Madrigal Pharmaceuticals (MDGL) 10-Q 31 Oct 24 2024 Q3 Quarterly report Financial data

Cover Page

Cover Page - shares	9 Months Ended
	Sep. 30, 2024	Oct. 28, 2024
Cover [Abstract]
Document Type	10-Q
Document Quarterly Report	true
Document Period End Date	Sep. 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		21,810,407
Amendment Flag	false
Document Fiscal Year Focus	2024
Entity Central Index Key	0001157601
Current Fiscal Year End Date	--12-31
Document Fiscal Period Focus	Q3

CONDENSED CONSOLIDATED BALANCE

CONDENSED CONSOLIDATED BALANCE SHEETS - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Current assets:
Cash and cash equivalents	$ 232,684	$ 99,915
Restricted cash	5,000	0
Marketable securities	765,943	534,216
Trade receivables, net	30,463	0
Inventory	8,715	0
Prepaid expenses and other current assets	20,861	3,150
Total current assets	1,063,666	637,281
Property and equipment, net	2,268	1,553
Intangible assets, net	4,819	0
Right-of-use asset	2,512	1,713
Total assets	1,073,265	640,547
Current liabilities:
Accounts payable	45,966	28,041
Accrued liabilities	131,003	89,980
Lease liability	878	527
Total current liabilities	177,847	118,548
Long term liabilities:
Loan payable, net of discount	117,087	115,480
Lease liability	1,176	1,186
Total long term liabilities	118,263	116,666
Total liabilities	296,110	235,214
Stockholders’ equity:
Preferred stock, par value $0.0001 per share authorized: 5,000,000 shares at September 30, 2024 and December 31, 2023; 2,369,797 and 2,369,797 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively	0	0
Common stock, par value $0.0001 per share authorized: 200,000,000 at September 30, 2024 and December 31, 2023; 21,806,436 and 19,875,427 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively	2	2
Additional paid-in-capital	2,518,648	1,741,153
Accumulated other comprehensive income	1,271	468
Accumulated deficit	(1,742,766)	(1,336,290)
Total stockholders’ equity	777,155	405,333
Total liabilities and stockholders’ equity	$ 1,073,265	$ 640,547

CONDENSED CONSOLIDATED BALANC_2

CONDENSED CONSOLIDATED BALANCE SHEETS (Parenthetical) - $ / shares	Sep. 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)	21,806,436	19,875,427
Common stock, shares outstanding (in shares)	21,806,436	19,875,427

CONDENSED CONSOLIDATED STATEMEN

CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Revenues:
Product revenue, net	$ 62,175	$ 0	$ 76,813	$ 0
Operating expenses:
Cost of sales	2,152	0	2,788	0
Research and development	68,742	70,951	211,070	201,710
Selling, general and administrative	107,585	27,583	293,834	61,610
Total operating expenses	178,479	98,534	507,692	263,320
Loss from operations	(116,304)	(98,534)	(430,879)	(263,320)
Interest income	13,019	3,298	35,575	10,625
Interest expense	(3,679)	(3,504)	(11,172)	(8,741)
Net loss	$ (106,964)	$ (98,740)	$ (406,476)	$ (261,436)
Net loss per common share:
Basic net loss per common share (in dollars per share)	$ (4.92)	$ (5.34)	$ (19.31)	$ (14.27)
Diluted net loss per common share (in dollars per share)	$ (4.92)	$ (5.34)	$ (19.31)	$ (14.27)
Basic weighted average number of common shares outstanding (in shares)	21,745,929	18,476,414	21,052,544	18,326,154
Diluted weighted average number of common shares outstanding (in shares)	21,745,929	18,476,414	21,052,544	18,326,154

CONDENSED CONSOLIDATED STATEM_2

CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS - USD ($) $ in Thousands	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Statement of Comprehensive Income [Abstract]
Net Loss	$ (106,964)	$ (98,740)	$ (406,476)	$ (261,436)
Other comprehensive income (loss):
Unrealized gain (loss) on available-for-sale securities	1,639	47	803	(10)
Comprehensive loss	$ (105,325)	$ (98,693)	$ (405,673)	$ (261,446)

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
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, 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) gain 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) gain on marketable securities	(10)
Net loss	(261,436)
Balance (in shares) at Sep. 30, 2023			2,369,797	18,494,192
Balance at Sep. 30, 2023	22,249		$ 0	$ 2		1,246,385		(42)	(1,224,096)
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
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, 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) gain 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)
Increase (Decrease) in Stockholders' Equity
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, net of transaction costs (in shares)				12,200	22,959
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, net of transaction costs	2,731	1,924				2,731	1,924
Stock-based compensation expense related to equity-classified awards and Compensation expense related to stock options for services	12,660					12,660
Unrealized (loss) gain on marketable securities	47							47
Hercules warrant	121					121
Net loss	(98,740)								(98,740)
Balance (in shares) at Sep. 30, 2023			2,369,797	18,494,192
Balance at Sep. 30, 2023	$ 22,249		$ 0	$ 2		1,246,385		(42)	(1,224,096)
Balance (in shares) at Dec. 31, 2023	19,875,427		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
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, 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) gain 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	19,875,427		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) gain on marketable securities	803
Net loss	$ (406,476)
Balance (in shares) at Sep. 30, 2024	21,806,436		2,369,797	21,806,436
Balance at Sep. 30, 2024	$ 777,155		$ 0	$ 2		2,518,648		1,271	(1,742,766)
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
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, 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) gain on marketable securities	(196)							(196)
Net loss	(151,971)								(151,971)
Balance (in shares) at Jun. 30, 2024			2,369,797	21,700,893
Balance at Jun. 30, 2024	857,120		$ 0	$ 2		2,493,288		(368)	(1,635,802)
Increase (Decrease) in Stockholders' Equity
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, net of transaction costs (in shares)					105,543
Issuance of common shares and sale of warrants in equity offerings, excluding to related parties, net of transaction costs		$ 7,462					$ 7,462
Stock-based compensation expense related to equity-classified awards and Compensation expense related to stock options for services	17,898					17,898
Unrealized (loss) gain on marketable securities	1,639							1,639
Net loss	$ (106,964)								(106,964)
Balance (in shares) at Sep. 30, 2024	21,806,436		2,369,797	21,806,436
Balance at Sep. 30, 2024	$ 777,155		$ 0	$ 2		$ 2,518,648		$ 1,271	$ (1,742,766)

CONDENSED CONSOLIDATED STATEM_4

CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS - USD ($) $ in Thousands	3 Months Ended	9 Months Ended		12 Months Ended
	Sep. 30, 2024	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023
Cash flows from operating activities:
Net loss		$ (406,476)	$ (261,436)
Adjustments to reconcile net loss to net cash used in operating activities:
Stock-based compensation expense		62,204	34,883
Depreciation and amortization expense		733	385
Amortization of debt issuance costs and discount		1,607	1,661
Amortization and interest accretion related to operating leases		(458)	0
Changes in operating assets and liabilities:
Trade receivables, net		(30,463)	0
Prepaid expenses and other current assets		(17,711)	(523)
Inventory		(8,715)	0
Accounts payable		17,925	(7,289)
Accrued liabilities		41,023	(8,851)
Accrued interest, net of interest received on maturity of investments		(10,755)	(3,127)
Net cash used in operating activities		(351,086)	(244,297)
Cash flows from investing activities:
Purchases of marketable securities		(781,021)	(359,549)
Sales and maturities of marketable securities		560,852	219,595
Acquisition of intangible asset		(5,000)	0
Purchases of property and equipment, net of disposals		(1,267)	(443)
Net cash used in investing activities		(226,436)	(140,397)
Cash flows from financing activities:
Proceeds from issuance of loan payable		0	65,000
Payment of debt issuance costs		0	(363)
Net cash provided by financing activities		715,291	115,200
Net increase (decrease) in cash, cash equivalents, and restricted cash		137,769	(269,494)
Cash, cash equivalents, and restricted cash at beginning of period		99,915	331,549	$ 331,549
Cash, cash equivalents, and restricted cash at end of period	$ 237,684	237,684	62,055	99,915
Supplemental disclosure of cash flow information:
Obtaining a right-of-use asset in exchange for a lease liability	$ 1,200	1,168	1,628	$ 1,600
Nonrelated Party
Cash flows from financing activities:
Proceeds from issuance or sale of equity		397,510	24,484
Related Party
Cash flows from financing activities:
Proceeds from issuance or sale of equity		$ 317,781	$ 26,079

Organization, Business and Basi

Organization, Business and Basis of Presentation	9 Months Ended
	Sep. 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 focused on delivering novel therapeutics for nonalcoholic steatohepatitis (“NASH”), a serious liver disease with high unmet medical need that can lead to cirrhosis, liver failure and premature mortality. NASH is expected to become the leading cause of liver transplantation in the U.S. and is already the leading cause of liver transplantation among women in the U.S. 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	9 Months Ended
	Sep. 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). Product Revenue, Net 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. Accruals are established for gross to net 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. Prompt Pay : Customers receive a 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 in accordance with the return goods policy defined within each customer agreement. A returns reserve is recorded as an accrued liability. Chargebacks : The Company estimates obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers at prices lower than the list prices charged to the customer who directly purchases from the Company. The customer charges the Company for the difference between what it pays to the Company for the product and the selling price to the qualified healthcare providers, with the difference recorded as a contra receivable. Co-Payment Assistance : Co-payment assistance 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 : The Company is subject to discount obligations under government programs, including Medicaid and Medicare. Reserves for rebates are recorded in the same period the related product revenue is recognized, resulting in a reduction of product revenues and a current liability that is included in accrued expenses on the consolidated balance sheet. The Company’s estimate for rebates is based on statutory discount rates, expected utilization or an estimated number of patients on treatment, as applicable. Concentrations of Credit Risk and Significant Customers 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 and nine months ended September 30, 2024. As Rezdiffra was made commercially available in April 2024, there were no sales and no corresponding customer concentrations in 2023. Three Months Ended Nine Months Ended 2024 2023 2024 2023 Customer A 38 % — % 38 % — % Customer B 21 % — % 21 % — % Customer C 16 % — % 16 % — % Customer D 11 % — % 12 % — % 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 nine months ended September 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 nine months ended September 30, 2024 and 2023, realized gains and losses on marketable securities were not material. 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 September 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 nine months ended September 30, 2024 and 2023, the Company did not have any transfers of financial assets between Levels 1 and 2. As of September 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, which consists of work in process and finished goods, 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 balance sheet classification of inventory as current or non-current is determined by whether it will be consumed within the Company’s normal operating cycle. 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 sales in the accompanying Consolidated Statements of Income. The Company capitalizes inventory costs when future commercial sale in the ordinary course of business is probable. 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, rather were expensed as incurred as research and development expenses. The Company began capitalizing inventory in March 2024 after FDA approval was granted. 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 cash compensation and 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 sales 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 stock-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 accounts for income taxes in accordance with ASC 740, "Income Taxes", which prescribes the use of the liability method where deferred tax asset and liability account balances are determined based on differences between financial reporting and tax bases of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. The Company provides a valuation allowance, if necessary, to reduce deferred tax assets to their estimated realizable value if it is more likely than not that a portion or all of the deferred tax assets will not be realized based on the weight of available positive and negative evidence. 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 nine months ended September 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 September 30, 2024 2023 Common stock options 1,701,618 2,518,612 Restricted stock units 502,867 311,767 Performance-based restricted stock units 235,520 150,000 Preferred stock 2,369,797 2,369,797 Warrants 3,625,244 19,454 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. The Company does not expect the adoption of ASU 2023-07 to have a significant impact 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 does not expect the adoption of ASU 2023-09 to have a significant impact on its financial statements.

Liquidity and Uncertainties

Liquidity and Uncertainties	9 Months Ended
	Sep. 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 FDA and other government regulations.

Cash, Cash Equivalents and Mark

Cash, Cash Equivalents and Marketable Securities	9 Months Ended
	Sep. 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 September 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 September 30, 2024 and December 31, 2023 is as follows (in thousands): September 30, 2024 Cost Unrealized gains Unrealized losses Fair value Cash and cash equivalents: Cash (Level 1) $ 25,242 $ — $ — $ 25,242 Money market funds (Level 1) 86,060 — — 86,060 U.S. government and government sponsored entities due within 3 months (Level 1) 67,541 — — 67,541 Corporate debt securities due within 3 months of date of purchase (Level 2) 58,841 — — 58,841 Total cash and cash equivalents 237,684 — — 237,684 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 436,277 573 (25) 436,825 U.S. government and government sponsored entities due within 1 year of date of purchase (Level 2) 297,160 411 (7) 297,564 U.S. government and government sponsored entities due within 1 to 2 years of date of purchase (Level 2) 31,235 320 (1) 31,554 Total cash, cash equivalents, restricted cash, and marketable securities $ 1,002,356 $ 1,304 $ (33) $ 1,003,627 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	9 Months Ended
	Sep. 30, 2024
Inventory Disclosure [Abstract]
Inventory	Inventory The following table summarizes the Company's inventory balances as of September 30, 2024 and December 31, 2023 (in thousands): September 30, 2024 December 31, 2023 Raw materials $ — $ — Work in process 5,389 — Finished goods 3,326 — Total $ 8,715 $ —

Accrued Liabilities

Accrued Liabilities	9 Months Ended
	Sep. 30, 2024
Accrued Liabilities, Current [Abstract]
Accrued Liabilities	Accrued Liabilities Accrued liabilities as of September 30, 2024 and December 31, 2023 consisted of the following (in thousands): September 30, December 31, Contract research organization costs $ 59,809 $ 50,737 Other clinical study related costs 1,496 3,724 Manufacturing and drug supply 2,291 9,705 Compensation and benefits 29,684 17,030 Professional fees 23,095 6,814 Gross to net accrued liabilities 7,816 — Other 6,812 1,970 Total accrued liabilities $ 131,003 $ 89,980

Long Term Debt

Long Term Debt	9 Months Ended
	Sep. 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, but subsequently entered into an amendment to extend the availability of these funds in August 2024. Coincident with the expansion of Tranche 2 borrowing capacity by $15.0 million, the First 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 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. On August 22, 2024, the Company entered into the Second Amendment (the “Second Amendment”) to the Loan Facility (as amended by the First Amendment and the Second Amendment, the “Second Amended Loan Facility”). Under the Second Amended Loan Facility, the Company’s borrowing capacity available under Tranche 4 is increased to include the $75.0 million available under Tranche 3 that was not utilized by the Company. After such increase, the Company’s current borrowing capacity is $135.0 million under Tranche 4, which is available subject to Hercules’ sole discretion. 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 may apply commencing at or after the time that financial reporting is due for the quarter ending September 30, 2024, subject to certain exceptions in the event that the Company maintains a specified market capitalization or an unrestricted cash balance as set forth in the Loan Facility. 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 September 30, 2024, the outstanding principal under the Loan Facility was $115.0 million. The interest rate as of September 30, 2024 was 10.45%. As of September 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 September 30, 2024 are as follows (in thousands): Period Ending September 30, 2024: Amount 2024 $ 3,067 2025 12,184 2026 79,197 Thereafter 53,247 $ 147,695 Less amount representing interest (26,542) Less unamortized discount (4,066) Loans payable, net of discount $ 117,087

Stockholders' Equity

Stockholders' Equity	9 Months Ended
	Sep. 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 nine months ended September 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	9 Months Ended
	Sep. 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 ten years until April 23, 2035. As of September 30, 2024, 1,233,829 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 September 30, 2024, 21,193 shares were available for future issuance under the 2023 Inducement Plan. Stock Options The following table summarizes stock option activity during the nine months ended September 30, 2024: Shares Weighted average exercise price Outstanding at December 31, 2023 2,355,779 $ 79.94 Options granted 162,517 234.84 Options exercised (748,143) 76.35 Options cancelled (68,535) 121.89 Outstanding at September 30, 2024 1,701,618 $ 94.62 Exercisable at September 30, 2024 1,297,563 $ 78.45 The total cash received by the Company as a result of stock option exercises was $57.1 million and $26.2 million, respectively, for the nine months ended September 30, 2024 and 2023. The total intrinsic value of options exercised was $132.5 million and $51.0 million, respectively, for the nine months ended September 30, 2024 and 2023. The weighted-average grant date fair values, based on the Black-Scholes option model, of options granted during the nine months ended September 30, 2024 and 2023 were $155.41 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 September 30, 2024, the Company had 502,867 restricted stock units outstanding, with a weighted average grant date fair value of $233.42 per unit. The following table summarizes RSU activity, excluding performance-based RSUs, during the nine months ended September 30, 2024: Shares Weighted average grant date fair value Outstanding at December 31, 2023 376,117 $ 241.45 RSUs granted 275,278 234.64 RSUs vested (93,172) 262.91 RSUs forfeited (55,356) 244.45 Outstanding at September 30, 2024 502,867 $ 233.42 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 nine months ended September 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 September 30, 2024 92,760 235,520 $ 257.77 Outstanding Awards As of September 30, 2024, the Company had restricted stock units, performance stock units, and options outstanding pursuant to which an aggregate of 2,440,005 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 nine months ended September 30, 2024 and 2023 was as follows (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Stock-based compensation expense by type of award: Stock options $ 4,978 $ 8,375 $ 21,891 $ 24,404 Restricted stock units 8,292 3,652 26,835 9,846 Performance-based restricted stock units 4,628 633 13,478 633 Total stock-based compensation expense $ 17,898 $ 12,660 $ 62,204 $ 34,883 Effect of stock-based compensation expense by line item: Research and development $ 5,570 $ 5,133 $ 17,059 $ 15,703 Selling, general and administrative 12,328 7,527 45,145 19,180 Total stock-based compensation expense included in net loss $ 17,898 $ 12,660 $ 62,204 $ 34,883

Commitments and Contingencies

Commitments and Contingencies	9 Months Ended
	Sep. 30, 2024
Commitments and Contingencies Disclosure [Abstract]
Commitments and Contingencies	Commitments and Contingencies The Company has entered into customary contractual arrangements and letters of intent in preparation for and in support of operations in the normal course of business. 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 began accruing for royalty payments following its commercial launch of Rezdiffra in April 2024. The Company did not achieve any product development or regulatory milestones for the nine months ended September 30, 2023. In 2019, the Company entered into an operating lease for office space located in West Conshohocken, PA (the “Office Lease”), which was later updated by three amendments entered into from 2019 to 2022. In May 2023, the Company entered into the Fourth Amendment to the Office Lease (the “Fourth Lease Amendment”), which did not have a financial impact. In August 2023, the Company entered into the Fifth Amendment to the Office Lease (the “Fifth Lease Amendment”). The Fifth Lease Amendment extended the term of the Office Lease through November 2026. As a result of the Fifth 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 Amendment (the “Sixth Lease Amendment”) and the Seventh Amendment (the “Seventh Lease Amendment”) to the Office Lease, respectively, leasing additional office space in the same premises under the Office Lease. The lease for the additional office space commenced in September 2024 and resulted in an incremental $1.2 million right-of-use asset and lease liability being recorded for the quarter ended September 30, 2024. In August 2024, the Company entered into the Eighth (the “Eighth Lease Amendment”) and in October 2024, the Company entered into the Ninth Amendment (the “Ninth Lease Amendment”) to the Office Lease further expanding the amount of office space in the same premises. The commencement date of the Eighth and Ninth Lease Amendments did not occur as of September 30, 2024, therefore had no impact to the financial statements.

Subsequent Events

Subsequent Events	9 Months Ended
	Sep. 30, 2024
Subsequent Events [Abstract]
Subsequent Events	Subsequent Events None.

Pay vs Performance Disclosure

Pay vs Performance Disclosure - USD ($) $ in Thousands	3 Months Ended						9 Months Ended
	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023
Pay vs Performance Disclosure
Net loss	$ (106,964)	$ (151,971)	$ (147,541)	$ (98,740)	$ (85,800)	$ (76,896)	$ (406,476)	$ (261,436)

Insider Trading Arrangements

Insider Trading Arrangements	3 Months Ended	9 Months Ended
	Sep. 30, 2024 shares	Sep. 30, 2024 shares
Trading Arrangements, by Individual
Non-Rule 10b5-1 Arrangement Adopted	false
Rule 10b5-1 Arrangement Terminated	false
Non-Rule 10b5-1 Arrangement Terminated	false
Richard Levy [Member]
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 October 28, 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
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)	9 Months Ended
	Sep. 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). Product Revenue, Net 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. Accruals are established for gross to net 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. Prompt Pay : Customers receive a 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 in accordance with the return goods policy defined within each customer agreement. A returns reserve is recorded as an accrued liability. Chargebacks : The Company estimates obligations resulting from contractual commitments with the government and other entities to sell products to qualified healthcare providers at prices lower than the list prices charged to the customer who directly purchases from the Company. The customer charges the Company for the difference between what it pays to the Company for the product and the selling price to the qualified healthcare providers, with the difference recorded as a contra receivable. Co-Payment Assistance : Co-payment assistance 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 : The Company is subject to discount obligations under government programs, including Medicaid and Medicare. Reserves for rebates are recorded in the same period the related product revenue is recognized, resulting in a reduction of product revenues and a current liability that is included in accrued expenses on the consolidated balance sheet. The Company’s estimate for rebates is based on statutory discount rates, expected utilization or an estimated number of patients on treatment, as applicable.
Concentrations of Credit Risk and Significant Customers	Concentrations of Credit Risk and Significant Customers
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.
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, which consists of work in process and finished goods, 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 balance sheet classification of inventory as current or non-current is determined by whether it will be consumed within the Company’s normal operating cycle. 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 sales in the accompanying Consolidated Statements of Income. The Company capitalizes inventory costs when future commercial sale in the ordinary course of business is probable. 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, rather were expensed as incurred as research and development expenses. The Company began capitalizing inventory in March 2024 after FDA approval was granted.
Research and Development Costs	Research and Development Costs
Patents	Patents
Intangible Assets	Intangible Assets Intangible assets with finite lives are amortized to cost of sales 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 stock-based compensation arrangements due to the fact that the Company does not believe it is more likely than not it will realize the related deferred tax assets.
Income Taxes	Income Taxes The Company accounts for income taxes in accordance with ASC 740, "Income Taxes", which prescribes the use of the liability method where deferred tax asset and liability account balances are determined based on differences between financial reporting and tax bases of assets and liabilities and are measured using the enacted tax rates and laws that will be in effect when the differences are expected to reverse. The Company provides a valuation allowance, if necessary, to reduce deferred tax assets to their estimated realizable value if it is more likely than not that a portion or all of the deferred tax assets will not be realized based on the weight of available positive and negative evidence. The Company currently maintains a 100% valuation allowance on its deferred tax assets.
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. The Company does not expect the adoption of ASU 2023-07 to have a significant impact 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 does not expect the adoption of ASU 2023-09 to have a significant impact on its financial statements.

Summary of Significant Accoun_3

Summary of Significant Accounting Policies (Tables)	9 Months Ended
	Sep. 30, 2024
Accounting Policies [Abstract]
Schedule of Concentrations of Credit Risk and Significant Customers	The following customers accounted for over 10% of total gross product revenue during three and nine months ended September 30, 2024. As Rezdiffra was made commercially available in April 2024, there were no sales and no corresponding customer concentrations in 2023. Three Months Ended Nine Months Ended 2024 2023 2024 2023 Customer A 38 % — % 38 % — % Customer B 21 % — % 21 % — % Customer C 16 % — % 16 % — % Customer D 11 % — % 12 % — %
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 September 30, 2024 2023 Common stock options 1,701,618 2,518,612 Restricted stock units 502,867 311,767 Performance-based restricted stock units 235,520 150,000 Preferred stock 2,369,797 2,369,797 Warrants 3,625,244 19,454

Cash, Cash Equivalents and Ma_2

Cash, Cash Equivalents and Marketable Securities (Tables)	9 Months Ended
	Sep. 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 September 30, 2024 and December 31, 2023 is as follows (in thousands): September 30, 2024 Cost Unrealized gains Unrealized losses Fair value Cash and cash equivalents: Cash (Level 1) $ 25,242 $ — $ — $ 25,242 Money market funds (Level 1) 86,060 — — 86,060 U.S. government and government sponsored entities due within 3 months (Level 1) 67,541 — — 67,541 Corporate debt securities due within 3 months of date of purchase (Level 2) 58,841 — — 58,841 Total cash and cash equivalents 237,684 — — 237,684 Marketable securities: Corporate debt securities due within 1 year of date of purchase (Level 2) 436,277 573 (25) 436,825 U.S. government and government sponsored entities due within 1 year of date of purchase (Level 2) 297,160 411 (7) 297,564 U.S. government and government sponsored entities due within 1 to 2 years of date of purchase (Level 2) 31,235 320 (1) 31,554 Total cash, cash equivalents, restricted cash, and marketable securities $ 1,002,356 $ 1,304 $ (33) $ 1,003,627 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)	9 Months Ended
	Sep. 30, 2024
Inventory Disclosure [Abstract]
Schedule of Inventory Balances	The following table summarizes the Company's inventory balances as of September 30, 2024 and December 31, 2023 (in thousands): September 30, 2024 December 31, 2023 Raw materials $ — $ — Work in process 5,389 — Finished goods 3,326 — Total $ 8,715 $ —

Accrued Liabilities (Tables)

Accrued Liabilities (Tables)	9 Months Ended
	Sep. 30, 2024
Accrued Liabilities, Current [Abstract]
Schedule of Accrued Liabilities	Accrued liabilities as of September 30, 2024 and December 31, 2023 consisted of the following (in thousands): September 30, December 31, Contract research organization costs $ 59,809 $ 50,737 Other clinical study related costs 1,496 3,724 Manufacturing and drug supply 2,291 9,705 Compensation and benefits 29,684 17,030 Professional fees 23,095 6,814 Gross to net accrued liabilities 7,816 — Other 6,812 1,970 Total accrued liabilities $ 131,003 $ 89,980

Long Term Debt (Tables)

Long Term Debt (Tables)	9 Months Ended
	Sep. 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 September 30, 2024 are as follows (in thousands): Period Ending September 30, 2024: Amount 2024 $ 3,067 2025 12,184 2026 79,197 Thereafter 53,247 $ 147,695 Less amount representing interest (26,542) Less unamortized discount (4,066) Loans payable, net of discount $ 117,087

Stock-based Compensation (Table

Stock-based Compensation (Tables)	9 Months Ended
	Sep. 30, 2024
Share-Based Payment Arrangement [Abstract]
Schedule of Stock Option Activity	The following table summarizes stock option activity during the nine months ended September 30, 2024: Shares Weighted average exercise price Outstanding at December 31, 2023 2,355,779 $ 79.94 Options granted 162,517 234.84 Options exercised (748,143) 76.35 Options cancelled (68,535) 121.89 Outstanding at September 30, 2024 1,701,618 $ 94.62 Exercisable at September 30, 2024 1,297,563 $ 78.45 Stock-based compensation expense during the three and nine months ended September 30, 2024 and 2023 was as follows (in thousands): Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Stock-based compensation expense by type of award: Stock options $ 4,978 $ 8,375 $ 21,891 $ 24,404 Restricted stock units 8,292 3,652 26,835 9,846 Performance-based restricted stock units 4,628 633 13,478 633 Total stock-based compensation expense $ 17,898 $ 12,660 $ 62,204 $ 34,883 Effect of stock-based compensation expense by line item: Research and development $ 5,570 $ 5,133 $ 17,059 $ 15,703 Selling, general and administrative 12,328 7,527 45,145 19,180 Total stock-based compensation expense included in net loss $ 17,898 $ 12,660 $ 62,204 $ 34,883
Schedule of RSU Activity	The following table summarizes RSU activity, excluding performance-based RSUs, during the nine months ended September 30, 2024: Shares Weighted average grant date fair value Outstanding at December 31, 2023 376,117 $ 241.45 RSUs granted 275,278 234.64 RSUs vested (93,172) 262.91 RSUs forfeited (55,356) 244.45 Outstanding at September 30, 2024 502,867 $ 233.42
Schedule of PSU Activity	The following table summarizes PSU activity during the nine months ended September 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 September 30, 2024 92,760 235,520 $ 257.77

Summary of Significant Accoun_4

Summary of Significant Accounting Policies - Concentrations of Credit Risk and Significant Customers (Details) - Revenue from Contract with Customer, Product and Service Benchmark - Customer Concentration Risk	3 Months Ended		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Customer A
Concentration Risk [Line Items]
Concentration risk, percentage (as a percent)	38%	0%	38%	0%
Customer B
Concentration Risk [Line Items]
Concentration risk, percentage (as a percent)	21%	0%	21%	0%
Customer C
Concentration Risk [Line Items]
Concentration risk, percentage (as a percent)	16%	0%	16%	0%
Customer D
Concentration Risk [Line Items]
Concentration risk, percentage (as a percent)	11%	0%	12%	0%

Summary of Significant Accoun_5

Summary of Significant Accounting Policies - Marketable Securities (Details) - USD ($)	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Accounting Policies [Abstract]
Realized gains or losses on marketable securities	$ 0	$ 0

Summary of Significant Accoun_6

Summary of Significant Accounting Policies - Income Taxes (Details)	9 Months Ended
	Sep. 30, 2024
Income Taxes
Valuation allowance on deferred tax assets (as a percent)	100%

Summary of Significant Accoun_7

Summary of Significant Accounting Policies - Basic and Diluted Loss Per Common Share (Details) - shares	9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023
Common 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)	1,701,618	2,518,612
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)	502,867	311,767
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	150,000
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	19,454

Liquidity and Uncertainties (De

Liquidity and Uncertainties (Details) - USD ($) $ in Thousands	1 Months Ended		3 Months Ended						9 Months Ended
	Apr. 30, 2024	Mar. 31, 2024	Sep. 30, 2024	Jun. 30, 2024	Mar. 31, 2024	Sep. 30, 2023	Jun. 30, 2023	Mar. 31, 2023	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023
Liquidity and Uncertainties
Net loss			$ (106,964)	$ (151,971)	$ (147,541)	$ (98,740)	$ (85,800)	$ (76,896)	$ (406,476)	$ (261,436)
Accumulated deficit			$ (1,742,766)						$ (1,742,766)		$ (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 ($)	Sep. 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 - Cash, Cash Equivalents and Available-for-Sale Marketable Securities (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Cash and cash equivalents:
Total cash and cash equivalents	$ 237,684	$ 99,915
Marketable securities:
Unrealized gains	1,304	476
Unrealized losses	(33)	(8)
Cost	1,002,356	633,663
Fair value	1,003,627	634,131
Corporate Debt Securities
Cash and cash equivalents:
Total cash and cash equivalents	58,841	3,664
Marketable securities:
Cost	436,277	382,028
Unrealized gains	573	195
Unrealized losses	(25)	(7)
Fair value	436,825	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
U.S. Government and Government Sponsored Entities
Cash and cash equivalents:
Total cash and cash equivalents	67,541	14,967
U.S. Government and Government Sponsored Entities Due Within 1 Year of Date of Purchase
Marketable securities:
Cost	297,160	150,743
Unrealized gains	411	280
Unrealized losses	(7)	(1)
Fair value	297,564	151,022
U.S. government and government sponsored entities due within 1 to 2 years of date of purchase
Marketable securities:
Cost	31,235
Unrealized gains	320
Unrealized losses	(1)
Fair value	31,554
Cash
Cash and cash equivalents:
Total cash and cash equivalents	25,242	2,729
Money Market Funds
Cash and cash equivalents:
Total cash and cash equivalents	$ 86,060	$ 78,555

Inventory (Details)

Inventory (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Inventory Disclosure [Abstract]
Raw materials	$ 0	$ 0
Work in process	5,389	0
Finished goods	3,326	0
Total	$ 8,715	$ 0

Accrued Liabilities (Details)

Accrued Liabilities (Details) - USD ($) $ in Thousands	Sep. 30, 2024	Dec. 31, 2023
Accrued Liabilities, Current [Abstract]
Contract research organization costs	$ 59,809	$ 50,737
Other clinical study related costs	1,496	3,724
Manufacturing and drug supply	2,291	9,705
Compensation and benefits	29,684	17,030
Professional fees	23,095	6,814
Gross to net accrued liabilities	7,816	0
Other	6,812	1,970
Total accrued liabilities	$ 131,003	$ 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 ($) tranche	Sep. 30, 2024 USD ($)	Aug. 22, 2024 USD ($)	Mar. 31, 2024 USD ($)	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						$ 117,087,000
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, (as a percent)					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 (as a percent)					7.45%
Accrued interest payments, term (in months)										36 months
Minimum liquidity covenant									$ 35,000,000
Loans payable, net of discount						$ 115,000,000
Line of credit facility, interest rate (as a percent)						10.45%
Secured Debt | Loan Facility | Line of Credit | Variable Rate Component One | Prime Rate
Debt Instrument [Line Items]
Basis spread on variable rate (as a percent)		2.45%
Secured Debt | Loan Facility | Line of Credit | Variable Rate Component Two | Prime Rate
Debt Instrument [Line Items]
Basis spread on variable rate (as a percent)		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)
Line of credit facility, current borrowing capacity							$ 135,000,000

Long Term Debt - Maturities of

Long Term Debt - Maturities of Long-Term Debt (Details) $ in Thousands	9 Months Ended
	Sep. 30, 2024 USD ($)
Debt Disclosure [Abstract]
2024	$ 3,067
2025	12,184
2026	79,197
Thereafter	53,247
Long-term debt	147,695
Less amount representing interest	(26,542)
Less unamortized discount	(4,066)
Loans payable, net of discount	$ 117,087

Stockholders' Equity- Common St

Stockholders' Equity- Common Stock (Details)	9 Months Ended
	Sep. 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)	Sep. 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 ($)	Mar. 21, 2024 USD ($) $ / shares 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 (in days)		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 dollars 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 dollars per share) | $ / shares		$ 259.9999
Stock price discount per share (in dollars 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 dollars 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 dollars per share) | $ / shares			$ 151.6899
Stock price discount per share (in dollars 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	9 Months Ended	17 Months Ended	36 Months Ended	37 Months Ended
	Apr. 30, 2024	Mar. 31, 2024	May 31, 2023	Jun. 30, 2021	Sep. 30, 2024	Sep. 30, 2024	Sep. 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	9 Months Ended
	Jun. 25, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023
Unrecognized stock-based compensation expense
Unrecognized stock compensation expense			$ 139.1
Weighted average remaining period (in years)			2 years 8 months 15 days
2015 Stock Plan
Unrecognized stock-based compensation expense
Expiration period (in years)	10 years
Shares available for future issuance (in shares)	750,000		1,233,829
2023 Inducement Plan
Unrecognized stock-based compensation expense
Expiration period (in years)		10 years
Shares available for future issuance (in shares)			21,193
Common stock, capital shares reserved for future issuance (in shares)			500,000
2015 Stock Plan And Inducement Plan
Unrecognized stock-based compensation expense
Common stock granted and issuable (in shares)			2,440,005
Stock Options
Unrecognized stock-based compensation expense
Proceeds resulting from exercise of stock options			$ 57.1	$ 26.2
Total intrinsic value of options exercised			$ 132.5	$ 51
Weighted-average grant date fair value of options (in dollars per share)			$ 155.41	$ 219.02
Stock Options | 2015 Stock Plan
Unrecognized stock-based compensation expense
Expiration period (in years)			10 years
Restricted Stock Units (RSUs)
Unrecognized stock-based compensation expense
Equity award other than options, nonvested (in shares)			502,867		376,117
Weighted average grant date fair value (in dollars per share)			$ 233.42		$ 241.45
Restricted Stock Units (RSUs) | 2015 Stock Plan
Unrecognized stock-based compensation expense
Equity award other than options, nonvested (in shares)			502,867
Weighted average grant date fair value (in dollars per share)			$ 233.42
Performance-Based Restricted Stock Units (PSUs)
Unrecognized stock-based compensation expense
Equity award other than options, nonvested (in shares)			92,760		50,000
Weighted average grant date fair value (in dollars per share)			$ 257.77		$ 146.37

Stock-based Compensation - Stoc

Stock-based Compensation - Stock Option (Details)	9 Months Ended
	Sep. 30, 2024 $ / shares shares
Shares
Outstanding at the beginning of the year (in shares) | shares	2,355,779
Options granted (in shares) | shares	162,517
Options exercised (in shares) | shares	(748,143)
Options cancelled (in shares) | shares	(68,535)
Outstanding at the ending of the year (in shares) | shares	1,701,618
Exercisable at the ending of the year (in shares) | shares	1,297,563
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	234.84
Options exercised (in dollars per share) | $ / shares	76.35
Options cancelled (in dollars per share) | $ / shares	121.89
Outstanding at the ending of the year (in dollars per share) | $ / shares	94.62
Exercisable at the ending of the year (in dollars per share) | $ / shares	$ 78.45

Stock-based Compensation - Rest

Stock-based Compensation - Restricted Stock Units (Details) - Restricted Stock Units (RSUs)	9 Months Ended
	Sep. 30, 2024 $ / shares shares
Shares
Outstanding, beginning balance (in shares) | shares	376,117
Granted (in shares) | shares	275,278
Vested (in shares) | shares	(93,172)
Forfeited (in shares) | shares	(55,356)
Outstanding, ending balance (in shares) | shares	502,867
Weighted average grant date fair value
Unvested, beginning balance (in dollars per share) | $ / shares	$ 241.45
Granted (in dollars per share) | $ / shares	234.64
Vested (in dollars per share) | $ / shares	262.91
Forfeited (in dollars per share) | $ / shares	244.45
Unvested, ending balance (in dollars per share) | $ / shares	$ 233.42

Stock-based Compensation - Perf

Stock-based Compensation - Performance-Based Restricted Stock Units (Details)	9 Months Ended
	Sep. 30, 2024 $ / shares shares
Performance-Based Restricted Stock Units (PSUs)
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 dollars per share) | $ / shares	$ 146.37
PSUs granted (in dollars per share) | $ / shares	388.02
PSUs attained (in dollars per share) | $ / shares	0
PSUs forfeited (in dollars per share) | $ / shares	388.02
Unvested, ending balance (in dollars 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		9 Months Ended
	Sep. 30, 2024	Sep. 30, 2023	Sep. 30, 2024	Sep. 30, 2023
Stock-based compensation expense
Stock-based compensation expense	$ 17,898	$ 12,660	$ 62,204	$ 34,883
Research and development
Stock-based compensation expense
Stock-based compensation expense	5,570	5,133	17,059	15,703
Selling, general and administrative
Stock-based compensation expense
Stock-based compensation expense	12,328	7,527	45,145	19,180
Stock options
Stock-based compensation expense
Stock-based compensation expense	4,978	8,375	21,891	24,404
Restricted stock units
Stock-based compensation expense
Stock-based compensation expense	8,292	3,652	26,835	9,846
Performance-based restricted stock units
Stock-based compensation expense
Stock-based compensation expense	$ 4,628	$ 633	$ 13,478	$ 633

Commitments and Contingencies (

Commitments and Contingencies (Details) - USD ($)	1 Months Ended	3 Months Ended	9 Months Ended		12 Months Ended
	Mar. 31, 2024	Sep. 30, 2024	Sep. 30, 2024	Sep. 30, 2023	Dec. 31, 2023
Commitments and Contingencies
Product revenue, net				$ 0
Obtaining a right-of-use asset in exchange for a lease liability		$ 1,200,000	$ 1,168,000	$ 1,628,000	$ 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	$ 3,000,000