Pridopidine was chosen in global competition as one of the top drug candidates to participate in the first ever ALS Platform Trial led by the Healey Center at Massachusetts General Hospital
This trial is the second late-stage clinical trial in neurodegenerative disorders with pridopidine, in addition to the global Phase 3 trial in Huntington’s disease
Positive data from this clinical study could lead to registration of pridopidine for the treatment of ALS
Prilenia Therapeutics B.V., a clinical stage biotech company focused on developing novel treatments for neurodegenerative and neurodevelopmental disorders, today announces the enrollment of the first participant for treatment with pridopidine in the pivotal HEALEY ALS Platform Trial.
The HEALEY ALS Platform trial (“HEALEY trial”; NCT04297683) is a multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of amyotrophic lateral sclerosis (ALS). It is the first platform trial for ALS, designed to accelerate development of promising new treatments by enabling investigators to simultaneously assess multiple therapeutic candidates.
The HEALEY ALS Platform Trial - Regimen D Pridopidine (NCT04615923) evaluates the safety and efficacy of pridopidine as a single agent for ALS patients. Pridopidine is a highly potent and selective Sigma-1 receptor (S1R) agonist, which was selected as a therapeutic candidate out of approximately 30 competing investigational treatments. It was chosen by an independent expert review committee based on compelling human genetic data, efficacy in preclinical models, favorable safety profile and readiness of drug supply. The trial treatment dose (45 mg bid) has an extensive safety record (based on >1,300 patient years), confirming placebo-like safety and tolerability profile.
Michael R. Hayden, MD, PhD, CEO of Prilenia, said: “Enrolling the first patient in the HEALEY ALS platform trial for treatment with pridopidine is great news for both the ALS patient community and Prilenia. Previous studies with pridopidine demonstrate it is a highly selective S1R agonist with an extensive safety record and a great therapeutic potential in multiple neurodegenerative disorders including HD and ALS. We are excited to be part of this platform trial which is designed to hasten development of new treatments to patients living with ALS.”
The HEALEY trial is being led by ALS specialists at the Sean M. Healey & AMG Center for ALS at Mass General Hospital. The study is recruiting adults with sporadic or familial ALS at 54 sites, all of which are part of the Northeast ALS (NEALS) Consortium, across the United States.