BrainStorm denied again in ALS as FDA issues Refuse-to-File Letter

Published: Nov 10, 2022

By Heather McKenzie

BioSpace

BrainStorm Cell Therapeutics ran into another stumbling block with ALS hopeful NurOwn Thursday as the FDA issued a Refusal to File Letter for its Biologics License Application.

For BrainStorm, the news must be eerily reminiscent of a day nearly two years ago when the regulator advised that the company’s Phase III trial did not meet the threshold of evidence to support a BLA.

BrainStorm spent the intervening period consulting with the FDA and ALS research community and further analyzing the data, and this summer announced it would seek a BLA.

The company hoped that a correction to analyses of the Phase III trial that resulted in a statistically significant treatment difference would buttress the therapy’s case.

The correction to the initial results, published in the journal Muscle and Nerve in December 2021, reflected a 2-point difference in a key secondary endpoint, average change from baseline on the ALSFRS-R scale. ALFSFRS-R is an established measure of disease progression where a top score of 48 equates to a healthy, fully functional individual.

The correction brought all subgroups with ALSFRS-R baseline scores of at least 26 to 35 to a place of statistical significance following treatment with NurOwn, BrainStorm said at the time.

In a statement Thursday, BrainStorm CEO Chaim Lebovits reaffirmed the company’s commitment to NurOwn’s advancement as a treatment for ALS. BrainStorm intends to request a Type A meeting with the FDA and “looks forward to continued discussions” with the regulator, Lebovits said.

In a statement, the trial’s co-primary investigators, Dr. Robert Brown, director of the program in neurotherapeutics at the University of Massachusetts Medical School, Dr. Merit Cudkowicz, chief of neurology at Mass Gen and Dr. Tony Windebank, professor of neurology at Mayo Clinic, said they continued to support discussions with the FDA on a path forward for NurOwn.

“While the pre-specified primary outcome measure was not met, there were participants with beneficial clinical effects and overall changes in relevant biomarkers of drug effect,” the investigators stated. 

While BrainStorm was not available to comment Thursday morning, the company noted it would outline its corporate strategy and plans to advance NurOwn at its third-quarter earnings call Monday morning. 

Source: BioSpace