China Medical System Holdings Limited announced positive results from its phase 3 clinical trial of ruxolitinib cream in patients with mild to moderate atopic dermatitis in China. The randomized, double-blind, placebo-controlled, multi-center trial enrolled 192 patients and was conducted at Shanghai Dermatology Hospital under the leadership of principal investigator Professor Shi Yuling.
Primary Endpoint Success
The trial met its primary endpoint, demonstrating that a significantly higher proportion of patients treated with ruxolitinib cream achieved an Investigator's Global Assessment (IGA) score of 0 or 1 with at least two grades of reduction from baseline at week 8, compared with placebo (63.0% vs 9.2%, P<0.001). This represents clear or almost clear skin with substantial improvement from the start of treatment.
Strong Secondary Endpoint Results
For the key secondary endpoint, the proportion of patients achieving at least a 75% improvement from baseline in the Eczema Area and Severity Index score (EASI 75) was significantly higher with ruxolitinib cream treatment compared to placebo at week 8 (78.0% vs 15.4%, P<0.001). This metric measures both the extent and severity of eczema lesions across the body.
Safety Profile
The safety analysis showed that treatment-emergent adverse events during the treatment period were mostly mild or moderate in severity. Notably, no treatment-emergent adverse events led to discontinuation of the study drug. Overall, the ruxolitinib cream was characterized as safe and well-tolerated by the study investigators.
Addressing Unmet Medical Need
Atopic dermatitis represents a significant healthcare burden in China, with an estimated 54 million patients as of 2024. Based on SCORAD scores, the disease severity distribution in China shows 73% mild cases, 25% moderate cases, and 2% severe cases. Traditional topical medications such as topical corticosteroids and topical calcineurin inhibitors present clinical limitations due to long-term adverse reactions or limited efficacy, creating an urgent need for novel treatments.
Drug Development and Regulatory Status
Ruxolitinib cream (Opzelura®) is a novel topical formulation containing the selective JAK1/JAK2 inhibitor ruxolitinib, originally developed by Incyte. The cream represents the first topical JAK inhibitor approved by the U.S. Food and Drug Administration. In the United States and Europe, it is indicated for topical treatment of nonsegmental vitiligo in patients 12 years and older, and for short-term, non-continuous chronic treatment of mild to moderate atopic dermatitis in non-immunocompromised patients 2 years and older whose disease is inadequately controlled with topical prescription therapies.
CMS is actively advancing the product's new drug application in China following these positive phase 3 results. The company obtained exclusive licensing rights for ruxolitinib cream through a collaboration and license agreement with Incyte signed on December 2, 2022, covering development, registration, and commercialization rights in Mainland China, Hong Kong, Macau, Taiwan, and eleven Southeast Asian countries.
Market Authorization Progress
The new drug application for the vitiligo indication has already been accepted by China's National Medical Products Administration. Additionally, marketing authorization applications have been approved in Hong Kong and Macau, and the product received approval from the Guangdong Provincial Medical Products Administration through the "Hong Kong and Macau Medicine and Equipment Connect" policy for treating non-segmental vitiligo with facial involvement in adults and adolescents 12 years and older.
