Document And Entity Information
Document And Entity Information - shares | 9 Months Ended | |
Sep. 30, 2023 | Oct. 27, 2023 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Sep. 30, 2023 | |
Document Fiscal Year Focus | 2023 | |
Document Fiscal Period Focus | Q3 | |
Current Fiscal Year End Date | --12-31 | |
Entity File Number | 001-38981 | |
Entity Registrant Name | Mirum Pharmaceuticals, Inc. | |
Entity Central Index Key | 0001759425 | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 83-1281555 | |
Entity Address, Address Line One | 950 Tower Lane, Suite 1050, | |
Entity Address, City or Town | Foster City | |
Entity Address, State or Province | CA | |
Entity Address, Postal Zip Code | 94404 | |
City Area Code | 650 | |
Local Phone Number | 667-4085 | |
Trading Symbol | MIRM | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Non-accelerated Filer | |
Entity Small Business | true | |
Entity Emerging Growth Company | true | |
Entity Ex Transition Period | true | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 46,614,274 | |
Document Quarterly Report | true | |
Document Transition Report | false | |
Title of 12(b) Security | Common stock, par value $0.0001 per share | |
Security Exchange Name | NASDAQ |
Condensed Consolidated Balance
Condensed Consolidated Balance Sheets - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Current assets: | ||
Cash and cash equivalents | $ 303,059 | $ 28,003 |
Short-term investments | 2,971 | 123,716 |
Accounts receivable | 47,834 | 23,994 |
Inventory | 22,250 | 5,565 |
Prepaid expenses and other current assets | 12,465 | 8,947 |
Total current assets | 388,579 | 190,225 |
Restricted cash equivalents | 0 | 100,000 |
Property and equipment, net | 787 | 914 |
Operating lease right-of-use assets | 1,428 | 1,431 |
Intangible assets, net | 258,338 | 58,954 |
Other assets | 1,510 | 1,382 |
Total assets | 650,642 | 352,906 |
Current liabilities: | ||
Accounts payable | 7,369 | 8,690 |
Accrued expenses | 66,213 | 54,018 |
Operating lease liabilities | 1,082 | 931 |
Derivative liability | 0 | 1,090 |
Total current liabilities | 74,664 | 64,729 |
Revenue interest liability, net | 0 | 140,351 |
Operating lease liabilities, noncurrent | 868 | 1,257 |
Convertible notes payable, net | 306,022 | 0 |
Other liabilities | 7 | 4,532 |
Total liabilities | 381,561 | 210,869 |
Commitments and contingencies | ||
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value; 10,000,000 shares authorized, and no shares issued and outstanding as of September 30, 2023 and December 31, 2022 | 0 | 0 |
Common stock, $0.0001 par value; 200,000,000 shares authorized; 46,595,745 and 36,956,345 shares issued and outstanding as of September 30, 2023 and December 31, 2022, respectively | 5 | 4 |
Additional paid-in capital | 791,114 | 535,074 |
Accumulated deficit | (520,580) | (392,824) |
Accumulated other comprehensive loss | (1,458) | (217) |
Total stockholders’ equity | 269,081 | 142,037 |
Total liabilities and stockholders’ equity | $ 650,642 | $ 352,906 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Sep. 30, 2023 | Dec. 31, 2022 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 10,000,000 | 10,000,000 |
Preferred stock, shares, issued | 0 | 0 |
Preferred stock, shares, outstanding | 0 | 0 |
Common stock, par value | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares, issued | 46,595,745 | 36,956,345 |
Common stock, shares, outstanding | 46,595,745 | 36,956,345 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Revenues [Abstract] | ||||
Total revenue | $ 47,725 | $ 18,780 | $ 116,820 | $ 49,156 |
Operating expenses: | ||||
Cost of sales | 10,228 | 2,932 | 22,019 | 7,880 |
Research and development | 26,117 | 26,217 | 71,674 | 75,737 |
Selling, general and administrative | 36,528 | 22,513 | 99,696 | 62,598 |
Total operating expenses | 72,873 | 51,662 | 193,389 | 146,215 |
Loss from operations | (25,148) | (32,882) | (76,569) | (97,059) |
Other income (expense): | ||||
Interest income | 4,061 | 1,352 | 9,960 | 1,714 |
Interest expense | (3,574) | (3,971) | (11,542) | (11,620) |
Change in fair value of derivative liability | 0 | 0 | 0 | 232 |
Loss from termination of revenue interest purchase agreement | 0 | 0 | (49,076) | 0 |
Other income (expense), net | 1,322 | (192) | 237 | 953 |
Net loss before provision for (benefit from) income taxes | (23,339) | (35,693) | (126,990) | (105,780) |
Provision for (benefit from) income taxes | 249 | 13 | 766 | (6,546) |
Net loss | $ (23,588) | $ (35,706) | $ (127,756) | $ (99,234) |
Net loss per share, basic | $ (0.57) | $ (1.02) | $ (3.28) | $ (3.03) |
Net loss per share, diluted | $ (0.57) | $ (1.02) | $ (3.28) | $ (3.03) |
Weighted-average shares of common stock outstanding, basic | 41,098,920 | 34,927,790 | 38,973,060 | 32,809,365 |
Weighted-average shares of common stock outstanding, diluted | 41,098,920 | 34,927,790 | 38,973,060 | 32,825,314 |
Product [Member] | ||||
Revenues [Abstract] | ||||
Total revenue | $ 47,725 | $ 18,780 | $ 109,320 | $ 47,156 |
License [Member] | ||||
Revenues [Abstract] | ||||
Total revenue | $ 0 | $ 0 | $ 7,500 | $ 2,000 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Statement of Comprehensive Income [Abstract] | ||||
Net loss | $ (23,588) | $ (35,706) | $ (127,756) | $ (99,234) |
Other comprehensive income (loss): | ||||
Unrealized gain (loss) on available-for-sale investments | 11 | (191) | 230 | (305) |
Cumulative translation adjustments | (1,545) | (20) | (1,471) | (73) |
Comprehensive loss | $ (25,122) | $ (35,917) | $ (128,997) | $ (99,612) |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Stockholders' Equity (Unaudited) - USD ($) $ in Thousands | Total | Equity Award Plan | At The Market Offering | Employee Stok Purchase Plan (ESPP) | Private Placement [Member] | Common Stock [Member] | Common Stock [Member] Equity Award Plan | Common Stock [Member] At The Market Offering | Common Stock [Member] Employee Stok Purchase Plan (ESPP) | Common Stock [Member] Private Placement [Member] | Additional Paid-In Capital | Additional Paid-In Capital Equity Award Plan | Additional Paid-In Capital At The Market Offering | Additional Paid-In Capital Employee Stok Purchase Plan (ESPP) | Additional Paid-In Capital Private Placement [Member] | Accumulated Deficit | Accumulated Other Comprehensive Income (Loss) |
Balance at Dec. 31, 2021 | $ 120,212 | $ 3 | $ 377,403 | $ (257,159) | $ (35) | ||||||||||||
Balance, Shares at Dec. 31, 2021 | 30,582,596 | ||||||||||||||||
Issuance of common stock in connection with equity award plans | $ 1,477 | $ 1,477 | |||||||||||||||
Issuance of common stock in connection with equity award plans, Shares | 100,951 | ||||||||||||||||
Issuance of common stock | $ 17,384 | $ 17,384 | |||||||||||||||
Issuance of common stock, shares | 995,897 | ||||||||||||||||
Restricted common stock vested in the period, Shares | 33,398 | ||||||||||||||||
Stock-based compensation | 6,561 | 6,561 | |||||||||||||||
Net loss | (36,606) | (36,606) | |||||||||||||||
Other comprehensive income (Loss) | (96) | (96) | |||||||||||||||
Balance at Mar. 31, 2022 | 108,932 | $ 3 | 402,825 | (293,765) | (131) | ||||||||||||
Balance, Shares at Mar. 31, 2022 | 31,712,842 | ||||||||||||||||
Balance at Dec. 31, 2021 | 120,212 | $ 3 | 377,403 | (257,159) | (35) | ||||||||||||
Balance, Shares at Dec. 31, 2021 | 30,582,596 | ||||||||||||||||
Net loss | (99,234) | ||||||||||||||||
Balance at Sep. 30, 2022 | 169,297 | $ 4 | 526,099 | (356,393) | (413) | ||||||||||||
Balance, Shares at Sep. 30, 2022 | 36,813,754 | ||||||||||||||||
Balance at Mar. 31, 2022 | 108,932 | $ 3 | 402,825 | (293,765) | (131) | ||||||||||||
Balance, Shares at Mar. 31, 2022 | 31,712,842 | ||||||||||||||||
Issuance of common stock in connection with equity award plans | 1,408 | $ 1,032 | 1,408 | $ 1,032 | |||||||||||||
Issuance of common stock in connection with equity award plans, Shares | 92,593 | 76,099 | |||||||||||||||
Issuance of common stock in connection with asset acquisition | 15,585 | 15,585 | |||||||||||||||
Issuance of common stock in connection with asset acquisition, Share | 609,305 | ||||||||||||||||
Issuance of common stock | 3,905 | 3,905 | |||||||||||||||
Issuance of common stock, shares | 165,018 | ||||||||||||||||
Restricted common stock vested in the period, Shares | 33,398 | ||||||||||||||||
Stock-based compensation | 6,818 | 6,818 | |||||||||||||||
Net loss | (26,922) | (26,922) | |||||||||||||||
Other comprehensive income (Loss) | (71) | (71) | |||||||||||||||
Balance at Jun. 30, 2022 | 110,687 | $ 3 | 431,573 | (320,687) | (202) | ||||||||||||
Balance, Shares at Jun. 30, 2022 | 32,689,255 | ||||||||||||||||
Issuance of common stock in connection with equity award plans | 1,378 | 1,378 | |||||||||||||||
Issuance of common stock in connection with equity award plans, Shares | 91,101 | ||||||||||||||||
Issuance of common stock | 86,078 | $ 1 | 86,077 | ||||||||||||||
Issuance of common stock, shares | 4,000,000 | ||||||||||||||||
Restricted common stock vested in the period, Shares | 33,398 | ||||||||||||||||
Stock-based compensation | 7,071 | 7,071 | |||||||||||||||
Net loss | (35,706) | (35,706) | |||||||||||||||
Other comprehensive income (Loss) | (211) | (211) | |||||||||||||||
Balance at Sep. 30, 2022 | 169,297 | $ 4 | 526,099 | (356,393) | (413) | ||||||||||||
Balance, Shares at Sep. 30, 2022 | 36,813,754 | ||||||||||||||||
Balance at Dec. 31, 2022 | 142,037 | $ 4 | 535,074 | (392,824) | (217) | ||||||||||||
Balance, Shares at Dec. 31, 2022 | 36,956,345 | ||||||||||||||||
Issuance of common stock in connection with equity award plans | 1,390 | 1,390 | |||||||||||||||
Issuance of common stock in connection with equity award plans, Shares | 197,703 | ||||||||||||||||
Issuance of common stock | $ 14,480 | $ 14,480 | |||||||||||||||
Issuance of common stock, shares | 658,206 | ||||||||||||||||
Issuance of common stock in connection with achievement of Contingent Milestone, Value | 4,292 | 4,292 | |||||||||||||||
Issuance of common stock in connection with achievement of Contingent Milestone, Shares | 199,993 | ||||||||||||||||
Stock-based compensation | 8,728 | 8,728 | |||||||||||||||
Net loss | (30,130) | (30,130) | |||||||||||||||
Other comprehensive income (Loss) | 353 | 353 | |||||||||||||||
Balance at Mar. 31, 2023 | 141,150 | $ 4 | 563,964 | (422,954) | 136 | ||||||||||||
Balance, Shares at Mar. 31, 2023 | 38,012,247 | ||||||||||||||||
Balance at Dec. 31, 2022 | $ 142,037 | $ 4 | 535,074 | (392,824) | (217) | ||||||||||||
Balance, Shares at Dec. 31, 2022 | 36,956,345 | ||||||||||||||||
Issuance of common stock in connection with settlement of Indemnification Holdback liability, Shares | 31,631 | ||||||||||||||||
Balance at Jun. 30, 2023 | $ 78,610 | $ 4 | 575,522 | (496,992) | 76 | ||||||||||||
Balance, Shares at Jun. 30, 2023 | 38,222,058 | ||||||||||||||||
Balance at Dec. 31, 2022 | 142,037 | $ 4 | 535,074 | (392,824) | (217) | ||||||||||||
Balance, Shares at Dec. 31, 2022 | 36,956,345 | ||||||||||||||||
Net loss | (127,756) | ||||||||||||||||
Balance at Sep. 30, 2023 | 269,081 | $ 5 | 791,114 | (520,580) | 1,458 | ||||||||||||
Balance, Shares at Sep. 30, 2023 | 46,595,745 | ||||||||||||||||
Balance at Mar. 31, 2023 | 141,150 | $ 4 | 563,964 | (422,954) | 136 | ||||||||||||
Balance, Shares at Mar. 31, 2023 | 38,012,247 | ||||||||||||||||
Issuance of common stock in connection with equity award plans | 803 | $ 1,294 | 803 | $ 1,294 | |||||||||||||
Issuance of common stock in connection with equity award plans, Shares | 101,699 | 76,481 | |||||||||||||||
Issuance of common stock in connection with settlement of Indemnification Holdback liability, Value | 896 | 896 | |||||||||||||||
Issuance of common stock in connection with settlement of Indemnification Holdback liability, Shares | 31,631 | ||||||||||||||||
Stock-based compensation | 8,565 | 8,565 | |||||||||||||||
Net loss | (74,038) | (74,038) | |||||||||||||||
Other comprehensive income (Loss) | (60) | (60) | |||||||||||||||
Balance at Jun. 30, 2023 | 78,610 | $ 4 | 575,522 | (496,992) | 76 | ||||||||||||
Balance, Shares at Jun. 30, 2023 | 38,222,058 | ||||||||||||||||
Issuance of common stock in connection with equity award plans | $ 4,766 | $ 4,766 | |||||||||||||||
Issuance of common stock in connection with equity award plans, Shares | 373,687 | ||||||||||||||||
Issuance of common stock | $ 202,239 | $ 1 | $ 202,238 | ||||||||||||||
Issuance of common stock, shares | 8,000,000 | ||||||||||||||||
Stock-based compensation | 8,588 | 8,588 | |||||||||||||||
Net loss | (23,588) | (23,588) | |||||||||||||||
Other comprehensive income (Loss) | (1,534) | (1,534) | |||||||||||||||
Balance at Sep. 30, 2023 | $ 269,081 | $ 5 | $ 791,114 | $ (520,580) | $ 1,458 | ||||||||||||
Balance, Shares at Sep. 30, 2023 | 46,595,745 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Stockholders' Equity (Unaudited) (Parenthetical) - Common Stock [Member] - USD ($) $ in Thousands | 3 Months Ended | ||||
Sep. 30, 2023 | Mar. 31, 2023 | Sep. 30, 2022 | Jun. 30, 2022 | Mar. 31, 2022 | |
At The Market Offering | |||||
Issuance Costs | $ 518 | $ 5,922 | $ 184 | $ 601 | |
Private Placement [Member] | |||||
Issuance Costs | $ 7,761 |
Condensed Consolidated Statem_5
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Operating activities | ||
Net loss | $ (127,756) | $ (99,234) |
Reconciliation of net loss to net cash used in operating activities: | ||
Stock-based compensation | 25,303 | 20,111 |
Depreciation and amortization | 5,335 | 2,037 |
Non-cash lease expense | 476 | 315 |
Net accretion of discounts on investments | (1,696) | (686) |
Non-cash interest expense related to the revenue interest liability | 5,060 | 11,620 |
Amortization of debt discount and offering costs | 718 | 0 |
Change in fair value of derivative liability | 0 | (232) |
Change in fair value of contingent liabilities associated with an acquisition | 671 | (1,125) |
Inventory reserves | 1,491 | 0 |
Loss from termination of revenue interest purchase agreement | 49,076 | 0 |
Unrealized foreign exchange gain | (1,230) | 0 |
Deferred income taxes associated with an acquisition | 0 | (6,580) |
Change in operating assets and liabilities: | ||
Accounts receivable | (23,840) | (13,070) |
Prepaid expenses and other current assets | (3,447) | (1,658) |
Inventory | (2,206) | (4,046) |
Other assets | 180 | (434) |
Accounts payable, accrued expenses and other liabilities | 18,250 | 7,644 |
Operating lease liabilities | (711) | (528) |
Net cash used in operating activities | (54,326) | (85,866) |
Investing activities | ||
Purchase of investments | (27,329) | (132,322) |
Proceeds from maturities of investments | 150,000 | 113,300 |
Purchase of property and equipment | (109) | (36) |
Cash paid for acquisition | (212,762) | 0 |
Payments made for additions to intangible assets | (15,000) | 0 |
Net cash used in investing activities | (105,200) | (19,058) |
Financing activities | ||
Proceeds from issuance of common stock in private placement, net of issuance costs | 202,363 | 0 |
Proceeds from issuance of common stock in at-the-market offerings, net of issuance costs | 14,480 | 21,289 |
Proceeds from issuance of common stock in public offerings, net of issuance costs | 0 | 86,358 |
Proceeds from the issuance of convertible notes, net | 305,304 | 0 |
Proceeds from issuance of common stock pursuant to equity plans | 8,253 | 5,295 |
Payments on revenue interest liability | (195,577) | (3,526) |
Net cash provided by financing activities | 334,823 | 109,416 |
Effect of exchange rate on cash, cash equivalents and restricted cash equivalents | (241) | (73) |
Net increase in cash, cash equivalents and restricted cash equivalents | 175,056 | 4,419 |
Cash, cash equivalents and restricted cash equivalents at beginning of period | 128,003 | 131,340 |
Cash, cash equivalents and restricted cash equivalents at end of period | 303,059 | 135,759 |
Supplemental disclosure of cash flow information: | ||
Operating cash flows paid for operating lease | 824 | 661 |
Non-cash operating, investing and financing activities: | ||
Commercial milestone included in accrued liabilities | 5,000 | 0 |
Private placement issuance costs included in accounts payable and accrued liabilities | 124 | 0 |
Inventory purchases included in accrued liabilities | 2,492 | 1,045 |
Right-of-use assets obtained in exchange for lease liability | 473 | 0 |
Stock-based compensation capitalized to inventory | 578 | 339 |
Reclassification of Contingent Milestone liability to equity in connection with issuance of common stock upon achievement of Contingent Milestone | 4,292 | 0 |
Issuance of common stock in connection with settlement of Indemnification Holdback liability | 896 | 0 |
Transaction costs from public offering included in accrued liabilities | 0 | 281 |
Issuance of common stock in exchange for acquired intangible assets | 0 | 15,585 |
Indemnification Holdback liability in exchange for acquired intangible assets | 0 | 831 |
Contingent milestone liability for common stock issuance tly issuable common stock for acquired intangible assets | 0 | 4,600 |
Deferred tax liability incurred from acquired intangible | $ 0 | $ 6,580 |
Organization and Description of
Organization and Description of Business | 9 Months Ended |
Sep. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization and Description of Business | 1. Organization and Description of Business Mirum Pharmaceuticals, Inc. (the “Company”) was incorporated in the State of Delaware on May 2, 2018 , and is headquartered in Foster City, California. The Company is a biopharmaceutical company focused on the identification, acquisition, development and commercialization of novel therapies for debilitating rare and orphan diseases. The Company has three approved medicines: LIVMARLI® (maralixibat) oral solution (“Livmarli”), Cholbam® (cholic acid) capsules, and Chenodal® (chenodiol) tablets. Livmarli is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (“ALGS”) three months of age and older in the United States and for the treatment of cholestatic pruritus in patients with ALGS two months of age and older in Europe. On July 16, 2023, the Company entered into an Asset Purchase Agreement (the “Purchase Agreement”) with Travere Therapeutics, Inc., a Delaware corporation (“Travere”), pursuant to which, upon the closing in August 2023, the Company acquired Travere’s bile acid product portfolio, including Chenodal and Cholbam (also known as Kolbam, and together with Chenodal, the “Bile Acid Medicines”), two therapies addressing rare diseases in high-need settings (such acquisition, the “Bile Acid Portfolio Acquisition”) (Note 7). Cholbam is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. Chenodal is approved for the treatment of radiolucent stones in the gallbladder and has received medical necessity recognition by the FDA for the treatment of cerebrotendinous xanthomatosis (“CTX”). The Company’s development pipeline consists of the clinical-stage product candidate volixibat and life-cycle extension opportunities for Livmarli. The Company commenced significant operations in November 2018. The Company views its operations and manages its business as one operating segment. The Company determined its operating segment on the same basis that it uses to evaluate its performance internally. Liquidity The Company has a limited operating history, has incurred significant operating losses since its inception, and the revenue and income potential of the Company’s business and market are unproven. As of September 30, 2023, the Company had an accumulated deficit of $ 520.6 million and cash, cash equivalents and investments of $ 306.0 million. The Company believes that its cash, cash equivalents and investments of $ 306.0 million as of September 30, 2023, provide sufficient capital resources to continue its operations for at least twelve months from the issuance date of the accompanying unaudited condensed consolidated financial statements. In April 2023, the Company completed a convertible notes offering, as further described in Note 10, with net proceeds of $ 305.3 million, after deducting the initial purchasers’ discounts and commissions and offering expenses. From the net proceeds, $ 192.7 million was used to repurchase all future revenue interests in connection with the Company’s Revenue Interest Purchase Agreement (“RIPA”) (Note 6). Upon repurchase and the termination of the RIPA, in accordance with its terms, the previously restricted cash equivalents of $ 100.0 million were no longer restricted from use. In August 2023, the Company completed the Bile Acid Portfolio Acquisition contemplated by the Purchase Agreement and acquired substantially all of the assets of Travere that are primarily related to the development, manufacture and commercialization of the Bile Acid Medicines. Under the terms of the Purchase Agreement, the Company paid an upfront purchase price of $ 210.4 million in cash to Travere and may pay additional amounts of up to $ 235.0 million upon the achievement of certain milestones based on specified amounts of net annual sales of the Bile Acid Medicines. In connection with and immediately prior to the closing of the Bile Acid Portfolio Acquisition, the Company completed the private placement of 8,000,000 shares of the Company's common stock at a price per share of $ 26.25 , resulting in net proceeds of approximately $ 202.2 million. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Basis of Presentation The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information and pursuant to Form 10-Q and Article 10 of Regulation S-X of the Securities and Exchange Commission (“SEC”). Accordingly, the accompanying unaudited condensed consolidated financial statements do not include all of the information and notes required by GAAP for complete financial statements. The unaudited interim financial statements reflect all adjustments which, in the opinion of management, are necessary for a fair statement of the results for the periods presented. All such adjustments are of a normal and recurring nature. The unaudited condensed consolidated balance sheet as of December 31, 2022 has been derived from the audited consolidated financial statements at that date but does not include all information and footnotes required by GAAP for complete financial statements. The operating results presented in these unaudited condensed consolidated financial statements are not necessarily indicative of the results that may be expected for any future periods. The accompanying unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All significant intercompany balances and transactions have been eliminated in consolidation. These unaudited condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and the notes thereto in the Company’s Annual Report on Form 10-K (“Annual Report”) for the fiscal year ended December 31, 2022, as filed with the SEC on March 8, 2023. Use of Estimates The preparation of consolidated financial statements in accordance with GAAP requires management to make estimates and assumptions that impact the reported amounts of assets, liabilities and expenses and the disclosure of contingent assets and liabilities in the financial statements and accompanying notes. These estimates and assumptions are based upon historical experience, knowledge of current events and various other factors believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities and the recording of expenses that are not readily apparent from other sources. Actual results could differ materially from those estimates. The Company’s unaudited condensed consolidated financial statements as of and for the three and nine months ended September 30, 2023 reflect the Company’s estimates of the impact of the geopolitical and macroeconomic environment, including the impact of inflation, bank failures, higher interest rates and foreign exchange rate fluctuations. The duration and the scope of these conditions cannot be predicted; therefore, the extent to which these conditions will directly or indirectly impact the Company’s business, results of operations and financial condition, is uncertain. The Company is not aware of any specific event or circumstance that would require an update to its estimates, judgments and assumptions or a revision of the carrying value of the Company’s assets or liabilities as of the date of this filing. Significant Accounting Policies There have been no significant changes to the accounting policies during the nine months ended September 30, 2023, as compared to the significant accounting policies described in Note 2 of the “Notes to Consolidated Financial Statements” in the Company’s audited consolidated financial statements included in the Annual Report, unless indicated below. Cash, Cash Equivalents and Restricted Cash Equivalents The Company considers all highly liquid investments that are readily convertible into cash without penalty and with original maturities of three months or less at the date of purchase to be cash equivalents. The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents are valued at cost, which approximate their fair value. Restricted cash equivalents as of December 31, 2022 consisted of deposits placed in a segregated bank account as required under the terms of the Company’s RIPA, as amended September 2021, with Mulholland SA LLC, an affiliate of Oberland Capital LLC, as agent for the purchasers party thereto (the “Purchasers”), and the Purchasers in connection with the sale of the Priority Review Voucher in December 2021. Upon repurchase and the termination of the RIPA in April 2023, in accordance with its terms, the previously restricted cash equivalents of $ 100.0 million were no longer restricted from use. The following table provides a reconciliation of cash, cash equivalents and restricted cash equivalents reported within the unaudited condensed consolidated balance sheets that together reflect the same amounts shown in the unaudited condensed consolidated statements of cash flows (in thousands): As of September 30, As of December 31, 2023 2022 Cash and cash equivalents $ 303,059 $ 28,003 Restricted cash equivalents — 100,000 Total cash, cash equivalents, and restricted cash equivalents $ 303,059 $ 128,003 Investments The Company classifies all investments in securities as available-for-sale. Management determines the appropriate classification of its investments in securities at the time of purchase. Investments with original maturities beyond three months at the date of purchase and which mature at, or less than twelve months from the balance sheet date, are classified as a current asset. Investments are recorded at fair value, with unrealized gains and losses reported as accumulated other comprehensive income (loss) until realized, with the exception of any declines in fair value below the cost basis that are a result of a credit loss, which, if any, are reported in other income (expense), net in the current period through an allowance for credit losses. Each reporting period, the Company evaluates whether declines in fair values of its available-for-sale securities below their cost basis are a result of credit loss or other factors and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. This evaluation consists of several qualitative and quantitative factors regarding the severity and duration of the unrealized loss, the creditworthiness of the security issuers, as well as the Company’s ability and intent to hold the available-for-sale security until a forecasted recovery occurs. Additionally, the Company assesses whether it has plans to sell the security or it is more likely than not it will be required to sell any available-for-sale securities before recovery of its amortized cost basis. The cost of debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, as well as interest and dividends, are included in interest income. Realized gains and losses from the sale of available-for-sale securities, if any, are determined on a specific identification basis and are also included in Other income (expense). To date, the Company has no t identified any declines in fair value of its investments related to credit loss. Concentrations of Credit Risk and Off-Balance Sheet Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, accounts receivable and investments. The Company limits the amount of credit exposure by investing cash that is not required for immediate operating needs in money market funds, government obligations and/or commercial paper with short maturities. Additionally, the Company has established guidelines regarding diversification of its investments and their maturities, which are designed to maintain principal and maximize liquidity. To date, the Company has not experienced any losses associated with this credit risk and continues to believe that this exposure is not significant. The Company relies on a single distributor and a specialty pharmacy for all of the Company’s sales of its approved medicines in the United States as well as a single distributor for sales of Livmarli outside the United States. The Company sources materials and services through several vendors. Certain materials are sourced from a single vendor. The loss of certain vendors could result in a temporary disruption of the Company’s commercialization efforts. As of September 30, 2023, the Company did no t have any customers that individually accounted for more than 10 % of accounts receivable. As of December 31, 2022, the Company had one customer that accounted for approximately 23 % of accounts receivable. For the three and nine months ended September 30, 2023 and 2022, the Company did not have revenue attributable to any one customer in excess of 10 % of sales. Accounts Receivable The Company has accounts receivable amounts due from product sales. The Company also has accounts receivable amounts due from license agreements for milestones achieved, but not yet paid. Amounts payable to the Company are recorded as accounts receivable when the Company’s right to consideration is unconditional. The Company estimates the allowance for credit losses using the current expected model. Under this model, the allowance for credit losses reflects the Company’s estimate of lifetime expected credit losses. The Company evaluates the collectability of the cash flows based on the risk of loss over the contractual life, even when that risk is remote, based on judgments about the creditworthiness of its customers, historical experience and other relevant information that is available to the Company. There was no allowance for credit losses as of September 30, 2023. There was no bad debt expense for the three and nine months ended September 30, 2023 and 2022. Intangible Assets, Net The Company accounts for asset acquisitions that do not meet the definition of a business using the cost accumulation method, whereby the cost of the acquisition, including certain transaction costs, is allocated to the asset (or assets) acquired on the basis of its (or their) relative fair value(s) on the measurement date. No goodwill is recognized in an asset acquisition. Intangible assets are measured at their fair values as of the acquisition date or, in the case of commercial milestone payments, the date they become due. The evaluation of intangible assets includes assessing the amortization period for which the asset is expected to contribute to the future cash flows of the Company. Intangible assets with finite useful lives are amortized over their estimated useful lives, primarily on a straight-line basis when the Company is unable to reliably estimate the pattern of cash flow. The Company tests its finite lived intangible assets for impairment annually or if events or changes in circumstances indicate that it is more likely than not that the asset is impaired. If it is determined that the asset is impaired, the carrying value is written down to its estimated fair value, with the related impairment charge recognized in the unaudited condensed consolidated statements of operations in the period in which the impairment occurs. The Company has not recorded any impairments to its intangible assets for any of the periods presented. The components of the Company’s intangible assets were as follows (in thousands, except for weighted-average remaining amortization period): September 30, 2023 Gross Carrying Value Accumulated Amortization Net Carrying Amount Weighted-Average Remaining Amortization Period Developed technology $ 226,620 $ ( 5,487 ) $ 221,133 11.9 Commercial milestones 39,000 ( 2,765 ) 36,235 16.4 Assembled workforce 970 — 970 2.9 Total intangible assets $ 266,590 $ ( 8,252 ) $ 258,338 11.7 December 31, 2022 Gross Carrying Value Accumulated Amortization Net Carrying Amount Weighted-Average Remaining Amortization Period Commercial milestones $ 34,000 $ ( 1,333 ) $ 32,667 17.1 Developed technology 28,107 ( 1,820 ) 26,287 8.4 Total intangible assets $ 62,107 $ ( 3,153 ) $ 58,954 11.9 Amortization expense was $ 2.6 million and $ 5.1 million for the three and nine months ended September 30, 2023, respectively, and $ 1.0 million and $ 1.8 million for the three and nine months ended September 30, 2022, respectively. Amortization expense was included in cost of sales in the accompanying unaudited condensed consolidated statements of operations. The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of September 30, 2023 (in thousands): Amount 2023 (remaining three months) 5,413 2024 21,545 2025 21,545 2026 21,436 2027 21,221 Thereafter 167,178 $ 258,338 Convertible Notes The Company evaluates all conversion, repurchase and redemption features contained in a debt instrument to determine if there are any embedded features that require bifurcation as a derivative. The Company accounts for its convertible notes (see Note 10) as a long-term liability equal to the proceeds received from issuance, including any embedded conversion features, net of the unamortized debt discount and offering costs in the accompanying unaudited consolidated balance sheets. The debt issuance and offering costs are amortized over the contractual term of the convertible notes, using the effective interest method, as interest expense in the accompanying unaudited consolidated statements of operations. Product Sales, Net The Company recognizes product sales, net when the customer obtains control of our product, which occurs at a point in time, typically upon delivery of the Company’s product to the customer. Revenues from product sales are recorded at the net sales price, or the transaction price, which may include fixed or variable consideration for discounts, government rebates, co-pay assistance, returns and other allowances that are offered within contracts with a customer relating to the sale of the Company's approved medicines. Estimates of variable consideration are calculated based on the actual product sales each reporting period and the nature of the variable consideration related to those sales. Overall, these estimates reflect the Company’s best estimate of the amount of consideration to which the Company expects to be entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in product sales, net only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. Estimates are reviewed and updated quarterly as additional information becomes known. Actual amounts of consideration ultimately received may differ materially from estimates. If actual results in the future vary from estimates, the Company will adjust these estimates, which would affect product sales, net and earnings in the period such variances are adjusted. Significant categories of sales discounts and allowances are as follows: Government Rebates : The Company records rebates payable under Medicaid and other government programs as a reduction of revenue at the time product revenues are generated. The Company’s rebate calculations may require estimates, including estimates of customer mix, to determine which sales will be subject to rebates and the amount of such rebates. The Company updates its estimates and assumptions on a quarterly basis and records any necessary adjustments to revenue in the period identified. The liability for unpaid rebates is included in accrued expenses on the accompanying unaudited condensed consolidated balance sheets. To date, actual government rebates have not differed materially from the Company’s estimates. Other Incentives : Other incentives include a branded co-pay assistance program for eligible patients with commercial insurance in the United States. The branded co-pay assistance program assists commercially insured patients who have coverage for the Company's approved medicines and is intended to reduce each participating patient’s portion of the financial responsibility of the purchase price up to a specified dollar amount of assistance. The calculation of the accrual for co-pay assistance is based upon an identification of claims and the cost per claims associated with product that has been recognized as revenue. The Company records amounts paid under the brand specific co-pay assistance program for each patient as a reduction of revenue from product sales. To date, actual other incentives have not differed materially from the Company’s estimates. Product Returns : The Company records revenue for product sales, net of estimated product returns. Customers have limited return rights related only to the product’s damage or defect identified upon delivery of the product. The Company estimates the amount of product sales that may be returned and records the estimate as a reduction of revenue and a refund liability in the period the related product revenue is recognized. To date, actual returns have not differed materially from the Company’s estimates. The following table disaggregates total Product sales, net: Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Product sales, net: Livmarli $ 38,708 $ 18,780 $ 100,303 $ 47,156 Bile Acid Medicines 9,017 — 9,017 — Total product sales, net 47,725 18,780 109,320 47,156 License revenue — — 7,500 2,000 Total revenues $ 47,725 $ 18,780 $ 116,820 $ 49,156 Net Loss Per Share Basic net loss per share is computed by dividing net loss by the weighted-average shares of common stock outstanding for the period, without consideration for potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss by the weighted-average shares of common stock and potentially dilutive securities outstanding for the period determined using the treasury-stock and if-converted methods. Diluted net loss per share excludes the potential impact of the Company’s common stock subject to repurchase, common stock options, restricted stock units, contingently issuable employee stock purchase plan shares and common stock issuable upon conversion of convertible notes because their effect would be anti-dilutive due to the Company’s net loss. Basic and diluted net loss per share were the same for the three and nine months ended September 30, 2023. The following table sets for the computation of basic and diluted earnings per share for the three and nine months ended September 30, 2022 (in thousands, except share and per share data): Three Months Ended Nine Months Ended 2022 2022 Numerator: Net loss, basic $ ( 35,706 ) $ ( 99,234 ) Less: Change in fair value of Holdback Indemnification liability — 149 Net loss, diluted $ ( 35,706 ) $ ( 99,383 ) Denominator: Weighted-average shares of common stock outstanding, basic 34,927,790 32,809,365 Effect of dilutive securities: Weighted-average Holdback Indemnification shares issuable — 15,949 Weighted-average shares of common stock outstanding, diluted 34,927,790 32,825,314 Net loss per share, basic $ ( 1.02 ) $ ( 3.03 ) Net loss per share, diluted $ ( 1.02 ) $ ( 3.03 ) The following outstanding potential dilutive shares have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: As of September 30, As of September 30, 2023 2022 Options to purchase common stock and restricted stock units 10,814,495 8,910,376 Common stock issuable upon conversion of convertible notes 9,964,247 — Employee stock purchase plan contingently issuable 49,868 46,760 Common stock subject to repurchase — 22,270 Shares issuable as contingent consideration in connection with asset acquisition — 199,993 Total 20,828,610 9,179,399 Recently Adopted Accounting Pronouncements On January 1, 2023, the Company adopted Accounting Standards Update (“ASU”) No. 2016-13, Financial Instruments-Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016-13”). ASU 2016-13 requires an entity to utilize a new impairment model that requires measurement and recognition of expected credit losses for most financial assets and certain other instruments, including but not limited to available-for-sale debt securities. Credit losses relating to available-for-sale debt securities will be recorded through an allowance for credit losses rather than as a direct write-down to the security. The new guidance also modifies the impairment models for available-for-sale debt securities and for purchased financial assets with credit deterioration since their origination. In March 2020, the Financial Accounting Standards Board (“FASB”) issued ASU No. 2020-3, Codification Improvements to Financial Instruments which makes narrow-scope improvements to various financial instruments topics, including the new credit losses standard and clarifies the following areas (i) the contractual term of a net investment in a lease should be the contractual term used to measure expected credit losses; (ii) when an entity regains control of financial assets sold, an allowance for credit losses should be recorded. There was no impact on the accompanying unaudited condensed consolidated financial statements as of the adoption date, January 1, 2023 . Recent Accounting Pronouncements Not Yet Adopted From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on the accompanying condensed consolidated financial statements and disclosures. |
Fair Value Measurements
Fair Value Measurements | 9 Months Ended |
Sep. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Fair Value Measurements | 3. Fair Value Measurements Financial assets and liabilities subject to fair value measurements on a recurring basis and the level of inputs used in such measurements by major security type are presented in the following table (in thousands): September 30, 2023 Level 1 Level 2 Level 3 Total Financial assets: Money market funds $ 297,316 $ — $ — 297,316 Commercial paper — 2,971 — 2,971 Total financial assets $ 297,316 $ 2,971 $ — $ 300,287 December 31, 2022 Level 1 Level 2 Level 3 Total Financial assets: Money market funds $ 124,227 $ — $ — $ 124,227 U.S. treasury bills 4,975 — — 4,975 Commercial paper — 74,386 — 74,386 U.S. government bonds — 44,354 — 44,354 Total financial assets $ 129,202 $ 118,740 $ — $ 247,942 Financial liabilities: Contingent milestone liability $ 3,900 $ — $ — $ 3,900 Derivative liability — — 1,090 1,090 Indemnification holdback — — 617 617 Total financial liabilities $ 3,900 $ — $ 1,707 $ 5,607 The carrying amounts of certain financial instruments such as cash and cash equivalents, restricted cash equivalents, accounts receivable, prepaid expenses, other current assets, accounts payable and accrued expenses as of September 30, 2023 and December 31, 2022 approximate their related fair values due to the short-term maturities of these instruments. Money market funds and U.S. treasury bills are highly liquid investments and are actively traded. The pricing information on these investment instruments is readily available and can be independently validated as of the measurement date. This approach results in the classification of these securities as Level 1 of the fair value hierarchy. U.S. government bonds and commercial paper are measured at fair value using Level 2 inputs. The Company reviews trading activity and pricing for these investments as of each measurement date. The carrying amount of the revenue interest liability as of December 31, 2022 approximates its fair value and is based on the Company’s contractual repayment obligation to the Purchasers, based on the current estimates of future revenues, over the life of the RIPA. The Contingent Milestone liability as of December 31, 2022 is considered a Level 1 input as the significant inputs were known and observable. The derivative liability and Indemnification Holdback liability (each as defined below) as of December 31, 2022 are each considered a Level 3 input based on the three-level hierarchy. Derivative Liability The debt pursuant to the RIPA (refer to Note 6 “Revenue Interest Purchase Agreement” for further information) contained an embedded derivative requiring bifurcation as a single compound derivative instrument. The Company estimated the fair value of the derivative liability using a “with-and-without” method. The “with-and-without” methodology involves valuing the whole instrument on an as-is basis and then valuing the instrument without the individual embedded derivative. The difference between the entire instrument with the embedded derivative compared to the instrument without the embedded derivative was the fair value of the derivative liability at December 31, 2022. The estimated probability and timing of underlying events triggering the exercisability of the put option contained within the RIPA, forecasted cash flows and the discount rate were significant unobservable inputs used to determine the estimated fair value of the entire instrument with the embedded derivative. As of December 31, 2022, the discount rate used for valuation of the derivative liability was 15.7 %. In 2023, there was no change in the fair value of the derivative liability prior to its extinguishment in connection with the termination of the RIPA in April 2023. Indemnification Holdback In May 2022, in connection with the acquisition of Satiogen (refer to Note 7 “Asset Acquisitions” for further information), the Company recorded at fair value liabilities related to the Company’s common stock issuable upon satisfaction of certain purchase price adjustments and indemnification obligations that may arise during the 12-month period following the asset acquisition date (“Indemnification Holdback”). The fair value of the Indemnification Holdback was classified within Level 3 of the fair value hierarchy and was estimated based upon the value of the Company’s common stock price. The fair value of the Indemnification Holdback was additionally determined based on management’s estimate of the probability of indemnification obligations being incurred during the one year following the acquisition date. The fair value of the Indemnification Holdback was initially measured on May 20, 2022, the date on which the Company completed the acquisition of Satiogen. The Company assessed the fair value of the Indemnification Holdback each reporting period until resolution of the related contingency and changes in fair value were recorded in other income (expense), net in the accompanying unaudited condensed consolidated statements of operations. In June 2023, the contingencies related to the Indemnification Holdback were resolved and the Company issued 31,631 shares of common stock. The following table provides a summary of the changes in the estimated fair value of the Indemnification Holdback liability (in thousands): Indemnification Holdback Liability Balance at December 31, 2022 $ 617 Change in fair value 279 Settlement of Indemnification Holdback liability ( 896 ) Balance at September 30, 2023 $ — The Indemnification Holdback is included in other liabilities on the accompanying unaudited condensed consolidated balance sheets as of December 31, 2022. There was no outstanding balance related to the Indemnification Holdback liability as of September 30, 2023. |
Financial Instruments
Financial Instruments | 9 Months Ended |
Sep. 30, 2023 | |
Financial Instruments, Owned, at Fair Value, by Type, Alternative [Abstract] | |
Financial Instruments | 4. Financial Instruments The fair value and amortized cost of cash equivalents and available-for-sale investments by major security type are presented in the following table (in thousands): September 30, 2023 Amortized Unrealized Unrealized Estimated Cash equivalents and investments: Money market funds $ 297,316 $ — $ — $ 297,316 Commercial paper 2,971 — — 2,971 Total cash equivalents and investments $ 300,287 $ — $ — $ 300,287 Classified as: Cash equivalents $ 297,316 Short-term investments 2,971 Total cash equivalents and investments $ 300,287 December 31, 2022 Amortized Unrealized Unrealized Estimated Cash equivalents and investments: Money market funds $ 124,227 $ — $ — $ 124,227 U.S. treasury bills 4,980 — ( 5 ) 4,975 Commercial paper 74,386 — — 74,386 U.S. government bonds 44,579 — ( 225 ) 44,354 Total cash equivalents and investments $ 248,172 $ — $ ( 230 ) $ 247,942 Classified as: Cash equivalents $ 24,226 Cash equivalents - restricted 100,000 Short-term investments 123,716 Total cash equivalents, restricted cash equivalents and investments $ 247,942 As of September 30, 2023, the remaining contractual maturities of available-for-sale debt securities were less than 12 months. During the three and nine months ended September 30, 2023 and 2022 , there have been no significant realized gains or losses on available-for-sale investments, no investments have been in a continuous unrealized loss position for more than 12 months, and the Company did no t recognize any material unrealized gains or losses on these securities. |
Balance Sheet Components
Balance Sheet Components | 9 Months Ended |
Sep. 30, 2023 | |
Balance Sheet Components [Abstract] | |
Balance Sheet Components | 5. Balance Sheet Components Inventory Inventory consists of the following (in thousands): September 30, December 31, 2023 2022 Raw materials $ 444 $ — Work in progress 10,529 5,351 Finished goods 11,277 214 Total inventory $ 22,250 $ 5,565 Accrued Expenses Accrued expenses consist of the following (in thousands): September 30, December 31, 2023 2022 Accrued compensation and related benefits $ 13,610 $ 14,660 Accrued sales deductions 14,557 4,284 Accrued clinical trials 6,351 8,319 Accrued professional service fees 9,502 5,372 Accrued contract manufacturing and non-clinical costs 7,004 3,927 Accrued royalties payable 4,426 2,456 Accrued interest 5,763 — Accrued milestone payments 5,000 15,000 Total accrued expenses $ 66,213 $ 54,018 |
Revenue Interest Purchase Agree
Revenue Interest Purchase Agreement | 9 Months Ended |
Sep. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Revenue Interest Purchase Agreement | 6. Revenue Interest Purchase Agreement In December 2020, the Company entered into the RIPA, as amended in September 2021, with Mulholland SA LLC, an affiliate of Oberland Capital LLC, as agent for the Purchasers, and the Purchasers to obtain financing for the commercialization and further development of Livmarli and other working capital needs. Pursuant to the RIPA, the Company has received $ 115.0 million consisting of an upfront payment of $ 50.0 million in December 2020 and $ 65.0 million in April 2021 associated with the acceptance for filing by the FDA of a New Drug Application for Livmarli for the treatment of cholestatic pruritus in patients with ALGS, less certain transaction expenses. Under the RIPA, the Company was entitled to receive an additional $ 35.0 million upon FDA approval of Livmarli, which it elected to forgo. The Company was also entitled to receive up to approximately $ 50.0 million at the option of the Purchasers to finance in-licenses or other acquisitions on or prior to December 31, 2022, which the Company did not request. As consideration for such payments, the Purchasers had the right to receive certain revenue interests (the “Revenue Interests”) from the Company based on annual product sales, net of Livmarli, in tiered payments (the “Revenue Interest Payments”) based on whether annual product sales, net are (i) less than or equal to $ 350.0 million (“Tier 1”), (ii) exceeding $ 350.0 million and less than or equal to $ 1.1 billion (“Tier 2”), or (iii) exceeding $ 1.1 billion (“Tier 3”). The Revenue Interest Payments were initially 9.75 % (at Tier 1) and 2.0 % (at Tier 2 and Tier 3) of such annual net sales. Under the RIPA, the Company had an option (the “Call Option”) to terminate the RIPA and repurchase future Revenue Interests at any time upon advance written notice. Additionally, the Purchasers had an option (the “Put Option”) to terminate the RIPA and to require the Company to repurchase future Revenue Interests upon enumerated events such as a bankruptcy event, an uncured material breach, a material adverse effect or a change of control, or upon the 12th anniversary of the first payment made by Purchasers. If the Put Option is exercised prior to the first anniversary of the closing date by the Purchasers (except pursuant to a change of control), the required repurchase price is 120.0 % of the Cumulative Purchaser Payments (minus all payments Company has made to the Purchasers in connection with the Revenue Interests). In all other cases, if the Put Option or the Call Option are exercised, the required repurchase price is 175.0 % of the Cumulative Purchaser Payments (minus all payments the Company has made to the Purchasers in connection with the Revenue Interests), if such option is exercised prior to the third anniversary of the closing date, and 195.0 % of the Cumulative Purchaser Payments (minus all payments Company has made to the Purchasers in connection with the Revenue Interests) if such option is exercised thereafter. In addition, the RIPA contained various representations and warranties, information rights, non-financial and financial covenants, indemnification obligations and other provisions that are customary for a transaction of this nature. The Purchasers’ obligations to fund the scheduled installments were subject to certain customary conditions as set forth in the RIPA. Concurrently with the RIPA, the Company entered into a Common Stock Purchase Agreement (“CSPA”) with certain affiliates of Oberland, pursuant to which the Company sold an aggregate of 509,164 shares of its common stock for an aggregate purchase price of $ 10.0 million. The $ 50.0 million upfront payment received pursuant to the RIPA and $ 10.0 million received pursuant to the CSPA was allocated between the resulting financial instruments on a relative fair value basis, with $ 49.2 million allocated to the debt under the RIPA and $ 10.8 million allocated to the common stock issued under the CSPA. The Put Option under the RIPA that was exercisable by Purchasers upon certain contingent events was determined to be an embedded derivative requiring bifurcation and separately accounted for as a single compound derivative instrument. The Company recorded the initial fair value of the derivative liability of $ 1.3 million as a debt discount, which was amortized to interest expense over the expected term of the debt using the effective interest method. The Company imputed interest expense associated with the revenue interest liability using the effective interest rate method. The effective interest rate was calculated based on the rate that would enable the debt to be repaid in full over the anticipated life of the arrangement. The interest rate on this liability was variable during the term of the agreement depending on a number of factors, including the level of forecasted product sales, net. The Company evaluated the interest rate quarterly based on each period's product sales, net forecasts utilizing the prospective method. The Company recorded interest expense related to this arrangement of $ 5.1 million for the nine months ended September 30, 2023, and $ 4.0 million and $ 11.6 million for the three and nine months ended September 30, 2022, respectively. No interest expense related to this arrangement was recorded for the three months ended September 30, 2023. The Company incurred $ 0.9 million of issuance costs in connection with the RIPA, which were amortized to interest expense over the estimated term of the debt. Revenue Interest Payments made as a result of the Company’s product sales, net reduce the revenue interest liability. During the nine months ended September 30, 2023, the Company made payments of $ 2.9 million in connection with the RIPA. In April 2023, the Company exercised the Call Option and repurchased all future Revenue Interests. In connection with such repurchase, the Company made a payment of $ 192.7 million, or 175 % of the Cumulative Purchaser Payments (minus all payments that were made to the Purchasers in connection with the Revenue Interests). As a result, the RIPA terminated in accordance with its terms. The following table summarizes the revenue interest liability activity and the related derivative liability activity prior to settlement (in thousands): Revenue Interest Liability Derivative Liability Balance at December 31, 2022 $ 140,351 $ 1,090 Interest expense recognized 5,060 — Revenue interest payments ( 2,883 ) — Revenue interest liability settlement ( 192,694 ) — Loss (gain) from termination of revenue interest purchase agreement 50,166 ( 1,090 ) Balance at September 30, 2023 $ — $ — The net loss from termination of RIPA of $ 49.1 million, comprised of the $ 50.2 million loss related to the settlement of the revenue interest liability and the $ 1.1 million gain on the derecognition of the related derivative liability, was recorded in the accompanying unaudited condensed consolidated statements of operations. As of September 30, 2023, there was no outstanding balance related to the revenue interest liability and the derivative liability. |
Asset Acquisitions
Asset Acquisitions | 9 Months Ended |
Sep. 30, 2023 | |
Asset Acquisitions [Abstract] | |
Asset Acquisitions | 7. Asset Acquisitions Asset Purchase Agreement with Travere Therapeutics, Inc. On August 31, 2023, the Company completed the Bile Acid Portfolio Acquisition. In accordance with the terms and conditions of the Purchase Agreement, the Company purchased from Travere substantially all of the assets related to its business of development, manufacturing (including synthesis, formulation, finishing or packaging) and commercialization of the Bile Acid Medicines. The Company paid $ 210.4 million upon closing of the transaction, and up to an additional $ 235.0 million is payable upon the achievement of certain milestones based on specified amounts of annual net sales of the Bile Acid Medicines. The Company and Travere concurrently entered into a transitional services agreement pursuant to which Travere is obligated to perform certain services for a period of time, not anticipated to last beyond 12 months post-close, with respect to the Company’s use and operation of the assets purchased. The Company accounted for the transaction as an asset acquisition as substantially all the fair value of the gross assets acquired was concentrated in a group of similar identifiable assets, namely, the developed technology related to the Bile Acid Medicines. The developed technology asset consists of certain processes and at-market contracts related to the manufacture and commercialization of the Bile Acid Medicines, regulatory approvals, and other assets, and are considered a single asset as they are interdependently linked. The Company estimated the fair value of the developed technology asset using the discounted cash flow method. This approach incorporates significant estimates and assumptions related to the forecasted results including, but not limited to, estimates and assumptions regarding revenues, expenses, and discount rates to estimate future cash flows. The Company determined that the developed technology asset acquired was commercially viable and the cost of the acquisition was recorded as an intangible asset in the accompanying unaudited condensed consolidated balance sheet as of September 30, 2023. The acquired assets are amortizable for tax purposes. The Company is obligated to pay tiered royalties, based on licensing agreements acquired with the Bile Acid Medicines, with rates ranging from high single digit to mid-teens based on net sales of the Bile Acid Medicines. The following represents the consideration paid and allocation of the purchase price for the acquisition of the Bile Acid Medicines (in thousands): Cash consideration $ 210,378 Transaction costs 2,384 Total purchase consideration 212,762 Assets acquired: Inventory 12,900 Intangible assets - developed technology 198,513 Intangible assets - assembled workforce 970 Other current and noncurrent assets 379 Total assets acquired $ 212,762 The Company amortizes the acquired intangible assets straight-line over their estimated useful lives (Note 2). Assignment and License Agreement with Shire International GmbH (Takeda) In November 2018, the Company entered into an Assignment and License Agreement (the “Shire Agreement”) with Shire International GmbH (“Shire”), which was subsequently acquired by Takeda Pharmaceutical Company Limited (“Takeda”). Under the terms of the Shire Agreement, Shire granted the Company an exclusive, royalty bearing worldwide license to develop and commercialize its two product candidates, Livmarli and volixibat. As part of the Shire Agreement, the Company was assigned license agreements held by Shire with Satiogen, Pfizer Inc. (“Pfizer”) and Sanofi-Aventis Deutschland GmbH (“Sanofi”). The Company has the right to sublicense under the Shire Agreement and additionally has the right to sublicense under the Satiogen, Pfizer and Sanofi licenses subject to the terms of those license agreements. The Company is obligated to pay Shire up to an aggregate of $ 109.5 million upon the achievement of certain clinical development and regulatory milestones for Livmarli in certain indications and an additional $ 25.0 million upon regulatory approval of Livmarli for each and every other indication. In addition, the Company is required to pay up to an aggregate of $ 30.0 million upon the achievement of certain clinical development and regulatory milestones for volixibat solely for the first indication sought. Upon commercialization, the Company is obligated to pay Shire product sales milestones on total licensed products up to an aggregate of $ 30.0 million. The Company is also obligated to pay tiered royalties with rates ranging from low double-digits to mid-teens based upon annual worldwide net sales for all licensed products; however, these royalties are reduced in part by royalties due under the Satiogen and Sanofi licenses, as discussed below, related to Livmarli and volixibat, as applicable. The Company’s royalty obligations will continue on a licensed product-by-licensed product and country-by-country basis until the later to occur of the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country, expiration of any regulatory exclusivity for the licensed product in a country and ten years after the first commercial sale of a licensed product in such country. In January 2023, the Company paid the accrued regulatory milestone of $ 15.0 million associated with approval of Livmarli by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months of age and older. As of September 30, 2023, the Company accrued $ 5.0 million for a commercial milestone associated with product sales. There were no development or regulatory milestones achieved for Livmarli or volixibat during the nine months ended September 30, 2023 and 2022. Satiogen License Through the Shire Agreement, the Company was assigned a license agreement with Satiogen pursuant to which the Company obtained an exclusive, worldwide license to certain patents and know-how, with the right to sublicense to a third party subject to certain financial considerations. Pursuant to the terms of the license agreement, the Company was obligated to pay to Satiogen up to an aggregate of $ 10.5 million upon the achievement of certain milestones, of which $ 0.5 million was for initiation of certain development activities, $ 5.0 million for the completion of regulatory approvals and $ 5.0 million for commercialization activities. Additionally, the Company was required to pay a low single-digit royalty on net sales. The Company’s royalty obligations continued on a licensed product-by-licensed product and country-by-country basis until the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country. Royalty obligations under the Satiogen license were creditable against the royalty obligations to Shire under the Shire Agreement. The Company has not paid milestone payments pursuant to this agreement for the periods presented. In May 2022, the Company completed the merger and acquisition of Satiogen for total consideration of approximately $ 24.2 million. At acquisition, Satiogen’s assets consisted of cash and intangible assets related to developed technology. The purchase consideration consisted of 609,305 shares of the Company’s common stock issued upon the closing of the acquisition and cash consideration of $ 2.6 million, excluding $ 0.2 million of stock option exercise prices deemed to have been paid immediately prior to the acquisition, in respect of an equivalent amount of cash on the books of Satiogen, with up to an additional 32,494 shares of common stock that would have been issued upon the closing of the acquisition except the parties agreed to such shares being held back by the Company for 12 months from the acquisition date to satisfy certain purchase price adjustments and indemnification obligations that may arise during this period. Specifically, purchase price adjustments and indemnification obligations that arise will reduce the number of shares issuable by the Company at settlement in accordance with the terms of the definitive acquisition agreement. The purchase consideration also included issuance of up to an additional 199,993 shares of the Company’s common stock, contingent upon the achievement of a certain milestone by June 30, 2025, subject to adjustment to satisfy certain purchase price adjustments and indemnification obligations that may arise. In December 2022, with the approval of Livmarli by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months of age and older, the milestone was achieved and the Company issued 199,993 shares of common stock in January 2023. Additionally, in June 2023, the contingencies related to the held back shares were resolved and the Company issued 31,631 shares of common stock. Through the transaction, the Company obtained all Satiogen licensing payments and Satiogen-owned intellectual property relating to Livmarli and volixibat. The transaction resulted in a reduction of total licensing royalty obligations for Livmarli and volixibat. The Company accounted for the transaction as an asset acquisition as the set of acquired assets did not constitute a business and substantially all the fair value of the gross assets acquired was concentrated in a group of similar identifiable assets, namely, the Satiogen intangible assets consisted of intellectual property. The Company evaluated that the intellectual property assets acquired were deemed to be commercially viable and the cost of the acquisition was recorded as an intangible asset. There was no gain or loss recognized from settlement of the preexisting contractual relationship with Satiogen as the pre-existing contract was determined to be at fair value on the date of acquisition. Through the date of settlement in June 2023, there were no significant expenses incurred that were approved for settlement against the Indemnification Holdback. As the number of shares potentially issuable upon the resolution of the Indemnification Holdback and the Contingent Milestone was variable, they were recorded as liabilities at their respective fair values on the date of acquisition using the Company’s common stock price. The fair value of the Indemnification Holdback was additionally determined based on management’s estimate of the probability of indemnification obligations being incurred during the one year following the acquisition date, while the fair value of the Contingent Milestone was additionally determined based upon management’s estimate of the probability of the milestone being met until the contingency was resolved in December 2022. The fair value of the Indemnification Holdback liability and the Contingent Milestone liability were remeasured at each reporting period until settled, with resulting changes in the fair value recorded in other income (expense) in the accompanying unaudited condensed consolidated statements of operations. The following represents the consideration paid and allocation of purchase price for the acquisition of Satiogen (in thousands, except per share data): Issued common stock $ 15,585 Cash consideration 2,600 Indemnification Holdback 831 Contingent consideration settled in common stock 4,600 Transaction costs 545 Total purchase consideration 24,161 Assets acquired: Intangible assets - developed technology 21,561 Cash consideration 2,600 Total assets acquired $ 24,161 Pfizer License Through the Shire Agreement, the Company was assigned a license agreement with Pfizer pursuant to which the Company obtained an exclusive, worldwide license to certain Pfizer know-how with a right to sublicense. Upon commercialization of any product utilizing the licensed product, the Company will be required to pay to Pfizer a low single-digit royalty on net sales of product sold by the Company, its affiliates or sublicensees. The Company’s royalty obligations continue on a licensed product-by-licensed product basis until the eighth anniversary of the first commercial sale of such licensed product anywhere in the world. Sanofi License Through the Shire Agreement, the Company was assigned a license agreement with Sanofi pursuant to which the Company obtained an exclusive, worldwide license to certain patents and know-how with the right to sublicense to a third party subject to certain financial considerations. The Company is obligated to pay up to an aggregate of $ 36.0 million upon the achievement of certain regulatory, commercialization and product sales milestones. Additionally, upon commercialization, the Company is required to pay tiered royalties in the mid to high single-digit range based upon net sales of licensed products sold by the Company and sublicensees in a calendar year, subject to adjustments in certain circumstances. The Company’s royalty obligations continue on a licensed product-by-licensed product and country-by-country basis until the later to occur of the expiration of the last valid claim in a licensed patent covering the applicable licensed product in such country and ten years after the first commercial sale of a licensed product in such country. Royalty obligations under the Sanofi license are creditable against the royalty obligations to Shire under the Shire Agreement. The Company has not paid milestone payments pursuant to this agreement for the periods presented. As of September 30, 2023 , no milestones had been accrued as there were no potential milestones considered probable. |
Collaboration and License Agree
Collaboration and License Agreements | 9 Months Ended |
Sep. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Collaboration and License Agreements | 8. Collaboration and License Agreements License and Collaboration Agreement with CANbridge In April 2021, the Company entered into an exclusive license and collaboration agreement with CANbridge Pharmaceuticals, Inc. (“CANbridge”). Under the terms of the agreement, CANbridge has obtained the exclusive right to develop and commercialize Livmarli within the Greater China regions (China, Hong Kong, Macau and Taiwan). In connection with the agreement, the Company received an upfront payment of $ 11.0 million, which, upon satisfaction of the performance obligation and receipt by CANbridge of the right to use and benefit from the license in May 2021, was recorded as license revenue. Additionally, the Company is eligible to receive up to $ 5.0 million in research and development funding, and up to $ 109.0 million for the achievement of future regulatory and commercial milestones, with double-digit tiered royalties based on product net sales. The Company concluded at inception of the agreement that the transaction price should not include the variable consideration related to unachieved developmental and regulatory milestones as this consideration was considered to be constrained as it is probable that the inclusion of such variable consideration could result in a significant reversal in cumulative revenue. The Company will recognize any consideration related to sales-based payments when the related sales occur, as the Company has determined that these amounts relate predominantly to the license granted and therefore will be recognized at the later of (i) when or as the related sales occur, or (ii) when the performance obligation to which some or all of the royalty has been allocated has been satisfied (or partially satisfied). The Company re-evaluates the transaction price at each reporting period as uncertain events are resolved and other changes in circumstances occur. In January 2022, CANbridge achieved a regulatory milestone, triggering a milestone payment to the Company of $ 2.0 million which was recorded as license revenue in the accompanying unaudited condensed consolidated statements of operations for the nine months ended September 30, 2022. In June 2023, CANbridge achieved another regulatory milestone, triggering a milestone payment to the Company of $ 5.0 million, which, upon the release of the constraint, was included in the transaction price and recorded as license revenue in the accompanying unaudited condensed consolidated statements of operations for the nine months ended September 30, 2023. For the three and nine months ended September 30, 2023 , the Company recorded research and development funding of $ 0.8 million and $ 2.2 million, respectively, and for the three and nine months ended September 30, 2022 , the Company recorded research and development funding of $ 0.2 million and $ 0.9 million, respectively, payable by CANbridge to the Company which is reflected as a reduction of research and development expense in the accompanying unaudited condensed consolidated statements of operations. As of September 30, 2023 and December 31, 2022 , such research and development funding of $ 2.4 million and $ 0.2 million, respectively, was recorded as a receivable which was included in accounts receivable on the accompanying unaudited condensed consolidated balance sheets. License and Collaboration Agreement with GC Biopharma In July 2021, the Company entered into an exclusive license and collaboration agreement with GC Biopharma. Under the terms of the agreement, GC Biopharma has obtained the exclusive right to develop and commercialize Livmarli within South Korea for ALGS, progressive familial intrahepatic cholestasis (“PFIC”), and biliary atresia (“BA”). In connection with the agreement, the Company received a $ 5.0 million upfront payment, which, upon satisfaction of the performance obligation and receipt by GC Biopharma of the right to use and benefit from the license, was recorded as license revenue. Additionally, the Company is entitled to certain research and development funding and up to $ 23.0 million for the achievement of future regulatory and commercial milestones, with double-digit tiered royalties based on product net sales. At inception of the agreement, the Company concluded that the transaction price should not include the variable consideration related to unachieved developmental and regulatory milestones as this consideration was considered to be constrained as it is probable that the inclusion of such variable consideration could result in a significant reversal in cumulative revenue for this contract when the uncertainty is resolved in the future. The Company will recognize any consideration related to sales-based payments (including milestones and royalties) when the related sales occur, as the Company has determined that these amounts relate predominantly to the license granted and therefore will be recognized on the later to occur of satisfaction of the performance obligation or the occurrence of the related sales. The Company re-evaluates the transaction price at each reporting period as uncertain events are resolved and other changes in circumstances occur. In February 2023, GC Biopharma achieved a regulatory milestone under this agreement triggering a milestone payment to the Company of $ 2.5 million, which upon the release of the constraint was included in the transaction price and recognized as license revenue in the accompanying unaudited condensed consolidated statements of operations for the nine months ended September 30, 2023. For the three and nine months ended September 30, 2023, the Company recorded research and development funding of $ 0.1 million and $ 0.3 million, respectively, payable by GC Biopharma to the Company which is reflected as a reduction of research and development expense in the accompanying unaudited condensed consolidated statements of operations. For the three and nine months ended September 30, 2022, research and development funding payable by GC Biopharma to the Company was insignificant. As of September 30, 2023, such research and development funding of $ 0.3 million was recorded as a receivable which is included in accounts receivable on the accompanying unaudited condensed consolidated balance sheets. Licensing Agreement with Takeda In September 2021, the Company entered into an exclusive licensing agreement with Takeda for the development and commercialization of Livmarli in Japan for ALGS, PFIC, and BA. Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of Livmarli in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications. The Company is responsible for commercial supply to Takeda. In exchange, the Company is eligible to receive a percentage of Takeda’s annualized net sales, which range from high double digits declining to mid double digits over the first four years from commercial launch and thereafter remains at mid double digits. The Company fully constrained all revenues upon transfer of control of the license to Takeda, which occurred when Takeda could use and benefit from the license, and will recognize any consideration related to sales-based payments when the related sales occur, as the Company has determined that these amounts relate predominantly to the license granted and therefore will be recognized on the later to occur of satisfaction of the performance obligation or the occurrence of the related sales. |
Leases
Leases | 9 Months Ended |
Sep. 30, 2023 | |
Leases [Abstract] | |
Leases | 9. Leases In January 2019, the Company entered into an operating lease agreement for office space which consisted of approximately 5,600 square feet (the “Initial Lease”). The lease term is approximately four years with an option to extend the term for one five-year term, which at the time was not reasonably assured of exercise and therefore, not included in the lease term. The lease contained a tenant improvement allowance of $ 0.4 million, which has been recorded as leasehold improvements on the accompanying unaudited condensed consolidated balance sheets with a corresponding reduction of the right-of-use (“ROU”) asset at inception of the lease. Rent payments commenced in August 2019. In November 2019, the Company amended the operating lease agreement (the “Amended Agreement”) to extend the term of the Initial Lease through March 2025. This extension was accounted for as a lease modification and the Company recorded an increase to the ROU asset and lease liability of $ 0.6 million at the time of the amendment. Additionally, pursuant to the Amended Agreement, the Company expanded the office space by 5,555 square feet for a five-year term expiring in March 2025 (the “Expanded Space”). The Company accounted for the Expanded Space as a separate contract as there were material additional rights of use that were not included in the Initial Lease. The Amended Agreement contained a tenant improvement allowance of $ 0.8 million in connection with the expanded space, which has been recorded as leasehold improvements within property and equipment, net on the accompanying unaudited condensed consolidated balance sheets with a corresponding reduction of the ROU asset at inception of the lease for the expanded space. The ROU and corresponding lease liabilities were estimated using a weighted-average incremental borrowing rate of 8.0 %. As of September 30, 2023, the Company recorded an aggregate ROU asset of $ 1.4 million and an aggregate lease liability of $ 2.0 million on the accompanying unaudited condensed consolidated balance sheets. The weighted-average remaining lease term is 2.2 years. As of September 30, 2023, undiscounted future minimum payments under the Company’s operating leases are as follows (in thousands): Undiscounted 2023 (remaining three months) $ 298 2024 1,187 2025 355 2026 113 2027 113 Thereafter 56 Total undiscounted lease payments 2,122 Less: imputed interest ( 172 ) Total lease liability $ 1,950 Rent expense was $ 0.2 million for each of the three months ended September 30, 2023 and 2022, and $ 0.6 million and $ 0.5 million for the nine months ended September 30, 2023 and 2022, respectively. Variable lease payments for operating expenses for the three and nine months ended September 30, 2023 and 2022 were immaterial. |
Convertible Notes
Convertible Notes | 9 Months Ended |
Sep. 30, 2023 | |
Debt Disclosure [Abstract] | |
Convertible Notes | 10. Convertible Notes In April 2023, the Company issued $ 316.3 million aggregate principal amount of its 4.00 % Convertible Senior Notes due 2029 (the "Notes") in a private placement to qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended. The Notes are subject to the terms and conditions of an Indenture (the “Indenture”) between the Company and U.S. Bank Trust Company, National Association, as trustee. The net proceeds from the issuance of the Notes were $ 305.3 million, after deducting the initial purchasers’ discounts and commissions and offering expenses. The Notes are the Company’s senior, unsecured obligations and are (i) senior in right of payment to any of the Company’s indebtedness that is expressly subordinated to the Notes in right of payment; (ii) equal in right of payment to any of the Company’s indebtedness that is not so subordinated; (iii) effectively subordinated to any of the Company’s secured indebtedness, to the extent of the value of the collateral securing that indebtedness; and (iv) structurally subordinated to all indebtedness and other liabilities, including trade payables, and (to the extent the Company is not a holder thereof) preferred equity, if any, of the Company’s subsidiaries. The Notes accrue interest at a rate of 4.00 % per annum, payable semi-annually in arrears on May 1 and November 1 of each year, beginning on November 1, 2023. The Notes will mature on May 1, 2029, unless earlier converted, redeemed or repurchased by the Company. Before January 2, 2029, noteholders will have the right to convert their Notes only in the following circumstances: (i) during any calendar quarter (and only during such calendar quarter) commencing after the calendar quarter ending on June 30, 2023, if the last reported sale price per share of the Company’s common stock exceeds 130 % of the conversion price for the Notes for each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the last trading day of the immediately preceding calendar quarter; (ii) during the five consecutive business days immediately after any 10 consecutive trading day period (such 10 consecutive trading day period, the “measurement period”) if the trading price per $1,000 principal amount of Notes for each trading day of the measurement period was less than 98 % of the product of the last reported sale price per share of the common stock on such trading day and the conversion rate on such trading day; (iii) upon the occurrence of certain corporate events or distributions on the common stock, as described in the Indenture; and (iv) if the Company calls such Notes for redemption. Additionally, noteholders will have the right to convert their Notes at any time from January 2, 2029 until the close of business on the scheduled trading day immediately before the maturity date. The Company may settle conversions by paying or delivering, as applicable, cash, shares of its common stock or a combination of cash and shares of its common stock, at the Company’s election. The initial conversion rate for the Notes is 31.5075 shares of common stock per $ 1,000 principal amount of Notes, which represents an initial conversion price of approximately $ 31.74 per share of common stock. The conversion rate and conversion price will be subject to customary adjustments upon the occurrence of certain events. In addition, if certain corporate events that constitute a “Make-Whole Fundamental Change” (as defined in the Indenture) occur, then the conversion rate will, in certain circumstances, be increased for a specified period of time. The Company may not redeem the Notes at its option at any time before May 5, 2026. The Notes are redeemable, in whole or in part (subject to the partial redemption limitation described below), at the Company’s option at any time, and from time to time, on or after May 5, 2026 and, in the case of a partial redemption, on or before the 50th scheduled trading day immediately before the maturity date, at a cash redemption price equal to the principal amount of the Notes to be redeemed, plus accrued and unpaid interest, if any, to, but excluding, the redemption date, but only if the last reported sale price per share of the Company's common stock exceeds 130 % of the conversion price on (i) each of at least 20 trading days, whether or not consecutive, during the 30 consecutive trading days ending on, and including, the trading day immediately before the date the Company sends the related redemption notice; and (ii) the trading day immediately before the date the Company sends such notice. In addition, calling any Note for redemption will constitute a Make-Whole Fundamental Change with respect to that Note, in which case the conversion rate applicable to the conversion of that Note will be increased in certain circumstances if it is converted after it is called for redemption. Pursuant to the partial redemption limitation, the Company may not elect to redeem less than all of the outstanding Notes unless at least $ 75.0 million aggregate principal amount of Notes are outstanding and not called for redemption as of the time the Company sends the related redemption notice. If a “Fundamental Change” (as defined in the Indenture) occurs, then, subject to a limited exception for certain cash mergers, noteholders may require the Company to repurchase their Notes at a cash repurchase price equal to the principal amount of the Notes to be repurchased, plus accrued and unpaid interest, if any, to, but excluding, the fundamental change repurchase date. The definition of Fundamental Change includes certain business combination transactions involving the Company and certain de-listing events with respect to the Company's common stock. The Indenture contains customary events of default with respect to the Notes and provides that upon certain events of default occurring and continuing, the Trustee may, and the Trustee at the request of holders of at least 25 % in principal amount of the Notes shall, declare all principal and accrued and unpaid interest, if any, of the Notes to be due and payable. In case of certain events of bankruptcy, insolvency or reorganization, involving the Company or a significant subsidiary, all of the principal of and accrued and unpaid interest on the Notes will automatically become due and payable. As of September 30, 2023, the Notes consisted of the following (in thousands): Convertible Notes Principal amount $ 316,250 Unamortized debt discount and issuance costs ( 10,228 ) Net carrying amount $ 306,022 The Company incurred $ 10.9 million of transaction costs related to the issuance of the Notes, which are being amortized to interest expense over the term of the Notes using the effective interest method. As of September 30, 2023, the remaining amortization period of the debt discount was approximately 5.6 years and the effective interest on the Notes was 4.6 %. The following table sets forth interest expense recognized related to the Notes (in thousands): Three Months Ended Nine Months Ended 2023 2023 Coupon interest expense $ 3,163 $ 5,764 Amortization of debt discount and issuance costs 411 718 Total interest expense on convertible notes $ 3,574 $ 6,482 As of September 30, 2023, the estimated fair value of the Notes was $ 402.5 million. The fair values were determined based on the quoted price of the convertible notes in an inactive market on the last trading day of the reporting period and has been classified as Level 2 in the fair value hierarchy. |
Stockholders' Equity
Stockholders' Equity | 9 Months Ended |
Sep. 30, 2023 | |
Equity [Abstract] | |
Stockholders' Equity | 11. Stockholders’ Equity Common Stock In August 2020, the SEC declared effective a registration statement on Form S-3 (“2020 Shelf Registration”) covering the sale of up to $ 300.0 million of the Company’s securities. Also, in August 2020, the Company entered into a sales agreement (“2020 Sales Agreement”) with SVB Securities LLC, recently acquired by Leerink Partners LLC (“Leerink”) pursuant to which the Company may elect to issue and sell, from time to time, shares of common stock having an aggregate offering price of up to $ 75.0 million under the 2020 Shelf Registration through Leerink acting as the sales agent and/or principal. During the nine months ended September 30, 2023, the Company issued and sold 658,206 shares of common stock pursuant to the 2020 Sales Agreement resulting in gross proceeds to the Company of $ 15.0 million. The net proceeds to the Company for the nine months ended September 30, 2023, after deducting sales commissions to Leerink and other issuance expenses were approximately $ 14.5 million. As of September 30, 2023 , the Company has issued and sold an aggregate of 2,125,090 shares of common stock pursuant to the 2020 Sales Agreement resulting in aggregate gross proceeds to the Company of $ 43.7 million. The 2020 Shelf Registration expired in August 2023, and no further sales may be made under the 2020 Sales Agreement. In August 2022, the Company completed an underwritten public offering of its common stock pursuant to the 2020 Shelf Registration. The Company issued and sold 3,478,261 shares of common stock at a public offering price of $ 23.00 per share. In addition, the Company granted the underwriters an option, exercisable for 30 days, to purchase up to 521,739 additional shares of its common stock at the public offering price, less the underwriting discounts, commissions and offering expenses, which the underwriters exercised in full. The underwritten public offering, including the underwriters’ exercise of their option, resulted in net proceeds to the Company of $ 86.1 million after deducting underwriting discounts, commissions and offering expenses. On September 9, 2022, the Company filed an automatic shelf registration statement on Form S-3 with the SEC (the “2022 Shelf Registration”), which became effective upon filing, pursuant to which the Company registered for sale from time to time in one or more offerings an unlimited amount of any combination of the Company’s common stock, preferred stock, debt securities and warrants, so long as the Company continues to satisfy the requirements of a “well-known seasoned issuer” under SEC rules. This automatic shelf registration statement will remain in effect for up to three years from the date it became effective. As of September 30, 2023, the Company had not issued any securities pursuant to the automatic shelf registration statement. On August 31, 2023, in connection with and immediately prior to the closing of the Bile Acid Portfolio Acquisition, the Company completed the private placement of 8,000,000 shares of the Company’s common stock at a price per share of $ 26.25 , resulting in net proceeds of approximately $ 202.2 million. Common Stock Reserved for Issuance Common stock reserved for issuance is as follows: As of September 30, As of December 31, 2023 2022 Stock options, restricted stock units and performance stock units issued and outstanding 10,814,495 8,955,557 Reserved for future stock awards or option grants 2,412,737 1,596,947 Reserved for employee stock purchase plan 1,081,089 1,157,570 Common stock issuable upon conversion of convertible notes 9,964,247 — Common stock held back in connection with asset acquisition — 31,638 Common stock issuable as contingent consideration in connection with asset acquisition — 199,993 24,272,568 11,941,705 |
Stock-Based Compensation
Stock-Based Compensation | 9 Months Ended |
Sep. 30, 2023 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-Based Compensation | 12. Stock-Based Compensation Equity Incentive Plans In November 2018, the Company adopted the 2018 Equity Incentive Plan (the “2018 Plan”), which permits the granting of stock awards and incentive and nonstatutory stock options to employees, directors and consultants of the Company. In July 2019, the Company’s board of directors and stockholders approved and adopted the 2019 Equity Incentive Plan (the “2019 Plan”). The 2019 Plan became effective on July 17, 2019. Under the 2019 Plan, the Company may grant stock options, stock appreciation rights, restricted stock, restricted stock units and other stock or cash-based awards to individuals who are then employees, officers, directors or consultants of the Company. Shares subject to outstanding awards under the 2018 Plan as of the effective date of the 2019 Plan that are subsequently canceled, forfeited or repurchased by the Company will be added to the shares reserved under the 2019 Plan. In addition, the number of shares of common stock available for issuance under the 2019 Plan will be automatically increased on the first day of each calendar year during the ten-year term of the 2019 Plan, beginning with January 1, 2020 and ending with January 1, 2029 , by an amount equal to 5 % of the outstanding number of shares of the Company’s common stock on December 31st of the preceding calendar year or such lesser amount as determined by the Company’s board of directors. As of September 30, 2023, 1,110,837 shares of common stock were available for issuance under the 2019 Plan. In March 2020, the compensation committee of the Company’s board of directors approved and adopted the 2020 Inducement Plan (the “2020 Inducement Plan”). Under the 2020 Inducement Plan, the Company may grant nonstatutory stock options, stock appreciation rights, restricted stock and restricted stock units to new employees entering into employment with the Company in accordance with Nasdaq Listing Rule 5635(c)(4). At adoption, the 2020 Inducement Plan authorized 750,000 shares of the Company’s common stock for future issuance. In 2023, 2021 and 2020, the Company’s board of directors authorized an additional 1,500,000 , 1,000,000 and 750,000 shares of the Company’s common stock for future issuance, respectively. As of September 30, 2023 , 1,301,900 shares of common stock were available for issuance under the 2020 Inducement Plan. Stock Options The following table summarizes stock option activity during the nine months ended September 30, 2023 (in thousands, except share and per share data): Number of Weighted- Weighted- Aggregate Outstanding as of December 31, 2022 8,340,083 $ 13.63 7.5 $ 51,645 Granted 1,925,589 $ 24.96 Exercised ( 437,959 ) $ 15.89 Canceled and forfeited ( 240,546 ) $ 18.40 Outstanding as of September 30, 2023 9,587,167 $ 15.68 7.1 $ 152,622 Vested and exercisable as of September 30, 2023 5,726,809 $ 11.85 6.1 $ 113,100 Intrinsic value is calculated as the difference between the exercise price of the underlying options and the fair value of the common stock for the options that had exercise prices that were lower than the per share fair value of the common stock on the date of exercise. The weighted-average grant date fair value per share of stock options granted during the nine months ended September 30, 2023 and 2022 was $ 17.96 and $ 12.71 per share, respectively. The total intrinsic value of options exercised during the nine months ended September 30, 2023 and 2022 was $ 4.7 million and $ 2.6 million, respectively . As of September 30, 2023, the total unrecognized stock-based compensation related to unvested stock option awards granted was $ 52.3 million, which the Company expects to recognize over a weighted-average period of approximately 2.6 years. The fair value of each employee and non-employee stock option grant is estimated on the date of grant using the Black-Scholes option-pricing model. Due to the Company’s limited operating history and a lack of company specific historical and implied volatility data, the expected stock price volatility was based upon the weighting of the Company’s historical volatility and the historical volatility of a peer group of publicly traded companies. The historical volatility data was computed using the daily closing prices for the Company’s and its peer companies’ shares during the equivalent period of the calculated expected term of the stock-based awards. Due to the lack of historical exercise history, the expected term of the Company’s stock options for employees has been determined utilizing the “simplified” method for awards. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve in effect at the time of grant of the award for time periods approximately equal to the expected term of the award. Expected dividend yield is zero based on the fact that the Company has never paid cash dividends and does not expect to pay any cash dividends in the foreseeable future. The following assumptions were used to estimate the fair value of stock option awards granted during the following periods: Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Expected term (in years) 6.08 6.08 5.31 - 6.08 5.50 - 6.08 Expected volatility 80.99 % - 81.63 % 81.13 %- 81.70 % 80.99 % - 85.24 % 80.86 %- 83.20 % Risk-free interest rate 3.97 % - 4.39 % 2.65 %- 3.44 % 3.35 - 4.39 1.46 %- 3.44 % Expected dividend yield — — — — Restricted Stock Units The following table summarizes the activity under the Company’s restricted stock units for the nine months ended September 30, 2023: Number of Weighted-Average Grant Date Unvested and outstanding as of December 31, 2022 615,474 $ 18.36 Granted 754,324 $ 25.00 Vested ( 235,130 ) $ 18.36 Cancelled/Forfeited ( 55,175 ) $ 20.29 Unvested and outstanding as of September 30, 2023 1,079,493 $ 22.89 The fair value of restricted stock unit awards granted to employees and nonemployees is equal to the closing market price of the Company’s common stock on the grant date. As of September 30, 2023, the total unrecognized stock-based compensation related to restricted stock unit awards granted was $ 20.0 million, which the Company expects to recognize over a weighted-average period of approximately 2.3 years. Performance Stock Units In January 2023, the Company granted an aggregate of 135,835 performance stock units to certain executive participants (“2023 Executive PSUs”). The 2023 Executive PSUs are subject to a performance condition of achieving certain net product sales levels related to Livmarli during the year ended December 31, 2024. If the performance condition is met, the first tranche of the award will vest on March 15, 2025 and the second tranche will vest on March 15, 2026, subject to the executive employees’ continuous service through each vesting date. The number of units to be vested in the first tranche of the 2023 Executive PSUs is calculated by multiplying two-thirds of the 2023 Executive PSUs granted by a percentage calculated based on attained Livmarli sales metrics, as certified by the Company’s Compensation Committee. The number of units to be vested in the second tranche of the 2023 Executive PSUs equals 50 % of the units vested in the first tranche. In June 2023, the Company granted an aggregate of 12,000 PSUs to certain employees ("2023 Employee PSUs"). The 2023 Employee PSUs are subject to a performance condition of achieving certain net product sales related to Livmarli in the US for the year ended December 31, 2023. The following table summarizes the activity under the Company's performance stock units for the nine months ended September 30, 2023: Number of Weighted-Average Grant Date Unvested and outstanding as of December 31, 2022 — $ — Granted 147,835 $ 23.64 Unvested and outstanding as of September 30, 2023 147,835 $ 23.64 As of September 30, 2023, the total unrecognized stock compensation related to performance stock units granted was $ 1.3 million, which the Company expects to recognize over a weighted-average period of approximately 1.7 years. 2019 Employee Stock Purchase Plan In July 2019, the Company’s board of directors and stockholders approved and adopted the 2019 Employee Stock Purchase Plan (“ESPP”). A total of 500,000 shares of common stock were approved to be initially reserved for issuance under the ESPP. In addition, the number of shares of common stock available for issuance under the ESPP will be automatically increased on the first day of each calendar year during the first ten years of the term of the ESPP, beginning with January 1, 2020 and ending with January 1, 2029 , by an amount equal to the lesser of (i) 1 % of the outstanding number of shares of common stock on December 31st of the preceding calendar year, (ii) 1,500,000 shares of common stock or (iii) such lesser amount as determined by the Company’s board of directors. The ESPP became effective on July 17, 2019 and generally provides for six-month consecutive offering periods beginning on May 11th and November 11th of each year. During the three months ended September 30, 2023, no shares were issued under the ESPP. During the nine months ended September 30, 2023, 76,481 shares were issued under the ESPP. As of September 30, 2023 , the Company had 1,081,089 shares available for future issuance under the ESPP. The stock-based compensation related to the ESPP was $ 0.2 million and $ 0.1 million for the three months ended September 30, 2023 and 2022, and $ 0.7 million and $ 0.5 million for the nine months ended September 30, 2023 and 2022, respectively. Restricted Common Stock In November 2018, in connection with the issuance of Series A Preferred Stock, the Company’s founders agreed to modify their outstanding shares of common stock to include vesting provisions that require continued service to the Company in order to vest in those shares. As such, the 562,500 modified shares of common stock became compensatory upon such modification. All restricted common stock was fully vested as of December 31, 2022. During the three and nine months ended September 30, 2022, 33,398 and 100,194 shares vested, respectively. Stock-Based Compensation Total stock-based compensation is reflected in the accompanying unaudited condensed consolidated statements of operations as follows (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Selling, general and administrative $ 5,759 $ 4,391 $ 17,290 $ 12,592 Research and development 2,626 2,517 8,013 7,519 Total $ 8,385 $ 6,908 $ 25,303 $ 20,111 Stock-based compensation capitalized into inventory was $ 0.2 million for each of the three months ended September 30, 2023 and 2022, and $ 0.6 million and $ 0.3 million for the nine months ended September 30, 2023, and 2022, respectively. |
Contingencies
Contingencies | 9 Months Ended |
Sep. 30, 2023 | |
Commitments and Contingencies Disclosure [Abstract] | |
Contingencies | 13. Contingencies The Company is subject to potential liabilities under government regulations and various claims and legal actions that are pending or may be asserted from time-to-time. These matters arise in the ordinary course and conduct of the Company’s business and may include, for example, commercial, intellectual property, and employment matters. The Company intends to defend itself vigorously in such matters and when warranted, take legal action against others. Furthermore, the Company regularly assesses contingencies to determine the degree of probability and range of possible loss for potential accrual in its financial statements. An estimated loss contingency is accrued in the Company’s financial statements if it is probable that a liability has been incurred and the amount of the loss can be reasonably estimated. The Company does not accrue amounts for liabilities that it does not believe are probable. Litigation is inherently unpredictable, and unfavorable resolutions could occur. As a result, assessing contingencies is highly subjective and requires judgment about future events. During the periods presented, the Company has not recorded any accrual for loss contingencies associated with such government regulations, claims or legal actions, determined that an unfavorable outcome is probable or reasonably possible, or determined that the amount or range of any possible loss is reasonably estimable. |
Subsequent Events
Subsequent Events | 9 Months Ended |
Sep. 30, 2023 | |
Subsequent Events [Abstract] | |
Subsequent Events | 14. Subsequent Events On November 2, 2023, the Company entered into a Sales Agreement (the “2023 Sales Agreement”) with Leerink and Cantor Fitzgerald & Co. (the “Sales Agents”), pursuant to which the Company may, from time to time, sell up to an aggregate amount of $ 200.0 million of its common stock through the Sales Agents in an “at-the-market” offering (the “ATM Offering”). The Company is not required to sell shares under the 2023 Sales Agreement. Sales of the Company’s common stock, if any, under the 2023 Sales Agreement may be made in any transactions that are deemed to be “at the market offerings” as defined in Rule 415 under the Securities Act. The Company will pay a given designated Sales Agent a commission of up to 3.0 % of the aggregate gross proceeds of any shares of common stock sold through such Sales Agent pursuant to the 2023 Sales Agreement. No sales may be made under the 2023 Sales Agreement until a prospectus supplement relating to the ATM Offering is filed with the Securities and Exchange Commission. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
Basis of Presentation | Basis of Presentation The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with U.S. generally accepted accounting principles (“GAAP”) for interim financial information and pursuant to Form 10-Q and Article 10 of Regulation S-X of the Securities and Exchange Commission (“SEC”). Accordingly, the accompanying unaudited condensed consolidated financial statements do not include all of the information and notes required by GAAP for complete financial statements. The unaudited interim financial statements reflect all adjustments which, in the opinion of management, are necessary for a fair statement of the results for the periods presented. All such adjustments are of a normal and recurring nature. The unaudited condensed consolidated balance sheet as of December 31, 2022 has been derived from the audited consolidated financial statements at that date but does not include all information and footnotes required by GAAP for complete financial statements. The operating results presented in these unaudited condensed consolidated financial statements are not necessarily indicative of the results that may be expected for any future periods. The accompanying unaudited condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiaries. All significant intercompany balances and transactions have been eliminated in consolidation. These unaudited condensed consolidated financial statements should be read in conjunction with the audited consolidated financial statements and the notes thereto in the Company’s Annual Report on Form 10-K (“Annual Report”) for the fiscal year ended December 31, 2022, as filed with the SEC on March 8, 2023. |
Use of Estimates | Use of Estimates The preparation of consolidated financial statements in accordance with GAAP requires management to make estimates and assumptions that impact the reported amounts of assets, liabilities and expenses and the disclosure of contingent assets and liabilities in the financial statements and accompanying notes. These estimates and assumptions are based upon historical experience, knowledge of current events and various other factors believed to be reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets and liabilities and the recording of expenses that are not readily apparent from other sources. Actual results could differ materially from those estimates. The Company’s unaudited condensed consolidated financial statements as of and for the three and nine months ended September 30, 2023 reflect the Company’s estimates of the impact of the geopolitical and macroeconomic environment, including the impact of inflation, bank failures, higher interest rates and foreign exchange rate fluctuations. The duration and the scope of these conditions cannot be predicted; therefore, the extent to which these conditions will directly or indirectly impact the Company’s business, results of operations and financial condition, is uncertain. The Company is not aware of any specific event or circumstance that would require an update to its estimates, judgments and assumptions or a revision of the carrying value of the Company’s assets or liabilities as of the date of this filing. |
Significant Accounting Policies | Significant Accounting Policies There have been no significant changes to the accounting policies during the nine months ended September 30, 2023, as compared to the significant accounting policies described in Note 2 of the “Notes to Consolidated Financial Statements” in the Company’s audited consolidated financial statements included in the Annual Report, unless indicated below. |
Cash, Cash Equivalents and Restricted Cash Equivalents | Cash, Cash Equivalents and Restricted Cash Equivalents The Company considers all highly liquid investments that are readily convertible into cash without penalty and with original maturities of three months or less at the date of purchase to be cash equivalents. The carrying amounts reported in the unaudited condensed consolidated balance sheets for cash and cash equivalents are valued at cost, which approximate their fair value. Restricted cash equivalents as of December 31, 2022 consisted of deposits placed in a segregated bank account as required under the terms of the Company’s RIPA, as amended September 2021, with Mulholland SA LLC, an affiliate of Oberland Capital LLC, as agent for the purchasers party thereto (the “Purchasers”), and the Purchasers in connection with the sale of the Priority Review Voucher in December 2021. Upon repurchase and the termination of the RIPA in April 2023, in accordance with its terms, the previously restricted cash equivalents of $ 100.0 million were no longer restricted from use. The following table provides a reconciliation of cash, cash equivalents and restricted cash equivalents reported within the unaudited condensed consolidated balance sheets that together reflect the same amounts shown in the unaudited condensed consolidated statements of cash flows (in thousands): As of September 30, As of December 31, 2023 2022 Cash and cash equivalents $ 303,059 $ 28,003 Restricted cash equivalents — 100,000 Total cash, cash equivalents, and restricted cash equivalents $ 303,059 $ 128,003 |
Investments | Investments The Company classifies all investments in securities as available-for-sale. Management determines the appropriate classification of its investments in securities at the time of purchase. Investments with original maturities beyond three months at the date of purchase and which mature at, or less than twelve months from the balance sheet date, are classified as a current asset. Investments are recorded at fair value, with unrealized gains and losses reported as accumulated other comprehensive income (loss) until realized, with the exception of any declines in fair value below the cost basis that are a result of a credit loss, which, if any, are reported in other income (expense), net in the current period through an allowance for credit losses. Each reporting period, the Company evaluates whether declines in fair values of its available-for-sale securities below their cost basis are a result of credit loss or other factors and whenever events or changes in circumstances indicate that the carrying amount of an asset may not be recoverable. This evaluation consists of several qualitative and quantitative factors regarding the severity and duration of the unrealized loss, the creditworthiness of the security issuers, as well as the Company’s ability and intent to hold the available-for-sale security until a forecasted recovery occurs. Additionally, the Company assesses whether it has plans to sell the security or it is more likely than not it will be required to sell any available-for-sale securities before recovery of its amortized cost basis. The cost of debt securities is adjusted for amortization of premiums and accretion of discounts to maturity. Such amortization and accretion, as well as interest and dividends, are included in interest income. Realized gains and losses from the sale of available-for-sale securities, if any, are determined on a specific identification basis and are also included in Other income (expense). To date, the Company has no t identified any declines in fair value of its investments related to credit loss. |
Concentrations of Credit Risk and Off-Balance Sheet Risk | Concentrations of Credit Risk and Off-Balance Sheet Risk Financial instruments that potentially subject the Company to a concentration of credit risk consist of cash and cash equivalents, accounts receivable and investments. The Company limits the amount of credit exposure by investing cash that is not required for immediate operating needs in money market funds, government obligations and/or commercial paper with short maturities. Additionally, the Company has established guidelines regarding diversification of its investments and their maturities, which are designed to maintain principal and maximize liquidity. To date, the Company has not experienced any losses associated with this credit risk and continues to believe that this exposure is not significant. The Company relies on a single distributor and a specialty pharmacy for all of the Company’s sales of its approved medicines in the United States as well as a single distributor for sales of Livmarli outside the United States. The Company sources materials and services through several vendors. Certain materials are sourced from a single vendor. The loss of certain vendors could result in a temporary disruption of the Company’s commercialization efforts. As of September 30, 2023, the Company did no t have any customers that individually accounted for more than 10 % of accounts receivable. As of December 31, 2022, the Company had one customer that accounted for approximately 23 % of accounts receivable. For the three and nine months ended September 30, 2023 and 2022, the Company did not have revenue attributable to any one customer in excess of 10 % of sales. |
Accounts Receivable | Accounts Receivable The Company has accounts receivable amounts due from product sales. The Company also has accounts receivable amounts due from license agreements for milestones achieved, but not yet paid. Amounts payable to the Company are recorded as accounts receivable when the Company’s right to consideration is unconditional. The Company estimates the allowance for credit losses using the current expected model. Under this model, the allowance for credit losses reflects the Company’s estimate of lifetime expected credit losses. The Company evaluates the collectability of the cash flows based on the risk of loss over the contractual life, even when that risk is remote, based on judgments about the creditworthiness of its customers, historical experience and other relevant information that is available to the Company. There was no allowance for credit losses as of September 30, 2023. There was no bad debt expense for the three and nine months ended September 30, 2023 and 2022. |
Intangibles Assets, Net | Intangible Assets, Net The Company accounts for asset acquisitions that do not meet the definition of a business using the cost accumulation method, whereby the cost of the acquisition, including certain transaction costs, is allocated to the asset (or assets) acquired on the basis of its (or their) relative fair value(s) on the measurement date. No goodwill is recognized in an asset acquisition. Intangible assets are measured at their fair values as of the acquisition date or, in the case of commercial milestone payments, the date they become due. The evaluation of intangible assets includes assessing the amortization period for which the asset is expected to contribute to the future cash flows of the Company. Intangible assets with finite useful lives are amortized over their estimated useful lives, primarily on a straight-line basis when the Company is unable to reliably estimate the pattern of cash flow. The Company tests its finite lived intangible assets for impairment annually or if events or changes in circumstances indicate that it is more likely than not that the asset is impaired. If it is determined that the asset is impaired, the carrying value is written down to its estimated fair value, with the related impairment charge recognized in the unaudited condensed consolidated statements of operations in the period in which the impairment occurs. The Company has not recorded any impairments to its intangible assets for any of the periods presented. The components of the Company’s intangible assets were as follows (in thousands, except for weighted-average remaining amortization period): September 30, 2023 Gross Carrying Value Accumulated Amortization Net Carrying Amount Weighted-Average Remaining Amortization Period Developed technology $ 226,620 $ ( 5,487 ) $ 221,133 11.9 Commercial milestones 39,000 ( 2,765 ) 36,235 16.4 Assembled workforce 970 — 970 2.9 Total intangible assets $ 266,590 $ ( 8,252 ) $ 258,338 11.7 December 31, 2022 Gross Carrying Value Accumulated Amortization Net Carrying Amount Weighted-Average Remaining Amortization Period Commercial milestones $ 34,000 $ ( 1,333 ) $ 32,667 17.1 Developed technology 28,107 ( 1,820 ) 26,287 8.4 Total intangible assets $ 62,107 $ ( 3,153 ) $ 58,954 11.9 Amortization expense was $ 2.6 million and $ 5.1 million for the three and nine months ended September 30, 2023, respectively, and $ 1.0 million and $ 1.8 million for the three and nine months ended September 30, 2022, respectively. Amortization expense was included in cost of sales in the accompanying unaudited condensed consolidated statements of operations. The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of September 30, 2023 (in thousands): Amount 2023 (remaining three months) 5,413 2024 21,545 2025 21,545 2026 21,436 2027 21,221 Thereafter 167,178 $ 258,338 |
Convertible Notes | Convertible Notes The Company evaluates all conversion, repurchase and redemption features contained in a debt instrument to determine if there are any embedded features that require bifurcation as a derivative. The Company accounts for its convertible notes (see Note 10) as a long-term liability equal to the proceeds received from issuance, including any embedded conversion features, net of the unamortized debt discount and offering costs in the accompanying unaudited consolidated balance sheets. The debt issuance and offering costs are amortized over the contractual term of the convertible notes, using the effective interest method, as interest expense in the accompanying unaudited consolidated statements of operations. |
Product Sales, Net | Product Sales, Net The Company recognizes product sales, net when the customer obtains control of our product, which occurs at a point in time, typically upon delivery of the Company’s product to the customer. Revenues from product sales are recorded at the net sales price, or the transaction price, which may include fixed or variable consideration for discounts, government rebates, co-pay assistance, returns and other allowances that are offered within contracts with a customer relating to the sale of the Company's approved medicines. Estimates of variable consideration are calculated based on the actual product sales each reporting period and the nature of the variable consideration related to those sales. Overall, these estimates reflect the Company’s best estimate of the amount of consideration to which the Company expects to be entitled based on the terms of the contract. The amount of variable consideration that is included in the transaction price may be constrained and is included in product sales, net only to the extent that it is considered probable that a significant reversal in the amount of the cumulative revenue recognized will not occur when the uncertainty associated with the variable consideration is subsequently resolved. Estimates are reviewed and updated quarterly as additional information becomes known. Actual amounts of consideration ultimately received may differ materially from estimates. If actual results in the future vary from estimates, the Company will adjust these estimates, which would affect product sales, net and earnings in the period such variances are adjusted. Significant categories of sales discounts and allowances are as follows: Government Rebates : The Company records rebates payable under Medicaid and other government programs as a reduction of revenue at the time product revenues are generated. The Company’s rebate calculations may require estimates, including estimates of customer mix, to determine which sales will be subject to rebates and the amount of such rebates. The Company updates its estimates and assumptions on a quarterly basis and records any necessary adjustments to revenue in the period identified. The liability for unpaid rebates is included in accrued expenses on the accompanying unaudited condensed consolidated balance sheets. To date, actual government rebates have not differed materially from the Company’s estimates. Other Incentives : Other incentives include a branded co-pay assistance program for eligible patients with commercial insurance in the United States. The branded co-pay assistance program assists commercially insured patients who have coverage for the Company's approved medicines and is intended to reduce each participating patient’s portion of the financial responsibility of the purchase price up to a specified dollar amount of assistance. The calculation of the accrual for co-pay assistance is based upon an identification of claims and the cost per claims associated with product that has been recognized as revenue. The Company records amounts paid under the brand specific co-pay assistance program for each patient as a reduction of revenue from product sales. To date, actual other incentives have not differed materially from the Company’s estimates. Product Returns : The Company records revenue for product sales, net of estimated product returns. Customers have limited return rights related only to the product’s damage or defect identified upon delivery of the product. The Company estimates the amount of product sales that may be returned and records the estimate as a reduction of revenue and a refund liability in the period the related product revenue is recognized. To date, actual returns have not differed materially from the Company’s estimates. The following table disaggregates total Product sales, net: Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Product sales, net: Livmarli $ 38,708 $ 18,780 $ 100,303 $ 47,156 Bile Acid Medicines 9,017 — 9,017 — Total product sales, net 47,725 18,780 109,320 47,156 License revenue — — 7,500 2,000 Total revenues $ 47,725 $ 18,780 $ 116,820 $ 49,156 |
Net Loss Per Share | Net Loss Per Share Basic net loss per share is computed by dividing net loss by the weighted-average shares of common stock outstanding for the period, without consideration for potentially dilutive securities. Diluted net loss per share is computed by dividing the net loss by the weighted-average shares of common stock and potentially dilutive securities outstanding for the period determined using the treasury-stock and if-converted methods. Diluted net loss per share excludes the potential impact of the Company’s common stock subject to repurchase, common stock options, restricted stock units, contingently issuable employee stock purchase plan shares and common stock issuable upon conversion of convertible notes because their effect would be anti-dilutive due to the Company’s net loss. Basic and diluted net loss per share were the same for the three and nine months ended September 30, 2023. The following table sets for the computation of basic and diluted earnings per share for the three and nine months ended September 30, 2022 (in thousands, except share and per share data): Three Months Ended Nine Months Ended 2022 2022 Numerator: Net loss, basic $ ( 35,706 ) $ ( 99,234 ) Less: Change in fair value of Holdback Indemnification liability — 149 Net loss, diluted $ ( 35,706 ) $ ( 99,383 ) Denominator: Weighted-average shares of common stock outstanding, basic 34,927,790 32,809,365 Effect of dilutive securities: Weighted-average Holdback Indemnification shares issuable — 15,949 Weighted-average shares of common stock outstanding, diluted 34,927,790 32,825,314 Net loss per share, basic $ ( 1.02 ) $ ( 3.03 ) Net loss per share, diluted $ ( 1.02 ) $ ( 3.03 ) The following outstanding potential dilutive shares have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: As of September 30, As of September 30, 2023 2022 Options to purchase common stock and restricted stock units 10,814,495 8,910,376 Common stock issuable upon conversion of convertible notes 9,964,247 — Employee stock purchase plan contingently issuable 49,868 46,760 Common stock subject to repurchase — 22,270 Shares issuable as contingent consideration in connection with asset acquisition — 199,993 Total 20,828,610 9,179,399 |
Recently Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements On January 1, 2023, the Company adopted Accounting Standards Update (“ASU”) No. 2016-13, Financial Instruments-Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments (“ASU 2016-13”). ASU 2016-13 requires an entity to utilize a new impairment model that requires measurement and recognition of expected credit losses for most financial assets and certain other instruments, including but not limited to available-for-sale debt securities. Credit losses relating to available-for-sale debt securities will be recorded through an allowance for credit losses rather than as a direct write-down to the security. The new guidance also modifies the impairment models for available-for-sale debt securities and for purchased financial assets with credit deterioration since their origination. In March 2020, the Financial Accounting Standards Board (“FASB”) issued ASU No. 2020-3, Codification Improvements to Financial Instruments which makes narrow-scope improvements to various financial instruments topics, including the new credit losses standard and clarifies the following areas (i) the contractual term of a net investment in a lease should be the contractual term used to measure expected credit losses; (ii) when an entity regains control of financial assets sold, an allowance for credit losses should be recorded. There was no impact on the accompanying unaudited condensed consolidated financial statements as of the adoption date, January 1, 2023 . |
Recent Accounting Pronouncements Not Yet Adopted | Recent Accounting Pronouncements Not Yet Adopted From time to time, new accounting pronouncements are issued by the FASB or other standard setting bodies and adopted by the Company as of the specified effective date. Unless otherwise discussed, the Company believes that the impact of recently issued standards that are not yet effective will not have a material impact on the accompanying condensed consolidated financial statements and disclosures. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Accounting Policies [Abstract] | |
Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash Reported within the Consolidated Balance Sheets | The following table provides a reconciliation of cash, cash equivalents and restricted cash equivalents reported within the unaudited condensed consolidated balance sheets that together reflect the same amounts shown in the unaudited condensed consolidated statements of cash flows (in thousands): As of September 30, As of December 31, 2023 2022 Cash and cash equivalents $ 303,059 $ 28,003 Restricted cash equivalents — 100,000 Total cash, cash equivalents, and restricted cash equivalents $ 303,059 $ 128,003 |
Schedule of Finite Lived Intangible Assets | The components of the Company’s intangible assets were as follows (in thousands, except for weighted-average remaining amortization period): September 30, 2023 Gross Carrying Value Accumulated Amortization Net Carrying Amount Weighted-Average Remaining Amortization Period Developed technology $ 226,620 $ ( 5,487 ) $ 221,133 11.9 Commercial milestones 39,000 ( 2,765 ) 36,235 16.4 Assembled workforce 970 — 970 2.9 Total intangible assets $ 266,590 $ ( 8,252 ) $ 258,338 11.7 December 31, 2022 Gross Carrying Value Accumulated Amortization Net Carrying Amount Weighted-Average Remaining Amortization Period Commercial milestones $ 34,000 $ ( 1,333 ) $ 32,667 17.1 Developed technology 28,107 ( 1,820 ) 26,287 8.4 Total intangible assets $ 62,107 $ ( 3,153 ) $ 58,954 11.9 |
Schedule of Estimated Future Amortization Expense Associated with Intangible Assets | The following table summarizes the estimated future amortization expense associated with the Company’s intangible assets as of September 30, 2023 (in thousands): Amount 2023 (remaining three months) 5,413 2024 21,545 2025 21,545 2026 21,436 2027 21,221 Thereafter 167,178 $ 258,338 |
Schedule of Disaggregation of Total Product Sales | The following table disaggregates total Product sales, net: Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Product sales, net: Livmarli $ 38,708 $ 18,780 $ 100,303 $ 47,156 Bile Acid Medicines 9,017 — 9,017 — Total product sales, net 47,725 18,780 109,320 47,156 License revenue — — 7,500 2,000 Total revenues $ 47,725 $ 18,780 $ 116,820 $ 49,156 |
Schedule of Computation of Basic and Diluted Earnings per Share | The following table sets for the computation of basic and diluted earnings per share for the three and nine months ended September 30, 2022 (in thousands, except share and per share data): Three Months Ended Nine Months Ended 2022 2022 Numerator: Net loss, basic $ ( 35,706 ) $ ( 99,234 ) Less: Change in fair value of Holdback Indemnification liability — 149 Net loss, diluted $ ( 35,706 ) $ ( 99,383 ) Denominator: Weighted-average shares of common stock outstanding, basic 34,927,790 32,809,365 Effect of dilutive securities: Weighted-average Holdback Indemnification shares issuable — 15,949 Weighted-average shares of common stock outstanding, diluted 34,927,790 32,825,314 Net loss per share, basic $ ( 1.02 ) $ ( 3.03 ) Net loss per share, diluted $ ( 1.02 ) $ ( 3.03 ) |
Summary of Outstanding Potentially Dilutive Shares of Common Stock Excluded from Calculation of Diluted Net Loss per Share | The following outstanding potential dilutive shares have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: As of September 30, As of September 30, 2023 2022 Options to purchase common stock and restricted stock units 10,814,495 8,910,376 Common stock issuable upon conversion of convertible notes 9,964,247 — Employee stock purchase plan contingently issuable 49,868 46,760 Common stock subject to repurchase — 22,270 Shares issuable as contingent consideration in connection with asset acquisition — 199,993 Total 20,828,610 9,179,399 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Summary of Financial Assets and Liabilities to Fair Value Measurements On Recurring Basis and Level of Input Measurements | Financial assets and liabilities subject to fair value measurements on a recurring basis and the level of inputs used in such measurements by major security type are presented in the following table (in thousands): September 30, 2023 Level 1 Level 2 Level 3 Total Financial assets: Money market funds $ 297,316 $ — $ — 297,316 Commercial paper — 2,971 — 2,971 Total financial assets $ 297,316 $ 2,971 $ — $ 300,287 December 31, 2022 Level 1 Level 2 Level 3 Total Financial assets: Money market funds $ 124,227 $ — $ — $ 124,227 U.S. treasury bills 4,975 — — 4,975 Commercial paper — 74,386 — 74,386 U.S. government bonds — 44,354 — 44,354 Total financial assets $ 129,202 $ 118,740 $ — $ 247,942 Financial liabilities: Contingent milestone liability $ 3,900 $ — $ — $ 3,900 Derivative liability — — 1,090 1,090 Indemnification holdback — — 617 617 Total financial liabilities $ 3,900 $ — $ 1,707 $ 5,607 |
Summary of Changes in Fair Value classified as Level 3 | The following table provides a summary of the changes in the estimated fair value of the Indemnification Holdback liability (in thousands): Indemnification Holdback Liability Balance at December 31, 2022 $ 617 Change in fair value 279 Settlement of Indemnification Holdback liability ( 896 ) Balance at September 30, 2023 $ — |
Financial Instruments (Tables)
Financial Instruments (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Fair Value Disclosures [Abstract] | |
Summary of Fair Value and Amortized Cost of Cash Equivalents and Available-for-sale Investments by Major Security Type | The fair value and amortized cost of cash equivalents and available-for-sale investments by major security type are presented in the following table (in thousands): September 30, 2023 Amortized Unrealized Unrealized Estimated Cash equivalents and investments: Money market funds $ 297,316 $ — $ — $ 297,316 Commercial paper 2,971 — — 2,971 Total cash equivalents and investments $ 300,287 $ — $ — $ 300,287 Classified as: Cash equivalents $ 297,316 Short-term investments 2,971 Total cash equivalents and investments $ 300,287 December 31, 2022 Amortized Unrealized Unrealized Estimated Cash equivalents and investments: Money market funds $ 124,227 $ — $ — $ 124,227 U.S. treasury bills 4,980 — ( 5 ) 4,975 Commercial paper 74,386 — — 74,386 U.S. government bonds 44,579 — ( 225 ) 44,354 Total cash equivalents and investments $ 248,172 $ — $ ( 230 ) $ 247,942 Classified as: Cash equivalents $ 24,226 Cash equivalents - restricted 100,000 Short-term investments 123,716 Total cash equivalents, restricted cash equivalents and investments $ 247,942 |
Balance Sheet Components (Table
Balance Sheet Components (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Balance Sheet Components [Abstract] | |
Schedule of Inventory | Inventory consists of the following (in thousands): September 30, December 31, 2023 2022 Raw materials $ 444 $ — Work in progress 10,529 5,351 Finished goods 11,277 214 Total inventory $ 22,250 $ 5,565 |
Schedule of Accrued Expenses | Accrued expenses consist of the following (in thousands): September 30, December 31, 2023 2022 Accrued compensation and related benefits $ 13,610 $ 14,660 Accrued sales deductions 14,557 4,284 Accrued clinical trials 6,351 8,319 Accrued professional service fees 9,502 5,372 Accrued contract manufacturing and non-clinical costs 7,004 3,927 Accrued royalties payable 4,426 2,456 Accrued interest 5,763 — Accrued milestone payments 5,000 15,000 Total accrued expenses $ 66,213 $ 54,018 |
Revenue Interest Purchase Agr_2
Revenue Interest Purchase Agreement (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Summary of Revenue Interest Liability and Derivative Liability | The following table summarizes the revenue interest liability activity and the related derivative liability activity prior to settlement (in thousands): Revenue Interest Liability Derivative Liability Balance at December 31, 2022 $ 140,351 $ 1,090 Interest expense recognized 5,060 — Revenue interest payments ( 2,883 ) — Revenue interest liability settlement ( 192,694 ) — Loss (gain) from termination of revenue interest purchase agreement 50,166 ( 1,090 ) Balance at September 30, 2023 $ — $ — |
Asset Acquisitions (Tables)
Asset Acquisitions (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Bile Acid Medicines | |
Asset Acquisitions [Line Items] | |
Schedule of Consideration Paid and Allocation of Costs | The following represents the consideration paid and allocation of the purchase price for the acquisition of the Bile Acid Medicines (in thousands): Cash consideration $ 210,378 Transaction costs 2,384 Total purchase consideration 212,762 Assets acquired: Inventory 12,900 Intangible assets - developed technology 198,513 Intangible assets - assembled workforce 970 Other current and noncurrent assets 379 Total assets acquired $ 212,762 |
Satiogen | |
Asset Acquisitions [Line Items] | |
Schedule of Consideration Paid and Allocation of Costs | The following represents the consideration paid and allocation of purchase price for the acquisition of Satiogen (in thousands, except per share data): Issued common stock $ 15,585 Cash consideration 2,600 Indemnification Holdback 831 Contingent consideration settled in common stock 4,600 Transaction costs 545 Total purchase consideration 24,161 Assets acquired: Intangible assets - developed technology 21,561 Cash consideration 2,600 Total assets acquired $ 24,161 |
Leases (Tables)
Leases (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Leases [Abstract] | |
Schedule of Undiscounted Future Minimum Payments under Operating Leases | As of September 30, 2023, undiscounted future minimum payments under the Company’s operating leases are as follows (in thousands): Undiscounted 2023 (remaining three months) $ 298 2024 1,187 2025 355 2026 113 2027 113 Thereafter 56 Total undiscounted lease payments 2,122 Less: imputed interest ( 172 ) Total lease liability $ 1,950 |
Convertible Notes (Tables)
Convertible Notes (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Debt Disclosure [Abstract] | |
Schedule Of Convertible Notes | As of September 30, 2023, the Notes consisted of the following (in thousands): Convertible Notes Principal amount $ 316,250 Unamortized debt discount and issuance costs ( 10,228 ) Net carrying amount $ 306,022 |
Schedule of Interest Expense Related to Convertible Notes | The following table sets forth interest expense recognized related to the Notes (in thousands): Three Months Ended Nine Months Ended 2023 2023 Coupon interest expense $ 3,163 $ 5,764 Amortization of debt discount and issuance costs 411 718 Total interest expense on convertible notes $ 3,574 $ 6,482 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Equity [Abstract] | |
Schedule of Common Stock Reserved for Issuance | Common stock reserved for issuance is as follows: As of September 30, As of December 31, 2023 2022 Stock options, restricted stock units and performance stock units issued and outstanding 10,814,495 8,955,557 Reserved for future stock awards or option grants 2,412,737 1,596,947 Reserved for employee stock purchase plan 1,081,089 1,157,570 Common stock issuable upon conversion of convertible notes 9,964,247 — Common stock held back in connection with asset acquisition — 31,638 Common stock issuable as contingent consideration in connection with asset acquisition — 199,993 24,272,568 11,941,705 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 9 Months Ended |
Sep. 30, 2023 | |
Summary of Stock Option Activity | The following table summarizes stock option activity during the nine months ended September 30, 2023 (in thousands, except share and per share data): Number of Weighted- Weighted- Aggregate Outstanding as of December 31, 2022 8,340,083 $ 13.63 7.5 $ 51,645 Granted 1,925,589 $ 24.96 Exercised ( 437,959 ) $ 15.89 Canceled and forfeited ( 240,546 ) $ 18.40 Outstanding as of September 30, 2023 9,587,167 $ 15.68 7.1 $ 152,622 Vested and exercisable as of September 30, 2023 5,726,809 $ 11.85 6.1 $ 113,100 |
Schedule of Assumptions Used to Estimate Fair Value of Stock Option Awards Granted | The following assumptions were used to estimate the fair value of stock option awards granted during the following periods: Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Expected term (in years) 6.08 6.08 5.31 - 6.08 5.50 - 6.08 Expected volatility 80.99 % - 81.63 % 81.13 %- 81.70 % 80.99 % - 85.24 % 80.86 %- 83.20 % Risk-free interest rate 3.97 % - 4.39 % 2.65 %- 3.44 % 3.35 - 4.39 1.46 %- 3.44 % Expected dividend yield — — — — |
Summary of RSU Activity | The following table summarizes the activity under the Company’s restricted stock units for the nine months ended September 30, 2023: Number of Weighted-Average Grant Date Unvested and outstanding as of December 31, 2022 615,474 $ 18.36 Granted 754,324 $ 25.00 Vested ( 235,130 ) $ 18.36 Cancelled/Forfeited ( 55,175 ) $ 20.29 Unvested and outstanding as of September 30, 2023 1,079,493 $ 22.89 |
Summary of PSU Activity | The following table summarizes the activity under the Company's performance stock units for the nine months ended September 30, 2023: Number of Weighted-Average Grant Date Unvested and outstanding as of December 31, 2022 — $ — Granted 147,835 $ 23.64 Unvested and outstanding as of September 30, 2023 147,835 $ 23.64 |
Summary of Stock-based Compensation Reflected in Unaudited Condensed Consolidated Statements of Operations | Total stock-based compensation is reflected in the accompanying unaudited condensed consolidated statements of operations as follows (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2023 2022 2023 2022 Selling, general and administrative $ 5,759 $ 4,391 $ 17,290 $ 12,592 Research and development 2,626 2,517 8,013 7,519 Total $ 8,385 $ 6,908 $ 25,303 $ 20,111 |
Organization and Description _2
Organization and Description of Business - Additional Information (Details) $ / shares in Units, $ in Thousands | 1 Months Ended | 9 Months Ended | |||
Aug. 31, 2023 USD ($) $ / shares shares | Apr. 30, 2023 USD ($) | Sep. 30, 2023 USD ($) Segment $ / shares shares | Sep. 30, 2022 USD ($) | Dec. 31, 2022 USD ($) | |
Restructuring Cost and Reserve [Line Items] | |||||
Date of incorporation | May 02, 2018 | ||||
Number of operating segments | Segment | 1 | ||||
Accumulated deficit | $ 520,580 | $ 392,824 | |||
Cash, cash equivalents and investments | 306,000 | ||||
Unrestricted cash, cash equivalents and investments | 306,000 | ||||
Proceeds from issuance of shares | $ 305,300 | 14,480 | $ 21,289 | ||
Proceeds to repurchase future revenue interests | 192,700 | ||||
Restricted cash equivalents | $ 100,000 | 0 | $ 100,000 | ||
Bile Acid Portfolio Acquisition | |||||
Restructuring Cost and Reserve [Line Items] | |||||
Upfront payment | 210,400 | ||||
Milestone payment | $ 235,000 | ||||
Shares issued, price per share | $ / shares | $ 26.25 | $ 26.25 | |||
Net proceeds from transaction | $ 202,200 | $ 202,200 | |||
Bile Acid Portfolio Acquisition | Private Placement | |||||
Restructuring Cost and Reserve [Line Items] | |||||
Issuance of common stock, shares | shares | 8,000,000 | 8,000,000 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Summary of Reconciliation of Cash, Cash Equivalents and Restricted Cash Reported Within the Consolidated Balance Sheets (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Apr. 30, 2023 | Dec. 31, 2022 | Sep. 30, 2022 | Dec. 31, 2021 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |||||
Cash and cash equivalents | $ 303,059 | $ 28,003 | |||
Restricted cash equivalents | 0 | $ 100,000 | 100,000 | ||
Total cash, cash equivalents, and restricted cash equivalents | $ 303,059 | $ 128,003 | $ 135,759 | $ 131,340 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Schedule Of Finite Lived Intangible Assets (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Summary Of Significant Accounting Policies [Line Items] | ||
Gross carrying value | $ 266,590 | $ 62,107 |
Accumulated Amortization | (8,252) | (3,153) |
Net Carrying Value | $ 258,338 | $ 58,954 |
Weighted-Average Remaining Amortization Period (Years) | 11 years 8 months 12 days | 11 years 10 months 24 days |
Developed technology | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Gross carrying value | $ 226,620 | $ 28,107 |
Accumulated Amortization | (5,487) | (1,820) |
Net Carrying Value | $ 221,133 | $ 26,287 |
Weighted-Average Remaining Amortization Period (Years) | 11 years 10 months 24 days | 8 years 4 months 24 days |
Commercial milestones | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Gross carrying value | $ 39,000 | $ 34,000 |
Accumulated Amortization | (2,765) | (1,333) |
Net Carrying Value | $ 36,235 | $ 32,667 |
Weighted-Average Remaining Amortization Period (Years) | 16 years 4 months 24 days | 17 years 1 month 6 days |
Assembled workforce | ||
Summary Of Significant Accounting Policies [Line Items] | ||
Gross carrying value | $ 970 | |
Accumulated Amortization | 0 | |
Net Carrying Value | $ 970 | |
Weighted-Average Remaining Amortization Period (Years) | 2 years 10 months 24 days |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Schedule of Estimated Future Amortization Expense Associated with Intangible Assets (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | ||
2023 (remaining three months) | $ 5,413 | |
2024 | 21,545 | |
2025 | 21,545 | |
2026 | 21,436 | |
2027 | 21,221 | |
Thereafter | 167,178 | |
Net Carrying Value | $ 258,338 | $ 58,954 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Schedule of Disaggregation of Total Product Sales (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Summary Of Significant Accounting Policies [Line Items] | ||||
Revenue | $ 47,725 | $ 18,780 | $ 116,820 | $ 49,156 |
Product [Member] | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Revenue | 47,725 | 18,780 | 109,320 | 47,156 |
Livmarli | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Revenue | 38,708 | $ 18,780 | 100,303 | 47,156 |
Bile Acid Medicines | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Revenue | $ 9,017 | 9,017 | ||
License revenue | ||||
Summary Of Significant Accounting Policies [Line Items] | ||||
Revenue | $ 7,500 | $ 2,000 |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Schedule of Computation of Basic and Diluted Earnings per Share (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Numerator: | ||||
Net loss, basic | $ (35,706) | $ (99,234) | ||
Less: Change in fair value of Holdback Indemnification liability | 0 | 149 | ||
Net loss, diluted | $ (35,706) | $ (99,383) | ||
Denominator: | ||||
Weighted-average shares of common stock outstanding, basic | 41,098,920 | 34,927,790 | 38,973,060 | 32,809,365 |
Effect of dilutive securities: | ||||
Weighted-average Holdback Indemnification shares issuable | 0 | 15,949 | ||
Weighted-average shares of common stock outstanding, diluted | 41,098,920 | 34,927,790 | 38,973,060 | 32,825,314 |
Net loss per share, basic | $ (0.57) | $ (1.02) | $ (3.28) | $ (3.03) |
Net loss per share, diluted | $ (0.57) | $ (1.02) | $ (3.28) | $ (3.03) |
Summary of Significant Accoun_9
Summary of Significant Accounting Policies - Summary of Outstanding Potentially Dilutive Shares of Common Stock Excluded from Calculation of Diluted Net Loss Per Share (Details) - shares | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2022 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 20,828,610 | 9,179,399 |
Common stock issuable upon conversion of convertible notes | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 9,964,247 | 0 |
Options to Purchase Common Stock and Restricted Stock Units | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 10,814,495 | 8,910,376 |
Employee Stok Purchase Plan (ESPP) | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 49,868 | 46,760 |
Common Stock Subject to Repurchase | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 0 | 22,270 |
Shares Issuable as Contingent Consideration as Part of Asset Acquisition | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 0 | 199,993 |
Summary of Significant Accou_10
Summary of Significant Accounting Policies - Additional Information (Details) | 3 Months Ended | 9 Months Ended | |||||
Sep. 30, 2023 USD ($) Customer | Sep. 30, 2022 USD ($) Customer | Sep. 30, 2023 USD ($) Customer | Sep. 30, 2022 USD ($) Customer | Apr. 30, 2023 USD ($) | Jan. 01, 2023 | Dec. 31, 2022 USD ($) Customer | |
Summary Of Significant Accounting Policies [Line Items] | |||||||
Restricted cash equivalents | $ 0 | $ 0 | $ 100,000,000 | $ 100,000,000 | |||
Investments with original maturities at date of purchase to be cash equivalents | 3 months | ||||||
Fair value of its investments related to credit loss | $ 0 | ||||||
Allowance for credit losses | 0 | 0 | |||||
Provision for doubtful accounts | 0 | $ 0 | 0 | $ 0 | |||
Amortization expense | $ 2,600,000 | $ 1,000,000 | $ 5,100,000 | $ 1,800,000 | |||
Accounts Receivable | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Number of customer | Customer | 0 | 0 | 1 | ||||
Accounts Receivable | Customer Concentration Risk | Minimum | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 10% | ||||||
Accounts Receivable | Customer Concentration Risk | Maximum [Member] | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 23% | ||||||
Revenue from Contract with Customer Benchmark | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Number of customer | Customer | 1 | 1 | 1 | 1 | |||
Revenue from Contract with Customer Benchmark | Customer Concentration Risk | Minimum | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Concentration of credit risk percentage | 10% | 10% | 10% | 10% | |||
Accounting Standards Update 2018-18 | |||||||
Summary Of Significant Accounting Policies [Line Items] | |||||||
Change in accounting principle, accounting standards update, adopted [true false] | true | ||||||
Change in accounting principle, accounting standards update, adoption date | Jan. 01, 2023 | ||||||
Change in accounting principle, accounting standards update, immaterial effect [true false] | true |
Fair Value Measurements - Summa
Fair Value Measurements - Summary of Financial Assets and Liabilities to Fair Value Measurement On Recurring Basis and Level of Input Measurement (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Financial liabilities: | ||
Derivative liability | $ 0 | $ 1,090 |
Derivative Liability, Statement of Financial Position [Extensible Enumeration] | Derivative Liability, Current | |
Fair Value, Recurring Basis | ||
Financial assets: | ||
Fair value measurements | 300,287 | $ 247,942 |
Financial liabilities: | ||
Derivative liability | 1,090 | |
Total financial liabilities | 5,607 | |
Fair Value, Recurring Basis | Level 1 | ||
Financial assets: | ||
Fair value measurements | 297,316 | 129,202 |
Financial liabilities: | ||
Derivative liability | 0 | |
Total financial liabilities | 3,900 | |
Fair Value, Recurring Basis | Level 2 | ||
Financial assets: | ||
Fair value measurements | 2,971 | 118,740 |
Financial liabilities: | ||
Derivative liability | 0 | |
Total financial liabilities | 0 | |
Fair Value, Recurring Basis | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Financial liabilities: | ||
Derivative liability | 1,090 | |
Total financial liabilities | 1,707 | |
Indemnification Holdback Liability | Fair Value, Recurring Basis | ||
Financial liabilities: | ||
Total financial liabilities | 617 | |
Indemnification Holdback Liability | Fair Value, Recurring Basis | Level 1 | ||
Financial liabilities: | ||
Total financial liabilities | 0 | |
Indemnification Holdback Liability | Fair Value, Recurring Basis | Level 2 | ||
Financial liabilities: | ||
Total financial liabilities | 0 | |
Indemnification Holdback Liability | Fair Value, Recurring Basis | Level 3 | ||
Financial liabilities: | ||
Total financial liabilities | 617 | |
Contingent Milestone Liability | Fair Value, Recurring Basis | ||
Financial liabilities: | ||
Total financial liabilities | 3,900 | |
Contingent Milestone Liability | Fair Value, Recurring Basis | Level 1 | ||
Financial liabilities: | ||
Total financial liabilities | 3,900 | |
Contingent Milestone Liability | Fair Value, Recurring Basis | Level 2 | ||
Financial liabilities: | ||
Total financial liabilities | 0 | |
Contingent Milestone Liability | Fair Value, Recurring Basis | Level 3 | ||
Financial liabilities: | ||
Total financial liabilities | 0 | |
Money Market Funds | Fair Value, Recurring Basis | ||
Financial assets: | ||
Fair value measurements | 297,316 | 124,227 |
Money Market Funds | Fair Value, Recurring Basis | Level 1 | ||
Financial assets: | ||
Fair value measurements | 297,316 | 124,227 |
Money Market Funds | Fair Value, Recurring Basis | Level 2 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Money Market Funds | Fair Value, Recurring Basis | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
U.S. treasury bills | Fair Value, Recurring Basis | ||
Financial assets: | ||
Fair value measurements | 4,975 | |
U.S. treasury bills | Fair Value, Recurring Basis | Level 1 | ||
Financial assets: | ||
Fair value measurements | 4,975 | |
U.S. treasury bills | Fair Value, Recurring Basis | Level 2 | ||
Financial assets: | ||
Fair value measurements | 0 | |
U.S. treasury bills | Fair Value, Recurring Basis | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | |
Commercial Paper | Fair Value, Recurring Basis | ||
Financial assets: | ||
Fair value measurements | 2,971 | 74,386 |
Commercial Paper | Fair Value, Recurring Basis | Level 1 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Commercial Paper | Fair Value, Recurring Basis | Level 2 | ||
Financial assets: | ||
Fair value measurements | 2,971 | 74,386 |
Commercial Paper | Fair Value, Recurring Basis | Level 3 | ||
Financial assets: | ||
Fair value measurements | $ 0 | 0 |
U.S. Government Bonds | Fair Value, Recurring Basis | ||
Financial assets: | ||
Fair value measurements | 44,354 | |
U.S. Government Bonds | Fair Value, Recurring Basis | Level 1 | ||
Financial assets: | ||
Fair value measurements | 0 | |
U.S. Government Bonds | Fair Value, Recurring Basis | Level 2 | ||
Financial assets: | ||
Fair value measurements | 44,354 | |
U.S. Government Bonds | Fair Value, Recurring Basis | Level 3 | ||
Financial assets: | ||
Fair value measurements | $ 0 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2023 shares | Jun. 30, 2023 shares | Sep. 30, 2023 USD ($) | Dec. 31, 2022 | |
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||||
Issuance of common stock in connection with settlement of Indemnification Holdback liability, Shares | 31,631 | |||
Indemnification Holdback Liability [Member] | ||||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||||
Indemnification Holdback liability | $ | $ 0 | |||
Common Stock [Member] | ||||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||||
Issuance of common stock in connection with settlement of Indemnification Holdback liability, Shares | 31,631 | |||
Discount Rate | ||||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||||
Discount rate used for valuation to derivative liability | 0.157 |
Fair Value Measurements - Sum_2
Fair Value Measurements - Summary of Changes in Fair Value of Indemnification Holdback (Details) - Indemnification Holdback Liability $ in Thousands | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
Fair Value, Liabilities Measured on Recurring Basis, Unobservable Input Reconciliation [Line Items] | |
Beginning Balance | $ 617 |
Change in fair value | 279 |
Settlement of Indemnification Holdback liability | (896) |
Ending Balance | $ 0 |
Financial Instruments - Summary
Financial Instruments - Summary of Fair Value and Amortized Cost of Cash Equivalents and Available-for-sale Investments by Major Security Type (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Cash equivalents and investments, Amortized cost | $ 300,287 | $ 248,172 |
Cash equivalents and investments, Unrealized gain | 0 | 0 |
Cash equivalents and investments, Unrealized loss | 0 | (230) |
Cash equivalents, Estimated Fair Value | 297,316 | 24,226 |
Restricted Cash and Cash Equivalents | 100,000 | |
Short-term investments | 2,971 | 123,716 |
Total cash equivalents and investments | 300,287 | 247,942 |
Money Market Funds | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Cash equivalents, Amortized Cost | 297,316 | 124,227 |
Cash equivalents, Estimated Fair Value | 297,316 | 124,227 |
U.S. treasury bills | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 4,980 | |
Investments, Unrealized Gain | 0 | |
Investments, Unrealized Loss | (5) | |
Investments, Estimated Fair Value | 4,975 | |
U.S. Government Bonds | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 44,579 | |
Investments, Unrealized Gain | 0 | |
Investments, Unrealized Loss | (225) | |
Investments, Estimated Fair Value | 44,354 | |
Commercial Paper | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 2,971 | 74,386 |
Investments, Unrealized Gain | 0 | 0 |
Investments, Unrealized Loss | 0 | 0 |
Investments, Estimated Fair Value | $ 2,971 | $ 74,386 |
Financial Instruments - Additio
Financial Instruments - Additional Information (Details) - USD ($) | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Financial Instruments, Owned, at Fair Value, by Type, Alternative [Abstract] | ||||
Realized gains or losses on available-for-sale investments | $ 0 | $ 0 | $ 0 | $ 0 |
Investments in continuous unrealized loss position for more than 12 months | $ 0 | $ 0 | 0 | 0 |
Other-than-temporary impairment losses | $ 0 | $ 0 |
Balance Sheet Components - Sche
Balance Sheet Components - Schedule of Inventory (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Inventory Disclosure [Abstract] | ||
Raw materials | $ 444 | $ 0 |
Work in progress | 10,529 | 5,351 |
Finished goods | 11,277 | 214 |
Total inventory | $ 22,250 | $ 5,565 |
Balance Sheet Components - Sc_2
Balance Sheet Components - Schedule of Accrued Expenses (Details) - USD ($) $ in Thousands | Sep. 30, 2023 | Dec. 31, 2022 |
Payables and Accruals [Abstract] | ||
Accrued compensation and related benefits | $ 13,610 | $ 14,660 |
Accrued sales deductions | 14,557 | 4,284 |
Accrued clinical trials | 6,351 | 8,319 |
Accrued professional service fees | 9,502 | 5,372 |
Accrued contract manufacturing and non-clinical costs | 7,004 | 3,927 |
Accrued royalties payable | 4,426 | 2,456 |
Accrued interest | 5,763 | 0 |
Accrued milestone payments | 5,000 | 15,000 |
Total accrued expenses | $ 66,213 | $ 54,018 |
Revenue Interest Purchase Agr_3
Revenue Interest Purchase Agreement - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||||
Apr. 30, 2021 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2020 | Apr. 30, 2023 | Dec. 31, 2022 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenue interest liability | $ 0 | $ 0 | $ 140,351 | |||||
Loss from termination of revenue interest purchase agreement | 0 | $ 0 | 49,076 | $ 0 | ||||
Revenue interest liability, net | 0 | 0 | 140,351 | |||||
Derivative liability | 0 | 0 | $ 1,090 | |||||
Sales | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Revenues | $ 2,900 | |||||||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Percentage of revenue interest payments on annual net sales at Tier 1 | 9.75% | |||||||
Percentage of revenue interest payments on annual net sales at Tier 2 and Tier 3 | 2% | |||||||
Required revenue interest payment percentage to cumulative purchaser payments to reduce interest rate | 175% | |||||||
Required repurchase price percentage of cumulative purchaser prior to first anniversary of closing date | 120% | |||||||
Required repurchase price percentage of cumulative purchaser payments after first anniversary and prior to third anniversary of closing date | 175% | |||||||
Required repurchase price percentage of cumulative purchaser after third anniversary of closing date | 195% | |||||||
Purchase agreement amount allocated to debt | $ 49,200 | |||||||
Initial fair value of derivative liability | 1,300 | 1,300 | ||||||
Debt issuance costs | 900 | 900 | ||||||
Interest expense | $ 0 | $ 4,000 | 5,100 | $ 11,600 | ||||
Payment on repurchase in connection with RIPA | $ 192,700 | |||||||
Loss from termination of revenue interest purchase agreement | 49,100 | |||||||
Loss related to settlement of revenue interest liability | 50,200 | |||||||
Gain on derecognition of related derivative liability | 1,100 | |||||||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | Maximum [Member] | On or Prior to December 31, 2022 | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Potential milestone payment to be received at the option of purchasers | 50,000 | |||||||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | Commercialization and Development of Product and Other Working Capital Needs | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Upfront payment received | 50,000 | |||||||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | Livmarli | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Milestone and upfront payments received | 115,000 | |||||||
Upfront payment received | $ 50,000 | |||||||
Milestone payment received | $ 65,000 | |||||||
Potential milestone payment received upon regulatory approval | 35,000 | |||||||
Tier I | Mulholland SA LLC, | Livmarli | Maximum [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Annual net sales | 350,000 | |||||||
Tier 2 | Mulholland SA LLC, | Livmarli | Minimum | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Annual net sales | 350,000 | |||||||
Tier 2 | Mulholland SA LLC, | Livmarli | Maximum [Member] | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Annual net sales | 1,100,000 | |||||||
Tier 3 | Mulholland SA LLC, | Livmarli | Minimum | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Annual net sales | $ 1,100,000 | |||||||
CSPA | Mulholland SA LLC, | ||||||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | ||||||||
Number of shares sold | 509,164 | |||||||
Aggregate purchase price | $ 10,000 | |||||||
Net proceeds from transaction | 10,000 | |||||||
Purchase agreement amount allocated to common stock issued | $ 10,800 |
Revenue Interest Purchase Agr_4
Revenue Interest Purchase Agreement - Summary of Revenue Interest Liability and Derivative Liability (Details) $ in Thousands | 9 Months Ended |
Sep. 30, 2023 USD ($) | |
Interest Expense, Interest-Bearing Liability [Abstract] | |
Revenue interest liability | $ 140,351 |
Interest expense recognized | 5,060 |
Revenue interest liability settlement | (192,694) |
Revenue interest payments | (2,883) |
Loss (gain) from termination of revenue interest purchase agreement | 50,166 |
Revenue interest liability | 0 |
Derivative Liability [Abstract] | |
Derivative liability | 1,090 |
Interest expense recognized | 0 |
Revenue interest payments | 0 |
Derivative Liability Settlement | 0 |
Loss (gain) from termination of revenue interest purchase agreement | (1,090) |
Derivative liabilities | $ 0 |
Asset Acquisitions - Additional
Asset Acquisitions - Additional Information (Details) | 1 Months Ended | 6 Months Ended | 9 Months Ended | ||||||
Aug. 31, 2023 USD ($) | Aug. 31, 2023 USD ($) | Jan. 31, 2023 shares | May 31, 2022 USD ($) shares | Nov. 30, 2018 USD ($) ProductCandidate | Jun. 30, 2023 shares | Sep. 30, 2023 USD ($) | Sep. 30, 2022 USD ($) | Jan. 01, 2023 USD ($) | |
Asset Acquisitions [Line Items] | |||||||||
Issuance of common stock in connection with settlement of Indemnification Holdback liability, Shares | shares | 31,631 | ||||||||
Satiogen | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Business Combination, Consideration Transferred | $ 24,161,000 | ||||||||
Business Acquisition, Equity Interest Issued or Issuable, Number of Shares | shares | 609,305 | ||||||||
Business Acquisition, Equity Interest Issued or Issuable, Number of Additional Shares | shares | 32,494 | ||||||||
Stock option exercise price | $ 200,000 | ||||||||
Business Acquisition Future Issuance Of Additional Shares | shares | 199,993 | 199,993 | |||||||
Cash consideration | $ 2,600,000 | ||||||||
Bile Acid Medicines | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Business Combination, Consideration Transferred | $ 212,762,000 | ||||||||
Cash consideration | 210,378,000 | ||||||||
Shire | Livmarli | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Development or Regulatory Milestones Incurred | $ 0 | $ 0 | |||||||
Shire Agreement | Livmarli | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Milestones accrued | $ 15,000,000 | ||||||||
License agreement milestone amount accrued | 5,000,000 | ||||||||
Shire Agreement | Shire | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Number of product candidates | ProductCandidate | 2 | ||||||||
Product sales milestone payments, payable | $ 30,000,000 | ||||||||
Shire Agreement | Shire | Volixibat | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Milestone payments, payable upon commercialization | 30,000,000 | ||||||||
Shire Agreement | Shire | Livmarli | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Milestone payments, payable | 109,500,000 | ||||||||
Milestone payments, payable upon approval | 25,000,000 | ||||||||
Assigned License Agreement | Satiogen Pharmaceuticals, Inc. | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Milestone payments, payable | 10,500,000 | ||||||||
Milestone payments, payable upon approval | 5,000,000 | ||||||||
Milestone payments, payable upon initiation | 500,000 | ||||||||
Milestone payments, payable upon commercialization | 5,000,000 | ||||||||
Assigned License Agreement | Sanofi-Aventis Deutschland GmbH | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Milestone payments, payable | $ 36,000,000 | ||||||||
Royalty obligations payment period | 10 years | ||||||||
Milestones accrued | $ 0 | ||||||||
Asset Purchase Agreement | Travere Therapeutics, Inc. | Bile Acid Medicines | |||||||||
Asset Acquisitions [Line Items] | |||||||||
Product sales milestone payments, payable | $ 235,000,000 | $ 235,000,000 | |||||||
Cash consideration | $ 210,400,000 |
Asset Acquisitions - Schedule o
Asset Acquisitions - Schedule of Consideration Paid and Allocation of Costs (Details) - USD ($) $ in Thousands | 1 Months Ended | |||
Aug. 31, 2023 | May 31, 2022 | Sep. 30, 2023 | Dec. 31, 2022 | |
Business Acquisition [Line Items] | ||||
Indemnification Holdback | $ 7 | $ 4,532 | ||
Satiogen | ||||
Business Acquisition [Line Items] | ||||
Issued common stock | $ 15,585 | |||
Cash consideration | 2,600 | |||
Indemnification Holdback | 831 | |||
Contingent consideration settled in common stock | 4,600 | |||
Transaction costs | 545 | |||
Total purchase consideration | 24,161 | |||
Assets acquired: | ||||
Cash consideration | 2,600 | |||
Total assets acquired | 24,161 | |||
Satiogen | Developed technology | ||||
Assets acquired: | ||||
Intangible assets | $ 21,561 | |||
Bile Acid Medicines | ||||
Business Acquisition [Line Items] | ||||
Cash consideration | $ 210,378 | |||
Transaction costs | 2,384 | |||
Total purchase consideration | 212,762 | |||
Assets acquired: | ||||
Inventory | 12,900 | |||
Cash consideration | 210,378 | |||
Other current and noncurrent assets | 379 | |||
Total assets acquired | 212,762 | |||
Bile Acid Medicines | Developed technology | ||||
Assets acquired: | ||||
Intangible assets | 198,513 | |||
Bile Acid Medicines | Assembled workforce | ||||
Assets acquired: | ||||
Intangible assets | $ 970 |
Collaboration and License Agr_2
Collaboration and License Agreements (Additional Information) (Details) - Exclusive Licensing Agreement - Livmarli - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended | 9 Months Ended | ||||
Jul. 31, 2021 | Apr. 30, 2021 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
CANbridge | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Upfront payment received | $ 11 | ||||||
Research and development funding received | $ 0.8 | $ 0.2 | $ 2.2 | $ 0.9 | |||
Achievement of future regulatory and commercial milestones payment | 5 | $ 2 | |||||
CANbridge | Prepaid Expenses and Other Current Assets [Member] | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Other research and development receivable | 2.4 | 2.4 | $ 0.2 | ||||
CANbridge | Maximum [Member] | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Potential regulatory and commercial milestone payment to be received | 109 | ||||||
Research and development funding received | $ 5 | ||||||
GC Biopharma | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Upfront payment received | $ 5 | ||||||
Research and development funding received | 0.1 | 0.3 | |||||
Achievement of future regulatory and commercial milestones payment | 2.5 | ||||||
GC Biopharma | Prepaid Expenses and Other Current Assets [Member] | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Other research and development receivable | $ 0.3 | $ 0.3 | |||||
GC Biopharma | Maximum [Member] | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Achievement of future regulatory and commercial milestones payment | $ 23 |
Leases - Additional Information
Leases - Additional Information (Detail) $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | |||||
Nov. 30, 2019 USD ($) ft² | Sep. 30, 2023 USD ($) | Sep. 30, 2022 USD ($) | Sep. 30, 2023 USD ($) | Sep. 30, 2022 USD ($) | Dec. 31, 2022 USD ($) | Jan. 31, 2019 USD ($) | Jan. 22, 2019 ft² | |
Lessee Lease Description [Line Items] | ||||||||
Area of office space | ft² | 5,600 | |||||||
Term of lease | 4 years | |||||||
Operating lease, option to extend, description | The lease term is approximately four years with an option to extend the term for one five-year term, which at the time was not reasonably assured of exercise and therefore, not included in the lease term. | |||||||
Existence of option to extend | true | |||||||
Term of extension of lease | 5 years | |||||||
Tenant improvement allowance | $ 400 | |||||||
Operating lease right-of-use assets | $ 1,428 | $ 1,428 | $ 1,431 | |||||
Lease liability | $ 1,950 | $ 1,950 | ||||||
Weighted-average incremental borrowing rate | 8% | 8% | ||||||
Weighted-average remaining lease term | 2 years 2 months 12 days | 2 years 2 months 12 days | ||||||
Rent expense | $ 200 | $ 600 | $ 500 | $ 500 | ||||
Amended Operating Lease Agreement | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Term of lease | 5 years | |||||||
Additional area of office space | ft² | 5,555 | |||||||
Lease expiration, month and year | 2025-03 | |||||||
Amended Operating Lease Agreement | Property, Plant and Equipment | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Tenant improvement allowance | $ 800 | |||||||
Amended Operating Lease Agreement | Restatement Adjustment | ||||||||
Lessee Lease Description [Line Items] | ||||||||
Operating lease right-of-use assets | 600 | |||||||
Lease liability | $ 600 |
Leases - Schedule of Undiscount
Leases - Schedule of Undiscounted Future Minimum Payments under Operating Leases (Detail) $ in Thousands | Sep. 30, 2023 USD ($) |
Leases [Abstract] | |
2023 (remaining three months) | $ 298 |
2024 | 1,187 |
2025 | 355 |
2026 | 113 |
2027 | 113 |
Thereafter | 56 |
Total undiscounted lease payments | 2,122 |
Less: imputed interest | (172) |
Total lease liability | $ 1,950 |
Convertible Notes - Additional
Convertible Notes - Additional Information (Detail) - Senior Notes Due At Two ThousandTwenty Nine [Member] $ / shares in Units, $ in Thousands | 1 Months Ended | 6 Months Ended | ||||
Apr. 30, 2023 USD ($) $ / shares | Apr. 30, 2023 USD ($) $ / shares | Apr. 30, 2023 USD ($) Days $ / shares | Apr. 30, 2023 USD ($) Tradingday $ / shares | Jun. 30, 2023 Days | Sep. 30, 2023 USD ($) | |
Debt Instrument [Line Items] | ||||||
Principle amount | $ 316,300 | $ 316,300 | $ 316,300 | $ 316,300 | $ 316,250 | |
Consecutive trading day | 30 | 10 | 30 | |||
Number of consecutive trading days before five business days during the measurement period | Tradingday | 10 | |||||
Trading days | Days | 20 | 20 | ||||
Net proceeds from debt issuance | $ 305,300 | |||||
Interest rate (percent) | 4% | 4% | 4% | 4% | ||
Consecutive trading days immediately after measurement period | Tradingday | 5 | |||||
Initial conversion price/rate | $ / shares | $ 31.5075 | $ 31.5075 | $ 31.5075 | $ 31.5075 | ||
Debt instrument conversion ratio multiple of principal | $ 1,000 | |||||
Common stock exceeds | 130% | 130% | ||||
Principal amount redeemable | $ 75,000 | $ 75,000 | $ 75,000 | $ 75,000 | ||
Percentage of principal amount of notes declared as accrued based on certain customary events of default | 25% | |||||
Debt instrument remaining life | 5 years 7 months 6 days | |||||
Effective interest rate (as a percent) | 4.60% | |||||
Convertible debt, fair value | $ 402,500 | |||||
Debt issuance costs | $ 10,900 | |||||
Common Stock [Member] | ||||||
Debt Instrument [Line Items] | ||||||
Initial conversion price/rate | $ / shares | $ 31.74 | $ 31.74 | $ 31.74 | $ 31.74 | ||
Maximum [Member] | ||||||
Debt Instrument [Line Items] | ||||||
Conversion premium percentage on sale price of common stock | 98% |
Convertible Notes - Schedule Of
Convertible Notes - Schedule Of Convertible Notes (Details) - Senior Notes Due At Two ThousandTwenty Nine [Member] - USD ($) $ in Thousands | Sep. 30, 2023 | Apr. 30, 2023 |
Debt Instrument [Line Items] | ||
Principle amount | $ 316,250 | $ 316,300 |
Unamortized debt discount and issuance costs | 10,228 | |
Net carrying amount | $ 306,022 |
Convertible Notes - Schedule _2
Convertible Notes - Schedule of Interest Expense Related to Convertible Notes (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | |
Sep. 30, 2023 | Sep. 30, 2023 | Sep. 30, 2022 | |
Debt Instrument [Line Items] | |||
Amortization of debt discount and issuance costs | $ 718 | $ 0 | |
Senior Notes Due At Two ThousandTwenty Nine [Member] | |||
Debt Instrument [Line Items] | |||
Coupon interest expense | $ 3,163 | 5,764 | |
Amortization of debt discount and issuance costs | 411 | 718 | |
Total interest expense on convertible notes | $ 3,574 | $ 6,482 |
Stockholders' Equity - Addition
Stockholders' Equity - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | |||||
Sep. 30, 2023 | Aug. 31, 2023 | Apr. 30, 2023 | Aug. 31, 2022 | Sep. 30, 2023 | Sep. 30, 2023 | Sep. 30, 2022 | Aug. 31, 2020 | |
Class Of Stock [Line Items] | ||||||||
Proceeds from issuance of shares | $ 305,300 | $ 14,480 | $ 21,289 | |||||
Maximum amount of sale covered in shelf registration statement | $ 300,000 | |||||||
Bile Acid Portfolio Acquisition | ||||||||
Class Of Stock [Line Items] | ||||||||
Shares issued, public offering price per share | $ 26.25 | $ 26.25 | $ 26.25 | $ 26.25 | ||||
Net proceeds from transaction | $ 202,200 | $ 202,200 | ||||||
Leerink Partners LLC [Member] | ||||||||
Class Of Stock [Line Items] | ||||||||
Maximum amount of offering issuance and sale covered in sales agreement | $ 75,000 | |||||||
Common Stock [Member] | Sales Agreement [Member] | ||||||||
Class Of Stock [Line Items] | ||||||||
Number of shares issued | 2,125,090 | 3,478,261 | 658,206 | |||||
Shares issued, public offering price per share | $ 23 | |||||||
Proceeds from issuance of shares | $ 14,500 | |||||||
Proceeds From Issuance Of Common Stock Gross | $ 43,700 | $ 15,000 | ||||||
Underwritten Public Offerings [Member] | Common Stock [Member] | Sales Agreement [Member] | Maximum [Member] | ||||||||
Class Of Stock [Line Items] | ||||||||
Number of shares issued | 521,739 | |||||||
Public Offering [Member] | ||||||||
Class Of Stock [Line Items] | ||||||||
Proceeds from issuance of shares | $ 86,100 | |||||||
Private Placement | Bile Acid Portfolio Acquisition | ||||||||
Class Of Stock [Line Items] | ||||||||
Number of shares issued | 8,000,000 | 8,000,000 | ||||||
Private Placement | Common Stock [Member] | ||||||||
Class Of Stock [Line Items] | ||||||||
Number of shares issued | 8,000,000 |
Stockholders' Equity - Schedule
Stockholders' Equity - Schedule of Common Stock Reserved for Issuance (Details) - shares | Sep. 30, 2023 | Dec. 31, 2022 |
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 24,272,568 | 11,941,705 |
Common stock issuable upon conversion of convertible notes | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 9,964,247 | 0 |
Stock options, restricted stock units and performance stock units issued and outstanding | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 10,814,495 | 8,955,557 |
Reserved for Future Stock Awards or Option Grants | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 2,412,737 | 1,596,947 |
Reserved for Employee Stock Purchase Plan | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 1,081,089 | 1,157,570 |
Common stock held back in connection with asset acquisition | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 0 | 31,638 |
Common stock issuable as contingent consideration in connection with asset acquisition | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 0 | 199,993 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Details) - USD ($) $ / shares in Units, $ in Thousands | 1 Months Ended | 3 Months Ended | 9 Months Ended | 12 Months Ended | |||||||||
Jun. 30, 2023 | Jan. 31, 2023 | Jul. 31, 2019 | Nov. 30, 2018 | Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2023 | Dec. 31, 2022 | Dec. 31, 2021 | Dec. 31, 2020 | Mar. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Number of common stock approved and reserved for issuance | 24,272,568 | 24,272,568 | 11,941,705 | ||||||||||
Stock-based compensation expense | $ 8,385 | $ 6,908 | $ 25,303 | $ 20,111 | |||||||||
Share-based compensation expenses capitalized amount | 200 | $ 200 | 600 | 300 | |||||||||
Stock Options | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation, Intrinsic value of options exercised | 4,700 | $ 2,600 | |||||||||||
Total unrecognized stock-based compensation related to unvested stock option awards granted | $ 52,300 | $ 52,300 | |||||||||||
Unrecognized stock-based compensation related to unvested stock, expected to recognize over weighted-average period | 2 years 7 months 6 days | ||||||||||||
Expected dividend yield | 0% | 0% | 0% | 0% | 0% | ||||||||
Weighted-average grant-date fair value | $ 17.96 | $ 12.71 | |||||||||||
2023 Executive PSUs | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Unrecognized stock-based compensation related to unvested stock, expected to recognize over weighted-average period | 1 year 8 months 12 days | ||||||||||||
Granted | 12,000 | 135,835 | |||||||||||
Total unrecognized stock-based compensation | $ 1,300 | $ 1,300 | |||||||||||
Award Vesting Rights Percentage | 50% | ||||||||||||
RSUs | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Total unrecognized stock-based compensation related to unvested stock option awards granted | $ 20,000 | $ 20,000 | |||||||||||
Unrecognized stock-based compensation related to unvested stock, expected to recognize over weighted-average period | 2 years 3 months 18 days | ||||||||||||
Restricted Common Stock | Founder | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Vested | 33,398 | 100,194 | |||||||||||
Stock issued for services | 562,500 | ||||||||||||
2019 Equity Incentive Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Equity incentive plans, description | Shares subject to outstanding awards under the 2018 Plan as of the effective date of the 2019 Plan that are subsequently canceled, forfeited or repurchased by the Company will be added to the shares reserved under the 2019 Plan. In addition, the number of shares of common stock available for issuance under the 2019 Plan will be automatically increased on the first day of each calendar year during the ten-year term of the 2019 Plan, beginning with January 1, 2020 and ending with January 1, 2029, by an amount equal to 5% of the outstanding number of shares of the Company’s common stock on December 31st of the preceding calendar year or such lesser amount as determined by the Company’s board of directors. As of September 30, 2023, 1,110,837 shares of common stock were available for issuance under the 2019 Plan. | ||||||||||||
Shares of common stock expiration term | 10 years | ||||||||||||
Shares of common stock beginning date | Jan. 01, 2020 | ||||||||||||
Shares of common stock ending date | Jan. 01, 2029 | ||||||||||||
Percentage of annual increase in common stock available for issuance | 5% | ||||||||||||
Number of common stock for future issuance | 1,110,837 | 1,110,837 | |||||||||||
2020 Inducement Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Number of common stock for future issuance | 1,301,900 | 1,301,900 | 750,000 | ||||||||||
Number of additional common stock for future issuance | 1,000,000 | 750,000 | |||||||||||
2020 Inducement Plan | Forecast | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Number of additional common stock for future issuance | 1,500,000 | ||||||||||||
2019 Employee Stock Purchase Plan | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Equity incentive plans, description | A total of 500,000 shares of common stock were approved to be initially reserved for issuance under the ESPP. In addition, the number of shares of common stock available for issuance under the ESPP will be automatically increased on the first day of each calendar year during the first ten years of the term of the ESPP, beginning with January 1, 2020 and ending with January 1, 2029, by an amount equal to the lesser of (i) 1% of the outstanding number of shares of common stock on December 31st of the preceding calendar year, (ii) 1,500,000 shares of common stock or (iii) such lesser amount as determined by the Company’s board of directors. The ESPP became effective on July 17, 2019 and generally provides for six-month consecutive offering periods beginning on May 11th and November 11th of each year. During the three months ended September 30, 2023, no shares were issued under the ESPP. During the nine months ended September 30, 2023, 76,481 shares were issued under the ESPP. As of September 30, 2023, the Company had 1,081,089 shares available for future issuance under the ESPP. | ||||||||||||
Shares of common stock expiration term | 10 years | ||||||||||||
Shares of common stock beginning date | Jan. 01, 2020 | ||||||||||||
Shares of common stock ending date | Jan. 01, 2029 | ||||||||||||
Percentage of annual increase in common stock available for issuance | 1% | ||||||||||||
Number of common stock approved and reserved for issuance | 1,081,089 | 1,081,089 | |||||||||||
Stock issued for services | 500,000 | 76,481 | |||||||||||
Annual increase in common stock available for issuance, shares | 1,500,000 | ||||||||||||
2019 Employee Stock Purchase Plan | Executive Performance Stock Units | |||||||||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||||||||
Stock-based compensation expense | $ 200 | $ 100 | $ 700 | $ 500 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Stock Option Activity (Details) - Stock Options - USD ($) $ / shares in Units, $ in Thousands | 9 Months Ended | 12 Months Ended |
Sep. 30, 2023 | Dec. 31, 2022 | |
Options, Outstanding | ||
Unvested and outstanding as of December 31, 2022 | 8,340,083 | |
Number of shares, Granted | 1,925,589 | |
Number of shares, Exercised | (437,959) | |
Number of shares, Canceled and forfeited | (240,546) | |
Unvested and outstanding as of September 30, 2023 | 9,587,167 | 8,340,083 |
Number of shares, Vested and exercisable | 5,726,809 | |
Weighted-average exercise price, Outstanding | ||
Weighted-average exercise price, Outstanding, Beginning balance | $ 13.63 | |
Weighted-average exercise price, Granted | 24.96 | |
Weighted-average exercise price, Exercised | 15.89 | |
Weighted-average exercise price, Canceled and forfeited | 18.4 | |
Weighted-average exercise price, Outstanding, Ending balance | 15.68 | $ 13.63 |
Weighted-average exercise price, Vested and exercisable | $ 11.85 | |
Share-based Payment Award, Options, Additional Disclosures | ||
Weighted-average remaining contractual life, Outstanding | 7 years 1 month 6 days | 7 years 6 months |
Weighted-average remaining contractual life, Vested and exercisable | 6 years 1 month 6 days | |
Aggregate intrinsic value, Outstanding | $ 152,622 | $ 51,645 |
Aggregate intrinsic value, Vested and exercisable | $ 113,100 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Assumptions Used to Estimate Fair Value of Stock Option Awards Granted (Details) - Stock Options | 3 Months Ended | 9 Months Ended | 12 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | Dec. 31, 2022 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Expected volatility, minimum | 80.99% | 81.13% | 80.99% | 80.86% | |
Expected volatility, maximum | 81.63% | 81.70% | 85.24% | 83.20% | |
Risk-free interest rate, minimum | 3.97% | 2.65% | 3.35% | 1.46% | |
Risk-free interest rate, maximum | 4.39% | 3.44% | 4.39% | 3.44% | |
Expected dividend yield | 0% | 0% | 0% | 0% | 0% |
Minimum | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Expected term (in years) | 6 years 29 days | 6 years 29 days | 5 years 3 months 21 days | 5 years 6 months | |
Maximum [Member] | |||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||
Expected term (in years) | 6 years 29 days | 6 years 29 days |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of RSU Activity (Details) - RSUs - $ / shares | 9 Months Ended | |
Sep. 30, 2023 | Dec. 31, 2022 | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | ||
Unvested and Outstanding as of December 31, 2022 | 1,079,493 | 615,474 |
Granted | 754,324 | |
Vested | (235,130) | |
Number of shares, Canceled and forfeited | (55,175) | |
Unvested and outstanding as of June 30, 2023 | 1,079,493 | 615,474 |
Weighted-average exercise price, Granted | $ 25 | |
Weighted-average exercise price, Vested | 18.36 | |
Weighted-average exercise price, Canceled and forfeited | 20.29 | |
Weighted-average exercise price, Vested and exercisable | $ 22.89 | $ 18.36 |
Stock-Based Compensation - Su_3
Stock-Based Compensation - Summary of PSU Activity (Details) - Performance Shares [Member] | 9 Months Ended |
Sep. 30, 2023 $ / shares shares | |
Share-Based Compensation Arrangement by Share-Based Payment Award [Line Items] | |
Unvested and outstanding as of December 31, 2022 | 0 |
Granted | 147,835 |
Unvested and outstanding as of September 30, 2023 | 147,835 |
Weighted-average exercise price, Granted | $ / shares | $ 23.64 |
Weighted-average exercise price, Vested and exercisable | $ / shares | $ 23.64 |
Stock-Based Compensation - Su_4
Stock-Based Compensation - Summary of Stock-based Compensation Reflected in Unaudited Condensed Consolidated Statements of Operations (Details) - USD ($) $ in Thousands | 3 Months Ended | 9 Months Ended | ||
Sep. 30, 2023 | Sep. 30, 2022 | Sep. 30, 2023 | Sep. 30, 2022 | |
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||||
Stock-based compensation expense | $ 8,385 | $ 6,908 | $ 25,303 | $ 20,111 |
Selling, General and Administrative | ||||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||||
Stock-based compensation expense | 5,759 | 4,391 | 17,290 | 12,592 |
Research and Development | ||||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||||
Stock-based compensation expense | $ 2,626 | $ 2,517 | $ 8,013 | $ 7,519 |
Subsequent Events - Additional
Subsequent Events - Additional Information (Details) - Subsequent Event - Sales Agreement [Member] $ in Millions | Nov. 02, 2023 USD ($) |
Business Acquisition [Line Items] | |
Net proceeds from transaction | $ 200 |
Percentage of gross proceeds | 3% |