Investigational Nonsteroidal Edaslonexent Treatment for Duchenne Muscular Dystrophy

Treatment of boys with Duchenne Muscular Dystrophy (DMD), age 4 to 7 years and never been exposed to steroids (n=31), led to growth patterns that were similar to children without DMD. Although the boys' growth rates were age normative, weight increase was less than the mean for boys at this age, resulting in overall normalized body mass index. In addition, treatment with the nonsteroidal edaslonexent was well tolerated and had no negative effects on bone health or adrenal function. The most common treatment-related

Disease progression as measured by the North Star Ambulatory Assessment, 4-stair climb, 10-meter walk or run, and time to stand from seated position, were all stable over a 72-week period. This a time frame during which boys with untreated DMD have steady declines in these motor tests. Edasalonexent has the potential to be disease-modifying in DMD patients and in this phase 2 trial did not have the adverse effects associated with high-dose steroids. An ongoing phase 3 trial of edasalonexent (NCT03703882) is fully enrolled and is further assessing safety and efficacy in young boys with DMD.

No serious treatment-related adverse events occurred and the majority of adverse events that did occur were gastrointestinal. No participants discontinued treatment. No low-impact fractures occurred during the study.

Edasalonexent, an oral inhibitor of nuclear factor kappa-light-chain-enhancer of activated B cells (NF-κB), is being investigated as a potential foundational therapy for DMD patients regardless of mutation. Importantly, as a nonsteroidal agent, edasalonexent does not affect the glucocorticoid receptor.

These data from the open-label extension of the MoveDMD trial (NCT02439216) were presented during the online presentation of the Muscular Dystrophy Association Clinical and Scientific Congress on March 24, 2020 in lieu of the annual meeting that was canceled due to the 2020 COVID-19 pandemic.