Ublituximab outperforms teriflunomide on ARRs, CNM-Au8 fails HEALEY ALS trial, benefits of aerobic exercise in multiple sclerosis


Welcome to this special edition of Neurology News Network. I am Marco Meglio.

Data from ULTIMATE I and II, a recent pair of phase 3 trials, showed that ublituximab, in the experimental treatment developed by TG Therapeutics, resulted in lower annualized relapse rates (ARR) and less brain damage on MRI than teriflunomide in patients with relapsing multiple sclerosis (MS) over 96 weeks. The ARR was 0.08 with ublituximab and 0.19 with teriflunomide in the ULTIMATE I trial, while the ARR was 0.09 and 0.18 in the ULTIMATE II. Ublituximab produces B-cell depletion and improves antibody-dependent cell cytolysis, which could be a beneficial treatment for patients with relapsing MS. The results further showed a significantly lower risk of disability worsening with ublituximab treatment. However, the experimental treatment was also associated with infusion-related reactions, which occurred in 47.7% of participants in the ublituximab group. One of the limitations of conducting the trials is that no inferences could be made regarding the effectiveness of ublituximab compared to other MS treatments more potent than teriflunomide.

Key results from the pivotal HEALEY ALS trial, the first-ever platform trial for ALS, demonstrated that CNM-Au8 failed to meet its primary and secondary endpoints; however, the therapy showed survival benefit in exploratory analyses. In total, over a period of 24 weeks, treatment with the investigational suspension of gold nanocrystals resulted in a 2% slowing of disease progression, as demonstrated by scores on the Revised Functional Rating Scale of L ALS (ALSFRS-R) adjusted for mortality. Pre-specified survival analyzes showed a significant survival benefit for people in the CNM-Au8 30 mg group, which the company says warrants continued development. No survival benefit was observed for the 60 mg group of CNM-Au8, the larger of the 2 doses evaluated. The news comes less than a week after another ALS hope, Biohaven’s enzyme inhibitor verdiperstat, also failed to statistically differentiate from placebo in HEALEY ALS.

Data from the randomized, controlled Early Multiple Sclerosis Exercise, or EMSES, study suggests that early supervised aerobic exercise positively affects the microstructural integrity of important motor pathways and nuclei in patients with multiple sclerosis. Notably, however, the supervised exercise regimen did not reduce relapse rates or global brain atrophy — the study’s primary outcome measures — but the researchers, including Ulrik Dalgas, PhD, MSc, associate professor of Public Health, University of Aarhus, noted that “exercise is a promising non-pharmacological approach,” and at this point the window of opportunity for an intervention that is suspected to exist early in the evolutionary of the disease has been largely unstudied. In particular, they wrote, “potential additional disease-modifying and neuroprotective treatment strategies are warranted in multiple sclerosis”. MRIs showed that microstructural integrity was higher in 50% of a priori defined motor pathways and nuclei in the exercise group (n=84) compared to the control group from the Danish population-based MS registry (n = 850) at 48 weeks groins.

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Richard V. Johnson