BrainStorm Therapeutics announces topline results from NurOwn phase III ALS study

BrainStorm Cell Therapeutics Inc. a leading developer of adult stem cell therapies for neurodegenerative diseases, announced topline results from the Company's randomized, double-blind placebo-controlled Phase III trial evaluating NurOwn (MSC-NTF cells) as a treatment for Amyotrophic lateral sclerosis (ALS). Results from the trial showed that NurOwn was generally well tolerated in this population of rapidly progressing ALS patients. While showing a numerical improvement in the treated group compared to placebo across the primary and key secondary efficacy endpoints, the trial did not reach statistically significant results. The Phase III clinical trial's primary efficacy endpoint, a responder analysis evaluating the proportion of participants who experienced a 1.25 points per month improvement in the post-treatment Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) slope, was powered on assumed treatment response rates of 35% on NurOwn versus 15% on Placebo. These estimates were based on available historical clinical trial data and the NurOwn Phase II data. The primary endpoint was achieved in 34.7% of NurOwn participants versus 27.7% for Placebo (p=0.453). Therefore, the trial met the expected 35% NurOwn treatment group efficacy response assumption, however the high placebo response exceeded placebo responses observed in contemporary ALS trials. The secondary efficacy endpoint measuring average change in ALSFRS-R total score from baseline to Week 28, was -5.52 with NurOwn versus -5.88 on Placebo, a difference of 0.36 (p= 0.693). In an important, pre-specified subgroup with early disease based on ALSFRS-R baseline score 35, NurOwn demonstrated a clinically meaningful treatment response across the primary and key secondary endpoints and remained consistent with the pre-trial, data-derived assumptions. In this subgroup, there were 34.6% responders who met the primary endpoint definition on NurOwn and 15.6% on Placebo (p=0.288), and the average change from baseline to week 28 in ALSFRS-R total score was -1.77 on NurOwn and -3.78 on Placebo (p=0.198), an improvement of 2.01 ALSFRS-R points favoring NurOwn. Cerebrospinal fluid (CSF) biomarker analyses confirmed that treatment with NurOwn resulted in a statistically significant increase of neurotrophic factors and reduction in neurodegenerative and neuroinflammatory biomarkers that was not observed in the placebo treatment group. The company also carried out pre-specified statistical modeling designed to predict clinical response with high sensitivity and specificity based on ALS biomarkers and ALS Function and confirmed that NurOwn treatment outcomes could be predicted by baseline ALS function as well as key CSF neurodegenerative and neuroinflammatory biomarkers.