Summit Therapeutics to expand clinical trials after healthy interim results

Summit Therapeutics will expand enrollment in its phase 2 clinical trials studying the effect of ezutromid treatment on Duchenne muscular dystrophy (DMD), the company said on Wednesday.

The PhaseOut DMD trial is a multi-centre, 48 week open-label clinical trial with 40 enrolled patients between the ages of five and ten who suffer from DMD which will be enhanced by the newly announced parallel running expansion.

David Roblin, chief operating and medical officer of Summit, said: "We are extremely grateful to the patients who participated in our Phase 1 clinical trials and contributed to ezutromid's clinical advancement, but were not initially eligible to participate in our Phase 2 clinical trial. Accordingly, we are pleased to open this additional group in our Phase 2 and provide these patients with the opportunity to receive ezutromid treatment."

Patients with DMD will be eligible for the expanded trial regardless of their age or ambulatory status.

DMD is a muscle wasting disease that affects approximately 50,000 men and boys in the developed world and is caused by genetic faults in the gene that codes dystrophin and thus preventing the healthy function of all muscles.

The orally administered small molecule drug is intended to modulate utrophin, a protein functionally and structurally similar to dystrophin, with the aim of slowing or even halting DMD.

Recently announced 24 week interim data from the trials demonstrated that compared to baseline, ezutromid significantly reduced muscle damage and muscle inflammation attributed to the disease.

"We expect the data collected from this additional group of patients will help expand our understanding of ezutromid's safety and efficacy profile across a broader patient population," said Roblin.

Ezutromid has been granted Fast Track designation and Rare Pediatric Disease designation by the US Food and Drug Administration (FDA) as well as orphan drug status which allows for additional regulatory support and a period of market exclusivity following approval.

As of 1221 GMT, Summit Therapeutics’ shares were up 2.58% at 184.65p.