Business
Disc Medicine Provides Update on Hematology Portfolio and Outlines Near-Term Business Objectives and Anticipated Milestones
Submitted a New Drug Application (NDA) for accelerated approval of bitopertin in erythropoietic protoporphyria (EPP) on September 29, 2025, and subsequently
About this update from Disc Medicine, Inc.
[{"type":"text","content":"Submitted a New Drug Application (NDA) for accelerated approval of bitopertin in erythropoietic protoporphyria (EPP) on September 29, 2025, and subsequently was awarded the FDA Commissioner’s National Priority Voucher (CNPV), a program designed to shorten the expected NDA review period to 1-2 monthsAcceleration of ongoing efforts to support the potential earlier approval and commercialization of bitopertin for EPP in the US, expected in late 2025 or early 2026Plans to advance DISC-0974 for anemia of myelofibrosis (MF) and DISC-3405 for polycythemia vera (PV) deeper into development and to continue exploring potential role of hepcidin in additional indicationsStrong financial position ending Q3 with approximately $616 million in cash, cash equivalents, and marketable securities WATERTOWN, Mass., Oct. 20, 2025 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today outlined its recent pipeline and operational progress and strategic priorities for the rest of 2025 and 2026. “2025 has been another eventful year for Disc Medicine, and we are accelerating development across three key indications—EPP and our myeloproliferative neoplasm portfolio, MF and PV. For bitopertin, with the recent grant of the FDA’s CNPV, our focus is on accelerating our commercial readiness to ensure access to patients as quickly as possible upon approval, if granted,” said John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc Medicine. “We’re also excited about the progress across our iron homeostasis portfolio, DISC-0974 and DISC-3405, as these programs will be important drivers of Disc’s future growth. We’re positioning DISC-0974 for development in anemia of MF and look forward to providing an initial set of data from the Phase 2 RALLY-MF trial of DISC-0974 in its lead indication, anemia of MF, by the end of this year. Additionally, we are advancing the development of DISC-3405 in PV with data expected next year. We also continue to drive our expansion strategy for both of these programs with a plan to initiate a Phase 2 trial of DISC-0974 in anemia of inflammatory bowel disease in 2026 and a Phase 1b trial of DISC-3405 in sickle cell disease by year end.”...