First US Patients Enrolled in PhaseOut DMD, a Phase 2 Clinical Trial of Ezutromid for DMD

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

gene therapy work

Summit Therapeutics has enrolled its first U.S. patients in its proof-of-concept Phase 2 clinical trial of ezutromid in patients with Duchenne muscular dystrophy (DMD). The study is actively recruiting patients.

“Ezutromid has shown great promise in preclinical testing as a universal treatment that has the potential to slow or stop disease progression in all patients with DMD, regardless of their underlying dystrophin gene mutation,” John Jefferies, MD, of Cincinnati Children’s Hospital Medical Center, and the U.S. coordinating investigator in PhaseOut DMD, said in a press release. ”We are excited to participate in PhaseOut DMD and contribute to the clinical development of this utrophin modulator.”

PhaseOut DMD is a 48-week proof-of-concept Phase 2 clinical trial (NCT02858362) designed to evaluate the activity and safety of utrophin modulation with ezutromid in 40 ambulatory paediatric male patients with DMD, ages 5 to 10 years.

Ralf Rosskamp, MD, chief medical officer of Summit added, “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 enrollment in this clinical trial, with enrollment ongoing in the U.K. and now in the U.S.”

The evidence that ezutromid is able to modulate utrophin and lead to clinical benefit will be evaluated through a range of measures, including looking at the amount of fat that infiltrates into muscle using magnetic resonance imaging (MRI), as well as measuring utrophin protein and muscle fibre regeneration in muscle biopsies.

The trial, will be conducted in a multi-center setting in both the U.K and the U.S. It is comprised of a screening and baseline phase of up to 28 days, a 48-week open label treatment phase, and a 30-day safety follow-up phase. Enrollment and dosing of patients in the U.K. is ongoing.



For more information about this clinical trial and how to enroll contact: Michele Cioffi (617) 401-2473, email: [email protected], or visit the trial website.

Ezutromid is an orally-administered, small-molecule modulator of utrophin, a protein that is structurally and functionally similar to the dystrophin, an essential protein for the healthy function of all muscles, that is missing in patients with DMD. Ezutromib represents a potential disease-modifying treatment for all patients with DMD, regardless of their underlying dystrophin gene mutation.