Core Insights - Disc Medicine, Inc. is advancing its clinical programs for bitopertin, DISC-0974, and DISC-3405, with positive data presented at the EHA 2025 annual meeting, indicating favorable long-term efficacy and safety profiles for these investigational therapies [1][5][6] Bitopertin - The HELIOS study demonstrated sustained reductions in protoporphyrin IX (PPIX) and improvements in quality of life for patients with erythropoietic protoporphyria (EPP) [1][6] - Continuous treatment with 60 mg of bitopertin resulted in significant reductions in ALT and other hepatobiliary biomarkers, with nearly all participants reporting substantial quality of life improvements [6][4] - The company plans to submit a New Drug Application (NDA) for bitopertin in the second half of 2025 and has initiated a confirmatory clinical trial, APOLLO, for EPP [1][3] DISC-0974 - Data from the Phase 1b trial in myelofibrosis (MF) anemia showed sustained activity on key biomarkers and durable anemia responses among major responders [2][7] - The trial included various patient cohorts, with long-term results indicating that 50% of non-transfusion dependent patients achieved a major response with a sustained mean hemoglobin increase of ≥1.5 g/dL [10][17] - Enrollment for the Phase 2 RALLY-MF trial is ongoing, with initial data expected in the second half of 2025 [2][5] DISC-3405 - The Phase 1 trial in healthy volunteers demonstrated deep, sustained reductions in serum iron and significant changes in hematologic parameters, supporting its mechanism of action [2][9] - An iron pulse study showed that DISC-3405 inhibited dietary iron uptake, resulting in an average 94% reduction in iron absorption at Day 2 and 68% at Day 15 [12][13] - A Phase 2 trial of DISC-3405 in polycythemia vera (PV) has been initiated, with initial results anticipated in 2026 [11][17] Overall Company Strategy - The data presented at EHA supports the advancement of all three clinical candidates and highlights potential expansion opportunities into new indications [3][5] - Management will host a corporate update call on June 16, 2025, to discuss these developments further [5][3]

Core Insights - Disc Medicine, Inc. is set to present data from its hematology portfolio at the European Hematology Association (EHA) 2025 Congress in Milan from June 12-15, 2025, highlighting progress in its clinical programs [1][2] Company Developments - The company will showcase data from the HELIOS long-term extension trial, supporting bitopertin as a potential first disease-modifying treatment for erythropoietic protoporphyria (EPP) [2] - Additional data will be presented from the Phase 1b study of DISC-0974 in patients with myelofibrosis (MF) anemia, along with new findings from DISC-3405 in healthy volunteers, which supports its progression into a Phase 2 study in polycythemia vera [2][5] Presentation Details - The HELIOS study results will be presented on June 14, 2025, by Melanie Chin, while multiple presentations on DISC-0974 and DISC-3405 will occur on June 13 and 14, 2025, featuring various authors [4][6] - Management will host a corporate update call on June 16, 2025, to review the presented data [5]

Core Insights - Disc Medicine, Inc. reported strong operational progress in Q1 2025, with a focus on advancing its lead program, bitopertin, for erythropoietic protoporphyria (EPP) [2][5] - The company has a solid financial foundation, with cash reserves expected to fund operations into 2028, following a public offering that raised $259 million [3][9] Financial Performance - Cash, cash equivalents, and marketable securities totaled $694.7 million as of March 31, 2025, compared to $489.9 million at the end of 2024 [20] - Research and Development (R&D) expenses increased to $27.8 million in Q1 2025 from $23.7 million in Q1 2024, driven by advancements in clinical studies and increased headcount [14] - Selling, General and Administrative (SG&A) expenses rose to $12.2 million in Q1 2025 from $7.8 million in Q1 2024, reflecting the establishment of infrastructure for potential commercialization [14] - The net loss for Q1 2025 was $34.1 million, compared to a net loss of $26.9 million in Q1 2024 [14] Clinical Development Updates - The APOLLO trial for bitopertin has been initiated, aimed at supporting the conversion of a potential accelerated approval to full approval [6] - The company plans to submit a New Drug Application (NDA) for bitopertin in EPP in the second half of 2025 [5][6] - Data readouts for the DISC-0974 program, including results from the Phase 2 study for myelofibrosis (MF) anemia and the Phase 1b study for non-dialysis dependent chronic kidney disease (NDD-CKD) anemia, are expected in the second half of 2025 [5][7] - A Phase 2 study of DISC-3405 in polycythemia vera (PV) is set to be initiated in the first half of 2025 [8]