Disc Medicine(IRON) - 2025 Q2 - Quarterly Results

Introduction This section provides an overview of Disc Medicine, its recent financial performance, and strategic pipeline advancements Company Overview and CEO Statement Disc Medicine reported Q2 2025 results, advancing bitopertin NDA and other pipeline programs, with cash runway into 2028 - Disc Medicine is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing novel treatments for patients suffering from serious hematologic diseases [2][11] - On track to submit an NDA for bitopertin in EPP under the accelerated approval pathway in October 2025, following positive feedback from a pre-NDA meeting [3][6] - Initiated a Phase 2 study of DISC-3405 in polycythemia vera and expects data from the Phase 2 trial of DISC-0974 in MF anemia and Phase 1b trial in NDD-CKD anemia in the second half of the year [3][6] - Maintains a strong balance sheet that provides cash runway into 2028, positioning the company for potential commercialization of bitopertin and pipeline advancement [3][6] Recent Highlights and Anticipated Milestones This section details Disc Medicine's progress across its key clinical programs and corporate developments, outlining significant achievements and upcoming milestones Bitopertin Program Update Bitopertin is on track for EPP NDA submission in October 2025, supported by positive pre-NDA feedback and favorable HELIOS data - Company on track to submit NDA for bitopertin in erythropoietic protoporphyria (EPP) under accelerated approval pathway in October 2025, supported by successful pre-NDA meeting [6][7] - Presented data from HELIOS, an ongoing open-label extension study, showing favorable long-term efficacy and safety with sustained protoporphyrin IX (PPIX) reductions, improvement in quality of life, and improved liver biomarkers [6][7] - Progressing confirmatory Phase 3 APOLLO clinical trial of bitopertin in adults and adolescents with EPP [7] - Publication of preclinical study results showing bitopertin may help prevent liver disease in EPP, in addition to ameliorating blood PPIX levels [7] DISC-0974 Program Update DISC-0974 is progressing with initial data expected in Q4 2025 from Phase 2 MF anemia and Phase 1b NDD-CKD - Expect initial data from Phase 2 study of DISC-0974 in patients with anemia of myelofibrosis (MF) and multiple dose data from Phase 1b study in NDD-CKD in Q4 2025 [6][13] - Presented clinical data from the continuation phase of the Phase 1b trial of DISC-0974 in MF anemia demonstrating durable hematologic response at EHA 2025 [6][13] - RALLY-MF Phase 2 study's exploratory cohort for patients on concomitant momelotinib or pacritinib is fully enrolled, and the trial protocol was updated to allow these patients into main study cohorts [13] DISC-3405 Program Update DISC-3405 initiated a Phase 2 study in PV with initial data expected in 2026, backed by Phase 1 proof-of-mechanism data - Initiated a Phase 2 study of DISC-3405 in patients with PV with initial data expected in 2026 [6][13] - Presented updated SAD/MAD data from the Phase 1 trial of DISC-3405 in healthy volunteers providing proof of mechanism to support advancement of the program at EHA 2025 [6][13] Corporate Developments Nadim Ahmed joined Disc Medicine's Board in July 2025, bringing extensive hematology development and commercial leadership - Appointed Nadim Ahmed, President and CEO of Cullinan Therapeutics, to the Company's Board of Directors in July, bringing over 25 years of development and commercial leadership experience including multiple product launches in the hematology space [9] Second Quarter 2025 Financial Results This section presents Disc Medicine's financial performance for the second quarter of 2025, detailing key income statement and balance sheet figures Financial Summary Disc Medicine reported a $55.2 million net loss in Q2 2025, driven by higher operating costs, maintaining $650.0 million cash into 2028 Financial Metric (Q2 2025) | Financial Metric (Q2 2025) | 2025 (in millions) | 2024 (in millions) | YoY Change | | :------------------------- | :----------------- | :----------------- | :--------- | | Research and Development | $46.3 | $23.5 | +97.0% | | Selling, General & Admin | $15.1 | $7.4 | +104.1% | | Net Loss | $(55.2) | $(26.4) | +109.1% | - Cash, cash equivalents, and marketable securities were $650.0 million as of June 30, 2025, which are expected to fund operational plans into 2028 [6][13] - The increase in R&D expenses was primarily driven by the progression of Disc's portfolio, including bitopertin's clinical studies and drug manufacturing, the advancement of the DISC-0974 program, increased headcount, and a $10 million milestone payment upon initiation of the APOLLO study [13] - The increase in SG&A expenses was primarily due to increased headcount including establishing infrastructure to support potential commercialization [13] Condensed Consolidated Statements of Operations The company reported a net loss of $55.2 million for Q2 2025, up from $26.4 million in Q2 2024, due to higher R&D and SG&A Statements of Operations (in thousands) | Metric (in thousands) | Three months ended June 30, 2025 | Three months ended June 30, 2024 | Six months ended June 30, 2025 | Six months ended June 30, 2024 | | :-------------------- | :------------------------------- | :------------------------------- | :----------------------------- | :----------------------------- | | Research and development | $46,319 | $23,485 | $74,082 | $47,189 | | Selling, general and administrative | $15,091 | $7,367 | $27,274 | $15,125 | | Total operating expenses | $61,410 | $30,852 | $101,356 | $62,314 | | Loss from operations | $(61,410) | $(30,852) | $(101,356) | $(62,314) | | Other income (expense), net | $6,215 | $4,560 | $12,195 | $9,078 | | Income tax expense | $(52) | $(60) | $(171) | $(65) | | Net loss | $(55,247) | $(26,352) | $(89,332) | $(53,301) | | Net loss per share, basic and diluted | $(1.58) | $(1.03) | $(2.61) | $(2.11) | | Weighted-average common shares outstanding, basic and diluted | 35,024,592 | 25,649,043 | 34,179,364 | 25,229,456 | Condensed Consolidated Balance Sheets Total assets increased to $665.1 million as of June 30, 2025, driven by $650.0 million cash, with equity rising to $614.2 million Balance Sheets (in thousands) | Metric (in thousands) | June 30, 2025 (Unaudited) | December 31, 2024 | | :-------------------- | :------------------------ | :---------------- | | Cash, cash equivalents, and marketable securities | $649,973 | $489,881 | | Total current assets | $661,592 | $493,615 | | Total assets | $665,061 | $496,773 | | Total liabilities | $50,896 | $53,186 | | Total stockholders' equity