Document and Entity Information
Document and Entity Information - shares | 6 Months Ended | |
Jun. 30, 2024 | Jul. 31, 2024 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Jun. 30, 2024 | |
Document Fiscal Year Focus | 2024 | |
Document Fiscal Period Focus | Q2 | |
Trading Symbol | AMLX | |
Entity Registrant Name | Amylyx Pharmaceuticals, Inc. | |
Entity Central Index Key | 0001658551 | |
Current Fiscal Year End Date | --12-31 | |
Entity File Number | 001-41199 | |
Entity Tax Identification Number | 46-4600503 | |
Entity Address, Address Line One | 43 Thorndike St. | |
Entity Address, City or Town | Cambridge | |
Entity Address, State or Province | MA | |
Entity Address, Postal Zip Code | 02141 | |
City Area Code | 617 | |
Local Phone Number | 682-0917 | |
Entity Current Reporting Status | Yes | |
Entity Filer Category | Accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 68,082,468 | |
Entity Interactive Data Current | Yes | |
Title of 12(b) Security | Common Stock, $0.0001 par value per share | |
Security Exchange Name | NASDAQ | |
Entity Incorporation, State or Country Code | DE | |
Document Quarterly Report | true | |
Document Transition Report | false |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Current assets: | ||
Cash and cash equivalents | $ 74,999 | $ 170,201 |
Short-term investments | 234,813 | 201,161 |
Accounts receivable, net | 4,091 | 40,050 |
Inventories | 38,323 | |
Prepaid expenses and other current assets | 12,819 | 14,931 |
Total current assets | 326,722 | 464,666 |
Property and equipment, net | 1,473 | 2,686 |
Restricted cash equivalents | 719 | 719 |
Operating lease right-of-use assets | 2,769 | 3,725 |
Long-term inventories | 44,957 | |
Other assets | 479 | 701 |
Total assets | 332,162 | 517,454 |
Current liabilities: | ||
Accounts payable | 4,955 | 22,061 |
Accrued expenses | 62,757 | 57,724 |
Operating lease liabilities, current portion | 2,369 | 2,257 |
Total current liabilities | 70,081 | 82,042 |
Operating lease liabilities, net of current portion | 772 | 1,980 |
Total liabilities | 70,853 | 84,022 |
Commitments and contingencies (Note 10) | ||
Stockholders' equity: | ||
Preferred stock, $0.0001 par value; 10,000,000 shares authorized | ||
Common stock, $0.0001 par value; 300,000,000 shares authorized; 68,079,057 and 67,707,432 shares issued and outstanding as of June 30, 2024 and December 31, 2023, respectively | 7 | 7 |
Additional paid-in capital | 757,841 | 738,177 |
Accumulated deficit | (496,442) | (304,949) |
Accumulated other comprehensive (loss) income | (97) | 197 |
Total stockholders' equity | 261,309 | 433,432 |
Total liabilities and stockholders' equity | $ 332,162 | $ 517,454 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Jun. 30, 2024 | Dec. 31, 2023 |
Statement of Financial Position [Abstract] | ||
Preferred stock par value | $ 0.0001 | $ 0.0001 |
Preferred stock shares authorized | 10,000,000 | 10,000,000 |
Common stock, stated par value per share | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 300,000,000 | 300,000,000 |
Common stock, shares, issued | 68,079,057 | 67,707,432 |
Common stock, shares, outstanding | 68,079,057 | 67,707,432 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Income Statement [Abstract] | ||||
Product revenue, net | $ (1,023) | $ 98,216 | $ 87,620 | $ 169,644 |
Revenue from Contract with Customer, Product and Service [Extensible Enumeration] | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember | us-gaap:ProductMember |
Operating expenses: | ||||
Cost of sales | $ 8 | $ 5,580 | $ 5,953 | $ 10,863 |
Cost of sales - inventory impairment and loss on firm purchase commitments | 7,410 | 117,871 | ||
Research and development | 23,347 | 29,044 | 59,955 | 53,236 |
Selling, general and administrative | 21,647 | 43,391 | 79,406 | 87,397 |
Restructuring expenses | 22,851 | 22,851 | ||
Total operating expenses | 75,263 | 78,015 | 286,036 | 151,496 |
Loss (income) from operations | (76,286) | 20,201 | (198,416) | 18,148 |
Other income, net: | ||||
Interest income | 4,069 | 3,887 | 8,395 | 7,605 |
Other expense, net | (483) | (81) | (1,230) | (343) |
Total other income, net | 3,586 | 3,806 | 7,165 | 7,262 |
(Loss) income before income taxes | (72,700) | 24,007 | (191,251) | 25,410 |
Provision for income taxes | 1,933 | 242 | 1,763 | |
Net (loss) income | $ (72,700) | $ 22,074 | $ (191,493) | $ 23,647 |
Net (loss) income per share - basic | $ (1.07) | $ 0.33 | $ (2.82) | $ 0.35 |
Net (loss) income per share - diluted | $ (1.07) | $ 0.31 | $ (2.82) | $ 0.34 |
Weighted-average shares used in computing net (loss) income per share-basic | 68,024,929 | 67,233,617 | 67,939,642 | 66,976,871 |
Weighted-average shares used in computing net (loss) income per share-diluted | 68,024,929 | 70,132,040 | 67,939,642 | 70,471,821 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE (LOSS) INCOME - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Statement of Comprehensive Income [Abstract] | ||||
Net (loss) income | $ (72,700) | $ 22,074 | $ (191,493) | $ 23,647 |
Other comprehensive (loss) income | ||||
Foreign currency translation (loss) gain | (43) | (18) | (201) | 60 |
Net unrealized gain (loss) on available-for-sale securities | 23 | (79) | (93) | (46) |
Other comprehensive (loss) income | (20) | (97) | (294) | 14 |
Comprehensive (loss) income | $ (72,720) | $ 21,977 | $ (191,787) | $ 23,661 |
CONDENSED CONSOLIDATED STATEM_3
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS' EQUITY - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-In Capital | Accumulated Other Comprehensive Income (Loss) | Accumulated Deficit |
Beginning Balance, Shares at Dec. 31, 2022 | 66,512,011 | ||||
Beginning Balance at Dec. 31, 2022 | $ 340,607 | $ 7 | $ 694,906 | $ (86) | $ (354,220) |
Issuance of common stock upon exercise of stock options, Shares | 451,298 | ||||
Issuance of common stock upon exercise of stock options | 2,777 | 2,777 | |||
Issuance of common stock upon vesting of RSUs | 132,294 | ||||
Stock-based compensation expense | 7,580 | 7,580 | |||
Other comprehensive loss | 111 | 111 | |||
Net (loss) income | 1,573 | 1,573 | |||
Ending Balance, Shares at Mar. 31, 2023 | 67,095,603 | ||||
Ending Balance at Mar. 31, 2023 | 352,648 | $ 7 | 705,263 | 25 | (352,647) |
Beginning Balance, Shares at Dec. 31, 2022 | 66,512,011 | ||||
Beginning Balance at Dec. 31, 2022 | 340,607 | $ 7 | 694,906 | (86) | (354,220) |
Net (loss) income | 23,647 | ||||
Ending Balance, Shares at Jun. 30, 2023 | 67,354,417 | ||||
Ending Balance at Jun. 30, 2023 | 386,365 | $ 7 | 717,003 | (72) | (330,573) |
Beginning Balance, Shares at Mar. 31, 2023 | 67,095,603 | ||||
Beginning Balance at Mar. 31, 2023 | 352,648 | $ 7 | 705,263 | 25 | (352,647) |
Issuance of common stock upon exercise of stock options, Shares | 226,138 | ||||
Issuance of common stock upon exercise of stock options | 1,687 | 1,687 | |||
Issuance of common stock upon vesting of RSUs | 32,676 | ||||
Stock-based compensation expense | 10,053 | 10,053 | |||
Other comprehensive loss | (97) | (97) | |||
Net (loss) income | 22,074 | 22,074 | |||
Ending Balance, Shares at Jun. 30, 2023 | 67,354,417 | ||||
Ending Balance at Jun. 30, 2023 | $ 386,365 | $ 7 | 717,003 | (72) | (330,573) |
Beginning Balance, Shares at Dec. 31, 2023 | 67,707,432 | 67,707,432 | |||
Beginning Balance at Dec. 31, 2023 | $ 433,432 | $ 7 | 738,177 | 197 | (304,949) |
Issuance of common stock upon exercise of stock options, Shares | 49,618 | ||||
Issuance of common stock upon exercise of stock options | 147 | 147 | |||
Issuance of common stock upon vesting of RSUs | 218,537 | ||||
Stock-based compensation expense | 9,924 | 9,924 | |||
Other comprehensive loss | (274) | (274) | |||
Net (loss) income | (118,793) | (118,793) | |||
Ending Balance, Shares at Mar. 31, 2024 | 67,975,587 | ||||
Ending Balance at Mar. 31, 2024 | $ 324,436 | $ 7 | 748,248 | (77) | (423,742) |
Beginning Balance, Shares at Dec. 31, 2023 | 67,707,432 | 67,707,432 | |||
Beginning Balance at Dec. 31, 2023 | $ 433,432 | $ 7 | 738,177 | 197 | (304,949) |
Issuance of common stock upon exercise of stock options, Shares | 109,599 | ||||
Net (loss) income | $ (191,493) | ||||
Ending Balance, Shares at Jun. 30, 2024 | 68,079,057 | 68,079,057 | |||
Ending Balance at Jun. 30, 2024 | $ 261,309 | $ 7 | 757,841 | (97) | (496,442) |
Beginning Balance, Shares at Mar. 31, 2024 | 67,975,587 | ||||
Beginning Balance at Mar. 31, 2024 | 324,436 | $ 7 | 748,248 | (77) | (423,742) |
Issuance of common stock upon exercise of stock options, Shares | 59,981 | ||||
Issuance of common stock upon exercise of stock options | 23 | 23 | |||
Issuance of common stock upon vesting of RSUs | 43,489 | ||||
Stock-based compensation expense | 9,570 | 9,570 | |||
Other comprehensive loss | (20) | (20) | |||
Net (loss) income | $ (72,700) | (72,700) | |||
Ending Balance, Shares at Jun. 30, 2024 | 68,079,057 | 68,079,057 | |||
Ending Balance at Jun. 30, 2024 | $ 261,309 | $ 7 | $ 757,841 | $ (97) | $ (496,442) |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
Cash flows (used in) provided by operating activities: | ||
Net (loss) income | $ (191,493) | $ 23,647 |
Adjustments to reconcile net (loss) income to net cash (used in) provided by operating activities: | ||
Stock-based compensation expense | 19,494 | 17,451 |
Depreciation expense | 523 | 485 |
Accretion of investment discounts, net | (5,760) | (4,886) |
Inventory impairment and loss on firm purchase commitments | 117,871 | |
Other non-cash items | 827 | |
Changes in operating assets and liabilities: | ||
Accounts receivable, net | 35,958 | (18,167) |
Inventories | (9,253) | (32,636) |
Interest receivable | 421 | (356) |
Prepaid expenses and other current assets | 1,657 | (1,318) |
Operating lease right-of-use assets | 957 | 881 |
Other assets | 217 | |
Accounts payable | (17,087) | 18,455 |
Accrued expenses | (20,305) | 971 |
Operating lease liabilities | (1,096) | (990) |
Net cash (used in) provided by operating activities | (67,069) | 3,537 |
Cash flows (used in) provided by investing activities: | ||
Purchases of property and equipment | (156) | (528) |
Purchases of investments | (231,986) | (9,756) |
Proceeds from maturities of short-term investments | 204,000 | 189,500 |
Net cash (used in) provided by investing activities | (28,142) | 179,216 |
Cash flows provided by financing activities: | ||
Follow-on offering costs paid | (136) | |
Proceeds from exercise of stock options and RSUs vesting | 1,493 | 5,663 |
Withholding taxes paid on stock-based awards | (1,335) | (3,103) |
Net cash provided by financing activities | 158 | 2,424 |
Effect of exchange rate changes on cash, cash equivalents and restricted cash equivalents | (149) | 57 |
Net (decrease) increase in cash, cash equivalents and restricted cash equivalents | (95,202) | 185,234 |
Cash, cash equivalents and restricted cash equivalents, beginning of period | 170,920 | 63,245 |
Cash, cash equivalents and restricted cash equivalents, end of period | 75,718 | 248,479 |
Supplemental disclosure of cash flow information: | ||
Taxes withheld on stock-based awards included in accrued expenses | 12 | 67 |
Purchases of property and equipment included in accounts payable | 81 | |
Income taxes paid | $ 281 | $ 2,562 |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2024 | Mar. 31, 2024 | Jun. 30, 2023 | Mar. 31, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Pay vs Performance Disclosure | ||||||
Net Income (Loss) | $ (72,700) | $ (118,793) | $ 22,074 | $ 1,573 | $ (191,493) | $ 23,647 |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended |
Jun. 30, 2024 | |
Trading Arrangements, by Individual | |
Material Terms of Trading Arrangement | During the three months ended June 30, 2024 , no officer or director of the Company (as defined in Rule 16a-1(f)) adopted or terminated a “Rule 10b5-1 trading arrangement” or a “non-Rule 10b5-1 trading arrangement,” as each term is defined in Item 408(a) of Regulation S-K of the Exchange Act. |
Rule 10b5-1 Arrangement Adopted | false |
Non-Rule 10b5-1 Arrangement Adopted | false |
Rule 10b5-1 Arrangement Terminated | false |
Non-Rule 10b5-1 Arrangement Terminated | false |
Nature of the Business
Nature of the Business | 6 Months Ended |
Jun. 30, 2024 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Nature of the Business | 1. Nature of the Business Amylyx Pharmaceuticals, Inc., together with its wholly owned subsidiaries, known as Amylyx or the Company, is a biotechnology company that is committed to the discovery and development of new treatment options for communities with high unmet needs, including people living with serious and fatal diseases. The Company has preclinical or clinical development programs underway in neurodegenerative, neuroendocrine and endocrine diseases. On July 9, 2024, Amylyx completed the acquisition of avexitide for the potential treatment of hyperinsulinemic hypoglycemia, a disease of the endocrine system. Avexitide is an investigational, first-in-class glucagon-like peptide-1, or GLP-1, receptor antagonist that has been evaluated in five clinical trials for post-bariatric hypoglycemia, or PBH, and has also been studied in congenital hyperinsulinism, or HI, two indications characterized by hyperinsulinemic hypoglycemia. The U.S. Food and Drug Administration, or FDA, has granted avexitide breakthrough therapy designation for both indications, rare pediatric disease designation in congenital HI, and orphan drug designation for the treatment of hyperinsulinemic hypoglycemia (which includes PBH and congenital HI). Amylyx expects to begin the Phase 3 program for avexitide in PBH in the first quarter of 2025. The Company is also investigating AMX0035, developed by Amylyx, in both neurodegenerative and neuroendocrine diseases where endoplasmic reticulum, or ER, stress and mitochondrial dysfunction are implicated, including progressive supranuclear palsy, or PSP, and Wolfram syndrome. The Company dosed the first participant in the HELIOS trial, a Phase 2 trial of AMX0035 for the treatment of Wolfram syndrome, in April 2023. Amylyx plans to present the HELIOS topline data for all 12 participants at Week 24, including all of the longer-term data available for participants who have reached their Week 36 or Week 48 visit at that time, at a medical meeting this fall. The Company is engaging with stakeholders, including the FDA, and planning for a single Phase 3 clinical trial and will share additional details on the clinical trial design once finalized. The Company dosed the first participant in the ORION trial, a Phase 2b/3 global clinical trial of AMX0035 for the treatment of PSP, in December 2023. Data from an interim analysis of the ORION are expected in mid-2025. The Company is also advancing additional drug candidates developed by Amylyx for neurodegenerative diseases including AMX0114, a potent antisense oligonucleotide targeting inhibition of Calpain-2, a key contributor to the axonal (also known as Wallerian) degeneration pathway. The Company is preparing to initiate a multiple ascending dose clinical trial of AMX0114 in people living with ALS in the second half of 2024. In April 2024, the Company announced it had started a process with the FDA and Heath Canada to voluntarily discontinue the marketing authorizations for RELYVRIO®/ALBRIOZA (AMX0035) for amyotrophic lateral sclerosis, or ALS, and remove the product from the market in the U.S. and Canada based on topline results from the global Phase 3 PHOENIX trial, which failed to meet its prespecified primary and secondary endpoints. The Company will continue to collect available data on survival and to share learnings from PHOENIX to help inform future ALS research. Amylyx is planning to wind down the Open Label Extension by the end of 2024. Separately, the PHOENIX survival data collection efforts will continue at the encouragement of ALS specialists. Risks and Uncertainties The Company is subject to risks and uncertainties common to companies in the biotechnology industry, including, but not limited to, the outcome of preclinical studies and clinical trials, potential difficulties with or delays in timing with respect to regulatory approval processes, development by competitors of new technological innovations, dependence on key personnel, protection of proprietary technology, compliance with government regulations, ability to secure additional capital to fund operations, and risks associated with the economic challenges caused by economic uncertainty in various global markets caused by geopolitical instability and conflict. The Company and its contractors may experience disruptions in supply of items that are essential for its research and development activities, including, for example, raw materials and bulk drug substances that the Company imports from Europe and Canada used in the manufacturing of AMX0035, avexitide and any additional or future product candidates . |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2024 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Significant Accounting Policies The Company’s significant accounting policies are disclosed in the audited consolidated financial statements for the year ended December 31, 2023 and the notes thereto, which are included in the Company’s most recent Annual Report on Form 10-K. Since the date of those consolidated financial statements, there have been no material changes to its significant accounting policies. Basis of Presentation and Consolidation The accompanying condensed consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the U.S., or GAAP, and include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. In the opinion of the Company's management, all normal and recurring adjustments necessary for a fair presentation have been reflected. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the ASC, and Accounting Standards Update, or ASU, of the Financial Accounting Standards Board, or FASB. Use of Estimates The preparation of the condensed consolidated financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the financial statements and the reported amount of expenses during the reporting period. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates . New Accounting Pronouncements Not Yet Adopted In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvement to Income Tax Disclosures , or ASU 2023-09, to enhance the transparency and decision usefulness of income tax disclosures. ASU 2023-09 is effective for annual periods beginning after December 15, 2024, on a prospective basis. Early adoption and retrospective application are permitted. The Company is currently evaluating the impact of this ASU on its consolidated financial statements and related disclosures. In November 2023, the FASB issued ASU No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures , or ASU 2023-09, which requires public entities to disclose information about their reportable segments’ significant expenses on an interim and annual basis. ASU 2023-07 is effective for the Company beginning the year ended December 31, 2024. The Company is currently evaluating the impact of this ASU on its consolidated financial statements and related disclosures. |
Product Revenue, Net
Product Revenue, Net | 6 Months Ended |
Jun. 30, 2024 | |
Revenue from Contract with Customer [Abstract] | |
Product Revenue, Net | 3. PRODUCT REVENUE, NET To date, the Company’s only source of product revenue had been from sales of RELYVRIO, known as ALBRIOZA in Canada. Significant judgment was required in estimating GTN adjustments considering historical experience, payer channel mix (e.g., Medicare or Medicaid), current contract prices under applicable programs, unbilled claims and processing time lags and inventory levels in the distribution channel. In April 2024, the Company announced it had started a process with the FDA and Health Canada to voluntarily discontinue the marketing authorizations for RELYVRIO®/ALBRIOZA and remove the product from the market in the U.S. and Canada based on topline results from the Phase 3 PHOENIX trial. As a result, the Company did no t generate significant revenue from product sales for the three months ended June 30, 2024 . The following table reconciles gross product revenue to net product revenue (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Product revenue, gross $ 605 $ 108,405 $ 114,265 $ 192,958 GTN adjustments ( 1,628 ) ( 10,189 ) ( 26,645 ) ( 23,314 ) Product revenue, net $ ( 1,023 ) $ 98,216 $ 87,620 $ 169,644 The activity and ending reserve balance for GTN adjustments were as follows for the six months ended June 30, 2024 and 2023 (in thousands): Chargebacks and Cash Discounts Medicaid and Medicare Rebates Other Rebates, Returns, Discounts and Adjustments Total Ending balance at December 31, 2023 $ 3,143 $ 4,946 $ 11,073 $ 19,162 Provision related to sales in the current year 4,983 3,504 20,916 29,403 Adjustments related to prior period sales ( 1,696 ) — ( 1,062 ) ( 2,758 ) Credits and payments made ( 6,430 ) ( 7,664 ) ( 8,828 ) ( 22,922 ) Ending balance at June 30, 2024 $ — $ 786 $ 22,099 $ 22,885 Chargebacks and Cash Discounts Medicaid and Medicare Rebates Other Rebates, Returns, Discounts and Adjustments Total Ending balance at December 31, 2022 $ 648 $ 1,992 $ 1,664 $ 4,304 Provision related to sales in the current year 10,053 6,516 7,147 23,716 Adjustments related to prior period sales — ( 236 ) ( 166 ) ( 402 ) Credits and payments made ( 5,831 ) ( 4,294 ) ( 5,988 ) ( 16,113 ) Ending balance at June 30, 2023 $ 4,870 $ 3,978 $ 2,657 $ 11,505 Included in the ending reserve balance for GTN adjustments are chargebacks 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 the Company, discounts to customers for prompt payment and estimates for product returns. Chargebacks, discounts and returns are recorded as reductions of accounts receivable, net on the condensed consolidated balance sheets to the extent there are receivable balances to reduce. If there are net balance owed to customers, they are recorded as a component of accrued expenses on the condensed consolidated balance sheets. In addition, included in the ending reserve balance for GTN adjustments are Medicaid and Medicare rebates, other rebates for obligations under voluntary patient assistance programs, and accrued fees payable to customers. Medicaid and Medicare rebates, other rebates and fees are recorded as a component of accrued expenses on the condensed consolidated balance sheets. |
Short-Term Investments
Short-Term Investments | 6 Months Ended |
Jun. 30, 2024 | |
Investments, Debt and Equity Securities [Abstract] | |
Short-Term Investments | 4. SHORT-TERM INVESTMENTS The Company has classified all of its marketable securities at June 30, 2024 as “available-for-sale”. The Company records available-for-sale securities at fair value, with the unrealized gains and losses included as a separate component of other accumulated comprehensive income (loss). There were no realized gains or losses recognized during the three and six months ended June 30, 2024 and 2023. The Company adjusts the cost of available-for-sale debt securities for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion are included in interest income. The cost of securities sold is based on the specific identification method. The Company includes interest and dividends on securities classified as available-for-sale in interest income. Accrued interest receivable relating to the Company's available-for-sale securities is presented within prepaid expenses and other current assets in the accompanying condensed consolidated balance sheets, and amounted to less than $ 0.1 million and $ 0.5 million at June 30, 2024 and December 31, 2023, respectively. As of June 30, 2024 , there were no securities in an unrealized loss position for greater than 12 months. The Company does not intend to sell the investments and it is not more likely than not that the Company will be required to sell the investments before recovery of their amortized cost bases. The Company did not record an allowance for credit losses as of June 30, 2024. Short-term investments, which are classified as available-for-sale, consisted of the following (in thousands): Balance at June 30, 2024: Amortized Unrealized Unrealized Fair Treasury bills $ 234,842 $ 1 $ ( 30 ) $ 234,813 Total short-term investments $ 234,842 $ 1 $ ( 30 ) $ 234,813 Balance at December 31, 2023: Amortized Unrealized Unrealized Fair Treasury bills $ 196,098 $ 67 $ — $ 196,165 U.S. agency bonds 4,999 — ( 3 ) 4,996 Total short-term investments $ 201,097 $ 67 $ ( 3 ) $ 201,161 |
Inventories
Inventories | 6 Months Ended |
Jun. 30, 2024 | |
Inventory Disclosure [Abstract] | |
Inventories | 5. INVENTORIES Inventories consisted of the following (in thousands): June 30, 2024 December 31, 2023 Raw materials $ — $ 53,144 Work in process — 18,945 Finished goods — 11,191 Total inventories $ — $ 83,280 In April, 2024, the Company announced it had started a process with the FDA and Health Canada to voluntarily discontinue the marketing authorizations for RELYVRIO ® /ALBRIOZA and remove the product from the market in the U.S. and Canada based on topline results from the global Phase 3 PHOENIX trial. As a result, the Company recorded approximately $ 92.5 million of charges associated with the write-down of inventory for the six months ended June 30, 2024. Inventory write-downs for the six months ended June 30, 2023 were immaterial. |
Accrued Expenses
Accrued Expenses | 6 Months Ended |
Jun. 30, 2024 | |
Payables and Accruals [Abstract] | |
Accrued Expenses | 6. Accrued Expenses Accrued expenses consisted of the following (in thousands): June 30, 2024 December 31, 2023 Accrued external research and development $ 7,466 $ 12,625 Accrued benefits and incentive compensation 6,067 16,790 Accrued manufacturing 9,008 1,652 Accrued consulting and other professional fees 2,424 6,506 Accrued returns, rebates and co-pay assistance 22,059 16,063 Accrued royalties — 3,111 Accrued loss on future purchase commitments 12,604 — Accrued restructuring expenses 2,648 — Other accrued expenses 481 977 Total accrued expenses $ 62,757 $ 57,724 |
Fair Value Measurements
Fair Value Measurements | 6 Months Ended |
Jun. 30, 2024 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 7 . FAIR VALUE MEASUREMENTS The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values (in thousands): June 30, 2024 Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ 536 $ — $ — $ 536 Short-term investments: Treasury bills 234,813 — — 234,813 Total short-term investments 234,813 — — 234,813 Restricted cash equivalents 719 — — 719 Total financial assets $ 236,068 $ — $ — $ 236,068 December 31, 2023 Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ 76,710 $ — $ — $ 76,710 Short-term investments: Treasury bills 196,165 — — 196,165 U.S. agency bonds — 4,996 — 4,996 Total short-term investments 196,165 4,996 — 201,161 Restricted cash equivalents 719 — — 719 Total financial assets $ 273,594 $ 4,996 $ — $ 278,590 The Company classifies its money market funds and treasury bills as Level 1 assets under the fair value hierarchy, as these assets have been valued using quoted market prices for identical assets in active markets without any valuation adjustment. The Company classifies its U.S. agency bonds as Level 2 assets under the fair value hierarchy, as these assets have been valued using information obtained through a third-party pricing service at each balance sheet date, using observable market inputs for similar assets that may include trade information, broker or dealer quotes, bids, offers, or a combination of these data sources . |
Stock Option and Grant Plan
Stock Option and Grant Plan | 6 Months Ended |
Jun. 30, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Stock Option and Grant Plan | 8. Stock Option and Grant Plan Stock Incentive Plan General Option Information A summary of option activity for the six months ended June 30, 2024, is as follows: Number of Weighted- Weighted- Aggregate Outstanding at December 31, 2023 9,823,248 $ 18.39 7.9 $ 27,639 Granted 3,053,811 $ 8.56 Exercised ( 109,599 ) $ 1.54 $ 745 Cancelled or forfeited ( 2,025,321 ) $ 20.73 Outstanding at June 30, 2024 10,742,139 $ 15.33 7.9 $ 287 Options exercisable as of June 30, 2024 5,147,314 $ 15.72 6.9 $ 272 Options unvested as of June 30, 2024 5,594,825 $ 14.95 8.9 $ 15 Weighted average grant-date fair value of options granted during the period $ 5.62 The aggregate intrinsic value of options exercised during the six months ended June 30, 2024 and 2023 was $ 0.7 million and $ 16.2 million , respectively. The total fair value of stock options vested during the six months ended June 30, 2024 and 2023 was $ 23.7 million and $ 19.9 million , respectively. Restricted Stock Unit Activity A summary of restricted stock unit activity for the six months ended June 30, 2024, is as follows: Number of Shares Weighted Average Grant Date Fair Value Nonvested as of December 31, 2023 1,112,542 $ 24.80 Granted 2,816,142 $ 8.82 Vested ( 263,901 ) $ 24.37 Forfeited ( 753,883 ) $ 20.23 Nonvested as of June 30, 2024 2,910,900 $ 10.56 Summary of Stock-Based Compensation Expense Stock-based compensation expense for the three and six months ended June 30, 2024 and 2023, is as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Research and development $ 2,449 $ 2,535 $ 5,161 $ 4,426 Selling, general and administrative 7,121 7,415 14,333 13,025 Total stock-based compensation expense $ 9,570 $ 9,950 $ 19,494 $ 17,451 The following table summarizes unrecognized stock-based compensation expense as of June 30, 2024, by type of awards (in thousands), and the weighted-average period over which that expense is expected to be recognized (in years). The total unrecognized stock-based compensation expense will be adjusted for actual forfeitures as they occur. June 30, 2024 Unrecognized Expense Weighted-average Recognition Period Stock options $ 53,765 2.48 Restricted stock units $ 26,996 2.92 |
Net (Loss) Income Per Share
Net (Loss) Income Per Share | 6 Months Ended |
Jun. 30, 2024 | |
Earnings Per Share [Abstract] | |
Net (Loss) Income Per Share | 9. NET (LOSS) INCOME PER SHARE Net (Loss) Income per Share Basic earnings per share is computed by dividing net income (loss) by the weighted average number of common shares outstanding during the period. Diluted earnings per share is calculated based on the combined weighted average number of common shares and potentially dilutive shares, which include the assumed exercise of employee stock options and unvested restricted stock units. In computing diluted earnings per share, the Company utilizes the treasury stock method. A summary of the numerator and denominators used in the computation of earnings per share follows (in thousands, except share and per share data): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Numerator: Net (loss) income $ ( 72,700 ) $ 22,074 $ ( 191,493 ) $ 23,647 Denominator: Weighted-average shares used to compute basic net (loss) income per share 68,024,929 67,233,617 67,939,642 66,976,871 Dilutive effect of employee stock options and restricted stock units — 2,898,423 — 3,494,950 Weighted-average shares used to compute diluted net (loss) income per share 68,024,929 70,132,040 67,939,642 70,471,821 Net (loss) income per share Basic $ ( 1.07 ) $ 0.33 $ ( 2.82 ) $ 0.35 Diluted $ ( 1.07 ) $ 0.31 $ ( 2.82 ) $ 0.34 Because the Company reported a net loss for the three and six months ended June 30, 2024, basic and diluted net loss per share attributable to common stockholders were the same. All stock options and restricted stock units were excluded from the computation of diluted weighted-average shares outstanding because such securities would have an antidilutive impact for the three and six months ended June 30, 2024 . The following stock options and restricted stock units outstanding at each period end have been excluded from the calculation of diluted net income (loss) per share because their inclusion would have been antidilutive: Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Options to purchase common stock 10,742,139 5,831,498 10,742,139 3,198,508 Restricted stock units 2,910,900 567,671 2,910,900 85,658 Total excluded common stock equivalents 13,653,039 6,399,169 13,653,039 3,284,166 |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 2024 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 10. Commitments and Contingencies Letter of Credit Restricted cash equivalents consist of $ 0.2 million of cash serving as collateral for a letter of credit issued for the Company’s office space, and $ 0.5 million as collateral for a corporate credit card program. As of June 30, 2024 and December 31, 2023, the Company’s restricted cash equivalents balance was $ 0.7 million on its condensed consolidated balance sheets. Legal Proceedings On February 9, 2024, a putative class action lawsuit was filed in the U.S. District Court for the Southern District of New York against the Company and certain of its current and former officers ( Shih v. Amylyx Pharmaceuticals, Inc., et al. , Case Number 1:24-CV-00988, or the Shih Complaint). Plaintiff filed an amended complaint on June 24, 2024. The Shih Complaint asserts a claim against all defendants for alleged violations of Section 10(b) of the Exchange Act and Rule 10b-5 promulgated thereunder and a claim under Section 20(a) against certain current and former officers as alleged controlling persons. The Shih Complaint alleges that defendants made materially false and misleading statements related to the commercial results and prospects for RELYVRIO. The Shih Complaint seeks unspecified damages, interest, costs and attorneys’ fees, and other unspecified relief that the court deems appropriate. The Company intends to defend against the Shih Complaint vigorously, and filed a motion to transfer venue on July 11, 2024. On July 12, 2024, the court granted the motion to transfer, ordering the clerk to transfer the case to the United States District Court for the District of Massachusetts, or the Order. The parties await a new docket number and assigned judge in light of the Order regarding the transfer to the District of Massachusetts. At this time, an estimate of the impact, if any, of the claims made in the Shih Complaint cannot be made. Royalty Payments Between August 2016 and February 2019, the Company entered into grant agreements with the ALS Association, ALS Finding a Cure Foundation, Alzheimer’s Drug Discovery Foundation, Alzheimer’s Association and Cure Alzheimer’s Fund, or Grantors. Under the terms of the respective grant agreements among the Company, Alzheimer’s Drug Discovery Foundation, the Alzheimer’s Association, and Cure Alzheimer’s Fund, the Company will make royalty payments up to the maximum amount of $ 15.0 million to each Grantor (or $ 45.0 million in aggregate). As the conditions that would trigger royalty payments under the agreements have not occurred, no amounts have been recorded in the condensed consolidated financial statements for the three and six months ended June 30, 2024 and 2023. Purchase Commitments The Company enters into agreements in the normal course of business with contract manufacturing organizations for raw material purchases and manufacturing services. As disclosed in Note 18 Commitments and Contingencies to the Company's financial statements for the year ended December 31, 2023, including in the Company’s Annual Report on Form 10-K, as of December 31, 2023, the Company had committed approximately $ 195.0 million under these agreements related to raw material purchases and manufacturing services, which were expected to be paid through 2028. While these commitments are estimates and are subject to changes based on the outcomes of ongoing negotiations with the contract manufacturing organizations, as of June 30, 2024, the Company estimated approximately $ 31.7 million of obligations under these agreements, which are expected to be paid through 2025. The actual obligations may differ from these estimates, and such differences will be reflected in the Company's financial statements in future periods as they become known. The Company recognized a loss on purchase commitments of $ 7.4 million and $ 25.3 million for the three and six months ended June 30, 2024 , respectively, which was recorded to cost of sales on the condensed consolidated statement of operations and as part of accrued expenses on the condensed consolidated balance sheet. The purchase commitment loss is based on an estimate of future commitments related to supply agreements with third party vendors for which the Company does not expect to have related sales. |
Restructuring
Restructuring | 6 Months Ended |
Jun. 30, 2024 | |
Restructuring and Related Activities [Abstract] | |
Restructuring | 11. Restructuring In April 2024, the Company announced a restructuring plan designed to focus the Company’s resources on key clinical and preclinical programs, or the Restructuring Plan. The Restructuring Plan includes a reduction in force which is expected to reduce the Company’s workforce by approximately 70 % and a decrease in external financial commitments outside of its priority areas. The Company substantially completed the Restructuring Plan in the second quarter of 2024. Restructuring expense consists primarily of employee severance and termination benefits, contract termination costs, impairment of long-lived assets and other costs. Liabilities for costs associated with a restructuring activity are recognized when the liability is incurred and are measured at fair value. According to ASC 420, Exit or Disposal Cost Obligations , one-time employee severance and termination benefits are expensed at the date the entity notifies the employee of the plan, unless the employee must provide future service, in which case the benefits are expensed in the period when the service ends. One-time termination benefits primarily include severance, continuation of health insurance coverage, and other benefits such as outplacement support services for a specified period of time. In connection with the Restructuring Plan, the Company performed an impairment evaluation of its long-lived assets resulting in an impairment charge of $ 0.9 million in three months ended June 30, 2024 related to the impairment of capitalized internal-use software. Restructuring expenses for the three months ended June 30, 2024 were as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Severance and employee benefit costs $ 21,812 $ — $ 21,812 $ — Contract termination costs, impairment of assets and other costs $ 1,039 — $ 1,039 — Total $ 22,851 $ — $ 22,851 $ — The Company’s accrued restructuring expenses were $ 2.6 million and are included in accrued expenses on its condensed consolidated balance sheet as of June 30, 2024 . These expenses are expected to be paid through December 31, 2024. |
Subsequent Events
Subsequent Events | 6 Months Ended |
Jun. 30, 2024 | |
Subsequent Events [Abstract] | |
Subsequent Events | 12. Subsequent Events On July 9, 2024, the Company completed the acquisition of substantially all the assets and interests in the development, manufacture and commercialization of avexitide from Eiger BioPharmaceuticals, Inc., (the “Seller”), for $ 35.1 million, subject to final adjustment. As part of the transaction, the Company assumed certain contractual obligations from the Seller, including royalty obligations between 4 % and 7 % on future sales owed to certain academic institutions and individuals. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2024 | |
Accounting Policies [Abstract] | |
Basis of Presentation and Consolidation | Basis of Presentation and Consolidation The accompanying condensed consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the U.S., or GAAP, and include the accounts of the Company and its wholly owned subsidiaries. All intercompany balances and transactions have been eliminated in consolidation. In the opinion of the Company's management, all normal and recurring adjustments necessary for a fair presentation have been reflected. Any reference in these notes to applicable guidance is meant to refer to the authoritative GAAP as found in the ASC, and Accounting Standards Update, or ASU, of the Financial Accounting Standards Board, or FASB. |
Use of Estimates | Use of Estimates The preparation of the condensed consolidated financial statements in conformity with GAAP requires the Company’s management to make estimates and assumptions that affect the reported amounts of assets and liabilities at the date of the financial statements and the reported amount of expenses during the reporting period. Actual results could differ from those estimates. Management considers many factors in selecting appropriate financial accounting policies in developing the estimates and assumptions that are used in the preparation of the financial statements. Management must apply significant judgment in this process. Management’s estimation process often may yield a range of potentially reasonable estimates and management must select an amount that falls within that range of reasonable estimates |
New Accounting Pronouncements Not Yet Adopted | New Accounting Pronouncements Not Yet Adopted In December 2023, the FASB issued ASU No. 2023-09, Income Taxes (Topic 740): Improvement to Income Tax Disclosures , or ASU 2023-09, to enhance the transparency and decision usefulness of income tax disclosures. ASU 2023-09 is effective for annual periods beginning after December 15, 2024, on a prospective basis. Early adoption and retrospective application are permitted. The Company is currently evaluating the impact of this ASU on its consolidated financial statements and related disclosures. In November 2023, the FASB issued ASU No. 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures , or ASU 2023-09, which requires public entities to disclose information about their reportable segments’ significant expenses on an interim and annual basis. ASU 2023-07 is effective for the Company beginning the year ended December 31, 2024. The Company is currently evaluating the impact of this ASU on its consolidated financial statements and related disclosures. |
Product Revenue, Net (Tables)
Product Revenue, Net (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Revenue from Contract with Customer [Abstract] | |
Summary of Gross Product Revenue to Net Product Revenue | The following table reconciles gross product revenue to net product revenue (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Product revenue, gross $ 605 $ 108,405 $ 114,265 $ 192,958 GTN adjustments ( 1,628 ) ( 10,189 ) ( 26,645 ) ( 23,314 ) Product revenue, net $ ( 1,023 ) $ 98,216 $ 87,620 $ 169,644 |
Schedule of Activity and Ending Reserve Balance for GTN Adjustments | The activity and ending reserve balance for GTN adjustments were as follows for the six months ended June 30, 2024 and 2023 (in thousands): Chargebacks and Cash Discounts Medicaid and Medicare Rebates Other Rebates, Returns, Discounts and Adjustments Total Ending balance at December 31, 2023 $ 3,143 $ 4,946 $ 11,073 $ 19,162 Provision related to sales in the current year 4,983 3,504 20,916 29,403 Adjustments related to prior period sales ( 1,696 ) — ( 1,062 ) ( 2,758 ) Credits and payments made ( 6,430 ) ( 7,664 ) ( 8,828 ) ( 22,922 ) Ending balance at June 30, 2024 $ — $ 786 $ 22,099 $ 22,885 Chargebacks and Cash Discounts Medicaid and Medicare Rebates Other Rebates, Returns, Discounts and Adjustments Total Ending balance at December 31, 2022 $ 648 $ 1,992 $ 1,664 $ 4,304 Provision related to sales in the current year 10,053 6,516 7,147 23,716 Adjustments related to prior period sales — ( 236 ) ( 166 ) ( 402 ) Credits and payments made ( 5,831 ) ( 4,294 ) ( 5,988 ) ( 16,113 ) Ending balance at June 30, 2023 $ 4,870 $ 3,978 $ 2,657 $ 11,505 |
Short-Term Investments (Tables)
Short-Term Investments (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Investments, Debt and Equity Securities [Abstract] | |
Schedule of Short-term Investments Classified as Available-for-sale | Short-term investments, which are classified as available-for-sale, consisted of the following (in thousands): Balance at June 30, 2024: Amortized Unrealized Unrealized Fair Treasury bills $ 234,842 $ 1 $ ( 30 ) $ 234,813 Total short-term investments $ 234,842 $ 1 $ ( 30 ) $ 234,813 Balance at December 31, 2023: Amortized Unrealized Unrealized Fair Treasury bills $ 196,098 $ 67 $ — $ 196,165 U.S. agency bonds 4,999 — ( 3 ) 4,996 Total short-term investments $ 201,097 $ 67 $ ( 3 ) $ 201,161 |
Inventories (Tables)
Inventories (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Inventory Disclosure [Abstract] | |
Summary of Inventories | Inventories consisted of the following (in thousands): June 30, 2024 December 31, 2023 Raw materials $ — $ 53,144 Work in process — 18,945 Finished goods — 11,191 Total inventories $ — $ 83,280 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Payables and Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consisted of the following (in thousands): June 30, 2024 December 31, 2023 Accrued external research and development $ 7,466 $ 12,625 Accrued benefits and incentive compensation 6,067 16,790 Accrued manufacturing 9,008 1,652 Accrued consulting and other professional fees 2,424 6,506 Accrued returns, rebates and co-pay assistance 22,059 16,063 Accrued royalties — 3,111 Accrued loss on future purchase commitments 12,604 — Accrued restructuring expenses 2,648 — Other accrued expenses 481 977 Total accrued expenses $ 62,757 $ 57,724 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Fair Value Disclosures [Abstract] | |
Schedule of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis | The following tables present information about the Company’s financial assets and liabilities measured at fair value on a recurring basis and indicates the level of the fair value hierarchy utilized to determine such fair values (in thousands): June 30, 2024 Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ 536 $ — $ — $ 536 Short-term investments: Treasury bills 234,813 — — 234,813 Total short-term investments 234,813 — — 234,813 Restricted cash equivalents 719 — — 719 Total financial assets $ 236,068 $ — $ — $ 236,068 December 31, 2023 Level 1 Level 2 Level 3 Total Assets: Cash equivalents $ 76,710 $ — $ — $ 76,710 Short-term investments: Treasury bills 196,165 — — 196,165 U.S. agency bonds — 4,996 — 4,996 Total short-term investments 196,165 4,996 — 201,161 Restricted cash equivalents 719 — — 719 Total financial assets $ 273,594 $ 4,996 $ — $ 278,590 |
Stock Option and Grant Plan (Ta
Stock Option and Grant Plan (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Summary of Stock Option Activity | A summary of option activity for the six months ended June 30, 2024, is as follows: Number of Weighted- Weighted- Aggregate Outstanding at December 31, 2023 9,823,248 $ 18.39 7.9 $ 27,639 Granted 3,053,811 $ 8.56 Exercised ( 109,599 ) $ 1.54 $ 745 Cancelled or forfeited ( 2,025,321 ) $ 20.73 Outstanding at June 30, 2024 10,742,139 $ 15.33 7.9 $ 287 Options exercisable as of June 30, 2024 5,147,314 $ 15.72 6.9 $ 272 Options unvested as of June 30, 2024 5,594,825 $ 14.95 8.9 $ 15 Weighted average grant-date fair value of options granted during the period $ 5.62 |
Summary of Restricted Stock Unit Activity | A summary of restricted stock unit activity for the six months ended June 30, 2024, is as follows: Number of Shares Weighted Average Grant Date Fair Value Nonvested as of December 31, 2023 1,112,542 $ 24.80 Granted 2,816,142 $ 8.82 Vested ( 263,901 ) $ 24.37 Forfeited ( 753,883 ) $ 20.23 Nonvested as of June 30, 2024 2,910,900 $ 10.56 |
Summary of Stock-Based Compensation Expense | Stock-based compensation expense for the three and six months ended June 30, 2024 and 2023, is as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Research and development $ 2,449 $ 2,535 $ 5,161 $ 4,426 Selling, general and administrative 7,121 7,415 14,333 13,025 Total stock-based compensation expense $ 9,570 $ 9,950 $ 19,494 $ 17,451 |
Summary of Unrecognized Stock-Based Compensation Expense and Weighted-Average Recognition Period | The following table summarizes unrecognized stock-based compensation expense as of June 30, 2024, by type of awards (in thousands), and the weighted-average period over which that expense is expected to be recognized (in years). The total unrecognized stock-based compensation expense will be adjusted for actual forfeitures as they occur. June 30, 2024 Unrecognized Expense Weighted-average Recognition Period Stock options $ 53,765 2.48 Restricted stock units $ 26,996 2.92 |
Net (Loss) Income Per Share (Ta
Net (Loss) Income Per Share (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Earnings Per Share [Abstract] | |
Summary of the Numerator and Denominators Used in Computation of Earnings Per Share | A summary of the numerator and denominators used in the computation of earnings per share follows (in thousands, except share and per share data): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Numerator: Net (loss) income $ ( 72,700 ) $ 22,074 $ ( 191,493 ) $ 23,647 Denominator: Weighted-average shares used to compute basic net (loss) income per share 68,024,929 67,233,617 67,939,642 66,976,871 Dilutive effect of employee stock options and restricted stock units — 2,898,423 — 3,494,950 Weighted-average shares used to compute diluted net (loss) income per share 68,024,929 70,132,040 67,939,642 70,471,821 Net (loss) income per share Basic $ ( 1.07 ) $ 0.33 $ ( 2.82 ) $ 0.35 Diluted $ ( 1.07 ) $ 0.31 $ ( 2.82 ) $ 0.34 |
Schedule of Antidilutive Shares Excluded from the Calculation of Diluted Net Income (Loss) Per Share | The following stock options and restricted stock units outstanding at each period end have been excluded from the calculation of diluted net income (loss) per share because their inclusion would have been antidilutive: Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Options to purchase common stock 10,742,139 5,831,498 10,742,139 3,198,508 Restricted stock units 2,910,900 567,671 2,910,900 85,658 Total excluded common stock equivalents 13,653,039 6,399,169 13,653,039 3,284,166 |
Restructuring (Tables)
Restructuring (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Restructuring and Related Activities [Abstract] | |
Schedule of Restructuring Expenses | Restructuring expenses for the three months ended June 30, 2024 were as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Severance and employee benefit costs $ 21,812 $ — $ 21,812 $ — Contract termination costs, impairment of assets and other costs $ 1,039 — $ 1,039 — Total $ 22,851 $ — $ 22,851 $ — |
Nature of the Business - Additi
Nature of the Business - Additional Information (Details) | 6 Months Ended |
Jun. 30, 2024 | |
Nature Of The Information [Abstract] | |
Participants dose description | The Company dosed the first participant in the HELIOS trial, a Phase 2 trial of AMX0035 for the treatment of Wolfram syndrome, in April 2023. Amylyx plans to present the HELIOS topline data for all 12 participants at Week 24, including all of the longer-term data available for participants who have reached their Week 36 or Week 48 visit at that time, at a medical meeting this fall. The Company is engaging with stakeholders, including the FDA, and planning for a single Phase 3 clinical trial and will share additional details on the clinical trial design once finalized. The Company dosed the first participant in the ORION trial, a Phase 2b/3 global clinical trial of AMX0035 for the treatment of PSP, in December 2023. Data from an interim analysis of the ORION are expected in mid-2025. The Company is also advancing additional drug candidates developed by Amylyx for neurodegenerative diseases including AMX0114, a potent antisense oligonucleotide targeting inhibition of Calpain-2, a key contributor to the axonal (also known as Wallerian) degeneration pathway. The Company is preparing to initiate a multiple ascending dose clinical trial of AMX0114 in people living with ALS in the second half of 2024. |
Product Revenue, Net - Addition
Product Revenue, Net - Additional Information (Details) - USD ($) | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | ||||
Product revenue, net | $ (1,023,000) | $ 98,216,000 | $ 87,620,000 | $ 169,644,000 |
Revenue from product sales | $ 0 |
Product Revenue, Net - Summary
Product Revenue, Net - Summary of Gross Product Revenue to Net product Revenue (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Revenue from Contract with Customer [Abstract] | ||||
Product revenue, gross | $ 605 | $ 108,405 | $ 114,265 | $ 192,958 |
GTN adjustments | (1,628) | (10,189) | (26,645) | (23,314) |
Product revenue, net | $ (1,023) | $ 98,216 | $ 87,620 | $ 169,644 |
Product Revenue, Net - Schedule
Product Revenue, Net - Schedule of Activity and Ending Reserve Balance for GTN Adjustments (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
Disaggregation of Revenue [Line Items] | ||
GTN Adjustments, Ending balance | $ 19,162 | $ 4,304 |
GTN Adjustments, Provision related to sales in the current year | 29,403 | 23,716 |
GTN Adjustments, Adjustments related to prior period sales | (2,758) | (402) |
GTN Adjustments, Credits and payments made | (22,922) | (16,113) |
GTN Adjustments, Ending balance | 22,885 | 11,505 |
Chargebacks and Cash Discounts | ||
Disaggregation of Revenue [Line Items] | ||
GTN Adjustments, Ending balance | 3,143 | 648 |
GTN Adjustments, Provision related to sales in the current year | 4,983 | 10,053 |
GTN Adjustments, Adjustments related to prior period sales | (1,696) | |
GTN Adjustments, Credits and payments made | (6,430) | (5,831) |
GTN Adjustments, Ending balance | 4,870 | |
Medicaid and Medicare Rebates | ||
Disaggregation of Revenue [Line Items] | ||
GTN Adjustments, Ending balance | 4,946 | 1,992 |
GTN Adjustments, Provision related to sales in the current year | 3,504 | 6,516 |
GTN Adjustments, Adjustments related to prior period sales | (236) | |
GTN Adjustments, Credits and payments made | (7,664) | (4,294) |
GTN Adjustments, Ending balance | 786 | 3,978 |
Other Rebates, Returns, Discounts and Adjustments | ||
Disaggregation of Revenue [Line Items] | ||
GTN Adjustments, Ending balance | 11,073 | 1,664 |
GTN Adjustments, Provision related to sales in the current year | 20,916 | 7,147 |
GTN Adjustments, Adjustments related to prior period sales | (1,062) | (166) |
GTN Adjustments, Credits and payments made | (8,828) | (5,988) |
GTN Adjustments, Ending balance | $ 22,099 | $ 2,657 |
Short-Term Investments - Additi
Short-Term Investments - Additional Information (Details) | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2024 USD ($) Security | Jun. 30, 2023 USD ($) | Jun. 30, 2024 USD ($) Security | Jun. 30, 2023 USD ($) | Dec. 31, 2023 USD ($) | |
Debt Securities, Available-for-Sale [Line Items] | |||||
Realized gains | $ 0 | $ 0 | $ 0 | $ 0 | |
Realized loss | $ 0 | $ 0 | $ 0 | $ 0 | |
Debt securities available-for-sale in an unrealized loss position for greater than 12 months | Security | 0 | 0 | |||
Available-for-Sale Securities | Maximum | |||||
Debt Securities, Available-for-Sale [Line Items] | |||||
Accrued interest receivable | $ 100,000 | $ 100,000 | $ 500,000 |
Short-Term Investments - Schedu
Short-Term Investments - Schedule of Short-term Investments Classified as Available-for-sale (Details) - Short-term Investments - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | $ 234,842 | $ 201,097 |
Unrealized Gain | 1 | 67 |
Unrealized Loss | (30) | (3) |
Fair Value | 234,813 | 201,161 |
Treasury Bills | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 234,842 | 196,098 |
Unrealized Gain | 1 | 67 |
Unrealized Loss | (30) | |
Fair Value | $ 234,813 | 196,165 |
Agency Bonds | ||
Schedule Of Available For Sale Securities [Line Items] | ||
Amortized Cost Basis | 4,999 | |
Unrealized Loss | (3) | |
Fair Value | $ 4,996 |
Inventories - Summary of Invent
Inventories - Summary of Inventory (Details) $ in Thousands | Dec. 31, 2023 USD ($) |
Inventory Disclosure [Abstract] | |
Raw materials | $ 53,144 |
Work in process | 18,945 |
Finished goods | 11,191 |
Total inventories | $ 83,280 |
Inventories - Additional Inform
Inventories - Additional Information (Details) $ in Millions | 6 Months Ended |
Jun. 30, 2024 USD ($) | |
Inventory Disclosure [Abstract] | |
Inventory write-downs | $ 92.5 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Payables and Accruals [Abstract] | ||
Accrued external research and development | $ 7,466 | $ 12,625 |
Accrued benefits and incentive compensation | 6,067 | 16,790 |
Accrued Manufacturing | 9,008 | 1,652 |
Accrued consulting and other professional fees | 2,424 | 6,506 |
Accrued returns, rebates and co-pay assistance | 22,059 | 16,063 |
Accrued royalties | 3,111 | |
Accrued loss on future purchase commitments | 12,604 | |
Accrued restructuring expenses | 2,648 | |
Other accrued expenses | 481 | 977 |
Total accrued expenses | $ 62,757 | $ 57,724 |
Fair Value Measurements - Sched
Fair Value Measurements - Schedule of Financial Assets and Liabilities Measured at Fair Value on Recurring Basis (Detail) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Short-term investments | $ 234,813 | $ 201,161 |
Fair Value Measurements, Recurring | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Short-term investments | 234,813 | 201,161 |
Assets measured at fair value | 236,068 | 278,590 |
Fair Value Measurements, Recurring | Cash equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 536 | 76,710 |
Fair Value Measurements, Recurring | Treasury Bills | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Short-term investments | 234,813 | 196,165 |
Fair Value Measurements, Recurring | U.S. Agency Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Short-term investments | 4,996 | |
Fair Value Measurements, Recurring | Restricted Cash Equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | 719 | 719 |
Fair Value Measurements, Recurring | Level 1 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Short-term investments | 234,813 | 196,165 |
Assets measured at fair value | 236,068 | 273,594 |
Fair Value Measurements, Recurring | Level 1 | Cash equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Cash equivalents | 536 | 76,710 |
Fair Value Measurements, Recurring | Level 1 | Treasury Bills | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Short-term investments | 234,813 | 196,165 |
Fair Value Measurements, Recurring | Level 1 | Restricted Cash Equivalents | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Assets measured at fair value | $ 719 | 719 |
Fair Value Measurements, Recurring | Level 2 | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Short-term investments | 4,996 | |
Assets measured at fair value | 4,996 | |
Fair Value Measurements, Recurring | Level 2 | U.S. Agency Bonds | ||
Fair Value Assets And Liabilities Measured On Recurring And Nonrecurring Basis [Line Items] | ||
Short-term investments | $ 4,996 |
Stock Option and Grant Plan - S
Stock Option and Grant Plan - Summary of Stock Option Activity (Details) - USD ($) $ / shares in Units, $ in Thousands | 6 Months Ended | 12 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | Dec. 31, 2023 | |
Share-Based Payment Arrangement [Abstract] | |||
Number of Options, Outstanding | 9,823,248 | ||
Number of Options, Granted | 3,053,811 | ||
Number of Options, Exercised | (109,599) | ||
Number of Options, Cancelled or forfeited | (2,025,321) | ||
Number of Options, Outstanding | 10,742,139 | 9,823,248 | |
Number of Options, Options exercisable | 5,147,314 | ||
Number of Options, Options unvested | 5,594,825 | ||
Weighted average grant-date fair value of options granted during the period | $ 5.62 | ||
Weighted-Average Exercise Price, Outstanding | 18.39 | ||
Weighted-Average Exercise Price, Granted | 8.56 | ||
Weighted-Average Exercise Price, Exercised | 1.54 | ||
Weighted-Average Exercise Price, Cancelled or forfeited | 20.73 | ||
Weighted-Average Exercise Price, Outstanding | 15.33 | $ 18.39 | |
Weighted-Average Exercise Price, Options exercisable | 15.72 | ||
Weighted-Average Exercise Price, Options unvested | $ 14.95 | ||
Weighted- Average Remaining Contractual Term (in years), Outstanding | 7 years 10 months 24 days | 7 years 10 months 24 days | |
Weighted- Average Remaining Contractual Term (in years), Options exercisable | 6 years 10 months 24 days | ||
Weighted- Average Remaining Contractual Term (in years), Options unvested | 8 years 10 months 24 days | ||
Aggregate Intrinsic Value, Outstanding | $ 27,639 | ||
Aggregate intrinsic value, exercised | 745 | $ 16,200 | |
Aggregate Intrinsic Value, Outstanding | 287 | $ 27,639 | |
Aggregate Intrinsic Value, Options exercisable | 272 | ||
Aggregate Intrinsic Value, Options unvested | $ 15 |
Stock Option and Grant Plan - A
Stock Option and Grant Plan - Additional Information (Details) - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | ||
Aggregate intrinsic value, exercised | $ 745 | $ 16,200 |
Total fair value of stock options vested | $ 23,700 | $ 19,900 |
Stock Option and Grant Plan -_2
Stock Option and Grant Plan - Summary of Restricted Stock Unit Activity (Details) - Restricted Stock Unit | 6 Months Ended |
Jun. 30, 2024 $ / shares shares | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Number of shares, Nonvested | shares | 1,112,542 |
Number of shares, Granted | shares | 2,816,142 |
Number of shares, Vested | shares | (263,901) |
Number of shares, Forfeited | shares | (753,883) |
Number of shares, Nonvested | shares | 2,910,900 |
Weighted Average Grant Date Fair Value, Nonvested | $ / shares | $ 24.8 |
Weighted Average Grant Date Fair Value, Granted | $ / shares | 8.82 |
Weighted Average Grant Date Fair Value, Vested | $ / shares | 24.37 |
Weighted Average Grant Date Fair Value, Forfeited | $ / shares | 20.23 |
Weighted Average Grant Date Fair Value, Nonvested | $ / shares | $ 10.56 |
Stock Option and Grant Plan -_3
Stock Option and Grant Plan - Summary of Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 9,570 | $ 9,950 | $ 19,494 | $ 17,451 |
Research and Development | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | 2,449 | 2,535 | 5,161 | 4,426 |
Selling, General and Administrative | ||||
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | ||||
Total stock-based compensation expense | $ 7,121 | $ 7,415 | $ 14,333 | $ 13,025 |
Stock Option and Grant Plan -_4
Stock Option and Grant Plan - Summary of Unrecognized Stock-Based Compensation Expense and Weighted-Average Recognition Period (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2024 USD ($) | |
Stock Options | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Unrecognized Expense | $ 53,765 |
Weighted-average Recognition Period | 2 years 5 months 23 days |
Restricted Stock Units | |
Share Based Compensation Arrangement By Share Based Payment Award [Line Items] | |
Unrecognized Expense | $ 26,996 |
Weighted-average Recognition Period | 2 years 11 months 1 day |
Net (Loss) Income Per Share - S
Net (Loss) Income Per Share - Summary of the Numerator and Denominators Used in Computation of Earnings Per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2024 | Mar. 31, 2024 | Jun. 30, 2023 | Mar. 31, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Numerator: | ||||||
Net (loss) income | $ (72,700) | $ (118,793) | $ 22,074 | $ 1,573 | $ (191,493) | $ 23,647 |
Denominator: | ||||||
Weighted-average shares used to compute basic net (loss) income per share | 68,024,929 | 67,233,617 | 67,939,642 | 66,976,871 | ||
Dilutive effect of employee stock options and restricted stock units | 2,898,423 | 3,494,950 | ||||
Weighted-average shares used to compute diluted net (loss) income per share | 68,024,929 | 70,132,040 | 67,939,642 | 70,471,821 | ||
Net (loss) income per share - basic | $ (1.07) | $ 0.33 | $ (2.82) | $ 0.35 | ||
Net (loss) income per share - diluted | $ (1.07) | $ 0.31 | $ (2.82) | $ 0.34 |
Net (Loss) Income Per Share -_2
Net (Loss) Income Per Share - Schedule of Antidilutive Shares Excluded from the Calculation of Diluted Net Income (Loss) Per Share (Details) - shares | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Total excluded common stock equivalents | 13,653,039 | 6,399,169 | 13,653,039 | 3,284,166 |
Options to Purchase Common Stock | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Total excluded common stock equivalents | 10,742,139 | 5,831,498 | 10,742,139 | 3,198,508 |
Restricted Stock Unit | ||||
Antidilutive Securities Excluded From Computation Of Earnings Per Share [Line Items] | ||||
Total excluded common stock equivalents | 2,910,900 | 567,671 | 2,910,900 | 85,658 |
Commitment and Contingencies -
Commitment and Contingencies - Additional Information (Details) - USD ($) | 3 Months Ended | 6 Months Ended | |||
Jun. 30, 2024 | Jun. 30, 2024 | Dec. 31, 2023 | Jun. 30, 2023 | Feb. 28, 2019 | |
Commitments And Contingencies [Line Items] | |||||
Restricted cash | $ 700,000 | $ 700,000 | $ 700,000 | ||
Purchase commitments and obligations | 31,700,000 | 31,700,000 | |||
Accrued expenses | 62,757,000 | 62,757,000 | 57,724,000 | ||
Raw Material Purchases and Manufacturing Services | |||||
Commitments And Contingencies [Line Items] | |||||
Purchase commitments and obligations | $ 195,000,000 | ||||
Purchase Commitments | |||||
Commitments And Contingencies [Line Items] | |||||
Loss on purchase commitments | 7,400,000 | 25,300,000 | |||
ALS Association and ALS Finding a Cure | |||||
Commitments And Contingencies [Line Items] | |||||
Accrued royalities grant agreements | 0 | 0 | $ 0 | ||
Royalty Payments Agreements with Grantors | Alzheimer's Drug Discovery Foundation, the Alzheimer's Association, and Cure Alzheimer's Fund | |||||
Commitments And Contingencies [Line Items] | |||||
Maximum Royalty amount payable to each grantor | $ 15,000,000 | ||||
Aggregate royalty amount payable to grantor | $ 45,000,000 | ||||
Collateral for Letter of Credit Issued | |||||
Commitments And Contingencies [Line Items] | |||||
Restricted cash | 200,000 | 200,000 | |||
Collateral for Corporate Credit Card Program | |||||
Commitments And Contingencies [Line Items] | |||||
Restricted cash | $ 500,000 | $ 500,000 |
Restructuring - Additional Info
Restructuring - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | |
Apr. 30, 2024 | Jun. 30, 2024 | |
Restructuring Cost and Reserve [Line Items] | ||
Accrued restructuring expenses | $ 2,648 | |
Restructuring Plan | ||
Restructuring Cost and Reserve [Line Items] | ||
Percentage of reduction in workforce | 70% | |
Impairment of capitalized internal-use software | 900 | |
Accrued restructuring expenses | $ 2,600 |
Restructuring - Schedule of Res
Restructuring - Schedule of Restructuring Expenses (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended |
Jun. 30, 2024 | Jun. 30, 2024 | |
Restructuring and Related Activities [Abstract] | ||
Severance and employee benefit costs | $ 21,812 | $ 21,812 |
Contract termination costs, impairment of assets and other costs | 1,039 | 1,039 |
Total | $ 22,851 | $ 22,851 |
Subsequent Events - Additional
Subsequent Events - Additional Information (Details) - Subsequent Event - Eiger BioPharmaceuticals, Inc. $ in Millions | Jul. 09, 2024 USD ($) |
Subsequent Event [Line Items] | |
Transaction costs | $ 35.1 |
Maximum | |
Subsequent Event [Line Items] | |
Percentage of royalty obligations on future sales | 7% |
Minimum | |
Subsequent Event [Line Items] | |
Percentage of royalty obligations on future sales | 4% |