BrainStorm Announces First Contracted US Clinical Site for Phase 2 Progressive MS Study

BrainStorm Cell Therapeutics Inc. recently announced Cleveland Clinic as the first US clinical site contracted for a planned Phase 2 open-label, multicenter study of repeated intrathecal administration of autologous MSC-NTF cells in participants with progressive Multiple Sclerosis (MS). The Phase 2 study (NCT03799718) will enroll progressive MS patients [Expanded Disability Status Scale (EDSS) 3.0-6.5] based on 2017 revised McDonald Criteria.

MS is a chronic neuroinflammatory and neurodegenerative disorder that affects the brain and spinal cord. MS affects approximately 1 million individuals in the US and 2.5 million individuals worldwide. Approximately half of affected individuals will eventually develop a progressive disease, which may lead to increasing levels of motor, visual, and cognitive functional impairment and disability.

“We are very excited to announce The Mellen Center for MS Treatment and Research at Cleveland Clinic as the first contracted US clinical site for this very important Phase 2 progressive MS study that we plan to initiate in early 2019,” said Chaim Lebovits, President and CEO of BrainStorm. “This is a crucial step forward toward rapidly enrolling a Phase 2 study to evaluate this innovative autologous cell therapy approach using our NurOwn technology platform in progressive MS patients.”

“Progressive MS treatment options are limited and do not directly address unmet need,” said Ralph Kern, MD MHSc, COO, and CMO of BrainStorm, “This phase 2 clinical trial is an important part of our commitment to bring a new treatment modality and hope to MS patients.”

Cleveland Clinic is currently ranked as the No. 2 hospital in the country, according to U.S. News & World Report (2017-2018). The Mellen Center for Multiple Sclerosis Treatment and Research at Cleveland Clinic is one of the largest and most comprehensive programs for MS care and clinical research worldwide, managing over 8,000 patients and over 21,000 total visits annually.

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 plans to start enrollment in early 2019.

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 US 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 US, supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for US FDA approval of autologous MSC-NTF cells in ALS. For more information, visit www.brainstorm-cell.com.