BrainStorm Cell Will Submit BLA to FDA after New Analysis of Trial Results

BrainStorm Cell Therapeutics, a developer of adult stem cell therapeutics for neurodegenerative diseases, said it would submit a Biologics License Application to the U.S. Food and Drug Administration for NurOwn for the treatment of amyotrophic lateral sclerosis (ALS) after new clinical analyses from its phase 3 trial despite an earlier notification from regulators that the phase 3 data did not support clinical benefit of the therapy.

Photo: Chaim Lebovits, CEO of BrainStorm Cell Therapeutics

“Brainstorm Cell Therapeutics is at a pivotal moment as a company as we finalize the regulatory filing for NurOwn in the treatment of ALS. The continued analysis and the feedback received from the many scientific presentations of NurOwn’s phase 3 data have uncovered key insights that furthered our understanding of the product mechanism of action and therapeutic potential and strengthened the conclusions of NurOwn’s efficacy,” said Chaim Lebovits, CEO of BrainStorm Cell Therapeutics. “After carefully considering these learnings, the totality of the evidence from NurOwn’s clinical studies, and the feedback received from key opinion leaders and the broader ALS community, we will submit a Biologics License Application to the FDA.”

In March 2021, the U.S. Food and Drug Administration, in response to a high-level data summary from the company’s ALS phase 3 clinical trial of its autologous stem cell therapy, said that the current level of clinical data does not provide the threshold of substantial evidence that the agency is seeking to support an application for approval. The agency, however, advised the company that its recommendation does not preclude Brainstorm from proceeding with an application submission seeking approval for the therapy.

BrainStorm said a correction was made to the Muscle and Nerve publication from December 2021 describing the results of NurOwn’s phase 3 clinical trial in ALS following new clinical analyses that strengthen the company’s original conclusions from the trial. The correction resulted in a statistically significant treatment difference of more than 2 points for an important secondary endpoint, average change from baseline in ALSFRS-R, in the pre-specified efficacy subgroup of participants with a baseline score of at least 35. Analyses reported in the original publication utilized an efficacy model that unintentionally deviated from the trial’s pre-specified statistical analysis plan by erroneously incorporating interaction terms between the subgroup and treatment. The newly published results, which include supporting information to the publication, employ the efficacy model as pre-specified in the trial’s statistical analysis plan, correcting the analyses. The correction also relates to the other subgroup analyses published for this endpoint, demonstrating that all subgroups with ALSFRS-R baseline scores of at least 26 to 35 showed a statistically significant benefit following treatment with NurOwn on this secondary endpoint. 

Amyotrophic lateral sclerosis (ALS) is a rare, fatal neurodegenerative disease that affects motor neurons (a type of nerve cell that controls voluntary movements) in the brain and spinal cord. When the nerve cells die, the brain can no longer initiate and control muscle movement, which results in severe disability, paralysis and eventually death. People with ALS may lose the ability to speak, eat, move and breathe. The rate of progression between individuals is variable and the natural history generally reflects progressive worsening over time until death occurs. The average life expectancy from symptom onset is between two and five years. Currently approved medications may slow disease progression, but ALS management is mostly supportive, palliative and symptom based.

Author: Rare Daily Staff