BrainStorm Cell Therapeutics is hauling itself off the mat for another run at amyotrophic lateral sclerosis (ALS). A piece of the latest comeback strategy slotted into place Tuesday when the biotech revealed a written agreement with the FDA on the design of a phase 3b trial.
When the FDA reaches a special protocol assessment agreement, it indicates the agency is aligned with the sponsor on the adequacy and acceptability of specific critical elements of protocol design such as the dose selection and endpoints. The FDA may still disagree with the sponsor on some protocol details.
BrainStorm said its agreement with the FDA validates the clinical trial protocol and statistical analysis of a planned phase 3b trial of its ALS cell therapy candidate NurOwn. BrainStorm CEO Chaim Lebovits added in a statement that the company believes the agreement “will help de-risk certain regulatory aspects of the NurOwn clinical program.”
The biotech is planning to enroll people who have developed ALS symptoms, including limb weakness, in the past 24 months in a two-part trial. Part one is a double-blind, placebo-controlled study that will track up to 200 patients for 24 weeks. The primary endpoint will look at changes in a disease-specific severity score, ALSFRS-R. The second part of the study is a 24-week open-label extension.
BrainStorm expects to start the phase 3b trial this year. First, the biotech said it needs to review the protocol with investigators, secure institutional review board approvals and “engage with appropriate members of the ALS community.”
The to-do list makes no mention of money. BrainStorm ended last year with $1.45 million. The biotech has previously named securing non-dilutive grants and cashing in on its exosome technology as options for how to fund the phase 3b trial.
The company is running the study to address failings identified in its most recent filing for approval, with FDA advisers pointing in September to the absence of evidence that NurOwn stymies the progression of mild-to-moderate ALS. The advisers offered their opinion days after the FDA savaged the filing in a briefing document.
BrainStorm, which had previously suffered two FDA rejections, responded by pulling the application and laying off 30% of its staff. The ever-optimistic biotech framed the layoffs as a way to accelerate its journey back to the FDA.