UK-based clinical-stage drug discovery and development company Summit Therapeutics has begun enrolment in the US for its Phase II PhaseOut DMD clinical trial of ezutromid to treat Duchenne muscular dystrophy (DMD).
Ezutromid, referred to as F6, is an orally administered utrophin modulator believed to slow down the progression of DMD regardless of the dystrophin gene mutation.
DMD is a genetic disorder primarily affecting boys by causing progressive muscle degeneration and weakness. It is caused due to the absence of dystrophin, a protein that keeps muscle cells intact.
The Phase II open-label, proof-of-concept study will test the safety and activity of ezutromidand utrophin modulation by measuring muscle fat infiltration, as well as by measuring utrophin protein and muscle fibre regeneration in muscle biopsies.
The study is expected to enrol 40 boys aged five to ten, each of who will receive two biopsies, one at baseline and the second either after 24 or 48 weeks.
Summit chief medical officer Ralf Rosskamp said: "Our PhaseOut DMD clinical trial is an important component of bringing ezutromid to patients and families who are in urgent need of a disease modifying therapy, and we are making progress with patient enrolment in this clinical trial, with enrolment ongoing in the UK and now in the US."
The company is expected to release data from the initial group receiving biopsies next year.