Enanta Begins Phase 1 Trial Of EDP-978 Study Of Oral KIT Inhibitor, Targeting Mast Cell-Driven Diseases

Enanta Pharmaceuticals has announced that the first participant has been dosed in its Phase 1 clinical trial evaluating EDP-978, an investigational oral KIT inhibitor. The therapy is being developed as a once-daily treatment for urticaria and other mast cell-driven diseases, marking an important early clinical milestone.

The Phase 1 trial is a randomised, double-blind, placebo-controlled, first-in-human study designed to assess the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of EDP-978 in healthy adult volunteers. The study will enroll approximately 98 participants between the ages of 18 and 65 to generate initial clinical data on the drug’s profile.

“We are excited to move our lead immunology program into clinical development with this Phase 1 study of EDP-978, a potent and selective once-daily KIT inhibitor in development for the treatment of chronic urticaria and potentially other mast cell-mediated diseases,” said Scott T. Rottinghaus, M.D., Chief Medical Officer of Enanta Pharmaceuticals. “These severely debilitating inflammatory skin diseases result in itchy, red hives and angioedema that can significantly impact a patient’s quality-of-life, causing sleep disturbance, fatigue, irritability, anxiety, and depression. Many patients are refractory to currently approved therapies, creating an unmet medical need for a new efficacious oral agent. We look forward to reporting topline Phase 1 data in the fourth quarter of this year, including effects on biomarkers such as serum tryptase, which will give us insight into the activity of EDP-978.”

The trial includes both single-ascending dose (SAD) and multiple-ascending dose (MAD) phases. The SAD portion also includes a two-part food-effect cohort to evaluate how food intake influences drug absorption, while the MAD phase will involve a 14-day treatment period to better understand repeat-dosing effects.

By targeting KIT, a key driver in mast cell activity, EDP-978 has the potential to address underlying mechanisms of diseases such as urticaria. The results from this early-stage trial will help determine the drug’s development path and inform future studies.