A year after FDA hit the breaks, BrainStorm hits the gas with updated data, approval plans

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Nearly a year and a half after the FDA publicly raised BrainStorm Cell Therapeutics’ stem cell-based amyotrophic lateral sclerosis (ALS) treatment, the company is back and asking regulators for approval.

BrainStorm said in Monday’s second-quarter earnings report that thanks to “ongoing analysis” and “feedback” from scientific presentations related to Phase 3 data for its Uncrown therapy, the company is more confident than ever that its treatment is having an impact.

Key to the biotech’s renewed attempts at approval is an error in a December 2021 publication in the journal Muscle & Nerve, which now shows that when phase 3 data was scored by patients based on rates of ALS progression, the treatment was actually found to be statistically significant. In other words, in patients with less progressive disease, the treatment significantly reduced the rate of disease progression compared to placebo.

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The release of the redacted data and subsequent decision to push for approval marks the company’s second act after a nearly year-and-a-half hiatus prompted by the FDA’s public rejection in March 2021. At the time, the agency said the data “were not statistically significant at all” and that a modest increase in response rates among treated group was “probably due to chance”.

In an interview with Fierce Biotech, BrainStorm Chief Development Officer Stacy Lindborg, Ph.D., would not say whether the company has received the FDA’s blessing for its latest plans to seek approval. “Negotiations with the FDA are certainly ongoing. We shared our mistakes before they were published,” said Lindborg.

A spokesperson for the agency did not respond to a request for comment by the time of publication on whether it had been informed of the company’s plans to apply for approval.

BrainStorm first reported phase 3 data in November 2020 and found that 34.7% of treated patients reported slowing the rate of disease progression compared to 27.7% on placebo.

When asked why the company isn’t conducting another NurOwn study, Lindborg said the urgency of the disease calls for quick action.
she said. The likelihood that a smaller study will detect a treatment effect given the heterogeneity of the disease is “very low,” Lindborg added.

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The BrainStorm decision revives a frequent debate about the clinical development of neurological diseases: Should any hint of success be enough to give patients the option of treatment? It’s a question that has also surfaced for fellow ALS developer Amylyx, as well as Alzheimer’s treatment Biogen Ad helm.

Asked how much the company plans to charge for the mode, Lindborg said the company had no comment before adding that it was aiming for “fair pricing.” In an earlier fireside chat, FDA Commissioner Robert Calif said the agency does not and will not consider pricing in its approval decisions.

Although it’s unclear whether approval was the path recommended by regulators for Uncrown, Brainstorm expects the data to be further reviewed by an advisory committee. An open forum for stakeholders to further analyze the data has “very positive aspects”, Lindborg said.