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].

BrainStorm's participation in this year's summit marks a critical juncture for the company as it prepares to launch its Phase 3b clinical trial of NurOwn, supported by a Special Protocol Assessment (SPA) from the U.S. Food and Drug Administration (FDA), which affirms that the trial's design and statistical analysis are adequate to support a future marketing application if successful. About Brainstorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous ...