Disc Medicine Initiates BEACON, a Phase 2 Clinical Study of Bitopertin in Patients with Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria (XLP)

• BEACON study will evaluate bitopertin as a potential disease-modifying treatment for patients with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP)
• Key assessments include changes in protoporphyrin IX levels, safety, tolerability and measures of photosensitivity; preliminary data expected by 1H 2023

Excerpt from the Press Release:

WATERTOWN, Mass., Aug. 10, 2022 /PRNewswire/ — Disc Medicine, a clinical-stage biotechnology company dedicated to the discovery and development of novel therapeutic candidates for the treatment of serious and debilitating hematologic diseases, announced today the initiation of BEACON, a phase 2 clinical study of bitopertin in patients with Erythropoietic Protoporphyria (EPP) or X-linked Protoporphyria (XLP). Bitopertin is an oral, selective inhibitor of glycine transporter 1 (GlyT1) designed to modulate heme biosynthesis, and has been shown in preclinical studies to reduce accumulation of protoporphyrin IX (PPIX), the toxic metabolite that causes disease pathology in EPP and XLP patients.

“The initiation of the BEACON phase 2 study is an important milestone as it marks the first time bitopertin will be evaluated as a potential, disease-modifying therapy in patients with porphyria. It builds upon our previous studies in animal models of EPP and XLP, which demonstrated that bitopertin can significantly reduce protoporphyrin IX,” said John Quisel, JD, PhD, Chief Executive Officer at Disc Medicine. “This is the second development program that Disc has successfully advanced into patient studies in the past few months. I want to thank our entire team for the tremendous effort that has brought us to this point and for their tireless dedication to patients.”

The BEACON Phase 2 study is a randomized, open-label, multiple dose clinical trial designed to evaluate the safety, tolerability, and efficacy of bitopertin in patients with EPP or XLP. It is designed to enroll approximately 20 patients at sites in Australia. The study will primarily assess changes in levels of PPIX as well as the pharmacokinetic profile, safety and tolerability of bitopertin in EPP or XLP patients. It will also include measures of photosensitivity, daylight tolerance, pain and exploratory biomarkers of hepatobiliary disease. Patients will receive orally-administered bitopertin for 24 weeks at doses of either 20 mg once-daily or 60 mg once-daily. Upon completion of the 24-week treatment period, patients may continue on bitopertin for an additional 24 weeks.

Click the button below to read the entire Press Release:

Continue Reading The Press Release