Protagonist Therapeutics, Inc 10-Q

ITEM 1. FINANCIAL STATEMENTS PROTAGONIST THERAPEUTICS, INC. Condensed Consolidated Balance Sheets (Unaudited) (In thousands, except share and per share data) ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2025 2024 Assets ​ ​ ​ ​ ​ ​ Current assets: ​ ​ ​ ​ ​ ​ Cash and cash equivalents ​ $ 168,543 ​ $ 97,249 Marketable securities ​ ​ 401,922 ​ ​ 321,664 Receivable from collaboration partner ​ ​ — ​ ​ 165,000 Contract asset ​ ​ 23,363 ​ ​ — Prepaid expenses and other current assets ​ ​ 8,698 ​ ​ 7,728 Total current assets ​ ​ 602,526 ​ ​ 591,641 Marketable securities - noncurrent ​ ​ 102,493 ​ ​ 140,252 Property and equipment, net ​ ​ 4,108 ​ ​ 3,190 Restricted cash - noncurrent ​ ​ 290 ​ ​ 225 Operating lease right-of-use asset ​ ​ 8,589 ​ ​ 9,417 Total assets ​ $ 718,006 ​ $ 744,725 Liabilities and Stockholders’ Equity ​ ​ ​ ​ ​ ​ Current liabilities: ​ ​ ​ ​ ​ Accounts payable ​ $ 3,249 ​ $ 1,615 Accrued expenses and other payables ​ ​ 15,397 ​ ​ 23,693 Income taxes payable ​ ​ — ​ ​ 2,689 Deferred revenue ​ ​ 14,688 ​ ​ 18,891 Operating lease liability ​ ​ 2,173 ​ ​ 510 Total current liabilities ​ ​ 35,507 ​ ​ 47,398 Deferred revenue - noncurrent ​ ​ 5,375 ​ ​ 11,676 Operating lease liability - noncurrent ​ ​ 9,106 ​ ​ 10,356 Total liabilities ​ ​ 49,988 ​ ​ 69,430 Commitments and contingencies ​ ​ ​ ​ ​ ​ Stockholders’ equity: ​ ​ ​ ​ ​ ​ Preferred stock, $ 0.00001 par value, 10,000,000 shares authorized; no shares issued and outstanding ​ ​ — ​ ​ — Common stock, $ 0.00001 par value, 180,000,000 shares authorized; 62,107,572 and 61,035,139 shares issued and outstanding as of June 30, 2025 and December 31, 2024, respectively ​ ​ 1 ​ ​ 1 Additional paid-in capital ​ ​ 1,054,980 ​ ​ 1,015,898 Accumulated other comprehensive loss ​ ​ ( 15 ) ​ ​ ( 82 ) Accumulated deficit ​ ​ ( 386,948 ) ​ ​ ( 340,522 ) Total stockholders’ equity ​ ​ 668,018 ​ ​ 675,295 Total liabilities and stockholders’ equity ​ $ 718,006 ​ $ 744,725 ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. ​ 1 Table of Contents PROTAGONIST THERAPEUTICS, INC. Condensed Consolidated Statements of Operations (Unaudited) (In thousands, except share and per share data) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2025 2024 2025 2024 License and collaboration revenue ​ $ 5,546 ​ $ 4,167 ​ $ 33,867 ​ $ 259,120 Operating expenses: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Research and development ​ ​ 37,036 ​ ​ 33,520 ​ ​ 72,929 ​ ​ 67,254 General and administrative ​ 10,551 ​ 9,440 ​ 22,289 ​ 24,350 Total operating expenses ​ 47,587 ​ 42,960 ​ 95,218 ​ 91,604 (Loss) income from operations ​ ( 42,041 ) ​ ( 38,793 ) ​ ( 61,351 ) ​ 167,516 Interest income ​ 7,406 ​ 7,404 ​ 14,979 ​ ​ 11,780 Other income, net ​ ​ 36 ​ ​ 97 ​ ​ 118 ​ ​ 78 (Loss) income before income tax expense (benefit) ​ ​ ( 34,599 ) ​ ​ ( 31,292 ) ​ ​ ( 46,254 ) ​ ​ 179,374 Income tax expense (benefit) ​ ​ 172 ​ ​ ( 676 ) ​ ​ 172 ​ ​ 2,650 Net (loss) income ​ $ ( 34,771 ) ​ $ ( 30,616 ) ​ $ ( 46,426 ) ​ $ 176,724 Net (loss) income per share, basic ​ $ ( 0.55 ) ​ $ ( 0.50 ) ​ $ ( 0.73 ) ​ $ 2.89 Net (loss) income per share, diluted ​ $ ( 0.55 ) ​ $ ( 0.50 ) ​ $ ( 0.73 ) ​ $ 2.77 Weighted-average shares used to compute net (loss) income per share, basic ​ 63,510,537 61,305,289 ​ 63,238,682 61,080,489 Weighted-average shares used to compute net (loss) income per share, diluted ​ 63,510,537 61,305,289 ​ 63,238,682 63,909,633 ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. ​ 2 Table of Contents PROTAGONIST THERAPEUTICS, INC. Condensed Consolidated Statements of Comprehensive (Loss ) Income (Unaudited) (In thousands) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2025 2024 2025 2024 Net (loss) income ​ $ ( 34,771 ) ​ $ ( 30,616 ) ​ $ ( 46,426 ) ​ $ 176,724 Other comprehensive (loss) income: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Unrealized (loss) gain on marketable securities ​ ​ ( 100 ) ​ ​ ( 20 ) ​ ​ 67 ​ ​ ( 144 ) Comprehensive (loss) income ​ $ ( 34,871 ) ​ $ ( 30,636 ) ​ $ ( 46,359 ) ​ $ 176,580 ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. ​ ​ 3 Table of Contents PROTAGONIST THERAPEUTICS, INC. Condensed Consolidated Statements of Stockholders’ Equity (Unaudited) (In thousands, except share data) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ Other ​ ​ ​ Total ​ ​ Common ​ Paid-In ​ Comprehensive ​ Accumulated ​ Stockholders’ ​ ​ Stock ​ Capital ​ (Loss) Income ​ Deficit ​ Equity Three months ended June 30, 2025 Shares Amount ​ ​ ​ ​ ​ ​ ​ Balance at March 31, 2025 61,928,760 $ 1 ​ $ 1,041,143 $ 85 ​ $ ( 352,177 ) $ 689,052 Issuance of common stock under equity incentive and employee stock purchase plans ​ 178,812 — ​ 2,925 — ​ — 2,925 Stock-based compensation expense — — ​ 10,912 — ​ — 10,912 Other comprehensive loss — — ​ — ( 100 ) ​ — ( 100 ) Net loss — — ​ — — ​ ( 34,771 ) ( 34,771 ) Balance at June 30, 2025 62,107,572 $ 1 ​ $ 1,054,980 $ ( 15 ) ​ $ ( 386,948 ) $ 668,018 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ Other ​ ​ ​ Total ​ ​ Common ​ Paid-In ​ Comprehensive ​ Accumulated ​ Stockholders’ ​ ​ Stock ​ Capital ​ (Loss) Income ​ Deficit ​ Equity Three months ended June 30, 2024 Shares Amount ​ ​ ​ ​ ​ ​ ​ Balance at March 31, 2024 ​ 58,600,787 $ 1 ​ $ 969,042 $ ( 229 ) ​ $ ( 408,370 ) $ 560,444 Issuance of common stock under equity incentive and employee stock purchase plans ​ 161,276 — ​ 2,572 — ​ — 2,572 Stock-based compensation expense — — ​ 8,944 — ​ — 8,944 Other comprehensive loss — — ​ — ( 20 ) ​ — ( 20 ) Net loss — — ​ — — ​ ( 30,616 ) ( 30,616 ) Balance at June 30, 2024 58,762,063 $ 1 ​ $ 980,558 $ ( 249 ) ​ $ ( 438,986 ) $ 541,324 ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. ​ ​ 4 Table of Contents PROTAGONIST THERAPEUTICS, INC. Condensed Consolidated Statements of Stockholders’ Equity (Unaudited) (In thousands, except share data) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ Other ​ ​ ​ Total ​ ​ Common ​ Paid-In ​ Comprehensive ​ Accumulated ​ Stockholders’ ​ ​ Stock ​ Capital ​ (Loss) Income ​ Deficit ​ Equity Six months ended June 30, 2025 Shares Amount ​ ​ ​ ​ ​ ​ ​ Balance at December 31, 2024 61,035,139 $ 1 ​ $ 1,015,898 $ ( 82 ) ​ $ ( 340,522 ) $ 675,295 Issuance of common stock under equity incentive and employee stock purchase plans ​ 1,085,069 ​ ​ — ​ ​ 14,847 ​ ​ — ​ ​ — ​ ​ 14,847 Shares withheld for net settlement of tax withholding upon vesting of restricted stock units ​ ( 12,636 ) ​ ​ — ​ ​ ( 479 ) ​ ​ — ​ ​ — ​ ​ ( 479 ) Stock-based compensation expense — — ​ 24,714 — ​ — 24,714 Other comprehensive income — — ​ — 67 ​ — 67 Net loss — — ​ — — ​ ( 46,426 ) ( 46,426 ) Balance at June 30, 2025 62,107,572 $ 1 ​ $ 1,054,980 $ ( 15 ) ​ $ ( 386,948 ) $ 668,018 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Accumulated ​ ​ ​ ​ ​ ​ ​ ​ Additional ​ Other ​ ​ ​ Total ​ ​ Common ​ Paid-In ​ Comprehensive ​ Accumulated ​ Stockholders’ ​ ​ Stock ​ Capital ​ (Loss) Income ​ Deficit ​ Equity Six months ended June 30, 2024 Shares Amount ​ ​ ​ ​ ​ ​ ​ Balance at December 31, 2023 ​ 57,708,613 ​ $ 1 ​ $ 952,491 ​ $ ( 105 ) ​ $ ( 615,710 ) ​ $ 336,677 Issuance of common stock under equity incentive and employee stock purchase plans 989,254 — ​ 10,371 — ​ — 10,371 Shares withheld for net settlement of tax withholding upon vesting of restricted stock units ​ ( 20,793 ) ​ ​ — ​ ​ ( 600 ) ​ ​ — ​ ​ — ​ ​ ( 600 ) Issuance of common stock upon exercise of Pre-Funded Warrants ​ 84,989 ​ ​ ​ ​ ​ — ​ ​ ​ ​ ​ ​ ​ ​ — Stock-based compensation expense — — ​ 18,296 — ​ — 18,296 Other comprehensive loss — — ​ — ( 144 ) ​ — ( 144 ) Net income — — ​ — — ​ 176,724 176,724 Balance at June 30, 2024 58,762,063 $ 1 ​ $ 980,558 $ ( 249 ) ​ $ ( 438,986 ) $ 541,324 ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. ​ 5 Table of Contents ​ PROTAGONIST THERAPEUTICS, INC. Condensed Consolidated Statements of Cash Flows (Unaudited) (In thousands) ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Six Months Ended ​ ​ June 30, ​ 2025 2024 Cash Flows from Operating Activities ​ ​ ​ Net (loss) income ​ $ ( 46,426 ) ​ $ 176,724 Adjustments to reconcile net (loss) income to net cash provided by operating activities: ​ ​ ​ ​ ​ ​ Stock-based compensation ​ ​ 24,714 ​ ​ 18,296 Non-cash lease expense ​ ​ 1,137 ​ ​ 1,235 Depreciation ​ ​ 543 ​ ​ 477 Accretion of discount on marketable securities ​ ​ ( 4,203 ) ​ ​ ( 3,277 ) Net realized loss on sale of marketable securities ​ ​ 5 ​ ​ — Changes in operating assets and liabilities: ​ ​ ​ ​ ​ ​ Receivable from collaboration partner ​ ​ 165,000 ​ ​ 9,957 Contract asset ​ ​ ( 22,287 ) ​ ​ — Prepaid expenses and other assets ​ ​ ( 2,045 ) ​ ​ ( 2,925 ) Accounts payable ​ ​ 1,612 ​ ​ 2,627 Accrued expenses and other payables ​ ​ ( 8,378 ) ​ ​ ( 4,229 ) Payable to collaboration partner ​ ​ — ​ ​ ( 3 ) Income taxes payable ​ ​ ( 2,689 ) ​ ​ 2,650 Deferred revenue ​ ​ ( 10,504 ) ​ ​ 40,880 Operating lease liability ​ ​ 104 ​ ​ ( 1,219 ) Net cash provided by operating activities ​ ​ 96,583 ​ ​ 241,193 Cash Flows from Investing Activities ​ ​ ​ ​ ​ ​ Purchase of marketable securities ​ ​ ( 281,194 ) ​ ​ ( 240,627 ) Proceeds from maturities of marketable securities ​ ​ 235,957 ​ ​ 158,849 Proceeds from sale of marketable securities ​ ​ 7,003 ​ ​ — Purchases of property and equipment ​ ​ ( 1,358 ) ​ ​ ( 270 ) Net cash used in investing activities ​ ​ ( 39,592 ) ​ ​ ( 82,048 ) Cash Flows from Financing Activities ​ ​ ​ ​ ​ ​ Proceeds from issuance of common stock upon exercise of stock options and purchases under employee stock purchase plan ​ ​ 14,847 ​ ​ 10,371 Tax withholding payments related to net settlement of restricted stock units ​ ​ ( 479 ) ​ ​ ( 600 ) Net cash provided by financing activities ​ ​ 14,368 ​ ​ 9,771 Net increase in cash, cash equivalents and restricted cash ​ ​ 71,359 ​ ​ 168,916 Cash, cash equivalents and restricted cash, beginning of period ​ 97,474 ​ 186,952 Cash, cash equivalents and restricted cash, end of period ​ $ 168,833 ​ $ 355,868 Supplemental Disclosure of Non-Cash Financing and Investing Information: ​ ​ ​ ​ ​ ​ Purchases of property and equipment in accounts payable and accrued liabilities ​ $ 103 ​ $ 275 Right-of-use asset obtained in exchange for lease obligation ​ $ — ​ $ 10,511 Leasehold improvements obtained under tenant improvement allowance ​ $ — ​ $ 516 ​ The accompanying notes are an integral part of these unaudited condensed consolidated financial statements. ​ ​ ​ 6 Table of Contents PROTAGONIST THERAPEUTICS, INC. Notes to Unaudited Condensed Consolidated Financial Statements Note 1. Organization and Description of Business Protagonist Therapeutics, Inc. (the “Company”) is a discovery through late-stage development biopharmaceutical company focused on peptide therapeutics. The Company’s clinical programs fall into two broad categories of diseases: (i) inflammatory and immunomodulatory (“I&I”) diseases and (ii) hematology and blood disorders. Two novel peptides derived from the Company’s proprietary discovery technology platform, icotrokinra and rusfertide, are currently in advanced Phase 3 clinical development. Icotrokinra is a first-in-class investigational targeted oral peptide that selectively blocks the Interleukin-23 receptor (“IL-23R”) and is licensed to J&J Innovative Medicines (“JNJ”), formerly Janssen Biotech, Inc. Following icotrokinra’s joint discovery by the Company and JNJ scientists pursuant to their IL-23R collaboration, the Company was primarily responsible for the development of icotrokinra through Phase 1, with JNJ assuming responsibility for development in Phase 2 and beyond. In July 2025, a New Drug Application (“NDA”) was submitted to the U.S. Food and Drug Administration (“FDA”) by JNJ seeking the first approval of icotrokinra for the treatment of adults and pediatric patients 12 years of age or older with moderate-to-severe plaque psoriasis. Rusfertide, a first-in-class investigational injectable mimetic of the natural hormone hepcidin, is currently in development for treatment of the rare blood disorder polycythemia vera (“PV”). Rusfertide is being co-developed and will be co-commercialized with Takeda Pharmaceuticals, Inc. (“Takeda”), with the Company remaining primarily responsible for clinical development through a potential NDA filing. The Company also has a number of pre-clinical stage drug discovery programs addressing biologically and commercially validated targets, including IL-17 oral peptide antagonist PN-881, obesity triple agonist peptide PN-477, and oral hepcidin. The Company is headquartered in Newark, California and has one wholly owned subsidiary, Protagonist Pty Limited (“Protagonist Australia”), located in Brisbane, Queensland, Australia. Operating Segments Operating segments are components of an enterprise for which separate financial information is available and is evaluated regularly by the Chief Executive Officer, the Company’s chief operating decision maker (“CODM”), in deciding how to allocate resources and assessing performance. The Company operates and manages its business as one operating segment. The Company’s Chief Executive Officer reviews financial information on an aggregate basis for the purpose of allocating and evaluating financial performance. See Note 10 to these condensed consolidated financial statements for the Company’s interim disclosures related to the adoption of ASU 2023-07 Segment Reporting (Topic 280) Improvements to Reporting Segment Disclosures for interim periods beginning on January 1, 2025. Liquidity As of June 30, 2025, the Company had cash, cash equivalents and marketable securities of $ 673.0 million. The Company has incurred an accumulated deficit from inception through June 30, 2025 of $ 386.9 million. The Company’s ultimate success depends upon the outcome of its research and development and collaboration activities. The Company may incur additional losses in the future as it continues rusfertide Phase 3 development activities through a potential NDA filing and invests in its pre-clinical discovery programs and may need to raise additional capital to continue to execute its long-range business plan. Since the Company’s initial public offering in August 2016, it has financed its operations primarily through proceeds from offerings of common stock and payments received under license and collaboration agreements. 7 Table of Contents Note 2. Summary of Significant Accounting Policies Basis of Presentation and Consolidation The accompanying unaudited condensed consolidated financial statements have been prepared in accordance with generally accepted accounting principles in the United States (“GAAP”), the instructions to Form 10-Q and Rule 10-01 of Regulation S-X and applicable rules and regulations of the Securities and Exchange Commission (“SEC”) regarding interim financial reporting. As permitted under those rules, certain footnotes or other financial information that are normally required by GAAP have been condensed or omitted and, accordingly, the condensed consolidated balance sheet as of June 30, 2025 has been derived from the Company’s unaudited consolidated financial statements at that date but does not include all of the information required by GAAP for complete consolidated financial statements. These unaudited interim condensed consolidated financial statements have been prepared on the same basis as the Company’s annual consolidated financial statements and, in the opinion of management, reflect all adjustments (consisting of normal recurring adjustments) that are necessary for a fair presentation of the Company’s condensed consolidated financial statements. The results of operations for the three and six months ended June 30, 2025 are not necessarily indicative of the results to be expected for the year ending December 31, 2025 or for any future period. The accompanying unaudited condensed consolidated financial statements and related financial information should be read in conjunction with the audited consolidated financial statements and the related notes thereto for the year ended December 31, 2024 included in the Company’s Annual Report on Form 10-K, filed with the SEC on February 21, 2025. Principles of Consolidation The accompanying unaudited interim condensed consolidated financial statements include the accounts of the Company and its wholly owned subsidiary. All intercompany transactions and balances have been eliminated upon consolidation. Use of Estimates The preparation of the condensed consolidated financial statements in conformity with GAAP requires management to make estimates, assumptions and judgments that affect the reported amounts of assets and liabilities and disclosure of contingent liabilities as of the date of the condensed consolidated financial statements and the reported amounts of revenues and expenses during the reporting period. On an ongoing basis, management evaluates its estimates, including those related to revenue recognition, accruals for research and development activities, stock-based compensation, income taxes, marketable securities and leases. Estimates related to revenue recognition include assumptions used to determine standalone selling price utilized to allocate the transaction price between distinct performance obligations, assumptions used to recognize revenue over time for certain performance obligations for which a cost-based input method is used as the measure of progress and estimates of whether contingent consideration should be included in the transaction price at each reporting period. Management bases these estimates on historical and anticipated results, trends, and various other assumptions that the Company believes are reasonable under the circumstances, including assumptions as to forecasted amounts and future events. Actual results may differ materially from these estimates. There has been uncertainty and disruption in the global economy and financial markets due to a number of factors, including but not limited to geopolitical instability, high interest rates, and changes in trade policies, including tariffs or other trade restrictions or the threat of such actions and retaliatory actions. Our business may also be impacted by changes or disruptions at the FDA and other government agencies. The Company has taken into consideration any known impacts to its accounting estimates to date and is not aware of any additional specific events or circumstances that would require any additional updates to its estimates or judgments or a revision of the carrying value of its assets or liabilities as of the filing date of this Quarterly Report on Form 10-Q. These estimates may change as new events occur and additional information is obtained. Actual results could differ materially from these estimates under different assumptions or conditions. 8 Table of Contents Cash as Reported in Condensed Consolidated Statements of Cash Flows Cash as reported in the condensed consolidated statements of cash flows includes the aggregate amounts of cash and cash equivalents and the restricted cash as presented on the condensed consolidated balance sheets. Cash as reported in the condensed consolidated statements of cash flows consisted of (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ 2025 2024 Cash and cash equivalents ​ $ 168,543 ​ $ 355,643 Restricted cash - noncurrent ​ 290 ​ 225 Total cash reported on condensed consolidated statements of cash flows ​ $ 168,833 ​ $ 355,868 ​ The increase in restricted cash – noncurrent as of June 30, 2025 was due to a $ 65.0 thousand deposit held as security in connection with the issuance of a bank guarantee in May 2025 to maintain the active status of the Company’s value-added tax registration. ​ Stock-Based Compensation Expense ​ The Company has granted stock options, restricted stock units (“RSUs”) and performance stock units (“PSUs”). ​ Stock-based compensation expense associated with stock options is based on the estimated grant date fair value using the Black-Scholes valuation model, which requires the use of subjective assumptions related to expected stock price volatility, option term, risk-free interest rate and dividend yield. The Company recognizes compensation expense over the vesting period of the awards that are ultimately expected to vest. ​ Stock-based compensation expense associated with RSUs is based on the fair value of the Company’s common stock on the grant date, which equals the closing market price of the Company’s common stock on the grant date. For RSUs, the Company recognizes compensation expense over the vesting period of the awards that are ultimately expected to vest. ​ PSUs allow the recipients of such awards to earn fully vested shares of the Company’s common stock upon the achievement of pre-established performance objectives. Stock-based compensation expense associated with PSUs is based on the fair value of the Company’s common stock on the grant date, which equals the closing market price of the Company’s common stock on the grant date and is recognized when the performance objective is expected to be achieved. The Company evaluates the probability of achieving the performance criteria on a quarterly basis. The cumulative effect on current and prior periods of a change in the estimated number of PSUs expected to be earned is recognized as compensation expense or as reduction of previously recognized compensation expense in the period of the revised estimate. The Company recognized $ 1.8 million of stock-based compensation expense related to PSUs for the six months ended June 30, 2025. ​ The Company recognizes forfeitures of stock-based awards as they occur. ​ Total stock-based compensation expense was as follows (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2025 2024 2025 2024 Research and development ​ $ 6,291 ​ $ 5,097 ​ $ 14,282 ​ $ 10,385 General and administrative ​ 4,621 ​ 3,847 ​ 10,432 ​ 7,911 Total stock-based compensation expense ​ $ 10,912 ​ $ 8,944 ​ $ 24,714 ​ $ 18,296 ​ ​ 9 Table of Contents Significant Accounting Policies ​ There have been no material changes to the Company’s significant accounting policies during the three and six months ended June 30, 2025, as compared to those disclosed in Note 2. Summary of Significant Accounting Policies included in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024. ​ Recently Issued Accounting Pronouncements Not Yet Adopted as of June 30, 2025 In December 2023, the Financial Accounting Standard Board (“FASB”) issued Accounting Standards Update No. 2023-09 Income Taxes (Topic 740) – Improvements to Income Tax Disclosures (“ASU 2023-09”), which requires public business entities to disclose specific categories in the income tax rate reconciliation annually and provide additional information for reconciling items that meet a qualitative threshold. ASU 2023-09 also requires that entities disclose annually additional information about income taxes paid and disaggregated information for certain items. ASU 2023-09 is effective for the Company for fiscal years beginning on January 1, 2025. The Company does not expect the adoption of this guidance to have a material impact on its financial position, results of operations or cash flows. ​ In November 2024, the FASB issued Accounting Standards Update No. 2024-03 Income Statement – Reporting Comprehensive Income – Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses (“ASU 2024-03”), which requires detailed disclosures about specified categories of expenses (including employee compensation, depreciation, and amortization) included in certain expense captions presented on the face of the income statement. In January 2025, the FASB issued an update to ASU 2024-03 clarifying that all public business entities should initially adopt the disclosure requirements in the first annual reporting period beginning after December 15, 2026, and interim reporting periods beginning after December 15, 2027. ASU 2024-03 is effective for the Company for fiscal years beginning on January 1, 2027, and for interim periods beginning on January 1, 2028. Early adoption is permitted. The guidance may be applied either (1) prospectively to financial statements issued for reporting periods after the effective date of ASU 2024-03 or (2) retrospectively to all prior periods presented in the financial statements. The Company does not expect the adoption of this guidance to have a material effect on its consolidated financial statements and continues to evaluate disclosure presentation alternatives. ​ Note 3. License and Collaboration Agreements JNJ License and Collaboration Agreement ​ In July 2021, the Company entered into an Amended and Restated License and Collaboration Agreement with JNJ, formerly Janssen Biotech, Inc., which amended and restated the License and Collaboration Agreement, effective July 2017, by and between the Company and JNJ, as amended in May 2019 (together, the “JNJ License and Collaboration Agreement”). The JNJ License and Collaboration Agreement relates to the development, manufacture and commercialization of oral IL-23R antagonist drug candidates and enables JNJ to develop collaboration compounds for multiple indications. Under the JNJ License and Collaboration Agreement, JNJ is required to use commercially reasonable efforts to develop at least one collaboration compound for at least two indications. ​ The JNJ License and Collaboration Agreement was further amended in November 2024 to: ​ ● Increase the milestone payment for a Phase 3 clinical trial of any licensed product for any indication meeting its primary endpoint by $ 50.0 million, from $ 115.0 million to $ 165.0 million; ● Eliminate the $ 35.0 million milestone payment previously due for the acceptance of an NDA filing by the FDA for use of a licensed product for any indication; and ● Eliminate the $ 15.0 million milestone payment previously due for the dosing of the third patient in the first Phase 3 clinical trial of a licensed product for a second indication. 10 Table of Contents The Company earned the $ 165.0 million milestone payment described above during the fourth quarter of 2024. The Company has earned a total of $ 337.5 million in non-refundable payments from JNJ from inception in 2017 through June 30, 2025. ​ Upcoming potential milestones under the JNJ License and Collaboration Agreement include: ● $ 50.0 million upon FDA approval of an NDA in any indication; ● $ 25.0 million upon the acceptance of an NDA filing by the FDA for a second indication; ● $ 45.0 million upon FDA approval of an NDA for a second indication; ● $ 35.0 million upon the acceptance of an NDA filing by the FDA for a third indication; and ● $ 50.0 million upon FDA approval of an NDA for a third indication. Pursuant to the agreement, the Company is eligible to receive future sales milestone payments and tiered royalties on net product sales at percentages ranging from 6 % to 10 %. Takeda Collaboration Agreement In January 2024, the Company entered into a worldwide license and collaboration agreement for rusfertide with Takeda, as amended March 2025 (the “Takeda Collaboration Agreement”), which became effective in March 2024. Pursuant to the Takeda Collaboration Agreement, the Company and Takeda are jointly developing and commercializing rusfertide and potentially other specified second-generation injectable hepcidin mimetic compounds (the “Licensed Products”) in the United States (the “Profit-Share Territory”). Takeda is solely and exclusively responsible for the development and commercialization of the Licensed Products in all other countries (the “Takeda Territory”). The Company and Takeda share the costs of the development, manufacture and commercialization activities for the Licensed Products in the Profit-Share Territory, provided that (i) the Company leads, and is solely responsible for its costs associated with, completion of the ongoing Phase 3 VERIFY trial evaluating rusfertide for the treatment of PV; (ii) Takeda leads, and is solely responsible for its costs associated with, U.S. regulatory and pre-commercialization activities related to rusfertide in the Profit-Share Territory; and (iii) Takeda leads commercialization of rusfertide in the Profit-Share Territory, with the Company holding an option to co-detail. Takeda is solely responsible for all costs for the development, manufacture and commercialization of the Licensed Products in the Takeda Territory. The Company granted Takeda a non-transferable, sublicensable and, except for certain specified exceptions, exclusive license to certain intellectual property of the Company to exercise its rights and perform its obligations under the Takeda Collaboration Agreement. In March 2025, the Company and Takeda agreed, pursuant to the provisions of the Takeda Collaboration Agreement, as amended, that Takeda would assume responsibility for leading and implementing the regulatory strategy and associated activities for preparation of the NDA related to rusfertide in PV, which is expected to be submitted to the FDA. The Company remains primarily responsible for clinical development activities through the NDA filing. ​ The Company received a one-time, non-refundable upfront payment of $ 300.0 million in April 2024. In March 2025, a $ 25.0 million milestone was deemed probable of achievement following positive topline results from the Phase 3 VERIFY trial of rusfertide in PV. In addition, the Company is eligible to receive additional worldwide development, regulatory and commercial milestone payments for rusfertide of up to $ 305.0 million, and tiered royalties from 10 % to 17 % on net sales of the Licensed Products in the Takeda Territory. The Company and Takeda will also share equally in profits and losses ( 50 % to the Company and 50 % to Takeda) for Licensed Products in the Profit-Share Territory. Takeda will book sales of the Licensed Products globally. ​ The Company has the right to opt-out entirely of profit- and loss-sharing in the Profit-Share Territory for rusfertide and all other Licensed Products (the “Full Opt-out Right”) (i) during the 90-day period beginning 120 days after the filing of an NDA with the FDA for rusfertide for PV (the “Initial Opt-out Period”); and (ii) for convenience without receipt of the Opt-out Payment (as defined below) (generally following the Initial Opt-out Period). In addition, if 11 Table of Contents the Company does not exercise the Full Opt-out Right, the Company may opt-out of any Licensed Product other than rusfertide on a Licensed Product-by-Licensed Product basis (each, a “Partial Opt-out Right” and either the Full Opt-out Right or a Partial Opt-out right being an “Opt-out Right”). Following the Company’s exercise of an Opt-out Right, the Company has agreed to transition applicable development and commercial activities to Takeda, and Takeda has agreed to assume sole operational and financial responsibility for such activities in the United States. The Takeda Collaboration Agreement provides for aggregate development, regulatory and commercial milestone payments from Takeda to the Company for rusfertide of up to $ 975.0 million if the Company exercises the Full Opt-out Right. In addition to these milestone payments, in the event the Company exercises the Full Opt-out Right during the Initial Opt-out Period, the Company will receive: (i) a $ 200.0 million payment following its exercise of the Full Opt-out Right; and (ii) an additional $ 200.0 million payment following FDA approval of the NDA for rusfertide for PV (together, the “Opt-out Payment”). If the Company exercises an Opt-out Right, Takeda has agreed to pay the Company royalties of 14 % to 29 % on worldwide net sales of the Licensed Products with respect to which the Company has exercised an Opt-out Right. Upcoming potential milestones under the Takeda Collaboration Agreement include: ● $ 50.0 million upon FDA approval of an NDA for rusfertide in PV (or $ 75.0 million if the Company exercises the Full Opt-out Right); ● $ 15.0 million upon first regulatory approval for rusfertide in PV in three European countries, after pricing and reimbursement approval; and ● $ 10.0 million upon first regulatory approval for rusfertide in PV in Japan. The Company evaluated the Takeda Collaboration Agreement and concluded that it has elements that are within the scope of ASC Topic 606 and ASC Topic 808. As of the effective date of the Takeda Collaboration Agreement, the Company identified two distinct performance obligations: (i) the rusfertide license delivered upon the effectiveness of the Takeda Collaboration Agreement and (ii) certain development services to be provided prior to the Initial Opt-out Period, including the Company’s responsibilities to complete the Phase 3 VERIFY trial in PV and associated manufacturing services. The Company determined that the initial transaction price totaled $ 300.0 million, which was comprised of the upfront payment. The Company initially excluded any future estimated milestones or royalties from this transaction price, all of which were either constrained or subject to the sales-and usage-based royalty exception. As part of the Company’s evaluation of this variable consideration constraint, it determined that the potential payments were contingent upon developmental and regulatory milestones that were uncertain and were highly susceptible to factors outside of its control. The Company allocated $ 254.1 million of the initial transaction price to the license and $ 45.9 million to the development services based upon the relative standalone selling price of each performance obligation. The estimate of standalone selling price for the license was determined based on discounted cash flows for the expected development and commercialization of rusfertide and included assumptions for forecasted revenues, development timelines and expenses, discount rates, and probabilities of technical and regulatory success. The estimate of standalone selling price for the development services was determined based on forecasted costs and expenses over the expected development period. For the license of rusfertide, the Company determined that Takeda could benefit from the license at the time the license was granted and therefore, the related performance obligation was satisfied at that point in time. ​ The amount allocated to the license, which represents functional intellectual property that was transferred at a point in time, was satisfied upon transfer of the license to Takeda. The amount allocated to development services will be recognized over time based on a measure of the Company’s efforts toward satisfying the performance obligation relative to the total expected efforts or inputs to satisfy the performance obligation (e.g., costs incurred compared to total budget). ​ The Company determined that the Takeda Collaboration Agreement met the definition of a collaborative arrangement under ASC Topic 808. Both parties are active participants in directing and carrying out the development of 12 Table of Contents the Licensed Products and both are exposed to the significant risk and rewards related to the commercial success of the Products. If the Company does not exercise an Opt-out Right (“Company Opt-in”), the Company and Takeda would co-detail the Licensed Products in the U.S. and share in the economic results through a profit-sharing structure. The Company determined that development costs subsequent to the Company Opt-in date are within the scope of ASC Topic 808, which does not provide recognition and measurement guidance. As such, the Company determined that Accounting Standards Codification Topic 730, “ Research and Development,” was an appropriate analogy based on the cost-sharing provisions of the agreement. The Company concluded that payments to or reimbursements from Takeda related to these services will be accounted for as an increase to or reduction of research and development expense, respectively. ​ In March 2025, the $ 25.0 million milestone was deemed probable of achievement due to the Phase 3 VERIFY trial meeting its primary endpoint, was no longer considered constrained and was added to the initial transaction price for a total transaction price of $ 325.0 million. The additional $ 25.0 million was then allocated proportionally to each performance obligation under the agreement, resulting in an additional $ 21.3 million allocated to the license and an additional $ 3.7 million allocated to the development services under the agreement. ​ Revenue Recognition ​ For the three months ended June 30, 2025, the Company recognized license and collaboration revenue of $ 5.5 million related to the Takeda Collaboration Agreement, including (i) $ 5.0 million related to the initial transaction price for development services provided by the Company during the period based on the cost-based input method and (ii) $ 0.5 million related to the proportional recognition of the $ 25.0 million milestone deemed probable of being achieved due to the Phase 3 VERIFY trial meeting its primary endpoint. For the six months ended June 30, 2025, the Company recognized license and collaboration revenue of $ 33.9 million related to the Takeda Collaboration Agreement, including (i) $ 23.4 million related to the proportional recognition of the $ 25.0 million milestone deemed probable of being achieved due to the Phase 3 VERIFY trial meeting its primary endpoint and (ii) $ 10.5 million related to the initial transaction price for development services provided by the Company during the period based on the cost-based input method. Revenue recognition for the $ 25.0 million milestone, which is payable upon completion of the VERIFY clinical study report, was allocated based on the initial standalone selling price of each performance obligation under the agreement in March 2025. As of June 30, 2025, the remaining $ 1.6 million in revenue related to the $ 25.0 million milestone will be recognized through the conclusion of the development services performance obligation. The Company recorded a corresponding contract asset of $ 23.4 million on its condensed consolidated balance sheet as of June 30, 2025. ​ For the three months ended June 30, 2024, the Company recognized license and collaboration revenue of $ 4.2 million related to the initial Takeda Collaboration Agreement transaction price for development services provided by the Company during the period based on the cost-based input method. For the six months ended June 30, 2024, the Company recognized license and collaboration revenue of $ 259.1 million related to the Takeda Collaboration Agreement transaction price, including (i) $ 254.1 million allocated to the rusfertide license delivered to Takeda upon effectiveness of the agreement in March 2024 and (ii) $ 5.0 million for development services provided by the Company during the period based on the cost-based input method. The remaining unrecognized transaction price amount of $ 40.9 million related to the Takeda Collaboration Agreement was recorded as deferred revenue on the Company’s condensed consolidated balance sheet as of June 30, 2024 to be recognized over time based on a measure of the Company’s efforts toward satisfying the performance obligation relative to the total expected efforts or inputs to satisfy the performance obligation (e.g. costs incurred compared to total budget). ​ For the three and six months ended June 30, 2025, the Company recognized $ 5.0 million and $ 10.5 million of revenue, respectively, that was included in the deferred revenue liability balance at the beginning of each period. For the three months ended June 30, 2024, the Company recognized $ 4.2 million of revenue that was included in the deferred revenue liability balance at the beginning of the period. For the six months ended June 30, 2024, the Company did no t recognize revenue from any amounts included in the deferred revenue contract liability balance at the beginning of the period. None of the costs to obtain or fulfill the contracts were capitalized. 13 Table of Contents Note 4. Fair Value Measurements Financial assets and liabilities are recorded at fair value. The accounting guidance for fair value provides a framework for measuring fair value, clarifies the definition of fair value and expands disclosures regarding fair value measurements. Fair value is defined as the price that would be received to sell an asset or paid to transfer a liability (an exit price) in an orderly transaction between market participants at the reporting date. The accounting guidance establishes a three-tiered hierarchy, which prioritizes the inputs used in the valuation methodologies in measuring fair value as follows: Level 1 —Inputs are unadjusted quoted prices in active markets for identical assets or liabilities at the measurement date. Level 2— Inputs (other than quoted market prices included in Level 1) are either directly or indirectly observable for the asset or liability through correlation with market data at the measurement date and for the duration of the instrument’s anticipated life. Level 3 —Inputs reflect management’s best estimate of what market participants would use in pricing the asset or liability at the measurement date. Consideration is given to the risk inherent in the valuation technique and the risk inherent in the inputs to the model. In determining fair value, the Company utilizes quoted market prices, broker or dealer quotations, or valuation techniques that maximize the use of observable inputs and minimize the use of unobservable inputs to the extent possible and considers counterparty credit risk in its assessment of fair value. The following tables present the fair value of the Company’s financial assets determined using the inputs defined above (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, 2025 ​ Level 1 Level 2 Level 3 Total Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 68,043 ​ $ — ​ $ — $ 68,043 Certificates of deposit ​ — ​ ​ 25,252 ​ — 25,252 U.S. Treasury and agency securities ​ ​ — ​ ​ 333,488 ​ ​ — ​ ​ 333,488 Commercial paper ​ — ​ ​ 98,608 ​ — 98,608 Corporate debt securities ​ ​ — ​ ​ 140,313 ​ ​ — ​ ​ 140,313 Total financial assets ​ $ 68,043 ​ $ 597,661 $ — $ 665,704 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ December 31, 2024 ​ Level 1 Level 2 Level 3 Total Assets: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Money market funds ​ $ 19,563 ​ $ — ​ $ — $ 19,563 Certificates of deposit ​ — ​ ​ 15,835 ​ — 15,835 U.S. Treasury and agency securities ​ ​ — ​ ​ 299,217 ​ ​ — ​ ​ 299,217 Commercial paper ​ — ​ 110,832 ​ — 110,832 Corporate debt securities ​ — ​ 102,705 — 102,705 Total financial assets ​ $ 19,563 ​ $ 528,589 $ — $ 548,152 ​ The Company’s certificates of deposit, U.S. Treasury and agency securities, including U.S. Treasury bills, commercial paper and corporate debt securities are classified as Level 2 as they were valued based upon quoted market prices for similar instruments in active markets, quoted prices for identical or similar instruments in markets that are not active and model-based valuation techniques, for which all significant inputs are observable in the market or can be corroborated by observable market data for substantially the full term of the assets. 14 Table of Contents The carrying amount of the Company’s remaining financial assets and liabilities, including cash, receivables and payables, approximates their fair value due to their short-term nature. Note 5. Cash Equivalents and Marketable Securities ​ Cash equivalents and marketable securities consisted of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, 2025 ​ ​ Amortized ​ Gross Unrealized ​ ​ Cost Gains Losses Fair Value Money market funds ​ $ 68,043 ​ $ — ​ $ — ​ $ 68,043 Certificates of deposit ​ ​ 25,252 ​ ​ 3 ​ ( 3 ) ​ 25,252 U.S. Treasury and agency securities ​ ​ 333,308 ​ ​ 332 ​ ​ ( 152 ) ​ ​ 333,488 Commercial paper ​ 98,630 ​ ​ — ​ ​ ( 22 ) ​ 98,608 Corporate debt securities ​ ​ 140,345 ​ ​ 23 ​ ​ ( 55 ) ​ ​ 140,313 Total cash equivalents and marketable securities ​ $ 665,578 ​ $ 358 $ ( 232 ) ​ $ 665,704 Classified as: ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash equivalents ​ ​ ​ ​ ​ ​ ​ $ 161,289 Marketable securities - current ​ ​ ​ ​ ​ ​ ​ 401,922 Marketable securities - noncurrent ​ ​ ​ ​ ​ ​ ​ 102,493 Total cash equivalents and marketable securities ​ ​ ​ ​ ​ ​ ​ $ 665,704 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ December 31, 2024 ​ ​ Amortized ​ Gross Unrealized ​ ​ Cost Gains Losses Fair Value Money market funds ​ $ 19,563 ​ $ — $ — ​ $ 19,563 Certificates of deposit ​ ​ 15,820 ​ ​ 22 ​ ​ ( 7 ) ​ 15,835 U.S. Treasury and agency securities ​ ​ 299,211 ​ ​ 429 ​ ​ ( 423 ) ​ ​ 299,217 Commercial paper ​ 110,815 ​ 28 ( 11 ) ​ 110,832 Corporate debt securities ​ ​ 102,714 ​ 103 ( 112 ) ​ 102,705 Total cash equivalents and marketable securities ​ $ 548,123 ​ $ 582 $ ( 553 ) ​ $ 548,152 Classified as: ​ ​ ​ ​ ​ ​ ​ ​ ​ Cash equivalents ​ ​ ​ ​ ​ ​ ​ $ 86,236 Marketable securities - current ​ ​ ​ ​ ​ ​ ​ 321,664 Marketable securities - noncurrent ​ ​ ​ ​ ​ ​ ​ 140,252 Total cash equivalents and marketable securities ​ ​ ​ ​ ​ ​ ​ $ 548,152 ​ All of the Company’s marketable securities are classified as available-for-sale. Current marketable securities of $ 401.9 million and $ 321.7 million held as of June 30, 2025 and December 31, 2024, respectively, had contractual maturities of less than one year . Noncurrent marketable securities of $ 102.5 million and $ 140.3 million held as of June 30, 2025 and December 31, 2024, respectively, had contractual maturities of at least one year but no more than two years . The Company does not intend to sell its securities that are in an unrealized loss position, and it is not more likely than not that the Company will be required to sell its securities before recovery of their amortized cost basis, which may be at maturity. ​ During the six months ended June 30, 2025, the Company sold $ 7.0 million of marketable securities and recognized a net realized loss of $ 5.0 thousand. There were no realized gains or realized losses on marketable securities for the three months ended June 30, 2025 and three and six months ended June 30, 2024. The Company evaluated securities with unrealized losses to determine whether such losses, if any, were due to credit-related factors and determined that there were no credit-related losses to be recognized as of June 30, 2025 and December 31, 2024. ​ 15 Table of Contents Note 6. Balance Sheet Components Prepaid Expenses and Other Current Assets Prepaid expenses and other current assets consisted of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2025 ​ 2024 Accrued interest receivable ​ $ 4,516 ​ $ 3,242 Prepaid clinical and research related expenses ​ ​ 1,805 ​ ​ 1,830 Prepaid licenses ​ ​ 819 ​ ​ 600 Prepaid insurance ​ ​ 582 ​ ​ 1,159 Other prepaid expenses ​ 884 ​ 649 Other receivable ​ ​ 92 ​ ​ 248 Prepaid expenses and other current assets ​ $ 8,698 ​ $ 7,728 ​ Property and Equipment, Net ​ Property and equipment, net consisted of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ ​ 2025 ​ 2024 Laboratory equipment ​ $ 7,309 ​ $ 6,354 Furniture and computer equipment ​ 1,455 ​ 1,447 Leasehold improvements ​ 2,922 ​ 2,424 Total property and equipment ​ 11,686 ​ 10,225 Accumulated depreciation ​ ( 7,578 ) ​ ( 7,035 ) Property and equipment, net ​ $ 4,108 ​ $ 3,190 ​ Accrued Expenses and Other Payables ​ Accrued expenses and other payables consisted of the following (in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ June 30, ​ December 31, ​ 2025 ​ 2024 Accrued clinical and research related expenses ​ $ 9,556 ​ $ 11,923 Accrued employee related expenses ​ 5,006 ​ 11,078 Accrued professional service fees ​ ​ 676 ​ ​ 618 Other ​ 159 ​ 74 Total accrued expenses and other payables ​ $ 15,397 ​ $ 23,693 ​ ​ Note 7. Stockholders’ Equity Shares of Common Stock Authorized for Issuance At the Company’s 2024 Annual Meeting of Stockholders held on June 20, 2024, the Company’s stockholders approved an amendment to the Company’s Amended and Restated Certificate of Incorporation (the “Certificate of Incorporation”) to increase the number of authorized shares of the Company’s common stock from 90,000,000 to 180,000,000 , which also has the effect of increasing the total number of authorized shares from 100,000,000 to 190,000,000 (the “Amendment”). On June 21, 2024, the Company filed a Certificate of Amendment to the Certificate of Incorporation with the Secretary of State of the State of Delaware, which became effective immediately upon such filing. 16 Table of Contents Pre-Funded Warrants In August 2018, the Company entered into a Securities Purchase Agreement with certain accredited investors (each, an “Investor” and, collectively, the “Investors”). In a concurrent private placement, the Company issued the Investors warrants to purchase an aggregate of 2,750,000 shares of its common stock (each, a “Warrant” and, collectively, the “Warrants”). Each Warrant was exercisable from August 8, 2018 through August 8, 2023 . In August 2023, prior to the expiration of the Warrants, the Company entered into certain agreements with the Investors and their affiliates under which the Company agreed to allow the Warrants to be exercised in exchange for pre-funded warrants representing the same number of Warrant Shares underlying the Warrants with an exercise price of $ 0.001 per share (the “Pre-Funded Warrants”). Subsequent to the execution of the agreements and prior to the expiration of the Warrants in August 2023, all outstanding Warrants were exercised for gross proceeds of $ 34.4 million in exchange for 44,748 shares of the Company’s common stock and Pre-Funded Warrants to purchase 2,705,252 shares of common stock (subject to adjustment in the event of any stock dividends and splits, reverse stock split, recapitalization, reorganization or similar transaction, as described in the Pre-Funded Warrants) with an exercise price of $ 0.001 per share. The Pre-Funded Warrants will expire on the day they are exercised in full. The Pre-Funded Warrants are exercisable at any time prior to expiration except that the Pre-Funded Warrants cannot be exercised by the Investors if, after giving effect thereto, the Investors would beneficially own more than 9.99 % of the Company’s common stock, subject to certain exceptions. In accordance with ASC Topic 260, “ Earnings Per Share” , outstanding Pre-Funded Warrants are included in the computation of basic net (loss) income per share because the exercise price is negligible, and they are fully vested and exercisable after the original issuance date. No Pre-Funded Warrants were exercised during the six months ended June 30, 2025. During the six months ended June 30, 2024, Pre-Funded Warrants to purchase 84,992 shares were net exercised, resulting in the issuance of 84,989 shares of common stock. As of June 30, 2025, Pre-Funded Warrants to purchase 1,500,000 shares of common stock remained outstanding. Note 8. Income Taxes The Company recorded income tax expense of $ 0.2 million for the three and six months ended June 30, 2025, which consisted of adjustments to estimated tax payments. The Company recorded an income tax benefit of $ 0.7 million and income tax expense of $ 2.7 million for the three and six months ended June 30, 2024, respectively. The primary difference in income tax expense as compared to the prior year was due to taxable income for the six months ended June 30, 2024 resulting from the recognition of revenue in connection with the Takeda Collaboration Agreement. The tax provision for the three and six months ended June 30, 2024 was determined using an estimated annual effective tax rate, adjusted for discrete items, if any. Note 9. Net (Loss) Income per Share The computation of basic net (loss) income per share of common stock is based on the weighted-average number of shares of common stock outstanding during each period. The computation of diluted net (loss) income per share of common stock is based on the weighted-average number of shares of common stock outstanding during the period plus, when their effect is dilutive, incremental shares consisting of shares subject to stock options, RSUs, PSUs, the Company’s employee stock purchase plan (“ESPP”), and warrants. In periods in which the Company reports a net loss, all common stock equivalents are deemed anti-dilutive such that basic net loss per share of common stock and diluted net loss per share of common stock are equal. In periods when the Company has net income, the dilutive effect of all potentially outstanding shares is computed using the treasury stock method. ​ 17 Table of Contents The following table reconciles the numerator and denominator used to calculate diluted net (loss) income per share of common stock (in thousands, except share and per share data): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2025 2024 2025 2024 Numerator: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Net (loss) income ​ $ ( 34,771 ) ​ $ ( 30,616 ) ​ $ ( 46,426 ) ​ $ 176,724 Denominator: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Weighted-average shares of common stock, basic ​ 63,510,537 ​ 61,305,289 ​ 63,238,682 61,080,489 Dilutive effect of common stock equivalents ​ — ​ — ​ — 2,829,144 Weighted-average shares of common stock, dilutive ​ ​ 63,510,537 ​ ​ 61,305,289 ​ ​ 63,238,682 ​ ​ 63,909,633 Net (loss) income per share of common stock ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Basic net (loss) income per share of common stock ​ $ ( 0.55 ) ​ $ ( 0.50 ) ​ $ ( 0.73 ) $ 2.89 Diluted net (loss) income per share of common stock ​ $ ( 0.55 ) ​ $ ( 0.50 ) ​ $ ( 0.73 ) ​ $ 2.77 ​ Approximately 9.5 million potentially dilutive shares of common stock (consisting of shares subject to outstanding stock options, RSUs, PSUs, and under the ESPP) were excluded from the diluted net loss per share of common stock computation for the three and six months ended June 30, 2025 due to the Company’s net losses for these periods. Approximately 9.9 million potentially dilutive shares of common stock (consisting of shares subject to outstanding stock options, RSUs, PSUs, and under the ESPP) were excluded from the diluted net loss per share of common stock computation for the three months ended June 30, 2024 due to the Company’s net loss for the period. Approximately 3.4 million potentially dilutive shares of common stock (consisting of shares subject to outstanding stock options, RSUs, PSUs, and under the ESPP) were excluded from the diluted net income per share of common stock computation for the six months ended June 30, 2024 because their effect was anti-dilutive. ​ Note 10. Segment Reporting Operating segments are components of an enterprise for which separate financial information is available and which are evaluated by a company’s CODM in deciding how to allocate resources and to assess performance. The Company operates and manages its business as one operating segment, which primarily focuses on the discovery and development of innovative medicines in areas of unmet medical need. The Company’s Chief Executive Officer serves as the Company’s CODM and manages and allocates resources to the operations of the Company on an entity-wide basis. Managing and allocating resources on an entity-wide basis enables the CODM to assess the overall level of resources available and how to best deploy these resources across functions and research and development projects based on unmet medical need, scientific data, probability of technical and regulatory successful development, market potential and other considerations, and, as necessary, reallocate resources among our internal research and development portfolio and external opportunities to best support the long-term growth of our business. The Company’s CODM reviews financial information on an aggregate basis for the purpose of allocating resources and evaluating financial performance, including segment net (loss) income, which is also reported on the condensed consolidated statement of operations as consolidated net (loss) income. 18 Table of Contents Segment information is as follows (dollars in thousands): ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Three Months Ended ​ Six Months Ended ​ ​ June 30, ​ June 30, ​ 2025 2024 2025 2024 ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Revenue ​ $ 5,546 ​ $ 4,167 ​ $ 33,867 ​ $ 259,120 Less: ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Discovery department expense (1)(2) ​ ​ ( 4,952 ) ​ ​ ( 3,832 ) ​ ​ ( 8,698 ) ​ ​ ( 6,492 ) Development department expense (1)(2) ​ ​ ( 15,504 ) ​ ​ ( 14,655 ) ​ ​ ( 28,409 ) ​ ​ ( 29,426 ) General and administrative expenses (1) ​ ​ ( 5,890 ) ​ ​ ( 5,051 ) ​ ​ ( 11,425 ) ​ ​ ( 14,975 ) Employee wages and benefits - discovery (2) ​ ​ ( 2,188 ) ​ ​ ( 1,808 ) ​ ​ ( 4,515 ) ​ ​ ( 3,677 ) Employee wages and benefits - development (2) ​ ​ ( 4,658 ) ​ ​ ( 5,268 ) ​ ​ ( 10,142 ) ​ ​ ( 11,646 ) Employee wages and benefits - general and administrative ​ ​ ( 3,483 ) ​ ​ ( 3,402 ) ​ ​ ( 7,315 ) ​ ​ ( 7,092 ) Stock-based compensation expense ​ ​ ( 10,912 ) ​ ​ ( 8,944 ) ​ ​ ( 24,714 ) ​ ​ ( 18,296 ) Other segment items (3) ​ ​ 36 ​ ​ 97 ​ ​ 118 ​ ​ 78 Interest income ​ ​ 7,406 ​ ​ 7,404 ​ ​ 14,979 ​ ​ 11,780 Income tax (expense) benefit ​ ​ ( 172 ) ​ ​ 676 ​ ​ ( 172 ) ​ ​ ( 2,650 ) Segment (loss) profit ​ $ ( 34,771 ) ​ $ ( 30,616 ) ​ $ ( 46,426 ) ​ $ 176,724 Reconciliation of (loss) profit ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ ​ Adjustments and reconciling items ​ $ — ​ $ — ​ $ — ​ $ — Consolidated net (loss) income ​ $ ( 34,771 ) ​ $ ( 30,616 ) ​ $ ( 46,426 ) ​ $ 176,724 (1) Amounts exclude employee wages and benefits, stock-based compensation and expense allocations. (2) As of April 1, 2025, the information regularly provided to the CODM was changed to reclassify pre-clinical expenses from development expense to discovery expense. Prior period segment information has been recast to reflect this change. (3) Other segment items include foreign currency related income (expense) and other miscellaneous income (expense). ​ The accounting policies of the Company’s operating segment are the same as those described in Note 2. Summary of Significant Accounting Policies. The measure of segment assets is reported as total assets on the Company’s condensed consolidated balance sheets for the periods presented. ​ 19 Table of Contents