BrainStorm Cell Therapeutics has won a $15.9 million grant from California’s regenerative medicine agency toward a Phase III trial of its amyotrophic lateral sclerosis (ALS) cell therapy candidate NurOwn®.
The governing board of the California Institute for Regenerative Medicine (CIRM) approved the grant yesterday—the second instance of CIRM funding a clinical trial program focused on ALS.
“CIRM’s mission is to accelerate stem cell treatments to patients with unmet medical needs, and, in keeping with this mission, our objective is to find a treatment for patients ravaged by this neurologic condition for which there is currently no cure,” Maria Millan, M.D., CIRM’s interim president and CEO, said in a statement.
NurOwn uses mesenchymal stem cells that are converted using BrainStem’s proprietary technology into cells that secrete a variety of neurotrophic factors (NTFs) designed to treat neurodegenerative diseases by allowing for delivery of several NTFs at or close to the site of injury. The resulting neurotrophic factors-secreting mesenchymal stromal cells (MSC-NTF) cells secrete a variety of NTFs, including glial cell-derived neurotrophic factor (GDNF), brain-derived neurotrophic factor (BDNF), vascular endothelial growth factor (VEGF), and hepatocyte growth factor (HGF).
NurOwn technology was developed in the laboratories of Dani Offen, Ph.D., and the late Eldad Melamed, M.D., at Tel Aviv University.
BrainStorm said it is in advanced stages of planning the Phase III trial, set to be conducted at six top ALS clinical sites in the U.S. BrainStorm announced two of those sites on Tuesday—Massachusetts General Hospital and California Pacific Medical Center (CPMC), with Merit Cudkowicz, M.D., and Robert G. Miller, M.D., of the respective institutions, agreeing to participate as investigators.
The Phase III trial is expected to enroll approximately 200 patients. The trial’s primary outcome measure will be the ALS Functional Rating Scale (ALSFRS) score responder analysis.
BrainStorm said the patient population will be optimized to include faster-progressing patients who showed superior outcomes in NurOwn’s Phase II ALS trial (NCT02017912).
The company reported positive topline results from that 48-patient trial in July 2016, saying that at week 12 post-treatment, 40% of subjects in the NurOwn arm, compared with 17% of placebo subjects, experienced an improvement of at least 50% in the ALSFRS slope post-treatment compared to pre-treatment.
The Phase II study also showed NurOwn to be safe and well tolerated in patients.
“CIRM is partnering with BrainStorm to follow up on the company’s promising Phase II trial in ALS,” Dr. Millan added.
Publicly traded BrainStorm is based in Hackensack, NJ, and Petach Tikva, Israel.
The FDA has approved two ALS treatments. The second was approved on May 5, when the agency authorized Mitsubishi Tanabe’s Radicava™ (edaravone) as an intravenous infusion treatment for the disease. The agency cited a 137-patient Phase III trial in which participants receiving Radicava declined less on a clinical assessment of daily functioning compared to those receiving a placebo at 24 weeks following treatment.
The first authorized ALS treatment, riluzole, was approved by the FDA in 1995, then marketed as Rilutek® by Sanofi until it sold U.S. rights to Covis Pharma in 2013.
NurOwn is also in preclinical development as a treatment for progressive multiple sclerosis and for autism.
In October 2016, CIRM awarded a $16,961,287 grant toward a clinical trial led by Clive Svendsen, Ph.D., director of the Board of Governors Regenerative Medicine Institute at Cedars-Sinai.
Dr. Svendsen and colleagues at Cedars-Sinai are using engineered stem cells to produce GDNF, then inject the GDNF-producing stem cells into motor neurons in one side of the spinal cord, into an area that controls movement in just one leg. The team’s objective is to complete preclinical studies with a neural progenitor cell line transfected with GDNF, file an Investigational New Drug (IND) application, and complete a Phase I clinical trial in ALS patients.