Cerebrospinal fluid (CSF) biomarker analyses confirmed that treatment with NurOwn resulted in a statistically significant increase of neurotrophic factors and reduction in neurodegenerative and neuroinflammatory biomarkers that was not observed in the placebo treatment group. The pivotal study was intended to support a filing for FDA approval of autologous MSC-NTF cells in ALS and discussion of potential regulatory pathways for approval are planned with the U.S. FDA. NEW YORK, Nov. 17, 2020 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, announced today topline results from the Company's randomized, double-blind placebo-controlled Phase 3 trial evaluating NurOwn® (MSC-NTF cells) as a treatment for Amyotrophic lateral sclerosis (ALS). For more information, visit the company's website at www.brainstorm-cell.com. The phase 3 program was one of … Researchers at Northwestern University have trained an AI algorithm to automatically detect the signs of COVID-19 on a basic X-ray of the lungs. Merit Cudkowicz, Dr. James Berry); University of Massachusetts Medical School (Prof. Robert Brown) and Mayo Clinic (Prof. Anthony Windebank, Dr. Nathan Staff). In its briefing document, the FDA said the attributes “do not have a demonstrated relationship to the clinical performance of specific [drug product] lots.” The advisory committee backed the drug, but the FDA issued a complete response letter. Evidence of the prospects of remestemcel-L, a mesenchymal stem cell therapy, in COVID-19-related ARDS is set to arrive when the 300-patient phase 3 trial wraps up early next year. We identified a superior treatment response in a pre-specified subgroup of patients with less advanced disease. AstraZeneca has scrapped development of savolitinib as a monotherapy treatment for papillary renal cell carcinoma. Limited. BrainStorm's management team will host a call and webinar to discuss the Phase 3 data today at 8.30 AM EST. Register Today. Secondary endpoints included the percentage of patients with disease progression halted or improved, ALSFRS-R change from baseline, combined analysis of function and survival, slow vital capacity, tracheostomy-free survival, overall survival and cerebrospinal fluid biomarker measurements. All rights reserved. ", "This clinical trial included a more severely affected ALS population compared to other recent ALS clinical trials. A Phase 3 clinical trial designed to evaluate if an investigational vaccine can prevent symptomatic coronavirus disease 2019 (COVID-19) in adults has begun. In an important, pre-specified subgroup with early disease based on ALSFRS-R baseline score ³ 35, NurOwn demonstrated a clinically meaningful treatment response across the primary and key secondary endpoints and remained consistent with our pre-trial, data-derived assumptions. These estimates were based on available historical clinical trial data and the NurOwn Phase 2 data. Overview, Clinical Development BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. Given the heterogeneity of ALS, it is not surprising that measurement of treatment effect may be influenced by disease severity including the behavior of disease progression rates at the lower end of the scale. We also carried out pre-specified statistical modeling designed to predict clinical response with high sensitivity and specificity based on ALS biomarkers and ALS Function and confirmed that NurOwn treatment outcomes could be predicted by baseline ALS function as well as key CSF neurodegenerative and neuroinflammatory biomarkers. We want to thank our partners, I AM ALS, and ALSA, who kindly supported the biomarker study". The FDA will review the data to see if there is a path forward to support approval" said Chaim Lebovits, Chief Executive Officer of BrainStorm. For more information on the trial, visit https://clinicaltrials.gov/ct2/show/NCT03280056. Participants were followed for 28 weeks after treatment. Production, Pipeline Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). Novartis has secured a global license to Mesoblast’s cell therapy remestemcel-L in the treatment of COVID-19. Last month, the FDA rejected a filing for approval of the cell therapy in children with steroid-resistant graft-versus-host disease (GvHD). NEW YORK, Nov. 17, 2020 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapies for neurodegenerative diseases, announced today topline results from the Company's randomized, double-blind placebo-controlled Phase 3 trial evaluating NurOwn® (MSC-NTF cells) as a treatment for Amyotrophic lateral sclerosis (ALS). Novartis, which has an approved cell therapy, may have the expertise to ensure remestemcel-L fares better when up for review by the FDA as a treatment for ARDS in COVID-19 patients. 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. 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. Therapy, MSC-NTF The secondary efficacy endpoint measuring average change in ALSFRS-R total score from baseline to Week 28, was -5.52 with NurOwn versus -5.88 on Placebo, a difference of 0.36 (p= 0.693). Join Thermo Fisher Scientific’s upcoming webinar to learn why softgels offer numerous benefits for Rx drug development, including enhanced bioavailability, patient compliance and easy scale-up. We are committed to learning as much as we can from this trial and to partner with the ALS community to progress our collective understanding of ALS, which in turn will help us to continue to bring forward new treatments for this unrelenting disease.". Results from the trial showed that NurOwn® was generally well tolerated in this population of rapidly progressing ALS patients. Chauk. The placebo response observed in this trial is unprecedented and the ability to show treatment benefit in this context provides evidence of the clinical value of NurOwn. 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. Indeed, few potential ALS treatments make it to Phase 3 trials—the final stage of clinical testing before a drug is approved by the FDA for treatment. The deal lands Mesoblast $50 million upfront and the support of a partner that could help address critical quality attribute concerns raised by the FDA. The Phase 2 study of autologous MSC-NTF cells in patients with progressive MS (NCT03799718) started enrollment in March 2019. 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). BrainStorm has completed the Phase 3 pivotal trial in ALS. Potential participants with ALS were screened during an 18-week run-in period and those who were rapid progressors (defined as patients with at least a 3 point decrease in ALSFRS-R score during the run-in period) were randomized 1:1 to receive three intrathecal injections (8 weeks between each injection) of NurOwn® or placebo. By then, Mesoblast had already begun testing the allogeneic cell therapy in COVID-19 patients with acute respiratory distress syndrome (ARDS). The replay will be available for 14 days. Novartis has stepped in to help Mesoblast take remestemcel-L forward in ARDS. of Use, https://clinicaltrials.gov/ct2/show/NCT03280056, https://www.webcaster4.com/Webcast/Page/2354/38723. "In addition to planned scientific engagements, biosamples from this study will be shared through the NEALS biorepository to enable additional scientific discovery efforts. "The consistency of effect observed across NurOwn treated patients, including within pre-specified subgroups, highlights an important treatment effect in a fatal disease with very limited treatment options. MS, Patients & The potential risks and uncertainties include, without limitation, the regulatory approval potential of BrainStorm's NurOwn® treatment candidate, the success of BrainStorm's product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorm's NurOwn® treatment candidate, if approved, to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorm's ability to manufacture and commercialize the NurOwn® treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorm's ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation BrainStorm's need to raise additional capital, BrainStorm's ability to continue as a going concern; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. Caregivers, Privacy The vaccine, known as mRNA-1273, was co-developed by the Cambridge, Massachusetts-based biotechnology company Moderna, Inc., and the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of … The Phase 3 NurOwn® trial was a multi-center, placebo-controlled, randomized, double-blind trial designed to evaluate the safety and efficacy of NurOwn® in 189 ALS patients. Evidence of the prospects of remestemcel-L, a mesenchymal stem cell therapy, in COVID-19-related ARDS is set to arrive when the 300-patient phase 3 trial wraps up early next year. Even after the bump, the stock remains down on the highs it hit before the complete response letter. We are in active discussions with the FDA who have expressed their eagerness to review the data and have committed to prioritize review of this data. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. Trials, Preapproval Access The primary endpoint was achieved in 34.7% of NurOwn participants versus 27.7% for Placebo (p=0.453). The Phase 3 clinical trial's primary efficacy endpoint, a responder analysis evaluating the proportion of participants who experienced a 1.25 points per month improvement in the post-treatment Revised Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) slope, was powered on assumed treatment response rates of 35% on NurOwn versus 15% on Placebo. Novartis plans to run a phase 3 trial in non-COVID-19 ARDS patients, setting it up to address the broader unmet medical need. Those interested in listening to the conference call live via the internet may do so by visiting the "Investors & Media" page of BrainStorm's website at www.ir.brainstorm-cell.com and clicking on the conference call link. It is important to fully explore this finding. BrainStorm Announces Topline Results from NurOwn® Phase 3 ALS Study, Clinical trial did not meet statistical significance in primary efficacy endpoint, NurOwn® showed a clinically meaningful treatment response compared to placebo in a pre-specified subgroup, CSF biomarker analyses confirmed NurOwn resulted in a statistically significant increase of neurotrophic factors and reduction in neurodegenerative and neuroinflammatory biomarkers, Company management to host conference call and live webcast today at 8:30 AM ET, Menu Caregivers, About Progressive SOURCE BrainStorm Cell Therapeutics Inc. Corporate Headquarters1325 Avenue of Americas, 28th FloorNew York City, NY 10019Phone: ARDS causes 40% mortality in the around 200,000 patients who develop the condition in the U.S. annually. Novartis will also provide “support to enable commercial manufacturing scale-up.” Mesoblast has previously said it needed to “substantially scale up manufacturing” to serve the COVID-19 market. I also want to thank the California Institute for Regenerative Medicine (CIRM) for their enormous support to conduct this trial.". MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. The phase 3 started in May is designed to validate signs of efficacy seen in a 12-patient compassionate use program. The focus on critical quality attributes reflects the problems Mesoblast encountered when it tried to win FDA approval for remestemcel-L in GvHD earlier this year. Toggle, Autologous Cellular "More detailed analyses will be shared at upcoming scientific conferences and in subsequent publications. If the trial is successful, Novartis plans to work with Mesoblast to “develop appropriate critical quality attributes that meet [FDA] requirements for remestemcel-L.”. Webcast URL: https://www.webcaster4.com/Webcast/Page/2354/38723. (NCT03280056); this trial investigated repeat-administration of autologous MSC-NTF cells at six U.S. sites supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). Toll Free: 877-481-4010International: 919-882-2331Replay Passcode: 38723. In addition, NurOwn was observed to have its clear intended biological effects with important changes in the pre-specified disease and drug related biomarkers. MS, Clinical The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. Therefore, the trial met the expected 35% NurOwn treatment group efficacy response assumption, however the high placebo response exceeded placebo responses observed in contemporary ALS trials. Novartis also has an option to distribute remestemcel-L outside of Japan in GvHD and to co-fund its development and commercialization in other non-respiratory indications. Policy, Terms We are committed to advancing discussions with the FDA to identify regulatory pathways that may support NurOwn in ALS," commented Ralph Kern MD MHSc, President and CMO of Brainstorm. "We would like to sincerely thank the patients, their families and caregivers, investigators and staff who participated in this study, as their dedication and hard work allowed for the study's on-time completion despite the ongoing COVID-19 pandemic. The robust changes in biomarkers of Neurodegeneration, including NfL and MCP-1, which allows identification of likely responders prior to treatment is encouraging", said Stacy Lindborg PhD, EVP and Head of Global Clinical Research. Autologous MSC-NTF cells can 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. Cells, MSC-NTF Cell Media, Patients & +1-201-488-0460. (Novartis), From Patient Adherence to Manufacturing Ease - Why Softgels Make Sense for Rx. In return for a $25 million upfront payment and $25 million investment, plus milestones and royalties, Mesoblast has given Novartis a global license to develop and commercialize remestemcel-L in ARDS. Statements in this announcement other than historical data and information, including statements regarding the topline results from the NurOwn® Phase 3 ALS study and future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Website by © 2020 Questex LLC. Program, Progressive A change in pre- to post- treatment slope of 1.25 or more is substantial and clinically important. 3 Speen Street, Suite 300, Framingham, MA 01701. Subscribe to FierceBiotech to get industry news and updates delivered to your inbox. 10-jun-2014 - James Becerra descrubrió este Pin. Policies, University of Massachusetts Medical School, Investors & In this subgroup, there were 34.6% responders who met the primary endpoint definition on NurOwn and 15.6% on Placebo (p=0.288), and the average change from baseline to week 28 in ALSFRS-R total score was -1.77 on NurOwn and -3.78 on Placebo (p=0.198), an improvement of 2.01 ALSFRS-R points favoring NurOwn. "The findings from this clinical trial demonstrated that NurOwn treatment was associated with a clinically meaningful treatment response and consistent biomarker effects in known ALS disease pathways and that the ability of the clinical trial to demonstrate treatment effects compared to placebo are influenced by baseline disease status, as revealed through ALS function and key biomarkers. exclude terms. © Copyright 2019, BrainStorm Cell These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. Enclose phrases in quotes. Descubre (y guarda) tus propios Pines en Pinterest. Novartis CEO Vas Narasimhan, M.D. Dr. Polyphor is developing an inhaled version of murepavadin, which targets Pseudomonas aeruginosa infections, but is currently given intravenously. The call can be accessed by dialing the numbers below: Toll Free: 877-407-9205International: 201-689-8054. 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. BrainStorm is also conducting an FDA-cleared Phase 2 open-label multicenter trial in progressive multiple sclerosis (MS). The relationship between the companies extends beyond COVID-19. The primary endpoints of the trial were safety assessments and a responder analysis of the rate of decline in ALSFRS-R score over 28 weeks, where response was defined as participants with a ³ 1.25 points/month improvement in the post-treatment versus pre-treatment slope in ALSFRS-R at 28 weeks following the first treatment. Reproduction in whole or part is prohibited. CONTACTS Investor Relations:Corey Davis, Ph.D.LifeSci Advisors, LLCPhone: [email protected], Media:Paul TyahlaSmithSolvePhone: + [email protected] It was conducted at six centers of excellence: University of California Irvine (Dr. Namita Goyal); Cedars-Sinai Medical Center (Dr. Matthew Burford); California Pacific Medical Center (Prof. Robert Miller); Massachusetts General Hospital (Prof. Merit Cudkowicz, one of the Principal Investigators of this trial and the Julianne Dorn Professor of Neurology at Harvard Medical School and the Director of the Healey Center for ALS and Chair of Neurology at Mass General Hospital said, "We found a clinically meaningful response to NurOwn in a pre-specified group of patients (greater than or equal to 35 ALSFRS-R at baseline). Shares in Mesoblast rose 18% in response to the news. Use a + to require a term in results and - to All rights reserved. It is this history of failure that has sparked so much enthusiasm in a new stem cell treatment for ALS. A COVID-19 antibody diagnostic developed through a joint venture between Mount Sinai Health System and RenalytixAI has been authorized by the FDA. Novartis licenses phase 3 COVID-19 cell therapy from Mesoblast, FDA OKs COVID-19 antibody test from Mount Sinai, RenalytixAI JV, Northwestern's open AI spots COVID-19 signs in chest X-rays, Polyphor snags $3.3M for inhaled antibiotic. There will also be a replay of the call which can be accessed by using the webcast link above or by dialing the numbers below. While showing a numerical improvement in the treated group compared to placebo across the primary and key secondary efficacy endpoints, the trial did not reach statistically significant results.