Disc Medicine's Bitopertin Reduces PPIX Levels in EPP Study

By Tamara Parker • Wednesday, April 03 06:02 • Permanent Link

Disc Medicine, Inc. has just released topline data from the Phase 2 Aurora study of bitopertin in patients with erythropoietic protoporphyria (EPP). The study showed that bitopertin met its primary endpoint by demonstrating dose-dependent, statistically significant reductions in protoporphyrin IX (PPIX) compared to placebo in both the 20 mg and 60 mg dose groups. The improvements in light tolerance, including the key secondary endpoint, were also observed in both dose groups, although they did not meet statistical significance compared to placebo.

The study revealed that bitopertin resulted in significant, dose-dependent, and sustained reductions in whole blood PPIX levels, with a reduction of 21.6% for the 20 mg dose and 40.7% for the 60 mg dose compared to placebo. Additionally, there were substantial and dose-dependent reductions in phototoxic reactions with pain during the study period. Bitopertin-treated patients also reported dose-dependent improvements in the patient global impression of change (PGIC), with statistical significance at the 60 mg dose group compared to placebo.

The Aurora study, a randomized, double-blind, placebo-controlled phase 2 study, enrolled 75 adult subjects with EPP who were randomized to receive 20 mg of bitopertin, 60 mg of bitopertin, or placebo once daily for 17 weeks.

The management of Disc Medicine, Inc. will host a call on April 1 at 8:30 am ET to review the data. Bitopertin is an investigational, clinical-stage, orally-administered inhibitor of glycine transporter 1 (GlyT1) designed to modulate heme biosynthesis, with potential as a treatment for a range of hematologic diseases including EPP.

Erythropoietic protoporphyria (EPP) is a rare, debilitating, and potentially life-threatening disease caused by mutations that affect heme biosynthesis, resulting in the accumulation of a toxic, photoactive intermediate called protoporphyrin IX (PPIX). There is currently no cure for EPP, and only one FDA-approved therapy exists.

Disc Medicine, Inc. is a clinical-stage biopharmaceutical company focused on discovering, developing, and commercializing novel treatments for patients suffering from serious hematologic diseases.

Today the company's shares have moved -6.4% to a price of $32.03. For the full picture, make sure to review Disc Medicine's 8-K report.

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