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BrainStorm Cell Therapeutics to Launch NurOwn Phase IIIb Trial This Year With FDA Feedback

NEW YORK – BrainStorm Cell Therapeutics on Tuesday said it will launch a Phase IIIb trial of its embattled stem cell therapy NurOwn (debamestrocel) in amyotrophic lateral sclerosis (ALS) this year after receiving feedback from the US Food and Drug Administration.

The New York City-based biotech said it has received written communication from the FDA under a special protocol assessment (SPA), a process through which drugmakers can get input from the regulator on clinical trial design. Based on the agency's feedback on the design of BrainStorm's proposed Phase IIIb trial for NurOwn, the company expects the results from this study may support a biologics license application (BLA). However, an SPA agreement does not indicate that the FDA agrees with every protocol detail.

"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 President and CEO Chaim Lebovits said in a statement. "We believe that having this SPA in place will help de-risk certain regulatory aspects of the NurOwn clinical program."

NurOwn uses multipotent stem cells derived from a patient's own bone marrow, which are induced to secrete high levels of neurotrophic factors that can support neuronal growth and survival.

BrainStorm has previously tried to take NurOwn through the FDA without success, most recently withdrawing its BLA in October on the heels of negative feedback from the FDA and an unfavorable reception from the agency's Cellular, Tissue, and Gene Therapies Advisory Committee. Despite setbacks, BrainStorm has said it is committed to developing NurOwn in ALS and that the stem cell therapy is essential to its business strategy, as it envisions it as a platform for advancing similar treatments for other neurodegenerative diseases.

BrainStorm said it expects to launch the Phase IIIb study sometime this year, after reviewing the protocol with investigators, securing institutional review board approvals, and engaging with the ALS community.

In the multicenter study, BrainStorm plans to evaluate the efficacy and safety of NurOwn in patients at an earlier stage of ALS than they were in previous trials. In the randomized-controlled part of the study, up to 200 patients will receive three repeated intrathecal injections of NurOwn or placebo every eight weeks. Patients in both study arms will be able to continue to receive other approved ALS treatments during the study.

Participants who complete that portion of the trial will have the option to enroll in an open-label extension study. 

Investigators will assess NurOwn's ability to slow disease progression based on change from baseline according to the Revised Amyotrophic Lateral Sclerosis Functional Rating score. They will also collect cerebrospinal fluid and blood samples for analysis of biomarkers related to neuroinflammation, neurodegeneration, and neuroprotection.