Ezutromid fails to hit targets in a mid-stage trial

Globally, there are about 50,000 people who have Duchenne muscular dystrophy (DMD), a rare muscle disease caused by a lack of dystrophin. Summit Therapeutics had hoped that their drug ezutromid would be an effective treatment of DMD by stimulating production of utrophin to substitute the missing protein dystrophin.

In initial trials, statistical decreases in developmental myosin and magnetic resonance T2 measures were recorded following 24 weeks of treatment, but the Phase II DMD trial did not meet its primary endpoint of change from baseline in magnetic resonance parameters related to the leg muscles, or secondary endpoints of biopsy measures evaluating utrophin and muscle damage after 48 weeks.

As a result, shares have fallen 80% and Summit will stop development of ezutromid and look for cost reductions.

Glyn Edwards, Summit’s chief executive said: “These data come as a great disappointment to us and to all those living with DMD. While we believe utrophin modulation could still have a place in the treatment of DMD, it is clear that ezutromid is not providing a benefit for patients. We therefore feel that our resources are better focussed on the development of our promising pipeline of new mechanism antibiotics.”