FDA clears additional NurOwn® dosage as part of expanded access program


NEW YORK, Dec. 27 2021 / PRNewswire / – BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cell therapy for neurodegenerative diseases, today announced plans to expand the dosage of NurOwn® for participants who have taken the Extended Access Protocol (EAP). The United States Food and Drug Administration (FDA) has recommended that BrainStorm submit an amendment to the EAP protocol to provide additional dosing for these participants. Under the original EAP protocol, participants who completed the Phase 3 NurOwn trial and met specific eligibility criteria were given the option of receiving 3 doses of NurOwn. Under the amended EAP protocol, these eligible participants will receive up to 3 additional doses. Data collected from the original EAP treatments informed the decision to go ahead with additional doses for participants who completed it.

“This assay extension for the Expanded Access Protocol is an appropriate next step following new assay and biomarker results from the Phase 3 study. It is deeply appreciated by our patients with ALS. Eligible patients now have the option of receiving up to 9 doses of NurOwn in total, allowing for the collection of additional data to better understand the potential benefits of longer-term treatment, ”said Robert Brown, MD DPhil, incumbent Leo P. and Theresa M. LaChance Chair in Medical Research and Chair of the Department of Neurology, University of Massachusetts Medical School and UMass Memorial Medical Center, and one of the principal investigators of the Phase 3 NurOwn® study.

“I congratulate BrainStorm for having the conviction to continue with their EAP, which has brought real hope to those who are part of it. I am also thankful that they recognize the value and importance of EAPs and are willing to invest to get them done right despite being a small business. I look forward to the day, hopefully very soon, when everyone living with ALS will have access to NurOwn, ”said Brian wallach, person living with ALS and co-founder of I AM ALS.

Chaim Lebovits, CEO of Brainstorm Cell Therapeutics, said, “We are delighted to be able to provide additional treatments to these patients. This program is the result of a successful collaboration between the FDA, patient advocacy groups and Brainstorm. We look forward to continuing this dialogue with the FDA for the best way forward. ”

The NurOwn EAP was established in 2020, following the conclusion of the Phase 3 trial, and the protocol was developed in partnership with the FDA. Patients were treated at one of six centers of clinical excellence that participated in the phase 3 trial. These six centers are: University of California, Irvine; Cedars-Sinai Medical Center; California Pacific Medical Center; Massachusetts General Hospital; University of Massachusetts School of Medicine; and Mayo Clinic. The FDA has approved BrainStorm to manufacture NurOwn for the EAP program at Catalent’s facility in Houston, Texas.

According to the FDA, EAPs, also known as “compassionate use” programs, provide appropriate patients with a route to receive an investigational drug for the treatment of a serious illness or condition other than a clinical trial when no comparable or satisfactory alternative treatment option is available. .

About NurOwn®

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 are designed to efficiently deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage in order to elicit the desired biological effect and ultimately to 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 ALS trial (NCT03280056) that was published in Muscle and nerve; this trial examined 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), in addition to a grant from I AM ALS and the ALS Association. BrainStorm has completed an investigational new drug application, a phase 2, open-label, multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS).

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 future clinical trials, the continued development of NurOwn® and the potential therapeutic effects and expected patient population of NurOwn®, 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 indicated or implied by these forward-looking statements. Terms and expressions such as “may”, “should”, “should”, “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 its business. operations, 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 offering significant protection, competitive and market developments, BrainStorm’s ability to protect our intellectual property from infringement by third parties, healthcare reform legislation, request 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 should circumstances or management’s beliefs, expectations or opinions 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.


Brainstorm on contacts

Investor Relations:
John mullaly
LifeSci Advisors, LLC
Telephone: +1 617-429-3548

Mary kay turner

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SOURCE Brainstorm Cell Therapeutics Inc


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