FDA Backs BrainStorm’s Design for Phase IIIb Trial in Early-Stage ALS

FDA Exterior/iStock, Grandbrothers

Pictured: Entrance to FDA building in Silver Spring, Maryland/iStock, Grandbrothers

BrainStorm Cell Therapeutics announced Tuesday that under a Special Protocol Assessment the FDA has agreed to the design of a Phase IIIb trial for the company’s NurOwn amyotrophic lateral sclerosis candidate. 

After an FDA advisory committee voted 17-1 against NurOwn in September 2023, BrainStorm withdrew its Biologics License Application in October 2023 and plotted a potential path forward with plans for a Phase IIIb trial. Tuesday’s announcement about the Special Protocol Assessment (SPA) agreement with the FDA validates the trial’s protocol and statistical analysis for the planned late-stage study. 

“We are pleased to have reached an agreement with the FDA on key elements of the Phase IIIb trial design that provides a potential path forward towards obtaining regulatory approval,” BrainStorm CEO Chaim Lebovits said in a statement. 

While the agreement holds no guarantee of approval or indicates FDA concurrence on every trial protocol, Lebovits contends that the SPA will “de-risk” aspects of the clinical program and potentially bring the asset one step closer to market. The trial will commence this year with two parts—a placebo-controlled period of 24 weeks followed by an open-label extension. 

The trial design validated by the agency’s SPA sets the primary endpoint as a change in the Revised Amyotrophic Lateral Sclerosis Functional Rating (ALSFRS-R) total score from baseline to Week 24. A combined assessment of function and survival will be utilized. Blood samples and spinal fluid collected will showcase biomarkers of neuroinflammation, neurodegeneration and neuroprotection. 

NurOwn is a stem cell therapeutic in which a patient’s bone marrow is drawn out and mesenchymal stem cells (MSC) are procured to manufacture each treatment. The MSC cells are induced to secrete neurotrophic factors, which support neuronal growth and survival to potentially combat the degeneration of the motor neurons affected by ALS. 

The FDA’s advisory committee in September 2023 voted overwhelmingly against approving NurOwn, citing “conflicting” information and a “lack of efficacy” in terms of survival. The trial showed a response rate only slightly higher than the placebo response—34.7% versus 27.7%, a higher than usual placebo response rate than other ALS trials. However, a subgroup of patients with earlier stage disease showed a wider margin with 34.6% of NurOwn treated patients showing a clinically significant response compared to only 15.6% in the placebo group. Early-stage ALS is the target population for BrainStorm’s upcoming Phase IIIb trial. 

BrainStorm’s stock traded higher in premarket trading Tuesday on news of the FDA’s trial design agreement. The company’s stock price nosedived last fall and is down 85% over the last year, though it has been slowly recovering since the submission of its SPA request in February 2024. 

Kate Goodwin is a freelance life science writer based in Des Moines, Iowa. She can be reached at kate.goodwin@biospace.com and on LinkedIn.   

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