Incyte (Nasdaq:INCY) today announced that data from the pivotal Phase 3 TRuE-V clinical trial program evaluating ruxolitinib cream (Opzelura™) 1.5% in patients 12 years of age and older with nonsegmental vitiligo have been published in The New England Journal of Medicine (NEJM). In the trials (TRuE-V1 and TRuE-V2), application of ruxolitinib cream resulted in significant facial and total body repigmentation versus vehicle-control as shown by greater proportions of patients reaching the facial and total body Vitiligo Area Scoring Index (F-VASI and T-VASI, respectively) endpoints at Week 24 vs. vehicle, with a higher proportion of patients responding at Week 521.
“Incyte is committed to developing innovative medicines for people with immune-mediated dermatologic conditions, and the publication of the TRuE-V results in NEJM is the culmination of years of work to advance science in vitiligo where there were no approved pharmacologic treatments for repigmentation,” said Jim Lee, M.D., Ph.D., Group Vice President, Inflammation & Autoimmunity, Incyte. “These pivotal results demonstrate significant improvements in facial and total body repigmentation with ruxolitinib cream, and we are proud that these data served as the foundation for the recent approval of ruxolitinib cream as the first and only U.S. Food and Drug Administration (FDA)-approved treatment for repigmentation in nonsegmental vitiligo.”
Results from the Phase 3 TRuE-V studies vehicle-controlled period (24 weeks) were previously announced, and Week 52 data were featured in an oral presentation at the late-breaking abstract session at the American Academy of Dermatology (AAD) Annual 2022 Meeting. Key findings from the studies include:
- Results at Week 24, which were consistent across both studies, showed that approximately 30% of patients treated with ruxolitinib cream achieved ≥75% improvement from baseline in F-VASI (F-VASI75), the primary endpoint, while approximately 13% of patients applying vehicle in one study achieved the primary endpoint. At Week 52, approximately 50% of patients who received ruxolitinib cream from Day 1 achieved F-VASI75.
- Additionally, at Week 24, more than 15% of patients treated with ruxolitinib cream achieved ≥90% improvement from baseline in F-VASI (F-VASI90) vs. approximately 2% of patients applying vehicle met F-VASI90. At Week 52, the percentage of ruxolitinib cream-treated patients who achieved F-VASI90 doubled to approximately 30%.
- A greater proportion of patients at Week 52 achieved ≥50% improvement in T-VASI (T-VASI50), and further improvement on percentage change from baseline in facial body surface area (F-BSA) with application of ruxolitinib cream was also observed.
In the vehicle-controlled period of the Phase 3 studies, the most common adverse reactions (incidence ≥ 1%) were application site acne, application site pruritus, nasopharyngitis, headache, urinary tract infection, application site erythema, and pyrexia2.
“The primary and key secondary analyses from the TRuE-V program demonstrate the improvement in repigmentation possible with ruxolitinib cream,” said David Rosmarin, M.D., Vice Chair of Research and Education, Department of Dermatology at Tufts Medical Center. “As a physician, this is exciting. Vitiligo has historically been challenging to treat, and these data reinforce the potential that this new medical treatment can have for people who are interested in repigmentation.”
Vitiligo is a chronic autoimmune disease characterized by depigmentation of skin that results from the loss of pigment-producing cells known as melanocytes. Overactivity of the JAK signaling pathway is believed to drive inflammation involved in the pathogenesis and progression of vitiligo. In the United States, more than 1.5 million people are diagnosed with vitiligo3. The overall prevalence of the condition is estimated to be approximately 2-3 million4, with the majority of patients (approximately 85%) suffering from nonsegmental vitiligo5. Vitiligo can occur at any age, although many patients with vitiligo will experience initial onset before the age of 306.
In July 2022, Opzelura (ruxolitinib) cream was approved by the FDA for the topical treatment of nonsegmental vitiligo in adult and pediatric patients 12 years of age and older. Previously, in 2021, Opzelura was approved by the FDA for the topical short-term and non-continuous chronic treatment of mild to moderate atopic dermatitis (AD) in non-immunocompromised patients 12 years of age and older whose disease is not adequately controlled with topical prescription therapies, or when those therapies are not advisable.
The NEJM publication of the TRuE-V results is available online.