Phase 3 VALOR Trial Published in NEJM: Brepocitinib Delivers Landmark Results in Dermatomyositis

Dermatomyositis is a rare autoimmune disease that causes chronic inflammation of the muscles and skin, leading to progressive muscle weakness, distinctive skin rashes, and in many cases serious complications including difficulty swallowing, lung disease, and cardiovascular problems. For decades, treatment has depended heavily on high-dose systemic corticosteroids like prednisone, which manage inflammation but carry significant long-term side effects including weight gain, bone loss, diabetes, and increased infection risk. The absence of a drug specifically approved for dermatomyositis has long been a frustration for patients and clinicians alike. That is now poised to change.

In March 2026, the results of the Phase 3 VALOR trial were published in the New England Journal of Medicine by researchers at Priovant Therapeutics. The trial enrolled 241 patients with dermatomyositis across 90 research sites globally and is the first large-scale, randomized, placebo-controlled study to evaluate a precision oral therapy specifically designed for this disease. The drug being studied, brepocitinib, is a first-in-class oral inhibitor of TYK2 and JAK1, two signaling proteins that drive the immune system dysregulation at the heart of dermatomyositis. By blocking both simultaneously, brepocitinib suppresses multiple cytokine pathways that are elevated in the disease, including type I and type II interferons, IL-6, IL-12, and IL-23.

The results were striking across every measure. Brepocitinib 30 mg once daily achieved a 15.3-point greater improvement in the Total Improvement Score (TIS) at Week 52 compared to placebo, a statistically significant result (P less than 0.001). TIS is a composite outcome measure developed specifically for myositis research that combines muscle strength, physician-assessed disease activity, and patient-reported function. Brepocitinib was superior to placebo on the primary endpoint and on all nine key secondary endpoints, including measures of skin disease activity, physical disability, itch, and skin-related quality of life. Improvements were visible as early as Week 4 and were sustained throughout the full 52-week trial period.

One of the most clinically significant findings was the drug's corticosteroid-sparing effect. Nearly twice as many patients in the brepocitinib 30 mg group were able to meaningfully reduce their background corticosteroid use compared to those on placebo. For the many dermatomyositis patients already experiencing steroid-related complications, or those facing years of ongoing treatment, the possibility of reducing corticosteroid dependence is a major quality-of-life consideration. More than two-thirds of patients taking brepocitinib 30 mg achieved a TIS score of at least 40, twice the minimum clinically important threshold, and more than half achieved this while also reducing steroids to 2.5 mg per day or less. The FDA has granted Priority Review to brepocitinib's New Drug Application for dermatomyositis, with a target action date in Q3 2026. If approved, it would become the first drug ever specifically indicated for dermatomyositis, marking a historic shift in how this disease is treated.

Source: GlobeNewswire / Priovant Therapeutics, March 28, 2026. Vleugels RA, et al. A Phase 3 Trial of Brepocitinib in Dermatomyositis. New England Journal of Medicine Publishes Positive Phase 3 VALOR Trial Results. NEJM, 2026. DOI: 10.1056/NEJMoa2503531. This article is for informational purposes only and does not constitute medical advice. Consult your healthcare provider for guidance specific to your situation.