US-based Sarepta Therapeutics has started dosing patients in a clinical trial of its lead exon-skipping therapeutic candidate eteplirsen to treat Duchenne muscular dystrophy (DMD).
The open-label trial (Study 204) will be carried out in DMD patients who are non-ambulant or who have advanced DMD and don’t meet a minimum six-minute walk test score at baseline.
Around 20 patients treated with eteplirsen who have genotypes amenable to exon 51 skipping and who meet other study inclusion criteria, will be enrolled in the trial.
The trial, to be carried out at several sites in the US, will evaluate the safety of eteplirsen in DMD patients over 96 weeks of dosing.
During the trial, patients will be given once weekly intravenous infusions of 30mg/kg of eteplirsen, and data will be collected across a number of safety parameters and secondary efficacy endpoints.
Sarepta chief medical officer Dr Edward Kaye said: "Expanding the DMD population to include patients who are older and non-ambulant demonstrates our strong commitment to develop eteplirsen for patients at all stages of DMD and will provide additional data to support our planned NDA filing."
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.
The company said that data from clinical studies of eteplirsen in DMD patients have showed a broadly favourable safety and tolerability profile and restoration of dystrophin protein expression.
Massachusetts General Hospital director of Pediatric Neuromuscular Service and principal investigator in the trial Fawn Leigh said: "Eteplirsen is a potential breakthrough treatment for patients with DMD. I am pleased to be able to offer this promising disease-modifying treatment to my patients and to potentially alter the course of this devastating disease."
The company is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.