|Indication||Compound||Discovery||Pre-clinical||Phase I/II||Phase III|
|Duchenne Muscular Dystrophy (DMD)||Drisapersen|
Drisapersen is Prosensa’s lead RNA-based product and is currently being developed in partnership with GlaxoSmithKline (GSK). Both parties are working closely together to make this drug available to patients as quickly as possible.
Drisapersen (previously PRO051/GSK2402968) induces exon 51 skipping in the dystrophin gene and is intended for approximately 13% of all Duchenne Muscular Dystrophy (DMD) patients, the largest known subpopulation of patients that includes those with deletions of exon 50, exon 52, exons 45-50, exons 48-50, and exons 49-50. It is highly sequence-specific minimizing the risk for off-target effects.
In human clinical trials, drisapersen has been shown to restore dystrophin expression and have a beneficial therapeutic effect on DMD patients. A Phase II placebo-controlled study of drisapersen in 53 DMD patients has been completed and has demonstrated a statistically significant and clinically important difference in its primary endpoint, which was the distance walked in the six minute walk test (6MWD), between the placebo group and the continuous active-treatment group at a dose of 6 mg/kg/week after 24 weeks. This clinically meaningful benefit was maintained after 48 weeks of treatment, and drisapersen was well tolerated throughout the duration of this study.
Drisapersen is currently in Phase III clinical trials, which were initiated in December 2010, and results are expected in the fourth quarter of 2013. This study is a randomized, double-blind and placebo-controlled trial, assessing drisapersen at a dose of 6 mg/kg/week in 186 boys. The primary endpoint is the 6MWD.
Drisapersen has also successfully completed a twelve patient Phase I/II clinical trial. Subcutaneous administration of the drug for a period of 5 weeks showed that the drug was well tolerated and that dystrophin expression was restored in all 12 treated patients. The open label extension of this study is on-going and the patients have been receiving the drug for more than 188 weeks.
Several other studies are on-going, such as a Phase II study comparing two doses to placebo (DMD114876) and an open-label study for patients that have previously participated in the 114044 or 114117 study. A Phase II dose regimen study (DMD114117) and a safety and PK study in non-ambulatory DMD boys (DMD114118) have recently been completed.
To date, over 300 patients have participated in clinical studies of drisapersen at more than 50 trial sites in 25 countries, and patient retention rates through March 2013 averaged 96% across all drisapersen clinical studies.
Details of the clinical trials, such as inclusion/exclusion criteria and trial sites, are posted on the website www.clinicaltrials.gov and all required approvals of authorities and ethics committees are granted. For further information on the clinical studies, you should contact the GSK call centre at +1 877-379-3718.
Drisapersen has been granted orphan drug status in the European Union and the United States.