A Two-Part Phase 3b Randomized, Double-Blind, Placebo-Controlled, Followed by Open-Label Extension, Multicenter Study of the Efficacy and Safety of MSC-NTF (NurOwn) in Participants With With Early Symptomatic and Moderate Disease Presentation in Amyotrophic Lateral Sclerosis (ALS)

The goal of this two-part clinical trial is:
1) to evaluate the safety and efficacy of Debamestrocel - MSC-NTF (NurOwn) compared to placebo in participants with early symptomatic ALS and moderate disease presentation in ALS; followed by 2) further evaluation by providing NurOwn to all participants in an open label extension period.
Researchers will compare NurOwn to a placebo (a look-alike substance that contains no drug) to evaluate the efficacy of NurOwn compared to placebo in the treatment of participants with ALS.
Participants will:
Receive NurOwn or a placebo every 8 weeks for 24 months. After that, every participant will receive NurOwn every 8 weeks for an additional 24 months.
They will visit the clinic approximately every 8 weeks for checkups and tests.

Start Date30 Jun 2025

Sponsor / Collaborator

Brainstorm Cell Therapeutics, Inc.

EUCTR2020-002872-11-NL

/ Unknown statusPhase 2

A Phase 2a, open-label, multicenter study to evaluate the safety and tolerability of repeated administration of NurOwn® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors; MSC-NTF cells) in participants with prodromal to mild Alzheimer’s Disease

Start Date26 Aug 2020

Sponsor / Collaborator

Brainstorm Cell Therapeutics, Inc.

NCT03799718

/ CompletedPhase 2

A Phase 2 Open-label Multicenter Study to Evaluate the Safety and Efficacy of Repeated Administration of NurOwn® [Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors (NTF), MSC-NTF] Cells in Participants With Progressive MS

A multidose open-label study with autologous Mesenchymal Stromal Stem Cells Secreting Neurotrophic Factors (MSC-NTF cells) involving 20 participants with progressive MS at multiple investigational study sites.

Start Date13 Mar 2019

Sponsor / Collaborator

Brainstorm Cell Therapeutics, Inc.

100 Clinical Results associated with Debamestrocel

100 Translational Medicine associated with Debamestrocel

100 Patents (Medical) associated with Debamestrocel

Literatures (Medical) associated with Debamestrocel

01 Jun 2024·Muscle & nerve

Debamestrocel multimodal effects on biomarker pathways in amyotrophic lateral sclerosis are linked to clinical outcomes

Article

Author: Mozaffar, Tahseen ; Owegi, Margaret Ayo ; Brown, Robert H ; Boulanger, Bruno ; Levy, Yossef S ; Windebank, Anthony J ; Lewis, Richard ; Bowser, Robert ; Gothelf, Yael ; Blondheim-Shraga, Netta R ; Miller, Robert ; Burford, Matthew ; Jenkins, Liberty ; Goyal, Namita A ; Li, Jenny ; Katz, Jonathan ; Kaspi, Haggai ; Nicholson, Katharine ; Cudkowicz, Merit E ; Lindborg, Stacy R ; Abramov, Natalie ; Kern, Ralph ; Berry, James D ; Baloh, Robert H ; Dagher, Bob ; Aricha, Revital ; Staff, Nathan P

Abstract:

Introduction/Aims:

Biomarkers have shown promise in amyotrophic lateral sclerosis (ALS) research, but the quest for reliable biomarkers remains active. This study evaluates the effect of debamestrocel on cerebrospinal fluid (CSF) biomarkers, an exploratory endpoint.

Methods:

A total of 196 participants randomly received debamestrocel or placebo. Seven CSF samples were to be collected from all participants. Forty‐five biomarkers were analyzed in the overall study and by two subgroups characterized by the ALS Functional Rating Scale‐Revised (ALSFRS‐R). A prespecified model was employed to predict clinical outcomes leveraging biomarkers and disease characteristics. Causal inference was used to analyze relationships between neurofilament light chain (NfL) and ALSFRS‐R.

Results:

We observed significant changes with debamestrocel in 64% of the biomarkers studied, spanning pathways implicated in ALS pathology (63% neuroinflammation, 50% neurodegeneration, and 89% neuroprotection). Biomarker changes with debamestrocel show biological activity in trial participants, including those with advanced ALS. CSF biomarkers were predictive of clinical outcomes in debamestrocel‐treated participants (baseline NfL, baseline latency‐associated peptide/transforming growth factor beta1 [LAP/TGFβ1], change galectin‐1, all p < .01), with baseline NfL and LAP/TGFβ1 remaining (p < .05) when disease characteristics (p < .005) were incorporated. Change from baseline to the last measurement showed debamestrocel‐driven reductions in NfL were associated with less decline in ALSFRS‐R. Debamestrocel significantly reduced NfL from baseline compared with placebo (11% vs. 1.6%, p = .037).

Discussion:

Following debamestrocel treatment, many biomarkers showed increases (anti‐inflammatory/neuroprotective) or decreases (inflammatory/neurodegenerative) suggesting a possible treatment effect. Neuroinflammatory and neuroprotective biomarkers were predictive of clinical response, suggesting a potential multimodal mechanism of action. These results offer preliminary insights that need to be confirmed.

01 Jan 2023·Multiple sclerosis (Houndmills, Basingstoke, England)

Evaluation of neurotrophic factor secreting mesenchymal stem cells in progressive multiple sclerosis

Article

Author: Lock, Christoper ; Aricha, Revital ; Mehra, Munish ; Chitnis, Tanuja ; Levy, Yossef ; Lublin, Fred D ; Lindborg, Stacy ; Pelletier, Daniel ; Gothelf, Yael ; Motamed Khorasani, Afsaneh ; Lebovits, Chaim ; Cohen, Jeffrey A ; Kern, Ralph

Background::

Autologous mesenchymal stem cell neurotrophic factor–secreting cells (NurOwn®) have the potential to modify underlying disease mechanisms in progressive multiple sclerosis (PMS).

Objective::

This open-label phase II study was conducted to evaluate safety/efficacy of three intrathecal cell treatments.

Methods::

Eighteen participants with non-relapsing PMS were treated. The primary endpoint was safety. Secondary endpoints included: cerebrospinal fluid (CSF) biomarkers; timed 25-foot walk speed, nine-hole peg test (9-HPT), low-contrast letter acuity, symbol digit modalities test, and 12-item multiple sclerosis (MS) walking scale. Seventeen participants received all treatments.

Results::

No deaths/adverse events related to worsening of MS, clinical/magnetic resonance imaging (MRI) evidence of disease activation, and clinically significant changes in safety lab results were reported. Two participants developed symptoms of low back and leg pain, consistent with a diagnosis of arachnoiditis, occurring in one of three intrathecal treatments in both participants. Nineteen percent of treated participants achieved pre-specified ⩾ 25% improvements in timed 25-foot walk speed/nine-HPT at 28 weeks compared to baseline, along with consistent efficacy signals for pre-specified response criteria across other secondary efficacy outcomes. CSF neuroprotective factors increased, and inflammatory biomarkers decreased after treatment, consistent with the proposed mechanism of action.

Conclusion::

Based on these encouraging preliminary findings, further confirmation in a randomized study is warranted.

01 Jul 2022·Experimental cell researchQ3 · MEDICINE

Various detailed characteristics of a new enhanced neurotrophic factor secreting rat derived bone marrow mesenchymal stem cells and its preliminary application in rat models of ischemic stroke

Q3 · MEDICINE

Article

Author: Cheng, Yanwen ; He, Zi ; Yuan, Chunmei ; Sun, Kaizhou ; Zhou, Hui ; Wang, Peishen ; Jiang, Ningjian ; Yang, Mingbin ; Meng, Peipei ; Han, Xinye ; An, Yihua ; Yang, Qing ; Wang, Lu ; Liu, Yang ; Jiang, Fenjun

Because MSC-NTF has a higher ability to secrete neurotrophic factors, it may have a greater potential than ordinary MSC in clinical applications. At present, research on MSC-NTF mainly focuses on clinical aspects, but its basic research is relatively few. In particular, the research on the comprehensive and detailed characteristics of MSC-NTF is missing. And its in vivo research in animals is also rare. Since the transplantation of human-derived MSC-NTF into rats is cross-species, its survival in the rat and the therapeutic effect may be seriously affected due to severe immune rejection. This will inevitably affect the research on the basic characteristics and the therapeutic mechanisms of MSC-NTF in vivo. Therefore, we chose the rat-derived MSCs to be induced as the MSC-NTF which had a stronger neurotrophic factor secretion function. This will also be helpful to perform the research of the basic therapeutic mechanisms of MSC-NTF in vivo. In addition, we have established some important characteristics that can be used to distinguish between MSC-NTF and MSCs: different multi-factor secretion ability and secretion characteristics, immunogenicity, three-line differentiation ability, stemness, etc. In addition to paying attention to their safety differences, this study also explored the differences in their in vivo survivability. Finally, we applied this newly induced rat-derived MSC-NTF in a rat model of ischemic stroke, and obtained beneficial therapeutic effects.

News (Medical) associated with Debamestrocel

15 May 2025

·www.prnewswire.com

BrainStorm Cell Therapeutics Announces First Quarter 2025 Financial Results and Provides Corporate Update

Conference call and webcast rescheduled for 8:30 a.m. Eastern Time on Monday, May 19 NEW YORK, May 15, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the first quarter ended March 31, 2025, and provided a corporate update. "BrainStorm continues to make meaningful progress in advancing our NurOwn® development program. Our priority continues to be initiation of a Phase 3b trial, designed to confirm the product's efficacy in early stage ALS patients and support a new BLA," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. "We have a clear path forward and ongoing support from the ALS community. We recently submitted an amendment to our IND, which includes updated documentation that is essential for regulatory compliance and trial integrity. We are in the process of finalization clinical trial agreements with leading academic centers and completing other necessary steps on trial execution and manufacturing. We believe that NurOwn, if approved, has the potential to become a valuable treatment option for ALS patients. Our team is fully aligned and executing with discipline to position BrainStorm for success." Recent Highlights NurOwn (MSC-NTF) for ALS IND amendment on NurOwn submitted to FDA BrainStorm submitted an Investigational New Drug (IND) amendment to the U.S. Food and Drug Administration for NurOwn. This important milestone sets the stage for the initiation of the planned Phase 3b clinical trial, which has been designed in collaboration with the FDA under a Special Protocol Assessment (SPA). The trial will have a primary efficacy endpoint assessing changes in ALSFRS-R scores from baseline to week 24, and is designed to enroll approximately 200 ALS participants with early stage disease. Successful completion of the double blind part of the study (Part A) is expected to generate the clinical data to support a new BLA submission. Phase 3b trial listed on Clinicaltrials.gov. Details of the trial, known as ENDURANCE, are now available on ClinicalTrials.gov ID NCT06973629. Included is a list of 15 clinical trial sites that are expected to participate in the trial. NurOwn® data selected as Breakthrough Science for Presentation at ISCT 2025 Meeting The new pharmacogenomic were delivered in a oral presentation at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting, in New Orleans. The data highlight the impact of the UNC13A genotype on clinical outcomes for ALS patients treated with NurOwn. The presentation was featured in the ISCT public announcement regarding the meeting, which indicated that the data were "carefully reviewed and selected by the ISCT 2025 Planning Faculty, to explore the latest breakthroughs in the clinical translation of Mesenchymal Stem/Stromal Cells and how they will shape the future of cell therapies." Biomarker insights supporting NurOwn's mechanism and clinical impact presented at the 2025 ALS Drug Development Summit BrainStorm's senior leadership team (Dr. Bob Dagher, Dr. Netta Blondheim-Shraga and May Kay Turner) participated in sessions at the summit that highlighted insights and expertise gained throughout the NurOwn® development program. These sessions included a presentation on cerebrospinal fluid (CSF) biomarker pathways associated with NurOwn treatment, including their relationship to clinical outcomes and disease heterogeneity in ALS. Financial Results for the First Quarter Ended March 31, 2025 Cash, cash equivalents, and restricted cash were approximately $1.8 million as of March 31, 2025. Research and development expenditures, net, for the quarter ended March 31, 2025 were $1.3 million, compared to $1.0 million for the quarter ended March 31, 2024. General and administrative expenses for the quarter ended March 31, 2025 were approximately $1.8 million, compared to approximately $1.5 million for the quarter ended March 31, 2024. Net loss for the quarter ended March 31, 2025, was approximately $2.9 million, as compared to a net loss of approximately $3.4 million for the quarter ended March 31, 2024. Net loss per share for the three months ended March 31, 2025, and 2024 was $0.45 and $0.75, respectively. Conference Call and Webcast Monday, May 19, 2025, at 8:30 a.m. U.S. Eastern Time Participant Numbers: U.S. dial in: 888-506-0062 International: 973-528-0011 Participant Access Code: 621608 Webcast URL: The replay of the conference call can be accessed by dialing the numbers below and will be available for 14 days. Teleconference Replay Number: Toll Free: 877-481-4010 International: 919-882-2331 Passcode: 52457 About NurOwn® The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been 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 (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. About BrainStorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a second Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A 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. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also 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 BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit . Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at . These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTS Investors: Michael Wood Phone: +1 646-597-6983 [email protected] Media: Uri Yablonka, Chief Business Officer Phone: +1 917-284-2911 [email protected] The accompanying notes are an integral part of the consolidated financial statements. The accompanying notes are an integral part of the consolidated financial statements. Logo: SOURCE BrainStorm Cell Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyPhase 2Phase 3Financial Statement

29 Apr 2025

·www.prnewswire.com

BrainStorm's NurOwn® Data Selected as Breakthrough Science for Presentation at ISCT 2025 Meeting

New findings highlight impact of UNC13A genotype on treatment response in ALS NEW YORK, April 29, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of cellular therapies for neurodegenerative diseases, in collaboration with leading ALS research centers, today announced the acceptance of new pharmacogenomic data for oral presentation of new findings on NurOwn® at the International Society for Cell & Gene Therapy (ISCT) 2025 Annual Meeting, to take place from May 6 - 10, 2025 in New Orleans. The data highlight the impact of the UNC13A genotype on clinical outcomes for amyotrophic lateral sclerosis (ALS) patients treated with NurOwn® (debamestrocel). The presentation was featured in the ISCT public announcement, which indicated that the data were "carefully reviewed and selected by the ISCT 2025 Planning Faculty, to explore the latest breakthroughs in the clinical translation of Mesenchymal Stem/Stromal Cells and how they will shape the future of cell therapies." "We look forward to sharing the latest scientific data on NurOwn at this year's ISCT meeting. These new results are particularly meaningful as they explore a genetic factor that may help predict treatment response. The feedback received from the ALS experts on these new data continues to be encouraging as we advance in our mission to address the unmet needs of patients with this devastating disease," said Chaim Lebovits, President and CEO of BrainStorm. Mr. Lebovits added, "Our immediate priority is to launch the Phase 3b trial of NurOwn to build on our clinical data and demonstrate its potential in early ALS. We are enjoying positive communication with the FDA, have submitted an IND amendment, and are implementing the SPA exactly as agreed with the Agency. We're fully prepared to begin the study." Dr. Bob Dagher, Executive Vice President & Chief Medical Officer at BrainStorm, commented, "These latest results offer additional scientific insights into our completed Phase 3 NurOwn trial and increase our understanding of treatment effects in patient subpopulations. These types of analysis provide important information for the development of future ALS treatments and add to the growing body of evidence supporting NurOwn®." Dr. Dagher added, "This represents a first-of-its-kind pharmacogenomic analysis from the Phase 3 trial in ALS and is among the first ALS studies to analyze how genetic differences, specifically the UNC13A genotype, affect patient response to therapy. Understanding how genetics can influence treatment response will help move us closer to truly personalized medicine for ALS." Presentation details: Title: Debamestrocel Effect on Clinical and Biomarker Endpoints by UNC13A Genotype in Phase 3 ALS Trial Lead Author Bob Dagher, MD Oral Session: Mesenchymal Stem/Stromal Cells #1 Date and time: Date & Time: Thursday, May 8, 2025 | 9:00 – 10:00 a.m. U.S. Central Time. Location: Ernest N. Morial Convention Center, 900 Convention Center Boulevard, New Orleans, Louisiana 70130 A copy of the poster will be available on the BrainStorm corporate website, at the conclusion of the ISCT meeting. About Brainstorm Cell Therapeutics Inc. BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI) is a leading developer of autologous adult stem cell therapies for debilitating neurodegenerative diseases. The company's proprietary NurOwn® platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. NurOwn® is BrainStorm's lead investigational therapy for amyotrophic lateral sclerosis (ALS) and has received Orphan Drug designation from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). A Phase 3 trial in ALS (NCT03280056) has been completed, and a confirmatory Phase 3b trial is set to launch under a Special Protocol Assessment (SPA) agreement with the FDA. The NurOwn clinical program has generated valuable insights into ALS disease biology, including pharmacogenomic response associated with the UNC13A 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. BrainStorm has published its findings in multiple peer-reviewed journals. In addition to ALS, BrainStorm has completed a Phase 2 open-label multicenter trial (NCT03799718) of MSC-NTF cells in progressive multiple sclerosis (MS), supported by a grant from the National MS Society. BrainStorm is also 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 BrainStorm's growing IP portfolio in this emerging area of regenerative medicine. To learn more, visit . Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at . These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACT: Michael Wood Phone: +1 646-597-6983 [email protected] Logo: SOURCE BrainStorm Cell Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyPhase 3Phase 2Orphan Drug

31 Mar 2025

·www.prnewswire.com

BrainStorm Cell Therapeutics Announces Full Year 2024 Financial Results and Provides Corporate Update

Conference call and webcast at 8:30 a.m. Eastern Time today NEW YORK, March 31, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced financial results for the fiscal year ended December 31, 2024, and provided a corporate update. "2024 was a year of resilience and progress for BrainStorm as the company remained focused on its mission to develop and commercialize innovative therapies for neurodegenerative diseases. We had important accomplishments that reinforced our goal to gain regulatory approval of NurOwn in ALS and we continue to work closely with the FDA to achieve this," said Chaim Lebovits, President and Chief Executive Officer of BrainStorm. "We are now in advanced preparations for the Phase 3b trial of NurOwn, to be conducted in approximately 200 participants with ALS. We have secured an agreement with the FDA on a Special Protocol Assessment (SPA), thereby significantly derisking the regulatory aspects of the program. We are also aligned with the FDA on the Chemistry, Manufacturing, and Controls (CMC) aspects of the trial. We believe that if approved, NurOwn has the potential to make a meaningful difference in the lives of ALS patients, and we are fully committed to executing on our clinical and corporate goals to realize that vision." Fourth Quarter 2024 and Recent Highlights December 30, 2024: Issued a letter to shareholders summarizing key corporate achievements and strategic priorities for 2025. NurOwn (MSC-NTF) for ALS December 11, 2024: Hosted a key opinion leader (KOL) webinar featuring Dr. Terry Heiman-Patterson, from the Lewis Katz School of Medicine at Temple University. Dr. Heiman-Patterson discussed the current treatment landscape and advances for patients with ALS (ALS). The replay can be accessed on the corporate website here. November 11, 2024: Announced a strategic partnership with Pluri to support manufacturing for the Phase 3b NurOwn trial, enhancing operational readiness and production capabilities. October 30, 2024: Regained compliance with Nasdaq's minimum bid price requirement, maintaining continued listing on the Nasdaq Capital Market. October 28, 2024: Presented two scientific posters at the 2024 Annual Northeastern Amyotrophic Lateral Sclerosis (NEALS) Meeting. The first poster highlighted findings from the Expanded Access Program (EAP) for NurOwn, showing a statistically significant survival benefit for treated patients. The second poster provided an overview of the planned Phase 3b trial design. October 7, 2024: Participated in the 2024 Maxim Healthcare Virtual Summit to provide a corporate update on the Phase 3b NurOwn trial . Exosome Technology December 3, 2024: Received a Notice of Allowance from the U.S. Patent and Trademark Office for a new patent covering exosome platform technology, further strengthening BrainStorm's IP portfolio. Corporate March 31, 2025: The Company is announcing today that it has entered into a warrant inducement agreement pursuant to which it is expected to raise approximately $1.64 million, to close on or about April 1, 2025. Financial Results for the Year Ended December 31, 2024 Cash, cash equivalents, and short-term bank deposits were approximately $0.4 million as of December 31, 2024, compared to $1.5 million as of December 31, 2023. Subsequent to year-end, on March 31, 2025, the Company entered into a warrant inducement agreement pursuant to which it is expected to raise approximately $1.64 million, to close on or about April 1, 2025, as described above. Research and development expenditures, net, for the year ended December 31, 2024 were $4.7 million, compared to $10.7 million for the year ended December 31, 2023. General and administrative expenses for the twelve months ended December 31, 2024, and 2023 were approximately $7.0 million and $10.7 million, respectively. Net loss for the twelve months ended December 31, 2024, was approximately $11.6 million, as compared to a net loss of approximately $17.2 million for the twelve months ended December 31, 2023. Net loss per share for the twelve months ended December 31, 2024, and 2023 was $2.31 and $6.00, respectively. Conference Call and Webcast Monday March 31, 2025, at 8:30 a.m. U.S. Eastern Time Those that wish to listen to the replay of the conference call can do so by dialing the numbers below. The replay will be available until April 14, 2025. About NurOwn® The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been 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 (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. About Brainstorm Cell Therapeutics Inc. Brainstorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn® technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). Brainstorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). Brainstorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive multiple sclerosis (MS) and was supported by a grant from the National MS Society (NMSS). Notice Regarding Forward-Looking Statements This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding meetings with the U.S. Food and Drug Administration (FDA), Special Protocol Assessment (SPA), ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital, BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at . These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements. CONTACTS Michael Wood Phone: +1 646-597-6983 [email protected] The accompanying notes are an integral part of the consolidated financial statements. The accompanying notes are an integral part of the consolidated financial statements. Logo - SOURCE BrainStorm Cell Therapeutics Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Cell TherapyPhase 3Financial StatementPhase 2Orphan Drug

100 Deals associated with Debamestrocel

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Amyotrophic Lateral Sclerosis	Phase 3	United States	Brainstorm Cell Therapeutics, Inc.	28 Aug 2017
Alzheimer Disease	Phase 2	Netherlands	Brainstorm Cell Therapeutics, Inc.	06 Dec 2020
Multiple Sclerosis, Chronic Progressive	Phase 2	United States	Brainstorm Cell Therapeutics, Inc.	13 Mar 2019
Multiple Sclerosis	Phase 2	-	Brainstorm Cell Therapeutics, Inc.	-
Respiratory Distress Syndrome, Acute	Preclinical	United States	Brainstorm Cell Therapeutics, Inc.	03 Apr 2024
Autism Spectrum Disorder	Preclinical	-	Brainstorm Cell Therapeutics, Inc.	22 Sep 2023
Peripheral Nerve Injuries	Preclinical	-	Brainstorm Cell Therapeutics, Inc.	22 Sep 2023
Acute Lung Injury	Preclinical	United States	Brainstorm Cell Therapeutics, Inc.	-

Clinical Result

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Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03280056 (Company_Website) Manual	Phase 3	Amyotrophic Lateral Sclerosis	10	Debamestrocel	jzgqjzwvnu(jrxpiqcbxp) = advsxlnvof iuzucnlhwt (htpnknprcr )	Positive	28 Oct 2024
				Placebo	jzgqjzwvnu(jrxpiqcbxp) = bcgovzolhd iuzucnlhwt (htpnknprcr ) View more
NCT02017912 (NurOwn \| NurOwn® Phase 2 ALS Trial, CTgov)	Phase 2	Amyotrophic Lateral Sclerosis	48	Nurown MSC-NTF cells (Nurown MSC-NTF Cells)	ninxitpwrc = yppsddnimk ftyttlwebq (lkdvaluqzn, bqirktvusj - xhlbptkggj) View more	-	06 Jun 2024
				Placebo (Excipient)	ninxitpwrc = jdlnikydyk ftyttlwebq (lkdvaluqzn, mcddsyvivq - hchxmanjqr) View more
NCT04681118 (PRNewswire) Manual	Phase 3	Amyotrophic Lateral Sclerosis	10	NurOwn	-	Positive	20 May 2024
NCT03280056 (CTgov)	Phase 3	Amyotrophic Lateral Sclerosis	196	Bone Marrow aspiration+NurOwn® (MSC-NTF cells) (NurOwn® (MSC-NTF Cells))	rlswfebflq: Odds Ratio (OR) = 1.330 (95% CI, 0.632 - 2.798), P-Value = 0.453 View more	-	29 Feb 2024
				Placebo (Placebo)
NCT03799718 (NurOwn (MSC-NTF), CTgov)	Phase 2	Multiple Sclerosis, Chronic Progressive	23	NurOwn (MSC-NTF cells)	lhajgzxbqi = sqqzhntbcn hgvjjqnudb (ndjbifusln, mutbsbogle - yrpmapexvs) View more	-	14 Nov 2022
NCT03799718 (NurOwn (MSC-NTF), ECTRIMS2022)	Phase 2	Multiple Sclerosis VEGF1 \| HGF2 \| NCAM-13	18	MSC-NTF(MSC-NTF) (MSC-NTF cells)	xfzggecwjj(xvezevprkm) = kqvivgpexx nwrjncowqz (ookhlutozs ) View more	Positive	12 Oct 2022
NCT03799718 (Corporate Publications) Manual	Phase 2	Multiple Sclerosis	20	NurOwn	ptsgghuupa(qbhqviatfc) = mqxgudtvar sxwcpozixp (ojaqjutsmk ) View more	Positive	15 Sep 2022
				Control	ptsgghuupa(qbhqviatfc) = bzygdwcxri sxwcpozixp (ojaqjutsmk ) View more
NCT03280056 (MDA2022)	Phase 3	Amyotrophic Lateral Sclerosis SOD1 \| TARDBP \| PSEN2 ... View more	189	NurOwn	btnsjnogzd(ebynroqdho) = hfxscajrwa pydrnzmxdn (eausnklqok )	-	13 Mar 2022
				Placebo	btnsjnogzd(ebynroqdho) = plthdqdscv pydrnzmxdn (eausnklqok )
NCT02017912 (Pubmed) Manual	Phase 2	Amyotrophic Lateral Sclerosis	48	Neurotrophic factor-producing mesenchymal stem cell therapy	jzlrszukpi(hgsojjywfi) = The study met its primary safety endpoint. mbsjixzkth (blhdsrqhdd ) View more	Positive	10 Dec 2019
				Placebo
NCT02017912 (NurOwn® Phase 2 ALS Trial, AAN2018)	Phase 2	Amyotrophic Lateral Sclerosis	48	NurOwn®	cxuobcvnea(gjszdopcbw) = owwvmzccrz phouynthst (jkcqzqfiuv ) View more	Positive	10 Apr 2018
				Placebo	bpgdrxpmsn(nvouoahzow) = yhqodsgboc petdqanhks (nviweekghj ) View more

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