Pralsetnib) Agreement | ||||||||||||||||
Collaboration Agreements | ||||||||||||||||
Collaborative arrangement, upfront payment, cash payment received | $ 675,000 | |||||||||||||||
Collaborative arrangement, milestones, specified regulatory and commercialization milestones, achieved | $ 105,000 | 105,000 | ||||||||||||||
Collaborative arrangement, percentage of global development costs shared, entity (as a percent) | 45% | |||||||||||||||
Collaborative arrangement, percentage of global development costs shared, counterparty (as a percent) | 55% | |||||||||||||||
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement | ||||||||||||||||
Collaboration Agreements | ||||||||||||||||
Collaborative arrangement, option rights, maximum | Right | 5 | |||||||||||||||
Collaborative arrangement, development programs, terminated | Program | 4 | |||||||||||||||
Collaborative arrangement, development programs | Program | 5 | |||||||||||||||
Collaborative Arrangement | Clementia | ||||||||||||||||
Collaboration Agreements | ||||||||||||||||
Collaborative arrangement, upfront payment, cash payment received | $ 25,000 | |||||||||||||||
Collaborative arrangement, milestones, cash payments received | $ 50,000 | |||||||||||||||
Collaborative arrangement, milestones, contingent payments eligible to receive | 460,000 | |||||||||||||||
Collaborative arrangement, inventory purchased by counterparty | $ 1,500 | |||||||||||||||
Collaborative arrangement, termination, counterparty written notice period | 12 months | |||||||||||||||
Collaborative Arrangement | C Stone | ||||||||||||||||
Collaboration Agreements | ||||||||||||||||
Collaborative arrangement, upfront payment, cash payment received | $ 40,000 | |||||||||||||||
Collaborative arrangement, milestones, contingent payments eligible to receive | $ 307,500 | |||||||||||||||
Collaborative arrangement, milestones, achieved | $ 38,500 | $ 38,500 | $ 38,500 | |||||||||||||
Collaborative arrangement, licensed product term from first commercial sale | 12 years | |||||||||||||||
Collaborative arrangement, license option rights, number | item | 3 | |||||||||||||||
Collaborative arrangement, collaboration programs with exclusive commercialization rights, number | item | 3 |
Collaboration and License Agr_4
Collaboration and License Agreements - Transaction Price (Details) - Collaborative Arrangement - USD ($) $ in Millions | 2 Months Ended | 9 Months Ended | |||
Jul. 13, 2020 | Oct. 15, 2019 | Jun. 01, 2018 | Dec. 31, 2021 | Sep. 30, 2023 | |
Zai Lab (Shanghai) Co., Ltd. | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 25 | ||||
Roche, Collaboration (Pralsetnib) Agreement | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 695.7 | ||||
Collaborative arrangement, transaction price, upfront payment | 675 | ||||
Collaborative arrangement, transaction price, stock issued, premium on sale of stock to counterparty | $ 20.7 | ||||
Roche, Collaboration and License (Immunotherapy) Agreement | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 64.7 | ||||
Collaborative arrangement, transaction price, upfront payment | 45 | ||||
Collaborative arrangement, transaction price, aggregate research milestone achieved | 25 | ||||
Collaborative arrangement, transaction price, reduction, payment for certain clinical development costs | $ 5.3 | ||||
Clementia | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 46.5 | ||||
C Stone | |||||
Collaboration Agreements | |||||
Collaborative arrangement, transaction price | $ 40 |
Collaboration and License Agr_5
Collaboration and License Agreements - Equity Investment (Details) - USD ($) $ in Thousands | 1 Months Ended | |||
Aug. 01, 2022 | Jul. 31, 2023 | Sep. 30, 2023 | Dec. 31, 2022 | |
Equity Investment | ||||
Equity investment | $ 27,789 | $ 27,789 | ||
Collaborative Arrangement | IDRx, Inc. | Director | ||||
Equity Investment | ||||
Equity investment, license agreement, shares received (in shares) | 4,509,105 | 192,282 | ||
Equity investment | $ 27,800 | $ 27,800 | ||
Equity investment, transaction costs | $ 300 |
Collaboration and License Agr_6
Collaboration and License Agreements - Sale of Stock (Details) - Private Placement $ / shares in Units, $ in Millions | Jul. 13, 2020 USD ($) $ / shares shares |
Sale of Stock | |
Stock issued (in shares) | shares | 1,035,519 |
Share price (in dollars per share) | $ / shares | $ 96.57 |
Purchase consideration | $ 100 |
Issuance of common stock | 79.3 |
Share purchase consideration, premium | $ 20.7 |
Collaboration and License Agr_7
Collaboration and License Agreements - Revenue Recognized (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | 12 Months Ended | 24 Months Ended | ||||
Aug. 01, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2021 | Dec. 31, 2018 | Dec. 31, 2020 | |
Revenues | ||||||||
Revenue | $ 56,566 | $ 65,977 | $ 177,423 | $ 165,255 | ||||
License | Related Party | ||||||||
Revenues | ||||||||
Revenue | 27,500 | 27,500 | ||||||
License | IDRx, Inc. | Director | ||||||||
Revenues | ||||||||
Revenue | $ 27,500 | |||||||
Collaborative Arrangement | Zai Lab (Shanghai) Co., Ltd. | ||||||||
Revenues | ||||||||
Revenue | $ 25,000 | |||||||
Collaborative Arrangement | Clementia | ||||||||
Revenues | ||||||||
Revenue | 0 | 30,000 | $ 46,500 | |||||
Collaborative Arrangement | Collaboration revenue | Roche, Collaboration (Pralsetnib) Agreement | ||||||||
Revenues | ||||||||
Revenue | 513 | 4,282 | 1,672 | 5,573 | ||||
Collaborative Arrangement | Collaboration revenue | Roche, Collaboration and License (Immunotherapy) Agreement | ||||||||
Revenues | ||||||||
Revenue | 0 | 2,500 | 25,700 | 1,700 | ||||
Cumulative revenue catch-up | 9,700 | |||||||
Collaborative Arrangement | Collaboration revenue | C Stone | ||||||||
Revenues | ||||||||
Revenue | 1,820 | 2,915 | 16,861 | 18,209 | $ 40,000 | |||
Collaborative Arrangement | License milestone revenue | C Stone | ||||||||
Revenues | ||||||||
Revenue | 0 | 0 | 9,000 | 4,000 | ||||
Collaborative Arrangement | Territory-specific activities | Zai Lab (Shanghai) Co., Ltd. | ||||||||
Revenues | ||||||||
Revenue | 200 | 800 | ||||||
Collaborative Arrangement | Territory-specific activities, manufacturing services and royalty | C Stone | ||||||||
Revenues | ||||||||
Revenue | 1,820 | 2,915 | 7,861 | 14,209 | ||||
Collaborative Arrangement | Territory-specific activities, manufacturing and research and development services | Roche, Collaboration (Pralsetnib) Agreement | ||||||||
Revenues | ||||||||
Revenue | 213 | 3,971 | 570 | 4,663 | ||||
Collaborative Arrangement | Royalty | Roche, Collaboration (Pralsetnib) Agreement | ||||||||
Revenues | ||||||||
Revenue | $ 300 | $ 311 | $ 1,102 | $ 910 |
Collaboration and License Agr_8
Collaboration and License Agreements - Collaboration Loss Sharing (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Collaboration Agreements | ||||
Collaboration loss sharing | $ 1,771 | $ 1,665 | $ 4,301 | $ 7,076 |
Collaborative Arrangement | Roche, Collaboration (Pralsetnib) Agreement | ||||
Collaboration Agreements | ||||
Collaboration loss sharing | $ 1,771 | $ 1,665 | $ 4,301 | $ 7,076 |
Collaboration and License Agr_9
Collaboration and License Agreements - Reduction in Expenses (Details) - Collaborative Arrangement - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Zai Lab (Shanghai) Co., Ltd. | ||||
Collaboration Agreements | ||||
Collaborative arrangement, reduction in expenses | $ 0 | $ 200 | $ 0 | $ 500 |
Roche, Collaboration (Pralsetnib) Agreement | ||||
Collaboration Agreements | ||||
Collaborative arrangement, commercialization, reduction in selling, general and administrative expenses | 3,063 | 3,560 | 8,618 | 12,808 |
Collaborative arrangement, global development activities, increase (reduction) in research and development expenses | $ 7,603 | 6,111 | $ 21,226 | 9,815 |
C Stone | ||||
Collaboration Agreements | ||||
Collaborative arrangement, reduction in manufacturing and research and development expenses | $ 500 | $ 1,600 |
Collaboration and License Ag_10
Collaboration and License Agreements - Change in Contract with Customer, Liability (Details) - USD ($) $ in Millions | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement | |||
Change in Contract with Customer, Liability | |||
Amounts included in the contact liability at the beginning of the period | $ 1.4 | $ 16 | $ 3.8 |
Collaboration and License Ag_11
Collaboration and License Agreements - Contract Assets and Liabilities (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Contract Assets | ||
Accounts receivable, net | $ 40,861 | $ 23,525 |
Unbilled accounts receivable | 393 | 13,413 |
Contract Liabilities | ||
Contract liabilities, current | 3,010 | 4,667 |
Contract liabilities, noncurrent | 5,000 | 13,624 |
Accrued expenses, current | 38,699 | 66,041 |
Collaborative Arrangement | Roche, Collaboration (Pralsetnib) Agreement | ||
Contract Assets | ||
Unbilled accounts receivable | 304 | |
Contract Liabilities | ||
Accrued expenses | 6,221 | 6,519 |
Collaborative Arrangement | Roche, Collaboration and License (Immunotherapy) Agreement | ||
Contract Liabilities | ||
Contract liabilities | 0 | 16,000 |
Contract liabilities, current | 2,400 | |
Accrued expenses | 0 | 11,200 |
Accrued expenses, current | 4,000 | |
Collaborative Arrangement | Clementia | ||
Contract Liabilities | ||
Contract liabilities | 0 | 0 |
Collaborative Arrangement | C Stone | ||
Contract Assets | ||
Accounts receivable, net | 5,338 | 4,991 |
Unbilled accounts receivable | 83 | 1,529 |
Contract Liabilities | ||
Contract liabilities | $ 3,010 | $ 2,265 |
Collaboration and License Ag_12
Collaboration and License Agreements - Performance Obligations (Details) - Collaborative Arrangement | 9 Months Ended |
Sep. 30, 2023 component item | |
IDRx, Inc. | Director | |
Performance Obligations | |
Collaborative arrangement, material promises, number | 2 |
Collaborative arrangement, material promises, combined into distinct performance obligation, number | 1 |
Zai Lab (Shanghai) Co., Ltd. | |
Performance Obligations | |
Collaborative arrangement, material promises, number | 3 |
Collaborative arrangement, material promises, combined into distinct performance obligation, number | 2 |
Collaborative arrangement, material components, number | component | 2 |
Roche, Collaboration (Pralsetnib) Agreement | |
Performance Obligations | |
Collaborative arrangement, material promises, number | 1 |
Collaborative arrangement, material promises, manufacturing activities, term | 24 months |
Collaborative arrangement, material components, number | component | 4 |
Roche, Collaboration and License (Immunotherapy) Agreement | |
Performance Obligations | |
Revenue, performance obligation, performance obligations, number | 1 |
Clementia | |
Performance Obligations | |
Collaborative arrangement, material promises, number | 4 |
Revenue, performance obligation, performance obligations, number | 3 |
C Stone | |
Performance Obligations | |
Collaborative arrangement, material promises, number | 6 |
Collaborative arrangement, material components, number | component | 2 |
Revenue, performance obligation, performance obligations, number | 3 |
Stock-based Compensation - 2015
Stock-based Compensation - 2015 Stock Option and Incentive Plan (Details) - 2015 Stock Option and Incentive Plan - shares | 9 Months Ended | ||
Jan. 01, 2023 | Sep. 30, 2023 | Apr. 08, 2015 | |
Stock-based compensation | |||
Initial shares of common stock authorized for issuance of stock awards (in shares) | 1,460,084 | ||
Increase in number of shares available for grant (as a percent) | 4% | ||
Increase in number of shares available for grant (in shares) | 2,398,356 | ||
Number of shares available for grant (in shares) | 4,896,334 |
Stock-based Compensation - 2020
Stock-based Compensation - 2020 Inducement Plan (Details) - 2020 Inducement Plan - shares | 1 Months Ended | ||
Jun. 30, 2022 | Sep. 30, 2023 | Mar. 31, 2020 | |
Stock-based compensation | |||
Initial shares of common stock authorized for issuance of stock awards (in shares) | 1,000,000 | ||
Increase in number of shares available for grant (in shares) | 1,500,000 | ||
Number of shares available for grant (in shares) | 1,366,923 |
Stock-based Compensation - Stoc
Stock-based Compensation - Stock Options - Activity (Details) | 9 Months Ended |
Sep. 30, 2023 $ / shares shares | |
Shares | |
Outstanding at beginning of period (in shares) | shares | 6,233,451 |
Granted (in shares) | shares | 1,182,245 |
Exercised (in shares) | shares | (154,433) |
Cancelled (in shares) | shares | (277,649) |
Outstanding at end of period (in shares) | shares | 6,983,614 |
Weighted-Average Exercise Price | |
Outstanding at beginning of period (in dollars per share) | $ / shares | $ 69.14 |
Granted (in dollars per share) | $ / shares | 45.31 |
Exercised (in dollars per share) | $ / shares | 22.02 |
Cancelled (in dollars per share) | $ / shares | 70.90 |
Outstanding at end of period (in dollars per share) | $ / shares | $ 66.08 |
Additional disclosures | |
Shares - Exercisable (in shares) | shares | 4,756,302 |
Weighted-Average Exercise Price - Exercisable (in dollars per share) | $ / shares | $ 68.88 |
Stock-based Compensation - Perf
Stock-based Compensation - Performance-based Restricted Stock Units (Details) - Performance-based restricted stock units | 9 Months Ended |
Sep. 30, 2023 | |
Stock-based compensation | |
Award service period | 3 years |
Award vesting period | 3 years |
Performance metric, cumulative relative total shareholder return, period | 3 years |
Minimum | |
Stock-based compensation | |
Award grants, percentage of target (as a percent) | 0% |
Maximum | |
Stock-based compensation | |
Award grants, percentage of target (as a percent) | 200% |
Stock-based Compensation - Unve
Stock-based Compensation - Unvested Awards - Activity (Details) | 9 Months Ended |
Sep. 30, 2023 $ / shares shares | |
Restricted Stock Units | |
Shares | |
Unvested at beginning of period (in shares) | shares | 1,893,944 |
Granted (in shares) | shares | 888,010 |
Vested (in shares) | shares | (569,385) |
Forfeited (in shares) | shares | (119,829) |
Unvested at end of period (in shares) | shares | 2,092,740 |
Weighted-Average Grant Date Fair Value | |
Unvested at beginning or period (in dollars per share) | $ / shares | $ 73.28 |
Granted (in dollars per share) | $ / shares | 44.78 |
Vested (in dollars per share) | $ / shares | 72.64 |
Forfeited (in dollars per share) | $ / shares | 64.97 |
Unvested at end of period (in dollars per share) | $ / shares | $ 61.85 |
Performance-based restricted stock units | |
Shares | |
Unvested at beginning of period (in shares) | shares | 0 |
Granted (in shares) | shares | 52,500 |
Unvested at end of period (in shares) | shares | 52,500 |
Weighted-Average Grant Date Fair Value | |
Unvested at beginning or period (in dollars per share) | $ / shares | $ 0 |
Granted (in dollars per share) | $ / shares | 59.32 |
Unvested at end of period (in dollars per share) | $ / shares | $ 59.32 |
Stock-based Compensation - Unre
Stock-based Compensation - Unrecognized Compensation Costs (Details) $ in Millions | Sep. 30, 2023 USD ($) |
Stock-based compensation | |
Total unrecognized compensation cost related to non-vested stock option awards | $ 69.2 |
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 | $ 99 |
Weighted-average period over which unrecognized compensation cost will be recognized | 2 years 6 months 18 days |
Performance-based restricted stock units | |
Stock-based compensation | |
Total unrecognized compensation cost related to non-vested stock awards | $ 2.5 |
Weighted-average period over which unrecognized compensation cost will be recognized | 2 years 5 months 1 day |
Stock-based Compensation - Empl
Stock-based Compensation - Employee Stock Purchase Plan (Details) - Employee Stock - shares | 1 Months Ended | |
May 31, 2015 | Jan. 01, 2023 | |
Stock-based compensation | ||
Number of common shares reserved for future issuance (in shares) | 243,347 | |
Annual increase for common stock for issuance (as a percent) | 1% | |
Increase of common shares reserved for future issuance (in shares) | 599,589 |
Stock-based Compensation - St_2
Stock-based Compensation - Stock-based Compensation Expense - General Information (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Total stock based compensation expense | ||||
Stock-based compensation expense | $ 23,071 | $ 24,109 | $ 70,087 | $ 72,855 |
Stock-based Compensation - St_3
Stock-based Compensation - Stock-based Compensation Expense - By Type (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Total stock based compensation expense | ||||
Subtotal | $ 23,212 | $ 24,268 | $ 70,497 | $ 73,402 |
Capitalized stock-based compensation costs | (141) | (159) | (410) | (547) |
Stock-based compensation expense included in total cost and operating expenses | 23,071 | 24,109 | 70,087 | 72,855 |
Employee Stock Option | ||||
Total stock based compensation expense | ||||
Subtotal | 9,998 | 12,062 | 31,195 | 37,819 |
Restricted Stock Units | ||||
Total stock based compensation expense | ||||
Subtotal | 12,536 | 11,802 | 37,389 | 34,583 |
Performance-based restricted stock units | ||||
Total stock based compensation expense | ||||
Subtotal | 261 | 605 | ||
Employee Stock | ||||
Total stock based compensation expense | ||||
Subtotal | $ 417 | $ 404 | $ 1,308 | $ 1,000 |
Stock-based Compensation - St_4
Stock-based Compensation - Stock-based Compensation Expense - By Classification (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Total stock based compensation expense | ||||
Stock-based compensation expense | $ 23,071 | $ 24,109 | $ 70,087 | $ 72,855 |
Research and Development | ||||
Total stock based compensation expense | ||||
Stock-based compensation expense | 11,169 | 10,010 | 31,512 | 30,548 |
General and Administrative Expense | ||||
Total stock based compensation expense | ||||
Stock-based compensation expense | $ 11,902 | $ 14,099 | $ 38,575 | $ 42,307 |
Net Loss Per Share (Details)
Net Loss Per Share (Details) - shares shares in Thousands | 3 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 9,177 | 8,200 |
Employee Stock Option | ||
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 6,984 | 6,316 |
Restricted Stock Units | ||
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 2,093 | 1,838 |
Performance-based restricted stock units | ||
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 53 | |
Employee Stock | ||
Antidilutive securities excluded from computation of earnings per share | ||
Antidilutive securities excluded from computation of earnings per share | 47 | 46 |
Income Taxes - Financing Arrang
Income Taxes - Financing Arrangements (Details) - Sale of Future Royalties and Revenues - USD ($) $ in Millions | 1 Months Ended | 12 Months Ended | |
Jun. 30, 2022 | Jul. 31, 2022 | Dec. 31, 2022 | |
Gross Proceeds | |||
Gross proceeds | $ 425 | ||
Royalty Pharma, Royalty Purchase Agreement | |||
Gross Proceeds | |||
Gross proceeds | $ 175 | ||
Sixth Street Partners, Future Revenue Purchase Agreement | |||
Gross Proceeds | |||
Gross proceeds | $ 250 |
Income Taxes - Expense (Benefit
Income Taxes - Expense (Benefit) (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Income tax expense (benefit) | ||||
Income tax expense | $ 197 | $ 886 | $ 907 | $ 4,200 |
Income Taxes - Unrecognized Tax
Income Taxes - Unrecognized Tax Benefits (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Unrecognized tax benefits | ||
Unrecognized tax benefits | $ 200 | $ 0 |
Leases - Components of Lease Ex
Leases - Components of Lease Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Operating leases: | ||||
Lease cost | $ 5,758 | $ 5,322 | $ 17,301 | $ 16,233 |
Sublease income | (600) | (2,132) | ||
Net lease cost | $ 5,758 | $ 4,722 | $ 17,301 | $ 14,101 |
Leases - Cash Flow Information
Leases - Cash Flow Information (Details) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Leases | ||
Cash paid for amounts included in the measurement of lease liabilities, operating cash flows from operating leases | $ 13,249 | $ 11,668 |
Lease liabilities arising from obtaining right-of-use assets, operating leases | $ 109 |
Leases - Weighted Average Remai
Leases - Weighted Average Remaining Lease-term and Weighted Average Discount Rate (Details) | Sep. 30, 2023 |
Leases | |
Weighted average remaining lease term in years | 6 years 1 month 6 days |
Weighted average discount rate | 7.40% |
Commitments and Contingencies -
Commitments and Contingencies - Purchase Commitments (Details) $ in Millions | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
Manufacturing Agreement, October 2020 | |
Manufacturing Agreements | |
Unrecorded unconditional purchase obligation, period increase (decrease) | $ (7.3) |
Commitments and Contingencies_2
Commitments and Contingencies - Indemnification Agreements (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Indemnification Agreement | ||
Indemnification Agreements | ||
Loss contingency accrual | $ 0 | $ 0 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||||||
Sep. 30, 2023 | Jun. 30, 2023 | Mar. 31, 2023 | Sep. 30, 2022 | Jun. 30, 2022 | Mar. 31, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Pay vs Performance Disclosure | ||||||||
Net Income (Loss) | $ (133,713) | $ (132,794) | $ (129,560) | $ (133,163) | $ (159,709) | $ (105,999) | $ (396,068) | $ (398,871) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended | 9 Months Ended |
Sep. 30, 2023 shares | Sep. 30, 2023 shares | |
Trading Arrangements, by Individual | ||
Material Terms of Trading Arrangement | Name and Title Action Taken Type of Trading Arrangement Nature of Trading Arrangement Duration of Trading Arrangement Aggregate Number of Securities Ariel Hurley ( Senior Vice President, Finance and Principal Accounting Officer ) Adoption ( 9/13/23 ) Trading plan intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c) Sale of the Company’s common stock pursuant to the terms of the trading plan 9/13/23 – 9/23/24 16,867 | |
Ariel Hurley | ||
Trading Arrangements, by Individual | ||
Name | Ariel Hurley | |
Title | Senior Vice President, Finance and Principal Accounting Officer | |
Rule 10b5-1 Arrangement Adopted | true | |
Adoption Date | Adoption | |
Termination Date | 9/23/24 | |
Aggregate Available | 16,867 | 16,867 |