- Positive APOLLO trial topline results demonstrated statistically significant and clinically meaningful benefit across all study endpoints
- Successfully met co-primary endpoints of reduction in protoporphyrin IX (PPIX) and increased total time in light
- All secondary endpoint results were also positive and statistically significant, including reductions in phototoxic reactions and improvements on multiple measures of light tolerance
- Exploratory endpoints of quality-of-life improvement were also statistically significant
- Bitopertin was generally well-tolerated with no new safety signals observed in APOLLO
- Disc plans to resubmit an NDA to FDA by end of 2026, with an FDA decision anticipated by mid-2027
- Disc to discuss topline results in a corporate webcast on October 19, 2026 at 8:00am ET
WATERTOWN, Mass., Oct. 19, 2026 (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 announced topline results from the APOLLO Phase 3 trial of bitopertin in erythropoietic protoporphyria (EPP), including X-linked protoporphyria (XLP). The trial met all its primary and secondary endpoints.
“Today represents a potential breakthrough in the science and treatment of EPP,” said John Quisel, J.D., Ph.D., President and Chief Executive Officer of Disc Medicine. “We could not have hoped for a better outcome, and we are grateful to the patients, caregivers, and physicians who have partnered with us throughout the clinical development of bitopertin. We look forward to engaging with regulators with the goal of bringing an important, innovative medicine to this community as efficiently as possible.”
“This is the first time a potential treatment for EPP has shown robust evidence of whole blood PPIX reduction alongside unequivocal improvement on important measures of disease severity: increased time in light, reduced phototoxic reactions, and improved Patient Reported Outcome scores,” said Herbert L. Bonkovsky, M.D., Director of Gastroenterology & Internal Medicine, Professor Emeritus of Medicine at Wake Forest University School of Medicine, and a Principal Investigator of APOLLO.
“These results are compelling evidence of the clinical benefit of a mechanism aimed at reducing the production of PPIX, the fundamental cause of disease,” added Will Savage, M.D., Ph.D., Chief Medical Officer of Disc Medicine. “It has been an incredible journey from establishing the original hypothesis of targeting heme synthesis in cellular models to seeing that biology translate into the clear and meaningful clinical effects from APOLLO. We plan to share more detailed data with additional insights from APOLLO at a future medical conference.”
APOLLO is a randomized, double-blind pivotal Phase 3 trial evaluating the safety and efficacy of bitopertin versus placebo in adults and adolescents aged 12 or older with EPP or XLP. The trial enrolled 183 patients across the United States, Canada, Europe, and Australia, who were stratified by baseline light tolerance and geography and randomized 1:1 to receive 60 mg oral bitopertin or placebo once daily for 6 months.
Summary of topline results:
- Bitopertin achieved statistical significance on both co-primary endpoints vs. placebo:
- Reduction in whole-blood metal-free PPIX from baseline (-41.4% vs. placebo, p