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news.Summit Therapeutics has reported positive phase I trial results for a new formulation of its Duchenne Muscular Dystrophy (DMD) candidate ezutromid.
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Ezutromid is an orally administered small molecule that is designed to modulate utrophin, a protein that is structurally and functionally similar to the dystrophin protein.
The new formulation, F6, achieved more than a six-fold increase in maximum plasma levels in DMD patients, compared to those achieved with the existing clinical formulation (F3), with only two fifths of the dose.
As a result, F6 will now be a part of the phase II trial of ezutromid, PhaseOutDMD.
Based on the positive data, Summit outlined its development strategy through to applications for market approval for ezutromid.
F6 is planned to be assessed in up to 10 of the 40 patients anticipated to be enrolled. It will be compared alongside the F3 formulation when dosed longer-term. Initial F3 24-week biopsy data is expected in the second or third quarters of 2017.
The development strategy also includes a randomized, placebo controlled trial designed with the potential to support accelerated and conditional regulatory approvals in the US and EU.
The trial is expected to begin in the second half of 2017 if PhaseOut DMD generates positive interim data.
Summit Therapeutics chief medical officer Ralf Rosskamp said: "The rigorous development of ezutromid has identified this new F6 formulation that achieved higher ezutromid plasma levels in patients in this trial allowing us to further explore the therapeutic effect of this promising treatment.
"Following these encouraging Phase 1 data, we plan to incorporate the F6 formulation of ezutromid into our ongoing Phase 2 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.”
Image: Histopathology of gastrocnemius muscle from patient who died of pseudohypertrophic muscular dystrophy, Duchenne type. Photo: courtesy of Dr. Edwin P. Ewing, Jr.