Summit Therapeutics to present at Duchenne conference

The AIM-traded firm said the conference would bring together families living with Duchenne and Becker muscular dystrophy, along with experts and industry members aiming to treat these diseases.

Summit’s scientific adviser and co-founder, professor Kay Davies from the University of Oxford, would present recently-published preclinical data highlighting the benefits of utrophin modulation on muscle health in animal models of DMD.

The data reportedly showed that continuously expressing utrophin in a dystrophin-deficient background could reduce mitochondrial aberration and oxidative stress.

Aberrant mitochondria drive, in part, oxidative stress, which Summit said is a contributing factor to muscle damage in DMD.

In addition, professor Davies was also expected to discuss previously-published preclinical data of utrophin modulators, including ezutromid, that had shown the potential of those compounds in preventing molecular disease, leading to functional improvements in mdx mice.

“My team at the University of Oxford continues to gather scientific evidence showing how utrophin can substitute for dystrophin in animal muscle and prevent many of the molecular hallmarks of DMD from occurring in these animal models,” commented professor Kay Davies.

“Importantly, these underlying changes in muscle health have the potential to lead to functional benefits, providing hope for a universal, disease-modifying treatment for families living with Duchenne.”

Separately, Summit said its presentations would focus on the ongoing Phase 2 proof-of-concept clinical trial, PhaseOut DMD, which was evaluating its lead utrophin modulator, ezutromid.

It described PhaseOut DMD as a 48-week open-label clinical trial that was fully enrolled with a total of 40 patients with DMD.

A key milestone of the clinical trial was the 24-week assessment, which could provide initial clinical proof-of-mechanism for ezutromid, and that data was expected to be reported in the first quarter of 2018.

“These new preclinical data from Professor Davies' team at the University of Oxford provide another piece of evidence highlighting the potential of utrophin modulation in being able to treat this devastating muscle wasting disease," said Summit Therapeutics chief operating and medical officer Dr David Roblin.

“We remain on-track to report the first results of ezutromid treatment in boys with DMD in PhaseOut DMD in the first quarter of 2018, and if results provide evidence of the mechanism of utrophin modulation in patients, we believe it would represent a major advancement for ezutromid.

“It would bring closer to all patients a therapy that has the potential to be disease modifying in DMD.”