FDA Fast Tracks BrainStorm Cell's ALS Stem Cell Treatment

BrainStorm Cell Therapeutics, an Israel-based biotech firm that develops adult stem cell treatments for neurodegenerative diseases, announced that its NurOwn has been given Fast Track status by the U.S. Food and Drug Administration (FDA) as treatment for amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease.

NurOwn is comprised of neurotrophic factor-secreting autologous mesenchymal stem cells. The product is currently undergoing investigation in a randomized, placebo controlled, double-blind, Phase 2 trial in patients with ALS.

Dr. Tony Fiorino, CEO of BrainStorm, said, “We are pleased that the FDA has granted Fast Track status for NurOwn as this will allow us greater and more frequent dialogue with the Agency as we continue the development of this ground-breaking cell therapy for the treatment of ALS.” Dr. Fiorino added that he expects the designation to help accelerate and enhance the company’s development program for NurOwn. Through the FDA Fast Track designation, drug makers may submit a New Drug Application (NDA) on a rolling basis.

Just a day before this announcement, the company declared that the last patient in its Phase 2A trial had completed their last visit to the trial site Hadassah Medical Center in Jerusalem. The primary endpoint of the single-arm, dose-ranging study is safety of NurOwn via intrathecal and intramuscular injection. Efficacy was measured by the ALS Functional Rating Score-Revised (ALSFRS-R) and forced vital capacity (FVC).

Professor Karussis said, “I am pleased to bring this second study of NurOwn to a successful conclusion. I have now treated over 30 ALS patients with NurOwn in two clinical trials, as well as in several compassionate use treatments, and I am enthusiastic about its emerging safety and efficacy profile.”

BrainStorm Cell anticipates the release of the final results of the study in the last quarter of the year upon conclusion of required statistical analysis.