BrainStorm's NurOwn® Data Selected as Breakthrough Science for Presentation at ISCT 2025 Meeting

Rhea-AI Summary

BrainStorm Cell Therapeutics announced that new pharmacogenomic data for their NurOwn® treatment will be presented at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting in New Orleans.

The breakthrough findings focus on how the UNC13A genotype affects treatment response in ALS patients. This represents a first-of-its-kind pharmacogenomic analysis from the Phase 3 trial, exploring genetic factors that may predict treatment outcomes.

Key developments include:

• Preparation for launching a Phase 3b trial of NurOwn for early ALS treatment
• Positive FDA communication and IND amendment submission
• Implementation of SPA as agreed with the Agency

The presentation, scheduled for May 8, 2025, will detail debamestrocel's effect on clinical and biomarker endpoints by UNC13A genotype in the Phase 3 ALS trial.

Positive

• NurOwn data selected as breakthrough science for ISCT 2025 presentation, indicating scientific recognition
• FDA communication is positive with submitted IND amendment
• Company is ready to launch Phase 3b trial for early ALS treatment
• Novel pharmacogenomic analysis from Phase 3 trial could help identify patient populations most likely to respond to treatment

Negative

• No immediate revenue generation potential mentioned
• Additional clinical trials required before potential commercialization
• Still in development phase without clear timeline to market

Insights

Biotech Analyst

BrainStorm's UNC13A genotype data adds precision medicine approach to NurOwn ALS program as they advance toward Phase 3b trial with FDA alignment.

BrainStorm's announcement represents meaningful progress for their NurOwn (debamestrocel) program in ALS. The acceptance of their pharmacogenomic data for oral presentation at ISCT 2025 demonstrates scientific recognition in a competitive selection process. This analysis connecting the UNC13A genotype to treatment response could help define responder populations—crucial for regulatory considerations.

The company has submitted an IND amendment and is implementing a Special Protocol Assessment (SPA) for their planned Phase 3b trial in early ALS, indicating alignment with FDA on trial design. This regulatory clarity reduces development risk significantly.

The pharmacogenomic approach is particularly noteworthy as it represents one of the first analyses examining genetic influence on treatment response in ALS. By potentially identifying genetic markers of response, BrainStorm could optimize patient selection for their therapy, addressing a common challenge in neurodegenerative disease trials where patient heterogeneity often complicates efficacy signals.

The focus on early ALS patients for the upcoming trial suggests a strategic refinement of their therapeutic approach based on insights from their completed Phase 3 study. This development pathway shows a methodical approach to addressing the complex challenges of ALS drug development.

Neurological Disease Specialist

The pharmacogenomic findings linking UNC13A genotype to NurOwn response marks an important advancement in personalized medicine for ALS. The UNC13A gene has been previously established as a genetic risk factor in ALS, with certain variants associated with more rapid disease progression and poorer outcomes.

This genotype-treatment response correlation could significantly impact the ALS treatment landscape. By identifying genetic predictors of response, clinicians could potentially direct treatments to patients most likely to benefit, improving both trial outcomes and eventual clinical use efficiency.

The oral presentation selection at ISCT indicates peer recognition of the significance of these findings. As one of the first ALS studies to analyze genetic differences in treatment response, this represents a pioneer approach in a field that has long struggled with therapeutic development.

From a scientific perspective, cellular therapies like NurOwn face unique challenges in neurodegenerative diseases, particularly regarding delivery, persistence, and mechanism of action. Genetic biomarkers could help overcome these hurdles by providing more homogeneous patient populations and clearer efficacy signals.

The planned Phase 3b focus on early ALS aligns with emerging consensus that intervention before significant neuronal loss occurs may be crucial for meaningful clinical benefit—a hypothesis that will be tested in their upcoming trial.

New findings highlight impact of UNC13A genotype on treatment response in ALS

NEW YORK, April 29, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, in collaboration with leading ALS research centers, today announced the acceptance of new pharmacogenomic data for oral presentation of new findings on NurOwn^® at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting, to take place from May 6 - 10, 2025 in New Orleans. The data highlight the impact of the UNC13A genotype on clinical outcomes for amyotrophic lateral sclerosis (ALS) patients treated with NurOwn^® (debamestrocel).

The presentation was featured in the ISCT public announcement, which indicated that the data were "carefully reviewed and selected by the ISCT 2025 Planning Faculty, to explore the latest breakthroughs in the clinical translation of Mesenchymal Stem/Stromal Cells and how they will shape the future of cell therapies."

"We look forward to sharing the latest scientific data on NurOwn at this year's ISCT meeting. These new results are particularly meaningful as they explore a genetic factor that may help predict treatment response. The feedback received from the ALS experts on these new data continues to be encouraging as we advance in our mission to address the unmet needs of patients with this devastating disease," said Chaim Lebovits, President and CEO of BrainStorm.

Mr. Lebovits added, "Our immediate priority is to launch the Phase 3b trial of NurOwn to build on our clinical data and demonstrate its potential in early ALS. We are enjoying positive communication with the FDA, have submitted an IND amendment, and are implementing the SPA exactly as agreed with the Agency. We're fully prepared to begin the study."

Dr. Bob Dagher, Executive Vice President & Chief Medical Officer at BrainStorm, commented, "These latest results offer additional scientific insights into our completed Phase 3 NurOwn trial and increase our understanding of treatment effects in patient subpopulations. These types of analysis provide important information for the development of future ALS treatments and add to the growing body of evidence supporting NurOwn^®." Dr. Dagher added, "This represents a first-of-its-kind pharmacogenomic analysis from the Phase 3 trial in ALS and is among the first ALS studies to analyze how genetic differences, specifically the UNC13A genotype, affect patient response to therapy. Understanding how genetics can influence treatment response will help move us closer to truly personalized medicine for ALS."

Presentation details:

Title:                            Debamestrocel Effect on Clinical and Biomarker Endpoints by UNC13A Genotype in Phase 3 ALS Trial
Lead Author                Bob Dagher, MD
Oral Session:              Mesenchymal Stem/Stromal Cells #1
Date and time:            Date & Time: Thursday, May 8, 2025 | 9:00 – 10:00 a.m. U.S. Central Time.
Location:                     Ernest N. Morial Convention Center, 900 Convention Center Boulevard, New Orleans, Louisiana 70130

A copy of the poster will be available on the BrainStorm corporate website, at the conclusion of the ISCT meeting.

About Brainstorm Cell Therapeutics Inc.    

BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection.           
NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a confirmatory Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA.     
The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A genotype, biomarker data collected at seven longitudinal time points, and a comprehensive analysis of the "Floor Effect" — a critical challenge in measuring clinical outcomes in advanced ALS. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also advancing a proprietary, allogeneic exosome-based platform designed to deliver therapeutic proteins and nucleic acids. The company recently received a Notice of Allowance from the U.S. Patent and Trademark Office for a foundational patent covering its exosome technology, further strengthening BrainStorm's growing IP portfolio in this emerging area of regenerative medicine.

To learn more, visit www.brainstorm-cell.com.

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.

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

Logo: https://mma.prnewswire.com/media/1166536/BrainStorm_Logo.jpg

 

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SOURCE BrainStorm Cell Therapeutics Inc.

FAQ

What new NurOwn data will BrainStorm (BCLI) present at ISCT 2025?

BrainStorm will present new pharmacogenomic data showing how UNC13A genotype affects treatment response in ALS patients treated with NurOwn (debamestrocel). The presentation is scheduled for May 8, 2025, at the ISCT Annual Meeting in New Orleans.

When is BrainStorm's (BCLI) Phase 3b NurOwn trial starting?

BrainStorm has submitted an IND amendment and is implementing the SPA as agreed with the FDA. The company states they are fully prepared to begin the Phase 3b trial of NurOwn in early ALS patients, though the exact start date is not specified in the release.

How does UNC13A genotype impact NurOwn treatment for ALS patients?

The specific impact of UNC13A genotype on NurOwn treatment response will be revealed at the ISCT 2025 meeting. This represents the first pharmacogenomic analysis from NurOwn's Phase 3 trial examining genetic influences on treatment outcomes.

Where and when will BrainStorm present the new NurOwn data in 2025?

The presentation will take place on Thursday, May 8, 2025, from 9:00-10:00 a.m. Central Time at the Ernest N. Morial Convention Center in New Orleans, during the ISCT 2025 Annual Meeting.

What makes BrainStorm's NurOwn presentation significant at ISCT 2025?

The presentation was selected as breakthrough science by ISCT 2025 Planning Faculty, focusing on how genetic factors may predict treatment response in ALS patients. It's among the first ALS studies analyzing UNC13A genotype's effect on therapy response.