Disc Medicine (NasdaqGM:IRON) FY Conference Transcript

Summary of Disc Medicine FY Conference Call Company Overview - Company: Disc Medicine (NasdaqGM:IRON) - Market Cap: Approximately $2 billion as of the call date [2] - Focus: Red blood cell biology, specifically treating diseases related to iron and heme metabolism [2] Lead Programs - Bidipertin: - Licensed from Roche, currently in the accelerated approval pathway with an NDA filing projected for October [3] - Confirmatory trial named APOLLO is underway [4] - Pipeline: - Two phase two programs targeting myeloproliferative diseases [4] - DISCO-nine 74: Focused on myelofibrosis anemia and anemia of chronic kidney disease, with readouts expected at ASN or ASH [4] - DISC 3,405: Aiming to manage hematocrit in polycythemia vera [4] Clinical Data and Regulatory Insights - Bidipertin: - Data from HELIOS study indicates a reduction in protoporphyrin nine (PP9) levels, which correlates with improved liver health outcomes [7] - KOLs (Key Opinion Leaders) are supportive of the drug's potential liver protection [8] - No expectation for liver protection to be included in the label claim without further studies [9][10] - Pediatric Plans: - Inclusion of adolescents in the approval process is planned, with ongoing enrollment in the APOLLO trial [14] - Future studies for younger patients are anticipated post-approval [17] Commercial Strategy - Commercial Team: - Building a team with experience in rare disease launches, including a Chief Commercial Officer and a medical affairs team [24] - Patient Engagement: - Identified 14,000 diagnosed patients in the US, with a focus on engaging the 6,000 who are actively seeking care [27] - Plans to utilize patient advocacy and social media to reach patients who have disengaged from treatment [28] Competitive Landscape - DISCO-nine 74: - Positioned as a unique treatment for anemia in myelofibrosis, especially as other therapies like momalotinib are not effectively addressing anemia [30][31] - Preliminary data suggests that DISCO-nine 74 may work effectively alongside momalotinib [35] Future Development Plans - CKD Anemia: - Ongoing studies to assess the efficacy of DISCO-nine 74 in chronic kidney disease anemia, with plans for a phase two trial depending on data outcomes [46][48] - DISC 3,405: - Currently in phase two, with data projections for the next year, aiming to provide a better dosing regimen for polycythemia vera patients [52] Key Takeaways - Disc Medicine is advancing its lead program bidipertin towards accelerated approval, with a strong pipeline in red blood cell-related diseases. - The company is actively engaging with KOLs and patients to build a robust commercial strategy. - Competitive positioning is strengthened by the unique therapeutic profile of DISCO-nine 74 in managing anemia in myelofibrosis and potential applications in CKD. - Future studies and data will be critical in determining the success of their ongoing and planned clinical trials.