Efgartigimod Meets Primary Endpoint, Neurologic Outcomes not Improved with EryDex, Elevidys Associated with Stabilization in DMD

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Welcome to the Neurology News Network. My name is Louie Pasculli. Here’s a look at this week’s top stories in neurology.

Beginning with some topline results, data from a phase 3 study dubbed ADAPT OCULUS showed that efgartigimod met its primary end point, demonstrating significant clinical improvements in adults with ocular myasthenia gravis (oMG). Based on these findings, argenx is expected to submit a biologics license application to expand efgartigimod’s label into oMG. ADAPT OCULUS, a phase 3, double-blind, placebo-controlled, parallel-group, registrational trial, featured 141 patients with oMG, using change in Myasthenia Gravis Impairment Index (MGII) ocular score at week 4 as the primary end point. After 4 weeks of treatment, patients on the approved medication demonstrated statistically significant improvement on this primary end point relative to placebo. Additional data from the study is expected to be presented at an upcoming meeting. Additional data from the release showed that efgartigimod-treated patients had a 4.04-point mean improvement in MGII patient-reported outcome whereas those on placebo showed changes of 1.99. Notably, those treated with the approved therapy also had marked reductions in key ocular symptoms, such as diplopia and ptosis. “Ocular myasthenia gravis significantly impacts patients’ daily lives, affecting vision, independence and the ability to do routine tasks, such as work or drive a car. Yet today, there are no approved targeted medicines for this disease,” Carolina Barnett-Tapia, MD, PhD, associate professor of medicine at the University of Toronto, said in a statement. “The improvements observed with VYVGART in the OCULUS trial offer hope to the thousands of myasthenia gravis patients with ocular involvement.”

Staying on the topic of topline results, the phase 3 NEAT trial showed that dexamethasone sodium phosphate encapsulated in autologous erythrocytes (EryDex;Quince Therapeutics) did not significantly improve neurologic outcomes compared with placebo in children and adolescents with ataxia-telangiectasia (A-T) over 6 months of treatment. Although the investigational therapy was generally well tolerated, the findings underscored the ongoing challenges in identifying effective disease-modifying treatments for A-T.1 The NEAT study was an international, multicenter, randomized, double-blind, placebo-controlled phase 3 trial that enrolled 105 participants with genetically confirmed A-T across sites in the US, UK, and Europe. Participants were randomized 1:1 to receive either EryDex or placebo infusions administered every 21 to 30 days for a total of six infusions over approximately 6 months. The primary efficacy endpoint was change from baseline to month 6 in the Rescored modified International Cooperative Ataxia Rating Scale (RmICARS), compared with placebo. The RmICARS is a refined version of the International Cooperative Ataxia Rating Scale (ICARS), a clinician-administered measure of ataxia severity that places greater emphasis on posture and gait and has been used in pediatric A-T populations.¹

Concluding with more phase 3 data, results from the 2-year EMBARK clinical trial suggest that treatment with delandistrogene moxeparvovec (marketed as Elevidys) was associated with stabilization or slowing of functional decline in ambulatory children with Duchenne muscular dystrophy (DMD), compared with a matched external control cohort receiving standard-of-care corticosteroids. Improvements in motor function measures including the North Star Ambulatory Assessment (NSAA), time to rise (TTR), and 10-meter walk/run (10MWR) were observed over 104 weeks, supporting the durability of the microdystrophin gene therapy approach. EMBARK is a phase 3, randomized, placebo-controlled crossover study evaluating Elevidys in ambulatory boys aged 4 to younger than 8 years with DMD. The therapy uses a recombinant adeno-associated virus serotype rh74 vector to deliver a microdystrophin gene to skeletal muscle. At 2 years, treated patients demonstrated statistically significant differences versus external controls across several functional outcomes. Mean change in NSAA score from baseline was +2.63 points in the gene therapy group compared with −0.25 points in the external control cohort. Improvements were also observed in TTR and 10MWR.

For more direct access to expert insight, head to NeurologyLive.com. Be sure to tune in next week to stay up to date on the latest in neurology. I’m Louie Pasculli and thanks for watching Neurology News network.