Core Insights - Disc Medicine, Inc. presented positive initial data from the RALLY-MF Phase 2 trial of DISC-0974 for treating anemia in myelofibrosis (MF) at the ASH Annual Meeting, showing significant reductions in hepcidin and increases in iron levels, leading to clinically meaningful improvements in anemia across various patient types [1][2]. Group 1: Trial Results - The ongoing Phase 2 trial enrolled 47 adult patients with MF and anemia, with 34 patients included in the responder analysis [2]. - DISC-0974 treatment resulted in over 75% reduction in hepcidin and significant increases in serum iron [6]. - 63% of non-transfusion dependent (nTD) patients achieved a hemoglobin increase of ≥1 g/dL for ≥12 weeks, while 50% had an increase of ≥1.5 g/dL for ≥12 weeks [6]. - 71% of low transfusion burden patients achieved transfusion independence over a 16-week period [6]. - 67% of high transfusion burden patients achieved a ≥50% reduction in transfusion requirement [6]. - 50% of patients receiving concomitant JAK inhibitor therapy achieved a major hematologic response [6]. Group 2: Treatment Administration and Tolerability - DISC-0974 was administered subcutaneously at a dose of 50 mg every 4 weeks for up to 6 treatments [2]. - The treatment was generally well-tolerated, with diarrhea and urinary tract infections being the only related adverse events reported in two or more subjects [6]. Group 3: Future Plans and Developments - The company plans to advance the DISC-0974 program to address the unmet need for anemia treatment in MF, as there are currently no approved therapies for this condition [2]. - Additional data from the trial is expected to be shared in the second half of 2026 [6].

Summary of Disc Medicine Conference Call Company Overview - **Company**: Disc Medicine (NasdaqGM:IRON) - **Focus**: Developing treatments for serious hematologic diseases, particularly targeting pathways fundamental to red blood cell biology [2][3] Key Programs and Developments - **Lead Program**: Bidapertin - **Type**: Oral small molecule inhibitor targeting GLIT1 - **Indication**: Erythropoietic protoporphyria (EPP) - **NDA Submission**: Submitted at the end of September 2025 - **FDA Priority Voucher**: Received, which accelerates review time from 10-12 months to 1-2 months [3][7][10] - **Potential Approval Timeline**: Expected by December 2025 or January 2026 [9] - **Iron Portfolio**: - **Disco 974**: Antibody against hemojuvelin, in Phase 2, targeting anemia of myelofibrosis [4] - **Disc 3405**: Antibody targeting Tempor S6, also in Phase 2, aimed at inducing hepcidin production and restricting iron [5] Commercialization and Launch Readiness - **Commercial Infrastructure**: Currently building out capabilities, with a focus on market access [16][17] - **Salesforce**: Hiring ongoing, with a staged approach to launch [18] - **Patient Identification**: Utilizing existing ICD-10 codes and claims data to target accounts effectively [21] Market Dynamics and Competitive Landscape - **EPP Market**: Recognized unmet medical need, with a specific diagnostic code aiding in patient identification [19][21] - **Competitive Products**: Cinesse and Mitsubishi Tanabe products are in the market; Disc Medicine's approach targets the underlying disease mechanism [30][31] Financial Position - **Balance Sheet**: Approximately $820 million available, projected to support operations through 2029, including commercialization of Bidapertin and completion of ongoing studies [63][64] Future Directions - **Exploratory Studies**: Plans to explore Bidapertin in other indications, including Diamond Blackfan anemia and inflammatory bowel disease (IBD) anemia [34][53] - **Phase 3 Apollo Trial**: Ongoing recruitment, with no expected negative impact from Bidapertin's commercial availability [27][29] Conclusion - Disc Medicine is positioned for significant growth with its innovative therapies targeting hematologic diseases, backed by a strong financial position and strategic plans for commercialization and market entry. The company is focused on leveraging its unique drug profiles to address unmet medical needs in rare diseases.

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]