Summary of Disc Medicine Conference Call Company Overview - **Company**: Disc Medicine - **Focus**: Red blood cell biology, specifically manipulating heme and iron metabolism to address various diseases, including rare and common anemias [3][4] Core Programs - **Lead Program**: Bitopertin for Erythropoietic Protoporphyria (EPP) - **Patient Population**: Estimated 14,000 patients in the U.S. with EPP [6] - **NDA Status**: Submitted and under accelerated review, with anticipated approval by late January or early February [7][14] - **Clinical Data**: - Reduction of protoporphyrin IX (PP-9) by 50%-60% in trials [10] - 75% reduction in phototoxic reactions in the Aurora study [12] - High patient satisfaction with reported improvements in disease state [13] - **Pipeline Programs**: - **DISC-0974**: Targets hemojuvelin, in late-stage Phase 2 development for myelofibrosis anemia [7][26] - **DISC-3405**: Targets TMPRSS6, designed to restrict iron for conditions like polycythemia vera and sickle cell disease [8][38] Market Opportunities - **EPP Market**: Potential market size of over $2 billion if all diagnosed patients are treated [46] - **Myelofibrosis Anemia**: Estimated 25,000 patients in the U.S., with a potential market exceeding $4 billion [29][47] - **Polycythemia Vera**: Approximately 75,000 patients, with significant unmet needs and potential for iron restriction therapy [39][40] Launch Strategy - **Sales Team**: 24 representatives ready for launch, focusing on top 120 accounts [19][22] - **Patient Engagement**: Strategies to identify and engage the 8,000 patients who are currently undiagnosed or untreated [48][49] - **Payer Mix**: Favorable payer landscape with 16% commercial, 25% Medicare, and 15% Medicaid [25] Regulatory and Clinical Milestones - **Upcoming Milestones**: - Anticipated FDA action date for bitopertin in late January or early February [46] - Top-line data from the confirmatory Apollo trial expected by late this year or early next year [15][43] - Plans to initiate pivotal trials for myelofibrosis and polycythemia vera in 2027 [42][44] Financial Position - **Capital**: Approximately $791 million available, providing runway into 2029 without assuming revenue from bitopertin [44][45] Conclusion - Disc Medicine is positioned for a significant year with the potential approval of bitopertin and a robust pipeline addressing critical unmet needs in hematology-oncology, with a strong financial foundation to support its growth strategy [46][47]