Summary of Disc Medicine FY Conference Call Company Overview - **Company**: Disc Medicine (NasdaqGM: IRON) - **Focus**: Red blood cell biology through manipulation of heme and iron metabolism - **Pipeline**: Three molecules in clinical stages, with lead program Bitopertin for erythropoietic protoporphyria (EPP) [2][6] Key Points Product Development and Pipeline - **Bitopertin**: NDA submission expected in October 2025, with potential launch in 2026 [2][6] - **Other Programs**: Two additional programs targeting myeloproliferative disorders, currently in phase two with data expected by year-end [2][6] - **Patient Population**: Estimated 14,000 patients in the U.S. with EPP, with 6,000 actively seeking therapy [9][10] Competitive Landscape - **Collaboration**: One program in-licensed from a Chinese biotech, indicating a positive collaboration experience [3] - **Market Position**: No significant competition identified in the U.S. or China for the unique biology being targeted [3] Regulatory Environment - **FDA Interactions**: Positive feedback from the FDA regarding rare disease development, aiding in the approval process [5] Commercial Strategy - **Launch Preparations**: Chief Commercial Officer hired, focusing on understanding the patient population and engaging with physicians [6][7] - **Sales Strategy**: Targeting 6,000 engaged patients initially, with plans to expand outreach through advocacy groups and social media [10][11] Clinical Data and Efficacy - **Dosing Strategy**: 60 mg dose of Bitopertin shows a 50% reduction in toxic metabolite PP-9, correlating with clinical improvement [14] - **Quality of Life**: Sustained PP-9 reductions lead to improved patient-reported outcomes [16] Upcoming Trials - **Apollo Trial**: Confirmatory trial for Bitopertin with 150 patients expected to enroll by May 2026 [19] - **RALI-MF Trial**: Focused on anemia in myelofibrosis, with data readout anticipated in Q4 2025 [34] Financial Position - **Cash Reserves**: $650 million as of Q2 2025, projected to fund operations into 2028, including commercial launch expenses for Bitopertin [61] Future Opportunities - **Potential Indications**: Exploring applications for iron overload conditions and sickle cell disease [59] Additional Insights - **AI Utilization**: Exploring AI for streamlining clinical and regulatory documentation [4] - **Patient Impact**: EPP significantly affects quality of life, with severe light sensitivity leading to lifestyle limitations [26][27] This summary encapsulates the critical aspects of Disc Medicine's conference call, highlighting the company's strategic direction, product pipeline, and market positioning.

Bitopertin in EPP - NDA preparation is on track with planned submission in H2 2025 under accelerated approval path[11] - HELIOS extension study data showed sustained PPIX reductions and improvements in QoL with favorable long-term efficacy and safety[8, 21, 25] - HELIOS data suggests potential for bitopertin to improve liver function, with continued treatment of 60 mg reducing ALT[8, 24] - The prevalence of EPP patients in the US is estimated to be around 20,000, with ~6,000 diagnosed and ~14,000 misdiagnosed or not seeking treatment[31] DISC-0974 - Phase 1b results in MF anemia showed durable anemia response through the continuation phase among major responders[8, 57] - Phase 1b data showed sustained activity on key biomarkers, supporting target engagement and iron mobilization[8, 53] - In MF Phase 1b study, major responders treated with DISC-0974 experienced a 43% and 29% reduction from baseline at Day 113 in erythropoietin and ferritin, respectively[53] - RALLY-MF Phase 2 enrollment is ongoing, with the momelotinib exploratory cohort fully enrolled[8, 62] DISC-3405 - Phase 1 data from healthy volunteers showed deep, sustained reductions in serum iron and meaningful changes in hematologic parameters[9, 78, 80] - Iron pulse study in healthy volunteers showed a 94% reduction in iron absorption at Day 2 and 68% at Day 15, confirming MOA and potential in diseases of iron overload[9, 86] - A Phase 2 study in polycythemia vera has been initiated[9, 87]