New England Journal of Medicine Publishes VALOR Phase 3 Brepocitinib Results in Dermatomyositis

The New England Journal of Medicine published Phase 3 VALOR results showing brepocitinib 30 mg improved mean Total Improvement Score by 15.3 points versus placebo at Week 52; the U.S. FDA has granted Priority Review with a PDUFA target date in the third quarter of 2026.

The New England Journal of Medicine published results from the Phase 3 VALOR trial of brepocitinib in adults with dermatomyositis. The trial enrolled 241 patients across 90 sites and compared brepocitinib 30 mg, brepocitinib 15 mg, and placebo over 52 weeks. The sponsor, Priovant Therapeutics, reported that the trial met its primary endpoint and presented additional skin-focused analyses at the 2026 AAD meeting. The U.S. Food and Drug Administration has granted Priority Review to brepocitinib's New Drug Application with a PDUFA target action date in the third quarter of 2026. Key findings: - Brepocitinib 30 mg achieved a 15.3-point greater improvement in mean Total Improvement Score (TIS) at Week 52 versus placebo (P<0.001). - Treatment effects were reported as early as Week 4 and were sustained through Week 52 across multiple measures, including muscle strength and skin activity. - Nearly twice as many patients in the brepocitinib 30 mg group reduced background systemic corticosteroids compared with placebo, and more than half who reached TIS40 also reduced steroid use to ≤2.5 mg/day (prednisone-equivalent). - Serious infections were reported more frequently with brepocitinib 30 mg than with placebo; these events were reported to have resolved with medical management and brepocitinib treatment was completed in most cases. - Additional analyses presented at AAD 2026 showed faster reductions in itch and improvements in skin-related quality of life measures with brepocitinib 30 mg. - The trial enrolled a broad dermatomyositis population, including patients with prior neoplasm history and multiple cardiovascular risk factors at baseline. Summary: The NEJM publication reports that the VALOR Phase 3 trial met its primary endpoint and showed sustained clinical effects through one year, alongside reductions in systemic corticosteroid use. Safety findings included an increase in serious infections with brepocitinib 30 mg, while some other adverse events were observed more often in the placebo arm; the authors note contextual factors such as baseline disease risks and concurrent immunosuppressive treatments. The next regulatory milestone is the FDA PDUFA target action date in the third quarter of 2026.