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BrainStorm Cell Therapeutics Reaches Alignment with FDA on CMC Aspects of Phase 3b NurOwn® Clinical Trial

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BrainStorm Cell Therapeutics (NASDAQ: BCLI) has reached alignment with the FDA on the Chemistry, Manufacturing, and Controls (CMC) aspects of its Phase 3b clinical trial for NurOwn, a therapy for amyotrophic lateral sclerosis (ALS). This development follows the FDA’s Special Protocol Assessment (SPA) agreement granted in April 2024, which provided clarity on the trial design and endpoints. The Type C meeting resolved all outstanding CMC questions, allowing BrainStorm to proceed with the pivotal trial. Chaim Lebovits, CEO, expressed satisfaction with the outcome, emphasizing the importance of alignment in manufacturing complexities and the company's commitment to advancing ALS treatment.

Positive
  • Reached alignment with FDA on CMC aspects of Phase 3b trial.
  • FDA granted Special Protocol Assessment (SPA) in April 2024.
  • Resolution of all outstanding CMC questions.
  • Positive momentum for pivotal Phase 3b trial.
Negative
  • No significant negative business aspects identified in the PR.

BrainStorm Cell Therapeutics securing alignment with the FDA on the Chemistry, Manufacturing and Controls (CMC) aspects of their Phase 3b clinical trial for NurOwn® marks a significant regulatory milestone. This de-risks the investment from a regulatory standpoint and provides more certainty on the progression timeline for the trial. For the financial health of the company, this can lead to increased investor confidence and potentially bolster the stock price in the short term.

It’s important to note that moving into a Phase 3b trial implies that the company is one step closer to potentially bringing NurOwn® to market. This can be seen as a positive signal for future revenue streams. However, it’s critical to monitor the company's burn rate and financial sustainability as clinical trials are capital intensive.

In the long term, if the Phase 3b trial results are positive and lead to FDA approval, BrainStorm could see substantial revenue growth as it taps into the ALS treatment market. On the flip side, investors should be aware of the risks involved with clinical trials, as negative results could significantly impact the stock adversely.

The alignment with the FDA on CMC aspects of the Phase 3b trial is key for BrainStorm's progression. CMC aspects focus on ensuring the product is consistently manufactured and controlled to meet quality standards. This is especially critical for cell therapy products like NurOwn® due to their inherent complexities.

This regulatory milestone reduces uncertainties regarding manufacturing processes, which is a major hurdle in the development of cell therapies. Furthermore, the Special Protocol Assessment (SPA) agreement previously secured ensures that the trial design and endpoints align with FDA expectations, which may expedite the approval process if the trial results are favorable.

For retail investors, understanding that these steps not only streamline the path to potential approval but also enhance the credibility and feasibility of NurOwn® is vital. However, the science behind stem cell therapy and its application in neurodegenerative diseases like ALS remains complex and high-risk, making it important to stay informed about ongoing trial results and any emerging scientific data.

NEW YORK, June 26, 2024 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced it has reached alignment with the U.S. Food and Drug Administration (FDA) on the Chemistry, Manufacturing, and Controls (CMC) aspects of Brainstorm's Phase 3b clinical trial for NurOwn®, its investigational therapy for amyotrophic lateral sclerosis (ALS).

 

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This Type C meeting builds upon the positive momentum established in April 2024, when the FDA granted BrainStorm a Special Protocol Assessment (SPA) agreement for its NurOwn Phase 3b trial. The SPA agreement provided clarity on the design and endpoints of the trial, significantly de-risking the regulatory aspects of the program.

"This in-person Type C meeting with the FDA was an important step as we finalize preparations for our pivotal Phase 3b trial of NurOwn. We are very pleased with the outcome and are now aligned with the FDA on resolving all previously outstanding CMC questions," said Chaim Lebovits, President & Chief Executive Officer of BrainStorm. "As with any cell therapy products, there are additional complexities in the manufacturing process, and it is important that we have alignment in advance with all the relevant groups within the FDA. We are committed to working closely with the FDA and to helping the ALS community, and are excited to move forward with the Phase 3b trial. Our ultimate goal is to achieve approval for NurOwn in order to address the unmet needs of patients."

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 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), and another grant from the ALS Association and I AM ALS. 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 meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. 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.

CONTACTS:

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

IR:
Michael Wood
Phone: +1 646-597-6983
mwood@lifesciadvisors.com 

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SOURCE BrainStorm Cell Therapeutics Inc.

FAQ

What recent alignment did BrainStorm Cell Therapeutics reach with the FDA?

BrainStorm Cell Therapeutics reached alignment with the FDA on the Chemistry, Manufacturing, and Controls (CMC) aspects of its Phase 3b NurOwn clinical trial for ALS.

What is the significance of the Special Protocol Assessment (SPA) agreement for BrainStorm's Phase 3b trial?

The SPA agreement provided clarity on the trial design and endpoints, significantly de-risking the regulatory aspects of the program.

What was resolved in the Type C meeting with the FDA?

The Type C meeting resolved all previously outstanding CMC questions regarding the Phase 3b trial for NurOwn.

What is BrainStorm Cell Therapeutics’ ultimate goal with NurOwn?

BrainStorm aims to achieve FDA approval for NurOwn to address the unmet needs of ALS patients.

Brainstorm Cell Therapeutics Inc.

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