Summit Therapeutics chief operating officer and medical officer David Roblin said: "In the interim data readout, we aim to show a positive change following ezutromid treatment in muscle structure and health through the evaluation of muscle biopsies.
“This could potentially provide the first evidence of proof of mechanism for utrophin modulators in patients and give hope to those living with DMD.”
Ezutromid is an orally administered small molecule drug. It has been designed to modulate utrophin, a protein that is identical, both structurally and functionally to the dystrophin protein.
The objective of the PhaseOut DMD will be to establish proof of concept of ezutromid. The phase 2 trial is assessing various muscle structure, muscle health and functional endpoints.
According to Summit, two muscle biopsies will be taken from each patient as part of the trial along with a baseline biopsy on enrolment followed by another one after 24 weeks or 48 weeks of dosing.
The company expects to release the top-line data from the complete 48-week phase 2 trial in the third quarter of 2018.
After the completion of the 48-week treatment, patients can continue onto an extension phase which will help the company record long term safety and efficacy data. The extension phase is likely to be carried on until ezutromid secures marketing approval in the relevant country or its development is stopped.
Last year, Ezutromid had succeeded in a phase 1 trial by registering more than a six-fold increase in maximum plasma levels in DMD patients, in comparison to the findings in patients who were subjected to existing clinical formulation (F3), with only two fifths of the dose.
Image: Summit has completed initial 24 weeks of dosing of ezutromid in PhaseOut DMD trial. Photo: courtesy of k1991/FreeDigitalPhotos.net.