Results ‘promising’ in phase 2 study of ruxolitinib cream for patients with vitiligo

David Rosmarin

Around half of patients with vitiligo treated with ruxolitinib cream achieved a 50% improvement in facial vitiligo area severity index scores at week 24, meeting the primary endpoint in a phase 2 study, David Rosmarin, MD, primary investigator of the study, said in an interview with Healio Dermatology.

The study results were announced at the World Congress of Dermatology in Milan.

“We see a response in as early as 8 weeks in most of the [ruxolitinib cream] treatment groups,” said Rosmarin, an assistant professor at Tufts University School of Medicine in Boston.

This is the largest vehicle-controlled, double-blind, randomized controlled trial completed in patients with vitiligo, he said.

He added that oral ruxolitinib (Jakafi, Incyte) is FDA approved for indications such as polycythemia vera, myelofibrosis and acute graft-versus-host disease. The topical formulation is not yet approved.

The study enrolled 157 adult patients (mean age: 48.9 years) with vitiligo with depigmented areas of 0.5% or greater of body surface area (BSA) on the face and 3% or greater of BSA on non-facial areas; 46.5% of patients were male and 84.1% were white.

Patients were randomly assigned to receive ruxolitinib cream 1.5% twice daily, 1.5% once daily, 0.5% once daily or 0.15% once daily or vehicle cream for 24 weeks.

At baseline, the mean percentage of total BSA involvement was 22.05%, and mean facial involvement was 1.48%. The mean baseline total Vitiligo Area Severity Index (VASI) score was 17.95, and facial-VASI (F-VASI) was 1.26, according to the study abstract.

A 75% improvement in F-VASI scores was found in 30% of patients who received ruxolitinib 1.5% twice a day and 17% who received ruxolitinib 1.5% once a day.

“As this is a topical medication and not an oral, we avoid the systemic side effects patients are subjected to,” Rosmarin said. “The medication was well tolerated in all treatment arms. There may be a slight signal for application site reactions and for acne, but overall, it is very well tolerated.”

Currently, there is no FDA-approved treatment for repigmenting the disease, Rosmarin said.

“[Our results] are very promising, but there are still some unanswered questions, [such as], will the medication work better with light treatment?” Rosmarin said.

These results encompass the first part of the phase 2 study, and results on treatment at 52 weeks and 2 years are forthcoming. In the third part of the study, patients receive the highest dose and phototherapy is no longer prohibited. Rosmarin said he is hopeful that those who receive phototherapy will have improved repigmentation.

A phase 3 trial is planned to start at the end of this year, he said. – by Abigail Sutton

For more information:

Rosmarin D, et al. Efficacy and safety of ruxolitinib cream for the treatment of vitiligo: Results of a 24-week randomized, double-blind, dose-ranging, vehicle-controlled study. Presented at: 24th World Congress of Dermatology; June 10-15, 2019; Milan.

Disclosure: Rosmarin reports he has received honoraria as a consultant for AbbVie, Celgene, Dermavant, Dermira, Janssen, Lilly, Novartis, Pfizer and Regeneron Pharmaceuticals; has received research support from AbbVie, Bristol Meyers Squibb, Celgene, Dermira, Incyte, Janssen, Lilly, Merck, Novartis, Pfizer and Regeneron Pharmaceuticals; and has served as a paid speaker for AbbVie, Celgene, Janssen, Lilly, Novartis, Pfizer, Regeneron Pharmaceuticals and Sanofi.