BrainStorm Cell Therapeutics has latched on to the recent accelerated FDA approval of Biogen and Ionis’ amyotrophic lateral sclerosis (ALS) drug to make the case that its cell therapy could join the rival product on the U.S. market.
As BrainStorm reported previously, the autologous cell therapy, NurOwn, was statistically no better at causing clinical responses than placebo in a phase 3 clinical trial. BrainStorm pushed past the setback and two rejections by the FDA to secure a third shot at the regulatory process earlier this year. The FDA has scheduled an advisory committee to discuss the application for September.
Ahead of the meeting, BrainStorm has used the 2023 ALS and Related Motor Neuron Diseases Gordon Research Conference to share a different look at the phase 3 data. The analysis was motivated by the accelerated approval of Biogen and Ionis’ antisense drug Qalsody in ALS.
With the FDA conditionally approving Qalsody on the strength of clinical evidence that it reduces plasma neurofilament light (NfL), BrainStorm has dug into its phase 3 data to assess the effect of NurOwn on the biomarker of nerve injury and neurodegeneration. The phase 3 investigators previously looked at the effect of NurOwn on NfL and other biomarkers in a paper published last year.
The new analysis linked treatment with NurOwn to increases in neuroprotection markers and reductions in neuroinflammation and neurodegeneration markers. Reductions in NfL correlated to better clinical outcomes.
“Time from first symptom to treatment and baseline physical function are important to understanding the treatment effect in clinical trials given the great heterogeneity in the disease, which can influence prognosis,” BrainStorm co-CEO Stacy Lindborg, Ph.D., said in the release. “The data we presented at the Gordon Research Conference show that it is equally important to examine biomarker data, particularly neurofilament light, which is a predictor of disease progression.”