Date: 2013-09-06
Type of information: Initiation of preclinical development
phase: 1-2
Announcement: dosing of the first patient
Company: Prosensa (The Netherlands)
Product: PRO053 (exon 53 specific phosphorothioate oligonucleotide)
Action
mechanism: PRO053 (exon 53 specific phosphorothioate oligonucleotide) is an antisense oligonucleotide. It is based on Prosensa’s exon skipping technology is directed at interference with exon inclusion signals within an exon during the splicing of the pre-mRNA, in order to prevent the incorporation of the targeted exon in the mature RNA. This selective removal of (mutated) exons increases the level of functional transcripts and reverse disease symptoms.PRO053, the company’s fourth drug development candidate, induces exon 53 skipping and may be applicable to approximately 8% of all DMD patients.
Disease: Duchenne muscular dystrophy (DMD)
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country: Europe
Trial
details: This open-label study has been designed to assess the safety, efficacy, tolerability and pharmacokinetics of multiple doses of PRO053 in a subset of patients with Duchenne muscular dystrophy. 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.
Latest
news: * On September 6, 2013, Prosensa, a Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, has 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 (DMD). This open-label study has been designed to assess the safety, efficacy, tolerability and pharmacokinetics of multiple doses of PRO053 in a subset of patients with Duchenne muscular dystrophy. The primary outcome measure of the study will be change from baseline in the six-minute walk test (6MWT) after 48 weeks of treatment.
PRO053 is Prosensa\'s fourth exon-skipping compound that has entered into clinical trials. Prosensa’s 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.
