Disc Medicine Advances Hematology Pipeline with Bitopertin NDA Plans and Phase 2 Trials
Key Insights
Disc Medicine is preparing for an NDA submission for bitopertin in erythropoietic protoporphyria (search) (EPP (search)) following positive FDA feedback, potentially leading to accelerated approval.
Phase 2 trials for DISC-0974 in anemia (search) of myelofibrosis (search) (MF) and chronic kidney disease (search) (CKD (search)) are progressing, with initial data expected in the second half of 2025.
A Phase 2 study of DISC-3405 in polycythemia vera (search) (PV (search)) is set to begin in H1 2025, expanding Disc Medicine's portfolio in hematologic diseases.
Disc Medicine, Inc. (NASDAQ:IRON) is making strides in its hematology portfolio, with plans to submit a New Drug Application (NDA) for bitopertin in erythropoietic protoporphyria (search) (EPP (search)) and advancing clinical trials for DISC-0974 and DISC-3405 in various anemia (search) conditions and polycythemia vera (search). The company's strategic priorities for 2025 focus on developing novel treatments for serious hematologic diseases.
Bitopertin: Potential Accelerated Approval for EPP
Following a Type C meeting with the FDA, Disc Medicine is moving forward with plans for an NDA submission for bitopertin, a GlyT1 inhibitor, in EPP (search). The FDA has indicated a potential accelerated approval path using protoporphyrin IX (PPIX) reduction as a surrogate endpoint. John Quisel, J.D., Ph.D., Chief Executive Officer and President of Disc, stated, "Our recent FDA interaction marks another step toward delivering a potentially life-altering therapy for EPP patients." The company plans to initiate the global, confirmatory APOLLO trial by mid-2025.
The APOLLO trial is designed as a double-blind, placebo-controlled study with approximately 150 patients aged 12 and older, including those with X-linked protoporphyria (XLP). Co-primary endpoints include average monthly total time in sunlight without pain and percent change from baseline in whole blood metal-free PPIX after 6 months of treatment. Secondary endpoints will assess phototoxic reactions, cumulative pain-free time in sunlight, patient global impression of change (PGIC), and time to prodrome. The selected dose for bitopertin is 60 mg, with a 6-month treatment duration.
Advancing Iron Homeostasis Portfolio: DISC-0974 and DISC-3405
Disc Medicine is also progressing its iron homeostasis portfolio with DISC-0974 and DISC-3405. Initial data from an ongoing phase 2 study of DISC-0974, an anti-hemojuvelin antibody, in anemia (search) of myelofibrosis (search) (MF) is expected in the second half of 2025. Additionally, data from higher doses and multiple dose cohorts of a phase 1b study of DISC-0974 in anemia of chronic kidney disease (search) (CKD (search)) are anticipated in the same timeframe.
DISC-3405, an anti-TMPRSS6 (search) antibody, is slated to enter a phase 2 clinical trial in polycythemia vera (search) (PV (search)) in the first half of 2025. This trial aims to broaden the role of iron restriction in other indications.
Financial Strength and Strategic Focus
Disc Medicine has strengthened its financial position through an equity offering with gross proceeds of approximately $178 million and a non-dilutive debt facility, providing a cash runway well into 2027. This financial stability supports the company's commitment to discovering, developing, and commercializing novel treatments for patients with serious hematologic diseases.
About Erythropoietic Protoporphyria (EPP)
Erythropoietic protoporphyria (search) (EPP (search)), including X-linked Protoporphyria (XLP), is a rare, debilitating, and potentially life-threatening disease caused by mutations affecting heme biosynthesis, leading to the accumulation of protoporphyrin IX (PPIX). This accumulation causes severe reactions to sunlight exposure, characterized by excruciating pain, edema, burning sensations, and potential blistering. Current treatments involve avoiding sunlight, managing pain, and, in some cases, using Scenesse® (afamelanotide), a surgically implanted synthetic hormone.