For its promising investigational therapeutic approach to neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), BrainStorm Cell Therapeutics is the Buzz of BIO 2020 winner in the Public Therapeutic Biotech category.
The Buzz of BIO contest identifies U.S. companies with groundbreaking, early-stage potential to improve lives. The event also is an opportunity to make investor connections that could take products to the next phase.
Ten biotechnology companies are nominated in each of the three categories of Buzz of BIO: Public Therapeutic Biotech, Private Therapeutic Biotech, and Diagnostics and Beyond. In the Public Therapeutic Biotech category that BrainStorm won, nominated companies must be actively developing a publicly traded human treatment intended for review by the U.S. Food and Drug Administration (FDA).
As a developer of autologous cellular therapies — treatments that use a patient’s own cells and tissues — for debilitating neurodegenerative diseases, BrainStorm is now testing its NurOwn therapy for safety and effectiveness. The treatment involves extracting, from human bone, marrow-derived mesenchymal stem cells (MSCs), which are capable of differentiating into other cell types. The MSCs are then matured into a specific cell type that produces neurotrophic factors — compounds that promote nervous tissue growth and survival. They are then reintroduced to the body via injection into muscles and/or the spinal canal.
Backed by a California Institute for Regenerative Medicine grant, Brainstorm has fully enrolled its randomized, double-blind, placebo-controlled Phase 3 clinical trial (NCT03280056) at six U.S. sites in California, Massachusetts, and Minnesota. Some 200 ALS patients are participating. A secondary safety analysis by the trial’s independent Data Safety Monitoring Board (DSMB) revealed no new concerns. Every two months, study subjects will be given three injections into the spinal canal of either NurOwn or placebo.
The trial is expected to conclude late this year. Results will be announced shortly afterward.
The use of autologous MSC cells to potentially treat ALS was given orphan drug status by both the FDA and the European Medicines Agency.
“Thanks to everyone who voted for BrainStorm during the Buzz of BIO competition,” Chaim Lebovits, BrainStorm president and CEO, said in a press release. “The entire management team at BrainStorm was very pleased with the results of this competition, and we look forward to presenting to an audience of accredited investors who may benefit from the company’s story. We thank the BIO [Biotechnology Innovation Organization] team for singling out BrainStorm’s NurOwn as a key technology with the potential to improve lives.”
As a contest winner, BrainStorm is invited to give a presentation at the Bio CEO & Investor Conference, to be held Feb. 10–11 in New York City, along with exposure to multiple industry elites and potential investors.
NurOwn cells also are being tested in a Phase 2 clinical study (NCT03799718) in patients with progressive multiple sclerosis.