Almost a year and a half after the FDA publicly spiked BrainStorm Cell Therapeutics’ stem cell-based amyotrophic lateral sclerosis (ALS) therapy, the company is back to ask regulators for approval.
BrainStorm said in a second-quarter earnings report Monday that thanks to “continued analysis” of and “feedback” from scientific presentations related to phase 3 data for the NurOwn therapy, the company is more confident than ever that its treatment is impactful.
Key to the biotech’s renewed attempts at approval is an erratum to a December 2021 publication in the journal Muscle & Nerve, which now shows that when the phase 3 data was delineated by patients’ scores on a measure of ALS progression, the treatment was in fact found to be statistically significant. In other words, among patients with less progressed disease, the treatment reduced the rate of disease progression significantly compared to placebo.
The release of the amended data and subsequent decision to push ahead with approval marks the second act for the company after a nearly year and a half long intermission spurred by the FDA’s public rejection in March 2021. At that time, the agency said that the company’s data “was not at all statistically significant” and that the slight increase in response rates among the treated group was “likely due to chance.”
In an interview with Fierce Biotech, BrainStorm’s chief development officer Stacy Lindborg, Ph.D., wouldn’t say whether the company has received the FDA’s blessing for its latest plans to apply for approval. “We certainly have ongoing discussions with the FDA. We did share our erratum before it was made public,” Lindborg said.
A spokesperson from the agency did not respond to a request for comment by the time of publication about whether it had been notified about the company’s plans to seek approval.
BrainStorm first reported the phase 3 data in November 2020, finding that 34.7% of treated patients had reported a slowing of the rate of disease progression compared to 27.7% for placebo.
When asked why the company isn’t pursuing an additional study of NurOwn, Lindborg said that the urgency of the disease requires quick action. “We know that if we start over with another trial, every single patient, or almost every patient, living with ALS today, will die in the process before we’re able to complete a trial.” she said. The likelihood of a smaller trial uncovering a treatment effect giving the heterogeneity of the disease is “very tenuous,” Lindborg added.
BrainStorm’s decision reinvigorates a frequent debate regarding the clinical development of neurological diseases: Should any hint of success be enough to give patients an option for treatment? It’s a question that’s also bubbled to the surface for fellow ALS developer Amylyx as well as Biogen’s Alzheimer’s treatment Aduhelm.
When asked how much the company was planning to charge for this regimen, Lindborg said the company wasn’t commenting before adding that it sought “fair pricing.” FDA commissioner Robert Califf had said in a previous fireside chat that the agency does not and would not consider pricing in its approval decisions.
Although it’s unclear whether or not approval was the route recommended by regulators for NurOwn, BrainStorm is anticipating an advisory committee will further assess the data. There are “very positive aspects” to an open forum for stakeholders to further dissect the data, Lindborg said.