Summit Therapeutics has opened enrollment for an additional group in its ongoing PhaseOut DMD trial to include patients with Duchenne muscular dystrophy (DMD) who participated in the previous Phase 1 clinical trials for the investigational therapy ezutromid (SMT C1100) but who, at the time of enrollment, failed to meet the enrollment criteria for PhaseOut DMD.
The new patients’ group will enhance the trial’s population, allowing Summit to test the therapeutic value of ezutromid in a larger group.
The new group will receive treatment in parallel to the ongoing participants of the PhaseOut DMD trial.
Ezutromid is an oral small-molecule modulator of utrophin, a protein that is structurally and functionally similar to the dystrophin protein, which is absent in DMD patients.
Ezutromid is a potential disease-modifying treatment for all patients with DMD, regardless of the type of mutation affecting the dystrophin gene, because previous studies have shown that expression of utrophin improves muscle performance.
The Phase 2 trial (NCT02858362) began enrolling participants in 2016 and completed it in May 2017 and was designed to evaluate the activity and safety of ezutromid in 40 ambulatory pediatric boys (5-10 years old) with DMD.
Involving centers in the United Kingdom and United States, enrolled patients receive a 2,500 mg oral administration of ezutromid. The study comprises a screening and baseline phase of up to 28 days, followed by a 48-week open-label treatment phase, and a 30-day safety follow-up phase.
Recent interim results from the 24-week analysis showed that, compared to baseline, ezutromid had a clinical meaningful impact by reducing muscle damage and inflammation, as shown by muscle biopsies and MRS transverse relaxation time T2, respectively.
The results from the 48-week trial of these 40 patients are expected by September.
“We are extremely grateful to the patients who participated in our Phase 1 clinical trials and contributed to ezutromid’s clinical advancement, but were not initially eligible to participate in our Phase 2 clinical trial. Accordingly, we are pleased to open this additional group in our Phase 2 and provide these patients with the opportunity to receive ezutromid treatment,” David Roblin, MD, chief operating officer and medical officer of Summit said in a press release.
“We expect the data collected from this additional group of patients will help expand our understanding of ezutromid’s safety and efficacy profile across a broader patient population,” he said.