BrainStorm Cell Therapeutics Inc (BCLI)

[Fmello]

BrainStorm Cell Therapeutics Submits Type A Meeting Request to U.S. Food and Drug Administration

https://seekingalpha.com/pr/19051717-brainstorm-cell-therapeutics-submits-type-meeting-request-to-u-s-food-and-drug-administration

Meeting expected to occur within 30 days of the FDA's receipt of the meeting request

NEW YORK, Dec. 12, 2022 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that the company has submitted a Type A Meeting Request to the U.S. Food and Drug Administration (FDA) to discuss the contents of a refusal to file letter previously issued by the FDA regarding the company's New Biologics License Application (BLA) for NurOwn® for the treatment of ALS. The Type A Meeting is expected to occur within 30 days of the FDA's receipt of the meeting request.

As previously reported, the contents of the refusal to file letter focus on topics related to chemistry, manufacturing, and controls (CMC), as well as clinical data and statistics. As part of the Type A Meeting, BrainStorm intends to discuss a path to an FDA Advisory Committee Meeting.

"Participating in a Type A meeting will be an important next step towards enabling NurOwn's advancement through the regulatory process," said Chaim Lebovits, Chief Executive Officer of BrainStorm. "The extensive briefing package submitted with our request contains a comprehensive strategy to fully address the CMC matters raised in the refusal to file letter. We anticipate achieving quick alignment with the FDA on the CMC strategy and expect that its execution will be straightforward. We therefore anticipate a Type A meeting focused primarily on discussing how we can secure an Advisory Committee Meeting, which we believe will be a critical step on NurOwn's path towards approval as an ALS therapy."

About the Phase 3 Trial of NurOwn in ALS

BrainStorm previously completed a Phase 3 trial in approximately 200 participants with ALS (Cudkowicz et al., 2022 Muscle and Nerve). In an 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, with baseline ALSFRS-R ≥35, 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 (p=0.050, Muscle and Nerve Supplemental File and Muscle and Nerve Erratum). In addition, post-hoc sensitivity analyses were presented in November 2022 (21st Annual NEALS Meeting 2022) which also showed a statistical trend towards a clinically meaningful treatment effect with NurOwn across subgroups, and one that is consistent with the pre-specified subgroup of participants with less advanced ALS at baseline. Finally, 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 indeed experiencing a positive biological effect (ALS ONE Research Symposia 2022).