The FDA was pretty clear a year and a half ago that BrainStorm Cell Therapeutics’ amyotrophic lateral sclerosis (ALS) therapy was not ready for prime time—but perhaps this latest rejection will really send the message home.
The New York biotech has received a refusal to file letter from the FDA, a rebuke reserved for when a drug application has significant deficiencies and the agency simply declines to review it. NurOwn was rejected back in March 2021 in a rare public letter from the FDA. The agency said the data provided to support the application were "not at all statistically significant."
BrainStorm’s shares tanked as the market opened Thursday, falling 60% to $1.15 compared to a closing price yesterday of $2.89.
The original application was based on a phase 3 trial that failed to slow disease progression when compared with placebo. But, after shifting the analysis, BrainStorm vowed to forge on, claiming patients with less progressed disease had a reduced rate of disease progression.
Armed with “continued analysis” and feedback from scientific presentations of the phase 3 data, the company resubmitted the application in August. It's unclear whether the company had the FDA's blessing or encouragement at the time of the resubmission.
Now, the agency is once again saying no with the refusal to file letter. The FDA has said that BrainStorm may request a meeting to discuss the reasons for the letter.
CEO Chaim Lebovits says the company will do just that, and he continued to tout the phase 3 data that backed up the new biologics license application. The company will provide further details on its corporate strategy and NurOwn's development in a Monday earnings call.
At the time of the initial refusal, the FDA acknowledged that ALS patients are in desperate need of treatment options for the debilitating and deadly disease that causes loss of muscle control. But the agency stressed that the data just did not support a clinical benefit, and therefore an approval of NurOwn.
Since then, the FDA has approved another ALS treatment from Amylyx Pharmaceuticals called Relyvrio. ALS patients and advocates urged the agency to approve the treatment despite shaky evidence of benefit in an effort to get a shot at slowing down the disease.