NEW YORK, June 16, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced new survival data from 10 participants in its Expanded Access Program (EAP) for NurOwn® (autologous MSC-NTF cells) in amyotrophic lateral sclerosis (ALS). The EAP enrolled 10 participants who had previously completed the Phase 3 clinical trial.The analysis reviewed survival from the time of first symptom onset through parti ...

Brainstorm Cell Therapeutics (BCLI) Q1 2025 Earnings Call May 19, 2025 08:30 AM ET Company Participants Joyce Lonergan - Managing DirectorChaim Lebovits - President & CEOHaro Hartounian - EVP & COOBob Dagher - EVP & Chief Medical OfficerNetta Blondheim-Shraga - SVP - Research & DevelopmentJason Mccarthy - Senior Managing Director & Head of Biotechnology Research Conference Call Participants David Bautz - Senior Analyst Operator Greetings, and welcome to the BrainStorm Cell Therapeutics Therapeutics first qu ...

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 Viewpoint - BrainStorm Cell Therapeutics Inc. has rescheduled the release of its financial results for Q1 2025 and an investor conference call, indicating ongoing engagement with investors and stakeholders [1][2]. Company Overview - BrainStorm Cell Therapeutics Inc. is a leading developer of adult stem cell therapies targeting neurodegenerative diseases, utilizing its proprietary NurOwn® platform to produce neurotrophic factor-secreting cells [5]. - The company's lead investigational therapy, NurOwn®, has received Orphan Drug designation from both the FDA and EMA, and a Phase 3 trial for ALS has been completed [6]. Upcoming Events - The financial results will be released post-market on May 15, 2025, and the investor conference call is scheduled for May 19, 2025, at 8:30 a.m. Eastern Time [1][2]. - Participants are encouraged to submit questions in advance, with specific contact details provided for access to the conference call [3]. Clinical Development - BrainStorm has completed a Phase 2 trial of MSC-NTF cells in progressive multiple sclerosis, supported by a grant from the National MS Society [6]. - The company is advancing an allogeneic exosome-based platform for delivering therapeutic proteins and nucleic acids, with a foundational patent recently allowed by the U.S. Patent and Trademark Office [6].

Company Overview - BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of adult stem cell therapeutics aimed at treating neurodegenerative diseases [3][4] - The company's proprietary NurOwn® platform utilizes autologous mesenchymal stem cells (MSCs) to create neurotrophic factor-secreting cells (MSC-NTF cells) [3][4] Upcoming Conference Call - A conference call is scheduled for May 15, 2025, at 8:30 a.m. Eastern Time to discuss the financial results for Q1 2025 and provide a corporate update [1][2] - The call will feature BrainStorm's President & CEO Chaim Lebovits, along with Dr. Bob Dagher, Chief Medical Officer, and Alla Patlis, Interim CFO [2] Clinical Development - NurOwn® is the lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the FDA and EMA [4] - A Phase 3 trial for ALS has been completed, and a confirmatory Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA [4] - The clinical program has provided insights into ALS disease biology, including pharmacogenomic responses and biomarker data [4] Additional Research and Development - BrainStorm has completed a Phase 2 open-label multicenter trial of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society [4] - The company is also advancing an allogeneic exosome-based platform for delivering therapeutic proteins and nucleic acids, with a foundational patent recently allowed by the U.S. Patent and Trademark Office [4]

Core Insights - New pharmacogenomic data on the impact of the UNC13A genotype on treatment response in ALS patients treated with NurOwn® will be presented at the ISCT 2025 Annual Meeting [1][2] - The findings are significant as they may help predict treatment responses based on genetic factors, advancing personalized medicine for ALS [2][3] Company Overview - BrainStorm Cell Therapeutics Inc. is a leading developer of autologous adult stem cell therapies for neurodegenerative diseases, with its proprietary NurOwn® platform utilizing mesenchymal stem cells to produce neurotrophic factor-secreting cells [4][5] - NurOwn® has received Orphan Drug designation from both the FDA and EMA, and a Phase 3 trial in ALS has been completed, with a confirmatory Phase 3b trial set to launch under a Special Protocol Assessment agreement with the FDA [5][6] Clinical Development - The upcoming Phase 3b trial aims to build on existing clinical data and demonstrate NurOwn's potential in early ALS, with positive communication ongoing with the FDA [3][5] - The recent findings provide insights into treatment effects in patient subpopulations and represent a first-of-its-kind pharmacogenomic analysis in ALS, focusing on how genetic differences affect therapy response [3][6] Presentation Details - The oral presentation titled "Debamestrocel Effect on Clinical and Biomarker Endpoints by UNC13A Genotype in Phase 3 ALS Trial" will take place on May 8, 2025, at the ISCT meeting in New Orleans [3]

Core Insights - BrainStorm Cell Therapeutics Inc. reported financial results for the fiscal year ended December 31, 2024, highlighting resilience and progress in developing therapies for neurodegenerative diseases, particularly ALS [2][7]. Financial Performance - Cash, cash equivalents, and short-term bank deposits were approximately $0.4 million as of December 31, 2024, down from $1.5 million as of December 31, 2023 [10]. - Research and development expenditures for the year were $4.7 million, a decrease from $10.7 million in 2023 [10][17]. - General and administrative expenses were approximately $7.0 million for 2024, compared to $10.7 million in 2023 [10][17]. - The net loss for the year was approximately $11.6 million, an improvement from a net loss of $17.2 million in 2023 [10][17]. - Net loss per share for 2024 was $2.31, compared to $6.00 in 2023 [10][17]. Corporate Developments - The company is preparing for a Phase 3b trial of NurOwn in ALS, with an agreement on a Special Protocol Assessment (SPA) from the FDA, which reduces regulatory risks [2]. - A new patent covering exosome platform technology was allowed by the U.S. Patent and Trademark Office, enhancing the company's intellectual property portfolio [5]. - A warrant inducement agreement is expected to raise approximately $1.64 million, closing around April 1, 2025 [6][10]. Clinical and Regulatory Updates - NurOwn technology, which utilizes autologous MSC-NTF cells, aims to target pathways in neurodegenerative disorders and has received Orphan Drug designation from the FDA and EMA for ALS treatment [12][13]. - The company has completed a Phase 3 pivotal trial in ALS and is actively working on the regulatory approval process for NurOwn [13].