Cover Page
Cover Page - shares | 6 Months Ended | |
Jun. 30, 2022 | Jul. 29, 2022 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Jun. 30, 2022 | |
Document Transition Report | false | |
Entity File Number | 001-36014 | |
Entity Registrant Name | AGIOS PHARMACEUTICALS, INC. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 26-0662915 | |
Entity Address, Address Line One | 88 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 | 649-8600 | |
Title of 12(b) Security | Common Stock, Par Value $0.001 per share | |
Trading Symbol | AGIO | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 54,818,049 | |
Document Fiscal Year Focus | 2022 | |
Document Fiscal Period Focus | Q2 | |
Amendment Flag | false | |
Entity Central Index Key | 0001439222 | |
Current Fiscal Year End Date | --12-31 |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets (Unaudited) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Current assets: | ||
Cash and cash equivalents | $ 88,543 | $ 203,126 |
Marketable securities | 730,766 | 816,892 |
Accounts receivable, net | 1,598 | 0 |
Other receivable | 2,704 | 4,378 |
Inventory | 4,060 | 0 |
Prepaid expenses and other current assets | 42,336 | 39,835 |
Total current assets | 870,007 | 1,064,231 |
Marketable securities | 280,845 | 266,375 |
Operating lease assets | 70,214 | 75,124 |
Property and equipment, net | 27,382 | 28,923 |
Financing lease assets | 64 | 183 |
Other non-current assets | 3,955 | 2,900 |
Total assets | 1,252,467 | 1,437,736 |
Current liabilities: | ||
Accounts payable | 7,790 | 16,700 |
Accrued expenses | 28,383 | 31,967 |
Operating lease liabilities | 12,056 | 10,828 |
Financing lease liabilities | 167 | 331 |
Total current liabilities | 48,396 | 59,826 |
Operating lease liabilities, net of current portion | 78,946 | 85,659 |
Other non-current liabilities | 1,055 | 276 |
Total liabilities | 128,397 | 145,761 |
Stockholders’ equity: | ||
Preferred stock, $0.001 par value; 25,000,000 shares authorized; no shares issued or outstanding at June 30, 2022 and December 31, 2021 | 0 | 0 |
Common stock, $0.001 par value; 125,000,000 shares authorized; 71,031,792 shares issued and 54,815,381 shares outstanding at June 30, 2022, and 70,550,631 shares issued and 54,334,220 shares outstanding at December 31, 2021 | 71 | 71 |
Additional paid-in capital | 2,362,327 | 2,334,348 |
Accumulated other comprehensive loss | (10,502) | (1,198) |
Treasury stock, at cost (16,216,411 shares at June 30, 2022 and December 31, 2021) | (802,486) | (802,486) |
Accumulated deficit | (425,340) | (238,760) |
Total stockholders’ equity | 1,124,070 | 1,291,975 |
Total liabilities and stockholders’ equity | $ 1,252,467 | $ 1,437,736 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Unaudited) (Parenthetical) - $ / shares | Jun. 30, 2022 | Dec. 31, 2021 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in usd per share) | $ 0.001 | $ 0.001 |
Preferred stock, shares authorized (in shares) | 25,000,000 | 25,000,000 |
Preferred stock, shares issued (in shares) | 0 | 0 |
Preferred stock, shares outstanding (in shares) | 0 | 0 |
Common stock, par value (in usd per share) | $ 0.001 | $ 0.001 |
Common stock, shares authorized (in shares) | 125,000,000 | 125,000,000 |
Common stock, shares issued (in shares) | 71,031,792 | 70,550,631 |
Common stock, shares outstanding (in shares) | 54,815,381 | 54,334,220 |
Treasury stock, shares (in shares) | 16,216,411 | 16,216,411 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Revenues: | ||||
Total revenue | $ 5,582 | $ 0 | $ 6,414 | $ 0 |
Cost and expenses: | ||||
Cost of sales | 435 | 0 | 774 | 0 |
Research and development | 74,523 | 62,007 | 144,646 | 119,674 |
Selling, general and administrative | 28,264 | 29,215 | 59,779 | 62,765 |
Total cost and expenses | 103,222 | 91,222 | 205,199 | 182,439 |
Loss from operations | (97,640) | (91,222) | (198,785) | (182,439) |
Royalty income from gain on sale of oncology business | 2,704 | 2,000 | 5,408 | 2,000 |
Interest income (expense), net | 1,793 | (92) | 2,487 | 248 |
Other income, net | 1,337 | 6,524 | 4,310 | 6,524 |
Net loss from continuing operations | (91,806) | (82,790) | (186,580) | (173,667) |
Net (loss) income from discontinued operations, net of tax | 0 | (3,427) | 0 | 1,961,775 |
Net (loss) income | $ (91,806) | $ (86,217) | $ (186,580) | $ 1,788,108 |
Net loss from continuing operations per share - basic (in usd per share) | $ (1.68) | $ (1.36) | $ (3.41) | $ (2.66) |
Net loss from continuing operations per share - diluted (in usd per share) | (1.68) | (1.36) | (3.41) | (2.66) |
Net (loss) income from discontinued operations per share - basic (in usd per share) | 0 | (0.06) | 0 | 30.05 |
Net (loss) income from discontinued operations per share - diluted (in usd per share) | 0 | (0.06) | 0 | 30.05 |
Net (loss) income per share - basic (in usd per share) | (1.68) | (1.41) | (3.41) | 27.39 |
Net (loss) income per share - diluted (in usd per share) | $ (1.68) | $ (1.41) | $ (3.41) | $ 27.39 |
Weighted-average number of common shares used in computing net loss per share from continuing operations, net (loss) income from discontinued operations and net (loss) income per share - basic (in shares) | 54,799,680 | 61,066,977 | 54,678,249 | 65,281,827 |
Weighted-average number of common shares used in computing net loss per share from continuing operations, net (loss) income from discontinued operations and net (loss) income per share - diluted (in shares) | 54,799,680 | 61,066,977 | 54,678,249 | 65,281,827 |
Product revenue, net | ||||
Revenues: | ||||
Total revenue | $ 3,082 | $ 0 | $ 3,914 | $ 0 |
Milestone revenue | ||||
Revenues: | ||||
Total revenue | $ 2,500 | $ 0 | $ 2,500 | $ 0 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Comprehensive (Loss) Income (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Statement of Comprehensive Income [Abstract] | ||||
Net (loss) income | $ (91,806) | $ (86,217) | $ (186,580) | $ 1,788,108 |
Other comprehensive loss | ||||
Unrealized loss on available-for-sale securities | (2,757) | (141) | (9,304) | (249) |
Comprehensive (loss) income | $ (94,563) | $ (86,358) | $ (195,884) | $ 1,787,859 |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Stockholders' Equity (Unaudited) - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-In Capital | Accumulated Other Comprehensive (Loss) Income | Accumulated Deficit | Treasury Stock |
Common stock, beginning balance (in shares) at Dec. 31, 2020 | 69,293,920 | |||||
Treasury stock, beginning balance (in shares) at Dec. 31, 2020 | 0 | |||||
Beginning balance at Dec. 31, 2020 | $ 399,500 | $ 69 | $ 2,242,801 | $ 105 | $ (1,843,475) | $ 0 |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Common stock issued under stock incentive plan and ESPP (in shares) | 518,285 | |||||
Common stock issued under stock incentive plan and ESPP | 7,347 | $ 1 | 7,346 | |||
Stock-based compensation expense | 14,854 | 14,854 | ||||
Other comprehensive loss | (108) | (108) | ||||
Net (loss) income | 1,874,325 | 1,874,325 | ||||
Disposition of oncology business | 712 | 712 | ||||
Common stock, ending balance (in shares) at Mar. 31, 2021 | 69,812,205 | |||||
Treasury stock, ending balance (in shares) at Mar. 31, 2021 | 0 | |||||
Ending balance at Mar. 31, 2021 | 2,296,630 | $ 70 | 2,265,713 | (3) | 30,850 | $ 0 |
Common stock, beginning balance (in shares) at Dec. 31, 2020 | 69,293,920 | |||||
Treasury stock, beginning balance (in shares) at Dec. 31, 2020 | 0 | |||||
Beginning balance at Dec. 31, 2020 | 399,500 | $ 69 | 2,242,801 | 105 | (1,843,475) | $ 0 |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Net (loss) income | 1,788,108 | |||||
Common stock, ending balance (in shares) at Jun. 30, 2021 | 70,404,782 | |||||
Treasury stock, ending balance (in shares) at Jun. 30, 2021 | (10,493,968,000) | |||||
Ending balance at Jun. 30, 2021 | 1,721,816 | $ 70 | 2,306,304 | (144) | (55,367) | $ (529,047) |
Common stock, beginning balance (in shares) at Mar. 31, 2021 | 69,812,205 | |||||
Treasury stock, beginning balance (in shares) at Mar. 31, 2021 | 0 | |||||
Beginning balance at Mar. 31, 2021 | 2,296,630 | $ 70 | 2,265,713 | (3) | 30,850 | $ 0 |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Common stock issued under stock incentive plan and ESPP (in shares) | 592,577 | |||||
Common stock issued under stock incentive plan and ESPP | 25,673 | 25,673 | ||||
Stock-based compensation expense | 14,885 | 14,885 | ||||
Repurchase of common stock (in shares) | (10,493,968,000) | |||||
Repurchase of common stock | (529,047) | $ (529,047) | ||||
Other comprehensive loss | (141) | (141) | ||||
Net (loss) income | (86,217) | (86,217) | ||||
Disposition of oncology business | 33 | 33 | ||||
Common stock, ending balance (in shares) at Jun. 30, 2021 | 70,404,782 | |||||
Treasury stock, ending balance (in shares) at Jun. 30, 2021 | (10,493,968,000) | |||||
Ending balance at Jun. 30, 2021 | 1,721,816 | $ 70 | 2,306,304 | (144) | (55,367) | $ (529,047) |
Common stock, beginning balance (in shares) at Dec. 31, 2021 | 70,550,631 | |||||
Treasury stock, beginning balance (in shares) at Dec. 31, 2021 | (16,216,411,000) | |||||
Beginning balance at Dec. 31, 2021 | 1,291,975 | $ 71 | 2,334,348 | (1,198) | (238,760) | $ (802,486) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Common stock issued under stock incentive plan and ESPP (in shares) | 442,646 | |||||
Common stock issued under stock incentive plan and ESPP | 1,289 | 1,289 | ||||
Stock-based compensation expense | 15,510 | 15,510 | ||||
Other comprehensive loss | (6,547) | (6,547) | ||||
Net (loss) income | (94,774) | (94,774) | ||||
Common stock, ending balance (in shares) at Mar. 31, 2022 | 70,993,277 | |||||
Treasury stock, ending balance (in shares) at Mar. 31, 2022 | (16,216,411,000) | |||||
Ending balance at Mar. 31, 2022 | 1,207,453 | $ 71 | 2,351,147 | (7,745) | (333,534) | $ (802,486) |
Common stock, beginning balance (in shares) at Dec. 31, 2021 | 70,550,631 | |||||
Treasury stock, beginning balance (in shares) at Dec. 31, 2021 | (16,216,411,000) | |||||
Beginning balance at Dec. 31, 2021 | $ 1,291,975 | $ 71 | 2,334,348 | (1,198) | (238,760) | $ (802,486) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Common stock issued under stock incentive plan and ESPP (in shares) | 4,074 | |||||
Net (loss) income | $ (186,580) | |||||
Common stock, ending balance (in shares) at Jun. 30, 2022 | 71,031,792 | |||||
Treasury stock, ending balance (in shares) at Jun. 30, 2022 | (16,216,411,000) | |||||
Ending balance at Jun. 30, 2022 | 1,124,070 | $ 71 | 2,362,327 | (10,502) | (425,340) | $ (802,486) |
Common stock, beginning balance (in shares) at Mar. 31, 2022 | 70,993,277 | |||||
Treasury stock, beginning balance (in shares) at Mar. 31, 2022 | (16,216,411,000) | |||||
Beginning balance at Mar. 31, 2022 | 1,207,453 | $ 71 | 2,351,147 | (7,745) | (333,534) | $ (802,486) |
Increase (Decrease) in Stockholders' Equity [Roll Forward] | ||||||
Common stock issued under stock incentive plan and ESPP (in shares) | 38,515 | |||||
Common stock issued under stock incentive plan and ESPP | 15 | 15 | ||||
Stock-based compensation expense | 11,165 | 11,165 | ||||
Other comprehensive loss | (2,757) | (2,757) | ||||
Net (loss) income | (91,806) | (91,806) | ||||
Common stock, ending balance (in shares) at Jun. 30, 2022 | 71,031,792 | |||||
Treasury stock, ending balance (in shares) at Jun. 30, 2022 | (16,216,411,000) | |||||
Ending balance at Jun. 30, 2022 | $ 1,124,070 | $ 71 | $ 2,362,327 | $ (10,502) | $ (425,340) | $ (802,486) |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2022 | Jun. 30, 2021 | |
Operating activities | ||
Net (loss) income | $ (186,580) | $ 1,788,108 |
Less: Net income from discontinued operations | 0 | 1,961,775 |
Net loss from continuing operations | (186,580) | (173,667) |
Adjustments to reconcile net loss from continuing operations to net cash used in operating activities: | ||
Depreciation and amortization | 4,769 | 4,829 |
Stock-based compensation expense | 26,675 | 29,739 |
Net amortization of premium (accretion of discount) on marketable securities | 1,382 | 3,422 |
Loss on disposal of property and equipment | 10 | 12 |
Non-cash operating lease expense | 4,919 | 4,760 |
Changes in operating assets and liabilities: | ||
Accounts receivable, net | (1,598) | 0 |
Inventory | (4,060) | 0 |
Other receivables | 1,674 | (8,131) |
Prepaid expenses and other current and non-current assets | (3,556) | (8,454) |
Accounts payable | (7,243) | (5,561) |
Accrued expenses and other current liabilities | (3,584) | (2,547) |
Operating lease liabilities | (5,551) | (3,631) |
Other non-current liabilities | 779 | 0 |
Net cash used in operating activities - continuing operations | (171,964) | (159,229) |
Net cash used in operating activities - discontinued operations | 0 | (78,814) |
Net cash used in operating activities | (171,964) | (238,043) |
Investing activities | ||
Purchases of marketable securities | (607,747) | (498,896) |
Proceeds from maturities and sales of marketable securities | 668,717 | 273,149 |
Purchases of property and equipment | (4,730) | (1,261) |
Net cash provided by (used in) investing activities - continuing operations | 56,240 | (227,008) |
Net cash provided by investing activities - discontinued operations | 0 | 1,802,936 |
Net cash provided by investing activities | 56,240 | 1,575,928 |
Financing activities | ||
Payments on financing lease obligations | (164) | (170) |
Purchase of treasury stock | 0 | (529,047) |
Net proceeds from stock option exercises and employee stock purchase plan | 1,305 | 33,020 |
Net cash provided by (used in) financing activities - continuing operations | 1,141 | (496,197) |
Net cash provided by financing activities - discontinued operations | 0 | 0 |
Net cash provided by (used in) financing activities | 1,141 | (496,197) |
Net change in cash and cash equivalents | (114,583) | 841,688 |
Cash and cash equivalents at beginning of the period | 203,126 | 127,436 |
Cash and cash equivalents at end of the period | 88,543 | 969,124 |
Supplemental disclosure of non-cash investing and financing transactions | ||
Additions to property and equipment in accounts payable and accrued expenses | 11 | 56 |
Financing lease liabilities arising from obtaining financing lease assets | 0 | 511 |
Cash taxes paid | $ 1,842 | $ 6,200 |
Overview and Basis of Presentat
Overview and Basis of Presentation | 6 Months Ended |
Jun. 30, 2022 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Overview and Basis of Presentation | Overview and Basis of Presentation References to Agios Throughout this Quarterly Report on Form 10-Q, “we,” “us,” and “our,” and similar expressions, except where the context requires otherwise, refer to Agios Pharmaceuticals, Inc. and its consolidated subsidiaries, and “our Board of Directors” refers to the board of directors of Agios Pharmaceuticals, Inc. Overview We are a biopharmaceutical company committed to transforming patients’ lives through leadership in the field of cellular metabolism, with the goal of creating differentiated, small molecule medicines for genetically defined diseases, or GDDs. With a history of focused study on cellular metabolism, we have a deep and mature understanding of this biology, which is involved in the healthy functioning of nearly every system in the body. We accelerate the impact of our portfolio by cultivating connections with patient communities, healthcare professionals, partners and colleagues to discover, develop and deliver potential therapies for GDDs. We are located in Cambridge, Massachusetts. The lead product candidate in our GDD portfolio, PYRUKYND® (mitapivat), is an activator of both wild-type and mutant pyruvate kinase, or PK, enzymes for the potential treatment of hemolytic anemias. On February 17, 2022, the U.S. Food and Drug Administration, or FDA, approved PYRUKYND® for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the United States. In June 2021, we submitted a marketing authorization application, or MAA, to the European Medicines Agency, or EMA, for the treatment of adults with PK deficiency in the European Union. The MAA has passed validation, and the regulatory review process is ongoing. We expect a regulatory decision by the end of 2022. In addition, we are currently evaluating PYRUKYND® in clinical trials for the treatment of thalassemia, sickle cell disease, or SCD, and in pediatric patients with PK deficiency. We are also developing AG-946, a novel, next-generation PK activator, for the potential treatment of hemolytic anemias and other indications. In addition to the aforementioned development programs, we continue to prioritize investment in advancing our late lead-optimization research programs – a branched chain amino acid aminotransferase-2, or BCAT2, inhibitor, for the treatment of propionic and methylmalonic acidemia, or MMA, and a phenylalanine hydroxylate, or PAH, stabilizer, for the treatment of phenylketonuria, or PKU. We believe this combination of assets represents an attractive portfolio of programs that aligns with our strategy and core expertise in non-malignant hematology and inborn errors of metabolism, and leaves room for continued growth of our pipeline. We are subject to risks common to companies in our industry including, but not limited to, uncertainties relating to conducting clinical research and development, the manufacture and supply of products for clinical and commercial use, obtaining and maintaining regulatory approvals and pricing and reimbursement for our products, market acceptance, managing global growth and operating expenses, availability of additional capital, competition, obtaining and enforcing patents, stock price volatility, dependence on collaborative relationships and third-party service providers, dependence on key personnel, potential litigation, product liability claims and government investigations. Sale of our Oncology Business to Servier On March 31, 2021, we completed the sale of our oncology business to Servier Pharmaceuticals, LLC, or Servier, which represented a discontinued operation. The transaction included the sale of our oncology business, including TIBSOVO®, our clinical-stage product candidates vorasidenib, AG-270 and AG-636, and our oncology research programs for a payment of approximately $1.8 billion in cash at the closing, subject to certain adjustments, and a payment of $200 million in cash, if, prior to January 1, 2027, vorasidenib is granted new drug application, or NDA, approval from the FDA with an approved label that permits vorasidenib’s use as a single agent for the adjuvant treatment of patients with Grade 2 glioma that have an isocitrate dehydrogenase 1 or 2 mutation (and, to the extent required by such approval, the vorasidenib companion diagnostic test is granted an FDA premarket approval), as well as a royalty of 5% of U.S. net sales of TIBSOVO® from the close of the transaction through loss of exclusivity, and a royalty of 15% of U.S. net sales of vorasidenib from the first commercial sale of vorasidenib through loss of exclusivity. Servier also acquired our co-commercialization rights for Bristol Myers Squibb’s IDHIFA® and the right to receive a $25.0 million potential milestone payment under our prior collaboration agreement with Celgene Corporation, and following the sale Servier will conduct certain clinical development activities within the IDHIFA® development program. We recorded income from royalties of approximately $2.7 million and $2.0 million on U.S. net sales of TIBSOVO® by Servier in the royalty income from gain on sale of oncology business line item within the condensed consolidated statements of operations, for the three months ended June 30, 2022 and 2021, respectively, and $5.4 million and $2.0 million for the six months ended June 30, 2022 and 2021, respectively. Basis of presentation The condensed consolidated balance sheet as of June 30, 2022, the condensed consolidated statements of operations, comprehensive (loss) income and stockholders' equity for the three and six months ended June 30, 2022 and 2021, and the condensed consolidated statements of cash flows for the six months ended June 30, 2022 and 2021 are unaudited. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of our management, reflect all adjustments, which include only normal recurring adjustments, necessary to fairly state our financial position as of June 30, 2022, our results of operations and stockholders' equity for the three and six months ended June 30, 2022 and 2021, and cash flows for the six months ended June 30, 2022 and 2021. The financial data and the other financial information disclosed in these notes to the condensed consolidated financial statements related to the three and six-month periods are also unaudited. The results of operations for the three and six months ended June 30, 2022 are not necessarily indicative of the results to be expected for the year ending December 31, 2022 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2021 was derived from our audited financial statements, but does not include all disclosures required by U.S. generally accepted accounting principles, or U.S. GAAP. The condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2021 that was filed with the Securities and Exchange Commission, or SEC, on February 24, 2022. Our condensed consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries. All intercompany transactions have been eliminated in consolidation. The condensed consolidated financial statements have been prepared in conformity with U.S. GAAP. Reclassifications Certain amounts in prior periods have been reclassified to reflect the impact of the discontinued operations treatment of the oncology business in order to conform to the current period presentation. Use of estimates The preparation of our condensed consolidated financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenues and expenses and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and methodologies. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making judgments about the carrying values of assets, liabilities and equity and the amount of revenues and expenses. The full extent to which the COVID-19 pandemic will directly or indirectly impact our business, results of operations and financial condition, including expenses, reserves and allowances, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain, including as a result of new information that may emerge concerning COVID-19 and any variant strains of the virus and the actions taken to contain the pandemic or treat COVID-19, as well as the economic impact on local, regional, national and international customers and markets. We have made estimates of the impact of COVID-19 within our financial statements and there may be changes to those estimates in future periods. Actual results may differ from these estimates. Liquidity On March 31, 2021, we completed the sale of our oncology business to Servier, and received approximately $1.8 billion in cash at closing. In connection with the sale, on March 25, 2021, we announced that our Board of Directors authorized the repurchase of up to $1.2 billion of our outstanding shares of common stock, or the Repurchase Program, using the proceeds from the sale of our oncology business to Servier. On March 31, 2021, in connection with the Repurchase Program, we entered into a definitive share repurchase agreement with Bristol-Myers Squibb Company, or BMS, to repurchase 7.1 million shares of our common stock held by certain subsidiaries of BMS for an aggregate purchase price of $344.5 million, or $48.38 per share. This repurchase was completed on April 5, 2021. Further, on April 2, 2021, in connection with the Repurchase Program, we entered into a Rule 10b5-1 repurchase plan pursuant to which we could repurchase up to $600.0 million of shares of our common stock. On October 5, 2021, we terminated our Rule 10b5-1 share repurchase program and on October 13, 2021 we entered into a Rule 10b-18 repurchase plan that allows us to conduct open market repurchases over time up to our remaining authorization. As of June 30, 2022 and December 31, 2021, we repurchased approximately 9.1 million shares of common stock for $458.0 million, or $50.35 per share, under the Rule 10b5-1 repurchase plan. As of June 30, 2022, we have not repurchased any shares under the Rule 10b-18 repurchase plan. In total, as of June 30, 2022, we repurchased 16.2 million shares of common stock for $802.5 million, or $49.49 per share, under the Repurchase Program. We have paused our share repurchases for the foreseeable future. As of June 30, 2022, we had cash, cash equivalents and marketable securities of $1.1 billion. Although we have incurred recurring losses and expect to continue to incur losses for the foreseeable future, we expect our cash, cash equivalents and marketable securities will be sufficient to fund current operations for at least the next twelve months from the issuance date of these financial statements. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2022 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | Summary of Significant Accounting Policies Accounts receivable, net Our trade accounts receivable arise from product sales and represent amounts due from specialty distributors and specialty pharmacy providers in the U.S. We monitor the financial performance and creditworthiness of our customers so that we can properly assess and respond to changes in their credit profile. We reserve against these receivables for estimated losses that may arise from a customer’s inability to pay. Amounts determined to be uncollectible are charged or written-off against the reserve. Inventory Inventory is stated at the lower of cost or estimated net realizable value on a first-in, first-out basis. Prior to the regulatory approval of our product candidates, we incur expenses for the manufacture of drug product that could potentially be available to support the commercial launch of those products. Until the date at which regulatory approval has been received or is otherwise considered probable, we record all such costs as research and development expenses. Upon approval of our wholly owned product, PYRUKYND® , by the FDA on February 17, 2022 for the treatment of hemolytic anemia in adults with PK deficiency in the United States, we began to capitalize inventories of PYRUKYND® . Revenue recognition Under Accounting Standards Codification 606, Revenue from Contracts with Customers , or ASC 606, revenue is recognized when the customer obtains control of promised goods or services, in an amount that reflects the consideration that we expect to receive in exchange for those goods or services. To determine revenue recognition for arrangements that have been determined to be within the scope of ASC 606, we perform the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy a performance obligation. We only apply the five-step model to contracts when it is probable that we will collect the consideration we are entitled to in exchange for the goods or services we transfer to the customer. This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, collaboration arrangements and financial instruments. Once the contract is determined to be within the scope of ASC 606, we assess the goods or services promised within each contract and determine those that are performance obligations, and assess whether each promised good or service is distinct. We will then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. Product Revenue We generate product revenue from sales of PYRUKYND® to a limited number of specialty distributors and specialty pharmacy providers, or collectively, the Customers. These Customers subsequently resell PYRUKYND® to pharmacies or dispense directly to patients. In addition to distribution agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND®. The performance obligation related to the sale of PYRUKYND® is satisfied and revenue is recognized when the Customer obtains control of the product, which occurs at a point in time, typically upon delivery to the Customer. Revenues from product sales are recorded at the net sales price, or transaction price, which includes estimates of variable consideration for which reserves are established and result from contractual adjustments, government rebates, returns and other allowances that are offered within the contracts with our Customers, healthcare providers, payors and other indirect customers relating to the sale of our products. Contractual Adjustments . We generally provide Customers with discounts, including prompt pay discounts, and allowances that are explicitly stated in the contracts and are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive sales order management, data and distribution services from certain Customers. Chargebacks and discounts represent the estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to Customers who directly purchase the product from us. Customers charge us for the difference between what they pay for the product and the ultimate selling price to the qualified healthcare providers. These reserves are estimated using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated channel mix and are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue. Government Rebates . Government rebates include Medicare, TriCare, and Medicaid rebates, which we estimate using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated payor mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we will owe an additional liability under the Medicare Part D program. Returns . We estimate the amount of product sales that may be returned by Customers and record this estimate as a reduction of revenue in the period the related product revenue is recognized. We currently estimate product return liabilities using the expected value method, based on available industry data, including our visibility into the inventory remaining in the distribution channel. Cost of sales Cost of sales consists primarily of manufacturing costs of PYRUKYND® . Based on our policy to expense costs associated with the manufacturing of our products prior to regulatory approval, certain of the manufacturing costs associated with product shipments of PYRUKYND® r ecorded during the three and six months ended June 30, 2022 were expensed prior to February 17, 2022 and, therefore, are not included in costs of sales during the three and six months ended June 30, 2022 . There have been no other material changes to the significant accounting policies previously disclosed in our Annual Report on Form 10-K for the year ended December 31, 2021. Recent accounting pronouncements Other accounting standards that have been issued by the Financial Accounting Standards Board or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on our financial statements upon adoption. |
Fair Value Measurements
Fair Value Measurements | 6 Months Ended |
Jun. 30, 2022 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | Fair Value Measurements We record cash equivalents and marketable securities at fair value. ASC 820, Fair Value Measurements and Disclosures , establishes a fair value hierarchy for those instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and our own assumptions (unobservable inputs). The hierarchy consists of three levels: Level 1 – Unadjusted quoted prices in active markets for identical assets or liabilities. Level 2 – Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, directly or indirectly, for substantially the full term of the asset or liability. Level 3 – Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or liability at the measurement date. The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of June 30, 2022: (In thousands) Level 1 Level 2 Level 3 Total Cash equivalents $ 31,062 $ 31,861 $ — $ 62,923 Total cash equivalents 31,062 31,861 — 62,923 Marketable securities: U.S. Treasuries — 153,231 — 153,231 Government securities — 260,712 — 260,712 Corporate debt securities — 597,668 — 597,668 Total marketable securities — 1,011,611 — 1,011,611 Total cash equivalents and marketable securities $ 31,062 $ 1,043,472 $ — $ 1,074,534 Cash equivalents and marketable securities have been initially valued at the transaction price and subsequently, at the end of each reporting period, valued utilizing third-party pricing services or other observable market data. The pricing services utilize industry standard valuation models, including both income and market-based approaches, and observable market inputs to determine value. After completing our validation procedures, we did not adjust or override any fair value measurements provided by the pricing services as of June 30, 2022. There have been no changes to the valuation methods during the six months ended June 30, 2022, and we had no financial assets or liabilities that were classified as Level 3 at any point during the six months ended June 30, 2022. |
Marketable Securities
Marketable Securities | 6 Months Ended |
Jun. 30, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
Marketable Securities | Marketable Securities Our marketable securities are classified as available-for-sale pursuant to ASC 320, Investments – Debt and Equity Securities , and are recorded at fair value. Unrealized gains are included as a component of accumulated other comprehensive loss in the condensed consolidated balance sheets and statements of stockholders’ equity and a component of total comprehensive loss in the condensed consolidated statements of comprehensive (loss) income, until realized. Unrealized losses are evaluated for impairment under ASC 326, Financial Instruments - Credit Losses , to determine if the impairment is credit-related or noncredit-related. Credit-related impairment is recognized as an allowance on the condensed consolidated balance sheets with a corresponding adjustment to earnings, and noncredit-related impairment is recognized in other comprehensive income, net of taxes. Realized gains and losses are included in investment income on a specific-identification basis. There were no material realized gains or losses on marketable securities for the three and six months ended June 30, 2022 or 2021. Marketable securities at June 30, 2022 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 122,984 $ 25 $ (537) $ 122,472 Government securities 99,715 — (928) 98,787 Corporate debt securities 512,636 — (3,129) 509,507 Total Current 735,335 25 (4,594) 730,766 Non-current: U.S. Treasuries 31,319 16 (576) 30,759 Government securities 165,797 23 (3,895) 161,925 Corporate debt securities 89,662 — (1,501) 88,161 Total Non-current 286,778 39 (5,972) 280,845 Total marketable securities $ 1,022,113 $ 64 $ (10,566) $ 1,011,611 Marketable securities at December 31, 2021 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 269,109 $ — $ (36) $ 269,073 Government securities 17,764 1 (10) 17,755 Corporate debt securities 530,490 3 (429) 530,064 Total Current 817,363 4 (475) 816,892 Non-current: U.S. Treasuries 40,607 — (23) 40,584 Government securities 148,820 — (470) 148,350 Corporate debt securities 77,675 — (234) 77,441 Total Non-current 267,102 — (727) 266,375 Total marketable securities $ 1,084,465 $ 4 $ (1,202) $ 1,083,267 As of June 30, 2022 and December 31, 2021, we held both current and non-current investments. Investments classified as current have maturities of less than one year. Investments classified as non-current are those that: (i) have a maturity of greater than one year, and (ii) we do not intend to liquidate within the next twelve months, although these funds are available for use and, therefore, are classified as available-for-sale. As of June 30, 2022 and December 31, 2021, we held 270 and 294 debt securities, respectively, that were in an unrealized loss position for less than one year. We did not record an allowance for credit losses as of June 30, 2022 and December 31, 2021 related to these securities. The aggregate fair value of debt securities in an unrealized loss position at June 30, 2022 and December 31, 2021 was $968.1 million and $950.5 million, respectively. There were no individual securities that were in a significant unrealized loss position as of June 30, 2022 and December 31, 2021. We regularly review the securities in an unrealized loss position and evaluate the current expected credit loss by considering factors such as historical experience, market data, issuer-specific factors, and current economic conditions. We do not consider these marketable securities to be impaired as of June 30, 2022 and December 31, 2021. |
Inventory
Inventory | 6 Months Ended |
Jun. 30, 2022 | |
Inventory Disclosure [Abstract] | |
Inventory | Inventory Inventory, which consists of commercial supply of PYRUKYND®, consisted of the following: (In thousands) June 30, December 31, Raw materials $ — $ — Work-in-process 3,401 — Finished goods 659 — Total inventory $ 4,060 $ — |
Leases
Leases | 6 Months Ended |
Jun. 30, 2022 | |
Leases [Abstract] | |
Leases | Leases Our building leases are comprised of office and laboratory space under non-cancelable operating leases. These lease agreements have remaining lease terms of six years and contain various clauses for renewal at our option. The renewal options were not included in the calculation of the operating lease assets and the operating lease liabilities as the renewal options are not reasonably certain of being exercised. The lease agreements do not contain residual value guarantees. The components of lease expense and other information related to leases were as follows: Three Months Ended Six Months Ended (In millions) 2022 2021 2022 2021 Operating lease costs $ 3.8 $ 3.8 $ 7.6 $ 7.6 Cash paid for amounts included in the measurement of operating lease liabilities $ 4.4 $ 3.6 $ 8.2 $ 7.2 We have not entered into any material short-term leases or financing leases as of June 30, 2022. In arriving at the operating lease liabilities as of June 30, 2022 and December 31, 2021, we applied the weighted-average incremental borrowing rate of 5.7% for both periods over a weighted-average remaining lease term of 5.7 years and 6.2 years, respectively. As of June 30, 2022, undiscounted minimum rental commitments under non-cancelable leases, for each of the next five years and total thereafter were as follows: (In thousands) Remaining 2022 $ 7,370 2023 18,126 2024 18,660 2025 19,507 2026 20,151 2027 20,755 Thereafter 3,479 Undiscounted minimum rental commitments $ 108,048 Interest (17,046) Operating lease liabilities $ 91,002 We provided our landlord a standby letter of credit of $2.9 million as security for our leases. We are not required to maintain any cash collateral for the standby letter of credit. In August 2021, we entered into a long-term sublease agreement for 13,000 square feet of the office space at 38 Sidney Street, Cambridge, Massachusetts, with the term of the lease running through December 2024. In April 2022, we entered into a long-term sublease agreement for 27,000 square feet of the office space at 64 Sidney Street, Cambridge, Massachusetts, with the term of the lease running through April 2025. We recorded operating sublease income of $1.3 million and $1.7 million for the three and six months ended June 30, 2022, respectively, in other income, net in the condensed consolidated statements of operations. We received a security deposit from our sublessee of approximately $1.1 million which is recorded within other non-current assets on our condensed consolidated balance sheet. As of June 30, 2022, the future minimum lease payments to be received under the long-term sublease agreements were as follows: (In thousands) Remaining 2022 $ 2,123 2023 4,329 2024 4,459 2025 1,101 Total $ 12,012 |
Accrued Expenses
Accrued Expenses | 6 Months Ended |
Jun. 30, 2022 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | Accrued Expenses Accrued expenses consisted of the following: (In thousands) June 30, December 31, Accrued compensation $ 13,213 $ 19,818 Accrued research and development costs 8,646 5,980 Accrued professional fees 2,523 2,335 Accrued other 4,001 3,834 Total accrued expenses $ 28,383 $ 31,967 |
Product Revenue
Product Revenue | 6 Months Ended |
Jun. 30, 2022 | |
Revenue from Contract with Customer [Abstract] | |
Product Revenue | Product Revenue We sell PYRUKYND®, our wholly owned product, to the Customers. The Customers subsequently resell PYRUKYND® to pharmacies or dispense directly to patients. In addition to distribution agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND®. The performance obligation related to the sale of PYRUKYND® is satisfied and revenue is recognized when the Customer obtains control of the product, which occurs at a point in time, typically upon delivery to the Customer. Product revenue, net, were as follows: Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2022 2021 2022 2021 Product revenue, net $ 3,082 $ — $ 3,914 $ — Reserves for Variable Consideration Revenues from product sales are recorded at the net sales price, or transaction price, which includes estimates of variable consideration for which reserves are established and result from contractual adjustments, government rebates, returns and other allowances that are offered within the contracts with our Customers, healthcare providers, payors and other indirect customers relating to the sale of our products. Contractual Adjustments We generally provide Customers with discounts, including prompt pay discounts, and allowances that are explicitly stated in the contracts and are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive sales order management, data and distribution services from certain Customers. Chargebacks and discounts represent the estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to Customers who directly purchase the product from us. Customers charge us for the difference between what they pay for the product and the ultimate selling price to the qualified healthcare providers. These reserves are estimated using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated channel mix and are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue. Government Rebates Government rebates include Medicare, TriCare, and Medicaid rebates, which we estimate using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated payor mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we will owe an additional liability under the Medicare Part D program. Returns We estimate the amount of product sales that may be returned by Customers and record this estimate as a reduction of revenue in the period the related product revenue is recognized. We currently estimate product return liabilities using the expected value method, based on available industry data, including our visibility into the inventory remaining in the distribution channel. The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the six months ended June 30, 2022: (In thousands) Contractual Adjustments Government Rebates Returns Total Balance at December 31, 2021 $ — $ — $ — $ — Current provisions relating to sales in the current year 152 250 44 446 Adjustments relating to prior years — — — — Payments/returns relating to sales in the current year (77) — — (77) Payments/returns relating to sales in the prior years — — — — Balance at June 30, 2022 $ 75 $ 250 $ 44 $ 369 Total revenue-related reserves above, included in our condensed consolidated balance sheets, are summarized as follows: (In thousands) June 30, 2022 December 31, 2021 Reduction of accounts receivable $ 36 $ — Component of accrued expenses 333 — Total revenue-related reserves $ 369 $ — The following table presents changes in our contract assets during the six months ended June 30, 2022: (In thousands) December 31, 2021 Additions Deductions June 30, 2022 Contract assets (1) Accounts receivable, net $ — $ 4,360 $ (2,762) $ 1,598 |
Share-Based Payments
Share-Based Payments | 6 Months Ended |
Jun. 30, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
Share-Based Payments | Share-Based Payments 2013 Stock Incentive Plan In June 2013, our Board of Directors adopted and, in July 2013 our stockholders approved, the 2013 Stock Incentive Plan, or the 2013 Plan. The 2013 Plan became effective upon the closing of our initial public offering and provides for the grant of incentive stock options, non-qualified stock options, stock appreciation rights, restricted stock awards, restricted stock units, or RSUs, performance-based share units, or PSUs, and other stock-based awards to employees, non-employees and non-employee directors. Following the adoption of the 2013 Plan, we granted no further stock options or other awards under the 2007 Stock Incentive Plan, or the 2007 Plan. Any options or awards outstanding under the 2007 Plan at the time of adoption of the 2013 Plan remain outstanding and effective. As of June 30, 2022, the total number of shares reserved under the 2007 Plan and the 2013 Plan was 12,989,404, and we had 6,000,638 shares available for future issuance under the 2013 Plan. Stock options The following table presents stock option activity for the six months ended June 30, 2022: Number of Weighted-Average Exercise Price Outstanding at December 31, 2021 4,798,826 $ 58.51 Granted 1,043,418 29.08 Exercised (4,074) 7.56 Forfeited/Expired (463,999) 68.38 Outstanding at June 30, 2022 5,374,171 $ 52.00 Exercisable at June 30, 2022 3,473,922 $ 58.44 Vested and expected to vest at June 30, 2022 5,374,171 $ 52.00 At June 30, 2022, there was approximately $41.7 million of total unrecognized compensation expense related to unvested stock option awards, which we expect to recognize over a weighted-average period of approximately 2.5 years. Restricted stock units The following table presents RSU activity for the six months ended June 30, 2022: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2021 1,002,924 $ 51.51 Granted 736,813 31.70 Vested (375,154) 53.95 Forfeited (124,097) 41.48 Unvested shares at June 30, 2022 1,240,486 $ 40.06 As of June 30, 2022, there was approximately $36.3 million of total unrecognized compensation expense related to RSUs, which we expect to recognize over a weighted-average period of approximately 1.9 years. Performance-based stock units The following table presents PSU activity for the six months ended June 30, 2022: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2021 234,059 $ 54.28 Granted 156,300 31.53 Vested (53,777) 54.28 Forfeited (5,168) 61.93 Unvested shares at June 30, 2022 331,414 $ 43.43 Stock-based compensation expense associated with these PSUs is recognized if the underlying performance condition is considered probable of achievement using our management’s best estimates. As of June 30, 2022, there was no unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered probable of achievement, and $14.4 million of total unrecognized compensation expense related to PSUs with performance-based vesting criteria that are considered not probable of achievement. Market-based stock units The following table presents market-based stock unit, or MSU, activity for the six months ended June 30, 2022: Number of Weighted-Average Unvested shares at December 31, 2021 42,695 $ 41.50 Granted — — Unvested shares at June 30, 2022 42,695 $ 41.50 The fair value of MSUs are estimated using a Monte Carlo simulation model. Assumptions and estimates utilized in the model include the risk-free interest rate, dividend yield, expected stock volatility and the estimated period to achievement of the market condition. As of June 30, 2022, there was no remaining unrecognized compensation expense related to MSUs. 2013 Employee Stock Purchase Plan In June 2013, our Board of Directors adopted, and in July 2013 our stockholders approved, the 2013 Employee Stock Purchase Plan, or the 2013 ESPP. We issued and sold 48,156 and 59,401 shares of common stock during the six months ended June 30, 2022 and 2021, respectively, under the 2013 ESPP. The 2013 ESPP provides participating employees with the opportunity to purchase up to an aggregate of 1,854,545 shares of our common stock. As of June 30, 2022, we had 1,346,491 shares of common stock available for future issuance under the 2013 ESPP. Stock-based compensation expense Stock-based compensation expense by award type included within the condensed consolidated statements of operations is as follows: Three Months Ended Six Months Ended (In thousands) 2022 2021 2022 2021 Stock options $ 5,655 $ 8,789 $ 11,856 $ 17,185 Restricted stock units 5,232 5,784 11,397 11,989 Performance-based stock units — — 2,919 — Employee stock purchase plan 278 312 503 565 Total stock-based compensation expense $ 11,165 $ 14,885 $ 26,675 $ 29,739 Expenses related to stock options and stock-based awards were allocated as follows in the condensed consolidated statements of operations: Three Months Ended Six Months Ended (In thousands) 2022 2021 2022 2021 Research and development expense $ 4,923 $ 6,422 $ 11,579 $ 13,395 Selling, general and administrative expense 6,242 8,463 15,096 16,344 Total stock-based compensation expense $ 11,165 $ 14,885 $ 26,675 $ 29,739 |
Loss per Share
Loss per Share | 6 Months Ended |
Jun. 30, 2022 | |
Earnings Per Share [Abstract] | |
Loss per Share | 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 the weighted average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury stock method. For purposes of the dilutive net loss per share calculation, stock options, RSUs, PSUs and MSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs and MSUs with performance and market vesting conditions, respectively, that were not deemed probable as of June 30, 2022 are not considered to be common stock equivalents. We utilize the control number concept in the computation of diluted earnings per share to determine whether potential common stock equivalents are dilutive. The control number used is net loss from continuing operations. The control number concept requires that the same number of potentially dilutive securities applied in computing diluted earnings per share from continuing operations be applied to all other categories of income or loss, regardless of their anti-dilutive effect on such categories. Since we had a net loss from continuing operations for all periods presented, no dilutive effect has been recognized in the calculation of income from discontinued operations per share. Basic and diluted net loss per share was the same for all periods presented. The following common stock equivalents were excluded from the calculation of diluted net loss per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: Three and Six Months Ended June 30, 2022 2021 Stock options 5,374,171 5,251,842 Restricted stock units 1,240,486 1,185,013 Performance-based stock units — — Employee stock purchase plan shares 60,589 29,422 Total common stock equivalents 6,675,246 6,466,277 |
Income Taxes
Income Taxes | 6 Months Ended |
Jun. 30, 2022 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | Income TaxesWe recorded no provision for income taxes for the three months ended June 30, 2022 or three months ended June 30, 2021, and recorded no provision for income taxes for the six months ended June 30, 2022 and $12.9 million for the six months ended June 30, 2021. The tax provision for the six months ended June 30, 2021 was recorded within discontinued operations as it related to the income tax impact on the sale of our oncology business to Servier. There is no income tax expense recorded in continuing operations for the three and six months ended June 30, 2022 and 2021, respectively. Cash taxes paid were $1.8 million and $6.2 million for the six months ended June 30, 2022 and 2021, respectively. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2022 | |
Accounting Policies [Abstract] | |
Basis of presentation | Basis of presentation The condensed consolidated balance sheet as of June 30, 2022, the condensed consolidated statements of operations, comprehensive (loss) income and stockholders' equity for the three and six months ended June 30, 2022 and 2021, and the condensed consolidated statements of cash flows for the six months ended June 30, 2022 and 2021 are unaudited. The unaudited condensed consolidated financial statements have been prepared on the same basis as the annual financial statements and, in the opinion of our management, reflect all adjustments, which include only normal recurring adjustments, necessary to fairly state our financial position as of June 30, 2022, our results of operations and stockholders' equity for the three and six months ended June 30, 2022 and 2021, and cash flows for the six months ended June 30, 2022 and 2021. The financial data and the other financial information disclosed in these notes to the condensed consolidated financial statements related to the three and six-month periods are also unaudited. The results of operations for the three and six months ended June 30, 2022 are not necessarily indicative of the results to be expected for the year ending December 31, 2022 or for any other future annual or interim period. The condensed consolidated balance sheet data as of December 31, 2021 was derived from our audited financial statements, but does not include all disclosures required by U.S. generally accepted accounting principles, or U.S. GAAP. The condensed consolidated interim financial statements should be read in conjunction with the audited consolidated financial statements and notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2021 that was filed with the Securities and Exchange Commission, or SEC, on February 24, 2022. Our condensed consolidated financial statements include our accounts and the accounts of our wholly owned subsidiaries. All intercompany transactions have been eliminated in consolidation. The condensed consolidated financial statements have been prepared in conformity with U.S. GAAP. |
Reclassifications | Reclassifications Certain amounts in prior periods have been reclassified to reflect the impact of the discontinued operations treatment of the oncology business in order to conform to the current period presentation. |
Use of estimates | Use of estimates The preparation of our condensed consolidated financial statements requires us to make estimates, judgments and assumptions that may affect the reported amounts of assets, liabilities, equity, revenues and expenses and related disclosure of contingent assets and liabilities. On an ongoing basis we evaluate our estimates, judgments and methodologies. We base our estimates on historical experience and on various other assumptions that we believe are reasonable, the results of which form the basis for making judgments about the carrying values of assets, liabilities and equity and the amount of revenues and expenses. The full extent to which the COVID-19 pandemic will directly or indirectly impact our business, results of operations and financial condition, including expenses, reserves and allowances, clinical trials, research and development costs and employee-related amounts, will depend on future developments that are highly uncertain, including as a result of new information that may emerge concerning COVID-19 and any variant strains of the virus and the actions taken to contain the pandemic or treat COVID-19, as well as the economic impact on local, regional, national and international customers and markets. We have made estimates of the impact of COVID-19 within our financial statements and there may be changes to those estimates in future periods. Actual results may differ from these estimates. |
Accounts receivable, net | Accounts receivable, net Our trade accounts receivable arise from product sales and represent amounts due from specialty distributors and specialty pharmacy providers in the U.S. We monitor the financial performance and creditworthiness of our customers so that we can properly assess and respond to changes in their credit profile. We reserve against these receivables for estimated losses that may arise from a customer’s inability to pay. Amounts determined to be uncollectible are charged or written-off against the reserve. |
Inventory | Inventory Inventory is stated at the lower of cost or estimated net realizable value on a first-in, first-out basis. Prior to the regulatory approval of our product candidates, we incur expenses for the manufacture of drug product that could potentially be available to support the commercial launch of those products. Until the date at which regulatory approval has been received or is otherwise considered probable, we record all such costs as research and development expenses. Upon approval of our wholly owned product, PYRUKYND® , by the FDA on February 17, 2022 for the treatment of hemolytic anemia in adults with PK deficiency in the United States, we began to capitalize inventories of PYRUKYND® . |
Revenue recognition | Revenue recognition Under Accounting Standards Codification 606, Revenue from Contracts with Customers , or ASC 606, revenue is recognized when the customer obtains control of promised goods or services, in an amount that reflects the consideration that we expect to receive in exchange for those goods or services. To determine revenue recognition for arrangements that have been determined to be within the scope of ASC 606, we perform the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligations in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligations in the contract; and (v) recognize revenue when (or as) we satisfy a performance obligation. We only apply the five-step model to contracts when it is probable that we will collect the consideration we are entitled to in exchange for the goods or services we transfer to the customer. This standard applies to all contracts with customers, except for contracts that are within the scope of other standards, such as leases, insurance, collaboration arrangements and financial instruments. Once the contract is determined to be within the scope of ASC 606, we assess the goods or services promised within each contract and determine those that are performance obligations, and assess whether each promised good or service is distinct. We will then recognize as revenue the amount of the transaction price that is allocated to the respective performance obligation when (or as) the performance obligation is satisfied. Product Revenue We generate product revenue from sales of PYRUKYND® to a limited number of specialty distributors and specialty pharmacy providers, or collectively, the Customers. These Customers subsequently resell PYRUKYND® to pharmacies or dispense directly to patients. In addition to distribution agreements with Customers, we enter into arrangements with healthcare providers and payors that provide for government-mandated and/or privately-negotiated rebates, chargebacks and discounts with respect to the purchase of PYRUKYND®. The performance obligation related to the sale of PYRUKYND® is satisfied and revenue is recognized when the Customer obtains control of the product, which occurs at a point in time, typically upon delivery to the Customer. Revenues from product sales are recorded at the net sales price, or transaction price, which includes estimates of variable consideration for which reserves are established and result from contractual adjustments, government rebates, returns and other allowances that are offered within the contracts with our Customers, healthcare providers, payors and other indirect customers relating to the sale of our products. Contractual Adjustments . We generally provide Customers with discounts, including prompt pay discounts, and allowances that are explicitly stated in the contracts and are recorded as a reduction of revenue in the period the related product revenue is recognized. In addition, we receive sales order management, data and distribution services from certain Customers. Chargebacks and discounts represent the estimated obligations resulting from contractual commitments to sell products to qualified healthcare providers at prices lower than the list prices charged to Customers who directly purchase the product from us. Customers charge us for the difference between what they pay for the product and the ultimate selling price to the qualified healthcare providers. These reserves are estimated using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated channel mix and are established in the same period that the related revenue is recognized, resulting in a reduction of product revenue. Government Rebates . Government rebates include Medicare, TriCare, and Medicaid rebates, which we estimate using the expected value method, based upon a range of possible outcomes that are probability-weighted for the estimated payor mix. These reserves are recorded in the same period the related revenue is recognized, resulting in a reduction of product revenue. For Medicare, we also estimate the number of patients in the prescription drug coverage gap for whom we will owe an additional liability under the Medicare Part D program. Returns . We estimate the amount of product sales that may be returned by Customers and record this estimate as a reduction of revenue in the period the related product revenue is recognized. We currently estimate product return liabilities using the expected value method, based on available industry data, including our visibility into the inventory remaining in the distribution channel. |
Cost of sales | Cost of sales Cost of sales consists primarily of manufacturing costs of PYRUKYND® . Based on our policy to expense costs associated with the manufacturing of our products prior to regulatory approval, certain of the manufacturing costs associated with product shipments of PYRUKYND® r ecorded during the three and six months ended June 30, 2022 were expensed prior to February 17, 2022 and, therefore, are not included in costs of sales during the three and six months ended June 30, 2022 . There have been no other material changes to the significant accounting policies previously disclosed in our Annual Report on Form 10-K for the year ended December 31, 2021. |
Recent accounting pronouncements | Recent accounting pronouncements Other accounting standards that have been issued by the Financial Accounting Standards Board or other standards-setting bodies that do not require adoption until a future date are not expected to have a material impact on our financial statements upon adoption. |
Fair value measurements | We record cash equivalents and marketable securities at fair value. ASC 820, Fair Value Measurements and Disclosures , establishes a fair value hierarchy for those instruments measured at fair value that distinguishes between assumptions based on market data (observable inputs) and our own assumptions (unobservable inputs). The hierarchy consists of three levels: Level 1 – Unadjusted quoted prices in active markets for identical assets or liabilities. Level 2 – Quoted prices for similar assets and liabilities in active markets, quoted prices in markets that are not active, or inputs which are observable, directly or indirectly, for substantially the full term of the asset or liability. Level 3 – Unobservable inputs that reflect our own assumptions about the assumptions market participants would use in pricing the asset or liability in which there is little, if any, market activity for the asset or liability at the measurement date. |
Marketable securities | Our marketable securities are classified as available-for-sale pursuant to ASC 320, Investments – Debt and Equity Securities , and are recorded at fair value. Unrealized gains are included as a component of accumulated other comprehensive loss in the condensed consolidated balance sheets and statements of stockholders’ equity and a component of total comprehensive loss in the condensed consolidated statements of comprehensive (loss) income, until realized. Unrealized losses are evaluated for impairment under ASC 326, Financial Instruments - Credit Losses |
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 the weighted average shares outstanding for the dilutive effect of common stock equivalents outstanding for the period, determined using the treasury stock method. For purposes of the dilutive net loss per share calculation, stock options, RSUs, PSUs and MSUs for which the performance and market vesting conditions, respectively, have been deemed probable, and 2013 ESPP shares are considered to be common stock equivalents, while PSUs and MSUs with performance and market vesting conditions, respectively, that were not deemed probable as of June 30, 2022 are not considered to be common stock equivalents. |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Fair Value Disclosures [Abstract] | |
Cash Equivalents and Marketable Securities Measured at Fair Value on a Recurring Basis | The following table summarizes our cash equivalents and marketable securities measured at fair value and by level on a recurring basis as of June 30, 2022: (In thousands) Level 1 Level 2 Level 3 Total Cash equivalents $ 31,062 $ 31,861 $ — $ 62,923 Total cash equivalents 31,062 31,861 — 62,923 Marketable securities: U.S. Treasuries — 153,231 — 153,231 Government securities — 260,712 — 260,712 Corporate debt securities — 597,668 — 597,668 Total marketable securities — 1,011,611 — 1,011,611 Total cash equivalents and marketable securities $ 31,062 $ 1,043,472 $ — $ 1,074,534 |
Marketable Securities (Tables)
Marketable Securities (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of Marketable Securities | Marketable securities at June 30, 2022 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 122,984 $ 25 $ (537) $ 122,472 Government securities 99,715 — (928) 98,787 Corporate debt securities 512,636 — (3,129) 509,507 Total Current 735,335 25 (4,594) 730,766 Non-current: U.S. Treasuries 31,319 16 (576) 30,759 Government securities 165,797 23 (3,895) 161,925 Corporate debt securities 89,662 — (1,501) 88,161 Total Non-current 286,778 39 (5,972) 280,845 Total marketable securities $ 1,022,113 $ 64 $ (10,566) $ 1,011,611 Marketable securities at December 31, 2021 consisted of the following: (In thousands) Amortized Unrealized Unrealized Fair Current: U.S. Treasuries $ 269,109 $ — $ (36) $ 269,073 Government securities 17,764 1 (10) 17,755 Corporate debt securities 530,490 3 (429) 530,064 Total Current 817,363 4 (475) 816,892 Non-current: U.S. Treasuries 40,607 — (23) 40,584 Government securities 148,820 — (470) 148,350 Corporate debt securities 77,675 — (234) 77,441 Total Non-current 267,102 — (727) 266,375 Total marketable securities $ 1,084,465 $ 4 $ (1,202) $ 1,083,267 |
Inventory (Tables)
Inventory (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventory | Inventory, which consists of commercial supply of PYRUKYND®, consisted of the following: (In thousands) June 30, December 31, Raw materials $ — $ — Work-in-process 3,401 — Finished goods 659 — Total inventory $ 4,060 $ — |
Leases (Tables)
Leases (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Leases [Abstract] | |
Schedule of Components of Lease Expense and Other Information | The components of lease expense and other information related to leases were as follows: Three Months Ended Six Months Ended (In millions) 2022 2021 2022 2021 Operating lease costs $ 3.8 $ 3.8 $ 7.6 $ 7.6 Cash paid for amounts included in the measurement of operating lease liabilities $ 4.4 $ 3.6 $ 8.2 $ 7.2 |
Schedule of Undiscounted Minimum Rental Commitments Under Non-cancelable Leases | As of June 30, 2022, undiscounted minimum rental commitments under non-cancelable leases, for each of the next five years and total thereafter were as follows: (In thousands) Remaining 2022 $ 7,370 2023 18,126 2024 18,660 2025 19,507 2026 20,151 2027 20,755 Thereafter 3,479 Undiscounted minimum rental commitments $ 108,048 Interest (17,046) Operating lease liabilities $ 91,002 |
Lessor, Operating Lease, Payment to be Received, Fiscal Year Maturity | As of June 30, 2022, the future minimum lease payments to be received under the long-term sublease agreements were as follows: (In thousands) Remaining 2022 $ 2,123 2023 4,329 2024 4,459 2025 1,101 Total $ 12,012 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following: (In thousands) June 30, December 31, Accrued compensation $ 13,213 $ 19,818 Accrued research and development costs 8,646 5,980 Accrued professional fees 2,523 2,335 Accrued other 4,001 3,834 Total accrued expenses $ 28,383 $ 31,967 |
Product Revenue (Tables)
Product Revenue (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Revenue from Contract with Customer [Abstract] | |
Schedule of Product Revenue | Product revenue, net, were as follows: Three Months Ended June 30, Six Months Ended June 30, (In thousands) 2022 2021 2022 2021 Product revenue, net $ 3,082 $ — $ 3,914 $ — |
Schedule of Product Revenue Allowance and Reserves | The following table summarizes balances and activity in each of the product revenue allowance and reserve categories for the six months ended June 30, 2022: (In thousands) Contractual Adjustments Government Rebates Returns Total Balance at December 31, 2021 $ — $ — $ — $ — Current provisions relating to sales in the current year 152 250 44 446 Adjustments relating to prior years — — — — Payments/returns relating to sales in the current year (77) — — (77) Payments/returns relating to sales in the prior years — — — — Balance at June 30, 2022 $ 75 $ 250 $ 44 $ 369 |
Schedule of Revenue Related Reserves | Total revenue-related reserves above, included in our condensed consolidated balance sheets, are summarized as follows: (In thousands) June 30, 2022 December 31, 2021 Reduction of accounts receivable $ 36 $ — Component of accrued expenses 333 — Total revenue-related reserves $ 369 $ — |
Schedule of Changes in Contract Assets and Liabilities, Product Revenue | The following table presents changes in our contract assets during the six months ended June 30, 2022: (In thousands) December 31, 2021 Additions Deductions June 30, 2022 Contract assets (1) Accounts receivable, net $ — $ 4,360 $ (2,762) $ 1,598 |
Share-Based Payments (Tables)
Share-Based Payments (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Share-Based Payment Arrangement [Abstract] | |
Schedule of Company's Stock Option Activity | The following table presents stock option activity for the six months ended June 30, 2022: Number of Weighted-Average Exercise Price Outstanding at December 31, 2021 4,798,826 $ 58.51 Granted 1,043,418 29.08 Exercised (4,074) 7.56 Forfeited/Expired (463,999) 68.38 Outstanding at June 30, 2022 5,374,171 $ 52.00 Exercisable at June 30, 2022 3,473,922 $ 58.44 Vested and expected to vest at June 30, 2022 5,374,171 $ 52.00 |
Unvested Stock Unit Activity | The following table presents RSU activity for the six months ended June 30, 2022: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2021 1,002,924 $ 51.51 Granted 736,813 31.70 Vested (375,154) 53.95 Forfeited (124,097) 41.48 Unvested shares at June 30, 2022 1,240,486 $ 40.06 |
Schedule of Performance-Based Units | The following table presents PSU activity for the six months ended June 30, 2022: Number of Weighted-Average Grant Date Fair Value Unvested shares at December 31, 2021 234,059 $ 54.28 Granted 156,300 31.53 Vested (53,777) 54.28 Forfeited (5,168) 61.93 Unvested shares at June 30, 2022 331,414 $ 43.43 |
Schedule of Market-Based Units Activity | The following table presents market-based stock unit, or MSU, activity for the six months ended June 30, 2022: Number of Weighted-Average Unvested shares at December 31, 2021 42,695 $ 41.50 Granted — — Unvested shares at June 30, 2022 42,695 $ 41.50 |
Schedule of Stock-Based Compensation Expense by Award Type Included Within the Condensed Consolidated Statements of Operations | Stock-based compensation expense by award type included within the condensed consolidated statements of operations is as follows: Three Months Ended Six Months Ended (In thousands) 2022 2021 2022 2021 Stock options $ 5,655 $ 8,789 $ 11,856 $ 17,185 Restricted stock units 5,232 5,784 11,397 11,989 Performance-based stock units — — 2,919 — Employee stock purchase plan 278 312 503 565 Total stock-based compensation expense $ 11,165 $ 14,885 $ 26,675 $ 29,739 |
Schedule of Allocated Stock-Based Compensation Expense | Expenses related to stock options and stock-based awards were allocated as follows in the condensed consolidated statements of operations: Three Months Ended Six Months Ended (In thousands) 2022 2021 2022 2021 Research and development expense $ 4,923 $ 6,422 $ 11,579 $ 13,395 Selling, general and administrative expense 6,242 8,463 15,096 16,344 Total stock-based compensation expense $ 11,165 $ 14,885 $ 26,675 $ 29,739 |
Loss per Share (Tables)
Loss per Share (Tables) | 6 Months Ended |
Jun. 30, 2022 | |
Earnings Per Share [Abstract] | |
Common Stock Excluded from Calculation of Diluted Earnings Per Share | The following common stock equivalents were excluded from the calculation of diluted net loss per share applicable to common stockholders for the periods indicated because including them would have had an anti-dilutive effect: Three and Six Months Ended June 30, 2022 2021 Stock options 5,374,171 5,251,842 Restricted stock units 1,240,486 1,185,013 Performance-based stock units — — Employee stock purchase plan shares 60,589 29,422 Total common stock equivalents 6,675,246 6,466,277 |
Overview and Basis of Present_2
Overview and Basis of Presentation (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||||||
Apr. 05, 2021 | Mar. 31, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | Apr. 02, 2021 | Mar. 25, 2021 | |
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Royalty income from gain on sale of oncology business | $ 2,704 | $ 2,000 | $ 5,408 | $ 2,000 | ||||
Stock repurchase program, authorized amount | $ 1,200,000 | |||||||
Repurchase of common stock | $ 529,047 | |||||||
Cash, cash equivalents, and marketable securities | $ 1,100,000 | $ 1,100,000 | ||||||
BMS Repurchase | ||||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Repurchase of common stock (in shares) | 7,100,000 | |||||||
Repurchase of common stock | $ 344,500 | |||||||
Shares repurchased (in usd per share) | $ 48.38 | |||||||
Rule 10b5-1 repurchase plan | ||||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Stock repurchase program, authorized amount | $ 600,000 | |||||||
Repurchase of common stock (in shares) | 9,100,000 | |||||||
Repurchase of common stock | $ 458,000 | |||||||
Shares repurchased (in usd per share) | $ 50.35 | |||||||
Rule 10b-18 repurchase program | ||||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Repurchase of common stock (in shares) | 0 | |||||||
Repurchase Program | ||||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Repurchase of common stock (in shares) | 16,200,000 | |||||||
Repurchase of common stock | $ 802,500 | |||||||
Shares repurchased (in usd per share) | $ 49.49 | |||||||
2010 Agreement | ||||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Milestone payment for achievement of specified ex-U.S. commercial milestone event | $ 25,000 | |||||||
Discontinued Operations, Disposed of by Sale | Agios Oncology Business | ||||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Cash proceeds | 1,800,000 | |||||||
Contingent milestone payment | $ 200,000 | |||||||
Discontinued Operations, Disposed of by Sale | Agios Oncology Business | TIBSOVO | UNITED STATES | ||||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Contingent royalty payment | 5% | |||||||
Discontinued Operations, Disposed of by Sale | Agios Oncology Business | Vorasidenib | UNITED STATES | ||||||||
Income Statement, Balance Sheet and Additional Disclosures by Disposal Groups, Including Discontinued Operations [Line Items] | ||||||||
Contingent royalty payment | 15% |
Fair Value Measurements - Cash
Fair Value Measurements - Cash Equivalents and Marketable Securities Measured at Fair Value on a Recurring Basis (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | $ 1,011,611 | $ 1,083,267 |
Fair Value, Measurements, Recurring | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 62,923 | |
Total marketable securities | 1,011,611 | |
Total cash equivalents and marketable securities | 1,074,534 | |
Fair Value, Measurements, Recurring | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 62,923 | |
Fair Value, Measurements, Recurring | U.S. Treasuries | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 153,231 | |
Fair Value, Measurements, Recurring | Government securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 260,712 | |
Fair Value, Measurements, Recurring | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 597,668 | |
Fair Value, Measurements, Recurring | Level 1 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 31,062 | |
Total marketable securities | 0 | |
Total cash equivalents and marketable securities | 31,062 | |
Fair Value, Measurements, Recurring | Level 1 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 31,062 | |
Fair Value, Measurements, Recurring | Level 1 | U.S. Treasuries | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 1 | Government securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 1 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 2 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 31,861 | |
Total marketable securities | 1,011,611 | |
Total cash equivalents and marketable securities | 1,043,472 | |
Fair Value, Measurements, Recurring | Level 2 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 31,861 | |
Fair Value, Measurements, Recurring | Level 2 | U.S. Treasuries | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 153,231 | |
Fair Value, Measurements, Recurring | Level 2 | Government securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 260,712 | |
Fair Value, Measurements, Recurring | Level 2 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 597,668 | |
Fair Value, Measurements, Recurring | Level 3 | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 0 | |
Total marketable securities | 0 | |
Total cash equivalents and marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 3 | Cash equivalents | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total cash equivalents | 0 | |
Fair Value, Measurements, Recurring | Level 3 | U.S. Treasuries | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 3 | Government securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | 0 | |
Fair Value, Measurements, Recurring | Level 3 | Corporate debt securities | ||
Fair Value, Balance Sheet Grouping, Financial Statement Captions [Line Items] | ||
Total marketable securities | $ 0 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) | Jun. 30, 2022 USD ($) |
Fair Value, Measurements, Recurring | Level 3 | |
Fair Value, Assets and Liabilities Measured on Recurring and Nonrecurring Basis [Line Items] | |
Fair value of assets (liabilities) | $ 0 |
Marketable Securities - Additio
Marketable Securities - Additional Information (Details) | Jun. 30, 2022 USD ($) security | Dec. 31, 2021 USD ($) security |
Investments, Debt and Equity Securities [Abstract] | ||
Number of debt securities in unrealized loss position for less than one year | security | 270 | 294 |
Allowance for credit losses | $ | $ 0 | $ 0 |
Aggregate fair value of debt securities in unrealized loss position | $ | $ 968,100,000 | $ 950,500,000 |
Number of debt securities in significant unrealized loss position | security | 0 | 0 |
Marketable Securities - Summary
Marketable Securities - Summary of Marketable Securities (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | $ 1,022,113 | $ 1,084,465 |
Unrealized Gains | 64 | 4 |
Unrealized Losses | (10,566) | (1,202) |
Fair Value | 1,011,611 | 1,083,267 |
Current: | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 735,335 | 817,363 |
Unrealized Gains | 25 | 4 |
Unrealized Losses | (4,594) | (475) |
Fair Value | 730,766 | 816,892 |
Current: | U.S. Treasuries | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 122,984 | 269,109 |
Unrealized Gains | 25 | 0 |
Unrealized Losses | (537) | (36) |
Fair Value | 122,472 | 269,073 |
Current: | Government securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 99,715 | 17,764 |
Unrealized Gains | 0 | 1 |
Unrealized Losses | (928) | (10) |
Fair Value | 98,787 | 17,755 |
Current: | Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 512,636 | 530,490 |
Unrealized Gains | 0 | 3 |
Unrealized Losses | (3,129) | (429) |
Fair Value | 509,507 | 530,064 |
Non-current: | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 286,778 | 267,102 |
Unrealized Gains | 39 | 0 |
Unrealized Losses | (5,972) | (727) |
Fair Value | 280,845 | 266,375 |
Non-current: | U.S. Treasuries | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 31,319 | 40,607 |
Unrealized Gains | 16 | 0 |
Unrealized Losses | (576) | (23) |
Fair Value | 30,759 | 40,584 |
Non-current: | Government securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 165,797 | 148,820 |
Unrealized Gains | 23 | 0 |
Unrealized Losses | (3,895) | (470) |
Fair Value | 161,925 | 148,350 |
Non-current: | Corporate debt securities | ||
Debt Securities, Available-for-sale [Line Items] | ||
Amortized Cost | 89,662 | 77,675 |
Unrealized Gains | 0 | 0 |
Unrealized Losses | (1,501) | (234) |
Fair Value | $ 88,161 | $ 77,441 |
Inventory - Schedule of Invento
Inventory - Schedule of Inventory (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 0 | $ 0 |
Work-in-process | 3,401 | 0 |
Finished goods | 659 | 0 |
Total inventory | $ 4,060 | $ 0 |
Leases - Additional Information
Leases - Additional Information (Details) ft² in Thousands, $ in Thousands | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2022 USD ($) | Jun. 30, 2022 USD ($) | Apr. 30, 2022 ft² | Dec. 31, 2021 USD ($) | Aug. 31, 2021 ft² | |
Lessor, Lease, Description [Line Items] | |||||
Remaining lease terms | 6 years | 6 years | |||
Weighted-average incremental borrowing rate | 5.70% | 5.70% | 5.70% | ||
Weighted-average remaining lease term | 5 years 8 months 12 days | 5 years 8 months 12 days | 6 years 2 months 12 days | ||
Other non-current assets | $ 3,955 | $ 3,955 | $ 2,900 | ||
Area of premises subleased (in square feet) | ft² | 27 | 13 | |||
Sublease income | 1,300 | 1,700 | |||
Other Noncurrent Assets | |||||
Lessor, Lease, Description [Line Items] | |||||
Other non-current assets | 2,900 | 2,900 | |||
Security deposit liability | $ 1,100 | $ 1,100 |
Leases - Schedule of Lease Expe
Leases - Schedule of Lease Expense and Other Information (Details) - USD ($) $ in Millions | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Leases [Abstract] | ||||
Operating lease costs | $ 3.8 | $ 3.8 | $ 7.6 | $ 7.6 |
Cash paid for amounts included in the measurement of operating lease liabilities | $ 4.4 | $ 3.6 | $ 8.2 | $ 7.2 |
Leases - Schedule of Undiscount
Leases - Schedule of Undiscounted Minimum Rental Commitments Under Non-cancelable Leases (Details) $ in Thousands | Jun. 30, 2022 USD ($) |
Leases [Abstract] | |
Remaining 2022 | $ 7,370 |
2023 | 18,126 |
2024 | 18,660 |
2025 | 19,507 |
2026 | 20,151 |
2027 | 20,755 |
Thereafter | 3,479 |
Undiscounted minimum rental commitments | 108,048 |
Interest | (17,046) |
Operating lease liabilities | $ 91,002 |
Leases - Future Minimum Lease P
Leases - Future Minimum Lease Payments to be Received (Details) $ in Thousands | Jun. 30, 2022 USD ($) |
Leases [Abstract] | |
Remaining 2022 | $ 2,123 |
2023 | 4,329 |
2024 | 4,459 |
2025 | 1,101 |
Total | $ 12,012 |
Accrued Expenses - Summary of A
Accrued Expenses - Summary of Accrued Expenses (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Payables and Accruals [Abstract] | ||
Accrued compensation | $ 13,213 | $ 19,818 |
Accrued research and development costs | 8,646 | 5,980 |
Accrued professional fees | 2,523 | 2,335 |
Accrued other | 4,001 | 3,834 |
Total accrued expenses | $ 28,383 | $ 31,967 |
Product Revenue - Schedule of P
Product Revenue - Schedule of Product Revenue (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Disaggregation of Revenue [Line Items] | ||||
Product revenue, net | $ 5,582 | $ 0 | $ 6,414 | $ 0 |
Product revenue, net | ||||
Disaggregation of Revenue [Line Items] | ||||
Product revenue, net | $ 3,082 | $ 0 | $ 3,914 | $ 0 |
Product Revenue - Schedule of_2
Product Revenue - Schedule of Product Revenue Allowance and Reserves (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2022 | Dec. 31, 2021 | |
Contractual Adjustments | ||
Contract adjustments, beginning balance | $ 0 | |
Contractual adjustments, current provisions relating to sales in the current year | 152 | |
Contractual adjustments, adjustments relating to prior year | 0 | |
Contractual adjustments, payments/returns relating to sales in the current year | (77) | |
Contractual adjustments, payments/returns relating to sales in the prior year | 0 | |
Contract adjustments, ending balance | 75 | |
Government Rebates | ||
Government rebates, beginning balance | 0 | |
Government rebates, current provisions relating to sales in the current year | 250 | |
Government rebates, adjustments relating to prior years | 0 | |
Government rebates, payments/returns relating to sales in the current year | 0 | |
Government rebates, payments/returns relating to sales in the prior years | 0 | |
Government rebates, ending balance | 250 | |
Returns | ||
Returns, beginning balance | 0 | |
Returns, current provisions relating to sales in the current year | 44 | |
Returns, adjustments relating to prior years | 0 | |
Returns, payments/returns relating to sales in the current year | 0 | |
Returns, payments/returns relating to sales in the prior years | 0 | |
Returns, ending balance | 44 | |
Total | ||
Total revenue-related reserves | 369 | $ 0 |
Total allowances and reserves, current provisions relating to sales in the current year | 446 | |
Total allowances and reserves, adjustments relating to prior years | 0 | |
Total allowances and reserves, payments/returns relating to sales in the current year | (77) | |
Total allowances and reserves, payments/returns relating to sales in the prior years | $ 0 |
Product Revenue - Schedule of R
Product Revenue - Schedule of Revenue-Related Reserves (Details) - USD ($) $ in Thousands | Jun. 30, 2022 | Dec. 31, 2021 |
Revenue from Contract with Customer [Abstract] | ||
Reduction of accounts receivable | $ 36 | $ 0 |
Component of accrued expenses | 333 | 0 |
Total revenue-related reserves | $ 369 | $ 0 |
Product Revenue - Schedule of C
Product Revenue - Schedule of Changes in Contract Assets and Liabilities, Product Revenue (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2022 USD ($) | |
Contract assets | |
Contract assets, beginning balance | $ 0 |
Additions | 4,360 |
Deductions | (2,762) |
Contract assets, ending balance | $ 1,598 |
Share-Based Payments - Addition
Share-Based Payments - Additional Information (Details) - USD ($) $ in Millions | 6 Months Ended | |
Jun. 30, 2022 | Jun. 30, 2021 | |
Restricted stock units | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Weighted-average period to recognize compensation expense (in years) | 1 year 10 months 24 days | |
Unrecognized compensation expense excluding options | $ 36.3 | |
Performance-based stock units | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Unrecognized compensation cost not expected to be recognized | $ 14.4 | |
2007 Plan and 2013 Plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Common stock reserved for issuance (in shares) | 12,989,404 | |
2013 Stock Incentive Plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Shares available for future issuance (in shares) | 6,000,638 | |
Unrecognized compensation expense related to options | $ 41.7 | |
2013 Stock Incentive Plan | Stock options | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Weighted-average period to recognize compensation expense (in years) | 2 years 6 months | |
Employee stock purchase plan | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Shares available for future issuance (in shares) | 1,346,491 | |
Shares issued under 2013 ESPP (in shares) | 48,156 | 59,401 |
Opportunity to purchase of common stock (in shares) | 1,854,545 |
Share-Based Payments - Summary
Share-Based Payments - Summary of Stock Option Activity (Details) - $ / shares | 6 Months Ended |
Jun. 30, 2022 | |
Number of Stock Options | |
Outstanding, beginning balance (in shares) | 4,798,826 |
Granted (in shares) | 1,043,418 |
Exercised (in shares) | (4,074) |
Forfeited/expired (in shares) | (463,999) |
Outstanding, ending balance (in shares) | 5,374,171 |
Number of stock options, exercisable (in shares) | 3,473,922 |
Number of stock options, vested and expected to vest (in shares) | 5,374,171 |
Weighted-Average Exercise Price | |
Outstanding, beginning balance (in usd per share) | $ 58.51 |
Granted (in usd per share) | 29.08 |
Exercised (in usd per share) | 7.56 |
Forfeited/expired (in usd per share) | 68.38 |
Outstanding, ending balance (in usd per share) | 52 |
Weighted-average exercise price, exercisable (in usd per share) | 58.44 |
Weighted-average exercise price, vested and expected to vest (in usd per share) | $ 52 |
Share-Based Payments - Summar_2
Share-Based Payments - Summary of Unvested RSUs Activity (Details) - Restricted stock units | 6 Months Ended |
Jun. 30, 2022 $ / shares shares | |
Number of Stock Units | |
Unvested shares beginning of period (in shares) | shares | 1,002,924 |
Granted (in shares) | shares | 736,813 |
Vested (in shares) | shares | (375,154) |
Forfeited (in shares) | shares | (124,097) |
Unvested shares end of period (in shares) | shares | 1,240,486 |
Weighted-Average Grant Date Fair Value | |
Unvested shares beginning of period (in usd per share) | $ / shares | $ 51.51 |
Granted (in usd per share) | $ / shares | 31.70 |
Vested (in usd per share) | $ / shares | 53.95 |
Forfeited (in usd per share) | $ / shares | 41.48 |
Unvested shares end of period (in usd per share) | $ / shares | $ 40.06 |
Share-Based Payments - Schedule
Share-Based Payments - Schedule of Performance-Based and Market- Based Units (Details) | 6 Months Ended |
Jun. 30, 2022 $ / shares shares | |
Performance-based stock units | |
Number of Stock Units | |
Unvested shares beginning of period (in shares) | shares | 234,059 |
Granted (in shares) | shares | 156,300 |
Vested (in shares) | shares | (53,777) |
Forfeited (in shares) | shares | (5,168) |
Unvested shares end of period (in shares) | shares | 331,414 |
Weighted-Average Grant Date Fair Value | |
Unvested shares beginning of period (in usd per share) | $ / shares | $ 54.28 |
Granted (in usd per share) | $ / shares | 31.53 |
Vested (in usd per share) | $ / shares | 54.28 |
Forfeited (in usd per share) | $ / shares | 61.93 |
Unvested shares end of period (in usd per share) | $ / shares | $ 43.43 |
Market-Based Stock Units | |
Number of Stock Units | |
Unvested shares beginning of period (in shares) | shares | 42,695 |
Granted (in shares) | shares | 0 |
Unvested shares end of period (in shares) | shares | 42,695 |
Weighted-Average Grant Date Fair Value | |
Unvested shares beginning of period (in usd per share) | $ / shares | $ 41.50 |
Granted (in usd per share) | $ / shares | 0 |
Unvested shares end of period (in usd per share) | $ / shares | $ 41.50 |
Share-Based Payments - Schedu_2
Share-Based Payments - Schedule of Stock-Based Compensation Expense by Award Type Included Within the Condensed Consolidated Statements of Operations (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 11,165 | $ 14,885 | $ 26,675 | $ 29,739 |
Stock options | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 5,655 | 8,789 | 11,856 | 17,185 |
Restricted stock units | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 5,232 | 5,784 | 11,397 | 11,989 |
Performance-based stock units | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 0 | 0 | 2,919 | 0 |
Employee stock purchase plan | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 278 | $ 312 | $ 503 | $ 565 |
Share-Based Payments - Expenses
Share-Based Payments - Expenses Related to Equity-Based Awards (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 11,165 | $ 14,885 | $ 26,675 | $ 29,739 |
Research and development expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 4,923 | 6,422 | 11,579 | 13,395 |
Selling, general and administrative expense | ||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 6,242 | $ 8,463 | $ 15,096 | $ 16,344 |
Loss per Share - Common Stock E
Loss per Share - Common Stock Excluded from Calculation of Diluted Net Loss Per Share (Details) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 6,675,246 | 6,466,277 | 6,675,246 | 6,466,277 |
Stock options | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 5,374,171 | 5,251,842 | 5,374,171 | 5,251,842 |
Restricted stock units | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 1,240,486 | 1,185,013 | 1,240,486 | 1,185,013 |
Performance-based stock units | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 0 | 0 | 0 | 0 |
Employee stock purchase plan shares | ||||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||||
Total common stock equivalents (in shares) | 60,589 | 29,422 | 60,589 | 29,422 |
Income Taxes - Additional Infor
Income Taxes - Additional Information (Details) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2022 | Jun. 30, 2021 | Jun. 30, 2022 | Jun. 30, 2021 | |
Income Tax Disclosure [Abstract] | ||||
Provision for income taxes recorded in discontinued operations | $ 0 | $ 0 | $ 0 | $ 12,900,000 |
Income tax expense (benefit) | $ 0 | $ 0 | 0 | 0 |
Income taxes paid | $ 1,800,000 | $ 6,200,000 |