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TOPLINE: 

In patients with dermatomyositis, once-daily oral brepocitinib 30 mg demonstrated significant improvement in the primary endpoint of multiple measures of disease activity over 52 weeks, compared with placebo. The drug enabled more patients to reduce or discontinue steroid use. 

METHODOLOGY:

• Brepocitinib, a dual selective inhibitor of TYK2 and JAK1, suppresses key cytokines associated with autoimmunity through dual inhibition.
• Researchers conducted a phase 3, multicenter, randomized controlled study of treatment with brepocitinib (VALOR trial), enrolling 241 adults with dermatomyositis (age, 18-75 years; BMI < 40) with active muscle and skin disease at screening and baseline.
• Participants were randomly assigned 1:1:1 to receive oral brepocitinib 30 mg, brepocitinib 15 mg, or placebo once daily for 1 year.
• Approximately 75% of patients were on background steroids, with mean baseline doses of 12.2 mg/d in the brepocitinib 30-mg arm and 11.3 mg/d in the placebo arm.
• The primary endpoint was the difference in mean total improvement score, a composite endpoint of multiple dermatomyositis disease activity measures, between the brepocitinib and placebo groups at week 52.

TAKEAWAY:

• Brepocitinib 30 mg achieved statistically significant improvement in the primary endpoint compared with placebo at week 52 (mean total improvement score, 46.5 vs 31.2; P = .0006), with significant differences observed as early as week 4.
• Among patients on background steroids, 62% of those in the brepocitinib 30-mg group vs 34% in the placebo group achieved a steroid dose ≤ 2.5 mg/d by the end of the study, while 42% of those on brepocitinib 30 mg vs 23% of those on placebo discontinued steroids completely.
• Among patients with moderate-to-severe skin disease, 44% of those receiving brepocitinib 30 mg achieved cutaneous clinical remission by week 52 compared with 21% of those receiving placebo. Significant improvements were observed in Cutaneous Dermatomyositis Area and Severity Index, motor strength, and Health Assessment Questionnaire Disability Index with brepocitinib compared with placebo.
• The safety profile aligned with previous brepocitinib trials, with adverse events of special interest not occurring more frequently in the brepocitinib 30-mg group than in the placebo group.

IN PRACTICE:

"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," Ruth Ann Vleugels, MD, MPH, MBA, Heidi and Scott C. Schuster Distinguished Chair in Dermatology, founding director of the Autoimmune Skin Disease Program and Connective Tissue Disease Clinics at Brigham and Women's Hospital, and program director for the Dermatology-Rheumatology Fellowship at Harvard Medical School, said in a press release.

SOURCE:

The study was conducted by Roivant and Priovant Therapeutics.

LIMITATIONS: 

The press release announcing the trial’s results did not discuss any limitations. 

DISCLOSURES:

This study was sponsored by Priovant Therapeutics, Inc.

This article was created using several editorial tools, including AI, as part of the process. Human editors reviewed this content before publication.