The US Food and Drug Administration (FDA) has granted clearance to BrainStorm Cell Therapeutics to commence a Phase IIIb trial of its NurOwn therapy for treating amyotrophic lateral sclerosis (ALS).
This development follows a special protocol assessment agreement, confirming that the trial's goals and statistical approach align with requirements for an upcoming biologics licence application (BLA).
The upcoming Phase IIIb trial will recruit approximately 200 subjects and comprises a 24-week double-blind, placebo-controlled, randomised phase, followed by an open-label extension for the same period.
Its primary goal is the change in ALS Functional Rating Scale-Revised (ALSFRS-R) scores from baseline to week 24.
This study also includes a comprehensive screening period of nine weeks, with a two-visit process leading to randomisation into the therapy and placebo groups, and subsequent bone marrow aspiration for all subjects.
According to the company, subjects will undergo a process where stem cells from their bone marrow are isolated and cryopreserved.
Before each intrathecal dose is administered, cells are said to be thawed, propagated, and induced into MSC-NTF cells (NurOwn).
In addition, the trial offers subjects the option to contribute to future biomarker and genetic research by providing blood, cerebrospinal fluid, and buccal samples.
The NurOwn platform uses autologous MSCs to produce NTF-secreting cells, aiming to modulate neuroinflammation and promote neuroprotection in ALS individuals.
The therapy has secured orphan drug designation from both the US regulator and the European Medicines Agency.
Beyond ALS, BrainStorm has also explored MSC-NTF cells in progressive multiple sclerosis, supported by a grant from the National Multiple Sclerosis Society in the US.
BrainStorm Cell Therapeutics CEO and president Chaim Lebovits said: "We are now positioned to swiftly activate clinical sites in a phased manner, diligently preparing to enrol the first patient in the Phase IIIb 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."
