Prosensa: First Patient Dosed In Phase I/II Clinical Trial Of PRO053

(RTTNews) - Prosensa Holding N.V. (RNA) announced the dosing of the first patient in its Phase I/II clinical trial of PRO053, an exon-skipping compound for the treatment of Duchenne muscular dystrophy or DMD.

Prosensa said PRO053, the company's fourth drug development candidate, induces exon 53 skipping and may be applicable to approximately 8% of all DMD patients.

This is an open-label study designed to assess the safety, efficacy, tolerability and pharmacokinetics of multiple doses of PRO053 in a subset of patients with DMD.

According to the company, the primary outcome measure of the study will be change from baseline in the six-minute walk test (6MWT) after 48 weeks of treatment. A number of secondary outcome measures will also be assessed, including safety, muscle function and strength, and functional outcome based on a questionnaire. The initial dose-finding portion of the study will be conducted at several clinical trial sites in Europe. The study may then be extended to additional territories in and outside of Europe.

The company noted that its current portfolio includes six compounds for the treatment of DMD, all of which have received orphan drug status in the United States and the European Union.