Summary of Disc Medicine Management Call - December 07, 2025 Company Overview - **Company**: Disc Medicine (NasdaqGM:IRON) - **Focus**: Development of therapies for hematologic diseases, particularly myelofibrosis (MF) and related anemias Key Updates on Products and Trials Bitopertin - **Status**: NDA accepted for review by the FDA for accelerated approval for erythropoietic protoporphyria (EPP) with a potential launch by the end of January 2026 [3][6][8] - **Review Process**: Engaged in a rigorous review process with the FDA, characterized by increased communication and collaboration [6][41] - **Commercialization**: Accelerated efforts for launch readiness, including drug supply expected by the end of January 2026 [7][8] DISC-0974 - **Indication**: Treatment for anemia of myelofibrosis (MF) - **Trial Updates**: Phase II RALLI-MF study shows significant decreases in hepcidin, leading to improved hemoglobin levels and reduced transfusion burden [4][10][12] - **Response Rates**: - Non-transfusion-dependent (NTD) group: 63% overall response, 50% major response - Transfusion-dependent (TD) low group: 71% overall response, with consistent results across different JAK inhibitors [12][14] - **Safety Profile**: Well tolerated with no treatment-related serious adverse events reported [14] DISC-3405 - **Mechanism**: Increases endogenous hepcidin production to limit iron availability, targeting conditions like polycythemia vera and sickle cell disease [21][22] - **Trial Initiation**: Phase II trials for polycythemia vera and sickle cell disease initiated, with initial data expected in 2026 [26][27] Market Opportunity - **Anemia of MF**: Affects approximately 22,000 patients in the U.S., representing a significant unmet need [17][18] - **Potential for Blockbuster Status**: DISC-0974 is positioned to address this unmet need effectively, with high response rates and the ability to work alongside existing therapies [19][18] Future Plans - **Regulatory Engagement**: Plans for an end-of-phase II meeting with the FDA for DISC-0974 to align on registrational trial design [19] - **Expansion of Trials**: Plans to initiate a phase II study in anemia of inflammatory bowel disease (IBD) and explore other anemias of inflammation [19][55] Financial and Operational Updates - **Funding**: Completed two follow-on offerings, providing a runway into 2029 without relying on revenue from bitopertin [29] - **Sales Force Preparation**: Hiring 24 sales representatives in anticipation of bitopertin's launch [90] Additional Insights - **Patient Experience**: Anecdotal reports indicate patients feel significantly better on DISC-0974, with improvements in fatigue and overall well-being [70] - **Combination Therapy Potential**: Ongoing discussions about potential combination studies with other agents to enhance efficacy [49][50] Conclusion Disc Medicine is making significant strides in the development of therapies for hematologic conditions, with promising data from ongoing trials and a clear path toward commercialization of its lead products. The company is well-positioned to address significant unmet needs in the market, particularly in the treatment of anemia associated with myelofibrosis.