BrainStorm’s ALS-combatting drug finally on its way to the FDA
pharmafile | August 16, 2022 | News story | Manufacturing and Production |
BrainStorm Cell Therapeutics’ NurOne was rejected by the Food and Drug Association in February 2021 but this year, after consulting with the regulator and the ALS community and analysing the data, they are ready to submit a Biologics License Application (BLA).
Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease characterised by selective deterioration of cortical, brainstem, and spinal cord motor neurons.
NurOne is a type of engineered stem cell therapy which delivers autologous MSC-NTF cells that can ‘effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to … ultimately slow or stabilise disease progression’. The MSC-NTF cells are produced by harvesting the patient’s bone marrow-derived MSCs, and differentiating them to secrete high levels of NTFs.
The issues with the original publication were that the initial data had an unsatisfactory placebo effect of 27.7%, and the analyses used an efficacy model which unintentionally deviated from the trial’s pre-specified statistical analysis plan.
However, the company looks to be confident moving forward after an update to the December publication of Phase III trial results in Muscle and Nerve. The correction shows a significant treatment difference of 2 points in a key secondary endpoint on the ALSFRS-R scale. Two points or more on the ALSFRS-R scale in a 28-week period is “a clinically very important difference,” Stacy Lindborg, Ph.D., EVP and Chief Development Officer at BrainStorm, said.
The ALSFRS-R scale is the validated measure of disease progression in ALS, with a score of 48 meaning a healthy individual. The correction brings all subgroups with ALSFRS-R baseline scores of at least 26-35 to a place of statistical significance following treatment with NurOne.
Dr Merit Cudkowicz, MD, Chief of the Department of Neurology at Massachusetts General Hospital and an investigator on the Phase III said: “The pre-specified secondary analyses are statistically significant for people earlier in illness. This doesn’t change the primary analysis (which was not statistically significant) but suggests that there may be better response in people with milder illness.”
BrainStorm must now wait and see if the FDA accepts their BLA. If accepted, they will receive a designated date for review.
James Spargo
