UK-based clinical-stage drug discovery and development company Summit Therapeutics has reported positive Phase I clinical trial results of ezutromid to treat Duchenne muscular dystrophy (DMD) and Clostridium difficile infection.
Ezutromid, referred to as F6, is an orally administered utrophin modulator believed to slow down the progression of DMD regardless of the dystrophin gene mutation.
The second part of the Phase I clinical trial has been designed to evaluate the pharmacokinetics and safety of three fixed doses of ezutromid in patients suffering from DMD, aged between five and nine years.
Results of the study displayed an increase in plasma levels within patients compared to the current clinical formulation of ezutromid referred to as F3.
Summit chief medical officer Dr Ralf Rosskamp said: “Following this encouraging Phase I data, we plan to incorporate the F6 formulation of ezutromid into our ongoing Phase II trial, PhaseOut DMD.
“This will allow us to directly compare the safety and efficacy of the F6 and F3 formulations of ezutromid, and help determine which to use in future clinical trials.
“Utrophin modulation focuses on maintaining expression of utrophin protein to protect muscle health and function and we believe these two formulations of ezutromid are capable of achieving this.
"It is therefore appropriate to now outline our clinical pathway to seek marketing approval of ezutromid.”
Ezutromid was tolerable across all dosages.
Summit is now planning to apply the new formulation of F6 into its ongoing, PhaseOut DMD, Phase II proof-of-concept trial.