- 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.
Vitiligo is a chronic autoimmune disease of the skin that causes patches of skin depigmentation, often in exposed areas like the face and the hands. “Despite its prevalence of up to 1% of the world´s population, up to now, there are no approved treatments,” Dr David Rosmarin (Tufts Medical Center Boston, MA, USA) explained1.
A cream formulation of ruxolitinib, a JAK1/JAK2 inhibitor, demonstrated substantial re-pigmentation in a phase 2 trial. Thus, the efficacy and safety of this treatment was assessed in adolescent (≥12 years) and adult patients with non-segmental vitiligo in 2 ongoing 52-week, randomised, double-blind phase 3 trials conducted in centres in the United States and Europe.
In the TRuE-V1 (NCT04052425) and TRuE-V2 (NCT04057573) studies, patients 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). Also assessed were several secondary endpoints and safety and tolerability. Dr Rosmarin presented the results of the double-blind phases. After 24 weeks, patients can continue in a 28-weeks treatment extension phase.
Baseline characteristics were similar for both studies, most participants had skin phototype II, III, or IV. 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 29.99% 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-related adverse events (TEAE) were reported; the most common TEAEs 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 results, which will hopefully show an even improved response from the week 24 results,” Dr Rosmarin concluded.