BrainStorm gets FDA green light for phase 3 trial of ALS cell therapy

BrainStorm Cell Therapeutics announced that the U.S. FDA has cleared the company to initiate its phase 3b clinical trial of its autologous MSC-NTF cell therapy, NurOwn, for the treatment of amyotrophic lateral sclerosis (ALS).

The clearance allows the company to proceed with patient enrollment. The trial design was agreed upon with the FDA last year under a Special Protocol Assessment, confirming the study's endpoints and statistical methodology are appropriate to support a future BLA submission.

"We are now positioned to swiftly activate clinical sites in a phased manner, diligently preparing to enroll the first patient in the phase 3b trial. At the same time, we are actively working to secure funding through multiple avenues, including non-dilutive grants, to ensure the timely and successful launch of this critical study," said Chaim Lebovits, BrainStorm president and CEO.

The company's proprietary NurOwn platform uses autologous mesenchymal stem cells (MSCs) to produce neurotrophic factor-secreting cells (MSC-NTF cells), designed to deliver targeted biological signals that modulate neuroinflammation and promote neuroprotection. The therapy has received orphan drug designation from both the U.S. FDA and the EMA.

But it’s been a long regulatory path for NurOwn. In February 2021, after reviewing data from a phase 3 ALS trial, senior leadership at the FDA advised BrainStorm that its clinical data did not meet the threshold of substantial evidence to support a BLA. In November 2022, the agency proceeded to reject NurOwn's BLA, citing phase 3 trial outcomes. BrainStorm submitted an amendment addressing the agency’s concerns and requested fast-tracking through a process known as ‘file over protest’ to secure an AdComm meeting. Almost a year later, in October 2023, BrainStorm opted to pull its BLA, cutting back resources and laying off 30% of its staff after an FDA AdComm voted 17-1 against approving NurOwn, noting concerns over the company’s data and its inability to demonstrate how they would safely manufacture the treatment.