NurOwn was well tolerated in the trial with no clinically significant differences in safety assessments observed between treatment groups. Prespecified statistical modeling utilizing machine learning identified biomarkers that were predictive of treatment response with >80% accuracy. Large and statistically significant improvements from baseline were observed in cerebrospinal fluid biomarkers related to neuroinflammation, neurodegeneration, and neuroprotection in NurOwn treated patients, while biomarker levels in placebo patients were unchanged. 20.5% of placebo-treated participants (p=.053). In a post-hoc subgroup of participants with ALSFRS-R >25 at baseline (77% of participants), 34.7% of NurOwn-treated participants achieved response vs.
15.6% of placebo-treated participants (p=0.29). In the pre-specified subgroup of participants with ALSFRS-R ≥ 35 at baseline (31% of trial participants), 34.6% of NurOwn-treated participants achieved response vs. Subgroup analyses based on disease severity at baseline consistently show larger treatment effects in patients with less advanced disease. 27.7% p=0.45), which was not statistically significant. In the trial's overall population, the NurOwn group had a higher percentage of responders compared to the placebo group (32.6% vs. The inclusion of trial participants with more advanced ALS disease appears to have had a dilutive effect that may have reduced the ability to show a treatment effect in the overall study population. Participants enrolled in the trial had more advanced disease than in other late-stage ALS trials (average baseline ALSFRS-R = 31 23% of participants with baseline ALSFRS-R ≤ 25). Key data and conclusions from the paper include:īaseline characteristics between treatment groups were generally well-balanced indicating randomization was effective. We look forward to discussing these findings with regulators and remain fully committed to pursuing the best and most expeditious path forward to bring NurOwn to patients." "When viewed in totality, we believe the trial's data provide evidence of NurOwn's effectiveness in driving meaningful clinical benefit for ALS patients with less advanced disease.
"Having these data peer reviewed and published in such a prestigious journal is an important milestone in the NurOwn development program, providing us with the ability to allow full transparency into the trial results and gain external validation from a journal devoted to publishing the most important findings on neuromuscular disorders and treatment options," said Chaim Lebovits, Chief Executive Officer, Brainstorm Cell Therapeutics.
Understanding how to use biomarkers to tailor treatments, like NurOwn, is an essential new direction in ALS therapy development." "There are both biological and clinical signals of a treatment effect - as a community, we must review these carefully. Healey & AMG Center for ALS at Mass General Hospital, and the study's lead author. "I am thankful for all the participants, faculty and staff at the six trial sites who all worked together to ensure the quality and rigor of this study," said Merit Cudkowicz, MD, MSc, Chief of Neurology Massachusetts General Hospital, Julieanne Dorn Professor of Neurology Harvard Medical School, Director Sean M. Although previously announced results showed the trial did not reach statistical significance on the primary or secondary endpoints, pre-specified and post hoc analyses show a NurOwn-induced treatment effect across both primary and secondary efficacy outcomes in those with less advanced disease. Data from the paper are from a randomized, double-blind, placebo-controlled, Phase 3 trial evaluating the safety and efficacy of repeat doses of NurOwn® in study participants.