Roivant and Priovant Therapeutics announced groundbreaking results from the Phase 3 VALOR study of brepocitinib in dermatomyositis (DM), marking the first-ever positive outcome for a 52-week placebo-controlled trial in this debilitating autoimmune disease. The once-daily oral therapy achieved statistically significant and clinically meaningful improvements across all primary and secondary endpoints, establishing a new potential standard of care for approximately 50,000 adults affected by DM in the United States.
Unprecedented Clinical Efficacy
The VALOR study, the longest and largest interventional DM study ever conducted with 241 subjects globally, demonstrated robust efficacy for brepocitinib 30 mg. On the primary endpoint, patients achieved a week 52 mean Total Improvement Score (TIS) of 46.5 compared to 31.2 for placebo (p=0.0006), representing a statistically significant difference that was sustained at all time points from week 4 through one year.
More than two-thirds of brepocitinib 30 mg patients experienced at least a moderate response (TIS≥40), while nearly half achieved a major response (TIS≥60). Among patients entering the trial with moderate-to-severe skin disease, 44% on brepocitinib 30 mg achieved cutaneous clinical remission by week 52, compared to only 21% on placebo.
"Dermatomyositis is an incredibly debilitating autoimmune disease for patients, and we urgently need novel, approved efficacious therapies," said Dr. Ruth Ann Vleugels, M.D., M.P.H., M.B.A., Heidi and Scott C. Schuster Distinguished Chair in Dermatology at Brigham and Women's Hospital. "The VALOR study's success represents a groundbreaking moment for the dermatomyositis field, and the results reinforce brepocitinib's potential to serve as a deeply impactful treatment option for a substantial number of dermatomyositis patients once approved."
Significant Steroid-Sparing Benefits
A key clinical advantage demonstrated in VALOR was brepocitinib's ability to reduce corticosteroid dependence. Approximately 75% of patients entered the study on background steroids, with mean baseline doses of 12.2 mg/day in the brepocitinib 30 mg arm and 11.3 mg/day in the placebo arm. Despite this steroid tapering challenge, 62% of brepocitinib 30 mg patients achieved a steroid dose ≤2.5 mg/day by study end compared to 34% for placebo, while 42% were able to discontinue steroids altogether versus 23% on placebo.
The drug demonstrated clinically meaningful improvements across multiple disease domains, including skin disease (CDASI), motor strength (MMT-8), and daily living activities (HAQ-Disability Index). The median time to achieve a TIS≥40 response was approximately 8 weeks, highlighting the therapy's rapid onset of action.
Novel Dual Inhibition Mechanism
Brepocitinib's therapeutic profile stems from its unique dual selective inhibition of TYK2 and JAK1, which distinctively suppresses key cytokines linked to autoimmunity including type I interferon, type II interferon, IL-6, IL-12, and IL-23. This targeted approach addresses the complex pathophysiology of dermatomyositis through a single, once-daily oral therapy.
The observed safety profile was consistent with previous brepocitinib clinical trials, with adverse events of special interest—including malignancy, cardiovascular events, and thromboembolic events—occurring no more frequently in the brepocitinib 30 mg arm than the placebo arm.
Addressing Critical Unmet Need
Dermatomyositis is a multi-organ idiopathic inflammatory condition characterized by debilitating muscle weakness and skin lesions that significantly impair daily living activities such as walking up stairs, carrying groceries, and getting dressed. The dermatomyositis rashes often affect large portions of a patient's body including the scalp and are frequently disfiguring and painful. Currently, no targeted therapies are approved for DM, and a majority of patients require high-dose chronic oral corticosteroids.
"We are thrilled with the results of the VALOR study, and I would like to thank all of the patients, investigators, and study site staff who contributed to this important research achievement," said Ben Zimmer, Priovant CEO. "We are excited to continue working towards the rapid approval of brepocitinib in dermatomyositis and our broader goal of developing brepocitinib as a transformational therapy for multiple highly morbid autoimmune diseases where the need for novel efficacious therapies is greatest."
Regulatory Timeline and Broader Pipeline
Priovant plans to file an NDA for brepocitinib in dermatomyositis in the first half of 2026. Beyond DM, brepocitinib is also being evaluated in non-infectious uveitis (Phase 3) and cutaneous sarcoidosis (Phase 2), potentially expanding its therapeutic applications across multiple autoimmune conditions.
"The VALOR study is a hallmark example of what Roivant does best: identify a high value program with a differentiated mechanism of action, focus on creative development plans in indications like DM with high unmet need, and deliver on clinical execution excellence," said Mayukh Sukhatme, Roivant President and Chief Investment Officer. "I am extremely proud of Roivant's continued track record with VALOR as the 12th consecutive positive Phase 3 study for the company."
The VALOR results position brepocitinib as a potential first-in-class targeted therapy for dermatomyositis, offering hope for patients who have long relied on broad immunosuppressive treatments with significant side effect profiles.
