Fmello:
It seems to me that any additional trials, including trials for MS, would need to focus on the “pre-specified group” and involve less severe patients. Also, is it possible that NurOwn may be useful as an adjunct therapy? I am not sure about any of this (maybe Midas is better suited to speak to all of this), but here is the discussion about the pre-specified group and less severe patients:
“In an important, pre-specified subgroup with early disease based on ALSFRS-R baseline score ³ 35, NurOwn demonstrated a clinically meaningful treatment response across the primary and key secondary endpoints and remained consistent with our pre-trial, data-derived assumptions. In this subgroup, there were 34.6% responders who met the primary endpoint definition on NurOwn and 15.6% on Placebo (p=0.288), and the average change from baseline to week 28 in ALSFRS-R total score was -1.77 on NurOwn and -3.78 on Placebo (p=0.198), an improvement of 2.01 ALSFRS-R points favoring NurOwn.
Cerebrospinal fluid (CSF) biomarker analyses confirmed that treatment with NurOwn resulted in a statistically significant increase of neurotrophic factors and reduction in neurodegenerative and neuroinflammatory biomarkers that was not observed in the placebo treatment group. We also carried out pre-specified statistical modeling designed to predict clinical response with high sensitivity and specificity based on ALS biomarkers and ALS Function and confirmed that NurOwn treatment outcomes could be predicted by baseline ALS function as well as key CSF neurodegenerative and neuroinflammatory biomarkers.
Dr. Merit Cudkowicz, one of the Principal Investigators of this trial and the Julianne Dorn Professor of Neurology at Harvard Medical School and the Director of the Healey Center for ALS and Chair of Neurology at Mass General Hospital said, "We found a clinically meaningful response to NurOwn in a pre-specified group of patients (greater than or equal to 35 ALSFRS-R at baseline). A change in pre- to post- treatment slope of 1.25 or more is substantial and clinically important. Given the heterogeneity of ALS, it is not surprising that measurement of treatment effect may be influenced by disease severity including the behavior of disease progression rates at the lower end of the scale. It is important to fully explore this finding. In addition, NurOwn was observed to have its clear intended biological effects with important changes in the pre-specified disease and drug related biomarkers."
"This clinical trial included a more severely affected ALS population compared to other recent ALS clinical trials. We identified a superior treatment response in a pre-specified subgroup of patients with less advanced disease.”
