Cover Page
Cover Page - shares | 6 Months Ended | |
Jun. 30, 2024 | Aug. 02, 2024 | |
Cover [Abstract] | ||
Document Type | 10-Q | |
Document Quarterly Report | true | |
Document Period End Date | Jun. 30, 2024 | |
Document Transition Report | false | |
Entity File Number | 001-33277 | |
Entity Registrant Name | MADRIGAL PHARMACEUTICALS, INC. | |
Entity Incorporation, State or Country Code | DE | |
Entity Tax Identification Number | 04-3508648 | |
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, State or Province | PA | |
Entity Address, Postal Zip Code | 19428 | |
City Area Code | 267 | |
Local Phone Number | 824-2827 | |
Title of 12(b) Security | Common Stock, $0.0001 Par Value Per Share | |
Trading Symbol | MDGL | |
Security Exchange Name | NASDAQ | |
Entity Current Reporting Status | Yes | |
Entity Interactive Data Current | Yes | |
Entity Filer Category | Large Accelerated Filer | |
Entity Small Business | false | |
Entity Emerging Growth Company | false | |
Entity Shell Company | false | |
Entity Common Stock, Shares Outstanding | 21,712,677 | |
Amendment Flag | false | |
Document Fiscal Year Focus | 2024 | |
Entity Central Index Key | 0001157601 | |
Current Fiscal Year End Date | --12-31 | |
Document Fiscal Period Focus | Q2 |
CONDENSED CONSOLIDATED BALANCE
CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Current assets: | ||
Cash and cash equivalents | $ 494,597 | $ 99,915 |
Restricted cash | 5,000 | 0 |
Marketable securities | 563,197 | 534,216 |
Trade receivables, net | 6,899 | 0 |
Inventory | 7,072 | 0 |
Prepaid expenses and other current assets | 14,963 | 3,150 |
Total current assets | 1,091,728 | 637,281 |
Property and equipment, net | 1,695 | 1,553 |
Intangible assets, net | 4,910 | 0 |
Right-of-use asset | 1,456 | 1,713 |
Total assets | 1,099,789 | 640,547 |
Current liabilities: | ||
Accounts payable | 8,994 | 28,041 |
Accrued expenses | 115,611 | 89,980 |
Lease liability | 557 | 527 |
Total current liabilities | 125,162 | 118,548 |
Long term liabilities: | ||
Loan payable, net of discount | 116,607 | 115,480 |
Lease liability | 900 | 1,186 |
Total long term liabilities | 117,507 | 116,666 |
Total liabilities | 242,669 | 235,214 |
Stockholders’ equity: | ||
Preferred stock, par value $0.0001 per share authorized: 5,000,000 shares at June 30, 2024 and December 31, 2023; 2,369,797 and 2,369,797 shares issued and outstanding at June 30, 2024 and December 31, 2023, respectively | 0 | 0 |
Common stock, par value $0.0001 per share authorized: 200,000,000 at June 30, 2024 and December 31, 2023; 20,684,663 and 19,875,427 shares issued and outstanding at June 30, 2024 and December 31, 2023, respectively | 2 | 2 |
Additional paid-in-capital | 2,493,288 | 1,741,153 |
Accumulated other comprehensive income (loss) | (368) | 468 |
Accumulated deficit | (1,635,802) | (1,336,290) |
Total stockholders’ equity | 857,120 | 405,333 |
Total liabilities and stockholders’ equity | $ 1,099,789 | $ 640,547 |
CONDENSED CONSOLIDATED BALANC_2
CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares | Jun. 30, 2024 | Dec. 31, 2023 |
Statement of Financial Position [Abstract] | ||
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Preferred stock, shares authorized (in shares) | 5,000,000 | 5,000,000 |
Preferred stock, shares issued (in shares) | 2,369,797 | 2,369,797 |
Preferred stock, shares outstanding (in shares) | 2,369,797 | 2,369,797 |
Common stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Common stock, shares authorized (in shares) | 200,000,000 | 200,000,000 |
Common stock, shares issued (in shares) | 19,875,427 | 21,700,893 |
Common stock, shares outstanding (in shares) | 19,875,427 | 21,700,893 |
CONDENSED CONSOLIDATED STATEMEN
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Revenues: | ||||
Product revenue, net | $ 14,638 | $ 0 | $ 14,638 | $ 0 |
Operating expenses: | ||||
Cost of sales | 636 | 0 | 636 | 0 |
Research and development | 71,091 | 68,605 | 142,328 | 130,759 |
Selling, general and administrative | 105,448 | 17,845 | 186,249 | 34,027 |
Total operating expenses | 177,175 | 86,450 | 329,213 | 164,786 |
Loss from operations | (162,537) | (86,450) | (314,575) | (164,786) |
Interest income | 14,222 | 3,551 | 22,556 | 7,327 |
Interest expense | (3,656) | (2,901) | (7,493) | (5,237) |
Net loss | $ (151,971) | $ (85,800) | $ (299,512) | $ (162,696) |
Net loss per common share: | ||||
Basic net loss per common share (in dollars per share) | $ (7.10) | $ (4.69) | $ (14.47) | $ (8.91) |
Diluted net loss per common share (in dollars per share) | $ (7.10) | $ (4.69) | $ (14.47) | $ (8.91) |
Basic weighted average number of common shares outstanding (in shares) | 21,402,646 | 18,310,952 | 20,702,041 | 18,249,778 |
Diluted weighted average number of common shares outstanding (in shares) | 21,402,646 | 18,310,952 | 20,702,041 | 18,249,778 |
CONDENSED CONSOLIDATED STATEM_2
CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Statement of Comprehensive Income [Abstract] | ||||
Net Loss | $ (151,971) | $ (85,800) | $ (299,512) | $ (162,696) |
Other comprehensive loss: | ||||
Unrealized loss on available-for-sale securities | (196) | (3) | (836) | (57) |
Comprehensive loss | $ (152,167) | $ (85,803) | $ (300,348) | $ (162,753) |
CONDENSED CONSOLIDATED STATEM_3
CONDENSED CONSOLIDATED STATEMENTS OF STOCKHOLDERS’ EQUITY - USD ($) $ in Thousands | Total | Related Party | Preferred stock | Common stock | Common stock Related Party | Additional paid-in Capital | Additional paid-in Capital Related Party | Accumulated other comprehensive income (loss) | Accumulated deficit |
Balance (in shares) at Dec. 31, 2022 | 2,369,797 | 18,102,523 | |||||||
Balance at Dec. 31, 2022 | $ 197,389 | $ 0 | $ 2 | $ 1,160,079 | $ (32) | $ (962,660) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs (in shares) | 180,551 | ||||||||
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, net of transaction costs | $ 17,903 | $ 17,903 | |||||||
Stock-based compensation expense related to equity-classified awards and Compensation expense related to stock options for services | 11,250 | 11,250 | |||||||
Unrealized loss on marketable securities | (54) | (54) | |||||||
Hercules warrant | 544 | 544 | |||||||
Net loss | (76,896) | (76,896) | |||||||
Balance (in shares) at Mar. 31, 2023 | 2,369,797 | 18,283,074 | |||||||
Balance at Mar. 31, 2023 | 150,136 | $ 0 | $ 2 | 1,189,776 | (86) | (1,039,556) | |||
Balance (in shares) at Dec. 31, 2022 | 2,369,797 | 18,102,523 | |||||||
Balance at Dec. 31, 2022 | 197,389 | $ 0 | $ 2 | 1,160,079 | (32) | (962,660) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Unrealized loss on marketable securities | (57) | ||||||||
Net loss | (162,696) | ||||||||
Balance (in shares) at Jun. 30, 2023 | 2,369,797 | 18,459,033 | |||||||
Balance at Jun. 30, 2023 | 103,506 | $ 0 | $ 2 | 1,228,949 | (89) | (1,125,356) | |||
Balance (in shares) at Mar. 31, 2023 | 2,369,797 | 18,283,074 | |||||||
Balance at Mar. 31, 2023 | 150,136 | $ 0 | $ 2 | 1,189,776 | (86) | (1,039,556) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs (in shares) | 85,901 | 90,058 | |||||||
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, net of transaction costs | 21,754 | 6,251 | 21,754 | 6,251 | |||||
Stock-based compensation expense related to equity-classified awards and Compensation expense related to stock options for services | 10,973 | 10,973 | |||||||
Unrealized loss on marketable securities | (3) | (3) | |||||||
Hercules warrant | 195 | 195 | |||||||
Net loss | (85,800) | (85,800) | |||||||
Balance (in shares) at Jun. 30, 2023 | 2,369,797 | 18,459,033 | |||||||
Balance at Jun. 30, 2023 | $ 103,506 | $ 0 | $ 2 | 1,228,949 | (89) | (1,125,356) | |||
Balance (in shares) at Dec. 31, 2023 | 21,700,893 | 2,369,797 | 19,875,427 | ||||||
Balance at Dec. 31, 2023 | $ 405,333 | $ 0 | $ 2 | 1,741,153 | 468 | (1,336,290) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs (in shares) | 750,000 | 59,236 | |||||||
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, net of transaction costs | 311,560 | 262,145 | 311,560 | 262,145 | |||||
Stock-based compensation expense related to equity-classified awards and Compensation expense related to stock options for services | 19,902 | 19,902 | |||||||
Unrealized loss on marketable securities | (640) | (640) | |||||||
Net loss | (147,541) | (147,541) | |||||||
Balance (in shares) at Mar. 31, 2024 | 2,369,797 | 20,684,663 | |||||||
Balance at Mar. 31, 2024 | $ 850,759 | $ 0 | $ 2 | 2,334,760 | (172) | (1,483,831) | |||
Balance (in shares) at Dec. 31, 2023 | 21,700,893 | 2,369,797 | 19,875,427 | ||||||
Balance at Dec. 31, 2023 | $ 405,333 | $ 0 | $ 2 | 1,741,153 | 468 | (1,336,290) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Unrealized loss on marketable securities | (836) | ||||||||
Net loss | $ (299,512) | ||||||||
Balance (in shares) at Jun. 30, 2024 | 19,875,427 | 2,369,797 | 21,700,893 | ||||||
Balance at Jun. 30, 2024 | $ 857,120 | $ 0 | $ 2 | 2,493,288 | (368) | (1,635,802) | |||
Balance (in shares) at Mar. 31, 2024 | 2,369,797 | 20,684,663 | |||||||
Balance at Mar. 31, 2024 | 850,759 | $ 0 | $ 2 | 2,334,760 | (172) | (1,483,831) | |||
Increase (Decrease) in Stockholders' Equity | |||||||||
Sale of common shares to related parties and exercise of common stock options, net of transaction costs (in shares) | 346,153 | 670,077 | |||||||
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, net of transaction costs | 85,950 | $ 48,174 | 85,950 | $ 48,174 | |||||
Stock-based compensation expense related to equity-classified awards and Compensation expense related to stock options for services | 24,404 | 24,404 | |||||||
Unrealized loss on marketable securities | (196) | (196) | |||||||
Net loss | $ (151,971) | (151,971) | |||||||
Balance (in shares) at Jun. 30, 2024 | 19,875,427 | 2,369,797 | 21,700,893 | ||||||
Balance at Jun. 30, 2024 | $ 857,120 | $ 0 | $ 2 | $ 2,493,288 | $ (368) | $ (1,635,802) |
CONDENSED CONSOLIDATED STATEM_4
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
Cash flows from operating activities: | ||
Net loss | $ (299,512) | $ (162,696) |
Adjustments to reconcile net loss to net cash used in operating activities: | ||
Stock-based compensation expense | 44,306 | 22,223 |
Depreciation and amortization expense | 435 | 250 |
Amortization of debt issuance costs and discount | 1,127 | 987 |
Changes in operating assets and liabilities: | ||
Trade receivables, net | (6,899) | 0 |
Prepaid expenses and other current assets | (11,813) | (582) |
Inventory | (7,072) | 0 |
Accounts payable | (19,047) | (6,297) |
Accrued expense | 25,631 | (9,700) |
Accrued interest, net of interest received on maturity of investments | (11,258) | (3,562) |
Net cash used in operating activities | (284,102) | (159,377) |
Cash flows from investing activities: | ||
Purchases of marketable securities | (364,988) | (290,696) |
Sales and maturities of marketable securities | 346,431 | 100,204 |
Acquisition of intangible asset | (5,000) | 0 |
Purchases of property and equipment, net of disposals | (488) | (104) |
Net cash used in investing activities | (24,045) | (190,596) |
Cash flows from financing activities: | ||
Proceeds from issuance of loan payable | 0 | 50,000 |
Payment of debt issuance costs | 0 | (288) |
Net cash provided by financing activities | 707,829 | 95,620 |
Net increase (decrease) in cash, cash equivalents, and restricted cash | 399,682 | (254,353) |
Cash, cash equivalents, and restricted cash at beginning of period | 99,915 | 331,549 |
Cash, cash equivalents, and restricted cash at end of period | 499,597 | 77,196 |
Nonrelated Party | ||
Cash flows from financing activities: | ||
Proceeds from issuance or sale of equity | 397,510 | 21,754 |
Related Party | ||
Cash flows from financing activities: | ||
Proceeds from issuance or sale of equity | $ 310,319 | $ 24,154 |
Organization, Business and Basi
Organization, Business and Basis of Presentation | 6 Months Ended |
Jun. 30, 2024 | |
Organization, Consolidation and Presentation of Financial Statements [Abstract] | |
Organization, Business and Basis of Presentation | Organization, Business, and Basis of Presentation Organization and Business Madrigal Pharmaceuticals, Inc. (the “Company” or “Madrigal”) is a biopharmaceutical company dedicated to transforming care for patients with nonalcoholic steatohepatitis (“NASH”), a serious liver disease that can lead to cirrhosis, liver failure and premature mortality. The Company's medication, Rezdiffra (resmetirom), is a once-daily, oral, liver-directed THR-β agonist designed to target key underlying causes of NASH. In March 2024, Rezdiffra became the first and only FDA-approved therapy for patients with NASH. Rezdiffra became commercially available in the United States in April 2024. Rezdiffra is indicated in conjunction with diet and exercise for the treatment of adults with noncirrhotic NASH with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). Basis of Presentation |
Summary of Significant Accounti
Summary of Significant Accounting Policies | 6 Months Ended |
Jun. 30, 2024 | |
Accounting Policies [Abstract] | |
Summary of Significant Accounting Policies | 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. Revenue Recognition The Company recognizes revenue in accordance with ASC Topic 606 - Revenue from Contracts with Customers. Revenue is recognized at a point in time when the customer obtains control of promised goods or services in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligation(s) in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligation(s) in the contract; and (v) recognize revenue when (or as) the Company satisfies its performance obligation(s). Revenue from product sales is recorded net of adjustments for estimated government-mandated rebates and chargebacks, distribution fees, estimated product returns, and other deductions. Accruals are established for these deductions, and actual amounts incurred are offset against applicable accruals. The Company reflects these accruals as either a reduction in the related account receivable from the customer or as an accrued liability, depending on the means by which the deduction is settled. Sales deductions are based on management’s estimates that involve a substantial degree of judgment. Net Product Revenue On March 14, 2024, the Company announced that the U.S. Food and Drug Administration (“FDA”) granted accelerated approval of Rezdiffra (resmetirom) in conjunction with diet and exercise for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (“NASH”) with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). The Company enters into agreements with specialty pharmacies and specialty distributors, each a “Customer” and collectively the “Customers”, to sell Rezdiffra in the U.S. Revenues from product sales are recognized when the Customer obtains control of the Company’s product, which occurs at a point in time, typically upon delivery to the Customer. Revenue is recorded net of variable consideration, which includes prompt pay discounts, returns, chargebacks, rebates, and co-payment assistance. The variable consideration is estimated based on contractual terms as well as management assumptions. The amount of variable consideration is calculated by using the expected value method, which is the sum of probability-weighted amounts in a range of possible outcomes, or the most likely amount method, which is the single most likely amount in a range of possible outcomes. Estimates are reviewed quarterly and adjusted as necessary. Prompt Pay : Customers receive a 2% prompt pay discount for payments made within a contractually agreed number of days before the due date. The discounts are accounted for as a reduction of the transaction price and recorded as a contra receivable. Returns : The Company records allowances for product returns as a reduction of revenue at the time product sales are recorded. Product returns are estimated based on forecasted sales and historical and industry data. Returns are permitted under certain contractual circumstances, including product expiration date. A returns reserve is recorded as an accrued liability. Chargebacks : Chargebacks are reserved for from eligible healthcare providers, resulting from discounted sales prices offered to qualified customers. The Company is charged back for the difference between the sale price and the discounted price, which is recorded as a contra receivable. Co-Payment Assistance : Co-payment assistant programs are offered to eligible end-users as price concessions and are recorded as accrued liabilities and a reduction of the transaction price. The Company uses a third-party to administer the co-payment program for pharmacy benefit claims. Rebates : Rebates include mandated discounts relating to the Medicaid Drug Rebate Program and Medicare coverage gap rebates. The discount obligations result in decreases to revenue and are recorded to accrued liabilities. The Company generates revenue from a small number of large, reputable customers. The following customers accounted for over 10% of total gross product revenue during three months ended June 30, 2024. As the Company launched Rezdiffra in April 2024, there were no sales and no corresponding customer concentrations in prior periods. Three Months Ended June 30, 2024 2023 Customer A 39 % — % Customer B 19 % — % Customer C 16 % — % Customer D 14 % — % 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. Since these securities are available to fund current operations, they are classified as current assets on the consolidated balance sheets. The Company adjusts the cost of available-for-sale debt securities for amortization of premiums and accretion of discounts to maturity. The Company includes such amortization and accretion as a component of interest income, net. Realized gains and losses and declines in value, if any, that the Company judges to be the result of impairment or as a result of recognizing an allowance for credit losses on available-for-sale securities are reported as a component of interest income. To determine whether an impairment exists, the Company considers whether it intends to sell the debt security and, if the Company does not intend to sell the debt security, it considers available evidence to assess whether it is more likely than not that it will be required to sell the security before the recovery of its amortized cost basis. During the six months ended June 30, 2024 and 2023, the Company determined it did not have any securities that were other-than-temporarily impaired. 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 six months ended June 30, 2024 and 2023, 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 June 30, 2024, the Company’s financial assets valued based on Level 1 inputs consisted of cash and cash equivalents in a money market fund, its financial assets valued based on Level 2 inputs consisted of high-grade corporate and government agency bonds and commercial paper, and it had no financial assets valued based on Level 3 inputs. During the six months ended June 30, 2024 and 2023, the Company did not have any transfers of financial assets between Levels 1 and 2. As of June 30, 2024 and December 31, 2023, the Company did not have any financial liabilities that were recorded at fair value on a recurring basis on the balance sheet. Inventory Inventory is stated at the lower of cost or estimated net realizable value, using actual cost, based on a first-in, first-out (“FIFO”) method. The Company analyzes its inventory levels quarterly and writes down inventory subject to expiry or in excess of expected requirements, or that has a cost basis in excess of its expected net realizable value. These write downs are charged to cost of goods sold in the accompanying Consolidated Statements of Income. The Company considered regulatory approval of its product candidate to be uncertain and product manufactured prior to regulatory approval could not have been sold unless regulatory approval was obtained. As such, the manufacturing costs incurred prior to regulatory approval were not capitalized as inventory, but were expensed as incurred as research and development expenses. 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 its drug, and conducted clinical trials, all of which are considered research and development expenditures. Management uses significant judgment in estimating the amount of research and development costs recognized in each reporting period. Management analyzes and estimates the progress of its clinical trials, completion of milestone events per underlying agreements, invoices received and contracted costs when estimating the research and development costs to accrue in each reporting period. Actual results could differ from the Company’s estimates. Patents Costs to secure and defend patents are expensed as incurred and are classified as selling, general and administrative expense in the Company’s consolidated statements of operations. Intangible Assets Intangible assets with finite lives are amortized to cost of goods sold over their estimated useful lives using the straight-line method. Intangible assets are tested for recoverability whenever events or changes in circumstances indicate that the carrying amounts may not be recoverable. Stock-Based Compensation The Company recognizes stock-based compensation expense based on the grant date fair value of stock options, restricted stock units, and other stock-based compensation awards granted to employees, officers, directors, and consultants. Awards that vest as the recipient provides service are expensed on a straight-line basis over the requisite service period. The Company uses the Black-Scholes option pricing model to determine the grant date fair value of stock options as management believes it is the most appropriate valuation method for its option grants. The Black-Scholes model requires inputs for risk-free interest rate, dividend yield, volatility and expected lives of the options. The expected lives for options granted represent the period of time that options granted are expected to be outstanding. The Company uses the simplified method for determining the expected lives of options. Expected volatility is based upon an industry estimate or blended rate including the Company’s historical trading activity. The risk-free rate for periods within the expected life of the option is based on the U.S. Treasury yield curve in effect at the time of the grant. The Company estimates the forfeiture rate based on historical data. This analysis is re-evaluated at least annually and the forfeiture rate is adjusted as necessary. For other stock-based compensation awards granted to employees and directors that vest based on market conditions, such as the trading price of the Company’s common stock achieving or exceeding certain price targets, the Company uses a Monte Carlo simulation model to estimate the grant date fair value and recognize stock compensation expense over the derived service period. The Monte Carlo simulation model requires key inputs for risk-free interest rate, dividend yield, volatility, and expected life. The assumptions used in computing the fair value of equity awards reflect the Company’s best estimates but involve uncertainties related to market and other conditions. Changes in any of these assumptions may materially affect the fair value of awards granted and the amount of stock-based compensation recognized. 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 sources. Changes in unrealized gains and losses on marketable securities represent the only difference between the Company’s net loss and comprehensive loss. 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. 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 six months ended June 30, 2024 and 2023, diluted net loss per share is the same as basic net loss per share because the inclusion of weighted average shares of common stock issuable upon the exercise of stock options and warrants or vesting of restricted stock units, and common stock issuable upon the conversion of preferred stock 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: Outstanding at June 30, 2024 2023 Common stock options 1,772,807 2,583,540 Restricted stock units 508,947 222,300 Performance-based restricted stock units 235,520 — Preferred stock 2,369,797 2,369,797 Warrants 3,625,244 18,403 Recent Accounting Pronouncements In November 2023, the Financial Accounting Standards Board (“FASB”) issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures, which enhances the disclosures required for operating segments in the Company's annual and interim consolidated financial statements. The amendments are effective for fiscal years beginning after December 15, 2023, and interim periods beginning after December 15, 2024. Early adoption is permitted. The Company is currently evaluating the impact of ASU 2023-07 on its financial statements. In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures, which enhances the disclosures required for income taxes in the Company's annual consolidated financial statements. The amendments are effective for annual periods beginning after December 15, 2024. Early adoption is permitted. The Company is currently evaluating the impact of ASU 2023-09 on its financial statements. |
Liquidity and Uncertainties
Liquidity and Uncertainties | 6 Months Ended |
Jun. 30, 2024 | |
Liquidity and Uncertainties | |
Liquidity and Uncertainties | Liquidity and Uncertainties The Company is subject to risks common to development stage companies and early commercial 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 (“FDA”) and other government regulations. |
Cash, Cash Equivalents and Mark
Cash, Cash Equivalents and Marketable Securities | 6 Months Ended |
Jun. 30, 2024 | |
Cash and Cash Equivalents [Abstract] | |
Cash, Cash Equivalents and Marketable Securities | Cash, Cash Equivalents and Marketable Securities The Company held restricted cash of $5.0 million as of June 30, 2024 as collateral to its corporate credit card program. The Company had no restricted cash as of December 31, 2023. A summary of cash, cash equivalents and available-for-sale marketable securities held by the Company as of June 30, 2024 and December 31, 2023 is as follows (in thousands): June 30, 2024 Cost Unrealized gains Unrealized losses Fair value Cash and cash equivalents: Cash (Level 1) $ 20,323 $ — $ — $ 20,323 Money market funds (Level 1) 476,755 — — 476,755 Corporate debt securities due within 3 months of date of purchase (Level 2) 2,519 — — 2,519 Total cash and cash equivalents 499,597 — — 499,597 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 344,202 5 (224) 343,983 U.S. government and government sponsored entities due within 1 year of date of purchase (Level 2) 219,363 — (149) 219,214 Total cash, cash equivalents, restricted cash, and marketable securities $ 1,063,162 $ 5 $ (373) $ 1,062,794 December 31, 2023 Cost Unrealized gains Unrealized losses Fair value Cash and cash equivalents: Cash (Level 1) $ 2,729 $ — $ — $ 2,729 Money market funds (Level 1) 78,555 — — 78,555 US government and government sponsored entities (Level 1) 14,967 — — 14,967 Corporate debt securities due within 3 months of date of purchase (Level 2) 3,664 — — 3,664 Total cash and cash equivalents 99,915 — — 99,915 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 382,028 195 (7) 382,216 US government and government sponsored entities due within 1 year of date of purchase (Level 2) 150,743 280 (1) 151,022 Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) 977 1 — 978 Total cash, cash equivalents and marketable securities $ 633,663 $ 476 $ (8) $ 634,131 |
Inventory
Inventory | 6 Months Ended |
Jun. 30, 2024 | |
Inventory Disclosure [Abstract] | |
Inventory | Inventory The following table summarizes the Company's inventory balances as of June 30, 2024 and December 31, 2023 (in thousands): June 30, 2024 December 31, 2023 Raw Materials $ — $ — Work In Process 5,728 — Finished Goods 1,344 — Total $ 7,072 $ — |
Accrued Liabilities
Accrued Liabilities | 6 Months Ended |
Jun. 30, 2024 | |
Accrued Liabilities, Current [Abstract] | |
Accrued Liabilities | Accrued Liabilities Accrued liabilities as of June 30, 2024 and December 31, 2023 consisted of the following (in thousands): June 30, December 31, Contract research organization costs $ 65,473 $ 50,737 Other clinical study related costs 4,206 3,724 Manufacturing and drug supply 2,045 9,705 Compensation and benefits 21,341 17,030 Professional fees 15,496 6,814 Gross to net accrued expenses 1,931 — Other 5,119 1,970 Total accrued liabilities $ 115,611 $ 89,980 |
Long Term Debt
Long Term Debt | 6 Months Ended |
Jun. 30, 2024 | |
Debt Disclosure [Abstract] | |
Long Term Debt | Long Term Debt In May 2022, the Company and its wholly-owned subsidiary, Canticle Pharmaceuticals, Inc., entered into the $250.0 million Loan Facility with the several banks and other financial institutions or entities party thereto (each, a “Lender” and collectively referred to as the “Lenders”), and Hercules Capital, Inc. (“Hercules”), in its capacity as administrative agent and collateral agent for itself and the Lenders. Under the terms of the Loan Facility, the first $50.0 million tranche was drawn at closing. The Company also could draw up to an additional $125.0 million in two separate tranches upon achievement of certain resmetirom clinical and regulatory milestones. A fourth tranche of $75.0 million could have been drawn by the Company, subject to the approval of Hercules. The Loan Facility had a minimum interest rate of 7.45% and adjusted with changes in the prime rate. The Company was originally scheduled to pay interest-only monthly payments of accrued interest under the Loan Facility through May 1, 2025, for a period of 36 months. In March 2024, the interest-only period was extended to May 1, 2026 when the Company achieved a milestone when Rezdiffra received FDA approval. The interest only period can further be extended to May 3, 2027, upon the achievement of future revenue milestones, subject to compliance with applicable covenants. The Loan Facility originally matured in May 2026, but the maturity date was extended to May 2027 when the Company achieved a milestone upon receipt of FDA approval in March 2024. The Loan Facility is secured by a security interest in substantially all of the Company’s assets, other than intellectual property. It includes an end of term charge of 5.35% of the aggregate principal amount, which is accounted for in the loan discount. In connection with the first tranche drawn at closing, the Company issued Hercules a warrant to purchase 14,899 shares of Company common stock, which had a Black-Scholes value of $0.6 million. On February 3, 2023, the Company entered into the First Amendment (the “First Amendment”) to the Loan Facility (as amended, the “Amended Loan Facility”). Under the Amended Loan Facility, an additional $35.0 million was drawn under a second, expanded, $65.0 million tranche (“Tranche 2”) in February of 2023 following the Company’s achievement of the Phase 3 clinical development milestone. An additional $15.0 million was drawn under Tranche 2 in June of 2023. The remaining $15.0 million available under Tranche 2 was drawn in September of 2023 in accordance with the Amended Loan Facility. The third tranche (“Tranche 3”) of $75.0 million was unchanged by the First Amendment, and such borrowings became available when the Company achieved a milestone with FDA approval for Rezdiffra in March 2024. The Company did not elect to draw Tranche 3 before it expired in June 2024. Coincident with the expansion of Tranche 2 borrowing capacity by $15.0 million, the Amendment reduced the fourth tranche under the Loan Facility (“Tranche 4”) by $15.0 million to $60.0 million. In connection with the $35.0 million drawn under Tranche 2 at the closing of the First Amendment, $15.0 million drawn in June of 2023, and $15.0 million drawn in September of 2023, the Company issued to Hercules and affiliates Tranche 2 Warrants to purchase an aggregate of 4,555 shares of common stock, which had a Black-Scholes value of $0.9 million. The First Amendment reduced the interest rate under the Amended Loan Facility to the greater of (i) the prime rate as reported in The Wall Street Journal plus 2.45% and (ii) 8.25%. The First Amendment and the Amended Loan Facility summary terms were disclosed in the Company’s Current Report on Form 8-K filed with the Securities and Exchange Commission on February 9, 2023. The Loan Facility includes affirmative and restrictive financial covenants commencing on January 1, 2023, including maintenance of a minimum cash, cash equivalents and liquid funds covenant of $35.0 million, which may decrease in certain circumstances if the Company achieves certain clinical milestones and a revenue milestone, and a revenue-based covenant that could apply commencing at or after the time that financial reporting is due for the quarter ending September 30, 2024. The Loan Facility contains event of default provisions for: the Company’s failure to make required payments or maintain compliance with covenants under the Loan Facility; the Company’s breach of certain representations or default under certain obligations outside the Loan Facility; insolvency, attachment or judgment events affecting the Company; and any circumstance which has occurred or could reasonably be expected to have a material adverse effect on the Company, provided that, any failure to achieve a clinical milestone or approval milestone under the Loan Facility shall not in and of itself constitute a material adverse effect. The Loan Facility also includes customary covenants associated with a secured loan facility, including covenants concerning financial reporting obligations, and certain limitations on indebtedness, liens (including a negative pledge on intellectual property and other assets), investments, distributions (including dividends), collateral, investments, distributions, transfers, mergers or acquisitions, taxes, corporate changes, and deposit accounts. As of June 30, 2024, the outstanding principal under the Loan Facility was $115.0 million. The interest rate as of June 30, 2024 was 10.95%. As of June 30, 2024, the Company was in compliance with all loan covenants and provisions. Future minimum payments, including interest and principal, under the loans payable outstanding as of June 30, 2024 are as follows (in thousands): Period Ending June 30, 2024: Amount 2024 $ 6,400 2025 12,768 2026 79,604 Thereafter 53,387 $ 152,159 Less amount representing interest (31,007) Less unamortized discount (4,545) Loans payable, net of discount $ 116,607 |
Stockholders' Equity
Stockholders' Equity | 6 Months Ended |
Jun. 30, 2024 | |
Equity [Abstract] | |
Stockholders' Equity | 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 “Board”). 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 and B Preferred Stock have a par value of $0.0001 per share and are convertible into shares of the common stock at a one-to-one ratio, subject to adjustment as provided in the Certificates of Designation of Preferences, Rights and Limitations of Series A Preferred Stock and Series B Preferred Stock that the Company filed with the Secretary of State of the State of Delaware on June 21, 2017 and December 22, 2022, respectively. The terms of the Series A and B Preferred Stock are set forth in such Certificates of Designation. Each share of the Series A and B Preferred Stock is convertible into shares of common stock following notice that may be given at the holder’s option. Upon any liquidation, dissolution or winding-up of the Company, whether voluntary or involuntary, after the satisfaction in full of the debts of the Company and the payment of any liquidation preference owed to the holders of shares of capital stock of the Company ranking prior to the Series A and B Preferred Stock upon liquidation, the holders of the Series A and B Preferred Stock shall participate pari passu with the holders of the common stock (on an as-if-converted-to-Common-Stock basis) in the net assets of the Company. Shares of the Series A and B Preferred Stock will generally have no voting rights, except as required by law. Shares of the Series A and B Preferred Stock will be entitled to receive dividends before shares of any other class or series of capital stock of the Company (other than dividends in the form of the common stock) equal to the dividend payable on each share of the common stock, on an as-converted basis. 2024 Public Offering On March 18, 2024, the Company entered into an Underwriting Agreement with Goldman Sachs & Co. LLC, Jefferies LLC, Cowen and Company, LLC, Evercore Group L.L.C. and Piper Sandler & Co, as representatives of the several underwriters named therein (the “2024 Underwriters”), pursuant to which the Company sold to the 2024 Underwriters in an underwritten public offering (the “2024 Offering”): (i) 750,000 shares of common stock at a public offering price of $260.00 per share, (ii) pre-funded warrants (the “2024 Pre-Funded Warrants”) to purchase 1,557,692 shares of common stock at a public offering price of $259.9999 per 2024 Pre-Funded Warrant, which represents the per share public offering price for the common stock less a $0.0001 per share exercise price for each such Pre-Funded Warrant, and (iii) a 30-day option for the 2024 Underwriters to purchase up to 346,153 additional shares of common stock at the public offering price of $260.00 per share (the “Underwriters’ Option”). The 2024 Offering closed on March 21, 2024. The gross proceeds of the 2024 Offering was $600.0 million, and the Company received net proceeds, after deducting the underwriting discount and commissions and other estimated offering expenses payable by the Company, of approximately $574.0 million. The Underwriters’ Option was later exercised in full, and closed on April 2, 2024. The net proceeds to the Company for the exercise of the Underwriters’ Option, after deducting the underwriting discounts and commissions and estimated offering expenses payable by the Company, was approximately $85.9 million. The Company intends to use the net proceeds from the 2024 Offering for its commercial activities in connection with the launch of Rezdiffra in the U.S. and for general corporate purposes, including, without limitation, research and development expenditures, ongoing clinical trial expenditures, manufacture and supply of drug substance and drug products, potential ex-U.S. commercialization or partnering opportunities, potential acquisitions or licensing of new technologies, capital expenditures and working capital. The 2024 Pre-Funded Warrants are exercisable at any time after the date of issuance. A holder of 2024 Pre-Funded Warrants may not exercise the warrant if the holder, together with its affiliates, would beneficially own more than 9.99% of the number of shares of common stock outstanding immediately after giving effect to such exercise. A holder of 2024 Pre-Funded Warrants may increase or decrease this percentage, but not in excess of 19.99%, by providing at least 61 days prior notice to the Company. 2023 Public Offering On September 28, 2023, the Company entered into an Underwriting Agreement with Goldman Sachs & Co. LLC, as representative of the several underwriters named therein (the “2023 Underwriters”), pursuant to which the Company sold to the 2023 Underwriters in an underwritten public offering (the “2023 Offering”): (i) 1,248,098 shares of common stock at a public offering price of $151.69 per share, and (ii) pre-funded warrants (the “2023 Pre-Funded Warrants”) to purchase 2,048,098 shares of common stock at a public offering price of $151.6899 per 2023 Pre-Funded Warrant, which represents the per share public offering price for the common stock less a $0.0001 per share exercise price for each such 2023 Pre-Funded Warrant. The 2023 Offering closed on October 3, 2023. The gross proceeds of the 2023 Offering was $500.0 million , and the Company received net proceeds, after deducting the underwriting discount and commissions and other estimated offering expenses payable by the Company, of approximately $472.0 million . The Company intends to use t he net proceeds from the Offering for its clinical and commercial activities in preparation for the launch of resmetirom in the U.S. and for general corporate purposes, including, without limitation, research and development expenditures, clinical trial expenditures, manufacture and supply of drug substance and drug products, potential acquisitions or licensing of new technologies, capital expenditures and working capital. The 2023 Pre-Funded Warrants are exercisable at any time afte r the date of issuance. A holder of 2023 Pre-Funded Warrants may not exercise the warrant if the holder, together with its affiliates, would beneficially own more than 9.99% of the number of shares of common stock outstanding immediately after giving effect to such exercise. A holder of 2023 Pre-Funded Warrants may increase or decrease this percentage, but not in excess of 19.99% , by providing at least 61 days prior notice to the Company. At-The-Market Issuance Sales Agreement In June 2021, the Company entered into an at-the-market sales agreement (the “2021 Sales Agreement”) with Cowen and Company, LLC (“Cowen”), pursuant to which the Company could, from time to time, issue and sell shares of its common stock. The 2021 Sales Agreement initially authorized an aggregate offering of up to $200.0 million in shares of the Company’s common stock, at the Company’s option, through Cowen as its sales agent. Sales of common stock through Cowen could be made by any method that is deemed an “at-the-market” offering as defined in Rule 415 promulgated under the Securities Act of 1933, as amended, including by means of ordinary brokers’ transactions at market prices, in block transactions or as otherwise agreed by the Company and Cowen. Subject to the terms and conditions of the 2021 Sales Agreement, Cowen would use commercially reasonable efforts consistent with its normal trading and sales practices to sell the common stock based upon the Company’s instructions (including any price, time or size limits or other customary parameters or conditions the Company imposed). In May 2023, the Company entered into an amendment to the 2021 Sales Agreement (the “Sales Agreement Amendment”), with Cowen, pursuant to which the Company could, from time to time, issue and sell an additional $200.0 million in shares of its common stock, until it was terminated in May 2024. The Company was not obligated to make any sales of its common stock under this arrangement. Any shares sold would be sold pursuant to the Registration Statement and prospectus supplement filed pursuant to the Registration Statement. The 2021 Sales Agreement, as amended by the Sales Agreement Amendment, authorized sales of shares of the Company’s common stock, from time to time, at the Company’s option, through Cowen as its sales agent. Sales of common stock through Cowen may be made by any method that is deemed an “at-the-market” offering as defined in Rule 415 promulgated under the Securities Act of 1933, as amended, and as described in the prospectus supplement. Since the entry into the Sales Agreement Amendment in May 2023, the Company sold 98,101 shares in total under the 2021 Sales Agreement, as amended by the Sales Agreement Amendment, for an aggregate of $25.2 million in gross proceeds, with net proceeds to the Company of approximately $24.5 million after deducting commissions and other transaction costs. All shares were sold pursuant to the Company’s effective Registration Statement and the prospectus supplement relating thereto. In total, the Company sold 1,334,044 shares of Common Stock having an aggregate offering price of $225.1 million pursuant to the 2021 Sales Agreement, as amended by the Sales Agreement Amendment. In May 2024, the Company entered into a Sales Agreement (the “2024 Sales Agreement”) with Cowen, replacing and superseding the 2021 Sales Agreement, as amended by the Sales Agreement Amendment which was terminated effective upon the entry into the 2024 Sales Agreement. The Company is authorized to issue and sell up to $300.0 million in shares of the Company’s common stock under the 2024 Sales Agreement. The Company sold no shares in the three and six months ended June 30, 2024 under either the 2021 Sales Agreement, as amended by the Sales Agreement Amendment, or the 2024 Sales Agreement. |
Stock-based Compensation
Stock-based Compensation | 6 Months Ended |
Jun. 30, 2024 | |
Share-Based Payment Arrangement [Abstract] | |
Stock-based Compensation | Stock-based Compensation 2015 Stock Plan The Company’s 2015 Stock Plan, as amended (the “2015 Stock Plan”), is one of the Company’s equity incentive compensation plans through which equity based grants are awarded. The 2015 Stock Plan provides for the grant of incentive stock options, non-statutory stock options, restricted stock, restricted stock units and other stock-based compensation awards to employees, officers, directors, and consultants of the Company. The administration of the 2015 Stock Plan is under the general supervision of the Compensation Committee of the Board. The terms of stock options awarded under the 2015 Stock Plan, in general, are determined by the Compensation Committee, provided the exercise price per share generally shall not be set at less than the fair market value of a share of the common stock on the date of grant and the term shall not be greater than ten years from the date the option is granted. On June 25, 2024, the Company’s stockholders approved an amendment to the 2015 Stock Plan to increase the total number of shares of common stock available for issuance by 750,000 and extend its duration by 10 years until April 23, 2035. As of June 30, 2024, 1,264,199 shares were available for future issuance under the 2015 Stock Plan. 2023 Inducement Plan In September 2023, the Company adopted the 2023 Inducement Plan (the “Inducement Plan”), pursuant to which the Company may from time to time make equity grants to new employees as a material inducement to their employment. The Inducement Plan was adopted without stockholder approval, pursuant to Nasdaq Listing Rule 5635(c)(4), and is administered by the Compensation Committee of the Board. The Inducement Plan provides for the granting of non-statutory stock options, restricted stock, restricted stock units, performance stock units and other stock-based compensation awards to new employees, but does not allow for the granting of incentive stock options. The terms of the stock options under the Inducement Plan, in general, are determined by the Compensation Committee, provided the exercise price per share generally shall not be set at less than the fair market value of a share of the common stock on the date of grant and the term shall not be greater than ten years from the date the option or award is granted. A total of 500,000 shares of the Company’s common stock were reserved for issuance under the Inducement Plan. As of June 30, 2024, 19,097 shares were available for future issuance under the 2023 Inducement Plan. Stock Options The following table summarizes stock option activity during the six months ended June 30, 2024: Shares Weighted average exercise price Outstanding at December 31, 2023 2,355,779 $ 79.94 Options granted 148,970 232.88 Options exercised (671,947) 73.91 Options cancelled (59,995) 118.98 Outstanding at June 30, 2024 1,772,807 $ 93.76 Exercisable at June 30, 2024 1,323,994 $ 79.14 The total cash received by the Company as a result of stock option exercises was $49.7 million and $21.8 million, respectively, for the six months ended June 30, 2024 and 2023. The total intrinsic value of options exercised was $121.1 million and $48.9 million, respectively, for the six months ended June 30, 2024 and 2023. The weighted-average grant date fair values, based on the Black-Scholes option model, of options granted during the six months ended June 30, 2024 and 2023 were $153.98 and $219.02, respectively. Restricted Stock Units The Company’s 2015 Stock Plan provides for awards of restricted stock units (“RSUs”) to employees, officers, directors and consultants to the Company. The Company’s Inducement Plan provides for awards of RSUs to new employees. RSUs vest over a period of months or years, or upon the occurrence of certain performance criteria or the attainment of stated goals or events, and are subject to forfeiture if employment or service terminates before vesting. As of June 30, 2024, the Company had 508,947 restricted stock units outstanding, with a weighted average grant date fair value of $230.61 per unit. The following table summarizes RSU activity, excluding performance-based RSUs, during the six months ended June 30, 2024: Shares Weighted average grant date fair value Outstanding at December 31, 2023 376,117 $ 241.45 RSUs granted 248,517 231.96 RSUs vested (63,825) 289.45 RSUs forfeited (51,862) 243.28 Outstanding at June 30, 2024 508,947 $ 230.61 Performance-Based Restricted Stock Units The Company has granted various performance-based restricted stock units (“PSUs”) to certain members of senior leadership. Depending on the terms of the PSUs and the outcome of the pre-established performance criteria, which may include a market and/or performance condition, a recipient may ultimately earn the target number of PSUs granted or a specified multiple thereof at the end of the vesting period. The following table summarizes PSU activity during the six months ended June 30, 2024: PSUs Eligible to Earn PSUs Weighted average grant date fair value Outstanding PSUs at December 31, 2023 50,000 150,000 $ 146.37 PSUs granted 51,202 102,404 388.02 PSUs attained — — — PSUs forfeited (8,442) (16,884) 388.02 Outstanding at June 30, 2024 92,760 235,520 $ 257.77 Outstanding Awards As of June 30, 2024, the Company had restricted stock units, performance stock units, and options outstanding pursuant to which an aggregate of 2,517,274 shares of its common stock may be issued pursuant to the terms of all awards granted under the 2015 Stock Plan and Inducement Plan. Stock-Based Compensation Expense Stock-based compensation expense during the three and six months ended June 30, 2024 and 2023 was as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Stock-based compensation expense by type of award: Stock options $ 10,027 $ 7,431 $ 16,913 $ 16,029 Restricted stock units 9,918 3,542 18,543 6,194 Performance-based restricted stock units 4,459 — 8,850 — Total stock-based compensation expense $ 24,404 $ 10,973 $ 44,306 $ 22,223 Effect of stock-based compensation expense by line item: Research and development $ 5,582 $ 5,184 $ 11,489 $ 10,570 Selling, general and administrative 18,822 5,789 32,817 11,653 Total stock-based compensation expense included in net loss $ 24,404 $ 10,973 $ 44,306 $ 22,223 |
Commitments and Contingencies
Commitments and Contingencies | 6 Months Ended |
Jun. 30, 2024 | |
Commitments and Contingencies Disclosure [Abstract] | |
Commitments and Contingencies | Commitments and Contingencies The Company has a Research, Development and Commercialization Agreement with Hoffmann-La Roche (“Roche”) which grants the Company a sole and exclusive license to develop, use, sell, offer for sale and import any Licensed Product as defined by the agreement. The agreement requires future milestone payments to Roche. In March 2024, upon receiving FDA approval of Rezdiffra, a milestone was achieved and $5.0 million became due to Roche. Remaining milestones under the agreement total $3.0 million and are payable upon the Company achieving specified objectives related to future regulatory approval in Europe of resmetirom or a product developed from resmetirom. Furthermore, a tiered single-digit royalty is payable on net sales of resmetirom or a product developed from resmetirom, subject to certain reductions. The Company launched Rezdiffra in the U.S. in April 2024. The company did not achieve any product development or regulatory milestones for the six months ended June 30, 2023. The Company has entered into customary contractual arrangements and letters of intent in preparation for and in support of clinical trials and commercialization. In August 2023, the Company entered into the Fifth Amendment to the Office Lease (the “Fifth Lease Amendment”). The Lease Amendment extended the term of the lease through November 2026. As a result of the Lease Amendment, an incremental $1.6 million right-of-use asset and lease liabilities were recorded during the year ended December 31, 2023. In April 2024 and May 2024, the Company entered into the Sixth (the “Sixth Lease Amendment”) and Seventh (the “Seventh Lease Amendment”) Amendments to the Office Lease, respectively, expanding the amount of office space available in the same premises. The lease commencement date had not yet occurred as of June 30, 2024 and therefore there was no impact to the financial statements. |
Subsequent Events
Subsequent Events | 6 Months Ended |
Jun. 30, 2024 | |
Subsequent Events [Abstract] | |
Subsequent Events | Subsequent Events None. |
Pay vs Performance Disclosure
Pay vs Performance Disclosure - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||||
Jun. 30, 2024 | Mar. 31, 2024 | Jun. 30, 2023 | Mar. 31, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Pay vs Performance Disclosure | ||||||
Net loss | $ (151,971) | $ (147,541) | $ (85,800) | $ (76,896) | $ (299,512) | $ (162,696) |
Insider Trading Arrangements
Insider Trading Arrangements | 3 Months Ended | 6 Months Ended |
Jun. 30, 2024 shares | Jun. 30, 2024 shares | |
Trading Arrangements, by Individual | ||
Material Terms of Trading Arrangement | The following table shows the number of shares of our common stock subject to the current Rule 10b5-1 trading arrangements of our Section 16 officers and directors in place as of August 2, 2024: Name of Director or Section 16 Officer Title of Director or Section 16 Officer Date of Adoption, Modification, or Termination Duration of the Plan Aggregate Number of Shares of Common Stock that may be Sold under the Plan Richard Levy, MD Director 11/30/2023 June 16, 2025 15,000 Fred B. Craves, Ph.D. Director 12/15/2023 August 31, 2024 30,000 | |
Non-Rule 10b5-1 Arrangement Adopted | false | |
Rule 10b5-1 Arrangement Terminated | false | |
Non-Rule 10b5-1 Arrangement Terminated | false | |
Fred B. Craves [Member] | ||
Trading Arrangements, by Individual | ||
Name | Fred B. Craves, Ph.D. | |
Title | Director | |
Rule 10b5-1 Arrangement Adopted | true | |
Adoption Date | 12/15/2023 | |
Arrangement Duration | 260 days | |
Aggregate Available | 30,000 | 30,000 |
Richard Levy [Member] | ||
Trading Arrangements, by Individual | ||
Name | Richard Levy, MD | |
Title | Director | |
Rule 10b5-1 Arrangement Adopted | true | |
Adoption Date | 11/30/2023 | |
Arrangement Duration | 564 days | |
Aggregate Available | 15,000 | 15,000 |
Summary of Significant Accoun_2
Summary of Significant Accounting Policies (Policies) | 6 Months Ended |
Jun. 30, 2024 | |
Accounting Policies [Abstract] | |
Principle of Consolidation | Principle of Consolidation |
Use of Estimates | Use of Estimates |
Revenue Recognition | Revenue Recognition The Company recognizes revenue in accordance with ASC Topic 606 - Revenue from Contracts with Customers. Revenue is recognized at a point in time when the customer obtains control of promised goods or services in an amount that reflects the consideration to which the entity expects to be entitled in exchange for those goods or services. To determine revenue recognition for arrangements that the Company determines are within the scope of ASC 606, the Company performs the following five steps: (i) identify the contract(s) with a customer; (ii) identify the performance obligation(s) in the contract; (iii) determine the transaction price; (iv) allocate the transaction price to the performance obligation(s) in the contract; and (v) recognize revenue when (or as) the Company satisfies its performance obligation(s). Revenue from product sales is recorded net of adjustments for estimated government-mandated rebates and chargebacks, distribution fees, estimated product returns, and other deductions. Accruals are established for these deductions, and actual amounts incurred are offset against applicable accruals. The Company reflects these accruals as either a reduction in the related account receivable from the customer or as an accrued liability, depending on the means by which the deduction is settled. Sales deductions are based on management’s estimates that involve a substantial degree of judgment. Net Product Revenue On March 14, 2024, the Company announced that the U.S. Food and Drug Administration (“FDA”) granted accelerated approval of Rezdiffra (resmetirom) in conjunction with diet and exercise for the treatment of adults with noncirrhotic nonalcoholic steatohepatitis (“NASH”) with moderate to advanced liver fibrosis (consistent with stages F2 to F3 fibrosis). The Company enters into agreements with specialty pharmacies and specialty distributors, each a “Customer” and collectively the “Customers”, to sell Rezdiffra in the U.S. Revenues from product sales are recognized when the Customer obtains control of the Company’s product, which occurs at a point in time, typically upon delivery to the Customer. Revenue is recorded net of variable consideration, which includes prompt pay discounts, returns, chargebacks, rebates, and co-payment assistance. The variable consideration is estimated based on contractual terms as well as management assumptions. The amount of variable consideration is calculated by using the expected value method, which is the sum of probability-weighted amounts in a range of possible outcomes, or the most likely amount method, which is the single most likely amount in a range of possible outcomes. Estimates are reviewed quarterly and adjusted as necessary. Prompt Pay : Customers receive a 2% prompt pay discount for payments made within a contractually agreed number of days before the due date. The discounts are accounted for as a reduction of the transaction price and recorded as a contra receivable. Returns : The Company records allowances for product returns as a reduction of revenue at the time product sales are recorded. Product returns are estimated based on forecasted sales and historical and industry data. Returns are permitted under certain contractual circumstances, including product expiration date. A returns reserve is recorded as an accrued liability. Chargebacks : Chargebacks are reserved for from eligible healthcare providers, resulting from discounted sales prices offered to qualified customers. The Company is charged back for the difference between the sale price and the discounted price, which is recorded as a contra receivable. Co-Payment Assistance : Co-payment assistant programs are offered to eligible end-users as price concessions and are recorded as accrued liabilities and a reduction of the transaction price. The Company uses a third-party to administer the co-payment program for pharmacy benefit claims. Rebates |
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 |
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. Since these securities are available to fund current operations, they are classified as current assets on the consolidated balance sheets. |
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. |
Inventory | Inventory Inventory is stated at the lower of cost or estimated net realizable value, using actual cost, based on a first-in, first-out (“FIFO”) method. The Company analyzes its inventory levels quarterly and writes down inventory subject to expiry or in excess of expected requirements, or that has a cost basis in excess of its expected net realizable value. These write downs are charged to cost of goods sold in the accompanying Consolidated Statements of Income. |
Research and Development Costs | Research and Development Costs |
Patents | Patents |
Intangible Assets | Intangible Assets Intangible assets with finite lives are amortized to cost of goods sold over their estimated useful lives using the straight-line method. Intangible assets are tested for recoverability whenever events or changes in circumstances indicate that the carrying amounts may not be recoverable. |
Stock-Based Compensation | Stock-Based Compensation The Company recognizes stock-based compensation expense based on the grant date fair value of stock options, restricted stock units, and other stock-based compensation awards granted to employees, officers, directors, and consultants. Awards that vest as the recipient provides service are expensed on a straight-line basis over the requisite service period. The Company uses the Black-Scholes option pricing model to determine the grant date fair value of stock options as management believes it is the most appropriate valuation method for its option grants. The Black-Scholes model requires inputs for risk-free interest rate, dividend yield, volatility and expected lives of the options. The expected lives for options granted represent the period of time that options granted are expected to be outstanding. The Company uses the simplified method for determining the expected lives of options. Expected volatility is based upon an industry estimate or blended rate including the Company’s historical trading activity. The risk-free rate for periods within the expected life of the option is based on the U.S. Treasury yield curve in effect at the time of the grant. The Company estimates the forfeiture rate based on historical data. This analysis is re-evaluated at least annually and the forfeiture rate is adjusted as necessary. For other stock-based compensation awards granted to employees and directors that vest based on market conditions, such as the trading price of the Company’s common stock achieving or exceeding certain price targets, the Company uses a Monte Carlo simulation model to estimate the grant date fair value and recognize stock compensation expense over the derived service period. The Monte Carlo simulation model requires key inputs for risk-free interest rate, dividend yield, volatility, and expected life. The assumptions used in computing the fair value of equity awards reflect the Company’s best estimates but involve uncertainties related to market and other conditions. Changes in any of these assumptions may materially affect the fair value of awards granted and the amount of stock-based compensation recognized. 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 |
Comprehensive Loss | Comprehensive Loss |
Basic and Diluted Loss Per Common Share | Basic and Diluted Loss Per Common Share |
Recent Accounting Pronouncements | Recent Accounting Pronouncements In November 2023, the Financial Accounting Standards Board (“FASB”) issued ASU 2023-07, Segment Reporting (Topic 280): Improvements to Reportable Segment Disclosures, which enhances the disclosures required for operating segments in the Company's annual and interim consolidated financial statements. The amendments are effective for fiscal years beginning after December 15, 2023, and interim periods beginning after December 15, 2024. Early adoption is permitted. The Company is currently evaluating the impact of ASU 2023-07 on its financial statements. In December 2023, the FASB issued ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures, which enhances the disclosures required for income taxes in the Company's annual consolidated financial statements. The amendments are effective for annual periods beginning after December 15, 2024. Early adoption is permitted. The Company is currently evaluating the impact of ASU 2023-09 on its financial statements. |
Summary of Significant Accoun_3
Summary of Significant Accounting Policies (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Accounting Policies [Abstract] | |
Schedule 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: Outstanding at June 30, 2024 2023 Common stock options 1,772,807 2,583,540 Restricted stock units 508,947 222,300 Performance-based restricted stock units 235,520 — Preferred stock 2,369,797 2,369,797 Warrants 3,625,244 18,403 |
Schedules of Concentration of Risk, by Risk Factor | The following customers accounted for over 10% of total gross product revenue during three months ended June 30, 2024. As the Company launched Rezdiffra in April 2024, there were no sales and no corresponding customer concentrations in prior periods. Three Months Ended June 30, 2024 2023 Customer A 39 % — % Customer B 19 % — % Customer C 16 % — % Customer D 14 % — % |
Cash, Cash Equivalents and Ma_2
Cash, Cash Equivalents and Marketable Securities (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Cash and Cash Equivalents [Abstract] | |
Schedule of cash, cash equivalents and available-for-sale marketable securities | A summary of cash, cash equivalents and available-for-sale marketable securities held by the Company as of June 30, 2024 and December 31, 2023 is as follows (in thousands): June 30, 2024 Cost Unrealized gains Unrealized losses Fair value Cash and cash equivalents: Cash (Level 1) $ 20,323 $ — $ — $ 20,323 Money market funds (Level 1) 476,755 — — 476,755 Corporate debt securities due within 3 months of date of purchase (Level 2) 2,519 — — 2,519 Total cash and cash equivalents 499,597 — — 499,597 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 344,202 5 (224) 343,983 U.S. government and government sponsored entities due within 1 year of date of purchase (Level 2) 219,363 — (149) 219,214 Total cash, cash equivalents, restricted cash, and marketable securities $ 1,063,162 $ 5 $ (373) $ 1,062,794 December 31, 2023 Cost Unrealized gains Unrealized losses Fair value Cash and cash equivalents: Cash (Level 1) $ 2,729 $ — $ — $ 2,729 Money market funds (Level 1) 78,555 — — 78,555 US government and government sponsored entities (Level 1) 14,967 — — 14,967 Corporate debt securities due within 3 months of date of purchase (Level 2) 3,664 — — 3,664 Total cash and cash equivalents 99,915 — — 99,915 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 382,028 195 (7) 382,216 US government and government sponsored entities due within 1 year of date of purchase (Level 2) 150,743 280 (1) 151,022 Corporate debt securities due within 1 to 2 years of date of purchase (Level 2) 977 1 — 978 Total cash, cash equivalents and marketable securities $ 633,663 $ 476 $ (8) $ 634,131 |
Inventory (Tables)
Inventory (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Inventory Disclosure [Abstract] | |
Schedule of Inventory Balances | The following table summarizes the Company's inventory balances as of June 30, 2024 and December 31, 2023 (in thousands): June 30, 2024 December 31, 2023 Raw Materials $ — $ — Work In Process 5,728 — Finished Goods 1,344 — Total $ 7,072 $ — |
Accrued Liabilities (Tables)
Accrued Liabilities (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Accrued Liabilities, Current [Abstract] | |
Schedule of Accrued Liabilities | Accrued liabilities as of June 30, 2024 and December 31, 2023 consisted of the following (in thousands): June 30, December 31, Contract research organization costs $ 65,473 $ 50,737 Other clinical study related costs 4,206 3,724 Manufacturing and drug supply 2,045 9,705 Compensation and benefits 21,341 17,030 Professional fees 15,496 6,814 Gross to net accrued expenses 1,931 — Other 5,119 1,970 Total accrued liabilities $ 115,611 $ 89,980 |
Long Term Debt (Tables)
Long Term Debt (Tables) | 6 Months Ended |
Jun. 30, 2024 | |
Debt Disclosure [Abstract] | |
Schedule of Maturities of Long-Term Debt | Future minimum payments, including interest and principal, under the loans payable outstanding as of June 30, 2024 are as follows (in thousands): Period Ending June 30, 2024: Amount 2024 $ 6,400 2025 12,768 2026 79,604 Thereafter 53,387 $ 152,159 Less amount representing interest (31,007) Less unamortized discount (4,545) Loans payable, net of discount $ 116,607 |
Stock-based Compensation (Table
Stock-based Compensation (Tables) | 3 Months Ended | 6 Months Ended |
Jun. 30, 2024 | Jun. 30, 2024 | |
Share-Based Payment Arrangement [Abstract] | ||
Schedule of stock option activity | Stock-based compensation expense during the three and six months ended June 30, 2024 and 2023 was as follows (in thousands): Three Months Ended June 30, Six Months Ended June 30, 2024 2023 2024 2023 Stock-based compensation expense by type of award: Stock options $ 10,027 $ 7,431 $ 16,913 $ 16,029 Restricted stock units 9,918 3,542 18,543 6,194 Performance-based restricted stock units 4,459 — 8,850 — Total stock-based compensation expense $ 24,404 $ 10,973 $ 44,306 $ 22,223 Effect of stock-based compensation expense by line item: Research and development $ 5,582 $ 5,184 $ 11,489 $ 10,570 Selling, general and administrative 18,822 5,789 32,817 11,653 Total stock-based compensation expense included in net loss $ 24,404 $ 10,973 $ 44,306 $ 22,223 | The following table summarizes stock option activity during the six months ended June 30, 2024: Shares Weighted average exercise price Outstanding at December 31, 2023 2,355,779 $ 79.94 Options granted 148,970 232.88 Options exercised (671,947) 73.91 Options cancelled (59,995) 118.98 Outstanding at June 30, 2024 1,772,807 $ 93.76 Exercisable at June 30, 2024 1,323,994 $ 79.14 |
Schedule of RSU activity | The following table summarizes RSU activity, excluding performance-based RSUs, during the six months ended June 30, 2024: Shares Weighted average grant date fair value Outstanding at December 31, 2023 376,117 $ 241.45 RSUs granted 248,517 231.96 RSUs vested (63,825) 289.45 RSUs forfeited (51,862) 243.28 Outstanding at June 30, 2024 508,947 $ 230.61 | |
Schedule of PSU activity | The following table summarizes PSU activity during the six months ended June 30, 2024: PSUs Eligible to Earn PSUs Weighted average grant date fair value Outstanding PSUs at December 31, 2023 50,000 150,000 $ 146.37 PSUs granted 51,202 102,404 388.02 PSUs attained — — — PSUs forfeited (8,442) (16,884) 388.02 Outstanding at June 30, 2024 92,760 235,520 $ 257.77 |
Summary of Significant Accoun_4
Summary of Significant Accounting Policies - Schedules of Concentration of Risk, by Risk Factor (Details) - Revenue from Contract with Customer, Product and Service Benchmark - Customer Concentration Risk | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
Customer A | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 39% | 0% |
Customer B | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 19% | 0% |
Customer C | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 16% | 0% |
Customer D | ||
Concentration Risk [Line Items] | ||
Concentration risk, percentage | 14% | 0% |
Summary of Significant Accoun_5
Summary of Significant Accounting Policies - Revenue Recognition (Details) | Jun. 30, 2024 |
Accounting Policies [Abstract] | |
Prompt payment percentage | 2% |
Summary of Significant Accoun_6
Summary of Significant Accounting Policies - Marketable Securities (Details) - USD ($) | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
Accounting Policies [Abstract] | ||
Marketable securities, realized gain (loss) | $ 0 | $ 0 |
Summary of Significant Accoun_7
Summary of Significant Accounting Policies - Income Taxes (Details) | 6 Months Ended |
Jun. 30, 2024 | |
Income Taxes | |
Valuation allowance on deferred tax assets (as a percent) | 100% |
Summary of Significant Accoun_8
Summary of Significant Accounting Policies - Basic and Diluted Loss Per Common Share (Details) - shares | 6 Months Ended | |
Jun. 30, 2024 | Jun. 30, 2023 | |
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) | 1,772,807 | 2,583,540 |
Restricted stock units | ||
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) | 508,947 | 222,300 |
Performance-based restricted stock units | ||
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) | 235,520 | 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) | 2,369,797 | 2,369,797 |
Warrants | ||
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) | 3,625,244 | 18,403 |
Liquidity and Uncertainties (De
Liquidity and Uncertainties (Details) - USD ($) $ in Thousands | 1 Months Ended | 3 Months Ended | 6 Months Ended | ||||||
Apr. 30, 2024 | Mar. 31, 2024 | Jun. 30, 2024 | Mar. 31, 2024 | Jun. 30, 2023 | Mar. 31, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | Dec. 31, 2023 | |
Liquidity and Uncertainties | |||||||||
Net loss | $ (151,971) | $ (147,541) | $ (85,800) | $ (76,896) | $ (299,512) | $ (162,696) | |||
Accumulated deficit | $ (1,635,802) | $ (1,635,802) | $ (1,336,290) | ||||||
Cash received on stock transaction after deduction of issuance costs | $ 85,900 | $ 574,000 |
Cash, Cash Equivalents and Ma_3
Cash, Cash Equivalents and Marketable Securities - Narrative (Details) - USD ($) | Jun. 30, 2024 | Dec. 31, 2023 |
Cash and Cash Equivalents [Abstract] | ||
Restricted cash | $ 5,000,000 | $ 0 |
Cash, Cash Equivalents and Ma_4
Cash, Cash Equivalents and Marketable Securities - Schedule of cash, cash equivalents and available-for-sale marketable securities (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Cash and cash equivalents: | ||
Total cash and cash equivalents | $ 499,597 | $ 99,915 |
Marketable securities: | ||
Unrealized gains | 5 | 476 |
Unrealized losses | (373) | (8) |
Cost | 1,063,162 | 633,663 |
Fair value | 1,062,794 | 634,131 |
Corporate debt securities | ||
Cash and cash equivalents: | ||
Total cash and cash equivalents | 2,519 | 3,664 |
Marketable securities: | ||
Cost | 219,363 | 382,028 |
Unrealized gains | 0 | 195 |
Unrealized losses | (149) | (7) |
Fair value | 219,214 | 382,216 |
Corporate debt securities due within 1 to 2 years of date of purchase | ||
Marketable securities: | ||
Cost | 977 | |
Unrealized gains | 1 | |
Unrealized losses | 0 | |
Fair value | 978 | |
US government and government sponsored entities | ||
Cash and cash equivalents: | ||
Total cash and cash equivalents | 14,967 | |
U.S. government and government sponsored entities due within 1 year of date of purchase | ||
Marketable securities: | ||
Cost | 344,202 | 150,743 |
Unrealized gains | 5 | 280 |
Unrealized losses | (224) | (1) |
Fair value | 343,983 | 151,022 |
Cash | ||
Cash and cash equivalents: | ||
Total cash and cash equivalents | 20,323 | 2,729 |
Money market funds | ||
Cash and cash equivalents: | ||
Total cash and cash equivalents | $ 476,755 | $ 78,555 |
Inventory (Details)
Inventory (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Inventory Disclosure [Abstract] | ||
Raw Materials | $ 0 | $ 0 |
Work In Process | 5,728 | 0 |
Finished Goods | 1,344 | 0 |
Total | $ 7,072 | $ 0 |
Accrued Liabilities (Details)
Accrued Liabilities (Details) - USD ($) $ in Thousands | Jun. 30, 2024 | Dec. 31, 2023 |
Accrued Liabilities, Current [Abstract] | ||
Contract research organization costs | $ 65,473 | $ 50,737 |
Other clinical study related costs | 4,206 | 3,724 |
Manufacturing and drug supply | 2,045 | 9,705 |
Compensation and benefits | 21,341 | 17,030 |
Professional fees | 15,496 | 6,814 |
Gross to net accrued expenses | 1,931 | 0 |
Other | 5,119 | 1,970 |
Total accrued liabilities | $ 115,611 | $ 89,980 |
Long Term Debt - Narrative (Det
Long Term Debt - Narrative (Details) | 1 Months Ended | |||||||
Feb. 03, 2023 USD ($) | Sep. 30, 2023 USD ($) shares | Jun. 30, 2023 USD ($) | Feb. 28, 2023 USD ($) | May 31, 2022 USD ($) tranche | Jun. 30, 2024 USD ($) | Jan. 01, 2023 USD ($) | May 01, 2022 USD ($) shares | |
Debt Instrument [Line Items] | ||||||||
Number of warrants issued for purchase of common shares (in shares) | shares | 14,899 | |||||||
Warrants and rights outstanding | $ 900,000 | $ 600,000 | ||||||
Loans payable, net of discount | $ 116,607,000 | |||||||
Hercules Warrants | ||||||||
Debt Instrument [Line Items] | ||||||||
Number of warrants issued for purchase of common shares (in shares) | shares | 4,555 | |||||||
Secured Debt | Line of Credit | ||||||||
Debt Instrument [Line Items] | ||||||||
Term charges on aggregate principal amount, percentage | 5.35% | |||||||
Secured Debt | Loan Facility | Line of Credit | ||||||||
Debt Instrument [Line Items] | ||||||||
Debt instrument, face amount | $ 250,000,000 | |||||||
Line of credit facility, number of withdrawals upon milestone achievements | tranche | 2 | |||||||
Long term debt floor interest rate percentage | 7.45% | |||||||
Accrued interest payments, term | 36 months | |||||||
Minimum liquidity covenant | $ 35,000,000 | |||||||
Loans payable, net of discount | $ 115,000,000 | |||||||
Line of credit facility, interest rate | 10.95% | |||||||
Secured Debt | Loan Facility | Line of Credit | Variable Rate Component One | Prime Rate | ||||||||
Debt Instrument [Line Items] | ||||||||
Basis spread on variable rate | 2.45% | |||||||
Secured Debt | Loan Facility | Line of Credit | Variable Rate Component Two | Prime Rate | ||||||||
Debt Instrument [Line Items] | ||||||||
Basis spread on variable rate | 8.25% | |||||||
Secured Debt | Loan Facility, Tranche 1 | Line of Credit | ||||||||
Debt Instrument [Line Items] | ||||||||
Proceeds from issuance of debt | $ 50,000,000 | |||||||
Secured Debt | Loan Facility, Tranches 2 and 3 | Line of Credit | ||||||||
Debt Instrument [Line Items] | ||||||||
Line of credit facility, maximum borrowing capacity | 125,000,000 | |||||||
Secured Debt | Loan Facility, Tranche 2 | Line of Credit | ||||||||
Debt Instrument [Line Items] | ||||||||
Proceeds from issuance of debt | $ 35,000,000 | $ 15,000,000 | $ 15,000,000 | |||||
Line of credit facility, maximum borrowing capacity | $ 65,000,000 | |||||||
Line of credit facility, increase In maximum borrowing capacity | $ 15,000,000 | |||||||
Secured Debt | Loan Facility, Tranche 3 | Line of Credit | ||||||||
Debt Instrument [Line Items] | ||||||||
Line of credit facility, remaining borrowing capacity | $ 75,000,000 | |||||||
Secured Debt | Loan Facility, Tranche 4 | Line of Credit | ||||||||
Debt Instrument [Line Items] | ||||||||
Line of credit facility, maximum borrowing capacity | 60,000,000 | $ 75,000,000 | ||||||
Line of credit facility, decrease In maximum borrowing capacity | $ (15,000,000) |
Long Term Debt - Schedule of Ma
Long Term Debt - Schedule of Maturities of Long-Term Debt (Details) $ in Thousands | 6 Months Ended |
Jun. 30, 2024 USD ($) | |
Debt Disclosure [Abstract] | |
2024 | $ 6,400 |
2025 | 12,768 |
2026 | 79,604 |
Thereafter | 53,387 |
Long-Term Debt | 152,159 |
Less amount representing interest | (31,007) |
Less unamortized discount | (4,545) |
Loans payable, net of discount | $ 116,607 |
Stockholders' Equity- Common St
Stockholders' Equity- Common Stock (Details) | 6 Months Ended |
Jun. 30, 2024 vote | |
Equity [Abstract] | |
Number of votes per share that common stockholders are entitled to receive | 1 |
Stockholders' Equity - Preferre
Stockholders' Equity - Preferred Stock (Details) | Jun. 30, 2024 vote $ / shares | Dec. 31, 2023 $ / shares |
Stockholders' Equity (Deficit) | ||
Preferred stock, par value (in dollars per share) | $ 0.0001 | $ 0.0001 |
Convertible Series A and B Preferred Stock | ||
Stockholders' Equity (Deficit) | ||
Preferred stock, par value (in dollars per share) | $ 0.0001 | |
Preferred stock conversion ratio (in shares) | 1 | |
Preferred shares number voting rights | vote | 0 |
Stockholders' Equity - Public O
Stockholders' Equity - Public Offering (Details) $ / shares in Units, $ in Millions | 1 Months Ended | ||||||
Apr. 02, 2024 USD ($) | Mar. 20, 2024 USD ($) $ / shares shares | Mar. 18, 2024 shares | Oct. 03, 2023 USD ($) $ / shares shares | Apr. 30, 2024 USD ($) | Mar. 31, 2024 USD ($) | May 01, 2022 shares | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Number of warrants issued for purchase of common shares (in shares) | shares | 14,899 | ||||||
Cash received on stock transaction after deduction of issuance costs | $ | $ 85.9 | $ 574 | |||||
Pre-funded warrants | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Beneficial ownership threshold percentage | 0.0999 | 0.0999 | |||||
Beneficial ownership maximum percentage | 0.1999 | 0.1999 | |||||
Beneficial ownership notice period | 61 days | 61 days | |||||
2024 Public Stock Offering | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Number of shares issued in transaction (in shares) | shares | 750,000 | ||||||
Shares issued (in USD per share) | $ / shares | $ 260 | ||||||
Sale of stock received on transaction, gross | $ | $ 600 | ||||||
Cash received on stock transaction after deduction of issuance costs | $ | $ 574 | ||||||
2024 Public Stock Offering | Pre-funded warrants | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Number of warrants issued for purchase of common shares (in shares) | shares | 1,557,692 | ||||||
Class of warrant or right, exercise price of warrants or rights (in USD per share) | $ / shares | $ 259.9999 | ||||||
Stock price discount per share (in USD per share) | $ / shares | $ 0.0001 | ||||||
Over-Allotment Option | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Number of shares issued in transaction (in shares) | shares | 346,153 | ||||||
Purchasing period | 30 days | ||||||
Cash received on stock transaction after deduction of issuance costs | $ | $ 85.9 | ||||||
2023 Public Stock Offering | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Number of shares issued in transaction (in shares) | shares | 1,248,098 | ||||||
Shares issued (in USD per share) | $ / shares | $ 151.69 | ||||||
Sale of stock received on transaction, gross | $ | $ 500 | ||||||
Cash received on stock transaction after deduction of issuance costs | $ | $ 472 | ||||||
2023 Public Stock Offering | Pre-funded warrants | |||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||
Number of warrants issued for purchase of common shares (in shares) | shares | 2,048,098 | ||||||
Class of warrant or right, exercise price of warrants or rights (in USD per share) | $ / shares | $ 151.6899 | ||||||
Stock price discount per share (in USD per share) | $ / shares | $ 0.0001 |
Stockholders' Equity - At The M
Stockholders' Equity - At The Market Issuances (Details) - USD ($) $ in Millions | 1 Months Ended | 3 Months Ended | 6 Months Ended | 14 Months Ended | 36 Months Ended | 37 Months Ended | |||
Apr. 30, 2024 | Mar. 31, 2024 | May 31, 2023 | Jun. 30, 2021 | Jun. 30, 2024 | Jun. 30, 2024 | Jun. 30, 2024 | May 07, 2024 | May 31, 2024 | |
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Cash received on stock transaction after deduction of issuance costs | $ 85.9 | $ 574 | |||||||
2021 Sales Agreement | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Maximum aggregate offering price | $ 225.1 | ||||||||
Number of shares issued in transaction (in shares) | 98,101 | ||||||||
Sale of stock received on transaction, gross | $ 25.2 | ||||||||
Cash received on stock transaction after deduction of issuance costs | $ 24.5 | ||||||||
2021 Sales Agreement | Common stock | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Number of shares issued in transaction (in shares) | 1,334,044 | ||||||||
2024 Sales Agreement | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Authorized capital | $ 300 | ||||||||
2024 Sales Agreement And Sales Agreement Amendment | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Number of shares issued in transaction (in shares) | 0 | 0 | |||||||
Cowen & Co. LLC | Maximum | 2021 Sales Agreement | |||||||||
Collaborative Arrangement and Arrangement Other than Collaborative [Line Items] | |||||||||
Maximum aggregate offering price | $ 200 | $ 200 |
Stock-based Compensation - Narr
Stock-based Compensation - Narrative (Details) - USD ($) $ / shares in Units, $ in Millions | 1 Months Ended | 6 Months Ended | |||
Jun. 25, 2024 | Sep. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | Dec. 31, 2023 | |
Unrecognized stock-based compensation expense | |||||
Unrecognized stock compensation expense | $ 149.1 | ||||
Weighted average remaining period (in years) | 2 years 1 month 2 days | ||||
2015 Stock Plan | |||||
Unrecognized stock-based compensation expense | |||||
Expiration period | 10 years | ||||
Shares available for future issuance (in shares) | 750,000 | 1,264,199 | |||
2023 stock inducement plan | |||||
Unrecognized stock-based compensation expense | |||||
Expiration period | 10 years | ||||
Shares available for future issuance (in shares) | 19,097 | ||||
Common stock, capital shares reserved for future issuance (in shares) | 500,000 | ||||
2015 Stock And Inducement Plan | |||||
Unrecognized stock-based compensation expense | |||||
Common stock granted and issuable (in shares) | 2,517,274 | ||||
Stock options | |||||
Unrecognized stock-based compensation expense | |||||
Proceeds resulting from exercise of stock options | $ 49.7 | $ 21.8 | |||
Total intrinsic value of options exercised | $ 121.1 | $ 48.9 | |||
Weighted-average grant date fair value of options (in dollars per share) | $ 153.98 | $ 219.02 | |||
Stock options | 2015 Stock Plan | |||||
Unrecognized stock-based compensation expense | |||||
Expiration period | 10 years | ||||
Restricted stock units | |||||
Unrecognized stock-based compensation expense | |||||
Equity award other than options, nonvested (in shares) | 508,947 | 376,117 | |||
Weighted average grant date fair value | $ 230.61 | $ 241.45 | |||
Granted (in shares) | 248,517 | ||||
Restricted stock units | 2015 Stock Plan | |||||
Unrecognized stock-based compensation expense | |||||
Equity award other than options, nonvested (in shares) | 508,947 | ||||
Weighted average grant date fair value | $ 230.61 | ||||
Performance-based restricted stock units | |||||
Unrecognized stock-based compensation expense | |||||
Equity award other than options, nonvested (in shares) | 92,760 | 50,000 | |||
Weighted average grant date fair value | $ 257.77 | $ 146.37 | |||
Granted (in shares) | 51,202 |
Stock-based Compensation - Stoc
Stock-based Compensation - Stock Option (Details) | 6 Months Ended |
Jun. 30, 2024 $ / shares shares | |
Shares | |
Outstanding at the beginning of the year (in shares) | shares | 2,355,779 |
Options granted (in shares) | shares | 148,970 |
Options exercised (in shares) | shares | (671,947) |
Options cancelled (in shares) | shares | (59,995) |
Outstanding at the ending of the year (in shares) | shares | 1,772,807 |
Exercisable at the ending of the year (in shares) | shares | 1,323,994 |
Weighted average exercise price | |
Outstanding at the beginning of the year (in dollars per share) | $ / shares | $ 79.94 |
Options granted (in dollars per share) | $ / shares | 232.88 |
Options exercised (in dollars per share) | $ / shares | 73.91 |
Options cancelled (in dollars per share) | $ / shares | 118.98 |
Outstanding at the ending of the year (in dollars per share) | $ / shares | 93.76 |
Exercisable at the ending of the year (in dollars per share) | $ / shares | $ 79.14 |
Stock-based Compensation - Rest
Stock-based Compensation - Restricted Stock Units (Details) - Restricted stock units | 6 Months Ended |
Jun. 30, 2024 $ / shares shares | |
Shares | |
Outstanding, beginning balance (in shares) | shares | 376,117 |
Granted (in shares) | shares | 248,517 |
Vested (in shares) | shares | (63,825) |
Forfeited (in shares) | shares | (51,862) |
Outstanding, ending balance (in shares) | shares | 508,947 |
Weighted average grant date fair value | |
Unvested, beginning balance (in USD per share) | $ / shares | $ 241.45 |
Granted (in USD per share) | $ / shares | 231.96 |
Vested (in USD per share) | $ / shares | 289.45 |
Forfeited (in USD per share) | $ / shares | 243.28 |
Unvested, ending balance (in USD per share) | $ / shares | $ 230.61 |
Stock-based Compensation - Perf
Stock-based Compensation - Performance-Based Restricted Stock Units (Details) | 6 Months Ended |
Jun. 30, 2024 $ / shares shares | |
Performance-based restricted stock units | |
Shares | |
Outstanding, beginning balance (in shares) | 50,000 |
PSUs granted (in shares) | 51,202 |
PSUs attained (in shares) | 0 |
PSUs forfeited (in shares) | (8,442) |
Outstanding, ending balance (in shares) | 92,760 |
Weighted average grant date fair value | |
Unvested, beginning balance (in USD per share) | $ / shares | $ 146.37 |
PSUs granted (in USD per share) | $ / shares | 388.02 |
PSUs attained (in USD per share) | $ / shares | 0 |
PSUs forfeited (in USD per share) | $ / shares | 388.02 |
Unvested, ending balance (in USD per share) | $ / shares | $ 257.77 |
Eligible to Earn PSUs | |
Shares | |
Outstanding, beginning balance (in shares) | 150,000 |
PSUs granted (in shares) | 102,404 |
PSUs attained (in shares) | 0 |
PSUs forfeited (in shares) | (16,884) |
Outstanding, ending balance (in shares) | 235,520 |
Stock-based Compensation - St_2
Stock-based Compensation - Stock-Based Compensation Expense (Details) - USD ($) $ in Thousands | 3 Months Ended | 6 Months Ended | ||
Jun. 30, 2024 | Jun. 30, 2023 | Jun. 30, 2024 | Jun. 30, 2023 | |
Stock-based compensation expense | ||||
Stock-based compensation expense | $ 24,404 | $ 10,973 | $ 44,306 | $ 22,223 |
Research and development | ||||
Stock-based compensation expense | ||||
Stock-based compensation expense | 5,582 | 5,184 | 11,489 | 10,570 |
Selling, general and administrative | ||||
Stock-based compensation expense | ||||
Stock-based compensation expense | 18,822 | 5,789 | 32,817 | 11,653 |
Stock options | ||||
Stock-based compensation expense | ||||
Stock-based compensation expense | 10,027 | 7,431 | 16,913 | 16,029 |
Restricted stock units | ||||
Stock-based compensation expense | ||||
Stock-based compensation expense | 9,918 | 3,542 | 18,543 | 6,194 |
Performance-based restricted stock units | ||||
Stock-based compensation expense | ||||
Stock-based compensation expense | $ 4,459 | $ 0 | $ 8,850 | $ 0 |
Commitments and Contingencies (
Commitments and Contingencies (Details) - USD ($) | 1 Months Ended | 6 Months Ended | 12 Months Ended | |
Mar. 31, 2024 | Jun. 30, 2023 | Dec. 31, 2023 | Jun. 30, 2024 | |
Commitments and Contingencies | ||||
Product revenue, net | $ 0 | |||
Right of use asset obtained in exchange for operating lease liability | $ 1,600,000 | |||
Hoffmann-La Roche ("Roche") | Research, Development and Commercialization Agreement | ||||
Commitments and Contingencies | ||||
Milestone payment due | $ 5,000,000 | |||
Remainder of future milestone payments | $ 3,000,000 |