Phase 3 study to assess safety, efficacy of Huntington’s disease therapeutic complete

Prilenia Therapeutics announced the completion of its global phase 3 study of pridopidine for Huntington’s disease, with topline results expected in the next few months.

According to a release from Prilenia, all participants in the PROOF-HD study have now completed the last planned visit of the blinded treatment period, and 98% of eligible participants will continue in an ongoing open-label extension period.

The randomized, double-blind, placebo-controlled study — a collaboration with the Huntington Study Group — was conducted in 11 countries across North America and Europe and evaluated the safety and efficacy of pridopidine, an oral sigma-1 receptor agonist. The trial enrolled 499 patients with Huntington’s disease who received twice daily pridopidine 45 mg for 65 to 78 weeks.

The primary endpoint of the study was change from baseline in the Unified Huntington Disease Rating Scale-Total Functional Capacity score at 65 weeks.

“There is an urgent need for treatments that can alter the course of HD and bring hope to individuals and families impacted by the disease,” Michael R. Hayden, PhD, MBChB, CEO and founder of Prilenia, stated in the release. “Our team is working diligently to bring topline results to the community as quickly as possible.”

Pridopidine was granted FDA orphan drug designation for Huntington’s disease and ALS in the U.S. and EU and also received fast track designation for Huntington’s.