BrainStorm Cell Therapeutics Inc. (NASDAQ:BCLI) announced that new data from the Company’s Phase 2 study of NurOwn® in ALS were presented by lead investigator Dr. James Berry at the 27th International Symposium on ALS/MND in Dublin, Ireland.
In the Phase 2 trial, levels of neurotrophic factors and inflammatory markers were measured in cerebral-spinal fluid (CSF) samples collected from patients. In the samples of those patients treated with NurOwn, a statistically significant increase in levels of neurotrophic factors VEGF, HGF and LIF was observed from pre- to post-transplantation. There was also a statistically significant reduction in inflammatory markers (MCP-1 and SDF-1) over this period, in patients treated with NurOwn® and this was not observed in the placebo group.
Dr. Berry also presented the pre-specified responder analyses from the Phase 2 trial which examined percentage improvements in post treatment of Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) slope compared to pre-treatment slope. These analyses showed that, in the NurOwn® treated group, a greater number of patients achieved the high threshold of 100% improvement in the post-treatment vs. pre-treatment slope, compared with the placebo group. The definition of these responders is that their disease symptoms were essentially halted for the period of the treatment effect or they achieved a positive improvement on their ALSFRS-R score. Moreover, in the pre-specified subgroup that was defined in order to exclude subjects whose disease was progressing slowly, this effect was even more pronounced.
“This Phase 2 trial demonstrated clinical meaningful improvements in disease symptoms as measured by the well-established ALSFRS-R scale”, stated Dr. James Berry, Unit Chief of the Mass. General ALS Multidisciplinary Clinic. “Importantly, there is evidence that NurOwn® may be halting disease progression or improving symptoms in some patients. The CSF biomarker profiles were also encouraging. The significant increases in neurotrophic factors and decrease in inflammatory markers observed in the treated group post-transplant provide a biological mechanism supporting the observed clinical effect.”
The Phase 2 trial was a randomized, double-blind, placebo-controlled multi-center study designed to evaluate the safety and efficacy of NurOwn® in 48 ALS patients. It was conducted at three sites in the U.S: Massachusetts General Hospital, UMass Medical School and the Mayo Clinic. Patients were randomized to receive NurOwn cells administered via combined intramuscular and intrathecal injection (n= 36), or placebo (n=12). The primary objective of the study was safety and tolerability. The pre-specified efficacy analyses were: change in the ALSFRS-R slope, change in Slow Vital Capacity (SVC) and muscle strength, responder analysis (the percentage of subjects who improved post-treatment compared with pre-treatment), and a subgroup analysis excluding slowly progressing patients who are less likely to have a detectable benefit from NurOwn. (Original Source)
Shares of Brainstorm are currently trading at $2.44, up $0.28 or 12.96%. BCLI has a 1-year high of $3.87 and a 1-year low of $1.90. The stock’s 50-day moving average is $2.20 and its 200-day moving average is $2.38.
Brainstorm Cell Therapeutics, Inc. operates as a biotechnology company, which develops innovative adult stem cell therapeutic products. It currently focuses on utilizing the patients own bone marrow stem cells to generate neuron-like cells that may provide an effective treatment initially for amyotrophic lateral sclerosis, Parkinson’s disease, multiple sclerosis and spinal cord injury.