Phase 2 study of systemic pegcetacoplan for ALS fails to meet efficacy endpoints

Key takeaways:

• Study of 250 adults with ALS did not meet primary endpoint of Combined Assessment of Function and Survival rank score at week 52.
• Apellis and Sobi plan to discontinue development of pegcetacoplan for ALS.

Apellis Pharmaceuticals Inc. announced that its phase 2 MERIDIAN study investigating systemic pegcetacoplan as a treatment for ALS failed to meet efficacy measures of overall function, survival, lung function and muscle strength.

According to a company press release, the multicenter, double-blind trial evaluated the efficacy and safety of systemic pegcetacoplan, a complement C3 inhibitor, in approximately 250 adults with sporadic ALS. Participants were randomized to receive pegcetacoplan or placebo with standard care for 52 weeks.

Although pegcetacoplan was well-tolerated with consistent safety data, the study failed to achieve its primary endpoint of Combined Assessment of Function and Survival rank scores at week 52. It also did not meet key secondary efficacy outcomes, including overall function, survival, lung function and muscle strength.

Based on the results, Apellis and its partner Sobi plan to discontinue development of pegcetacoplan for ALS. The companies discontinued treatment in the open-label portion of the study in April, after a recommendation from an independent data monitoring committee.

“We are disappointed in the outcome of the MERIDIAN study, especially on behalf of the ALS community who has been waiting for new treatments for this complex and unrelenting disease,” Jeffrey Eisele, PhD, Apellis chief development officer, said in the release. “Our hope is that the data generated from this study will continue to support future research and development in ALS.”