Despite red flag from FDA, controversial ALS cell therapy developer says it may still march ahead anyway
When BrainStorm Cell Therapeutics broke out the abysmal numbers in its Phase III trial, the biotech insisted that the FDA was eager to review the data — given the signal they had identified in a subgroup of amyotrophic lateral sclerosis with less advanced disease.
Three months later, regulators did give a look to the high-level summary of the data. But they didn’t have too many good things to say.
“The FDA concluded from their initial review that the current level of clinical data does not provide the threshold of substantial evidence that FDA is seeking to support a Biologics License Application (BLA),” BrainStorm wrote in a release.
Yet — and this is giving the company reason to push ahead — “the FDA advised that this recommendation does not preclude Brainstorm from proceeding with a BLA submission.”
Chaim Lebovits, the CEO, says they will consult with principal investigators, ALS experts, statisticians, regulatory advisors and advocacy groups before making a final decision on whether to file.
While filing after such disclosure would be risky and highly unorthodox, BrainStorm stresses that ALS is a devastating disease with few to no treatment options, and commits to fully analyzing biomarkers from the Phase III trial to determine the effects of its NurOwn cell therapy, which consists of autologous mesenchymal stem cells that are engineered to produce neurotrophic factors.
Besides, the executive team is no stranger to controversy.
The New York-based crew ignited a ring of criticism more than two years ago by indicating that it was preparing to sell their drug to ALS patients under the “Right to Try” law that was about to be passed. They eventually dropped the plan, but not before Lebovits disclosed that they were thinking of charging around $300,000 for the experimental treatment, which had generated less than encouraging results in Phase II.
He now notes that BrainStorm has over $40 million in cash and plans to announce its next flagship product soon.
It wasn’t enough to stem a plunge for the stock $BCLI, which fell 28.70% to $4.92.