BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell technologies for neurodegenerative diseases, is presenting results from its phase 2a study of NurOwn® in amyotrophic lateral sclerosis (ALS) at a poster session today at the American Academy of Neurology annual meeting, taking place in Washington D.C. In addition to previously disclosed topline results, further data and analyses are being presented for the first time today.
Among the new results is a piecewise linear regression analysis of all subjects who received intrathecal (IT) administration in the phase 2a study and the prior phase 1/2 study. At six months post-treatment, there was a statistically significant improvement in the estimated rate of decline in Forced Vital Capacity (FVC), from -5.1% per month pre-treatment to -1.2% per month post-treatment (two-sided p=0.036) and a nearly significant improvement in the rate of ALS Functional Rating Score-Revised (ALSFRS-R) decline, from -1.2 points per month pre-treatment to -0.6 points per month post treatment (two-sided p=0.052).
Also being reported for the first time are local positive effects of intramuscular administration. 3D volumetric analysis using MRI revealed an improvement in the rate of decline in muscle mass in the right arm, the site of NurOwn® administration, through one month post-treatment, as compared to the left arm. Electromyography demonstrated a trend of stabilization of the compound motor axon potential in the right musculocutaneous nerve as compared to deterioration observed in the left.
BrainStorm’s CEO Tony Fiorino, MD, PhD, commented “These results represent further validation for our NurOwn® platform. In this study, we observed a large and clinically meaningful benefit after treatment with NurOwn®. Moreover, our analysis of subjects who received IT administration in our two completed trials showed a statistically significant improvement in the rate of FVC decline, and a nearly significant improvement in the rate of ALSFRS-R decline, at six months post-treatment, a notable achievement given the small sample size. With our US phase 2 trial now more than half-enrolled and a multi-dose study being planned, BrainStorm is well-positioned to confirm and extend these findings over the coming year.”
Professor Dimitrios Karussis of Hadassah University Medical Center, the principal investigator for the study, stated “We have observed in our two studies clear indications that a single intrathecal administration of NurOwn® can induce clinically meaningful beneficial effects in ALS patients. In these studies, 88% of subjects with 3 months follow-up and 73% of those with six months follow-up responded to the cells, showing a post-treatment improvement in either ALSFRS-R or FVC, or both. We eagerly await the results of current and planned studies that will define the safety and efficacy profile of NurOwn®, and we are particularly hopeful that the administration of repeated doses will increase the magnitude or duration of benefit, or both.”