Topical Ruxolitinib Improves Vitiligo Severity in Phase 2 Study

Data from a phase 2 study demonstrated that topical ruxolitinib cream significantly improved repigmentation of facial lesions in adults with vitiligo.

Topical ruxolitinib cream significantly improved repigmentation of facial lesions in adult patients with vitiligo, according to results from a phase 2 study.

Vitiligo is a chronic autoimmune condition characterized by the progressive loss of pigmentation in patches of skin, which occurs when the immune system attacks the body’s pigment cells. The disease can range from mild to severe and typically affects exposed areas of the skin, such as the face and hands. Overactivity of the Janus kinase (JAK) signaling pathway has been shown to drive inflammation involved in the pathogenesis and progression of vitiligo, according to Incyte.

Ruxolitinib cream, which has been formulated for topical application, is also used as an oral treatment for certain blood disorders. Current treatment methods for vitiligo, including phototherapy, corticosteroids, and calcineurin inhibitors, have limited efficacy.

The phase 2 study program evaluating topical ruxolitinib for vitiligo began in April 2017 and is comprised of 3 parts spanning 104 weeks. The first part of the study, which spanned 24 weeks, included 157 adults diagnosed with vitiligo and with depigmented areas of at least 0.5% of the body surface area (BSA) on the face and at least 3% of the total BSA on nonfacial areas.

For the study, patients were randomized across 5 treatment arms: ruxolitinib cream 1.5%, 0.5%, or 0.15% administered once daily (QD); ruxolitinib cream 1.5% administered twice daily (BID); or vehicle control for 24 weeks. The primary efficacy endpoint at week 24 was the percentage of patients treated with ruxolitinib cream who achieved facial vitiligo area severity index (F-VASI50) score compared with patients treated with vehicle control.

According to the data, significantly more patients achieved F-VASI50 after 24 weeks of treatment with all-ruxolitinib cream regimens compared with the vehicle control group. Patients treated with ruxolitinib cream 1.5% QD and BID achieved the highest F-VASI50 response compared with the vehicle control (50% and 45% versus 3%, respectively; P<0.001).

Additionally, a ≥75% improvement from baseline in the facial vitiligo area severity index score was achieved by 17%, 30%, and 0 patients treated with ruxolitinib cream 1.5% QD, BID, and vehicle cream, respectively. Facial Physician Global Vitiligo Assessment scores of clear or almost clear skin were achieved by 13%, 9%, and 0 patients receiving ruxolitinib cream 1.5% QD, BID, and vehicle cream, respectively.

Overall, the adverse effects of ruxolitinib cream were mild and included redness and irritation at the application site and mild acne, according to the study.

“Topical ruxolitinib has the potential to change the way vitiligo is treated,” lead study author David Rosmarin, MD, dermatologist at Tufts Medical Center, said in a press release about the study. “Not only is it effective at repigmenting the skin, but it has an excellent safety profile. We also are optimistic that many vitiligo patients may see an even better response with continuous ruxolitinib usage over an extended period of time, combined with phototherapy and sunlight exposure. Our hope is that this treatment ultimately will be a game-changer for the millions of people worldwide affected by vitiligo.”

The positive 24-week data were presented at the World Congress of Dermatology. Ruxolitinib cream is also currently in phase 3 development for the treatment of mild-to-moderate atopic dermatitis and is expected to enter phase 3 for vitiligo in the second half of 2019.

References

Incyte Announces Positive Results from a Phase 2 Study of Ruxolitinib Cream in Patients with Vitiligo [news release]. Incyte. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-positive-results-phase-2-study-ruxolitinib. Accessed June 17, 2019.

Topical cream for vitiligo [news release]. Tufts Medical Center. https://www.tuftsmedicalcenter.org/News-Events-Media/Press-Releases/2019/Topical-cream-for-vitiligo. Accessed June 17, 2019.