Initial Phase 4 RESPOND Results, Omaveloxolone Continues to Perform in FA, Wearable Sensors Safe for FSHD

Neurology News Network for the week ending March 19, 2022.

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

At the 2022 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, March 13-16, in Nashville, Tennessee, new data from the phase 4 RESPOND study showed no adverse events (AEs) or serious AEs occurred in children with spinal muscular atrophy (SMA) treated with nusinersen. The first patient was treated in January 2021, and the main goal of the trial is to understand if the proven efficacy of nusinersen and its mechanism of action, which leads to continuous production of survival motor neuron protein, may also benefit patients who have been insufficiently treated with gene therapy. The initial safety data showed that 4 participants had a total of 7 infection-related events. They included ear infection, viral gastroenteritis, parainfluenza virus infection, pneumonia, upper respiratory tract infection (URTI), and viral URTI. Two participants reported vomiting as well. Serious AEs, which included parainfluenza virus infection (2 events in 1 participant) and URTI, were unrelated to treatment with nusinersen. No deaths or post-lumbar puncture syndrome events occurred.

Findings from the delayed-start period of the MOXIe Extension study support positive primary end point findings from the pivotal MOXIe Part 2 trial, as data is consistent with the persistent effect of omaveloxolone (Reata Pharmaceuticals) treatment on disease course in patients with Friedrich ataxia. All patients in the full analysis set who completed MOXIePart 2 enrolled in the open-label, delayed-start period (MOXIe Extension). Both patients and investigators remained blinded to prior patient treatment assignments, and MOXIe Extension included 42 patients who had previously been randomized to receive placebo and 50 patients previously randomized to receive omaveloxolone. When comparing modified FA Rating Scale (mFARS) scores between both groups, investigators found that the difference at the end of the placebo-controlled MOXIe Part 2 study, which was –2.25 points (SD, 1.07; P = .037),was preserved at the conclusion of the delayed-start period, at –3.51 points.

The use of wearable sensors to measure functional performance in patients with facioscapulohumeral muscular dystrophy (FSHD) is feasible and reliable, with investigators further concluding that data correlated with multiple clinical outcome assessments, activities of daily living, and mobility. Findings from the assessment may provide data on disease progression and treatment efficacy in future clinical trials in FSHD.There was a 99% compliance rate for wearing the devices, and monitoring averaged 2626 hours per patient, with all participants monitored for a total of 2941 days, or 36,758 hours. There were multiple parameters calculated from patients’ upper and lower extremities, which were found to be reliable (ICC > .90), and investigators noted that analysis of correlations with clinical assessments would be reported.

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