Document And Entity Information
Document And Entity Information - shares | 3 Months Ended | |
Mar. 31, 2021 | Apr. 30, 2021 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Amendment Flag | false | |
Document Period End Date | Mar. 31, 2021 | |
Document Fiscal Year Focus | 2021 | |
Document Fiscal Period Focus | Q1 | |
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 | 30,453,374 | |
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 | Mar. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 90,677 | $ 142,086 |
Short-term investments | 122,436 | 89,734 |
Prepaid expenses and other current assets | 4,642 | 4,530 |
Total current assets | 217,755 | 236,350 |
Property and equipment, net | 1,206 | 1,293 |
Operating lease right-of-use assets | 1,854 | 1,949 |
Other assets | 1,426 | 1,272 |
Total assets | 222,241 | 240,864 |
Current liabilities: | ||
Accounts payable | 5,658 | 3,151 |
Accrued expenses | 27,781 | 13,411 |
Operating lease liabilities | 653 | 636 |
Derivative liability | 930 | 1,264 |
Total current liabilities | 35,022 | 18,462 |
Revenue interest liability, net | 50,860 | 47,651 |
Operating lease liabilities, noncurrent | 2,452 | 2,627 |
Other liabilities | 24 | 29 |
Total liabilities | 88,358 | 68,769 |
Commitments and contingencies | 0 | 0 |
Stockholders’ equity: | ||
Preferred stock, $0.0001 par value; 10,000,000 shares authorized as of March 31, 2021 and December 31, 2020, respectively; and zero shares issued and outstanding as of March 31, 2021 and December 31, 2020, respectively | 0 | 0 |
Common stock, $0.0001 par value; 200,000,000 shares authorized as of March 31, 2021 and December 31, 2020, respectively; 30,420,789 shares issued and 30,198,129 shares outstanding, excluding 222,660 shares subject to repurchase as of March 31, 2021; and 30,032,600 shares issued and 29,776,544 shares outstanding, excluding 256,056 shares subject to repurchase as of December 31, 2020 | 3 | 3 |
Additional paid-in capital | 357,583 | 345,180 |
Accumulated deficit | (223,703) | (173,171) |
Accumulated other comprehensive income | 0 | 83 |
Total stockholders’ equity | 133,883 | 172,095 |
Total liabilities and stockholders’ equity | $ 222,241 | $ 240,864 |
Condensed Consolidated Balanc_2
Condensed Consolidated Balance Sheets (Parenthetical) - $ / shares | Mar. 31, 2021 | Dec. 31, 2020 |
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 | 30,420,789 | 30,032,600 |
Common stock, shares, outstanding | 30,198,129 | 29,776,544 |
Common stock, subject to repurchase | 222,660 | 256,056 |
Condensed Consolidated Statemen
Condensed Consolidated Statements of Operations (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Operating expenses: | ||
Research and development | $ 38,134 | $ 17,340 |
General and administrative | 9,479 | 4,692 |
Total operating expenses | 47,613 | 22,032 |
Loss from operations | (47,613) | (22,032) |
Other income (expense): | ||
Interest income | 149 | 749 |
Interest expense | (3,381) | 0 |
Change in fair value of derivative liability | 334 | 0 |
Other expense, net | (16) | (23) |
Net loss before provision for income taxes | (50,527) | (21,306) |
Provision for income taxes | 5 | 4 |
Net loss | $ (50,532) | $ (21,310) |
Net loss per share, basic and diluted | $ (1.68) | $ (0.86) |
Weighted-average shares of common stock outstanding, basic and diluted | 30,105,017 | 24,704,651 |
Condensed Consolidated Statem_2
Condensed Consolidated Statements of Comprehensive Loss (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Statement Of Income And Comprehensive Income [Abstract] | ||
Net loss | $ (50,532) | $ (21,310) |
Other comprehensive gain (loss): | ||
Unrealized loss on available-for-sale investments | (74) | (149) |
Cumulative translation adjustments | (9) | (4) |
Comprehensive loss | $ (50,615) | $ (21,463) |
Condensed Consolidated Statem_3
Condensed Consolidated Statements of Stockholders' Equity (Unaudited) - USD ($) $ in Thousands | Total | Common Stock | Additional Paid-In Capital | Accumulated Deficit | Accumulated Other Comprehensive Income(Loss) |
Balance at Dec. 31, 2019 | $ 130,349 | $ 2 | $ 200,119 | $ (69,901) | $ 129 |
Balance, Shares at Dec. 31, 2019 | 22,600,338 | ||||
Issuance of common stock | 44,659 | $ 1 | 44,658 | ||
Issuance of common stock, shares | 2,400,000 | ||||
Restricted common stock vested in the period, Shares | 33,398 | ||||
Stock-based compensation | 2,573 | 2,573 | |||
Net loss | (21,310) | (21,310) | |||
Other comprehensive loss | (153) | (153) | |||
Balance at Mar. 31, 2020 | 156,118 | $ 3 | 247,350 | (91,211) | (24) |
Balance, Shares at Mar. 31, 2020 | 25,033,736 | ||||
Balance at Dec. 31, 2019 | 130,349 | $ 2 | 200,119 | (69,901) | 129 |
Balance, Shares at Dec. 31, 2019 | 22,600,338 | ||||
Balance at Dec. 31, 2020 | 172,095 | $ 3 | 345,180 | (173,171) | 83 |
Balance, Shares at Dec. 31, 2020 | 29,776,544 | ||||
Issuance of common stock in connection with common stock option exercises | 80 | 80 | |||
Issuance of common stock in connection with common stock option exercises, Shares | 12,535 | ||||
Issuance of common stock | 7,038 | 7,038 | |||
Issuance of common stock, shares | 375,654 | ||||
Restricted common stock vested in the period, Shares | 33,396 | ||||
Stock-based compensation | 5,285 | 5,285 | |||
Net loss | (50,532) | (50,532) | |||
Other comprehensive loss | (83) | $ (83) | |||
Balance at Mar. 31, 2021 | $ 133,883 | $ 3 | $ 357,583 | $ (223,703) | |
Balance, Shares at Mar. 31, 2021 | 30,198,129 |
Condensed Consolidated Statem_4
Condensed Consolidated Statements of Stockholders' Equity (Unaudited) (Parenthetical) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Common Stock | ||
Issuance of common stock upon public offering, issuance costs | $ 25 | $ 3,342 |
Condensed Consolidated Statem_5
Condensed Consolidated Statements of Cash Flows (Unaudited) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Operating activities | ||
Net loss | $ (50,532) | $ (21,310) |
Reconciliation of net loss to net cash used in operating activities: | ||
Stock-based compensation | 5,285 | 2,573 |
Depreciation and amortization | 90 | 73 |
Amortization of operating lease right-of-use assets | 95 | 83 |
Non-cash interest expense related to the revenue interest liability | 3,381 | 0 |
Net amortization (accretion) on investments | 6 | (52) |
Change in fair value of derivative liability | (334) | 0 |
Change in operating assets and liabilities: | ||
Prepaid and other current assets | (112) | (158) |
Other assets | (160) | 0 |
Accounts payable, accrued expenses and other liabilities | 17,228 | 476 |
Operating lease liabilities | (157) | (36) |
Net cash used in operating activities | (25,210) | (18,351) |
Investing activities | ||
Proceeds from maturities of investments | 48,600 | 29,500 |
Proceeds from paydown of investments | 2,000 | 4,359 |
Purchase of investments | (83,381) | 0 |
Purchase of property and equipment | (3) | (98) |
Net cash (used in) provided by investing activities | (32,784) | 33,761 |
Financing activities | ||
Proceeds from issuance of common stock in public offerings, net of issuance costs | 6,914 | 44,659 |
Proceeds from issuance of common stock pursuant to equity award plans | 80 | 0 |
Payment of issuance costs related to the revenue interest liability | (400) | 0 |
Net cash provided by financing activities | 6,594 | 44,659 |
Effect of exchange rate on cash and cash equivalents | (9) | (4) |
Net (decrease) increase in cash and cash equivalents | (51,409) | 60,065 |
Cash and cash equivalents at beginning of period | 142,086 | 11,970 |
Cash and cash equivalents at end of period | 90,677 | 72,035 |
Supplemental disclosure of cash flow information: | ||
Operating cash flows paid for operating lease | $ 212 | $ 107 |
Organization and Description of
Organization and Description of Business | 3 Months Ended |
Mar. 31, 2021 | |
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 development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. The Company’s pipeline consists of two clinical-stage product candidates, maralixibat and volixibat, with mechanisms of action that have potential utility across a wide range of orphan liver diseases. The Company commenced significant operations in November 2018. The unaudited condensed consolidated financial statements include the accounts of the Company and its wholly-owned subsidiaries. All intercompany balances and transactions among the consolidated entities have been eliminated in consolidation. The Company views its operations and manages its business as one operating segment. 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 March 31, 2021, the Company had an accumulated deficit of $223.7 million and cash, cash equivalents and investments of $213.1 million, which are available to fund future operations. The Company believes that its cash, cash equivalents and investments as of March 31, 2021 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. The unaudited condensed consolidated financial statements have been prepared on a going concern basis, which contemplates the realization of assets and satisfaction of liabilities in the normal course of business. The unaudited condensed consolidated financial statements do not include any adjustments relating to the recoverability and classification of recorded asset amounts or the amounts and classification of liabilities that might result from the outcome of this uncertainty. Management expects to continue to incur additional substantial losses in the foreseeable future as a result of the Company’s research and development activities. |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 3 Months Ended |
Mar. 31, 2021 | |
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 consolidated balance sheet as of December 31, 2020 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. 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, 2020, as filed with the SEC on March 9, 2021. 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. The most significant estimates in the Company’s unaudited condensed consolidated financial statements relate to the revenue interest liability, accrued research and development expenses, the valuation of derivative liabilities, equity award compensation and the valuation allowance of deferred tax assets resulting from net operating losses. These estimates and assumptions are based upon historical experience, . Significant Accounting Policies There have been no significant changes to the accounting policies during the three months ended March 31, 2021, 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 Stock-Based Compensation The Company recognizes stock-based compensation for all stock-based awards based on the grant date fair value of the award. For stock-based awards with service conditions, the fair value of the awards is amortized on a straight-line basis over the requisite service period Net Loss Per Share Basic net loss per share is computed by dividing net loss attributable to common stockholders 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 attributable to common stockholders 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, contingently issuable employee stock purchase plan shares, contingently issuable performance stock units (“PSUs”) and contingently issuable underwriter option shares because their effect would be anti-dilutive due to the Company’s net loss. Since the Company incurred a net loss in each of the periods presented, basic and diluted net loss per share were the same. The following outstanding potentially dilutive shares of common stock have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: March 31, December 31, 2021 2020 Options to purchase common stock 6,263,306 5,071,740 Common stock subject to repurchase 222,660 256,056 Employee stock purchase plan contingently issuable 32,578 12,931 PSUs contingently issuable 227,537 — Underwriter option shares contingently issuable — 562,500 Total 6,746,081 5,903,227 Recently Adopted Accounting Pronouncements In November 2018, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) Collaborative Arrangements (Topic 818): Clarifying the Interaction Between Topic 808 and Topic 606, There was no impact on the accompanying consolidated financial statements as of the adoption date. Recent Accounting Pronouncements Not Yet Adopted In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments-Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments Codification Improvements to Topic 326, Financial Instruments - Credit Losses Codification Improvements to Topic 326, Financial Instruments—Credit Losses, Derivatives and Hedging (Topic 815), and Financial Instruments (Topic 842), Codification Improvements to Financial Instruments is effective for fiscal years, and interim periods within those years, beginning after December 15, 2019 for public business entities, excluding smaller reporting companies. . In August 2020, the FASB issued ASU No. 2020-06, Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging-Contracts in Entity’s Own Equity (Subtopic 815-40) The Company is currently evaluating the impact this change will have on its consolidated financial statements. |
Fair Value Measurements
Fair Value Measurements | 3 Months Ended |
Mar. 31, 2021 | |
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) : March 31, 2021 Level 1 Level 2 Level 3 Total Financial assets: Money market fund $ 89,221 $ — $ — $ 89,221 U.S. treasury bills 6,000 — — 6,000 Corporate debt securities — 6,506 — 6,506 Commercial paper — 104,892 — 104,892 U.S. government bonds — 5,038 — 5,038 Total financial assets $ 95,221 $ 116,436 $ — $ 211,657 Financial liabilities: Derivative liability — — $ 930 $ 930 Total financial liabilities $ — $ — $ 930 $ 930 December 31, 2020 Level 1 Level 2 Level 3 Total Financial assets: Money market fund $ 127,783 $ — $ — $ 127,783 U.S. treasury bills 29,997 — — 29,997 Corporate debt securities — 23,201 — 23,201 Commercial paper — 41,460 — 41,460 U.S. government bonds — 5,066 — 5,066 Asset-backed securities — 2,006 — 2,006 Total financial assets $ 157,780 $ 71,733 $ — $ 229,513 Financial liabilities: Derivative liability $ — $ — $ 1,264 $ 1,264 Total financial liabilities $ — $ — $ 1,264 $ 1,264 The carrying amounts of certain financial instruments such as cash and cash equivalents, prepaid expenses, other current assets, accounts payable, accrued expenses, and other current liabilities as of March 31, 2021 and December 31, 2020 approximate their related fair values due to the short-term maturities of these instruments. Certain financial instruments classified within Level 2 of the fair value hierarchy include the types of instruments that trade in markets that are not considered to be active, but are valued based on quoted market prices, broker or dealer quotations, or alternative pricing sources with reasonable levels of price transparency. The carrying amount of the revenue interest liability as of March 31, 2021 and December 31, 2020 approximates its fair value and is based on the Company’s contractual repayment obligation to the Purchasers (as defined below), based on the current estimates of future revenues, over the life of the Revenue Interest Purchase Agreement (“RIPA”) Derivative Liability The debt pursuant to the RIPA contains embedded derivatives requiring bifurcation as a single compound derivative instrument. The Company estimated the fair value of the derivative liability at March 31, 2021 and December 31, 2020 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 March 31, 2021 and December 31, 2020. The estimated probability and timing of underlying events triggering the exercisability of the put options contained within the RIPA, forecasted cash flows and the discount rate are significant unobservable inputs used to determine the estimated fair value of the entire instrument with the embedded derivative. As of March 31, 2021 and December 31, 2020, the discount rate used for valuation of the derivative liability is 15.7% and 15.9%, respectively. The following table provides a summary of the change in the estimated fair value of the Company’s derivative liability, classified as Level 3 in the fair value hierarchy: Balance at December 31, 2020 $ 1,264 Change in fair value of derivative liability (334 ) Balance at March 31, 2021 $ 930 |
Financial Instruments
Financial Instruments | 3 Months Ended |
Mar. 31, 2021 | |
Financial Instruments Owned At Fair Value [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): March 31, 2021 Amortized Cost Unrealized Gain Unrealized Loss Estimated Fair Value Cash equivalents and investments: Money market fund $ 89,221 $ — $ — $ 89,221 U.S. treasury bills 6,000 — — 6,000 Corporate debt securities 6,500 6 — 6,506 Commercial paper 104,892 — — 104,892 U.S. government bonds 5,037 1 — 5,038 Total cash equivalents and investments $ 211,650 $ 7 $ — $ 211,657 Classified as: Cash equivalents $ 89,221 Short-term investments 122,436 Total cash equivalents and investments $ 211,657 December 31, 2020 Amortized Cost Unrealized Gain Unrealized Loss Estimated Fair Value Cash equivalents and investments: Money market fund $ 127,783 $ — $ — $ 127,783 U.S. treasury bills 29,995 2 — 29,997 Corporate debt securities 23,126 75 — 23,201 Commercial paper 41,460 — — 41,460 U.S. government bonds 5,067 — (1 ) 5,066 Asset-backed securities 2,001 5 — 2,006 Total cash equivalents and investments $ 229,432 $ 82 $ (1 ) $ 229,513 Classified as: Cash equivalents $ 139,779 Short-term investments 89,734 Total cash equivalents and investments $ 229,513 As of March 31, 2021, the remaining contractual maturities of available-for-sale debt securities were less than 12 months. During the three months ended March 31, 2021 and 2020, there have been no significant realized gains or losses on available-for-sale investments , no investments had been in a continuous unrealized loss position for more than 12 months, and the Company did not recognize any other-than-temporary impairment losses on these securities |
Accrued Expenses
Accrued Expenses | 3 Months Ended |
Mar. 31, 2021 | |
Payables And Accruals [Abstract] | |
Accrued Expenses | 5. Accrued Expenses Accrued expenses consist of the following (in thousands): March 31, December 31, 2021 2020 Accrued clinical trials $ 3,705 $ 3,673 Accrued professional service fees 2,822 2,157 Accrued contract manufacturing and non-clinical costs 3,215 2,780 Accrued compensation and related benefits 3,039 4,801 Accrued milestone payments 15,000 — Total accrued expenses $ 27,781 $ 13,411 |
Revenue Interest Purchase Agree
Revenue Interest Purchase Agreement | 3 Months Ended |
Mar. 31, 2021 | |
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 with Mulholland SA LLC, an affiliate of Oberland Capital LLC, as agent for the purchasers party thereto (the “Purchasers”), and the Purchasers to obtain financing for the commercialization and further development of maralixibat and other working capital needs. The Company may be entitled to receive up to approximately $85.0 million in subsequent installments as follows : As consideration for such payments, the Purchasers have the right to receive certain revenue interests (the “Revenue Interests”) from the Company based on annual net sales of maralixibat, if approved, which will be tiered payments (the “Revenue Interest Payments”) based on whether such annual net sales 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 will initially be 9.75% (at Tier 1) and 2.00% (at Tier 2 and Tier 3) of such annual net sales. If the Purchasers have received Revenue Interest Payments in an amount equal to or greater than 110.0% of the total payments actually made by the Purchasers to the Company, exclusive of transaction expenses (the “Cumulative Purchaser Payments”), on or prior to December 31, 2026, the Revenue Interests shall be reduced to 2.00% at Tier 1 and 0.00% at Tier 3 for all subsequent calendar years beginning on January 1, 2027. If the Purchasers have not received Revenue Interest Payments in an amount equal to or greater than 110.0% of the Cumulative Purchaser Payments on or prior to December 31, 2026, the Revenue Interests shall be increased for all subsequent calendar years beginning on January 1, 2027 to a single defined rate (with no separate tiers) that would have provided the Purchasers with an amount equal to 110.0% of the Cumulative Purchaser Payments on or prior to December 31, 2026 had such rate applied to Tier 1 of initial Revenue Interest Payments. The Purchasers’ rights to receive the Revenue Interest Payments shall terminate on the date on which the Purchasers have received Revenue Interest Payments of 195.0% of the Cumulative Purchaser Payments, unless the RIPA is terminated earlier. Under the RIPA, the Company has an option (the “Call Option”) to terminate the RIPA and repurchase future Revenue Interests at any time upon advance written notice. Additionally, the Purchasers have 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 12 th In addition, the RIPA contains various representations and warranties, information rights, non-financial covenants, indemnification obligations and other provisions that are customary for a transaction of this nature. The Purchaser’s obligations to fund the scheduled installments are 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 Options under the RIPA that are exercisable by Purchasers upon certain contingent events were determined to be embedded derivatives 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 will be amortized to interest expense over the expected term of the debt using the effective interest method. In connection with the RIPA, as of March 31, 2021 and December 31, 2020, $50.9 million and $47.7 million, respectively, was recorded as a revenue interest liability on the accompanying unaudited condensed consolidated balance sheets. The Company imputes interest expense associated with this liability using the effective interest rate method. The effective interest rate is 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 may vary during the term of the agreement depending on a number of factors, including the level of forecasted net sales. The Company evaluates the interest rate quarterly based on its current net sales forecasts utilizing the prospective method. A significant increase or decrease in net sales will materially impact the revenue interest liability, interest expense and the time period for repayment. The Company recorded $3.4 million in interest expense related to this arrangement for the three months ended March 31, 2021. The Company incurred $0.8 million of issuance costs in connection with the RIPA, which will be amortized to interest expense over the estimated term of the debt. Revenue Interest Payments made as a result of the Company’s net sales will reduce the revenue interest liability. The following table summarizes the revenue interest liability activity during the three months ended March 31, 2021 (in thousands): Revenue interest liability at December 31, 2020 $ 47,651 Interest expense recognized 3,381 Capitalized issuance costs (172 ) Revenue interest liability at March 31, 2021 $ 50,860 |
Asset Acquisitions
Asset Acquisitions | 3 Months Ended |
Mar. 31, 2021 | |
Asset Acquisitions [Abstract] | |
Asset Acquisitions | 7. Asset Acquisitions 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, and made an upfront payment to Shire of $7.5 million and issued Shire 1,859,151 shares of redeemable common stock with an estimated fair value of $7.0 million, or $3.76 per share. Under the terms of the Shire 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 maralixibat in certain indications and an additional $25.0 million upon regulatory approval of maralixibat 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 maralixibat 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. The Company paid development milestones of $2.5 million in July 2019 related to the initiation of the Phase 3 MARCH clinical trial of maralixibat in progressive familial intrahepatic cholestasis (“PFIC”), $10.0 million in December 2020 upon acceptance of a marketing authorization application filing to the European Medicines Agency for maralixibat for the treatment of PFIC2 and $2.0 million in January 2021 associated with the initiation of the VISTAS Phase 2b clinical trial of volixibat in primary sclerosing cholangitis. Development milestones achieved prior to regulatory approval of the product are expensed by the Company in the period incurred as research and development. As of March 31, 2021, the Company accrued $15.0 million of additional milestones which were subsequently paid in April 2021, consisting of (1) $5.0 million which would have been due had the Company initiated a Phase 3 clinical trial in ALGS, and (2) $10.0 million associated with the acceptance of the Company’s NDA filing by the FDA for maralixibat for the treatment of cholestatic pruritus in patients with ALGS based upon the results of the Company’s Phase 2b ICONIC clinical trial. 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. The Company is 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 will be required to pay a low single-digit royalty on net sales. The Company’s royalty obligations continue 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 are creditable against the royalty obligations to Shire under the Shire Agreement. In July 2019, the Company achieved a development milestone related to the initiation of the Phase 3 MARCH-PFIC clinical trial of maralixibat in PFIC and made a $0.5 million payment to Satiogen, which was expensed by the Company as research and development expense. As of March 31, 2021, no additional milestones had been accrued as there were no other potential milestones yet considered probable. 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 T hrough 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 |
Leases
Leases | 3 Months Ended |
Mar. 31, 2021 | |
Leases [Abstract] | |
Leases | 8. 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 in the accompanying unaudited condensed consolidated balance sheets with a corresponding reduction of the r ight-of-use (“ROU”) 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 lease 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 March 31, 2021, the Company recorded an aggregate ROU asset of $1.9 million and an aggregate lease liability of $3.1 million in the accompanying unaudited condensed consolidated balance sheet. The weighted-average remaining lease term is 3.9 years. As of March 31, 2021, undiscounted future minimum payments under the Company’s operating leases are as follows (in thousands): Year Ending December 31, Undiscounted Rent Payments 2021 (remaining nine months) $ 653 2022 891 2023 920 2024 928 2025 230 Total undiscounted lease payments 3,622 Less: imputed interest (517 ) Total lease liability $ 3,105 Rent expense was $0.2 million and $0.2 million for the three months ended March 31, 2021 and 2020, respectively. Variable lease payments for operating expenses were immaterial for the three months ended March 31, 2021 and 2020. |
Stockholders' Equity
Stockholders' Equity | 3 Months Ended |
Mar. 31, 2021 | |
Equity [Abstract] | |
Stockholders' Equity | 9. Stockholders’ Equity Common Stock In January 2020, the Company completed a follow-on public offering of its common stock, pursuant to which the Company sold 2,400,000 shares of common stock at a public offering price of $20.00 per share, resulting in net proceeds of $44.7 million after deducting underwriting discounts, commissions and offering expenses. In August 2020, the SEC declared effective a registration statement on Form S-3 (“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 (“Sales Agreement”) with SVB Leerink LLC (“SVB 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 Shelf Registration through SVB Leerink acting as the sales agent and/or principal. During the three months ended March 31, 2021, the Company did not sell any common stock pursuant to the Sales Agreement. As of December 31, 2020, the Company had sold 305,969 shares of common stock in an at-the-market offering pursuant to the Sales Agreement at a weighted-average price of $21.55 per share, resulting in gross proceeds of $6.6 million. The net proceeds after deducting sales commissions to SVB Leerink and other issuance expenses were approximately $6.3 million. The remaining capacity under the Sales Agreement is approximately $68.4 million as of March 31, 2021. In December 2020, the Company completed an underwritten public offering of its common stock pursuant to the Shelf Registration. The Company sold 3,750,000 shares of common stock at a public offering price of $20.00 per share, resulting in net proceeds of $70.0 million after deducting underwriting discounts, commissions and offering expenses. In addition, the Company granted the underwriters an option, exercisable for 30 days, to purchase up to 562,500 additional shares of its common stock at the public offering price, less the underwriting discounts and commissions. In January 2021, the underwriters partially exercised their option and purchased 375,654 shares of the Company’s common stock at a public offering price of $20.00 per share, resulting in net proceeds of $7.1 million after deducting underwriting discounts. The remaining options expired unexercised. As of March 31, 2021 and December 31, 2020, 222,660 shares and 256,056 shares of common stock, respectively, were subject to repurchase by the Company. The unvested stock liability related to these shares is immaterial to all periods presented. Common Stock Reserved for Issuance Common stock reserved for issuance is as follows: March 31, December 31, 2021 2020 Stock options issued and outstanding 6,263,306 5,071,740 Reserved for future stock awards or option grants 2,079,402 2,009,410 Reserved for employee stock purchase plan 1,000,349 700,023 Reserved for PSUs 227,537 — Reserved for underwriter option shares — 562,500 9,570,594 8,343,673 |
Stock-Based Compensation
Stock-Based Compensation | 3 Months Ended |
Mar. 31, 2021 | |
Disclosure Of Compensation Related Costs Sharebased Payments [Abstract] | |
Stock-Based Compensation | 10. 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 (“RSUs”) 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 March 31, 2021, 1,393,102 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 RSUs 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 December 2020, the Company’s board of directors authorized an additional 750,000 shares of the Company’s common stock for future issuance. As of March 31, 2021, shares of common stock were available for issuance under the 2020 Inducement Plan. Stock Options The following table summarizes stock option activity during the three months ended March 31, 2021 (in thousands, except share and per share data): Number of Shares Weighted- Average Exercise Price Weighted- Average Remaining Contractual Life (in Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 5,071,740 $ 9.99 8.6 $ 40,631 Granted 1,306,400 $ 19.19 Exercised (12,535 ) $ 6.39 Canceled and forfeited (102,299 ) $ 11.56 Outstanding as of March 31, 2021 6,263,306 $ 11.89 8.4 $ 50,284 Vested and exercisable as of March 31, 2021 1,948,720 $ 6.87 7.4 $ 25,312 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 three months ended March 31, 2021 and 2020 was $14.55 and $12.67 per share, respectively . The total intrinsic value of options exercised during the three months ended March 31, 2021 was $0.2 million. As of March 31, 2021, the total unrecognized stock-based compensation related to unvested stock option awards granted was $46.5 million, which the Company expects to recognize over a weighted-average period of approximately 3.0 years. During the three months ended March 31, 2021, in connection with a separation agreement with an employee, the Company accelerated the vesting of certain unvested stock options and extended the exercise period related to the vested awards. The modification resulted in incremental stock-based compensation of $0.3 million, which was recognized immediately. 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 Company estimated expected volatility based on the historical volatility of a group of similar companies that are publicly traded. The historical volatility data was computed using the daily closing prices for the selected 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 March 31, 2021 2020 Exercise price $18.41-$21.22 $10.40 - $24.52 Expected term (in years) 6.08 5.50 - 6.08 Expected volatility 93.24%-94.06% 77.07% - 88.11% Risk-free interest rate 0.62%-1.00% 0.51% - 1.73% Expected dividend yield — — Performance Stock Units In March 2021, the Company granted an aggregate of 151,849 PSUs to employees which are subject to a performance condition of FDA marketing approval of maralixibat for the treatment of cholestatic pruritis in patients with ALGS (“NDA Approval”) by a certain date. If the performance condition is met, 50% will vest immediately and 50% will vest on June 30, 2023, subject to the employees’ continuous service through each vesting date. The weighted-average grant date fair value of these PSUs is $18.53 per share. Additionally, in March 2021, the Company granted an aggregate of 75,688 PSUs to certain executive participants (“Executive PSUs”). The Executive PSUs are subject to performance and market conditions: (1) NDA Approval by a certain date and (2) achievement of a specified stock price. The Executive PSU’s will vest as follows: • 25% of the granted Executive PSUs will vest upon NDA Approval and achievement of a specified stock price as measured by the volume-weighted average price of the Company’s common stock for the 30 trading days ended December 31, 2021 (“VWAP”). Up to an additional 25% of the granted Executive PSUs will vest as determined by the incremental performance of the common stock over a specified stock price measured by the VWAP (altogether the “Base Units”). • An equal number of units to the Base Units will vest on June 30, 2023, subject to the executive participants’ continued service with the Company. As the Executive PSU’s contained a market condition, the grant date fair value was determined using a Monte Carlo simulation model and the following input assumptions were used to estimate the Executive PSUs granted during the following period: Three Months Ended March 31, 2021 Common stock price on grant date $ 18.50 Expected term (in years) 0.80 Expected volatility 80% Risk-free interest rate 0.09% Expected dividend yield — The weighted-average grant date fair value of the Executive PSUs is $7.09 per share. As of March 31, 2021, the Company determined achievement of the NDA Approval performance condition was not met as applied under the Accounting Standards Codification 718, Compensation — Stock Compensation (Topic 718) . As a result, none of the PSUs or Executive PSUs vested and no associated expense was recognized during the three months ended March 31, 2021. The total unrecognized stock-based compensation related to the PSUs and the Executive PSUs was $3.4 million as of March 31, 2021 . 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”). The ESPP became effective on July 17, 2019. 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. During the three months ended March 31, 2021, no shares were issued under the ESPP. As of March 31, 2021, the Company had 1,000,349 shares available for future issuance under the ESPP. The stock-based compensation related to the ESPP for the three months ended March 31, 2021 was $0.2 million. Restricted 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. The modified shares have a four-year vesting period and a measurement date fair value of $2.936 per share. For the three months ended March 31, 2021 and 2020, 33,396 shares and 33,398 shares vested, respectively. Total stock-based compensation is reflected in the unaudited condensed consolidated statements of operations as follows (in thousands): Three Months Ended March 31, 2021 2020 General and administrative $ 2,542 $ 1,532 Research and development 2,743 1,041 Total $ 5,285 $ 2,573 |
Contingencies
Contingencies | 3 Months Ended |
Mar. 31, 2021 | |
Commitments And Contingencies Disclosure [Abstract] | |
Contingencies | 11. 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 Event
Subsequent Event | 3 Months Ended |
Mar. 31, 2021 | |
Subsequent Events [Abstract] | |
Subsequent Event | 12. Subsequent Event In April 2021, the Company entered into an Option, License and Collaboration Agreement (“Vivet Collaboration Agreement”) with Vivet Therapeutics SAS (“Vivet”). Pursuant to the Vivet Collaboration Agreement, Vivet granted the Company the exclusive option to develop and subsequently commercialize Vivet’s two proprietary AAV gene therapy programs for PFIC, subtypes 3 and 2. Under the terms of the Vivet Collaboration Agreement, the Company agreed to provide funding to support certain research and development costs associated with the two gene therapy programs. Pursuant to the Vivet Collaboration Agreement, the Company paid an upfront fee of €3.5 million and initial research and development funding of €5.3 million ($4.2 million and $6.4 million, respectively, based on the exchange rate in effect at the time of payment). In April 2021, the Company entered into an exclusive licensing agreement with CANbridge Pharmaceuticals (“CANbridge”). Under the terms of the agreement, CANbridge has obtained the exclusive right to develop and commercialize maralixibat within the Greater China regions (China, Hong Kong, Macau and Taiwan) for ALGS, PFIC, and biliary atresia. The Company is entitled to receive an $11.0 million upfront payment, research and development funding, and up to $109.0 million for the achievement of future regulatory and commercial maralixibat milestones, with significant double-digit tiered royalties based on product net sales. |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 3 Months Ended |
Mar. 31, 2021 | |
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 consolidated balance sheet as of December 31, 2020 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. 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, 2020, as filed with the SEC on March 9, 2021. |
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. The most significant estimates in the Company’s unaudited condensed consolidated financial statements relate to the revenue interest liability, accrued research and development expenses, the valuation of derivative liabilities, equity award compensation and the valuation allowance of deferred tax assets resulting from net operating losses. These estimates and assumptions are based upon historical experience, . |
Significant Accounting Policies | Significant Accounting Policies There have been no significant changes to the accounting policies during the three months ended March 31, 2021, 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 |
Stock-Based Compensation | Stock-Based Compensation The Company recognizes stock-based compensation for all stock-based awards based on the grant date fair value of the award. For stock-based awards with service conditions, the fair value of the awards is amortized on a straight-line basis over the requisite service period |
Net Loss Per Share | Net Loss Per Share Basic net loss per share is computed by dividing net loss attributable to common stockholders 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 attributable to common stockholders 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, contingently issuable employee stock purchase plan shares, contingently issuable performance stock units (“PSUs”) and contingently issuable underwriter option shares because their effect would be anti-dilutive due to the Company’s net loss. Since the Company incurred a net loss in each of the periods presented, basic and diluted net loss per share were the same. The following outstanding potentially dilutive shares of common stock have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: March 31, December 31, 2021 2020 Options to purchase common stock 6,263,306 5,071,740 Common stock subject to repurchase 222,660 256,056 Employee stock purchase plan contingently issuable 32,578 12,931 PSUs contingently issuable 227,537 — Underwriter option shares contingently issuable — 562,500 Total 6,746,081 5,903,227 |
Recently Adopted Accounting Pronouncements | Recently Adopted Accounting Pronouncements In November 2018, the Financial Accounting Standards Board (“FASB”) issued Accounting Standards Update (“ASU”) Collaborative Arrangements (Topic 818): Clarifying the Interaction Between Topic 808 and Topic 606, There was no impact on the accompanying consolidated financial statements as of the adoption date. |
Recent Accounting Pronouncements Not Yet Adopted | Recent Accounting Pronouncements Not Yet Adopted In June 2016, the FASB issued ASU No. 2016-13, Financial Instruments-Credit Losses (Topic 326): Measurement of Credit Losses on Financial Instruments Codification Improvements to Topic 326, Financial Instruments - Credit Losses Codification Improvements to Topic 326, Financial Instruments—Credit Losses, Derivatives and Hedging (Topic 815), and Financial Instruments (Topic 842), Codification Improvements to Financial Instruments is effective for fiscal years, and interim periods within those years, beginning after December 15, 2019 for public business entities, excluding smaller reporting companies. . In August 2020, the FASB issued ASU No. 2020-06, Debt with Conversion and Other Options (Subtopic 470-20) and Derivatives and Hedging-Contracts in Entity’s Own Equity (Subtopic 815-40) The Company is currently evaluating the impact this change will have on its consolidated financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Outstanding Potentially Dilutive Shares of Common Stock Excluded from Calculation of Diluted Net Loss Per Share | The following outstanding potentially dilutive shares of common stock have been excluded from the calculation of diluted net loss per share for the periods presented due to their anti-dilutive effect: March 31, December 31, 2021 2020 Options to purchase common stock 6,263,306 5,071,740 Common stock subject to repurchase 222,660 256,056 Employee stock purchase plan contingently issuable 32,578 12,931 PSUs contingently issuable 227,537 — Underwriter option shares contingently issuable — 562,500 Total 6,746,081 5,903,227 |
Fair Value Measurements (Tables
Fair Value Measurements (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
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) : March 31, 2021 Level 1 Level 2 Level 3 Total Financial assets: Money market fund $ 89,221 $ — $ — $ 89,221 U.S. treasury bills 6,000 — — 6,000 Corporate debt securities — 6,506 — 6,506 Commercial paper — 104,892 — 104,892 U.S. government bonds — 5,038 — 5,038 Total financial assets $ 95,221 $ 116,436 $ — $ 211,657 Financial liabilities: Derivative liability — — $ 930 $ 930 Total financial liabilities $ — $ — $ 930 $ 930 December 31, 2020 Level 1 Level 2 Level 3 Total Financial assets: Money market fund $ 127,783 $ — $ — $ 127,783 U.S. treasury bills 29,997 — — 29,997 Corporate debt securities — 23,201 — 23,201 Commercial paper — 41,460 — 41,460 U.S. government bonds — 5,066 — 5,066 Asset-backed securities — 2,006 — 2,006 Total financial assets $ 157,780 $ 71,733 $ — $ 229,513 Financial liabilities: Derivative liability $ — $ — $ 1,264 $ 1,264 Total financial liabilities $ — $ — $ 1,264 $ 1,264 |
Summary of Change in Estimated Fair Value of Company's Derivative Liability Classified as Level 3 | The following table provides a summary of the change in the estimated fair value of the Company’s derivative liability, classified as Level 3 in the fair value hierarchy: Balance at December 31, 2020 $ 1,264 Change in fair value of derivative liability (334 ) Balance at March 31, 2021 $ 930 |
Financial Instruments (Tables)
Financial Instruments (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
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): March 31, 2021 Amortized Cost Unrealized Gain Unrealized Loss Estimated Fair Value Cash equivalents and investments: Money market fund $ 89,221 $ — $ — $ 89,221 U.S. treasury bills 6,000 — — 6,000 Corporate debt securities 6,500 6 — 6,506 Commercial paper 104,892 — — 104,892 U.S. government bonds 5,037 1 — 5,038 Total cash equivalents and investments $ 211,650 $ 7 $ — $ 211,657 Classified as: Cash equivalents $ 89,221 Short-term investments 122,436 Total cash equivalents and investments $ 211,657 December 31, 2020 Amortized Cost Unrealized Gain Unrealized Loss Estimated Fair Value Cash equivalents and investments: Money market fund $ 127,783 $ — $ — $ 127,783 U.S. treasury bills 29,995 2 — 29,997 Corporate debt securities 23,126 75 — 23,201 Commercial paper 41,460 — — 41,460 U.S. government bonds 5,067 — (1 ) 5,066 Asset-backed securities 2,001 5 — 2,006 Total cash equivalents and investments $ 229,432 $ 82 $ (1 ) $ 229,513 Classified as: Cash equivalents $ 139,779 Short-term investments 89,734 Total cash equivalents and investments $ 229,513 |
Accrued Expenses (Tables)
Accrued Expenses (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Payables And Accruals [Abstract] | |
Schedule of Accrued Expenses | Accrued expenses consist of the following (in thousands): March 31, December 31, 2021 2020 Accrued clinical trials $ 3,705 $ 3,673 Accrued professional service fees 2,822 2,157 Accrued contract manufacturing and non-clinical costs 3,215 2,780 Accrued compensation and related benefits 3,039 4,801 Accrued milestone payments 15,000 — Total accrued expenses $ 27,781 $ 13,411 |
Revenue Interest Purchase Agr_2
Revenue Interest Purchase Agreement (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Summary of Revenue Interest Liability | The following table summarizes the revenue interest liability activity during the three months ended March 31, 2021 (in thousands): Revenue interest liability at December 31, 2020 $ 47,651 Interest expense recognized 3,381 Capitalized issuance costs (172 ) Revenue interest liability at March 31, 2021 $ 50,860 |
Leases (Tables)
Leases (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Leases [Abstract] | |
Schedule of Undiscounted Future Minimum Payments under Operating Leases | As of March 31, 2021, undiscounted future minimum payments under the Company’s operating leases are as follows (in thousands): Year Ending December 31, Undiscounted Rent Payments 2021 (remaining nine months) $ 653 2022 891 2023 920 2024 928 2025 230 Total undiscounted lease payments 3,622 Less: imputed interest (517 ) Total lease liability $ 3,105 |
Stockholders' Equity (Tables)
Stockholders' Equity (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Equity [Abstract] | |
Schedule of Common Stock Reserved for Issuance | Common stock reserved for issuance is as follows: March 31, December 31, 2021 2020 Stock options issued and outstanding 6,263,306 5,071,740 Reserved for future stock awards or option grants 2,079,402 2,009,410 Reserved for employee stock purchase plan 1,000,349 700,023 Reserved for PSUs 227,537 — Reserved for underwriter option shares — 562,500 9,570,594 8,343,673 |
Stock-Based Compensation (Table
Stock-Based Compensation (Tables) | 3 Months Ended |
Mar. 31, 2021 | |
Summary of Stock Option Activity | The following table summarizes stock option activity during the three months ended March 31, 2021 (in thousands, except share and per share data): Number of Shares Weighted- Average Exercise Price Weighted- Average Remaining Contractual Life (in Years) Aggregate Intrinsic Value Outstanding as of December 31, 2020 5,071,740 $ 9.99 8.6 $ 40,631 Granted 1,306,400 $ 19.19 Exercised (12,535 ) $ 6.39 Canceled and forfeited (102,299 ) $ 11.56 Outstanding as of March 31, 2021 6,263,306 $ 11.89 8.4 $ 50,284 Vested and exercisable as of March 31, 2021 1,948,720 $ 6.87 7.4 $ 25,312 |
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 March 31, 2021 2020 Exercise price $18.41-$21.22 $10.40 - $24.52 Expected term (in years) 6.08 5.50 - 6.08 Expected volatility 93.24%-94.06% 77.07% - 88.11% Risk-free interest rate 0.62%-1.00% 0.51% - 1.73% Expected dividend yield — — |
Summary of Stock-based Compensation Reflected in Unaudited Condensed Consolidated Statements of Operations | Total stock-based compensation is reflected in the unaudited condensed consolidated statements of operations as follows (in thousands): Three Months Ended March 31, 2021 2020 General and administrative $ 2,542 $ 1,532 Research and development 2,743 1,041 Total $ 5,285 $ 2,573 |
Executive Performance Stock Units | |
Schedule of Assumptions Used to Estimate Fair Value of Stock Option Awards Granted | As the Executive PSU’s contained a market condition, the grant date fair value was determined using a Monte Carlo simulation model and the following input assumptions were used to estimate the Executive PSUs granted during the following period: Three Months Ended March 31, 2021 Common stock price on grant date $ 18.50 Expected term (in years) 0.80 Expected volatility 80% Risk-free interest rate 0.09% Expected dividend yield — |
Organization and Description _2
Organization and Description of Business - Additional Information (Details) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021USD ($)Segment | Dec. 31, 2020USD ($) | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | ||
Date of incorporation | May 2, 2018 | |
Number of operating segments | Segment | 1 | |
Accumulated deficit | $ 223,703 | $ 173,171 |
Cash, cash equivalents and investments | $ 213,100 |
Summary of Significant Accoun_4
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 | 3 Months Ended | 12 Months Ended |
Mar. 31, 2021 | Dec. 31, 2020 | |
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 6,746,081 | 5,903,227 |
Options to Purchase Common Stock | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 6,263,306 | 5,071,740 |
Common Stock Subject to Repurchase | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 222,660 | 256,056 |
Employee Stock Purchase Plan Contingently Issuable | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 32,578 | 12,931 |
PSUs Contingently Issuable | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 227,537 | 0 |
Underwriter Option Shares Contingently Issuable | ||
Antidilutive Securities Excluded from Computation of Earnings Per Share [Line Items] | ||
Anti-dilutive shares of stock | 0 | 562,500 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Additional Information (Details) - Accounting Standards Update 2018-18 | Mar. 31, 2021 |
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. 1, 2021 |
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) - Fair Value, Recurring Basis - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Financial assets: | ||
Fair value measurements | $ 211,657 | $ 229,513 |
Financial liabilities: | ||
Derivative liability | 930 | 1,264 |
Total financial liabilities | 930 | 1,264 |
Level 1 | ||
Financial assets: | ||
Fair value measurements | 95,221 | 157,780 |
Financial liabilities: | ||
Derivative liability | 0 | 0 |
Total financial liabilities | 0 | 0 |
Level 2 | ||
Financial assets: | ||
Fair value measurements | 116,436 | 71,733 |
Financial liabilities: | ||
Derivative liability | 0 | 0 |
Total financial liabilities | 0 | 0 |
Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Financial liabilities: | ||
Derivative liability | 930 | 1,264 |
Total financial liabilities | 930 | 1,264 |
Money Market Fund | ||
Financial assets: | ||
Fair value measurements | 89,221 | 127,783 |
Money Market Fund | Level 1 | ||
Financial assets: | ||
Fair value measurements | 89,221 | 127,783 |
Money Market Fund | Level 2 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Money Market Fund | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
U.S. treasury bills | ||
Financial assets: | ||
Fair value measurements | 6,000 | 29,997 |
U.S. treasury bills | Level 1 | ||
Financial assets: | ||
Fair value measurements | 6,000 | 29,997 |
U.S. treasury bills | Level 2 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
U.S. treasury bills | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Corporate Debt Securities | ||
Financial assets: | ||
Fair value measurements | 6,506 | 23,201 |
Corporate Debt Securities | Level 1 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Corporate Debt Securities | Level 2 | ||
Financial assets: | ||
Fair value measurements | 6,506 | 23,201 |
Corporate Debt Securities | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Commercial Paper | ||
Financial assets: | ||
Fair value measurements | 104,892 | 41,460 |
Commercial Paper | Level 1 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
Commercial Paper | Level 2 | ||
Financial assets: | ||
Fair value measurements | 104,892 | 41,460 |
Commercial Paper | Level 3 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
U.S. Government Bonds | ||
Financial assets: | ||
Fair value measurements | 5,038 | 5,066 |
U.S. Government Bonds | Level 1 | ||
Financial assets: | ||
Fair value measurements | 0 | 0 |
U.S. Government Bonds | Level 2 | ||
Financial assets: | ||
Fair value measurements | 5,038 | 5,066 |
U.S. Government Bonds | Level 3 | ||
Financial assets: | ||
Fair value measurements | $ 0 | 0 |
Asset Backed Securities | ||
Financial assets: | ||
Fair value measurements | 2,006 | |
Asset Backed Securities | Level 1 | ||
Financial assets: | ||
Fair value measurements | 0 | |
Asset Backed Securities | Level 2 | ||
Financial assets: | ||
Fair value measurements | 2,006 | |
Asset Backed Securities | Level 3 | ||
Financial assets: | ||
Fair value measurements | $ 0 |
Fair Value Measurements - Addit
Fair Value Measurements - Additional Information (Details) | Mar. 31, 2021 | Dec. 31, 2020 |
Discount Rate | ||
Fair Value Assets Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Discount rate used for valuation to derivative liability | 0.157 | 0.159 |
Fair Value Measurements - Sum_2
Fair Value Measurements - Summary of Change in Estimated Fair Value of Company's Derivative Liability Classified as Level 3 (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Change in fair value of derivative liability | $ 334 | $ 0 |
Level 3 | ||
Fair Value Liabilities Measured On Recurring Basis Unobservable Input Reconciliation [Line Items] | ||
Balance at December 31, 2020 | 1,264 | |
Change in fair value of derivative liability | (334) | |
Balance at March 31, 2021 | $ 930 |
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 | Mar. 31, 2021 | Dec. 31, 2020 |
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Cash equivalents and investments, Amortized cost | $ 211,650 | $ 229,432 |
Cash equivalents and investments, Unrealized gain | 7 | 82 |
Cash equivalents and investments, Unrealized loss | (1) | |
Cash equivalents and investments, Estimated Fair Value | 211,657 | 229,513 |
Cash equivalents, Estimated Fair Value | 89,221 | 139,779 |
Short-term investments | 122,436 | 89,734 |
Money Market Fund | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Cash equivalents, Amortized Cost | 89,221 | 127,783 |
Cash equivalents, Estimated Fair Value | 89,221 | 127,783 |
U.S. treasury bills | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 6,000 | 29,995 |
Investments, Unrealized Gain | 0 | 2 |
Investments, Unrealized Loss | 0 | 0 |
Investments, Estimated Fair Value | 6,000 | 29,997 |
Corporate Debt Securities | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 6,500 | 23,126 |
Investments, Unrealized Gain | 6 | 75 |
Investments, Unrealized Loss | 0 | 0 |
Investments, Estimated Fair Value | 6,506 | 23,201 |
U.S. Government Bonds | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 5,037 | 5,067 |
Investments, Unrealized Gain | 1 | 0 |
Investments, Unrealized Loss | 0 | (1) |
Investments, Estimated Fair Value | 5,038 | 5,066 |
Commercial Paper | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 104,892 | 41,460 |
Investments, Unrealized Gain | 0 | 0 |
Investments, Unrealized Loss | 0 | 0 |
Investments, Estimated Fair Value | $ 104,892 | 41,460 |
Asset Backed Securities | ||
Fair Value Balance Sheet Grouping Financial Statement Captions [Line Items] | ||
Investments, Amortized Cost | 2,001 | |
Investments, Unrealized Gain | 5 | |
Investments, Unrealized Loss | 0 | |
Investments, Estimated Fair Value | $ 2,006 |
Financial Instruments - Additio
Financial Instruments - Additional Information (Details) - USD ($) | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Financial Instruments Owned At Fair Value [Abstract] | ||
Realized gains or losses on available-for-sale investments | $ 0 | $ 0 |
Investments in continuous unrealized loss position for more than 12 months | 0 | 0 |
Other-than-temporary impairment losses | $ 0 | $ 0 |
Accrued Expenses - Schedule of
Accrued Expenses - Schedule of Accrued Expenses (Details) - USD ($) $ in Thousands | Mar. 31, 2021 | Dec. 31, 2020 |
Payables And Accruals [Abstract] | ||
Accrued clinical trials | $ 3,705 | $ 3,673 |
Accrued professional service fees | 2,822 | 2,157 |
Accrued contract manufacturing and non-clinical costs | 3,215 | 2,780 |
Accrued compensation and related benefits | 3,039 | 4,801 |
Accrued milestone payments | 15,000 | |
Total accrued expenses | $ 27,781 | $ 13,411 |
Revenue Interest Purchase Agr_3
Revenue Interest Purchase Agreement - Additional Information (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 5 Months Ended | 12 Months Ended | |
Apr. 30, 2021 | Dec. 31, 2020 | Mar. 31, 2021 | May 06, 2021 | Dec. 31, 2020 | |
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Revenue interest liability | $ 47,651 | $ 50,860 | $ 47,651 | ||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Potential milestone payment to be received | 85,000 | ||||
Potential milestone payment to be received at the option of purchasers | $ 50,000 | ||||
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 | 2.00% | ||||
Revenue interest rate percentage shall be reduced, if revenue interest payment percentage greater than or equal to 110% at Tier | 2.00% | ||||
Revenue interest rate percentage shall be reduced, if revenue interest payment percentage greater than or equal to 110% at Tier 3 | 0.00% | ||||
Required revenue interest payment percentage of cumulative purchaser payments for termination | 195.00% | ||||
Required repurchase price percentage of cumulative purchaser prior to first anniversary of closing date | 120.00% | ||||
Required repurchase price percentage of cumulative purchaser payments after first anniversary and prior to third anniversary of closing date | 175.00% | ||||
Required repurchase price percentage of cumulative purchaser after third anniversary of closing date | 195.00% | ||||
Purchase agreement amount allocated to debt | 49,200 | ||||
Initial fair value of derivative liability | $ 1,300 | ||||
Revenue interest liability | 47,700 | 50,900 | $ 47,700 | ||
Interest expense | 3,400 | ||||
Debt issuance costs | $ 800 | ||||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | On or Prior to December 31, 2026 | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Minimum required revenue interest payment percentage to cumulative purchaser payments to reduce interest rate | 110.00% | ||||
Revenue Interest Purchase Agreement | Mulholland SA LLC, | Maralixibat | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Upfront payment received | $ 50,000 | ||||
Potential milestone payment received upon regulatory approval | $ 35,000 | ||||
Tier I | Mulholland SA LLC, | Maralixibat | Maximum | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Annual net sales | 350,000 | ||||
Tier 2 | Mulholland SA LLC, | Maralixibat | Minimum | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Annual net sales | 350,000 | ||||
Tier 2 | Mulholland SA LLC, | Maralixibat | Maximum | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Annual net sales | 1,100,000 | ||||
Tier 3 | Mulholland SA LLC, | Maralixibat | 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 | ||||
Subsequent Event | Revenue Interest Purchase Agreement | Mulholland SA LLC, | Maralixibat | |||||
Collaborative Arrangements And Noncollaborative Arrangement Transactions [Line Items] | |||||
Milestone and upfront payments received | $ 115,000 | ||||
Milestone payment received | $ 65,000 |
Revenue Interest Purchase Agr_4
Revenue Interest Purchase Agreement - Summary of Revenue Interest Liability (Details) $ in Thousands | 3 Months Ended |
Mar. 31, 2021USD ($) | |
Organization Consolidation And Presentation Of Financial Statements [Abstract] | |
Revenue interest liability | $ 47,651 |
Interest expense recognized | 3,381 |
Capitalized issuance costs | (172) |
Revenue interest liability | $ 50,860 |
Asset Acquisitions - Additional
Asset Acquisitions - Additional Information (Details) | 1 Months Ended | 3 Months Ended | ||
Dec. 31, 2020USD ($) | Jul. 31, 2019USD ($) | Nov. 30, 2018USD ($)ProductCandidate$ / sharesshares | Mar. 31, 2021USD ($) | |
Asset Acquisitions [Line Items] | ||||
Accrued milestones | $ 15,000,000 | |||
Shire Agreement | Shire | ||||
Asset Acquisitions [Line Items] | ||||
Number of product candidates | ProductCandidate | 2 | |||
Upfront payment | $ 7,500,000 | |||
Product sales milestone payments, payable | 30,000,000 | |||
Milestone payment | $ 2,500,000 | |||
Milestone payments, upon clinical trial treatment | 2,000,000 | |||
Accrued milestones | 15,000,000 | |||
Shire Agreement | Shire | Maralixibat | ||||
Asset Acquisitions [Line Items] | ||||
Milestone payments, payable | 109,500,000 | |||
Milestone payments, payable upon approval | 25,000,000 | |||
Milestone payment | $ 10,000,000 | |||
Accrued milestones | 10,000,000 | |||
Shire Agreement | Shire | Volixibat | ||||
Asset Acquisitions [Line Items] | ||||
Milestone payments, payable | $ 30,000,000 | |||
Shire Agreement | Shire | Clinical Trial in ALGS | ||||
Asset Acquisitions [Line Items] | ||||
Accrued milestones | 5,000,000 | |||
Shire Agreement | Shire | Redeemable Common Stock | ||||
Asset Acquisitions [Line Items] | ||||
Temporary equity, shares issued | shares | 1,859,151 | |||
Issuance of Series A redeemable convertible preferred stock, net of issuance costs | $ 7,000,000 | |||
Shares issued, price per share | $ / shares | $ 3.76 | |||
Assigned License Agreement | Satiogen Pharmaceuticals, Inc. | ||||
Asset Acquisitions [Line Items] | ||||
Milestone payments, payable | $ 10,500,000 | |||
Milestone payments, payable upon approval | 5,000,000 | |||
Accrued milestones | 0 | |||
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 | |||
Assigned License Agreement | Maralixibat | Satiogen Pharmaceuticals, Inc. | ||||
Asset Acquisitions [Line Items] | ||||
Milestone payment | $ 500,000 |
Leases - Additional Information
Leases - Additional Information (Detail) $ in Thousands | 1 Months Ended | 3 Months Ended | ||||
Nov. 30, 2019USD ($)ft² | Mar. 31, 2021USD ($) | Mar. 31, 2020USD ($) | Dec. 31, 2020USD ($) | Jan. 31, 2019USD ($) | Jan. 22, 2019ft² | |
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 | $ 1,854 | $ 1,949 | ||||
Lease liability | $ 3,105 | |||||
Weighted-average incremental borrowing rate | 8.00% | |||||
Weighted-average remaining lease term | 3 years 10 months 24 days | |||||
Rent expense | $ 200 | $ 200 | ||||
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 | Mar. 31, 2021USD ($) |
Leases [Abstract] | |
2021 (remaining nine months) | $ 653 |
2022 | 891 |
2023 | 920 |
2024 | 928 |
2025 | 230 |
Total undiscounted lease payments | 3,622 |
Less: imputed interest | (517) |
Total lease liability | $ 3,105 |
Stockholders' Equity - Addition
Stockholders' Equity - Additional Information (Details) - USD ($) | 1 Months Ended | 3 Months Ended | 12 Months Ended | ||||
Jan. 31, 2021 | Dec. 31, 2020 | Jan. 31, 2020 | Mar. 31, 2021 | Mar. 31, 2020 | Dec. 31, 2020 | Aug. 31, 2020 | |
Class Of Stock [Line Items] | |||||||
Maximum amount of sale covered in shelf registration statement | $ 300,000,000 | ||||||
Common stock aggregate offering price, remaining | $ 68,400,000 | $ 68,400,000 | |||||
SVB Leerink LLC | |||||||
Class Of Stock [Line Items] | |||||||
Maximum amount of offering issuance and sale covered in sales agreement | $ 75,000,000 | ||||||
Common Stock | |||||||
Class Of Stock [Line Items] | |||||||
Number of shares issued | 375,654 | 2,400,000 | |||||
Number of shares subject to repurchase | 256,056 | 222,660 | 256,056 | ||||
Common Stock | Sales Agreement | |||||||
Class Of Stock [Line Items] | |||||||
Proceeds from issuance of shares | $ 6,300,000 | ||||||
Gross proceeds from issuance of common stock | $ 6,600,000 | ||||||
Follow-on Public Offering | |||||||
Class Of Stock [Line Items] | |||||||
Proceeds from issuance of shares | $ 44,700,000 | ||||||
Follow-on Public Offering | Common Stock | |||||||
Class Of Stock [Line Items] | |||||||
Number of shares issued | 2,400,000 | ||||||
Shares issued, public offering price per share | $ 20 | ||||||
At The Market Offering | Common Stock | Sales Agreement | |||||||
Class Of Stock [Line Items] | |||||||
Number of shares issued | 3,750,000 | 0 | 305,969 | ||||
Shares issued, public offering price per share | $ 20 | $ 20 | |||||
Proceeds from issuance of shares | $ 70,000,000 | ||||||
At The Market Offering | Common Stock | Sales Agreement | Weighted Average | |||||||
Class Of Stock [Line Items] | |||||||
Shares issued, public offering price per share | $ 21.55 | $ 21.55 | |||||
Underwritten Public Offerings | Common Stock | Sales Agreement | Maximum | |||||||
Class Of Stock [Line Items] | |||||||
Number of shares issued | 562,500 | ||||||
Public Offering | |||||||
Class Of Stock [Line Items] | |||||||
Proceeds from issuance of shares | $ 7,100,000 | ||||||
Public Offering | Common Stock | |||||||
Class Of Stock [Line Items] | |||||||
Shares issued, public offering price per share | $ 20 | ||||||
Number of shares purchased | 375,654 |
Stockholders' Equity - Schedule
Stockholders' Equity - Schedule of Common Stock Reserved for Issuance (Details) - shares | Mar. 31, 2021 | Dec. 31, 2020 |
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 9,570,594 | 8,343,673 |
Stock Options | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 6,263,306 | 5,071,740 |
Reserved for Future Stock Awards or Option Grants | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 2,079,402 | 2,009,410 |
Reserved for Employee Stock Purchase Plan | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 1,000,349 | 700,023 |
Reserved for PSUs | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 227,537 | |
Reserved for Underwriter Option Shares | ||
Class Of Stock [Line Items] | ||
Common stock reserved for issuance | 562,500 |
Stock-Based Compensation - Addi
Stock-Based Compensation - Additional Information (Details) | Jun. 30, 2023 | Nov. 05, 2018$ / sharesshares | Mar. 31, 2021USD ($)d$ / sharesshares | Dec. 31, 2020shares | Jul. 31, 2019shares | Mar. 31, 2021USD ($)$ / sharesshares | Mar. 31, 2020$ / sharesshares |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Number of common stock approved and reserved for issuance | 9,570,594 | 8,343,673 | 9,570,594 | ||||
Incremental stock-based compensation | $ | $ 300,000 | ||||||
Stock Options | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Number of common stock approved and reserved for issuance | 6,263,306 | 5,071,740 | 6,263,306 | ||||
Grant date fair value of options granted | $ / shares | $ 14.55 | $ 12.67 | |||||
Stock-based compensation, Intrinsic value of options exercised | $ | $ 200,000 | ||||||
Total unrecognized stock-based compensation related to unvested stock option awards granted | $ | $ 46,500,000 | $ 46,500,000 | |||||
Unrecognized stock-based compensation related to unvested stock, expected to recognize over weighted-average period | 3 years | ||||||
Expected dividend yield | 0.00% | 0.00% | |||||
PSUs | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Number of common stock approved and reserved for issuance | 227,537 | 227,537 | |||||
Granted | 151,849 | ||||||
Weighted-average grant-date fair value | 50.00% | ||||||
Granted | $ / shares | $ 18.53 | $ 7.09 | |||||
Vested | 0 | ||||||
Vested and associated expenses | $ | $ 0 | ||||||
Total unrecognized stock-based compensation | $ | $ 3,400,000 | $ 3,400,000 | |||||
PSUs | Forecast | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Weighted-average grant-date fair value | 50.00% | ||||||
Executive Performance Stock Units | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Expected dividend yield | 0.00% | ||||||
Granted | $ / shares | $ 75,688 | ||||||
Common stock trading days | d | 30 | ||||||
Executive Performance Stock Units | NDA Approval and Achievement | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Weighted-average grant-date fair value | 25.00% | ||||||
Executive Performance Stock Units | Incremental Performance of Common Stock | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Weighted-average grant-date fair value | 25.00% | ||||||
Restricted Stock | |||||||
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 | ||||||
Vested | 33,396 | 33,398 | |||||
Total unrecognized compensation expense related to unvested restricted stock | $ | $ 700,000 | $ 700,000 | |||||
Restricted Stock | Founder | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Granted | $ / shares | $ 2.936 | ||||||
Restricted Stock | Common Stock | Founder | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Stock issued for services | 562,500 | ||||||
Vesting period | 4 years | ||||||
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 March 31, 2021, 1,393,102 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. 1, 2020 | ||||||
Shares of common stock ending date | Jan. 1, 2029 | ||||||
Percentage of annual increase in common stock available for issuance | 5.00% | ||||||
Number of common stock approved and reserved for issuance | 1,393,102 | 1,393,102 | |||||
2020 Inducement Plan | |||||||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |||||||
Number of common stock for future issuance | 750,000 | ||||||
Common stock available for issuance | 686,300 | ||||||
Number of additional common stock for future issuance | 750,000 | ||||||
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. During the three months ended March 31, 2021, no shares were issued under the ESPP. As of March 31, 2021, the Company had 1,000,349 shares available for future issuance under the ESPP. | ||||||
Shares of common stock expiration term | 10 years | ||||||
Shares of common stock beginning date | Jan. 1, 2020 | ||||||
Shares of common stock ending date | Jan. 1, 2029 | ||||||
Percentage of annual increase in common stock available for issuance | 1.00% | ||||||
Number of common stock approved and reserved for issuance | 1,000,349 | 1,000,349 | |||||
Stock issued for services | 500,000 | 0 | |||||
Annual increase in common stock available for issuance, shares | 1,500,000 |
Stock-Based Compensation - Summ
Stock-Based Compensation - Summary of Stock Option Activity (Details) - Stock Options - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended |
Mar. 31, 2021 | Dec. 31, 2020 | |
Options, Outstanding | ||
Number of shares, Outstanding, Beginning balance | 5,071,740 | |
Number of shares, Granted | 1,306,400 | |
Number of shares, Exercised | (12,535) | |
Number of shares, Canceled and forfeited | (102,299) | |
Number of shares, Outstanding, Ending balance | 6,263,306 | 5,071,740 |
Number of shares, Vested and exercisable | 1,948,720 | |
Weighted-average exercise price, Outstanding | ||
Weighted-average exercise price, Outstanding, Beginning balance | $ 9.99 | |
Weighted-average exercise price, Granted | 19.19 | |
Weighted-average exercise price, Exercised | 6.39 | |
Weighted-average exercise price, Canceled and forfeited | 11.56 | |
Weighted-average exercise price, Outstanding, Ending balance | 11.89 | $ 9.99 |
Weighted-average exercise price, Vested and exercisable | $ 6.87 | |
Share-based Payment Award, Options, Additional Disclosures | ||
Weighted-average remaining contractual life, Outstanding | 8 years 4 months 24 days | 8 years 7 months 6 days |
Weighted-average remaining contractual life, Vested and exercisable | 7 years 4 months 24 days | |
Aggregate intrinsic value, Outstanding | $ 50,284 | $ 40,631 |
Aggregate intrinsic value, Vested and exercisable | $ 25,312 |
Stock-Based Compensation - Sche
Stock-Based Compensation - Schedule of Assumptions Used to Estimate Fair Value of Stock Option Awards Granted (Details) - Stock Options - $ / shares | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Exercise price, minimum | $ 18.41 | $ 10.40 |
Exercise price, maximum | $ 21.22 | $ 24.52 |
Expected term (in years) | 6 years 29 days | |
Expected volatility, minimum | 93.24% | 77.07% |
Expected volatility, maximum | 94.06% | 88.11% |
Risk-free interest rate, minimum | 0.62% | 0.51% |
Risk-free interest rate, maximum | 1.00% | 1.73% |
Expected dividend yield | 0.00% | 0.00% |
Minimum | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term (in years) | 5 years 6 months | |
Maximum | ||
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | ||
Expected term (in years) | 6 years 29 days |
Stock-Based Compensation - Inpu
Stock-Based Compensation - Input Assumptions used to Estimate Executive PSUs Granted During Period (Details) - Executive Performance Stock Units | 3 Months Ended |
Mar. 31, 2021$ / shares | |
Share-based Compensation Arrangement by Share-based Payment Award [Line Items] | |
Common stock price on grant date | $ 18.50 |
Expected term (in years) | 9 months 18 days |
Expected volatility | 80.00% |
Risk-free interest rate | 0.09% |
Expected dividend yield | 0.00% |
Stock-Based Compensation - Su_2
Stock-Based Compensation - Summary of Stock-based Compensation Reflected in Unaudited Condensed Consolidated Statements of Operations (Details) - USD ($) $ in Thousands | 3 Months Ended | |
Mar. 31, 2021 | Mar. 31, 2020 | |
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | $ 5,285 | $ 2,573 |
General and Administrative | ||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | 2,542 | 1,532 |
Research and Development | ||
Share-based Payment Arrangement, Expensed and Capitalized, Amount [Line Items] | ||
Stock-based compensation expense | $ 2,743 | $ 1,041 |
Subsequent Event - Additional I
Subsequent Event - Additional Information (Details) - Subsequent Event € in Millions | 1 Months Ended | |
Apr. 30, 2021USD ($) | Apr. 30, 2021EUR (€) | |
Vivet Collaboration Agreement | ||
Subsequent Event [Line Items] | ||
License and collaboration agreement date | Apr. 30, 2021 | Apr. 30, 2021 |
Upfront fee | $ 4,200,000 | € 3.5 |
Initial research and development funding | 6,400,000 | € 5.3 |
Exclusive Licensing Agreement | CANbridge | Maralixibat | ||
Subsequent Event [Line Items] | ||
Potential upfront payment to be received | 11,000,000 | |
Exclusive Licensing Agreement | CANbridge | Maralixibat | Maximum | ||
Subsequent Event [Line Items] | ||
Potential regulatory and commercial milestone payment to be received | $ 109,000,000 |