US-based biopharmaceutical firm Sarepta Therapeutics has dosed the first patient in a clinical trial of its lead exon-skipping therapeutic candidate eteplirsen to treat Duchenne muscular dystrophy (DMD).
The open-label study trial 4658-301 (PROMOVI) will be carried out in ambulatory DMD patients who meet specific criteria on their baseline six-minute walk test score.
Around 160 patients will be enrolled in the trial, which will evaluate the efficacy and safety of eteplirsen in DMD patients over 48 weeks of dosing, and will be conducted at about 39 sites across the US.
The trial will enrol 60-80 boys, aged between seven and 16 years with genotypes amenable to exon 51 skipping who will be treated with eteplirsen, and an additional 60-80 patients with genotypes not amenable to exon 51 skipping will serve as a concurrent control group.
Patients in the trial's treatment group will receive once weekly intravenous infusions of 30mg/kg of eteplirsen and data will be collected across a number of efficacy and safety parameters, including dystrophin assessments.
Sarepta chief medical officer Dr Edward Kaye said: "This confirmatory study with eteplirsen in ambulatory patients is an important step in the process of confirming the promising clinical results we achieved in our previous studies 201/202.
"By evaluating a larger population of boys and examining both functional and biochemical endpoints, we expect to further support the evidence of eteplirsen's potential benefit to patients."
Eteplirsen uses the company's new phosphorodiamidate morpholino oligomer (PMO)-based chemistry and proprietary exon-skipping technology to skip exon 51 of the dystrophin gene enabling the repair of specific genetic mutations that affect about 13% of the total DMD population.
Seattle Children's Hospital principal investigator Susan Apkon said: "The commencement of this study is a significant achievement for the DMD community.
"Eteplirsen shows great potential as an important therapeutic, and I am delighted to be a part of this larger confirmatory study to further understand the impact that eteplirsen may have on families dealing with this terrible disease."