Design of Phase 2 Open-Label Multicenter Study in Progressive MS Outlined
NEW YORK and SEATTLE, May 31, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in developing innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, yesterday, on World MS Day, presented a poster of the Company’s “Phase 2 Open-Label, Multicenter Study of Repeated Intrathecal Administration of Autologous MSC-NTF cells in Progressive Multiple Sclerosis (MS)” at the Annual Meeting of the Consortium of Multiple Sclerosis Centers, in Seattle.
- Study Design: Open label, single-arm Phase 2 study, at 5 US MS clinical centers.
- Study Population: 20 Progressive MS patients with Expanded Disability Status Scale (EDSS) 3.0-6.5, based on the 2017 revised McDonald Criteria.
- Primary Endpoint: To evaluate the safety and tolerability of 3 repeated intrathecal doses of NurOwn® (MSC-NTF cells).
- Secondary Endpoints: To evaluate the efficacy of MSC-NTF cells using validated MS clinical outcome assessments as well as paired CSF and blood biomarker analyses.
- Study Status: Enrollment and treatment has been initiated with topline clinical data expected in the first half of 2020.
- Conclusion: This Phase 2 open label study was designed to provide preliminary data on the safety and efficacy of repeated intrathecal doses of MSC-NTF cells in progressive MS patients to inform the design of a Phase 3 pivotal trial.
“Remembering that World MS Day is a global event that raises awareness of the invisible symptoms of MS, we shared the design of our Phase 2 clinical study during the Annual Meeting of the Consortium of Multiple Sclerosis Centers," said Ralph Kern, MD, MHSc, Chief Operating Officer and Chief Medical Officer of BrainStorm. He added, "CMSC is the largest North American gathering for healthcare professionals and researchers engaged in MS care and we were pleased to share our investigational study design with leading members of the MS medical community.”
NurOwn® (autologous MSC-NTF) cells represent 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. 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. BrainStorm is currently conducting a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also recently received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.
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 status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm is currently enrolling a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at www.brainstorm-cell.com.
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Source: BrainStorm Cell Therapeutics Inc.