Disc Medicine (NasdaqGM:IRON) 2025 Conference November 13, 2025 08:40 AM ET Company ParticipantsJonathan Yu - COOConference Call ParticipantsStephen Willey - Managing Director and Senior Equity Research AnalystStephen WilleySo we're going to go ahead and get started. I'm Stephen Willie, one of the senior biotech analysts here at Steve Full, and glad to have with us from Disc Medicine Johnathan Yu, who is the chief operating officer. we're going to have a fireside chat. If there's any questions, feel free to ...

Core Insights - Disc Medicine, Inc. is set to present initial data from the RALLY-MF Phase 2 trial of DISC-0974 for anemia of myelofibrosis at the ASH Annual Meeting in December 2025 [1][2] - The company aims to provide updates on the trial results, including data from a broader patient population and the drug's efficacy as a monotherapy and in combination with JAK inhibitors [2] - Additionally, a poster on the ongoing Phase 2 trial of DISC-3405 in polycythemia vera will be presented, with initial readouts expected in 2026 [2] Presentation Details - The DISC-0974 poster presentation is scheduled for December 6, 2025, from 5:30 pm to 7:30 pm EST, with Naseema Gangat as the presenting author [4] - The DISC-3405 poster presentation will also occur on December 6, 2025, during the same time frame, presented by Marcus Carden [5] Company Overview - Disc Medicine is a clinical-stage biopharmaceutical company focused on developing novel treatments for serious hematologic diseases, targeting key biological pathways related to red blood cell biology [6]

Core Insights - Disc Medicine, Inc. is set to present clinical data on its drug DISC-0974 at the 2025 American Society of Nephrology Kidney Week, focusing on patients with non-dialysis-dependent chronic kidney disease (NDD-CKD) and anemia [1][2] Company Overview - Disc Medicine is a clinical-stage biopharmaceutical company dedicated to developing novel treatments for serious hematologic diseases, with a focus on innovative therapeutic candidates targeting red blood cell biology, heme biosynthesis, and iron homeostasis [5] Clinical Study Details - The upcoming presentation will include data from a Phase 1b, randomized, placebo-controlled study assessing the safety, tolerability, and pharmacokinetics of DISC-0974, along with pharmacodynamic activity measures such as hepcidin and iron levels [2][4] - The presentation is scheduled for November 8, 2025, at 10:00 am CST, with the abstract available on the ASN Kidney Week conference website [3][4]

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]