Brepocitinib shows groundbreaking results in dermatomyositis Phase 3 trial

A Phase 3 trial of brepocitinib has shown promising results for patients with dermatomyositis (DM). The VALOR study, published in the New England Journal of Medicine, found the drug significantly improved symptoms compared to placebo. The findings have led to an FDA Priority Review, with a decision expected by late 2026. The VALOR trial involved 241 adults with dermatomyositis across 90 global sites. After 52 weeks, patients on brepocitinib 30 mg saw a 15.3-point greater improvement in the Total Improvement Score (TIS) than those on placebo. This difference was statistically significant, with a P value of less than 0.001.

Brepocitinib is a first-in-class therapy that blocks TYK2 and JAK1, two enzymes linked to autoimmune inflammation. The drug also met all nine secondary endpoints, with benefits appearing as early as Week 4 and lasting through the full year. Nearly twice as many patients in the treatment group were able to reduce their corticosteroid use compared to placebo. However, serious infections occurred more often in the brepocitinib group. Most cases resolved with standard medical care. Dr. Ruth Ann Vleugels, a dermatology specialist, called the results 'potentially practice-changing,' noting brepocitinib's role as a steroid-sparing option. Priovant Therapeutics, a Roivant company focused on autoimmune diseases, leads the drug's development. The U.S. Food and Drug Administration has now granted Priority Review to brepocitinib's New Drug Application, setting a target decision date in the third quarter of 2026.

The VALOR trial's success positions brepocitinib as a potential new treatment for dermatomyositis. With FDA review underway, the drug could offer an alternative for patients who rely on long-term steroids. Final approval will determine its place in clinical practice.