BrainStorm Cell Therapeutics (Nasdaq: BCLI), which is developing a stem cell-based treatment for amyotrophic lateral sclerosis ( ALS ), announced yesterday that its NurOwn product had not met the main endpoint of its Phase III clinical trial. In the trial, in which BrainStorm's product was tested against control treatments in 189 patients, no statistically significant difference was found between the two groups in the symptoms of the disease. The company said that there was a difference, but that it was too small to be considered significant.
The company's share price fell by more than 70% yesterday. In early trading today, the share price is stable at the new level.
ALS is a progressive nervous system disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control. It is known as Lou Gehrig's disease in Canada and the US, and as motor neurone disease in the UK and Australia.
The company said that the response to the placebo in the control group was especially high, and that this may have been the reason that the difference between it and the group that received the treatment was not statically significant.
The company added that in a pre-specified subgroup, NurOwn demonstrated a clinically meaningful treatment response across the primary and key secondary endpoints, and 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.
Although the trial failed to meet its primary endpoint, the company is in talks with the US Food and Drug Administration (FDA) to examine how to proceed. Occasionally, companies manage to persuade the FDA that a product can be used on the basis of meeting secondary endpoints in a sub-group in a trial. It is more likely, however, that the FDA will tell BrainStorm to carry out another trial or give up.
The trial carried out by BrainStorm took three years, and apparently cost tens of millions of dollars. If it chooses to carry out a further trail, it will have to invest the time and money again.
BrainStorm is in the midst of another trial, for a treatment for multiple sclerosis.
BrainStorm CEO Chaim Lebovits said, "This clinical trial included a more severely affected ALS population compared to other recent ALS clinical trials. We identified a superior treatment response in a pre-specified subgroup of patients with less advanced disease. We are in active discussions with the FDA who have expressed their eagerness to review the data and have committed to prioritize review of this data. The FDA will review the data to see if there is a path forward to support approval." p>Published by Globes, Israel business news - en.globes.co.il - on November 18, 2020
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