Ruxolitinib Cream 1.5% Demonstrates Efficacy in Prurigo Nodularis: Phase 3 Findings

Shawn Kwatra, M.D.,

Ruxolitinib cream 1.5%, a topical Janus kinase (JAK) 1/2 inhibitor, met all primary and key secondary endpoints in full trial results from one Phase 3 trial in adult patients with prurigo nodularis (PN), according to a company news release.¹

Topline results from a second phase 3 trial did not reach statistical significance in the primary endpoint, the primary and all key secondary endpoints were favorable, the company said.¹

Data from the TRuE-PN trial program were presented at the 2025 American Academy of Dermatology (AAD) Annual Meeting in Orlando, offering important insights into potential new therapeutic options for this debilitating condition.

"This program includes the first clinical trials evaluating a topical JAK inhibitor for the treatment of patients with PN, a condition associated with the formation of cutaneous nodules across the body that cause persistent itch and discomfort," said Shawn Kwatra, M.D., Joseph W. Burnett Endowed Professor and Chair of Dermatology at University of Maryland School of Medicine and Chief of Service Dermatology at the University of Maryland Medical Center, in a news release. "In TRuE-PN1, ruxolitinib cream 1.5% demonstrated its ability to significantly improve itch and PN lesions, and I believe it has the potential to be a much-needed novel approach for PN patients."¹

The TRuE-PN program comprises two double-blind, vehicle-controlled studies, TRuE-PN1 and TRuE-PN2, each followed by a 40-week open-label extension.

TRuE-PN1 successfully met its primary endpoint: significantly more patients in the ruxolitinib cream 1.5% group achieved at least a 4-point improvement on the Worst-Itch Numeric Rating Scale (WI-NRS4) at Week 12 compared with vehicle (44.6% vs 20.6%; P=0.0003). Furthermore, ruxolitinib cream displayed evidence of early itch relief, with significantly higher proportions of patients meeting the WI-NRS4 threshold as early as Day 7. The active treatment arm also outperformed vehicle across all key secondary endpoints, including the Investigator’s Global Assessment for Stage of Chronic Prurigo Treatment Success (IGA-CPG-S-TS) at Week 12, overall treatment success, and itch reduction by Week 4.¹

Topline findings from TRuE-PN2 similarly favored ruxolitinib cream over vehicle for the primary endpoint, although the difference did not reach statistical significance due to an unexpectedly high placebo response. Nevertheless, strong positive trends in key secondary endpoints, most notably IGA-CPG-S-TS at Week 12 and WI-NRS4 at Day 7, underscore the potential efficacy of ruxolitinib cream.¹

Safety findings from both studies were consistent with prior data on ruxolitinib cream in other dermatologic indications, including atopic dermatitis and vitiligo. No new safety signals emerged.¹

Prurigo nodularis is a chronic inflammatory disease marked by the formation of itchy nodules, often on the arms, legs, and trunk, which can significantly affect patients’ quality of life.

"PN is a challenging condition characterized by intensely itchy nodules that significantly impact patients' quality of life," said Jim Lee, M.D., Ph.D., Group Vice President, Inflammation and Autoimmunity, Incyte, in the news release. "These positive results display improvement in itch reduction and skin clearance, reinforcing the potential of ruxolitinib cream to become an effective topical treatment option that will help people living with PN."¹

Ruxolitinib cream 1.5% is already approved by the US Food and Drug Administration as Opzelura for the topical treatment of nonsegmental vitiligo in patients aged 12 years and older and for the short-term and noncontinuous chronic treatment of mild to moderate atopic dermatitis in certain populations.

Reference

1. Incyte. Incyte Announces Results of Phase 3 Clinical Trials Evaluating Ruxolitinib Cream 1.5% (Opzelura®) in Patients with Prurigo Nodularis (PN) at 2025 American Academy of Dermatology Annual Meeting. Press release. March 8, 2025. Accessed March 8, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-announces-results-phase-3-clinical-trials-evaluating