What's Happening?
Prilenia Therapeutics B.V. and Ferrer have received clearance from the U.S. Food and Drug Administration (FDA) to commence a pivotal Phase 3 study of pridopidine in patients with rapidly progressive amyotrophic
lateral sclerosis (ALS). The study, named PREVAiLS, will involve 500 participants and will be conducted across 60 ALS treatment centers globally, including in the U.S., Canada, and Europe. The trial aims to confirm the efficacy and safety of pridopidine, building on promising results from a previous Phase 2 trial. The study will include a 48-week double-blind placebo-controlled phase followed by a 48-week open-label extension. The primary endpoint is the change in ALSFRS-R score adjusted for mortality, with secondary endpoints including speech and respiratory function. Recruitment is expected to begin in early 2026.
Why It's Important?
The approval of the Phase 3 study is a significant step forward in the search for effective treatments for ALS, a debilitating neurodegenerative disease with limited treatment options. Pridopidine has shown potential in improving multiple domains of disease progression, including speech and survival, which could offer hope to patients and their families. The study's success could lead to a new, well-tolerated oral therapy for ALS, potentially improving the quality of life and extending survival for those affected. This development also highlights the ongoing commitment to addressing rare neurological disorders and the importance of regulatory support in advancing clinical research.
What's Next?
Following the FDA's clearance, Prilenia and Ferrer will begin recruiting participants for the PREVAiLS study in early 2026. The study's outcomes will be closely monitored by the ALS community, researchers, and regulatory bodies. If successful, the results could pave the way for regulatory approval and commercialization of pridopidine as a treatment for ALS. Additionally, the study's findings may influence future research directions and funding allocations for ALS and similar neurodegenerative diseases.
