BrainStorm to Meet With FDA on Confirmatory Trial for NurOwn ALS Therapy


After a negative AdComm meeting and withdrawing its BLA, the company seeks guidance on a phase 3 trial.

BrainStorm Cell Therapeutics is meeting with the FDA to discuss a possible regulatory path forward for its NurOwn cell therapy for the potential treatment of amyotrophic lateral sclerosis (ALS) on December 6, 2023.1

Brainstorm is seeking approval of its overall protocol design for a confirmatory Phase 3 trial of NurOwn and will discuss plans for a Special Protocol Assessment (SPA) with the FDA.

"We are pleased that the FDA has granted this expedited in-person meeting to discuss the best path forward for NurOwn for ALS," Chaim Lebovits, president and chief executive officer, BrainStorm, said in a statement.1 "Our proposed plan is to conduct a confirmatory Phase 3b trial and it is important that we are aligned with the Agency on the expected requirements for re-submitting a Biologics License Application (BLA). We believe that reaching an agreement through a SPA on the overall protocol design and the adequacy to address the requirements for marketing approval will be a key step to position the company for success and to potentially de-risk the program. We are grateful for the FDA's support and quick response in granting this meeting as we remain committed to our goal of making NurOwn available to the ALS community."

NurOwn, also known as debamestrocel is an investigational autologous mesenchymal stem cell (MSC) neurotrophic factor–secreting cell therapy product. The therapy previously had a Prescription Drug User Fee Act action date of December 8, 2023 before BrainStorm pulled its BLA from consideration after an FDA Cellular, Tissue, and Gene Therapies Advisory Committee voted against its approval.2,3

READ MORE: New FDA Pilot Program Will Provide Hands-On Regulatory Guidance for Cell and Gene Therapy Trials in Rare Disease

“It's possible that this therapy has some benefit for some patients,” committee member Lynn A. Raymond, MD, PhD, the director of the Djavad Mowafaghian Centre for Brain Health, said after voting no.“But we look at the total when we have to decide whether this goes forward to public market and there wasn't evidence for the whole group that this was effective and there was evidence to potentially suggest it was actually deleterious at least for those who are maybe more advanced with ALS, causing more deaths and causing more bulbar dysfunction. So for that reason, I vote no...”

A prior phase 3 trial of NurOwn had previously failed to meet its primary endpoint in 2021 of responder rates on ALSFRS-R scores in all stages of disease progression, but a newer erratum later revealed that analyses that adhered more closely to prespecified sub-analyses showed statistically significant improvements (= .050) of more than 2 points on ALSFRS-R scores in participants with a baseline score of at least 35.4 The newer analyses prompted BrainStorm to seek approval for the therapy despite receiving a refusal to file letter from the FDA.

1. BrainStorm Cell Therapeutics announces in-person meeting with the FDA to discuss confirmatory phase 3 trial for NurOwn® in ALS. News release. BrainStorm. November 20, 2023.
2. BrainStorm cell therapeutics to meet with US FDA to discuss development plan for NurOwn as a treatment of ALS. News release. BrainStorm Cell Therapeutics Inc. October 18, 2023. Accessed October 20, 2023.
3. 74th Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC) Meeting. September 27, 2023.
4. BrainStorm Cell Therapeutics announces second quarter 2022 financial results and provides a corporate update. News release. BrainStorm Cell Therapeutics. August 15, 2022.
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