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Sarepta starts dosing DMD patients in eteplirsen trial

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 trial (Study 204) is designed to evaluate the safety of eteplirsen over 96 weeks of dosing, and will be conducted 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.

The trial, which will be carried out at several sites in the US, will enroll around 20 patients treated with eteplirsen who have genotypes amenable to exon 51 skipping and who meet other study inclusion criteria.

In 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: "The initiation of this eteplirsen study represents an important milestone for patients, their families, and the DMD community.

"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 is also developing other PMO-based exon-skipping drug candidates intended to treat additional patients with DMD.