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Topical JAK1/JAK2 inhibitor effective in vitiligo

Presented by
Dr David Rosmarin, Tufts Medical Center Boston, USA
EADV 2021
Phase 3, TRuE-V1; TruE-V2
Ruxolitinib cream led to a 75% re-pigmentation in almost a third of patients in the phase 3 TRuE-V1 and TruE-V2 trials. The topical Janus kinase (JAK)1/JAK2 inhibitor also led to improvement of vitiligo in difficult-to-treat areas such as the hands and was well tolerated.

“Despite a vitiligo prevalence of up to 1% of the world´s population, up to now, there are no approved treatments,” said Dr David Rosmarin (Tufts Medical Center Boston, MA, USA) [1]. In a phase 2 trial, a cream formulation of ruxolitinib, a JAK1/JAK2 inhibitor, demonstrated substantial re-pigmentation [2]. In the 2 current, 52-week, randomised, double-blind, phase 3 trials, the efficacy and safety of this treatment was assessed in adolescent (≥12 years) and adult patients with non-segmental vitiligo in centres in the United States and Europe [1]. Dr Rosmarin presented the 24-week results.

In the TRuE-V1 (NCT04052425) and TRuE-V2 (NCT04057573) studies, a total of 674 participants were randomly assigned to twice-daily treatment with a vehicle cream or a cream containing 1.5% ruxolitinib. The primary endpoint was the proportion of patients achieving ≥75% improvement in the Vitiligo Area Scoring Index in the face (F-VASI). After 24 weeks, patients could continue in a 28-week treatment extension phase. Baseline characteristics were similar for both studies.

At week 24, a 75% improvement in F-VASI was achieved by a significantly greater proportion of participants applying ruxolitinib cream versus vehicle. In the TRuE-V1 trial, 29.9% of participants treated with ruxolitinib compared with 7.5% treated with the vehicle achieved this endpoint. The corresponding numbers in the TRuE-V2 trial were 29.9% and 12.9%, respectively. Significant results were also observed for an improvement by 50% and by 90% at week 24.

“All primary and secondary endpoints were met. The results were similar to the ones we have seen in our phase 2 study,” Dr Rosmarin commented. Ruxolitinib also showed good results in challenging-to-treat areas like the hands. “Ruxolitinib cream does not just improve the face but works on the whole body as well,” Dr Rosmarin said. Improvements were also noticed in patient-reported outcomes.

Ruxolitinib was well tolerated. No clinically significant application site reactions or serious treatment-emergent adverse events were reported; the most common treatment-emergent adverse events were acne and pruritus at the application site. “Important to know is that the plasma concentration with this local treatment was well below half the inhibitory concentration (IC50) for JAK2-mediated changes in the bone marrow,” Dr Rosmarin explained. There were also no clinically significant changes in haemoglobin or platelet levels.

“Re-pigmentation can take time. Thus, I look forward to the 52-week results, which will hopefully show an even improved response from the week 24 results,” Dr Rosmarin said.

    1. Rosmarin D. Efficacy and safety of ruxolitinib cream for the treatment of vitiligo: 24-week results from 2 randomized, double-blind phase 3 studies. D3T01.2A, EADV Congress 2021, 29 Sept–2 Oct.
    2. Rosmarin D, et al. Lancet. 2020;396:110-20.


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