WALTHAM, Mass. — Prilenia Therapeutics and Ferrer have enrolled the first participant in a global Phase 3 clinical trial evaluating the experimental drug pridopidine for amyotrophic lateral sclerosis, marking a key step in efforts to develop new treatments for the progressive neurodegenerative disease.
The study, known as PREVAiLS, is a randomized, placebo-controlled trial expected to enroll about 500 participants with rapidly progressing ALS early in their disease course. Researchers will assess whether pridopidine can slow disease progression, preserve function, and improve survival outcomes.
The first patient was enrolled at Mass General Brigham under the supervision of Sabrina Paganoni, M.D., Ph.D., co-director of the Massachusetts General Hospital Neurological Clinical Research Institute and the trial’s principal investigator.
“Pridopidine is a sigma-1 receptor (S1R) agonist. The S1R has been shown to play a role in stimulating multiple neuroprotective pathways impaired in neurodegenerative diseases, such as ALS and Huntington’s disease (HD). Enrolling the first participant in this confirmatory study is a milestone in our search for potential new therapeutic options that may help to slow disease progression, preserve function, maintain speech and prolong survival — key aims of early ALS therapy,” Paganoni said.
The PREVAiLS trial is designed to build on findings from a subgroup analysis in the Phase 2 HEALEY ALS Platform Trial, which suggested potential benefits of pridopidine in patients with rapidly progressing ALS, though the broader study did not meet its primary or secondary endpoints.
“The ALS community urgently needs new treatment options that can delay the disease’s relentless progression, and awaits the outcome of this study,” said Kuldip Dave, Ph.D., senior vice president of research at the ALS Association. “The earlier we can diagnose and treat ALS, the greater the potential to preserve function and maintain quality of life for longer, which are key to making ALS livable until we can cure it.”
The 48-week study will take place across as many as 60 ALS treatment centers in 13 countries, with additional sites in the U.S., Canada, Europe, the U.K., and Israel expected to begin recruiting in the coming months. Participants will be randomly assigned to receive pridopidine or placebo, followed by an open-label extension phase.
Researchers will measure changes in ALS progression using the ALS Functional Rating Scale-Revised (ALSFRS-R), along with secondary outcomes including survival, respiratory and speech function, and quality of life.
Pridopidine is an investigational, orally administered therapy targeting the sigma-1 receptor, a pathway believed to play a role in neuroprotection. The drug has shown a favorable safety and tolerability profile in prior clinical studies involving more than 1,600 participants, primarily in Huntington’s disease trials.
Amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, affects motor neurons in the brain and spinal cord, leading to progressive muscle weakness, paralysis, and ultimately death. The disease affects an estimated 500,000 people worldwide, and current treatment options remain limited.
