Disc Medicine (NasdaqGM:IRON) FY Conference Transcript

Summary of Disc Medicine Conference Call Company Overview - Company: Disc Medicine - Ticker Symbol: IRON - Public Trading Since: End of 2022 - Focus: Development of therapies for blood disorders, particularly related to iron and heme metabolism [4][3] Key Programs and Developments Bitopertin - Indication: Erythropoietic protoporphyria (EPP) - Regulatory Status: Received a Commissioner's National Priority Voucher (CNPV), projecting potential approval by end of 2023 or early 2024 [2][5] - Clinical Trials: Completed a Phase 2 program with 100 participants; FDA aligned on an accelerated approval pathway using the toxic metabolite PPIX as a surrogate endpoint [5][6] - Sales Force: Planning to hire 24 sales representatives targeting 6,000 engaged patients identified through claims data [9][11] - Market Size: Estimated 14,000 diagnosed patients in the US, with a potential market size of $800 million to $1.4 billion [50] Competitive Landscape - Existing Treatment: Scenesse, which requires surgical implantation every two months, limiting its accessibility to about 3% of patients [12][14] - Pricing Comparison: Scenesse costs approximately $300,000 annually, while another treatment, Givlaari, is priced at $575,000 [18] Apollo Study - Confirmatory Trial: Enrolling 150 patients across the US, Europe, Canada, and Australia, expected to complete enrollment in about one year [21] - Endpoints: Co-primary endpoints include PPIX reduction and a measure of time in light at the end of the six-month trial [21] Other Assets 0974 - Mechanism: Monoclonal antibody targeting hemojuvelin to release iron for red blood cell production, primarily for myelofibrosis [22][23] - Phase 1b Results: Showed response rates of 50% in non-transfused patients, 80% in low transfusion burden patients, and 40% in high transfusion burden patients [26] - Next Steps: Interim results expected at ASH; aiming for a pivotal trial post-data analysis [37] Polycythemia Vera Program - Goal: Achieve iron restriction with a patient-friendly administration schedule [43] - Phase 2 Study: Aiming for a target hematocrit of 45% without phlebotomy, with a benchmark set against Rusfertide's 75% achievement [44][46] Financial Overview - Cash Position: Approximately $826 million post-stock offering, providing a runway until early 2029 without revenue from bitopertin [49] - Investment Outlook: Strong financial backing supports ongoing clinical trials and potential market entry [49] Additional Considerations - Patient Engagement: Focus on engaging the 6,000 identified patients initially, with broader outreach planned for the remaining diagnosed population [11] - Regulatory Strategy: Discussions with the FDA regarding the use of existing drug batches for potential early launch [6][8] This summary encapsulates the critical aspects of Disc Medicine's current status, focusing on their lead program, competitive landscape, financial health, and future plans.