Israel’s BrainStorm Cell Therapeutics has announced positive top-line Phase 2 data on NurOwn, a customized stem-cell therapy technology to treat amyotrophic lateral sclerosis (ALS).
BrainStorm has been developing and commercializing NurOwn in partnership with Ramot, the business engagement unit of Tel Aviv University. NurOwn enables the production of growth factors that support the survival of neurons, a critical step in treating neurodegenerative diseases.
In July 2016, BrainStorm announced positive results from its phase 2 randomized, placebo-controlled study of NurOwn in ALS patients, and presented the results at the 27th International Symposium on ALS/MND in Dublin, Ireland. That study mainly showed that NurOwn was safe and well tolerated. It also achieved multiple efficacy endpoints when given to 48 ALS patients, including the ability to halt or reverse disease progression over the course of a six-month follow-up.
Subsequently, BrainStorm received U.S. Food and Drug Administration (FDA) permission to start a Phase 3 trial. This will be a randomized, double-blind, placebo-controlled multi-dose trial conducted at multiple sites in the United States and Israel. Patient enrollment is expected to begin later this year.
“Following our end of Phase 2 meeting with the FDA, we are making final preparations to begin a Phase 3 clinical trial, which, when successful, will position us to bring to ALS patients and their families a much-needed and innovative ALS disease modifying therapy,” BrainStorm President and CEO Chaim Lebovits said in a press release.
The company plans to use cryopreservation to enable repeated doses of autologous NurOwn from a single bone marrow aspirate.
The FDA has already accepted key elements of the Phase 3 program to support a Biologic License Application (BLA) for NurOwn, which is a necessary step in commercializing the product.
Separately, BrainStorm announced plans to partner with City of Hope’s Center for Biomedicine and Genetics in the Los Angeles area to produce clinical supplies needed for its Phase 3 study. That study also involves Israel’s Tel Aviv Sourasky Medical Center.