Core Viewpoint - BrainStorm Cell Therapeutics Inc. is making progress in stabilizing its financial situation and advancing the Phase 3b study of its investigational therapy NurOwn for ALS, with FDA clearance obtained for the clinical study [2][8]. Financial Results - As of September 30, 2025, the company reported cash, cash equivalents, and restricted cash of approximately $0.23 million [7]. - Research and development expenditures for Q3 2025 were $0.9 million, a decrease from $1 million in Q3 2024 [7]. - General and administrative expenses for Q3 2025 were approximately $1.1 million, down from approximately $2.0 million in Q3 2024 [7]. - The net loss for Q3 2025 was approximately $2.1 million, compared to a net loss of approximately $2.7 million in Q3 2024 [7]. NurOwn Therapy Development - NurOwn is designed to deliver neurotrophic factors and immunomodulatory cytokines to slow disease progression in ALS [6][8]. - The upcoming Phase 3b ENDURANCE study will enroll about 200 participants and will include a 24-week randomized, double-blind, placebo-controlled period followed by a 24-week open-label extension [7]. - The primary efficacy measure will be the change from baseline to Week 24 on the ALSFRS-R scale [7]. Regulatory Engagement - The company is actively engaging with regulatory authorities and has received FDA clearance for the planned clinical study [2][8]. - A Citizen Petition was filed with the FDA by ALS community representatives, requesting a renewed review of NurOwn's data, which the company views as a constructive development [7]. Future Communications - BrainStorm plans to host a conference call and webcast for the investment community later in Q4 2025 or early Q1 2026 to provide further updates on the NurOwn Phase 3 program [4].

Financial Data and Key Metrics Changes - The company reported significant progress in preparing for the Phase 3b trial of NurOwn, with a focus on securing funding to support trial initiation [24][25] - Financial constraints have been acknowledged, but the company has made strides in regulatory submissions and site selections despite limited resources [13][24] Business Line Data and Key Metrics Changes - The primary focus remains on the clinical development of NurOwn, with the initiation of a pivotal Phase 3b trial aimed at ALS patients [6][10] - The trial design has been agreed upon with the FDA, which is expected to de-risk the regulatory pathway for NurOwn [6][7] Market Data and Key Metrics Changes - The company is actively negotiating with approximately 15 leading clinical centers across the United States for the Phase 3b trial [10] - The trial, named "Endurance," aims to resonate with the ALS community and reflects the company's commitment to developing a therapeutic option for ALS [26] Company Strategy and Development Direction - The company is focused on executing the clinical development plan for NurOwn and is preparing for a significant trial that could lead to regulatory approval [6][12] - There is an emphasis on expanding manufacturing capabilities in the U.S. to support future commercialization and supply chain security [28] Management's Comments on Operating Environment and Future Outlook - Management highlighted the urgency felt by patients and clinicians for innovative therapeutic options in ALS, reinforcing their commitment to executing the trial with scientific rigor [11][12] - The company is pursuing multiple funding avenues, including a promising $15 million non-dilutive grant, to ensure timely trial commencement [25][14] Other Important Information - The company participated in the annual ALF Drug Development Summit, discussing critical areas in ALS therapeutic development [12] - The scientific team is engaged with the academic community to share insights and data, which is crucial for advancing the NurOwn program [12] Q&A Session Summary Question: Can you start the trial without proper funding? - Management emphasized that while significant progress has been made, securing proper funding is essential to commence the trial [24][25] Question: What is the meaning of the trial name "Endurance"? - The name reflects the strength and resilience of the ALS community and the company's commitment to delivering a therapeutic option [26] Question: Will the company also be producing in the U.S.? - The company confirmed plans to expand manufacturing in the U.S. and will announce a letter of intent with a U.S.-based facility soon [28] Question: Can you update on any advances in the exosome program? - The company is encouraged by the progress of its exosome program and is preparing a manuscript detailing its efficacy in preclinical models [32]

Financial Data and Key Metrics Changes - The company is focused on executing the clinical development plan for NurOwn and has received FDA clearance to initiate a pivotal Phase 3b trial for ALS [6][10] - Financial constraints have been acknowledged, but the company has made significant strides in trial preparations despite limited resources [13][25] Business Line Data and Key Metrics Changes - The NurOwn technology platform is central to the company's strategy, with ongoing clinical trials aimed at demonstrating its therapeutic benefits [6][12] - The company is actively negotiating clinical trial agreements with approximately 15 leading clinical centers across the U.S. for the Phase 3b trial [10][25] Market Data and Key Metrics Changes - The ALS community's strong interest in NurOwn is highlighted by the engagement of renowned ALS clinicians and researchers [10] - The company participated in the annual ALF Drug Development Summit, indicating its active involvement in the ALS therapeutic development landscape [12] Company Strategy and Development Direction - The company aims to secure strategic funding to support the trial, with a focus on partnerships and non-dilutive grants [14][26] - Expansion of manufacturing capabilities in the U.S. is a key strategic objective, with plans to announce a letter of intent with a U.S.-based facility [30] Management's Comments on Operating Environment and Future Outlook - Management emphasized the urgency for innovative therapeutic options in ALS and the commitment to executing the trial with scientific rigor [11][12] - The company is optimistic about the potential of NurOwn and is preparing for various funding opportunities to ensure timely trial commencement [14][26] Other Important Information - The trial, named "Endurance," is designed to resonate with the ALS community, reflecting the strength and resilience of ALS patients [27] - The company is encouraged by progress in its exosome program, which shows potential in treating respiratory and inflammatory diseases [34] Q&A Session Summary Question: Can you start the trial without proper funding? - Management stated that while significant progress has been made, proper funding is essential to commence the trial [25][26] Question: What is the meaning of the trial name "Endurance"? - The name was chosen to resonate with the ALS community and reflects the company's commitment to delivering a therapeutic option for ALS [27] Question: Will the company also be producing in the U.S.? - The company confirmed plans to expand its manufacturing footprint in the U.S. and will announce a letter of intent with a U.S.-based facility [30] Question: Can you update on any advances in the exosome program? - Management expressed encouragement regarding the exosome program's progress and is preparing to advance it towards clinical development [34] Question: Have you had any communications with FDA regarding stratification by UNC13A? - Management indicated that while the FDA has not approved any biomarker as a surrogate, they are excited about the genetic discoveries and will explore them further [39][40] Question: Can the mechanism of action be used as part of the data package for the next BLA? - Management confirmed that the protective effect observed in cell cultures could support the data package for future filings [43] Question: How many clinical trial sites are you looking to open? - Management confirmed plans to open approximately 15 clinical trial sites and is finalizing agreements to begin patient enrollment [48][50]

Core Viewpoint - BrainStorm Cell Therapeutics Inc. is making significant progress in the development of its NurOwn® therapy for ALS, with plans for a Phase 3b trial aimed at confirming efficacy in early-stage ALS patients and supporting a new Biologics License Application (BLA) [2][4]. Financial Results - As of March 31, 2025, the company reported cash, cash equivalents, and restricted cash of approximately $1.8 million [4]. - Research and development expenditures for Q1 2025 were $1.3 million, an increase from $1.0 million in Q1 2024 [4]. - General and administrative expenses for Q1 2025 were approximately $1.8 million, compared to $1.5 million in Q1 2024 [4]. - The net loss for Q1 2025 was approximately $2.9 million, an improvement from a net loss of approximately $3.4 million in Q1 2024 [4][14]. - Net loss per share for Q1 2025 was $0.45, compared to $0.75 for Q1 2024 [4][14]. Corporate Updates - The company submitted an IND amendment for NurOwn to the FDA, which is a critical step for the upcoming Phase 3b trial [4]. - The Phase 3b trial, named ENDURANCE, is designed to enroll approximately 200 early-stage ALS participants and will assess changes in ALSFRS-R scores [4]. - NurOwn data was selected for presentation at the ISCT 2025 Meeting, highlighting the impact of the UNC13A genotype on clinical outcomes for ALS patients [4]. - Insights on biomarkers related to NurOwn treatment were presented at the 2025 ALS Drug Development Summit [4]. About NurOwn® - NurOwn® is an investigational therapy utilizing autologous MSC-NTF cells, which are derived from bone marrow and designed to deliver neurotrophic factors to slow or stabilize neurodegenerative diseases [6][8]. - The therapy has received Orphan Drug designation from both the FDA and EMA, and a Phase 3 trial has been completed [8]. Company Overview - BrainStorm Cell Therapeutics Inc. focuses on developing autologous adult stem cell therapies for neurodegenerative diseases, with its proprietary NurOwn® platform aimed at modulating neuroinflammation and promoting neuroprotection [7].

Core Insights - BrainStorm Cell Therapeutics Inc. is participating in the 4th Annual ALS Drug Development Summit, showcasing its expertise in ALS clinical research and the NurOwn® development program [1][5] Company Developments - Netta Blondheim-Shraga, PhD, will present new insights into cerebrospinal fluid biomarker pathways related to Debamestrocel (NurOwn), focusing on their connection to clinical outcomes and disease heterogeneity in ALS [2] - The company is preparing to launch its Phase 3b clinical trial of NurOwn, which has received a Special Protocol Assessment (SPA) from the FDA, indicating that the trial's design is adequate for future marketing application if successful [5] - Dr. Bob Dagher will moderate a workshop on adaptive and decentralized clinical trial models aimed at improving enrollment and reducing the burden on ALS patients [3][4] Research and Development - Biomarker data from a prior Phase 3 study suggests a multimodal mechanism of action for NurOwn, enhancing understanding of its influence on ALS disease pathways [3] - The upcoming Phase 3b trial will refine patient selection criteria and trial design based on learnings from previous studies [4] - The NurOwn clinical program has provided valuable insights into ALS biology, including pharmacogenomic responses and biomarker data collected over time [6] Industry Context - A panel discussion will address the role of patient and caregiver engagement in clinical trials and the impact of the diagnostic journey on trial decision-making [4] - The company is advancing a proprietary exosome-based platform for therapeutic delivery, further strengthening its intellectual property portfolio in regenerative medicine [8]