Disc Medicine (IRON) Update / Briefing Transcript

Summary of DISC Medicine Corporate Call Company Overview - Company: DISC Medicine - Focus: Development of therapies for rare hematologic diseases, particularly Erythropoietic Protoporphyria (EPP) and Myelofibrosis (MF) anemia Key Points Industry and Product Pipeline - Bidapertin: - NDA submission planned for the second half of the year for EPP [11] - Positive data from HELIOS study showing long-term efficacy and safety, with significant PP9 reduction and quality of life improvement [7][20] - Evidence of liver function improvement indicated by sustained ALT level reduction [7][20] - DISCO-974: - Monoclonal antibody targeting anemia of inflammation, with ongoing Phase II RALEY MF trial [28][50] - Durable efficacy observed in Phase 1b study, with sustained activity on key biomarkers [29][50] - Initial data from the RALEY MF trial expected later this year [50] - DISC-3405: - Monoclonal antibody for polycythemia vera, Phase II trial initiated [44][48] - Demonstrated ability to suppress iron absorption, with significant reductions in dietary iron uptake [47][48] Clinical Data and Efficacy - Bidapertin: - 86 patients in HELIOS study showed approximately 40% reduction in PP9 levels [17][19] - Patients on the 60 mg dose exhibited the greatest benefits [8][19] - Positive patient-reported outcomes with nearly universal positive responses regarding disease severity and quality of life [20][21] - DISCO-974: - Strong responses across various anemia patient categories, with major responders maintaining hemoglobin increases [31][32] - Evidence of synergistic effects when combined with other anemia-targeted agents [33][34] - DISC-3405: - Phase I study showed significant reductions in iron levels and hematologic responses in healthy volunteers [46][48] Commercialization and Market Opportunity - EPP Market: - Approximately 6,000 engaged EPP patients in the U.S. with a total of 14,000 diagnosed patients [24][25] - Focused commercial model targeting key treatment centers and healthcare professionals [25][26] - Disease state education campaign launched to raise awareness among patients and physicians [27] - MF Anemia Market: - Estimated 22,000 addressable patients in the U.S. with a significant unmet need for anemia treatment [39][40] - DISCO-974 aims to address anemia across all levels of transfusion dependence [41] Regulatory and Future Outlook - Regulatory Progress: - Pre-NDA meeting with the FDA scheduled to align on NDA package [12][49] - Enrollment for the APOLLO trial expected to be completed by Q2 next year [83] - Future Milestones: - Initial data from the RALEY MF trial and multiple dose portion of the Phase 1b study in chronic kidney disease expected later this year [50] - Initial results from the Phase II study in polycythemia vera anticipated in 2026 [48] Additional Insights - Physician Engagement: - Physicians are optimistic about the potential liver health benefits of bidapertin, which may influence prescription decisions [63] - Market research indicates that many EPP patients have been disengaged from healthcare due to a lack of treatment options, but awareness campaigns may drive re-engagement [84] - Combination Therapies: - Potential for combination therapies with existing anemia treatments like luspatercept, which may enhance patient outcomes [75][78] This summary encapsulates the key points discussed during the DISC Medicine corporate call, highlighting the company's pipeline, clinical data, market opportunities, and future regulatory steps.