New England Journal of Medicine Publishes Positive Phase 3 VALOR Trial Results of Brepocitinib in Dermatomyositis
Rhea-AI Summary
Priovant Therapeutics (NASDAQ:ROIV) announced publication in the New England Journal of Medicine of positive Phase 3 VALOR results for brepocitinib 30 mg in dermatomyositis. VALOR enrolled 241 patients, met the primary endpoint with a 15.3-point TIS improvement versus placebo at Week 52 (P<0.001), and showed benefits across nine key secondary endpoints. The FDA granted Priority Review with a PDUFA target action date in Q3 2026. Safety signals included increased serious infections with brepocitinib 30 mg; many events resolved and most patients completed treatment.
Positive
- TIS +15.3 points vs placebo at Week 52 (P<0.001)
- 241 patients enrolled across 90 global sites
- FDA Priority Review granted; PDUFA target Q3 2026
- Rapid onset of effect: treatment effects as early as Week 4
- Steroid-sparing: nearly twice as many steroid reductions vs placebo
Negative
- Serious infections increased with brepocitinib 30 mg versus placebo
- Adverse events led to discontinuation more often in placebo group, complicating safety interpretation
- Baseline risks (neoplasm, cardiovascular factors) may affect event rates and trial generalizability
Key Figures
Previous Clinical trial Reports
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| Mar 03 | NDA priority review | Positive | -0.9% | FDA accepted brepocitinib NDA in dermatomyositis with Priority Review and Q3 2026 PDUFA. |
| Feb 06 | Phase 2 CS data | Positive | +22.1% | Positive Phase 2 BEACON results for brepocitinib in cutaneous sarcoidosis with strong efficacy signals. |
| Sep 17 | Phase 3 VALOR topline | Positive | +7.8% | Breakthrough Phase 3 VALOR data in dermatomyositis showing TIS superiority and steroid reduction. |
| Dec 03 | Namilumab Phase 2 fail | Negative | -2.9% | Kinevant’s Phase 2 RESOLVE-Lung study of namilumab failed primary and secondary endpoints. |
| Dec 03 | Roivant namilumab update | Negative | -2.9% | Roivant reported namilumab’s RESOLVE-Lung failure and discontinuation of development in sarcoidosis. |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Across recent clinical trial headlines, ROIV has generally moved in the same direction as the news tone, with four aligned reactions and one divergence.
Over the last 18 months, Roivant/Priovant have built a consistent clinical narrative. Positive Phase 3 VALOR results in dermatomyositis and subsequent NDA plans were followed by Phase 2 success for brepocitinib in cutaneous sarcoidosis, both drawing strong share reactions on some days. FDA Priority Review acceptance for brepocitinib in dermatomyositis and prior NAMilumab trial failure show that negative clinical readouts have produced modest declines. Today’s NEJM VALOR publication and additional skin-focused data extend this brepocitinib story without introducing new directional surprises relative to earlier disclosures.
Key Terms
phase 3 medical
new drug application (nda) regulatory
pdufa regulatory
total improvement score (tis) medical
placebo medical
corticosteroids medical
skindex-16 medical
AI-generated analysis. How Rhea-AI works. Not financial advice.
- The results of the Phase 3 VALOR trial were published in the New England Journal of Medicine, underscoring the practice-changing potential of brepocitinib 30 mg once-daily in dermatomyositis
- Brepocitinib 30 mg was superior to placebo on the primary and all nine key secondary endpoints, with statistically significant and clinically meaningful improvements observed across measures of global disease activity, muscle strength, skin disease, physical function, and corticosteroid reduction
- Additional analyses from VALOR, presented at the 2026 American Academy of Dermatology (AAD) Meeting, demonstrated meaningful improvements in itch and skin-related quality of life with brepocitinib 30 mg
- The U.S. Food and Drug Administration has granted Priority Review to brepocitinib’s New Drug Application (NDA) and assigned a Prescription Drug User Fee Act (PDUFA) target action date in the third quarter of 2026
DURHAM, N.C., March 28, 2026 (GLOBE NEWSWIRE) -- Priovant Therapeutics, a clinical-stage biotechnology company focused on developing targeted therapeutics in autoimmune disease, announced today the publication of results from the Phase 3 VALOR trial evaluating brepocitinib in adults with dermatomyositis (DM) in the New England Journal of Medicine (NEJM).
As reported in the NEJM publication, “A Phase 3 Trial of Brepocitinib in Dermatomyositis,” VALOR enrolled 241 patients across 90 sites globally and met its primary endpoint, with brepocitinib 30 mg achieving a 15.3-point greater improvement in mean Total Improvement Score (TIS) at Week 52 compared to placebo (P<0.001). This clinical improvement was observed alongside meaningful corticosteroid tapering in the brepocitinib treatment arms, with nearly twice as many patients reducing background corticosteroids in the brepocitinib 30 mg group compared to placebo. Brepocitinib 30 mg also demonstrated statistically significant and clinically meaningful improvements on all nine key secondary endpoints, with treatment effects evident as early as Week 4 and sustained through Week 52.
VALOR enrolled a broad, representative DM population that included patients with prior history of benign or malignant neoplasm and patients with multiple cardiovascular risk factors at baseline. 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. Adverse events leading to treatment discontinuation occurred more frequently with placebo, as did malignancies, cardiovascular events, and thromboembolic events. The NEJM authors hypothesize in the publication that the elevated rates of these events in the placebo group reflect the baseline risks associated with dermatomyositis itself and the immunosuppressive agents — particularly systemic steroids — commonly used in its treatment. They also note the importance of the potential dual benefit of brepocitinib seen in the trial’s efficacy data – namely, reducing disease activity while simultaneously enabling substantial reductions in systemic corticosteroid exposure.
“I anticipate that the VALOR trial results, which my collaborators and I were fortunate to publish in the New England Journal of Medicine, will be practice-changing for patients with dermatomyositis,” said Dr. Ruth Ann Vleugels, M.D., M.P.H., M.B.A., Heidi and Scott C. Schuster Distinguished Chair in Dermatology, Founding Director of the Autoimmune Skin Disease Center, Connective Tissue Disease Clinics, and Dermatology-Rheumatology Fellowship at Mass General Brigham, and Professor at Harvard Medical School. “These findings underscore the need to move beyond the historical paradigm of suboptimal disease control and reliance on systemic corticosteroids toward a patient-centric model focused on rapid, sustained, steroid-sparing efficacy with a modern, targeted therapy. Pending FDA approval, brepocitinib represents a monumental step forward in this direction, demonstrating impressive efficacy over 52 weeks across multiple measures of disease activity while also enabling meaningful corticosteroid reduction.”
The full publication entitled, “A Phase 3 Trial of Brepocitinib in Dermatomyositis,” appears in the New England Journal of Medicine.
Additional Skin-Focused Analyses Presented at the 2026 Annual American Academy of Dermatology (AAD) Meeting
Additional analyses presented during the late-breaking abstract session at AAD 2026 provide deeper insight into the skin-specific and patient-reported benefits of brepocitinib beyond those reported in NEJM. These data demonstrate rapid and statistically significant reductions in itch, with an
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).
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 suggests a safety profile similar to approved JAK and TYK2 inhibitors.
The U.S. FDA has granted Priority Review to brepocitinib’s New Drug Application (NDA) and assigned a Prescription Drug User Fee Act (PDUFA) target action date in the third quarter of 2026.
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 dual 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. The New Drug Application for brepocitinib in dermatomyositis is under review at FDA. Brepocitinib is also being evaluated in a Phase 3 program in non-infectious uveitis and recently generated positive Phase 2 data in cutaneous sarcoidosis, with a Phase 3 study to begin in calendar year 2026. Priovant Therapeutics is a Roivant (Nasdaq: ROIV) company.
About Roivant
Roivant (Nasdaq: ROIV) is a biopharmaceutical company that aims to improve the lives of patients by accelerating the development and commercialization of medicines that matter. Roivant’s pipeline includes brepocitinib, a potent small molecule inhibitor of TYK2 and JAK1 in development for the treatment of dermatomyositis, non-infectious uveitis and cutaneous sarcoidosis; IMVT-1402 and batoclimab, fully human monoclonal antibodies targeting FcRn in development across several IgG-mediated autoimmune indications; and mosliciguat, an inhaled sGC activator in development for pulmonary hypertension associated with interstitial lung disease. We advance our pipeline by creating nimble subsidiaries or “Vants” to develop and commercialize our medicines and technologies. Beyond therapeutics, Roivant also incubates discovery-stage companies and health technology startups complementary to its biopharmaceutical business. For more information, visit www.roivant.com.
Contact:
Stephanie Lee, stephanie.lee@priovant.com
