Brainstorm Cell Therapeutics Inc BCLI shares are sinking after the FDA released briefing documents ahead of the adcomm meeting scheduled on 27 September. PDUFA action date is targeted to occur by 8 December.
The FDA staff reviewers raised concerns over the safety and efficacy of BrainStorm Cell Therapeutics' amyotrophic lateral sclerosis (ALS) therapy.
Brainstorm's therapy, NurOwn (debamestrocel, MSC-NTF), is being reviewed to treat ALS, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
In its briefing documents, the FDA said it does not believe there is sufficient evidence to support NurOwn's clinical benefit, adding there were several missing data.
The FDA does not believe sufficient evidence supports that any of the assessed biomarkers is reasonably likely to predict clinical benefit. C
"We interpret your Phase 2 data as evidence that your product is not effective in the treatment of ALS. Your proposal that your Phase 2 data suggest benefit for the 'rapid progressors' is most likely over interpretation of your subgroup analyses. In subgroup analyses, the results for the 'slow progressors' could be interpreted to suggest that your product is harmful to some patients with ALS," the document noted.
The health regulator also identified a higher incidence of deaths in the treatment group, suggesting the lack of efficacy of MSC-NTF on the survival of patients with ALS.
Last year, BrainStorm Cell received a refusal to file letter from the FDA regarding its marketing application seeking approval for NurOwn.
Price Action: BCLI shares are down 41.6% at $0.48 on the last check Monday.
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