BrainStorm Cell Withdraws Application for Approval of ALS Stem Cell Therapy as it Readies to Meet with FDA

Rare Daily Staff

BrainStorm Cell Therapeutics said that it is withdrawing its application with the U.S. Food and Drug Administration for approval of its NurOwn stem cell therapy for the rare neurodegenerative condition amyotrophic lateral sclerosis as it readies to meet the with the agency to discuss the path forward for the therapy.

BrainStorm said it is exploring the next steps in support of NurOwn, including publication of emerging clinical data and development of a protocol for an additional clinical study, which it said will be needed.

In November 2022, the FDA sent BrainStorm a refusal to file letter in response to its application seeking approval.

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis, and eventually, death. More than 90 percent of people with ALS have sporadic disease, showing no clear family history. ALS affects approximately 29,000 people in the U.S.

Brainstorm’s NurOwn technology platform represents a promising therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors (NTFs). Autologous MSC-NTF cells are designed to effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.

BrainStorm completed a phase 3 trial in 200 participants with ALS. In the attempt to examine a real-world population, the study enrolled people with more advanced disease than other late-stage ALS trials. In fact, more than a third of these participants with advanced disease entered the trial with one or more dimensions of physical function (e.g., dressing/hygiene, cutting food, walking) starting at the lowest possible score of 0 on the ALSFRS-R; thereby preventing the measurement of further deterioration.

A pre-specified subgroup of participants, which controls for this “scale effect” showed a trend to a meaningful increase in the clinical response with NurOwn compared to placebo. The secondary endpoint, average ALSFRS-R change from baseline to 28 weeks in this subgroup, was statistically significant. Biomarker data in all trial participants also showed consistent patterns of NurOwn reducing markers of inflammation and neurodegeneration, and increasing neuroprotective and anti-inflammatory markers relative to placebo, further supporting the notion that trial participants taking NurOwn are experiencing a positive biological effect.

“We are confident in the data supporting the value of NurOwn as an addition to the options for treating ALS, and we have every desire to bring it to the ALS community,” said Chaim Lebovits, president and CEO of BrainStorm. “We look forward to working with the FDA to define the path forward. We understand that an additional pivotal trial will be necessary, and we are doing everything in our power to execute on this as quickly as possible.”

Photo: Chaim Lebovits, president and CEO of BrainStorm