Summary of Disc Medicine Conference Call Company Overview - **Company**: Disc Medicine - **Focus**: Development of treatments for diseases related to red blood cell metabolism, specifically through manipulation of iron and heme metabolism [5][6] Key Programs 1. **Bidipertin** - **NDA Filing**: Scheduled for October 2025 with expected acceptance in December 2025, leading to a PDUFA date in 2026 [5] - **Patient Data**: Approximately 6,000 engaged patients identified from a claims database, with a total of 14,000 diagnosed patients [8][9] - **Market Strategy**: MSLs (Medical Science Liaisons) are engaging with physicians to confirm patient existence and raise disease awareness [10][13] - **Patient Education**: Emphasis on educating patients about the new therapy post-approval, with a focus on transitioning from clinical trials to commercial therapy [19][20] 2. **DISCO-974** - **Indication**: Targeting anemia of myelofibrosis (MF) with data cuts expected to be presented at ASH [6] - **Competitive Landscape**: Comparison with luspatercept, which failed in Phase 3 trials, highlighting the potential for DISCO-974 to address anemia more effectively across various patient subgroups [46][48] 3. **Chronic Kidney Disease (CKD) Anemia** - **Phase 1b SADMAD Program**: Data expected to be presented at ASN or ASH, focusing on iron mobilization and hemoglobin response [61][62] - **Future Trials**: Plans for a Phase 2 trial to confirm efficacy based on initial data, with a focus on identifying responding populations [63][64] Regulatory and Market Considerations - **Regulatory Alignment**: Confidence in the design of the Apollo confirmatory trial based on successful Phase 2 data, with a focus on minimizing placebo effects [38][41] - **Market Entry Strategy**: Anticipation of a gradual launch process, with emphasis on payer approval and physician engagement [22][24] - **Patient Population**: Targeting a significant unmet need in rare diseases, with a focus on educating both patients and physicians about new treatment options [19][57] Competitive Landscape - **Comparative Analysis**: Insights drawn from other rare disease launches, particularly Solano in rare obesity, and the challenges faced by luspatercept in treating anemia [33][46] - **Market Size**: Potential to address a population of approximately 22,000 anemic patients in the U.S. [56] Additional Insights - **Patient Advocacy**: Strong support from patient advocacy groups, which aids in raising awareness and sharing patient success stories [37] - **Data Presentation**: Upcoming data presentations at ASH are expected to provide further clarity on the efficacy and competitive positioning of DISCO-974 and other programs [56][65] This summary encapsulates the key points discussed during the conference call, highlighting the strategic direction and anticipated developments for Disc Medicine.

Summary of Disc Medicine Conference Call Company Overview - **Company**: Disc Medicine - **Focus**: Hematology, specifically targeting pathways of red blood cell biology and iron metabolism - **Lead Programs**: Bidipertin for erythropoietin protoporphyria and DISCO-nine 74 for anemia of myelofibrosis [3][6][8] Industry Context - **Disease Focus**: Myelofibrosis, a type of blood cancer characterized by anemia, splenomegaly, and constitutional symptoms [16][22] - **Current Treatment Landscape**: - Four JAK inhibitors approved for myelofibrosis, primarily targeting spleen size reduction and symptom improvement [46][49] - No approved therapies specifically for anemia of myelofibrosis, leading to significant unmet medical needs [19][45] Key Points from the Call Myelofibrosis and Anemia - **Prevalence**: Approximately 25,000 patients in the U.S. diagnosed with myelofibrosis, with a five-year survival rate around 50% [26][27] - **Anemia Statistics**: - 40% of patients present with significant anemia at diagnosis, increasing to 60% within one year [36] - Nearly all patients will develop anemia over the disease course, with transfusion dependency rising from 25% at diagnosis to 45% within a year [36][37] - **Prognostic Importance**: Anemia is a significant prognostic factor, with lower hemoglobin levels correlating with worse survival outcomes [30][38] Current Treatment Limitations - **JAK Inhibitors**: While effective for managing symptoms, they often exacerbate anemia, leading to treatment discontinuation in many patients [60][61] - **Existing Treatments for Anemia**: Use of erythropoietin, steroids, and androgens, but these are not specifically approved for myelofibrosis-related anemia [49][50] Emerging Therapies - **DISCO-nine 74**: - A monoclonal antibody targeting hepcidin suppression to mobilize iron for red blood cell production [8][10] - Phase 1b data shows promise in treating anemia of myelofibrosis [14][19] - **Luspatercept and Elitracept**: - Both are in development as potential treatments for anemia, with luspatercept already approved for MDS [79][80] - Phase 3 trials for luspatercept are ongoing, focusing on patients requiring transfusions while on stable doses of JAK inhibitors [80] Market Opportunity - **Anemia of Myelofibrosis**: Significant market opportunity exists for effective treatments targeting anemia, given the high prevalence and unmet needs [19][45] - **Potential Impact of New Therapies**: New treatments could improve patient quality of life, reduce healthcare costs associated with transfusions, and address the underlying pathophysiology of anemia in myelofibrosis [40][41][44] Conclusion - The call emphasized the critical need for new therapies specifically targeting anemia in myelofibrosis, highlighting the potential of DISCO-nine 74 and other emerging treatments to fill this gap in the current treatment landscape [19][45][76]