Brepocitinib met the primary endpoint in the Phase 3 VALOR trial in dermatomyositis, showing sustained efficacy, steroid-sparing benefits, and improvements across key disease measures.
Written By: Samiksha Jadhav, BPharm
Reviewed By: Pharmacally Editorial Team
Priovant Therapeutics has reported positive results from the Phase 3 VALOR trial evaluating brepocitinib in adults with dermatomyositis (DM), with findings published in the New England Journal of Medicine. The study met its primary endpoint, demonstrating significant and sustained improvements in disease activity over one year.
Dermatomyositis (DM) is a rare autoimmune disease-causing muscle weakness and characteristic skin rashes such as heliotrope rash and Gottron’s papules. It may also involve systemic complications and is commonly treated with long-term corticosteroids, which carry safety concerns.
In the global VALOR trial (NCT05437263), brepocitinib 30 mg achieved a 15.3-point greater improvement in Total Improvement Score (TIS) at Week 52 compared with placebo (P<0.001). Clinical benefits were observed as early as Week 4 and sustained through the 52-week treatment period. The therapy also demonstrated statistically significant improvements across all nine key secondary endpoints, including muscle strength, skin disease activity, and functional outcomes.
Brepocitinib also enabled meaningful corticosteroid reduction, with nearly twice as many patients in the 30 mg group tapering background steroids compared to placebo. More than two-thirds of treated patients achieved a TIS40 response, and over half reached this threshold while reducing corticosteroid use to ≤2.5 mg/day, highlighting its steroid-sparing potential.
The VALOR study enrolled 241 patients across 90 global sites, including a broad population with baseline comorbidities such as prior malignancies and cardiovascular risk factors. While serious infections were more frequent in the brepocitinib 30 mg arm, these were manageable and treatment was generally completed. Notably, malignancies, cardiovascular events, and thromboembolic events occurred more often in the placebo group, reflecting underlying disease risks and corticosteroid exposure.
Additional analyses presented at the American Academy of Dermatology Annual Meeting 2026 highlighted significant skin-specific benefits. Brepocitinib demonstrated rapid reductions in itch, with an 18.9% higher rate of itch remission at Week 4 versus placebo. Improvements in skin-related quality of life were sustained over one year, and among patients with moderate-to-severe skin disease, the therapy achieved a 26.6% higher rate of functional skin remission compared to placebo.
Brepocitinib is an oral dual TYK2 and JAK1 inhibitor that targets key immune signaling pathways. By reducing pro-inflammatory cytokine activity, including type I interferons and interleukins, it helps control inflammation in both muscle and skin tissues in dermatomyositis.
Commenting on the findings, Ruth Ann Vleugels, Professor of Dermatology at Harvard Medical School, noted that brepocitinib could shift treatment toward a targeted, steroid-sparing approach with sustained efficacy and improved long-term disease control.
Brepocitinib’s safety profile across studies involving more than 2,000 patients appears consistent with other JAK and TYK2 inhibitors. Based on these data, the U.S. Food and Drug Administration has granted Priority Review to its New Drug Application, with a target action date in the third quarter of 2026.
Overall, the VALOR results position brepocitinib as a potential disease-modifying therapy in dermatomyositis, offering rapid and sustained efficacy alongside reduced reliance on systemic corticosteroids.
Reference
New England Journal of Medicine Publishes Positive Phase 3 VALOR Trial Results of Brepocitinib in Dermatomyositis, 28 March 2026, New England Journal of Medicine Publishes Positive Phase 3
Ruth Ann Vleugels et al, A Phase 3 Trial of Brepocitinib in Dermatomyositis, New Eng J Med, Published March 28, 2026, https://www.nejm.org/doi/full/10.1056/NEJMoa2503531
A Study to Investigate the Efficacy and Safety of Brepocitinib in Adults with Dermatomyositis (VALOR), ClinicalTrials.gov ID NCT05437263, https://clinicaltrials.gov/study/NCT05437263
About the Writer
Samiksha Vikram Jadhav is a B.Pharm graduate with a strong academic foundation in pharmaceutical sciences, pharmacology, and drug development. She has a keen interest in healthcare advancements, clinical research, medical writing, and emerging therapies. Her work focuses on presenting developments in the pharmaceutical and healthcare sectors through clear and accurate scientific communication.