JAMA Dermatology Publishes Skin-Specific Outcomes from Phase 3 VALOR Trial Of Brepocitinib in Dermatomyositis
- JAMA Dermatology publication includes results from VALOR skin-specific secondary endpoints, with rapid and durable improvements seen for brepocitinib 30 mg compared to placebo across multiple dimensions of cutaneous dermatomyositis (DM), including measurements of disease activity, itch, and skin-related quality of life
- In patients with at least moderate itch at baseline, clinically meaningful improvements were observed as early as Week 4 in 54% of brepocitinib 30 mg patients versus 10% with placebo, increasing to 74% versus 33%, respectively, by Week 52
- Nearly half of brepocitinib 30 mg treated patients with moderate-to-severe skin disease at baseline achieved remission-level outcomes by Week 52, with 46% demonstrating “Clear” or “Almost Clear” skin on the Investigators Global Assessment (IGA) and 44% achieving functional skin remission on the Cutaneous Dermatomyositis Activity and Severity Index – Activity Score (CDASI-A), more than two-fold higher than with placebo (22% and 21%, respectively)
- Results complement the primary efficacy and safety results from the VALOR trial previously published in the New England Journal of Medicine and reinforce brepocitinib’s potential as an important treatment for signs and symptoms of skin disease in dermatomyositis, regardless of muscle involvement
DURHAM, N.C., Aug. 26, 2026 (GLOBE NEWSWIRE) -- Priovant Therapeutics announced today the publication in JAMA Dermatology of skin-specific outcomes from the Phase 3 VALOR trial evaluating brepocitinib, a first-in-class oral TYK2 and JAK1 inhibitor, in adults with dermatomyositis (DM). Primary efficacy and safety results from the trial were previously published in the New England Journal of Medicine, including benefit on measures of skin disease, muscle strength, physical function, and steroid-sparing.
“Skin disease is a major and often underappreciated driver of morbidity in dermatomyositis, with an impact on quality of life that exceeds most other inflammatory skin diseases,” said Victoria P. Werth, MD, Professor of Dermatology and Medicine at the Perelman School of Medicine at the University of Pennsylvania, Chief of the Division of Dermatology at the Philadelphia Veterans Administration Hospital, and one of the lead investigators of the Phase 3 VALOR Trial. “The rapid and sustained improvements in cutaneous disease activity and itch seen in the VALOR trial, together with the achievement of functional skin remission for many patients with moderate-to-severe skin disease at baseline, represent a monumental finding for patients with dermatomyositis. These results are particularly meaningful given how difficult cutaneous dermatomyositis manifestations and symptoms have historically been to control with conventional therapies.”
In the analyses published in JAMA Dermatology, brepocitinib 30 mg produced rapid, durable and clinically meaningful improvements across multiple dimensions of cutaneous dermatomyositis, including skin disease activity, itch and skin-related quality of life. Treatment effects were evident as early as Week 4 and sustained through Week 52, with significantly more brepocitinib-treated patients achieving clinically meaningful improvements in skin disease activity and itch, as well as remission-level skin outcomes, compared with placebo. The table below summarizes the results published in JAMA Dermatology:
| Brepocitinib 30 mg | Placebo | Delta (95% CI) | |
| Disease Activity1 | |||
| Achievement of Clinically Meaningful CDASI-A Response (≥40% Improvement and ≥ 4-Point Improvement) at Week 52 | 61.7% | 44.3% | 16.8% (1.1–32.5, P=0.04) |
| Remission2 | |||
| Achievement of Gold Standard ≥ 2-category improvement on IGA to “Clear” / “Almost Clear” Skin at Week 52 | 45.7% | 21.8% | 21.1% (2.5 to 39.7) |
| Achievement of Functional Skin Remission (CDASI-A ≤ 5) at Week 52 | 43.5% | 20.8% | 26.6% (7.6 to 45.5) |
| Itch3 | |||
| Achievement of Clinically Meaningful Itch Reduction (≥ 2-point improvement in PP-NRS) by Week 4 | 54.0% | 9.5% | 47.3% (30.4 to 64.1) |
| Achievement of Clinically Meaningful Itch Reduction (≥ 2-point Improvement in PP-NRS) by Week 52 | 74.0% | 33.3% | 39.8% (18.9-60.6) |
| Skin-Related QoL1 | |||
| Improvement in Skindex-164 by Week 4 | 12.9 | 0.9 | 11.9 (6.0 to 17.9) |
1Among all participants
2Among participants with at least moderate skin disease at baseline
3Among participants with at least moderate itch at baseline
4Minimal clinically important difference defined as 10 units of improvement
Abbreviations: CDASI-A, Cutaneous Dermatomyositis Disease Area and Severity Index - Activity; CDA-IGA, Cutaneous Dermatomyositis Activity-Investigator’s Global Assessment; PP-NRS, Peak Pruritus-Numerical Rating Scale; Skindex-16, skin-related quality of life
Improvements in skin disease occurred alongside reductions in oral corticosteroid (OCS) use. Among patients receiving OCS at baseline, 61.7% of patients treated with brepocitinib 30 mg tapered to 2.5 mg/day (prednisone-equivalent) or less by Week 52 compared to 34.4% receiving placebo, while 41.7% discontinued OCS altogether compared with 23.4% receiving placebo. These findings support brepocitinib’s potential to deliver meaningful control of skin disease alongside substantial tapering of OCS, an important treatment goal in DM given the cumulative toxicity associated with systemic corticosteroid use.
As previously published in the New England Journal of Medicine, the VALOR trial enrolled a broad, representative DM population including patients with prior history of benign or malignant neoplasm and patients with multiple cardiovascular risk factors. Serious infections in the study were increased in brepocitinib 30 mg compared to placebo; these events resolved with medical management, and brepocitinib treatment was completed in most cases. New or recurrent malignancy, cardiovascular events, and thromboembolic events in the study occurred more frequently in the placebo arm than the brepocitinib 30 mg arm. The brepocitinib safety database across all studies includes over 2,000 patients and subjects and supports a safety profile consistent with the known safety profile of JAK inhibitors.
About the Phase 3 VALOR Study
The VALOR study was a global Phase 3 trial that enrolled 241 subjects with dermatomyositis across 90 sites. Subjects were randomized 1:1:1 to brepocitinib 30 mg, brepocitinib 15 mg, and placebo. Brepocitinib 30 mg demonstrated statistically significant and clinically meaningful improvement compared to placebo on the primary endpoint of Total Improvement Score (TIS) at Week 52. TIS is a composite endpoint of six core set measures of myositis disease activity. Benefit compared to placebo was seen as early as Week 4 and sustained at every visit thereafter through the end of the one-year double-blind treatment period. Brepocitinib 30 mg also demonstrated statistically significant and clinically meaningful improvement compared to placebo on all nine key secondary endpoints evaluated, including measures of muscle strength, skin disease activity, functional disability, and steroid tapering. More than two thirds of brepocitinib 30 mg patients achieved a Total Improvement Score of at least 40 (TIS40), twice the minimum clinically important difference. More than half achieved this TIS40 threshold while also reducing systemic corticosteroid use to ≤2.5 mg/day (prednisone-equivalent). Brepocitinib exhibited a safety profile consistent with the known safety profile of JAK inhibitors, with no new safety signals identified.
About Priovant
Priovant Therapeutics is a biotechnology company dedicated to developing novel therapies for autoimmune diseases with high morbidity and few available treatment options. The company's lead asset is brepocitinib, a first-in-class, selective inhibitor of TYK2 and JAK1. Through selective TYK2/JAK1 inhibition, brepocitinib distinctively suppresses key cytokines linked to autoimmunity—including type I IFN, type II IFN, IL-6, IL-12 and IL-23—with a single, targeted, once-daily oral therapy. Brepocitinib recently generated positive Phase 3 data in dermatomyositis. Brepocitinib is also being evaluated in a Phase 3 program in non-infectious uveitis, a Phase 3 program in cutaneous sarcoidosis, and a Phase 2b/3 program in lichen planopilaris. Priovant Therapeutics is a Roivant (Nasdaq: ROIV) company.
Contacts:
Stephanie Lee: stephanie.lee@priovant.com
