BrainStorm Advances NurOwn Toward Phase 3b ENDURANCE Trial in ALS Under First-Ever SPA for an ALS Therapeutic
核心洞察
BrainStorm Cell Therapeutics is completing preparations to launch the Phase 3b ENDURANCE trial of NurOwn (MSC-NTF) in ALS, conducted under an FDA Special Protocol Assessment.
The ENDURANCE trial plans to enroll about 200 ALS patients across a randomized, double-blind, placebo-controlled Part A and an open-label extension Part B, with ALSFRS-R change as the primary endpoint.
BrainStorm appointed former FDA Associate Commissioner Peter J. Pitts as Executive Chairman to lead regulatory, operational, partnership, and capital-markets initiatives for the NurOwn program.
BrainStorm Cell Therapeutics Inc. (OTCQB: BCLI), a developer of adult stem cell therapeutics for neurodegenerative diseases, announced on August 14, 2026 that it is completing the operational and regulatory work needed to open enrollment for the Phase 3b ENDURANCE trial of NurOwn in amyotrophic lateral sclerosis (搜索) (ALS). The trial will be conducted under a Special Protocol Assessment (SPA) with the U.S. Food and Drug Administration (FDA), which the company describes as the first ever granted for an ALS therapeutic candidate.
"We continue to advance and complete the preparations necessary to initiate the Phase 3b ENDURANCE study of NurOwn in ALS, supported by an FDA-agreed protocol under a Special Protocol Assessment," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. "ALS imposes an enormous burden on patients, families and caregivers, and we remain resolute in our commitment to the ALS community and to advancing what we believe will be a valuable new therapeutic option."
ENDURANCE Trial Design
The Phase 3b ENDURANCE trial is planned to enroll about 200 patients with ALS in two 24-week parts. Part A is a randomized, double-blind, placebo-controlled portion, followed by Part B, an open-label extension in which all participants receive NurOwn. The primary endpoint is change on the ALSFRS-R scale, and Part A results are intended to support a new Biologics License Application (BLA). Additional details are available at ClinicalTrials.gov under identifier NCT06973629.
NurOwn Platform and Clinical Background
NurOwn is BrainStorm's lead investigational therapy for ALS and has received Orphan Drug designation from both the FDA and the European Medicines Agency (EMA). The NurOwn technology platform (autologous MSC-NTF cells) uses autologous, bone marrow-derived mesenchymal stem cells (MSCs) that are 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 (搜索) (NTFs). These autologous MSC-NTF cells are designed to deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to slow or stabilize disease progression.
A Phase 3 trial in ALS (NCT03280056) has been completed. The NurOwn clinical program has generated insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A (搜索) 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. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (搜索) (MS), supported by a grant from the National MS Society.
Leadership and Regulatory Strategy
In July 2026, BrainStorm named Peter J. Pitts, a former FDA Associate Commissioner and co-founder of the Center for Medicine in the Public Interest, as Executive Chairman and Chief Strategic Regulatory and Policy Officer. Mr. Pitts, who joined the Board in May 2026, will lead the company's daily operations, partnerships, investor engagement, and regulatory strategy, focused primarily on advancing the Phase 3b ENDURANCE trial. Professor Jacob Frenkel, in BrainStorm's leadership since 2007 and Board Chairman since 2020, will transition to Senior Advisor.
"The recent appointment of Peter Pitts as Executive Chairman represents an important enhancement to our leadership," said Lebovits. "His experience as a former senior FDA official brings an additional level of regulatory insight and strategic perspective as we prepare to enter this next phase."
In June 2026, BrainStorm announced a peer-reviewed article co-authored by Peter J. Pitts and CEO Chaim Lebovits in the Journal of the Academy of Public Health, titled "Restoring Regulatory Fairness and Reclaiming Biomedical Leadership: ALS, Rare Disease Regulation, and the Future of Regenerative Medicine." The authors argue that regulation should keep pace with advances in biomarkers, genomics, artificial intelligence, and precision medicine, and that tools such as accelerated approval, adaptive trial design, and Bayesian statistics can improve therapy development for ALS and other rare diseases. The article highlights the company's Special Protocol Assessment as a model of sponsor and regulator collaboration.
Financial Results
For the second quarter ended June 30, 2026, BrainStorm reported cash, cash equivalents, and restricted cash of approximately $0.2 million, compared to approximately $1.03 million as of June 30, 2025. Research and development expenditures, net, were approximately $1 million for the second quarter of 2026, compared to approximately $1.1 million for the second quarter of 2025. General and administrative expenses were approximately $2.7 million, compared to approximately $1.5 million in the prior-year period.
Net loss for the second quarter of 2026 was approximately $3.9 million, compared to a net loss of approximately $2.9 million for the second quarter of 2025. Net loss per share was $(0.35) for the second quarter of 2026, compared to $(0.34) for the second quarter of 2025.
In May 2026, BrainStorm entered into private placement agreements for gross proceeds of $400,000, priced at a premium to market. Together with the $2.0 million raised in February 2026, capital raised in first-half 2026 totals approximately $2.4 million.
Broader Pipeline
Beyond ALS, BrainStorm is 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 its intellectual property portfolio in regenerative medicine.