, /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a starring developer of cellular therapies for neurodegenerative diseases, contiguous announced that the institution has received a refusal to record missive from the U.S. Food and Drug Administration (FDA) regarding the company's New Biologics License Application (BLA) for NurOwn for the attraction of ALS. The FDA has indicated that the institution tin petition a Type A gathering to sermon the contented of the refusal to record letter.
"While we are disappointed that the FDA has not accepted our BLA for NurOwn successful ALS, we stay committed to NurOwn's advancement arsenic a attraction for this devastating disease. The institution intends to petition a Type A gathering and looks guardant to continued discussions with the FDA," said Chaim Lebovits, Chief Executive Officer of BrainStorm. "We proceed to judge that NurOwn's Phase 3 proceedings represents a important publication to ALS therapy and volition proceed to enactment tirelessly to code the needs of radical surviving with ALS by advancing subject and partnering with researchers astir the world."
The three, co-principal investigators of the NurOwn Phase 3 survey were Dr. Robert Brown, Director of the Program successful Neurotherapeutics astatine the University of Massachusetts Medical School, Dr. Merit Cudkowicz, Chief of Neurology astatine Massachusetts General Hospital, Julieanne Dorn Professor of Neurology astatine Harvard Medical School, Director of the Sean M. Healey & AMG Center for ALS astatine Massachusetts General Hospital and Dr. Tony Windebank, Professor of Neurology and Judith and James Pape Adams Foundation Professor of Neuroscience astatine Mayo Clinic.
Drs. Brown, Cudkowicz and Windebank jointly stated, "While the pre-specified superior result measurement was not met, determination were participants with beneficial clinical effects and wide changes successful applicable biomarkers of cause effect. Understanding whether determination are radical with ALS who mightiness respond amended to NurOwn is important fixed the unmet therapeutic need. As the 3 co-PIs of the Phase 3 survey of NurOwn, we enactment continued discussions with the FDA connected the champion way forward."
BrainStorm completed a Phase 3 proceedings successful 200 participants with ALS (Cudkowicz et al., 2022 Muscle and Nerve). In the effort to analyse a real-world population, the survey enrolled radical with much precocious illness than different late-stage ALS trials. In fact, much than a 3rd of these participants with precocious illness entered the proceedings with the 1 oregon much dimensions of carnal relation (e.g., dressing/hygiene, cutting food, walking) starting astatine the lowest imaginable people of 0 connected the ALSFRS-R; thereby preventing the measurement of further deterioration. A pre-specified subgroup of participants, with baseline ALSFRS-R³35, which controls for this "scale effect" showed a inclination to a meaningful summation successful the objective effect with NurOwn compared to placebo. The secondary endpoint, mean ALSFRS-R alteration from baseline to 28 weeks successful this subgroup, was statistically important (p=0.050, Muscle and Nerve Supplemental File and Muscle and Nerve Erratum). In addition, post-hoc sensitivity analyses were presented past week (21st Annual NEALS Meeting 2022) which besides showed a statistical inclination towards a clinically meaningful attraction effect with NurOwn crossed subgroups, and 1 that is accordant with the pre-specified subgroup of participants with little precocious ALS astatine baseline. Finally, biomarker information successful each proceedings participants besides showed accordant patterns of NurOwn reducing markers of inflammation and neurodegeneration, and expanding neuroprotective and anti-inflammatory markers comparative to placebo, further supporting the conception that proceedings participants taking NurOwn are so experiencing a affirmative biologic effect (ALS ONE Research Symposia 2022).
BrainStorm volition sermon its firm strategy and plans to beforehand NurOwn's improvement connected its 3rd 4th 2022 net call, to instrumentality spot astatine 8:00 a.m. Eastern Time connected Monday, November 14, 2022. Those funny successful joining the telephone tin find further accusation here.
Phase 3 NurOwn Study Design
The Phase 3 NurOwn proceedings was a multi-center, placebo-controlled, randomized, double-blind trial designed to measure the information and efficacy of repetition doses of NurOwn successful 189 ALS participants. It was conducted astatine six centers of excellence: University of California Irvine (Dr. Namita Goyal); Cedars-Sinai Medical Center (Dr. Matthew Burford, Dr. Robert Baloh); California Pacific Medical Center (Prof. Robert Miller, Dr. Jonathan Katz); Massachusetts General Hospital (Prof. Merit Cudkowicz, Dr. James Berry); University of Massachusetts Medical School (Prof. Robert Brown) and Mayo Clinic (Prof. Anthony Windebank, Dr. Nathan Staff). Potential participants with ALS were screened during an 18-week run-in play and those who were accelerated progressors (defined arsenic participants with astatine slightest a 3-point alteration successful ALSFRS-R people during the run-in period) were randomized 1:1 to person 3 intrathecal injections (8 weeks betwixt each injection) of NurOwn oregon placebo. Participants were followed for 28 weeks aft treatment. The superior endpoints of the proceedings were information assessments and a responder investigation of the complaint of diminution successful ALSFRS-R people implicit 28 weeks, wherever effect was defined arsenic participants with a 1.25 points/month betterment successful the post-treatment versus pre-treatment slope successful ALSFRS-R astatine 28 weeks pursuing the archetypal treatment. Secondary endpoints included the percent of participants with illness progression halted oregon improved, ALSFRS-R alteration from baseline, combined investigation of relation and survival, dilatory captious capacity, tracheostomy-free survival, wide endurance and cerebrospinal fluid biomarker measurements. For much accusation connected the trial, visit https://clinicaltrials.gov/ct2/show/NCT03280056.
The NurOwn® exertion level (autologous MSC-NTF cells) represents a promising investigational therapeutic attack to targeting illness pathways important successful neurodegenerative disorders. MSC-NTF cells are produced from autologous, bony marrow-derived mesenchymal stem cells (MSCs) that person been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by increasing them nether patented conditions that induce the cells to secrete precocious levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to efficaciously present aggregate NTFs and immunomodulatory cytokines straight to the tract of harm to elicit a desired biologic effect and yet dilatory oregon stabilize illness progression.
About BrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc. is simply a starring developer of innovative autologous big stem compartment therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to objective improvement and commercialization of the NurOwn® exertion level utilized to nutrient autologous MSC-NTF cells done an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells person received Orphan Drug designation presumption from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the attraction of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal proceedings successful ALS (NCT03280056); this proceedings investigated the information and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a assistance from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed nether an investigational caller cause exertion a Phase 2 open-label multicenter proceedings (NCT03799718) of autologous MSC-NTF cells successful progressive MS and was supported by a assistance from the National MS Society (NMSS).
Safe-Harbor Statement
Statements successful this announcement different than humanities information and information, including statements regarding aboriginal objective proceedings enrollment and data, represent "forward-looking statements" and impact risks and uncertainties that could origin BrainStorm Cell Therapeutics Inc.'s existent results to disagree materially from those stated oregon implied by specified forward-looking statements. Terms and phrases specified arsenic "may," "should," "would," "could," "will," "expect," "likely," "believe," "plan," "estimate," "predict," "potential," and akin presumption and phrases are intended to place these forward-looking statements. The imaginable risks and uncertainties include, without limitation, BrainStorm's request to rise further capital, BrainStorm's quality to proceed arsenic a going concern, prospects for aboriginal regulatory support of BrainStorm's NurOwn® attraction candidate, the occurrence of BrainStorm's merchandise improvement programs and research, regulatory and unit issues, improvement of a planetary marketplace for our products and services, the quality to unafraid and support probe institutions to behaviour our objective trials, the quality to make important revenue, the quality of BrainStorm's NurOwn® attraction campaigner to execute wide acceptance arsenic a attraction enactment for ALS oregon different neurodegenerative diseases, BrainStorm's quality to manufacture and commercialize the NurOwn® attraction candidate, obtaining patents that supply meaningful protection, contention and marketplace developments, BrainStorm's quality to support our intelligence spot from infringement by 3rd parties, heath betterment legislation, request for our services, currency speech rates and merchandise liability claims and litigation; the impacts of the COVID-19 pandemic connected our objective trials, proviso chain, and operations; and different factors elaborate successful BrainStorm's yearly study connected Form 10-K and quarterly reports connected Form 10-Q disposable at http://www.sec.gov. These factors should beryllium considered carefully, and readers should not spot undue reliance connected BrainStorm's forward-looking statements. The forward-looking statements contained successful this property merchandise are based connected the beliefs, expectations, and opinions of absorption arsenic of the day of this property release. We bash not presume immoderate work to update forward-looking statements to bespeak existent results oregon assumptions if circumstances oregon management's beliefs, expectations oregon opinions should change, unless different required by law. Although we judge that the expectations reflected successful the forward-looking statements are reasonable, we cannot warrant aboriginal results, levels of activity, performance, oregon achievements.
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