Disc Medicine Announces Success on All Primary and Secondary Endpoints in the Pivotal Phase 3 APOLLO Trial of Bitopertin in Adults and Adolescents with Erythropoietic Protoporphyria (EPP)
Key Highlights
- ➤APOLLO met co-primary endpoints, reducing PPIX 41.4% versus placebo
- ➤Bitopertin increased average monthly pain-free light exposure 12.2 hours versus placebo
- ➤Phototoxic reaction incidence declined significantly versus placebo over six months
- ➤Disc plans NDA resubmission by end-2026, with FDA decision anticipated mid-2027
- ➤Bitopertin was generally well-tolerated; no treatment-emergent adverse events exceeded Grade 3
Expert Statements
John Quisel, President and Chief Executive Officer of Disc Medicine
“Today represents a potential breakthrough in the science and treatment of EPP. 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.”
Herbert L. Bonkovsky, Director of Gastroenterology & Internal Medicine and Professor Emeritus of Medicine at Wake Forest University School of Medicine; Principal Investigator of APOLLO
“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”
Will Savage, Chief Medical Officer of Disc Medicine
“These results are compelling evidence of the clinical benefit of a mechanism aimed at reducing the production of PPIX, the fundamental cause of disease”
* 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.
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