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- BrainStorm Cell Therapeutics has named former FDA Associate Commissioner Peter J. Pitts as Executive Chairman and Chief Strategic Regulatory and Policy Officer to drive the upcoming Phase 3b trial for NurOwn. - NurOwn is advancing under the first FDA Special Protocol Assessment (SPA) ever granted for an amyotrophic lateral sclerosis (ALS) therapeutic candidate, providing a clear regulatory blueprint. - Longtime Board Chairman Professor Jacob Frenkel will transition to Senior Advisor after nearly two decades of leadership, marking a strategic succession aligned with the company's late-stage clinical execution phase. - The leadership expansion aims to strengthen corporate partnerships, investor engagements, and regulatory strategy as the company moves toward potential marketing approval for its autologous stem cell therapy.
- 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. - The company reported cash of approximately $0.2 million as of June 30, 2026, and a net loss of approximately $3.9 million for the second quarter of 2026.
- A 309-page Citizens' Petition has been filed with the FDA seeking accelerated approval of NurOwn, a mesenchymal stem cell therapy for ALS, based on unprecedented survival and respiratory data from trial participants. - The petition presents compelling real-world evidence including 100% five-year survival in the Expanded Access Program versus 20% natural history (p-value ~0.000335), and median 7-year tracheostomy-free survival compared to 30 months in natural history. - Neurologist Dr. Danielle Geraldi-Samara documented clinical improvements in multiple NurOwn recipients, including patients who regained motor function and maintained respiratory independence years beyond typical ALS progression timelines. - The petition argues that the FDA's focus on primary endpoints overlooked the totality of evidence, including patient experiences and expert clinical observations that demonstrate NurOwn's efficacy in slowing disease progression.
- A coalition of ALS patients and families has filed a 309-page Citizens' Petition with the FDA requesting approval of NurOwn, a neurotrophically-enhanced stem cell therapy developed by BrainStorm Cell Therapeutics. - The petition presents unprecedented survival data showing 100% five-year survival in NurOwn's Expanded Access Program participants compared to 20% in natural ALS history, with median tracheostomy-free survival of 7 years. - NurOwn demonstrated statistically significant changes in 23 cerebrospinal fluid biomarkers and up to 85% slowing of ALS progression, supported by real-world evidence from clinical trials and expanded access programs. - The petition argues NurOwn meets approval thresholds for traditional, accelerated, or conditional approval pathways based on survival data, respiratory function preservation, and biomarker evidence.
- Brainstorm Cell Therapeutics has received FDA clearance to initiate a Phase 3b trial of NurOwn for the treatment of Amyotrophic Lateral Sclerosis (ALS), marking a significant advancement in their clinical development program. - The company's proprietary NurOwn technology utilizes Mesenchymal Stem Cells (MSC) that are differentiated into neurotrophic factor-secreting cells and transplanted near damaged areas, offering a novel approach to treating neurodegenerative diseases. - Following the FDA announcement, Brainstorm's stock surged by 31.82% in premarket trading, reflecting strong investor confidence in the potential of this stem cell therapy for ALS patients.
- BrainStorm Cell Therapeutics has submitted an IND amendment to the FDA for NurOwn, its autologous mesenchymal stem cell therapy for ALS, setting the stage for a pivotal Phase 3b clinical trial. - The trial design received FDA validation through a Special Protocol Assessment agreement, confirming its potential to support a future Biologics License Application if successful. - The Phase 3b study will enroll approximately 200 ALS patients in a two-part design, with the primary endpoint measuring changes in ALSFRS-R scores from baseline to week 24.
- The neuroregeneration therapy market is projected to grow at a 5.3% CAGR from 2024 to 2034, with North America leading the expansion due to advanced healthcare infrastructure and strong research funding. - Recent breakthroughs include Biogen's promising gene therapy trials for Parkinson's disease and BrainStorm Cell Therapeutics' regulatory approval for stem cell-based ALS treatment. - Artificial intelligence, stem cell research, and gene therapy applications are reshaping the therapeutic landscape, with major pharmaceutical companies forming strategic partnerships to accelerate innovation.
- The market for gene and cell therapies targeting CNS disorders was valued at approximately USD 1 billion in 2021 and is projected to grow significantly by 2034. - Several companies are developing gene and cell therapies for CNS disorders, with some therapies already approved for conditions like spinal muscular atrophy (SMA). - Clinical trials are showing positive trends, such as uniQure's AMT-130 for Huntington's disease demonstrating encouraging results in mean CSF NfL levels.
- BrainStorm Cell Therapeutics presented data from its NurOwn Expanded Access Program (EAP) at the 2024 NEALS meeting, showing potential long-term survival benefits for ALS patients. - The EAP data revealed a statistically significant survival difference favoring debamestrocel, with a median survival time of 46.6 months compared to 41.1 months in the matched control group. - Biomarker analysis showed continual reduction in neurofilament light (NfL) levels among early-start participants, indicating reduced neurodegeneration with NurOwn treatment. - BrainStorm is advancing NurOwn through a Phase 3b trial, aiming to confirm its efficacy in early-stage ALS patients, with a Special Protocol Assessment agreement with the FDA.
- BrainStorm Cell Therapeutics will provide an update on its Phase 3b clinical trial for NurOwn, an investigational cell therapy for amyotrophic lateral sclerosis (ALS). - The update will be presented by CEO Chaim Lebovits at the Maxim Healthcare Virtual Summit on October 15, 2024. - NurOwn is an autologous MSC-NTF cell therapy targeting key disease pathways in neurodegenerative disorders like ALS. - BrainStorm's NurOwn program has provided insights into ALS pathology, disease progression, and potential treatment strategies through rigorous peer-reviewed analyses.
