FDA advisory committee (ADCOM) meeting to review NurOwn^® for ALS

NEW YORK, Sept. 27, 2023 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that Nasdaq has halted trading of the Company's common stock.

The U.S. Food and Drug Administration's (FDA) Cellular, Tissue and Gene Therapies Advisory Committee is meeting today to review BrainStorm's Biologics License Application (BLA) for NurOwn^® for the treatment of amyotrophic lateral sclerosis (ALS). The ADCOM meeting is scheduled to take place 10:00 am to 6:00 pm ET.

The FDA has posted briefing materials, agendas and webcast information for the meeting that can be accessed on the Agency's website here. The online web conference meeting can be viewed through this link.

BrainStorm's BLA for NurOwn^® has a Prescription Drug User Fee Act (PDUFA) action date targeted to occur by December 8, 2023.

About NurOwn^®

The NurOwn^® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are harvested from each person with ALS and are manufactured using an innovative and proprietary process to secrete neurotrophic factors to target specific neurodegenerative diseases. The lead program for NurOwn® is for the treatment of ALS, which is under FDA review. BrainStorm's long-term commitment to ALS is demonstrated in preclinical research and a series of clinical studies, all of which have been published in peer-reviewed journals.

The Phase 3 pivotal trial of NurOwn® did not reach statistical significance on the primary or secondary endpoints, likely due to a "floor effect," which confounds measurement of disease progression in patients with more advanced disease. A thorough analysis of NurOwn® Phase 3 data shows evidence of clinically meaningful effectiveness in ALS participants who have not progressed to advanced levels of disease progression. In a pre-specified group of participants with an ALSFRS-R score ³35, there was a larger treatment effect across all endpoints with NurOwn® compared to placebo, which aligned with historical trials and the study power assumptions and resulted in a statistically significant difference on a key endpoint (change from baseline in ALSFRS-R). Additionally, a post-hoc sensitivity analysis of patients across threshold of >26 through ≥35 on the ALSFRS-R highlighted that NurOwn®-treated patients retain, on average, two points of function more compared to placebo–clinically meaningful preservation and important for quality of life for a person living with ALS and their loved ones. 

NurOwn®'s clinical program also included the most robust cerebrospinal fluid (CSF) biomarker study ever done in ALS, strong and consistent biomarker data, which are predictive of clinical response in the trial, span pathways that are important to ALS (neuroinflammation, neurodegeneration, neuroprotection), and align with NurOwn®'s mechanism of action. Biomarker data in all trial participants showed consistent biological patterns of NurOwn® reducing markers of inflammation and neurodegeneration and increasing neuroprotective markers relative to placebo. Biomarker patterns were consistent across all NurOwn® participants, including in those with Advanced ALS disease where clinical scales, such as the ALS Functional Rating Scale, have demonstrated measurement challenges. Three CSF biomarkers were predictive of clinical outcomes in NurOwn®-treated participants–NfL, galectin-1, latency associated peptide of TGF-beta1 (LAP or TGF-b). 

The NurOwn® clinical program has generated valuable insights into the pathology of ALS, as well as disease progression and treatment. Since the initial Phase 3 readout, BrainStorm has shared the full dataset through rigorous peer-reviewed analysis, including: quantification of Floor Effect, which had been noted but never before explored in depth; evaluation of multiple pre-specified biomarkers, collected at seven different points across 20 weeks during the trial, allowing a longitudinal view; and analysis of genetic data, which represents one of the first ALS trials to prospectively invoke pharmacogenomic analysis of clinical outcome, offering great promise for the development of future treatments for ALS.

BrainStorm previously announced the FDA's intention to hold an ADCOM meeting to review NurOwn® for the treatment of ALS. BrainStorm filed a BLA for NurOwn® on September 9, 2022, and received a Refusal to File (RTF) letter from FDA on November 8, 2022. Following a Type A meeting and subsequent discussions with the FDA BrainStorm requested that CBER utilize the FDA's "File Over Protest" procedure, which offers the shortest amount of time to complete the regulatory process. The BLA was filed over protest, allowing completion of the regulatory process in the shortest time possible, and active review resumed on February 7, 2023.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. BrainStorm holds the rights to clinical development and commercialization of the NurOwn® technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive MS and was supported by a grant from the National MS Society (NMSS).

Notice Regarding Forward-Looking Statements

This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding the ADCOM meeting related to NurOwn®, the timing of a PDUFA action date for the BLA for NurOwn®, and the clinical development of NurOwn® as a therapy for the treatment of ALS. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn®, whether BrainStorm's future interactions with the FDA will have productive outcomes, 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 considered carefully, 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 do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements.

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CONTACTS

Investor Relations: 
John Mullaly
LifeSci Advisors, LLC
Phone: +1 617-429-3548
jmullaly@lifesciadvisors.com

Media: 
Lisa Guiterman
Phone: +1 202-330-3431
lisa.guiterman@gmail.com 

SOURCE BrainStorm Cell Therapeutics Inc.