Cover Page
Cover Page - USD ($) | 12 Months Ended | ||
Dec. 31, 2021 | Feb. 21, 2022 | Jun. 30, 2021 | |
Cover [Abstract] | |||
Document Type | 10-K | ||
Document Annual Report | true | ||
Document Transition Report | false | ||
Entity Interactive Data Current | Yes | ||
Amendment Flag | false | ||
Document Fiscal Year Focus | 2021 | ||
Document Period End Date | Dec. 31, 2021 | ||
Document Fiscal Period Focus | FY | ||
Entity Registrant Name | MADRIGAL PHARMACEUTICALS, INC. | ||
Entity Central Index Key | 0001157601 | ||
Entity File Number | 001-33277 | ||
Current Fiscal Year End Date | --12-31 | ||
Entity Tax Identification Number | 04-3508648 | ||
Entity Incorporation, State or Country Code | DE | ||
Entity Well-known Seasoned Issuer | Yes | ||
Entity Voluntary Filers | No | ||
Entity Current Reporting Status | Yes | ||
Entity Public Float | $ 819,348,240 | ||
Entity Filer Category | Large Accelerated Filer | ||
Entity Emerging Growth Company | false | ||
Entity Small Business | false | ||
Entity Shell Company | false | ||
Entity Common Stock, Shares Outstanding | 17,103,395 | ||
City Area Code | 267 | ||
Local Phone Number | 824-2827 | ||
Trading Symbol | MDGL | ||
Security Exchange Name | NASDAQ | ||
Title of 12(b) Security | Common Stock, $0.0001 Par Value Per Share | ||
Entity Address, Address Line One | Four Tower Bridge | ||
Entity Address, Address Line Two | 200 Barr Harbor Drive, Suite 200 | ||
Entity Address, City or Town | West Conshohocken | ||
Entity Address, Postal Zip Code | 19428 | ||
Entity Address, State or Province | PA | ||
ICFR Auditor Attestation Flag | true | ||
Auditor Name | PricewaterhouseCoopers LLP | ||
Auditor Firm ID | 238 | ||
Auditor Location | Philadelphia, Pennsylvania |
Consolidated Balance Sheets
Consolidated Balance Sheets - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Current assets: | ||
Cash and cash equivalents | $ 36,269 | $ 54,004 |
Marketable securities | 234,077 | 230,145 |
Prepaid expenses and other current assets | 1,338 | 1,014 |
Total current assets | 271,684 | 285,163 |
Property and equipment, net | 851 | 1,047 |
Right-of-use asset | 797 | 785 |
Total assets | 273,332 | 286,995 |
Current liabilities: | ||
Accounts payable | 21,380 | 1,017 |
Accrued expenses | 55,048 | 45,222 |
Lease liability | 410 | 318 |
Total current liabilities | 76,838 | 46,557 |
Long-term liabilities: | ||
Lease liability | 387 | 468 |
Total long term liabilities | 387 | 468 |
Total liabilities | 77,225 | 47,025 |
Stockholders' equity: | ||
Preferred stock, par value $0.0001 per share authorized: 5,000,000 shares at December 31, 2020 and December 31, 2019; 1,969,797 shares issued and outstanding at December 31, 2020 and December 31, 2019 | ||
Common stock, par value $0.0001 per share authorized: 200,000,000 at December 31, 2020 and December 31, 2019; 15,508,146 and 15,429,154 shares issued and outstanding at December 31, 2020 and December 31, 2019, respectively | 2 | 2 |
Additional paid-in-capital | 863,495 | 665,385 |
Accumulated other comprehensive gain (loss) | (80) | 47 |
Accumulated deficit | (667,310) | (425,464) |
Total stockholders' equity | 196,107 | 239,970 |
Total liabilities and stockholders' equity | $ 273,332 | $ 286,995 |
Consolidated Balance Sheets (Pa
Consolidated Balance Sheets (Parenthetical) - $ / shares | Dec. 31, 2021 | Dec. 31, 2020 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized | 5,000,000 | 5,000,000 |
Preferred stock, shares issued | 1,969,797 | 1,969,797 |
Preferred stock, shares outstanding | 1,969,797 | 1,969,797 |
Common stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized | 200,000,000 | 200,000,000 |
Common stock, shares issued | 17,103,395 | 15,508,146 |
Common stock, shares outstanding | 17,103,395 | 15,508,146 |
Consolidated Statements of Oper
Consolidated Statements of Operations - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Revenues: | |||
Total revenues | $ 0 | $ 0 | $ 0 |
Operating expenses: | |||
Research and development | 205,164 | 184,809 | 72,324 |
General and administrative | 37,318 | 21,864 | 22,648 |
Total operating expenses | 242,482 | 206,673 | 94,972 |
Loss from operations | (242,482) | (206,673) | (94,972) |
Interest income | 363 | 4,329 | 11,024 |
Other income | 273 | 100 | 0 |
Net loss | $ (241,846) | $ (202,244) | $ (83,948) |
Net loss per common share: | |||
Basic and diluted net loss per common share (in dollars per share) | $ (14.63) | $ (13.09) | $ (5.45) |
Basic and diluted weighted average number of common shares outstanding | 16,535,188 | 15,446,638 | 15,394,659 |
Consolidated Statements of Comp
Consolidated Statements of Comprehensive Loss - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Statement of Comprehensive Income [Abstract] | |||
Net Loss | $ (241,846) | $ (202,244) | $ (83,948) |
Other comprehensive income (loss): | |||
Unrealized gain (loss) on available-for-sale securities | (127) | (169) | 535 |
Comprehensive loss | $ (241,973) | $ (202,413) | $ (83,413) |
Consolidated Statements of Stoc
Consolidated Statements of Stockholders' Equity - USD ($) $ in Thousands | Total | Preferred Stock | Common stock | Additional paid-in Capital | Accumulated other comprehensive income (loss) | Accumulated deficit |
Balance at Dec. 31, 2018 | $ 476,984 | $ 2 | $ 616,573 | $ (319) | $ (139,272) | |
Balance (in shares) at Dec. 31, 2018 | 1,969,797 | 15,409,023 | ||||
Increase (Decrease) in Stockholders' Equity | ||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction cost | 235 | 235 | ||||
Sale of common shares to related parties and exercise of common stock options, net of transaction cost ( in shares) | 20,131 | |||||
Compensation expense related to stock options for services | 22,759 | 22,759 | ||||
Unrealized gain (loss) on marketable securities | 535 | 535 | ||||
Net loss | (83,948) | (83,948) | ||||
Balance at Dec. 31, 2019 | 416,565 | $ 2 | 639,567 | 216 | (223,220) | |
Balance (in shares) at Dec. 31, 2019 | 1,969,797 | 15,429,154 | ||||
Increase (Decrease) in Stockholders' Equity | ||||||
Issuance of common shares in equity offering, excluding to related parties, net of transaction costs | 4,421 | $ 0 | 4,421 | |||
Issuance of common shares in equity offering, excluding to related parties, net of transaction costs (in shares) | 39,607 | |||||
Sale of common shares to related parties and exercise of common stock options, net of transaction cost | 667 | 667 | ||||
Sale of common shares to related parties and exercise of common stock options, net of transaction cost ( in shares) | 39,385 | |||||
Compensation expense related to stock options for services | 20,730 | 20,730 | ||||
Unrealized gain (loss) on marketable securities | (169) | (169) | ||||
Net loss | (202,244) | (202,244) | ||||
Balance at Dec. 31, 2020 | $ 239,970 | $ 2 | 665,385 | 47 | (425,464) | |
Balance (in shares) at Dec. 31, 2020 | 15,508,146 | 1,969,797 | 15,508,146 | |||
Increase (Decrease) in Stockholders' Equity | ||||||
Issuance of common shares in equity offering, excluding to related parties, net of transaction costs | $ 170,207 | 170,207 | ||||
Issuance of common shares in equity offering, excluding to related parties, net of transaction costs (in shares) | 1,584,169 | |||||
Sale of common shares to related parties and exercise of common stock options, net of transaction cost | 1,030 | 1,030 | ||||
Sale of common shares to related parties and exercise of common stock options, net of transaction cost ( in shares) | 11,080 | |||||
Compensation expense related to stock options for services | 26,873 | 26,873 | ||||
Unrealized gain (loss) on marketable securities | (127) | (127) | ||||
Net loss | (241,846) | (241,846) | ||||
Balance at Dec. 31, 2021 | $ 196,107 | $ 2 | $ 863,495 | $ (80) | $ (667,310) | |
Balance (in shares) at Dec. 31, 2021 | 17,103,395 | 1,969,797 | 17,103,395 |
Consolidated Statements of Cash
Consolidated Statements of Cash Flows - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Cash flows from operating activities: | |||
Net loss | $ (241,846) | $ (202,244) | $ (83,948) |
Adjustments to reconcile net loss to net cash used in operating activities: | |||
Stock-based compensation expense | 26,873 | 20,730 | 22,759 |
Depreciation and amortization expense | 405 | 471 | 112 |
Changes in operating assets and liabilities: | |||
Prepaid expenses and other current assets | (325) | 138 | 332 |
Accounts payable | 20,363 | (161) | (1,309) |
Accrued expense | 9,826 | 21,585 | 16,783 |
Accrued interest, net of interest received on maturity of investments | 787 | 1,920 | 3,647 |
Net cash used in operating activities | (183,917) | (157,561) | (41,624) |
Cash flows from investing activities: | |||
Purchases of marketable securities | (394,120) | (329,342) | (619,303) |
Sales and maturities of marketable securities | 389,274 | 489,456 | 650,182 |
Purchases of property and equipment, net of disposals | (209) | (334) | (172) |
Net cash provided by (used in) investing activities | (5,055) | 159,780 | 30,707 |
Cash flows from financing activities: | |||
Proceeds from issuances of stock, excluding related parties, net of transaction costs | 170,207 | 4,421 | |
Proceeds from the sale of related party stock and exercise of common stock options, net of transaction costs | 1,030 | 667 | 235 |
Net cash provided by financing activities | 171,237 | 5,088 | 235 |
Net increase (decrease) in cash and cash equivalents | (17,735) | 7,307 | (10,682) |
Cash and cash equivalents at beginning of period | 54,004 | 46,697 | 57,379 |
Cash and cash equivalents at end of period | 36,269 | 54,004 | 46,697 |
Supplemental disclosure of cash flow information: | |||
Obtaining a right-of-use asset in exchange for a lease liability | $ 376 | $ 451 | 900 |
Purchases of property and equipment in accounts payable at period end | $ 897 |
Organization, Business and Basi
Organization, Business and Basis of Presentation | 12 Months Ended |
Dec. 31, 2021 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization, Business and Basis of Presentation | 1. Organization, Business and Basis of Presentation Organization and Business Madrigal Pharmaceuticals, Inc. (the “Company” or “Madrigal”) is a clinical-stage pharmaceutical company developing novel, high-quality, small-molecule drugs addressing major unmet needs in cardiovascular, metabolic, and liver diseases. The Company’s lead compound, resmetirom, is being advanced for non-alcoholic non-alcoholic MAESTRO-NAFLD-1 |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 2. Summary of Significant Accounting Policies Principle of Consolidation The consolidated financial statements include the financial statements of the Company and its wholly owned subsidiaries. All significant intercompany balances and transactions have been eliminated in consolidation. Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect certain reported amounts of assets and liabilities, and the reported amounts of revenues and expenses during the reporting periods. The Company bases its estimates on historical experience and various other assumptions that management believes to be reasonable under the circumstances. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. Cash and Cash Equivalents The Company considers all highly liquid investments with original maturities of three months or less at the date of purchase to be cash equivalents. The Company maintains its cash in bank accounts, the balance of which, at times, exceeds Federal Deposit Insurance Corporation insured limits. The primary objective of the Company’s investment activities is to preserve its capital for the purpose of funding operations and the Company does not enter into investments for trading or speculative purposes. The Company’s cash is deposited in highly rated financial institutions in the United States. The Company invests in money market funds and high-grade, commercial paper and corporate bonds, which management believes are subject to minimal credit and market risk. Marketable Securities Marketable securities consist of investments in high-grade corporate obligations, and government and government agency obligations that are classified as available-for-sale. The Company adjusts the cost of available-for-sale than-temporary on available-for-sale Marketable securities are stated at fair value, including accrued interest, with their unrealized gains and losses included as a component of accumulated other comprehensive income or loss, which is a separate component of stockholders’ equity. The fair value of these securities is based on quoted prices and observable inputs on a recurring basis. Realized gains and losses are determined on the specific identification method. During the years ended December 31, 2021, 2020 and 2019, the Company did not have any realized gains or losses on marketable securities. Fair Value of Financial Instruments The carrying amounts of the Company’s financial instruments, which include cash equivalents, and marketable securities, approximate their fair values. The fair value of the Company’s financial instruments reflects the amounts that would be received upon sale of an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. The fair value hierarchy has the following three levels: Level 1—quoted prices in active markets for identical assets and liabilities. Level 2—observable inputs other than Level 1 inputs. Examples of Level 2 inputs include quoted prices in active markets for similar assets or liabilities and quoted prices for identical assets or liabilities in markets that are not active. Level 3—unobservable inputs that reflect the Company’s own assumptions about the assumptions market participants would use in pricing the asset or liability. Financial assets and liabilities are classified in their entirety within the fair value hierarchy based on the lowest level of input that is significant to the fair value measurement. The Company measures the fair value of its marketable securities by taking into consideration valuations obtained from third-party pricing sources. The pricing services utilize industry standard valuation models, including both income and market-based approaches, for which all significant inputs are observable, either directly or indirectly, to estimate fair value. These inputs include reported trades of and broker-dealer quotes on the same or similar securities, issuer credit spreads, benchmark securities and other observable inputs. As of December 31, 2021 and 2020, the Company’s financial assets valued based on Level 1 inputs consisted of cash and cash equivalents in a money market fund and its financial assets valued based on Level 2 inputs consisted of high-grade corporate bonds and commercial paper. During the years ended December 31, 2021, 2020 and 2019, the Company did not have any transfers of financials assets between Levels 1 and 2. As of December 31, 2021 and 2020, the Company did not have any financial liabilities that were recorded at fair value on a recurring basis on the balance sheet. Research and Development Costs Research and development costs are expensed as incurred. Research and development costs are comprised of costs incurred in performing research and development activities, including internal costs (including stock-based compensation), costs for consultants, milestone payments under licensing agreements, and other costs associated with the Company’s preclinical and clinical programs. In particular, the Company has conducted safety studies in animals, optimized and implemented the manufacturing of our drug, and conducted Phase 1-3 Patents Costs to secure and defend patents are expensed as incurred and are classified as general and administrative expense in the Company’s statements of operations. Patent expenses were approximately $0.5 million, $0.4 million and $0.4 million for the years ended December 31, 2021, 2020 and 2019, respectively. Stock-Based Compensation The Company recognizes re-evaluated Certain of the employee stock options granted by the Company are structured to qualify as incentive stock options (ISOs). Under current tax regulations, the Company does not receive a tax deduction for the issuance, exercise or disposition of ISOs if the employee meets certain holding requirements. If the employee does not meet the holding requirements, a disqualifying disposition occurs, at which time the Company may receive a tax deduction. The Company does not record tax benefits related to ISOs unless and until a disqualifying disposition is reported. In the event of a disqualifying disposition, the entire tax benefit is recorded as a reduction of income tax expense. The Company has not recognized any income tax benefit for its share-based compensation arrangements due to the fact that the Company does not believe it is more likely than not it will realize the related deferred tax assets. Income Taxes The Company uses the asset and liability method to account for income taxes. Deferred tax assets and liabilities are determined based on the expected future tax consequences of temporary differences between the Company’s financial statement carrying amounts and the tax basis of assets and liabilities using enacted tax rates expected to be in effect in the years in which the differences are expected to reverse. The Company currently maintains a 100% valuation allowance on its deferred tax assets. Comprehensive Loss Comprehensive loss is defined as the change in equity of a business enterprise during a period from transactions and other events and circumstances from non-owner Basic and Diluted Loss Per Common Share Basic net loss per share is computed using the weighted average number of common shares outstanding during the period, excluding restricted stock that has been issued but is not yet vested. Diluted net loss per common share is computed using the weighted average number of common shares outstanding and the weighted average dilutive potential common shares outstanding using the treasury stock method. However, for the years ended December 31, 2021, 2020 and 2019, diluted net loss per share is the same as basic net loss per share as the inclusion of weighted average shares of unvested restricted common stock and common stock issuable upon the exercise of stock options would be anti-dilutive. The following table summarizes outstanding securities not included in the computation of diluted net loss per common share as their inclusion would be anti-dilutive: As of December 31, 2021 2020 2019 Common stock options 2,301,574 1,837,540 1,461,987 Unvested restricted common stock 0 0 0 Preferred stock 1,969,797 1,969,797 1,969,797 Recent Accounting Pronouncements None. |
Liquidity and Uncertainties
Liquidity and Uncertainties | 12 Months Ended |
Dec. 31, 2021 | |
Liquidity and Uncertainties | |
Liquidity and Uncertainties | 3. Liquidity and Uncertainties The Company is subject to risks common to development stage companies in the biopharmaceutical industry including, but not limited to, uncertainty of product development and commercialization, dependence on key personnel, uncertainty of market acceptance of products and product reimbursement, product liability, uncertain protection of proprietary technology, potential inability to raise additional financing necessary for development and commercialization, and compliance with the U.S. Food and Drug Administration and other government regulations. The Company has incurred losses since inception, including approximately $241.8 million for the year ended December 31, 2021, resulting in an accumulated deficit of approximately $667.3 million and $425.5 million as of December 31, 2021 and 2020, respectively. Management expects to incur losses for the foreseeable future. To date, the Company has funded its operations primarily through proceeds from sales of the Company’s capital stock. The Company believes that its cash, cash equivalents and marketable securities at December 31, 2021 will be sufficient to fund operations past one year from the issuance of these financial statements. To meet its future capital needs, the Company intends to raise additional capital through debt or equity financings, collaborations, partnerships or other strategic transactions. However, there can be no assurance that the Company will be able to complete any such transactions on acceptable terms or otherwise. The inability of the Company to obtain sufficient funds on acceptable terms when needed could have a material adverse effect on the Company’s business, results of operations and financial condition. The Company has the ability to delay certain research activities and related clinical expenses if necessary due to liquidity concerns until a date when those concerns are relieved. |
Cash, Cash Equivalents and Mark
Cash, Cash Equivalents and Marketable Securities | 12 Months Ended |
Dec. 31, 2021 | |
Cash and Cash Equivalents [Abstract] | |
Cash, Cash Equivalents and Marketable Securities | 4. Cash, Cash Equivalents and Marketable Securities A summary of cash, cash equivalents and available-for-sale December 31, 2021 Cost Unrealized Unrealized Fair value Cash and cash equivalents: Cash (Level 1) $ 18,877 $ — $ — $ 18,877 Money market funds (Level 1) 17,392 — — 17,392 Total cash and cash equivalents 36,269 — — 36,269 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 228,348 6 (66 ) 228,288 Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) 5,809 — (20 ) 5,789 Total cash, cash equivalents and marketable securities $ 270,426 $ 6 $ (86 ) $ 270,346 December 31, 2020 Cost Unrealized Unrealized Fair value Cash and cash equivalents: Cash (Level 1) $ 716 $ — $ — $ 716 Money market funds (Level 1) 53,288 — — 53,288 Total cash and cash equivalents 54,004 — — 54,004 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 227,172 80 (36 ) 227,216 Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) 2,926 4 (1 ) 2,929 Total cash, cash equivalents and marketable securities $ 284,102 $ 84 $ (37) $ 284,149 |
Accrued Liabilities
Accrued Liabilities | 12 Months Ended |
Dec. 31, 2021 | |
Accrued Liabilities, Current [Abstract] | |
Accrued Liabilities | 5. Accrued Liabilities Accrued liabilities as of December 31, 2021 and 2020 consisted of the following (in thousands): December 31, December 31, Contract research organization costs $ 38,349 $ 31,646 Other clinical study related costs 3,957 3,901 Compensation and benefits 6,769 4,686 Professional fees 2,455 830 Other 3,518 4,159 Total accrued liabilities $ 55,048 $ 45,222 |
Stockholders' Equity
Stockholders' Equity | 12 Months Ended |
Dec. 31, 2021 | |
Stockholders' Equity Note [Abstract] | |
Stockholders' Equity | 6. Stockholders’ Equity Common Stock Each common stockholder is entitled to one vote for each share of common stock held. The common stock will vote together with all other classes and series of stock of the Company as a single class on all actions to be taken by the Company’s stockholders. Each share of common stock is entitled to receive dividends, as and when declared by the Company’s board of directors. The Company has never declared cash dividends on its common stock and does not expect to do so in the foreseeable future. Preferred Stock The Series A Preferred Stock has a par value of $0.0001 per share and is convertible into shares of the common stock at a one-to-one winding-up as-if-converted-to-Common-Stock as-converted At-The-Market In November 2020, the Company entered into an at-the-market “at-the-market” Under the 2020 Sales Agreement the Company sold 1,126,733 shares for an aggregate of approximately $137.4 million in gross proceeds, with net proceeds to the Company of approximately $134.8 million after deducting commissions and other transaction costs. Of those shares sold, 1,087,126 were sold in 2021, and 39,607 were sold in 2020. In June 2021, the Company filed with the SEC and had declared effective a new shelf registration statement on Form S-3 at-the-market As of December 31, 2021, 497,043 shares had been sold under the 2021 Sales Agreement for an aggregate of approximately $40.8 million in gross proceeds, with net proceeds to the Company of approximately $39.8 million after deducting commissions and other transaction costs. As of December 31, 2021, $159.2 million remained reserved and available for sale under the 2021 Sales Agreement and the Company’s related prospectus supplement. |
Stock-based Compensation
Stock-based Compensation | 12 Months Ended |
Dec. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Stock-based Compensation | 7. Stock-based Compensation The 2015 Stock Plan, as amended, is our primary plan through which non-statutory The following table summarizes stock option activity during the twelve months ended December 31, 2021: Shares Weighted Weighted Aggregate Outstanding at January 1, 2021 1,837,540 $ 71.80 Options granted 687,059 109.03 Options exercised (11,080 ) 92.96 Options cancelled (211,945 ) 114.34 Outstanding at December 31, 2021 2,301,574 $ 78.89 6.51 $ 62,566 Exercisable at December 31, 2021 1,432,144 $ 62.66 5.38 $ 60,932 The total cash received by the Company as a result of stock option exercises was $1.0 million, $0.7 million and $0.2 million for the years ended December 31, 2021, 2020, and 2019. The total intrinsic value of options exercised was $0.1 million $4.1 million and $2.2 million for the years ended December 31, 2021, 2020, and 2019. The weighted-average grant date fair values, based on the Black-Scholes option model, of options granted during the year ended December 31, 2021, 2020 and 2019 was $ 73.29, $68.07, and $91.19, respectively. Restricted Common Stock The Company’s share-based compensation plan provides for awards of restricted shares of common stock to employees, officers, directors and consultants to the Company. Restricted stock awards are subject to forfeiture if employment or service terminates during the prescribed retention period. Restricted shares vest over the service period. There were no outstanding restricted shares or activity in 2021. Stock-Based Compensation Expense Stock-based compensation expense during the years ended December 31, 2021, 2020 and 2019 was as follows (in thousands): Years Ended December 31, 2021 2020 2019 Stock-based compensation expense by type of award: Stock options $ 26,873 $ 20,730 $ 22,487 Restricted stock — — 272 Total stock-based compensation expense $ 26,873 $ 20,730 $ 22,759 Effect of stock-based compensation expense by line item: Research and development $ 10,698 $ 8,833 $ 8,277 General and administrative 16,175 11,897 14,482 Total stock-based compensation expense included in net loss $ 26,873 $ 20,730 $ 22,759 Unrecognized stock-based compensation expense on stock options as of December 31, 2021 was $48.9 million with a weighted average remaining period of 2.79 years. |
Leases
Leases | 12 Months Ended |
Dec. 31, 2021 | |
Leases [Abstract] | |
Leases | 8. Leases In 2019, the Company entered into an operating lease for office space, which was renewed and extended in 2020. We adopted ASU 2016-02, right-of-use non-cancelable Future minimum payments under the Company’s operating leases related to the ROU asset and lease liability as of December 31, 2021 was as follows (in thousand): Operating 2022 436 2023 400 Thereafter — Total minimum payments $ 836 Less: imputed interest 39 Present value of lease liabilities $ 797 As of December 31, 2021, the weighted average remaining operating lease term was 1.92 years and the weighted average discount rate used to determine the operating lease liabilities was 3.25%. Cash paid related to the lease liability was $0.4 and $0.3 million for years ended December 31, 2021 and 2020 respectively. Operating lease costs were $0.4 and $0.3 million for years ended December 31, 2021 and 2020 respectively. Rent, short term and variable leases costs were immaterial during the years ended December 31, 2021, 2020 and 2019. |
Commitments and Contingencies
Commitments and Contingencies | 12 Months Ended |
Dec. 31, 2021 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | 9. Commitments and Contingencies The Company has a Research, Development and Commercialization Agreement with Hoffmann-La The agreement requires future milestone payments to Roche. In 2019, the Company commenced a Phase 3 study in Non-Alcoholic The Company has entered into customary contractual arrangements and letters of intent in preparation for and in support of the Phase 3 clinical trials. |
Income Taxes
Income Taxes | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Income Taxes | 10. Income Taxes At December 31, 2021, the Company had federal net operating loss (“NOL”) carryforwards of approximately $168.8 million available to reduce future taxable income, of which $40.4 million will expire between 2031 and 2037. The Company also has state operating loss carryforwards of approximately $157.5 million, available to reduce future taxable income, which expire between 2031 and 2041. The Company has unused federal research and development carryforwards of approximately $22.4 million which will begin to expire in 2031. The Internal Revenue Code (“IRC”) limits the amounts of NOL carryforwards that a Company may use in any one year in the event of certain cumulative changes in ownership over a three-year period as described in Section 382 of the IRC. Such change in ownership could limit the Company’s utilization of the NOL, and could be triggered by subsequent sales of securities by the Company or stockholders. The deferred tax asset related to the NOL reflected on the financial statements could be affected by this limitation. Although a formal analysis has not been completed, the Company has determined that an ownership change likely occurred for Madrigal during the year ended December 31, 2017. In assessing the realizability of deferred tax assets, management considers whether it is more likely than not that some portion of the deferred tax assets will not be realized. The ultimate realization of deferred tax assets is dependent upon the generation of future taxable income during the periods in which those temporary differences become deductible. As there is no assurance of future taxable income, a full valuation allowance has been established to offset the deferred tax assets. The valuation allowance increased $79.3 million for the year ended December 31, 2021. Changes in the deferred tax asset will be recorded as an income tax benefit or expense on the accompanying consolidated statements of operations. Entities are also required to evaluate, measure, recognize and disclose any uncertain income tax provisions taken on their income tax returns. The Company has analyzed its tax positions and has concluded that as of December 31, 2021 there were no uncertain positions. The 2017 through 2021 tax returns are open to review by the IRS and state taxing authorities. Interest and penalties, if any, as they relate to income taxes assessed, are included in the income tax provision. There was no income tax related interest and penalties included in the income tax provision for 2021. Temporary differences that give rise to deferred tax assets and liabilities are as follows (in thousands): For the years ended December 31, 2021 2020 2019 Deferred Tax Liabilities Unrealized gains on investments $ — $ 14 $ 62 Total Deferred Tax Liabilities $ — $ 14 $ 62 Deferred Tax Assets Charitable contributions $ 53 $ 51 $ 15 Accrued expenses 1,857 1,318 759 Intangibles 783 883 983 Stock compensation 24,335 16,812 10,943 Property, plant & equipment 80 68 13 Unrealized loss on investment 23 — — Net operating losses 47,864 27,933 17,635 Capitalized R&D 112,848 71,128 29,364 R&D credit 23,799 14,205 6,141 Total deferred tax assets before valuation allowance 211,642 132,398 65,853 Valuation allowance (211,642 ) (132,384 ) (65,791 ) Total deferred tax assets — 14 62 Net deferred tax assets $ — $ — $ — Differences between the effective income tax rate and the US statutory rate were as follows (in thousands): For the years ended December 31, 2021 2020 2019 Tax benefit at U.S. federal statutory rate $ (50,788 ) $ (42,471 ) $ (17,629 ) Stock based compensation — (68 ) (47 ) Other nondeductible expenses 5 1 14 State income taxes benefit before valuation allowance, net of federal benefit (19,622 ) (16,580 ) (6,613 ) Increase in domestic valuation allowance 79,258 66,593 26,843 Research and development credit (9,002 ) (7,472 ) (2,636 ) Other adjustments 149 (3 ) 68 Income tax expense (benefit) $ — $ — $ — |
Quarterly Financial Data (unaud
Quarterly Financial Data (unaudited) | 12 Months Ended |
Dec. 31, 2021 | |
Quarterly Financial Information Disclosure [Abstract] | |
Quarterly Financial Data (unaudited) | 11. Quarterly Financial Data (unaudited) The following tables present a summary of quarterly results of operations for 2021 and 2020 (in thousands, except shares and per share data): Three months ended March 31, June 30, September 30, December 31, Revenues: Total revenues $ — $ — $ — $ — Operating expenses: Research and development 45,770 51,632 54,873 52,889 General and administrative 7,209 10,110 8,287 11,712 Total operating expenses 52,979 61,742 63,160 64,601 Loss from operations (52,979 ) (61,742 ) (63,160 ) (64,601 ) Interest income 160 91 60 52 Other income 273 — — — Net loss $ (52,546) $ (61,651) $ (63,100) $ (64,549) Net loss per common share: Basic and diluted net loss per common share $ (3.32) $ (3.72) $ (3.79) $ (3.78) Basic and diluted weighted average number of common shares outstanding 15,840,401 16,571,322 16,639,776 17,074,543 Three months ended March 31, June 30, September 30, December 31, Revenues: Total revenues $ — $ — $ — $ — Operating expenses: Research and development 33,400 44,688 53,292 53,429 General and administrative 4,605 5,639 5,494 6,126 Total operating expenses 38,005 50,327 58,786 59,555 Loss from operations (38,005 ) (50,327 ) (58,786 ) (59,555 ) Interest income 1,870 1,204 823 432 Other income — 100 — — Net loss $ (36,135) $ (49,023) $ (57,963) $ (59,123) Net loss per common share: Basic and diluted net loss per common share $ (2.34) $ (3.18) $ (3.75) $ (3.82) Basic and diluted weighted average number of common shares outstanding 15,429,154 15,433,348 15,448,425 15,475,291 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Principle of Consolidation | Principle of Consolidation The consolidated financial statements include the financial statements of the Company and its wholly owned subsidiaries. All significant intercompany balances and transactions have been eliminated in consolidation. |
Use of Estimates | Use of Estimates The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect certain reported amounts of assets and liabilities, and the reported amounts of revenues and expenses during the reporting periods. The Company bases its estimates on historical experience and various other assumptions that management believes to be reasonable under the circumstances. Changes in estimates are recorded in the period in which they become known. Actual results could differ from those estimates. |
Cash and Cash Equivalents | Cash and Cash Equivalents The Company considers all highly liquid investments with original maturities of three months or less at the date of purchase to be cash equivalents. The Company maintains its cash in bank accounts, the balance of which, at times, exceeds Federal Deposit Insurance Corporation insured limits. The primary objective of the Company’s investment activities is to preserve its capital for the purpose of funding operations and the Company does not enter into investments for trading or speculative purposes. The Company’s cash is deposited in highly rated financial institutions in the United States. The Company invests in money market funds and high-grade, commercial paper and corporate bonds, which management believes are subject to minimal credit and market risk. |
Marketable Securities | Marketable Securities Marketable securities consist of investments in high-grade corporate obligations, and government and government agency obligations that are classified as available-for-sale. The Company adjusts the cost of available-for-sale than-temporary on available-for-sale Marketable securities are stated at fair value, including accrued interest, with their unrealized gains and losses included as a component of accumulated other comprehensive income or loss, which is a separate component of stockholders’ equity. The fair value of these securities is based on quoted prices and observable inputs on a recurring basis. Realized gains and losses are determined on the specific identification method. During the years ended December 31, 2021, 2020 and 2019, the Company did not have any realized gains or losses on marketable securities. |
Fair Value of Financial Instruments | Fair Value of Financial Instruments The carrying amounts of the Company’s financial instruments, which include cash equivalents, and marketable securities, approximate their fair values. The fair value of the Company’s financial instruments reflects the amounts that would be received upon sale of an asset or paid to transfer a liability in an orderly transaction between market participants at the measurement date. The fair value hierarchy has the following three levels: Level 1—quoted prices in active markets for identical assets and liabilities. Level 2—observable inputs other than Level 1 inputs. Examples of Level 2 inputs include quoted prices in active markets for similar assets or liabilities and quoted prices for identical assets or liabilities in markets that are not active. Level 3—unobservable inputs that reflect the Company’s own assumptions about the assumptions market participants would use in pricing the asset or liability. Financial assets and liabilities are classified in their entirety within the fair value hierarchy based on the lowest level of input that is significant to the fair value measurement. The Company measures the fair value of its marketable securities by taking into consideration valuations obtained from third-party pricing sources. The pricing services utilize industry standard valuation models, including both income and market-based approaches, for which all significant inputs are observable, either directly or indirectly, to estimate fair value. These inputs include reported trades of and broker-dealer quotes on the same or similar securities, issuer credit spreads, benchmark securities and other observable inputs. As of December 31, 2021 and 2020, the Company’s financial assets valued based on Level 1 inputs consisted of cash and cash equivalents in a money market fund and its financial assets valued based on Level 2 inputs consisted of high-grade corporate bonds and commercial paper. During the years ended December 31, 2021, 2020 and 2019, the Company did not have any transfers of financials assets between Levels 1 and 2. As of December 31, 2021 and 2020, the Company did not have any financial liabilities that were recorded at fair value on a recurring basis on the balance sheet. |
Research and Development Costs | Research and Development Costs Research and development costs are expensed as incurred. Research and development costs are comprised of costs incurred in performing research and development activities, including internal costs (including stock-based compensation), costs for consultants, milestone payments under licensing agreements, and other costs associated with the Company’s preclinical and clinical programs. In particular, the Company has conducted safety studies in animals, optimized and implemented the manufacturing of our drug, and conducted Phase 1-3 |
Patents | Patents Costs to secure and defend patents are expensed as incurred and are classified as general and administrative expense in the Company’s statements of operations. Patent expenses were approximately $0.5 million, $0.4 million and $0.4 million for the years ended December 31, 2021, 2020 and 2019, respectively. |
Stock-Based Compensation | Stock-Based Compensation The Company recognizes re-evaluated Certain of the employee stock options granted by the Company are structured to qualify as incentive stock options (ISOs). Under current tax regulations, the Company does not receive a tax deduction for the issuance, exercise or disposition of ISOs if the employee meets certain holding requirements. If the employee does not meet the holding requirements, a disqualifying disposition occurs, at which time the Company may receive a tax deduction. The Company does not record tax benefits related to ISOs unless and until a disqualifying disposition is reported. In the event of a disqualifying disposition, the entire tax benefit is recorded as a reduction of income tax expense. The Company has not recognized any income tax benefit for its share-based compensation arrangements due to the fact that the Company does not believe it is more likely than not it will realize the related deferred tax assets. |
Income Taxes | Income Taxes The Company uses the asset and liability method to account for income taxes. Deferred tax assets and liabilities are determined based on the expected future tax consequences of temporary differences between the Company’s financial statement carrying amounts and the tax basis of assets and liabilities using enacted tax rates expected to be in effect in the years in which the differences are expected to reverse. The Company currently maintains a 100% valuation allowance on its deferred tax assets. |
Comprehensive Loss | Comprehensive Loss Comprehensive loss is defined as the change in equity of a business enterprise during a period from transactions and other events and circumstances from non-owner |
Basic and Diluted Loss Per Common Share | Basic and Diluted Loss Per Common Share Basic net loss per share is computed using the weighted average number of common shares outstanding during the period, excluding restricted stock that has been issued but is not yet vested. Diluted net loss per common share is computed using the weighted average number of common shares outstanding and the weighted average dilutive potential common shares outstanding using the treasury stock method. However, for the years ended December 31, 2021, 2020 and 2019, diluted net loss per share is the same as basic net loss per share as the inclusion of weighted average shares of unvested restricted common stock and common stock issuable upon the exercise of stock options would be anti-dilutive. The following table summarizes outstanding securities not included in the computation of diluted net loss per common share as their inclusion would be anti-dilutive: As of December 31, 2021 2020 2019 Common stock options 2,301,574 1,837,540 1,461,987 Unvested restricted common stock 0 0 0 Preferred stock 1,969,797 1,969,797 1,969,797 |
Recent Accounting Pronouncements | Recent Accounting Pronouncements None. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Accounting Policies [Abstract] | |
Summary of the outstanding securities not included in the computation of diluted net loss per common share as their inclusion would be anti-dilutive | The following table summarizes outstanding securities not included in the computation of diluted net loss per common share as their inclusion would be anti-dilutive: As of December 31, 2021 2020 2019 Common stock options 2,301,574 1,837,540 1,461,987 Unvested restricted common stock 0 0 0 Preferred stock 1,969,797 1,969,797 1,969,797 |
Cash, Cash Equivalents and Ma_2
Cash, Cash Equivalents and Marketable Securities (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Cash and Cash Equivalents [Abstract] | |
Summary of cash, cash equivalents and available-for-sale marketable securities | A summary of cash, cash equivalents and available-for-sale December 31, 2021 Cost Unrealized Unrealized Fair value Cash and cash equivalents: Cash (Level 1) $ 18,877 $ — $ — $ 18,877 Money market funds (Level 1) 17,392 — — 17,392 Total cash and cash equivalents 36,269 — — 36,269 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 228,348 6 (66 ) 228,288 Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) 5,809 — (20 ) 5,789 Total cash, cash equivalents and marketable securities $ 270,426 $ 6 $ (86 ) $ 270,346 December 31, 2020 Cost Unrealized Unrealized Fair value Cash and cash equivalents: Cash (Level 1) $ 716 $ — $ — $ 716 Money market funds (Level 1) 53,288 — — 53,288 Total cash and cash equivalents 54,004 — — 54,004 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 227,172 80 (36 ) 227,216 Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) 2,926 4 (1 ) 2,929 Total cash, cash equivalents and marketable securities $ 284,102 $ 84 $ (37) $ 284,149 |
Accrued Liabilities (Tables)
Accrued Liabilities (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Accrued Liabilities, Current [Abstract] | |
Schedule of Accrued Liabilities | Accrued liabilities as of December 31, 2021 and 2020 consisted of the following (in thousands): December 31, December 31, Contract research organization costs $ 38,349 $ 31,646 Other clinical study related costs 3,957 3,901 Compensation and benefits 6,769 4,686 Professional fees 2,455 830 Other 3,518 4,159 Total accrued liabilities $ 55,048 $ 45,222 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Share-based Payment Arrangement [Abstract] | |
Summary of stock option activity | The following table summarizes stock option activity during the twelve months ended December 31, 2021: Shares Weighted Weighted Aggregate Outstanding at January 1, 2021 1,837,540 $ 71.80 Options granted 687,059 109.03 Options exercised (11,080 ) 92.96 Options cancelled (211,945 ) 114.34 Outstanding at December 31, 2021 2,301,574 $ 78.89 6.51 $ 62,566 Exercisable at December 31, 2021 1,432,144 $ 62.66 5.38 $ 60,932 |
Schedule of stock-based compensation expense | Stock-based compensation expense during the years ended December 31, 2021, 2020 and 2019 was as follows (in thousands): Years Ended December 31, 2021 2020 2019 Stock-based compensation expense by type of award: Stock options $ 26,873 $ 20,730 $ 22,487 Restricted stock — — 272 Total stock-based compensation expense $ 26,873 $ 20,730 $ 22,759 Effect of stock-based compensation expense by line item: Research and development $ 10,698 $ 8,833 $ 8,277 General and administrative 16,175 11,897 14,482 Total stock-based compensation expense included in net loss $ 26,873 $ 20,730 $ 22,759 |
Leases (Tables)
Leases (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Leases [Abstract] | |
Schedule of future minimum payments for operating leases | Future minimum payments under the Company’s operating leases related to the ROU asset and lease liability as of December 31, 2021 was as follows (in thousand): Operating 2022 436 2023 400 Thereafter — Total minimum payments $ 836 Less: imputed interest 39 Present value of lease liabilities $ 797 |
Income Taxes (Tables)
Income Taxes (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Income Tax Disclosure [Abstract] | |
Schedule of temporary differences that give rise to deferred tax assets and liabilities | Temporary differences that give rise to deferred tax assets and liabilities are as follows (in thousands): For the years ended December 31, 2021 2020 2019 Deferred Tax Liabilities Unrealized gains on investments $ — $ 14 $ 62 Total Deferred Tax Liabilities $ — $ 14 $ 62 Deferred Tax Assets Charitable contributions $ 53 $ 51 $ 15 Accrued expenses 1,857 1,318 759 Intangibles 783 883 983 Stock compensation 24,335 16,812 10,943 Property, plant & equipment 80 68 13 Unrealized loss on investment 23 — — Net operating losses 47,864 27,933 17,635 Capitalized R&D 112,848 71,128 29,364 R&D credit 23,799 14,205 6,141 Total deferred tax assets before valuation allowance 211,642 132,398 65,853 Valuation allowance (211,642 ) (132,384 ) (65,791 ) Total deferred tax assets — 14 62 Net deferred tax assets $ — $ — $ — |
Schedule of differences between the effective income tax rate and the US statutory rate | Differences between the effective income tax rate and the US statutory rate were as follows (in thousands): For the years ended December 31, 2021 2020 2019 Tax benefit at U.S. federal statutory rate $ (50,788 ) $ (42,471 ) $ (17,629 ) Stock based compensation — (68 ) (47 ) Other nondeductible expenses 5 1 14 State income taxes benefit before valuation allowance, net of federal benefit (19,622 ) (16,580 ) (6,613 ) Increase in domestic valuation allowance 79,258 66,593 26,843 Research and development credit (9,002 ) (7,472 ) (2,636 ) Other adjustments 149 (3 ) 68 Income tax expense (benefit) $ — $ — $ — |
Quarterly Financial Data (una_2
Quarterly Financial Data (unaudited) (Tables) | 12 Months Ended |
Dec. 31, 2021 | |
Quarterly Financial Information Disclosure [Abstract] | |
Schedule of quarterly results of operations | The following tables present a summary of quarterly results of operations for 2021 and 2020 (in thousands, except shares and per share data): Three months ended March 31, June 30, September 30, December 31, Revenues: Total revenues $ — $ — $ — $ — Operating expenses: Research and development 45,770 51,632 54,873 52,889 General and administrative 7,209 10,110 8,287 11,712 Total operating expenses 52,979 61,742 63,160 64,601 Loss from operations (52,979 ) (61,742 ) (63,160 ) (64,601 ) Interest income 160 91 60 52 Other income 273 — — — Net loss $ (52,546) $ (61,651) $ (63,100) $ (64,549) Net loss per common share: Basic and diluted net loss per common share $ (3.32) $ (3.72) $ (3.79) $ (3.78) Basic and diluted weighted average number of common shares outstanding 15,840,401 16,571,322 16,639,776 17,074,543 Three months ended March 31, June 30, September 30, December 31, Revenues: Total revenues $ — $ — $ — $ — Operating expenses: Research and development 33,400 44,688 53,292 53,429 General and administrative 4,605 5,639 5,494 6,126 Total operating expenses 38,005 50,327 58,786 59,555 Loss from operations (38,005 ) (50,327 ) (58,786 ) (59,555 ) Interest income 1,870 1,204 823 432 Other income — 100 — — Net loss $ (36,135) $ (49,023) $ (57,963) $ (59,123) Net loss per common share: Basic and diluted net loss per common share $ (2.34) $ (3.18) $ (3.75) $ (3.82) Basic and diluted weighted average number of common shares outstanding 15,429,154 15,433,348 15,448,425 15,475,291 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Patents (Details) - USD ($) $ in Millions | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Accounting Policies [Abstract] | |||
Patent expenses | $ 0.5 | $ 0.4 | $ 0.4 |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Income Taxes (Details) | 12 Months Ended |
Dec. 31, 2021 | |
Income Taxes | |
Valuation allowance on deferred tax assets (as a percent) | 100.00% |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Basic and Diluted Loss Per Common Share (Details) - shares | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Stock options | |||
Anti-dilutive securities | |||
Outstanding securities not included in the computation of diluted net loss per common share as their inclusion would be anti-dilutive (in shares) | 2,301,574 | 1,837,540 | 1,461,987 |
Unvested restricted common stock | |||
Anti-dilutive securities | |||
Outstanding securities not included in the computation of diluted net loss per common share as their inclusion would be anti-dilutive (in shares) | 0 | 0 | 0 |
Preferred Stock | |||
Anti-dilutive securities | |||
Outstanding securities not included in the computation of diluted net loss per common share as their inclusion would be anti-dilutive (in shares) | 1,969,797 | 1,969,797 | 1,969,797 |
Liquidity and Uncertainties (De
Liquidity and Uncertainties (Details) - USD ($) $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2021 | Sep. 30, 2021 | Jun. 30, 2021 | Mar. 31, 2021 | Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Liquidity and Uncertainties | |||||||||||
Net loss | $ (64,549) | $ (63,100) | $ (61,651) | $ (52,546) | $ (59,123) | $ (57,963) | $ (49,023) | $ (36,135) | $ (241,846) | $ (202,244) | $ (83,948) |
Accumulated deficit | $ (667,310) | $ (425,464) | $ (667,310) | $ (425,464) |
Cash, Cash Equivalents and Ma_3
Cash, Cash Equivalents and Marketable Securities (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Cash, Cash Equivalents and Marketable Securities | ||
Total cash, cash equivalents and marketable securities, Unrealized gains | $ 6 | $ 84 |
Total cash, cash equivalents and marketable securities, Unrealized losses | (86) | (37) |
Carrying value | ||
Cash, Cash Equivalents and Marketable Securities | ||
Cash and cash equivalents | 36,269 | 54,004 |
Total cash, cash equivalents and marketable securities, Cost | 270,426 | 284,102 |
Fair value | ||
Cash, Cash Equivalents and Marketable Securities | ||
Cash and cash equivalents | 36,269 | 54,004 |
Total cash, cash equivalents and marketable securities, Fair value | $ 270,346 | $ 284,149 |
Corporate debt securities due within 1 year of date of purchase | ||
Cash, Cash Equivalents and Marketable Securities | ||
Maturity period from date of purchase to classify an investment as marketable securities | 1 year | 1 year |
Corporate debt securities due within 1 year of date of purchase | Level 2 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Marketable securities, unrealized gains | $ 6 | $ 80 |
Marketable securities, unrealized losses | (66) | (36) |
Corporate debt securities due within 1 year of date of purchase | Carrying value | Level 2 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Marketable securities, cost | 228,348 | 227,172 |
Corporate debt securities due within 1 year of date of purchase | Fair value | Level 2 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Marketable securities, fair value | $ 228,288 | $ 227,216 |
Corporate debt securities due within 1 to 2 years of date of purchase | Maximum [Member] | ||
Cash, Cash Equivalents and Marketable Securities | ||
Maturity period from date of purchase to classify an investment as marketable securities | 2 years | 2 years |
Corporate debt securities due within 1 to 2 years of date of purchase | Minimum [Member] | ||
Cash, Cash Equivalents and Marketable Securities | ||
Maturity period from date of purchase to classify an investment as marketable securities | 1 year | 1 year |
Corporate debt securities due within 1 to 2 years of date of purchase | Level 2 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Marketable securities, unrealized gains | $ 4 | |
Marketable securities, unrealized losses | $ (20) | (1) |
Corporate debt securities due within 1 to 2 years of date of purchase | Carrying value | Level 2 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Marketable securities, cost | 5,809 | 2,926 |
Corporate debt securities due within 1 to 2 years of date of purchase | Fair value | Level 2 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Marketable securities, fair value | 5,789 | 2,929 |
Cash | Carrying value | Level 1 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Cash and cash equivalents | 18,877 | 716 |
Cash | Fair value | Level 1 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Cash and cash equivalents | 18,877 | 716 |
Money market funds | Carrying value | Level 1 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Cash and cash equivalents | 17,392 | 53,288 |
Money market funds | Fair value | Level 1 | ||
Cash, Cash Equivalents and Marketable Securities | ||
Cash and cash equivalents | $ 17,392 | $ 53,288 |
Accrued Liabilities (Details)
Accrued Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 |
Accrued Liabilities, Current [Abstract] | ||
Contract research organization costs | $ 38,349 | $ 31,646 |
Other clinical study related costs | 3,957 | 3,901 |
Compensation and benefits | 6,769 | 4,686 |
Professional fees | 2,455 | 830 |
Other | 3,518 | 4,159 |
Total accrued liabilities | $ 55,048 | $ 45,222 |
Stockholders' Equity (Deficit)
Stockholders' Equity (Deficit) - Common Stock (Details) | 12 Months Ended |
Dec. 31, 2021Vote | |
Stockholders' Equity Note [Abstract] | |
Number of votes per share that common stockholders are entitled to receive | 1 |
Stockholders' Equity (Deficit_2
Stockholders' Equity (Deficit) - Preferred Stock (Details) | 1 Months Ended | ||
Jun. 30, 2017Vote$ / sharesshares | Dec. 31, 2021$ / shares | Dec. 31, 2020$ / shares | |
Stockholders' Equity (Deficit) | |||
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 | |
Series A Convertible Preferred Stock | |||
Stockholders' Equity (Deficit) | |||
Preferred stock, par value (in dollars per share) | $ 0.0001 | ||
Preferred stock conversion ratio | shares | 1 | ||
Preferred shares number voting rights | Vote | 0 |
Stockholders' Equity (Deficit_3
Stockholders' Equity (Deficit) - At The Market Issuances (Details) - USD ($) $ in Millions | 1 Months Ended | 12 Months Ended | |
Nov. 30, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | |
Stockholders' Equity (Deficit) | |||
Number of shares sold by the entity | 39,607 | ||
At-The-Market Issuance Sales Agreement | |||
Stockholders' Equity (Deficit) | |||
Number of shares sold by the entity | 1,087,126 | 1,126,733 | |
Value of shares sold by the entity | $ 137.4 | ||
Net proceeds after deducting commissions and other transactions costs | $ 134.8 | ||
At-The-Market Issuance Sales Agreement | 2021 Sales Agreement [Member] | |||
Stockholders' Equity (Deficit) | |||
Number of shares sold by the entity | 497,043 | ||
Value of shares sold by the entity | $ 40.8 | ||
Net proceeds after deducting commissions and other transactions costs | 39.8 | ||
Remaining reserved amount under shelf registration | 159.2 | ||
Cowen & Co. LLC | Maximum | At-The-Market Issuance Sales Agreement | |||
Stockholders' Equity (Deficit) | |||
Maximum aggregate offering price | $ 200 | $ 200 |
Stock-based Compensation - Stoc
Stock-based Compensation - Stock Option (Details) - USD ($) $ / shares in Units, $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Maximum | |||
Stock-based compensation expense | |||
Expiration period | 10 years | ||
Stock options | |||
Stock-based compensation expense | |||
Options outstanding to purchase of common stock | 2,301,574 | 1,837,540 | |
Shares available for future issuance | 1,661,293 | ||
Shares | |||
Outstanding at the beginning of the year (in shares) | 1,837,540 | ||
Options granted (in shares) | 687,059 | ||
Options exercised (in shares) | (11,080) | ||
Options cancelled (in shares) | (211,945) | ||
Outstanding at the end of the year (in shares) | 2,301,574 | 1,837,540 | |
Exercisable at the end of the year (in shares) | 1,432,144 | ||
Weighted average exercise price | |||
Outstanding at the beginning of the year (in dollars per share) | $ 71.80 | ||
Options granted (in dollars per share) | 109.03 | ||
Options exercised (in dollars per share) | 92.96 | ||
Options cancelled (in dollars per share) | 114.34 | ||
Outstanding at the end of the year (in dollars per share) | 78.89 | $ 71.80 | |
Exercisable at the end of the year (in dollars per share) | $ 62.66 | ||
Weighted average remaining contractual life (years) | |||
Outstanding at the end of the year | 6 years 6 months 3 days | ||
Exercisable at the end of the year | 5 years 4 months 17 days | ||
Aggregate intrinsic value | |||
Outstanding at the end of the year | $ 62,566 | ||
Exercisable at the end of the year | 60,932 | ||
Additional disclosures | |||
Total intrinsic value of options exercised | 100 | $ 4,100 | $ 2,200 |
Proceeds resulting from exercise of stock options | $ 1,000 | $ 700 | $ 200 |
Weighted-average grant date fair value of options (in dollars per share) | $ 73.29 | $ 68.07 | $ 91.19 |
Stock-based Compensation - Rest
Stock-based Compensation - Restricted Common Stock (Details) | Dec. 31, 2021shares |
Unvested restricted common stock | |
Shares | |
Outstanding restricted shares | 0 |
Stock-based Compensation - Expe
Stock-based Compensation - Expense (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Stock-based compensation expense | |||
Stock-based compensation expense | $ 26,873 | $ 20,730 | $ 22,759 |
Research and development | |||
Stock-based compensation expense | |||
Stock-based compensation expense | 10,698 | 8,833 | 8,277 |
General and administrative | |||
Stock-based compensation expense | |||
Stock-based compensation expense | 16,175 | 11,897 | 14,482 |
Stock options | |||
Stock-based compensation expense | |||
Stock-based compensation expense | 26,873 | 20,730 | 22,487 |
Unvested restricted common stock | |||
Stock-based compensation expense | |||
Stock-based compensation expense | $ 0 | $ 0 | $ 272 |
Stock-based Compensation - Unre
Stock-based Compensation - Unrecognized Expense (Details) - Stock options $ in Millions | 12 Months Ended |
Dec. 31, 2021USD ($) | |
Unrecognized stock-based compensation expense | |
Unrecognized stock compensation expense | $ 48.9 |
Weighted average remaining period (in years) | 2 years 9 months 14 days |
Leases (Details)
Leases (Details) - USD ($) $ in Thousands | 12 Months Ended | |
Dec. 31, 2021 | Dec. 31, 2020 | |
Operating Leases, Future Minimum Payments Due, Fiscal Year Maturity [Abstract] | ||
2022 | $ 436 | |
2023 | 400 | |
Total minimum payments | 836 | |
Less: imputed interest | 39 | |
Present value of lease liabilities | $ 797 | |
Weighted average, operating lease term | 1 year 11 months 1 day | |
Weighted average, operating discount rate | 3.25% | |
Lease Liability paid in cash | $ 400 | $ 300 |
Operating lease costs | $ 400 | $ 300 |
Commitments and Contingencies (
Commitments and Contingencies (Details) - USD ($) | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Commitments and Contingencies | |||
Licensed product sales | $ 0 | $ 0 | $ 0 |
Research, Development and Commercialization Agreement | Hoffmann-La Roche ("Roche") | |||
Commitments and Contingencies | |||
Remainder of future milestone payments | 8,000,000 | ||
Milestone payment | $ 2,000,000 |
Income Taxes - Net Operating Lo
Income Taxes - Net Operating Loss Carryforwards (Details) $ in Millions | Dec. 31, 2021USD ($) |
Federal | |
Income Taxes | |
Net operating loss | $ 168.8 |
Federal | Expire In 2037 [Member] | |
Income Taxes | |
Net operating loss | 40.4 |
State | |
Income Taxes | |
Net operating loss | $ 157.5 |
Income Taxes - Research and Dev
Income Taxes - Research and Development Carryforward (Details) $ in Millions | Dec. 31, 2021USD ($) |
Federal | Research and development carryforward | |
Income Taxes | |
Research and development tax credit carryforwards | $ 22.4 |
Income Taxes - Valuation Allowa
Income Taxes - Valuation Allowance and Uncertain Tax Position (Details) $ in Millions | 12 Months Ended |
Dec. 31, 2021USD ($) | |
Income Tax Disclosure [Abstract] | |
Increase in valuation allowance | $ 79.3 |
Liability for uncertain tax positions | 0 |
Income tax related interest and penalties | $ 0 |
Income Taxes - Deferred Tax Ass
Income Taxes - Deferred Tax Assets and Liabilities (Details) - USD ($) $ in Thousands | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 |
Deferred Tax Liabilities | |||
Unrealized gains on investments | $ 14 | $ 62 | |
Total Deferred Tax Liabilities | 14 | 62 | |
Deferred Tax Assets | |||
Charitable Contributions | $ 53 | 51 | 15 |
Accrued Expenses | 1,857 | 1,318 | 759 |
Intangibles | 783 | 883 | 983 |
Stock Compensation | 24,335 | 16,812 | 10,943 |
Property, Plant & Equipment | 80 | 68 | 13 |
Unrealized Loss on Investment | 23 | ||
Net Operating Losses | 47,864 | 27,933 | 17,635 |
Capitalized R&D | 112,848 | 71,128 | 29,364 |
R&D Credit | 23,799 | 14,205 | 6,141 |
Total deferred tax assets before valuation allowance | 211,642 | 132,398 | 65,853 |
Valuation Allowance | (211,642) | (132,384) | (65,791) |
Total deferred tax assets | $ 14 | $ 62 | |
Net deferred tax assets | $ 0 |
Income Taxes - Effective Income
Income Taxes - Effective Income Tax Rate (Details) - USD ($) $ in Thousands | 12 Months Ended | ||
Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Effective income tax rate reconciliation | |||
Tax benefit at U.S. federal statutory rate | $ (50,788) | $ (42,471) | $ (17,629) |
Stock based compensation | (68) | (47) | |
Other nondeductible expenses | 5 | 1 | 14 |
State income taxes benefit before valuation allowance, net of federal benefit | (19,622) | (16,580) | (6,613) |
Increase in domestic valuation allowance | 79,258 | 66,593 | 26,843 |
Research and development credit | (9,002) | (7,472) | (2,636) |
Other adjustments | 149 | (3) | 68 |
Income tax expense (benefit) | $ 0 | $ 0 |
Quarterly Financial Data (una_3
Quarterly Financial Data (unaudited) (Details) - USD ($) $ / shares in Units, $ in Thousands | 3 Months Ended | 12 Months Ended | |||||||||
Dec. 31, 2021 | Sep. 30, 2021 | Jun. 30, 2021 | Mar. 31, 2021 | Dec. 31, 2020 | Sep. 30, 2020 | Jun. 30, 2020 | Mar. 31, 2020 | Dec. 31, 2021 | Dec. 31, 2020 | Dec. 31, 2019 | |
Operating expenses: | |||||||||||
Research and development | $ 52,889 | $ 54,873 | $ 51,632 | $ 45,770 | $ 53,429 | $ 53,292 | $ 44,688 | $ 33,400 | $ 205,164 | $ 184,809 | $ 72,324 |
General and administrative | 11,712 | 8,287 | 10,110 | 7,209 | 6,126 | 5,494 | 5,639 | 4,605 | 37,318 | 21,864 | 22,648 |
Total operating expenses | 64,601 | 63,160 | 61,742 | 52,979 | 59,555 | 58,786 | 50,327 | 38,005 | 242,482 | 206,673 | 94,972 |
Loss from operations | (64,601) | (63,160) | (61,742) | (52,979) | (59,555) | (58,786) | (50,327) | (38,005) | (242,482) | (206,673) | (94,972) |
Interest income | 52 | 60 | 91 | 160 | 432 | 823 | 1,204 | 1,870 | |||
Other income | 0 | 0 | 0 | 273 | 0 | 0 | 100 | 0 | 273 | 100 | 0 |
Net loss | $ (64,549) | $ (63,100) | $ (61,651) | $ (52,546) | $ (59,123) | $ (57,963) | $ (49,023) | $ (36,135) | $ (241,846) | $ (202,244) | $ (83,948) |
Basic and diluted net loss per common share (in dollars per share) | $ (3.78) | $ (3.79) | $ (3.72) | $ (3.32) | $ (3.82) | $ (3.75) | $ (3.18) | $ (2.34) | $ (14.63) | $ (13.09) | $ (5.45) |
Basic and diluted weighted average number of common shares outstanding | 17,074,543 | 16,639,776 | 16,571,322 | 15,840,401 | 15,475,291 | 15,448,425 | 15,433,348 | 15,429,154 | 16,535,188 | 15,446,638 | 15,394,659 |