Prilenia's lead compound selected to participate in first-ever ALS platform trial

Reviewed by James Ives, M.Psych. (Editor)Sep 19 2019

Prilenia, a newly formed biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, announces that Pridopidine, its lead compound, has been selected as the first of three potential new treatments to be included in the launch of the first ever platform trial in amyotrophic lateral sclerosis (ALS). Two additional compounds were chosen to join the trial at a later stage.

Pridopidine was chosen by an independent review committee out of 30 competing investigational treatments based on human genetic data, efficacy in preclinical models, favorable safety profile and readiness of drug supply. Pridopidine is a highly selective S1R agonist, and this mechanism has already been shown to provide some benefit in ALS patients.

This innovative platform trial is led by the Sean M. Healey & AMG Center for ALS at Mass General Hospital. The trial design aims to accelerate the development of effective treatments for people living with ALS, by testing and evaluating multiple treatments simultaneously.

This groundbreaking trial brings hope to ALS patients. The selection of Pridopidine validates the potential for this drug to impact common pathways across neurodegenerative diseases. We are honored to be a part of this innovative platform trial hosted by the Healey Center and the NEALS organization, a world leader in running ALS clinical trials."

Michael R. Hayden, MD, PhD, Executive Chairman of Prilenia

"A subset of the Healey Science Advisory Committee and NEALS scientific advisory board reviewed the almost 30 applications from 10 countries to select the top candidates ready for testing in people today," says Merit Cudkowicz, MD, MSc, director of the Sean M. Healey & AMG Center for ALS at Mass General. "We believe that this platform trial approach will accelerate how therapies are tested and look forward to advancing this important work."

The HEALEY ALS Platform Trial will be the second clinical trial initiated by Prilenia. Earlier this year, Prilenia launched a phase 2 clinical trial in the US to evaluate the safety and efficacy of Pridopidine in treating Levodopa Induced Dyskinesia in patients with Parkinson's Disease.

Partial financial support to initiate these first treatments is made possible thanks to the generosity of the Healey family and friends and the AMG Charitable foundation along with partners at TackleALS.