BrainStorm Announces Completion of All Dosing in NurOwn® Phase 3 Clinical Trial in ALS

Rhea-AI Summary

BrainStorm Cell Therapeutics (NASDAQ: BCLI) completed participant dosing in its Phase 3 trial of NurOwn® for amyotrophic lateral sclerosis (ALS). Approximately 200 participants received either MSC-NTF cells or a placebo over four months. The company anticipates top-line data by Q4 2020, aligning with their enrollment timeline. CEO Chaim Lebovits emphasized this milestone's importance for a potential Biologics License Application. The trial is being conducted at six renowned medical centers, and the company has a recent FDA clearance for a Phase 2 trial in progressive multiple sclerosis.

Positive

• Completion of participant dosing in Phase 3 trial.
• Anticipated release of top-line data by Q4 2020.
• CEO's statement on potential Biologics License Application for MSC-NTF cells.
• Conducted at six prestigious medical centers.
• FDA clearance for Phase 2 trial in progressive multiple sclerosis.

Negative

• Risks associated with regulatory approvals.
• Need for additional capital to support trials.
• Dependence on ongoing clinical trial success for future revenue.

NEW YORK, July 2, 2020 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, announced today that final participant dosing has been administered in the Phase 3 pivotal trial of NurOwn® (MSC-NTF cells) in amyotrophic lateral sclerosis (ALS).  The trial enrolled approximately 200 participants, randomized 1:1 to receive three doses of MSC-NTF cells or placebo, administered over four months.  As previously reported, the Company expects top-line data from the trial to be available in the fourth quarter of 2020, consistent with the timeline established upon trial enrollment.

 

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"Completion of participant dosing in this clinical trial is an important milestone and brings us a step closer to potentially filing a Biologics License Application to make MSC-NTF cells available to people with ALS," Chaim Lebovits, CEO of BrainStorm stated. "I would like to thank the investigators and their staff at the participating sites for their clinical excellence, especially for enabling this trial to complete on time in the middle of the ongoing COVID-19 pandemic. I must also express my complete gratitude to the trial participants and their loved ones who fully devoted themselves to the challenges of bringing an investigational therapeutic forward. We look forward to the data readout later in 2020."

The Phase 3 NurOwn trial is being conducted at six centers of excellence: University of California, Irvine; Cedars-Sinai Medical Center; California Pacific Medical Center; Massachusetts General Hospital; University of Massachusetts Medical School and Mayo Clinic.

About NurOwn®       

The NurOwn technology platform (autologous Mesenchymal stem cells, MSC-NTF cells) represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. With this press release, BrainStorm has now fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received acceptance from the U.S. Food and Drug Administration (FDA) to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS) and initiated enrollment in March 2019.

About BrainStorm Cell Therapeutics Inc.   

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company 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 status designation 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 fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm also recently received U.S. FDA clearance to initiate a Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. For more information, visit the company's website at www.brainstorm-cell.com

Safe-Harbor Statement       

Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern, regulatory approval of BrainStorm's NurOwn treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; 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:
Preetam Shah, MBA, PhD
Chief Financial Officer
BrainStorm Cell Therapeutics Inc.
Phone: + 1.862.397.1860
pshah@brainstorm-cell.com

Media:

Paul Tyahla
SmithSolve
Phone: + 1.973.713.3768
Paul.tyahla@smithsolve.com

 

 

 

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SOURCE Brainstorm Cell Therapeutics Inc

FAQ

What is the purpose of the Phase 3 trial for BCLI?

The Phase 3 trial aims to evaluate the efficacy of NurOwn® (MSC-NTF cells) in treating amyotrophic lateral sclerosis (ALS).

When will data from the Phase 3 trial of BCLI be available?

Top-line data from the Phase 3 trial is expected in the fourth quarter of 2020.

How many participants were involved in the BCLI trial?

Approximately 200 participants were enrolled in the Phase 3 trial.

What are the next steps for BCLI after the trial?

Following the trial, BCLI may file for Biologics License Application based on the trial results.

What recent FDA actions have impacted BCLI?

BCLI received FDA clearance to initiate a Phase 2 trial for progressive multiple sclerosis.