Latest news with #NurOwn
Yahoo
27-05-2025
- Business
- Yahoo
BrainStorm Secures Key Manufacturing Partnership with Minaris for Upcoming NurOwn® Phase 3b ALS Clinical Trials
NEW YORK, May 27, 2025 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, today announced that it has signed a Letter of Intent (LOI) with Minaris Advanced Therapies, a global contract development and manufacturing organization (CDMO) specializing in cell and gene therapies, to manufacture NurOwn® for its upcoming clinical trial. The LOI marks the beginning of a strategic collaboration between BrainStorm and Minaris to initiate the technology transfer of NurOwn® in preparation for clinical trial manufacturing at Minaris' state-of-the-art facility in Allendale, New Jersey. This partnership supports BrainStorm's readiness for its planned Phase 3b clinical trial. "We are excited to support BrainStorm in the next phase of NurOwn development," said Eytan Abraham, Ph.D., Chief Commercial and Technology Officer at Minaris Advanced Therapies. "Our team looks forward to applying our expertise in cell therapy manufacturing to enable BrainStorm to conduct its clinical trial to the highest standards and move efficiently to commercial manufacturing." This partnership enhances BrainStorm's U.S. based manufacturing capabilities and supports its multicenter Phase 3b clinical trial in the United States. It complements BrainStorm's recently announced collaboration with Pluri Inc. (Nasdaq: PLUR) in Israel, which will also contribute to the production of clinical trial material. Together, these strategic relationships reinforce BrainStorm's commitment to building a robust and flexible manufacturing network to advance the clinical development of NurOwn®. Chaim Lebovits, President and Chief Executive Officer of BrainStorm Cell Therapeutics, commented, "This partnership reflects our ongoing commitment to the ALS community and to advancing a therapy with the potential to meaningfully impact patients' lives. NurOwn® represents a scientifically advanced and differentiated approach to treating ALS, and by joining forces with Minaris Advanced Therapies, we are ensuring that our clinical and manufacturing strategies are tightly aligned to support a successful Phase 3b trial and beyond," 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. CONTACTSInvestors:Michael Wood Phone: +1 646-597-6983mwood@ Media:Uri Yablonka, Chief Business Officer Phone: +1 917-284-2911 uri@ Logo: View original content: SOURCE BrainStorm Cell Therapeutics Inc. Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
20-05-2025
- Business
- Yahoo
Brainstorm Cell Therapeutics Inc (BCLI) Q1 2025 Earnings Call Highlights: Pivotal Phase 3b ...
Phase 3b Trial Initiation: FDA clearance received to initiate pivotal Phase 3b trial for NurOwn. Manufacturing and Site Selection: Initial manufacturing at Tel Aviv Sourasky Medical Center; technology transfer to Pluri for additional facilities. Clinical Trial Agreements: Negotiations with approximately 15 leading clinical centers in the U.S. for Phase 3b trial sites. Trial Design: Phase 3b trial includes a 24-week double-blind period followed by a 24-week open-label extension. Primary Endpoint: Change from baseline to week 24 in ALSFRS-R total score. Biomarker Analysis: NurOwn associated with reduction in neuroinflammatory and neurodegenerative biomarkers. Genetic Substudy: UNC13A genotype may influence response to NurOwn therapy. Warning! GuruFocus has detected 2 Warning Signs with BCLI. Release Date: May 19, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. The U.S. FDA has cleared Brainstorm Cell Therapeutics Inc (NASDAQ:BCLI) to initiate a pivotal Phase 3b trial for NurOwn, marking a significant milestone in their ALS treatment development. The trial design has been agreed upon with the FDA under a special protocol assessment (SPA), which de-risks the regulatory pathway for NurOwn. Brainstorm Cell Therapeutics Inc (NASDAQ:BCLI) has secured a leading U.S. clinical site that has passed FDA inspection, enhancing their operational readiness for the upcoming trial. The company is actively engaged in negotiations with approximately 15 leading clinical centers across the United States for the Phase 3b trial, indicating strong interest from the ALS research community. Brainstorm Cell Therapeutics Inc (NASDAQ:BCLI) is pursuing multiple funding avenues, including a promising $15 million non-dilutive grant, to ensure the timely commencement of the trial. The company faces significant financial constraints, which are common in the biotech industry, and securing proper funding is essential to commence the trial. The initiation and successful execution of the clinical trial demand a robust and sustainable cash flow, which is currently a challenge for Brainstorm Cell Therapeutics Inc (NASDAQ:BCLI). The company has not yet signed clinical trial agreements (CTAs) with the clinical centers, which could delay the trial's start. The manufacturing capacity at the Tel Aviv facility is limited, and the company is still in the process of expanding its manufacturing footprint to the United States. The exosome program, while promising, is still in the preclinical stage and requires strategic partnerships to advance towards clinical development. Q: Can you start the trial without proper funding? A: Chaim Lebovits, President and CEO, stated that while significant progress has been made in preparing for the trial, initiating and executing it requires robust funding. They are actively pursuing multiple funding avenues, including a $15 million non-dilutive grant and strategic partnerships, to ensure the trial's commencement. Q: What is the significance of naming the trial ENDURANCE? A: Chaim Lebovits explained that the name ENDURANCE resonates with the ALS community, symbolizing the strength and resilience of individuals with ALS and their families. It also reflects Brainstorm's commitment to generating robust data for NurOwn's regulatory approval. Q: Will the company also be producing in the U.S.? A: Hartoun Hartounian, COO, confirmed that expanding manufacturing to the U.S. is a strategic objective. They plan to announce a letter of intent with a U.S.-based facility that has passed FDA inspection, which is crucial for future commercialization and supply chain security. Q: Can you update on any advances in the exosome program? A: Netta Blondheim-Shraga, SVP of R&D, shared that the exosome program is progressing well, showing potential in treating respiratory and inflammatory diseases. They are preparing a manuscript on its efficacy in a COPD model and are pursuing strategic partnerships for clinical development. Q: Have you discussed stratifying by UNC13A with the FDA for the Phase 3b trial? A: Ibrahim Dagher, CMO, noted that while the FDA has not approved any biomarker as a surrogate, the UNC13A findings are exploratory. The trial design under the SPA is agreed upon, but they will continue to explore genetic factors in post hoc analyses. Q: How does the hypoxic stress co-culture with neuron cells support NurOwn's mechanism of action? A: Netta Blondheim-Shraga explained that the enriched media from NurOwn cells showed a protective effect on cells under hypoxic conditions, restoring them to near-normal levels. This supports NurOwn's mechanism and was included in their IND. Q: What are the plans for clinical trial sites and patient enrollment? A: Chaim Lebovits mentioned plans to open approximately 15 clinical trial sites, with details on They aim to enroll 200 patients over two to three years, with the BLA filing contingent on significant results from the first part of the trial. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
Yahoo
15-05-2025
- Business
- Yahoo
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 Details of the trial, known as ENDURANCE, are now available on 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. CONTACTSInvestors:Michael WoodPhone: +1 646-597-6983mwood@ Media: Uri Yablonka, Chief Business OfficerPhone: +1 917-284-2911uri@ BRAINSTORM CELL THERAPEUTICS INC. AND SUBSIDIARIES INTERIM CONDENSED CONSOLIDATED BALANCE SHEETS U.S. dollars in thousands (Except share data)March 31,December 31, 20252024 UnauditedAudited U.S. $ in thousands ASSETSCurrent Assets: Cash and cash equivalents$ 1,644$ 187 Other accounts receivable 67 63 Prepaid expenses and other current assets 621 135 Total current assets$ 2,332$ 385Long-Term Assets: Prepaid expenses and other long-term assets $ 22$ 22 Restricted Cash 182 184 Operating lease right of use asset (Note 4) 653 807 Property and Equipment, Net 382 434 Total Long-Term Assets$ 1,239$ 1,447Total assets$ 3,571$ 1,832LIABILITIES AND STOCKHOLDERS' EQUITY (DEFICIT)Current Liabilities: Accounts payables$ 6,797$ 6,080 Accrued expenses 538 619 Short-term loans (Note 7) 1,200 300 Operating lease liability (Note 4) 443 549 Employees related liability 1,923 1,430 Total current liabilities$ 10,901$ 8,978Long-Term Liabilities: Operating lease liability (Note 4) 127 171 Warrants liability (Note 5) - 447 Total long-term liabilities$ 127$ 618Total liabilities$ 11,028$ 9,596Stockholders' Deficit: Stock capital: (Note 6) 15 14 Common Stock of $0.00005 par value - Authorized: 250,000,000 shares at March 31, 2025 and at December 31, 2024 respectively; Issued and outstanding: 7,911,204 and 6,141,762 shares at March31, 2025 and December 31, 2024 respectively (*) Additional paid-in-capital 222,144 218,974 Treasury stocks (116) (116) Accumulated deficit (229,500) (226,636) Total stockholders' deficit$ (7,457)$ (7,764)Total liabilities and stockholders' deficit$ 3,571$ 1,832 The accompanying notes are an integral part of the consolidated financial statements. BRAINSTORM CELL THERAPEUTICS INC. AND SUBSIDIARIES INTERIM CONDENSED CONSOLIDATED STATEMENTS OF COMPREHENSIVE LOSS (UNAUDITED) U.S. dollars in thousands (Except share data) Three months ended March 31, 20252024Unaudited Operating expenses:Research and development, net $ 1,304$ 961 General and administrative1,785 1,513 Operating loss(3,089) (2,474) Financial income, net46 13 Loss on change in fair value of Warrants liability (Note 6)(179) (940) Net loss $ (2,864)$ (3,401) Basic and diluted net loss per share from continuing operations $ (0.45)$ (*) (0.75) Weighted average number of shares outstanding used in computing basic and diluted net loss per share6,342,002 (*) 4,315,903 The accompanying notes are an integral part of the consolidated financial statements. Logo: View original content: SOURCE BrainStorm Cell Therapeutics Inc.
Yahoo
06-05-2025
- Business
- Yahoo
BrainStorm to Present Biomarker Insights Supporting NurOwn's Mechanism of Action and Clinical Impact at the 2025 ALS Drug Development Summit
"Biomarker data from our prior Phase 3 study suggest a potential multimodal mechanism of action and deepen our understanding of how NurOwn may influence ALS disease pathways," said Blondheim-Shraga. "These insights are helping to refine the biomarkers that will be further evaluated in the upcoming Phase 3b trial of NurOwn, under a Special Protocol Assessment (SPA) agreement with the U.S. Food and Drug Administration (FDA)." Story Continues 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 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. CONTACTS Investors: Michael Wood Phone: +1 646-597-6983 mwood@ Media: Uri Yablonka, Chief Business Officer Phone: +1 917-284-2911 uri@ Logo - Cision View original content: SOURCE BrainStorm Cell Therapeutics Inc.
Yahoo
29-04-2025
- Health
- Yahoo
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 TrialLead Author Bob Dagher, MDOral Session: Mesenchymal Stem/Stromal Cells #1Date and time: Date & Time: Thursday, May 8, 2025 | 9:00 – 10:00 a.m. U.S. Central 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 WoodPhone: +1 646-597-6983mwood@ Logo: View original content: SOURCE BrainStorm Cell Therapeutics Inc. Sign in to access your portfolio
