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Upadacitinib: A novel treatment option for vitiligo

Presented by
Prof. Thierry Passeron, Nice University Hospital, France
AAD 2024
Phase 2
In a phase 2 trial, the JAK1 inhibitor upadacitinib led to a fast improvement in repigmentation in patients with non-segmental vitiligo. After 52 weeks, participants showed continuous further pigmentation in both facial and body areas.

Previous research has shown that disruption of IFN-γ signalling by inhibiting the JAK/STAT pathway is an attractive therapeutic target for vitiligo [1,2]. Thus, the objective of the current phase 2 dose-ranging study (NCT04927975) was to evaluate the efficacy and safety of the JAK1 inhibitor upadacitinib for the treatment of adults with non-segmental vitiligo, a disease with only limited treatment options [3]. Prof. Thierry Passeron (Nice University Hospital, France) presented the results.

The 184 participants were randomised to placebo or upadacitinib at either 6 mg, 11 mg, or 22 mg once daily for 24 weeks. The primary endpoint was the percentage change from baseline in the Facial Vitiligo Area Scoring Index (F-VASI) at week 24. After the double-blind period at week 24, participants on placebo were switched to either 11 mg or 22 mg upadacitinib and continuously treated until week 52.

The study met its primary endpoint with a significantly greater percentage change with the 2 highest upadacitinib doses (i.e. 11 mg and 22 mg). F-VASI was reduced by -34% in the 22 mg group (P<0.05 vs placebo) and -35.6% in the 11 mg group (P<0.01 vs placebo), compared with -14.4% in the placebo group (see Figure). Significant reductions were also noted for the change in total VASI (T-VASI).

Figure: Primary endpoint of percentage change from baseline in F-VASI at week 24 (MMRM) [3]

BL, baseline; CI, confidence interval; F-VASI, Facial Vitiligo Area Scoring Index; LS, least squares; MMRM, mixed-effects model for repeated measures; PBO, placebo; UPA, upadacitinib.

Moreover, participants treated with upadacitinib experienced continued improvement in F-VASI through week 52. At this time, participants achieved an approximately 60% to 65% reduction in F-VASI with 11 mg and 22 mg upadacitinib, respectively. Similar improvements in F-VASI were observed in participants who switched at week 24 from placebo to upadacitinib 11 mg or 22 mg. T-VASI values also improved up to week 52.

The safety assessment disclosed similar rates of treatment-emergent adverse events between upadacitinib and placebo groups with the most frequently reported treatment-emergent adverse events being a COVID-19 infection, acne, headache, and nasopharyngitis.

Relevant reading:

    1. Qi F, et al. Front Immunol 2021;12:790125.
    2. Boniface K, et al. Front Immunol 2021;12:613056.
    3. Passeron T. Efficacy and safety after 52 weeks of once-daily upadacitinib in adults with extensive non-segmental vitiligo (NSV): final results from a phase 2, multicenter, randomized, double-blind, placebo-controlled, dose-ranging study. LB1, 2024 AAD Annual Meeting, 08–12 March, San Diego, USA.

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