BrainStorm Cell Therapeutics Inc (BCLI)

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BrainStorm Announces High-Level FDA Feedback on NurOwn® ALS Clinical Development Program
FDA recommended generating additional clinical data to meet FDA's BLA requirements

https://finance.yahoo.com/news/brainstorm-announces-high-level-fda-130500410.html

NEW YORK, Feb. 22, 2021 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, announced today that it recently met with senior leadership from the U.S. Food and Drug Administration (FDA) and received FDA feedback on a high-level data summary from the NurOwn (autologous MSC-NTF cells) ALS Phase 3 clinical trial. The FDA concluded from their initial review that the current level of clinical data does not provide the threshold of substantial evidence that FDA is seeking to support a Biologics License Application (BLA). In addition, the FDA advised that this recommendation does not preclude Brainstorm from proceeding with a BLA submission.

"Brainstorm will first consult with principal investigators, ALS experts, expert statisticians, regulatory advisors, and ALS advocacy groups to assess the benefit/risk of a BLA submission before making a final decision," said Chaim Lebovits, CEO Brainstorm Cell Therapeutics. "We want to thank our independent advisors for their candor and unwavering commitment to our ALS program. We also would like to give a heartfelt thank you to all ALS patients and their families that were involved in this clinical research program. The company will continue to build on the strength of its wonderful, professional and dedicated team while being well funded at a current position of over $40M in cash. We will soon announce our next flagship product and program for other diseases with unmet needs."

"Many of us with longstanding experience in ALS therapy development agree that there was evidence of benefit from NurOwn cell therapy and hope that there will be an opportunity for further assessment of this modality in ALS," said Robert Brown, DPhil, MD, Director of the Program in Neurotherapeutics at the University of Massachusetts Medical School.

"ALS is a devastating disease with worse outcomes than most forms of cancer," said Anthony Windebank MD, Jean and Judith Pape Adams Professor of Neuroscience, Mayo Clinic College of Medicine and Science. "The clear signal in this trial that some patients with ALS respond to treatment with NurOwn is a light at the end of the tunnel. The careful study of biomarkers associated with response will help lead us forward towards a broadly effective therapy. The patients and their families who have brought us to this point are true heroes."

"I share my profound gratitude and thanks to the patients and families that participated in this phase 3 trial and all the teams at the six sites," said Merit Cudkowicz MD, Chief of Neurology and Director of the Healey & AMG Center for ALS at Mass General Hospital, and the Julieanne Dorn Professor of Neurology, Harvard Medical School. "We learned a lot about the efficacy and safety of NurOwn in people with ALS in this well conducted trial. We also learned some of the challenges with the use of ALSFRS-R at the lower end of the scale. Additional discussions with the community and sharing all the data in a peer reviewed publication are critical next steps."

"We will complete all ALS phase 3 study analyses to support scientific communication of the phase 3 data, including a peer-reviewed manuscript," said Ralph Kern MD MHSc, President and Chief Medical Officer Brainstorm Cell Therapeutics. "The results of the phase 3 clinical trial are very important, and we are confident that the analysis of key ALS disease biomarkers will contribute to our understanding of ALS. Our focus is to advance our proprietary cellular technology platform and to prioritize product development efforts based on data and business priorities."