FDA Rejects BrainStorm Cell Therapeutics’ BLA For ALS Drug

FDA has refused to accept BrainStorm Cell Therapeutics’ New Biologics License Application (BLA) for NurOwn for the treatment of amyotrophic lateral sclerosis, or ALS. 

The company in August asked the FDA to approve its stem cell therapy despite missing primary endpoints in a Phase 3 trial in 2020. 

The New York-based drugmaker has stated its intent to request a Type A meeting with the regulator to discuss the content of the refusal to file letter. 

“We continue to believe that NurOwn’s Phase 3 trial represents a significant contribution to ALS therapy and will continue to work tirelessly to address the needs of people living with ALS by advancing science and partnering with researchers around the world,” said Chaim Lebovits, CEO of BrainStorm. 

The company will discuss its corporate strategy and plans for NurOwn’s development on its Q3 2022 earnings call on November 14, 2022. 

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Missing Phase 3 Primary Endpoints 

NurOwn is a stem cell therapy where mesenchymal stem cells (MSCs) are engineered to produce high levels of neurotrophic factors (NTF) which encourage neuronal differentiation, growth, and survival. Autologous MSC-NTF cells are designed to deliver multiple NTFs and immunomodulatory cytokines to the site of damage to slow disease progression. 

The placebo-controlled Phase 3 NurOwn trial evaluated 189 ALS patients. While clinical response was shown to improve with NurOwn treatment, it did not meet the primary endpoint as the response rate in the placebo group was higher than expected. 

Additional analyses of the study led to BrainStorm discovering an error in the statistical methods used to assess the results of NurOwn. The correction meant NurOwn met the secondary goal regarding the average change on a rating scale that measures the severity of ALS symptoms in a prespecified group of participants, BrainStorm said. 

NurOwn also reduced biomarkers of inflammation and neurodegeneration, which supported the idea that trial participants are experiencing positive biological effects from the treatment, the company said. 

The three principal investigators (PIs) of the Phase 3 study, Drs. Brown, Cudkowicz, and Windebank jointly expressed, “While the pre-specified primary outcome measure was not met, there were participants with beneficial clinical effects and overall changes in relevant biomarkers of drug effect. 

“Understanding whether there are people with ALS who might respond better to NurOwn is important given the unmet therapeutic need. As the three co-PIs of the Phase 3 study of NurOwn, we support continued discussions with the FDA on the best path forward.”

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Joy Lin

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