- Brainstorm Cell Therapeutics successfully completed a phase 2 trial of NurOwn, a stem-cell therapy to treat amyotrophic lateral sclerosis (ALS). The trial involved 48 patients and achieved its primary endpoint of demonstrating the drug's safety and tolerability, in addition to secondary endpoints supporting some "clinically meaningful" benefits, the company reported Monday.
- ALS is a progressive neurodegenerative disease in which motor neurons die, eventually leaving the patient unable to walk, eat or breathe. In the U.S., 6,000 people are diagnosed with the fatal disease each year, although some patients may temporarily regain some neurological functions.
- The only approved drug for ALS is Riluzole, which is co-marketed by Sanofi and Martindale Pharmaeuticals. Riluzole was approved in 1995 and modestly decreases disease progression, giving patients the ability to retain function for a longer period of time, and delay death by several months.
In a release Monday, Brainstorm signaled its readiness to move into phase 3 with its stem-cell-based therapy for ALS.
"This study met its objectives, demonstrating both the safety of NurOwn and its ability to provide clinical benefit to ALS patients, and most importantly, will help us to determine the study population and design for a pivotal study of multiple doses of NurOwn in ALS," said Brainstorm CEO Chaim Lebovits
Thirty-six patients received the NurOwn injection, while 12 received a placebo. All patients had been assessed for three months prior to receiving treatment to establish a baseline and were measured at six points over six months following treatment for improvement, as measured by ALSFRS-R score and change in muscle strength and slow vital capacity.
Using a responder analysis, Brainstorm's analysis showed 74% of patients receiving treatment improved after two weeks, compared to 45% of those on placebo. This edge in percentage response persisted throughout the six months, except at 8 weeks where the placebo response percentage beat out that of the treatment arm.
No deaths or serious adverse events were reported in the study. But treatment-related adverse events occurred in nearly all actively treated patients (97.2% of treated patients compared to 75% on placebo). Many of these were considered minor, with the most common being injection site pain, fever, and headache.