CAGE Bio, Inc. has announced positive topline results from its Phase 2 study evaluating CGB-500, an ionic liquid formulation of 1% tofacitinib, for the treatment of Atopic Dermatitis (AD). The randomized, vehicle-controlled trial demonstrated significant efficacy and a favorable safety profile, suggesting a potential new option for patients with moderate to severe AD and low body surface area (BSA) involvement.
The study met all primary and secondary endpoints, with the active group experiencing an average reduction of 98% in lesional Eczema Area Severity Index (EASI) scores at 8 weeks, compared to a 28% reduction in the vehicle group. Treatment success, defined as a rating of clear or almost clear with ≥ 2 grade improvement from baseline, was achieved in 95% of patients treated with CGB-500, versus 24% in the vehicle group. Furthermore, a significantly higher proportion of patients in the active group achieved a greater than 90% improvement in their EASI score (EASI-90) throughout the study. Notably, 84% of patients in the active group had a reduction of 4 or greater on the Numeric Rating Scale (NRS) for itching compared to 43% in the vehicle group.
Safety and Tolerability
Both CGB-500 and the vehicle were well-tolerated, with no treatment-emergent adverse events reported. There were no reports of pain or skin irritation at the application site, and no serious adverse events (SAEs) occurred during the study.
Ionic Liquid Platform Advantage
The positive results highlight the potential of CAGE Bio's ionic liquid platform to enhance topical drug delivery. The platform aims to achieve higher local bioavailability of the drug without increasing systemic exposure, addressing a critical need for AD patients who may benefit from localized therapy while avoiding systemic side effects.
Expert Commentary
"Effectiveness and the potential of tofacitinib in treating immune-mediated skin diseases has been known. The strong data from this study further underscore its potential when combined with the right formulation science. Ionic-liquid based formulations of JAK inhibitors like tofacitinib could address a critical need for treating AD patients with higher severity and low BSA involvement", said dermatologist Dr. Justin Ko, chair of CAGE Bio's scientific advisory board.
Nitin Joshi, Ph.D., CEO of CAGE Bio, stated, "We are elated with the results from our phase 2 study. This product could fill a key gap in the treatment of AD patients. We remain committed to advancing the development of CGB-500 for AD and multiple other indications that will benefit from localized therapy."
Next Steps
CAGE Bio plans to initiate dose-ranging and registration trials to further validate the efficacy and safety of CGB-500. These pivotal trials will pave the way for regulatory submissions and the potential commercial availability of this therapy.
