Brainstorm Cell Therapeutics (NSDQ:BCLI) said today that the first patients have been enrolled in its Phase III trial evaluating NurOwn as a treatment for amyotrophic lateral sclerosis, or ALS, at Massachusetts General Hospital and UC Irvine Medical Center.
The trial, which is slated to enroll 200 patients, is designed to include the pre-specified patient subgroups who saw superior outcomes in the NurOwn Phase II ALS trial. BrainStorm said it expects top-line data from the trial in 2019.
A $16 million non-dilutive grant from the California Institute of Regenerative Medicine is helping support BrainStorm’s late-stage trial and the company said it has received a milestone payment of $5.5 million, or nearly 30% of the grant.
“NurOwn is a highly innovative and advanced stem cell therapy now being studied in a Phase III trial in ALS. The support of this trial by the California Institute of Regenerative Medicine highlights the importance of addressing ALS unmet need beyond currently available therapies, which slow disease progression but do not maintain or restore function,” president & CEO Chaim Lebovits said in prepared remarks. “BrainStorm plans to quickly advance the Phase III trial to confirm promising Phase II efficacy findings and bring much needed hope to ALS patients and their families.”
“Stem cells are emerging as a potential viable candidate to treat ALS, and we are very excited to be participating in this important Phase III trial,” Dr. Robert Miller, director of the Forbes Norris ALS Research Center at the California Pacific Medical Center, added.
“The clinical data from NurOwn have been encouraging, demonstrating clear evidence of clinical benefit and supported by analysis of inflammatory markers and neurotrophic factors in treated patients. NurOwn cells have the important advantage of being autologous, so patients do not need to be immunosuppressed. In addition, the production process is relatively straightforward, and the harvesting and delivery of cells is much less invasive than other stem cell systems that have been tested in the clinic. We have been conducting ALS clinical trials for more than two decades at California Pacific Medical Center and this is, by far, the most exciting trial in which we have been involved to date.”
“CIRM’s partnership with Brainstorm Therapeutics represents an international collaboration to find a treatment for ALS or ‘Lou Gehrig’s Disease,’ a devastating neurologic and fatal condition for which there is currently no durable treatment or cure,” CIRM president & CEO Dr. Maria Millan said. “CIRM is partnering with Brainstorm to follow up on the Company’s promising Phase II trial in patients with ALS.”