BrainStorm Cell Therapeutics announced that it has reached an agreement with the FDA on key elements of the phase 3b trial design for the company's ALS treatment hopeful, NurOwn.
According to BrainStorm, the company received written agreement from the FDA, under a Special Protocol Assessment (SPA), on the trial design. The SPA indicated that the trial adequately addresses the objectives needed to support a future BLA.
It's good news for NurOwn, which has stumbled more than once along the regulatory path. Back in October, BrainStorm opted to pull its BLA, cutting back resources and laying off 30% of its staff after an FDA advisory committee voted 17-1 against approving NurOwn the month prior.
The AdComm had expressed concerns regarding study findings and BrainStorm's capability to safely manufacture NurOwn which involves utilizing a patient's own stem cells. NurOwn uses autologous mesenchymal stem cells (MSC) cells to secrete neurotrophic factors targeting ALS, and while it showed promise for those with less advanced ALS, it fell short of statistical significance in its phase 3 pivotal trial.
The FDA had previously rejected NurOwn's BLA in November 2022, due to its phase 3 trial outcomes. The company had submitted an amendment addressing their concerns and requested fast-tracking through a process known as "File Over Protest" to secure an AdComm meeting.
Now it seems the company is back on track with a plan to launch a two-part, multicenter, study designed to assess the efficacy and safety of NurOwn in patients with ALS this year.
The ALS treatment landscape doesn't offer many options for the approximately 30,000 ALS patients in the U.S. Last week, Amylyx announced that it will discontinue its controversial ALS treatment, Relyvrio, after phase 3 trial results failed to demonstrate the drug’s efficacy.
