BrainStorm Announces High Level FDA Feedback on NurOwn® ALS Clinical Development Program
NEW YORK, February 22, 2021 / PRNewswire / – BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that it has recently met with senior management at Food and US Drug Administration (FDA) and receiving FDA feedback on a high-level data summary from the NurOwn phase 3 clinical trial for ALS (MSC-NTF autologous cells). The FDA concluded from its initial review that the current level of clinical data does not provide the threshold for substantial evidence that the FDA seeks to support a Biologics License Application (BLA). Additionally, the FDA has indicated that this recommendation does not prevent Brainstorm from proceeding with a BLA submission.
“Brainstorm will first consult with principal investigators, ALS experts, expert statisticians, regulatory advisers, 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 would like to thank our independent advisors for their frankness and unwavering commitment to our SLA program. We also wish to warmly thank all ALS patients and their families who participated in this clinical research program. The company will continue to rely on the strength of its wonderful professional and dedicated team while being well funded in a current position of over $ 40 million in liquid. We will soon be announcing our next flagship product and program for other diseases with unmet needs. “
“Many of us with long experience in the development of therapy for ALS agree that there was evidence for the benefits of NurOwn cell therapy and hope that there will be an opportunity for further evaluation of this. modality in ALS, “said Robert brown, DPhil, MD, director of the neurotherapy program at University of Massachusetts School of Medicine.
“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 are responding to treatment with NurOwn is a light at the end of the tunnel. Careful study of the biomarkers associated with the response will help us move towards a widely effective therapy. The patients and their families who have brought us to this point are true heroes. ”
“I share my deep gratitude and thanks to the patients and families who participated in this Phase 3 trial and to 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 Julieanne Dorn Professor of Neurology, Harvard Medical School. “We learned a lot about the effectiveness and safety of NurOwn in people with ALS in this well-conducted trial. We also learned some of the challenges of using ALSFRS-R on the lower end of scale. Further discussions with the community and sharing all the data in a peer-reviewed publication are critical next steps. “
“We will complete all analyzes from the Phase 3 ALS study to support scientific communication of the Phase 3 data, including a peer-reviewed manuscript,” said Ralph kern MD MHSc, President and Medical Director Brainstorm Cell Therapeutics. “The results of the Phase 3 clinical trial are very important and we are confident that the analysis of the major biomarkers of ALS disease will contribute to our understanding of ALS. Our goal is to advance our proprietary cellular technology platform and prioritize data-driven product development efforts and business priorities. “
The NurOwn® Technology platform (autologous MSC-NTF cells) represents a promising experimental therapeutic approach to target disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous bone marrow-derived mesenchymal stem cells (MSC) that have been grown and differentiated ex vivo. MSCs are converted into MSC-NTF cells by culturing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTF). Autologous MSC-NTF cells can efficiently deliver several NTFs and immunomodulatory cytokines directly to the site of damage to induce a desired biological effect and ultimately slow or stabilize disease progression.
About BrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapies for debilitating neurodegenerative diseases. The Company owns the clinical development and commercialization rights for NurOwn® technology platform used to produce autologous MSC-NTF cells under an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have been granted orphan drug status from the United States Food and Drug Administration (FDA) and European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a pivotal Phase 3 trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeated administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm is also conducting an FDA-approved, multi-center, open-label, phase 2 trial in progressive multiple sclerosis (MS). The phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) completed the assay in December 2020, and the first results are expected by the end of the first quarter of 2021.
For more information, visit the company’s website at www.brainstorm-cell.com.
Safe Harbor Declaration
Statements in this announcement other than historical data and information, including statements regarding the future NurOwn® manufacturing and clinical development plans, constitute “forward-looking statements” and involve risks and uncertainties which could cause the actual results of BrainStorm Cell Therapeutics Inc. to differ materially from those stated or implied by these forward-looking statements. . Terms and expressions such as “may”, “should”, “could”, “could”, “will”, “expect”, “probably”, “believe”, “plan”, “estimate”, “predict” “, Potential”, and similar terms and expressions are intended to identify such forward-looking statements. Potential risks and uncertainties include, but are not limited to, BrainStorm’s need to raise additional capital, BrainStorm’s ability to continue as a going concern , the outlook for regulatory approval for NurOwn from BrainStorm® candidate for treatment, initiation, completion and success of BrainStorm’s product development and research programs, regulatory and personnel issues, development of a global market for our services, ability to secure and to maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm’s NurOwn® candidate for treatment to gain wide acceptance as a treatment option for ALS or other neurodegenerative diseases, the ability of BrainStorm to manufacture or use third parties to manufacture and market the NurOwn® candidate for treatment, obtaining patents that offer significant protection, competition and market developments, BrainStorm’s ability to protect our intellectual property from third party infringement, healthcare reform legislation, demand for our services, exchange rates and product liability claims and disputes; and other factors detailed in BrainStorm’s annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be carefully considered and readers should not place undue reliance on BrainStorm’s forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We assume no obligation to update any forward-looking statements to reflect actual results or assumptions if circumstances or management’s beliefs, expectations or opinions should change, except as required by law otherwise. Although we believe that the expectations reflected in forward-looking statements are reasonable, we cannot guarantee future results, activity levels, performance or achievements.
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SOURCE Brainstorm Cell Therapeutics Inc