Date: 2014-03-17
Type of information: Results
phase: 2
Announcement: results
Company: Prosensa (The Netherlands)
Product: drisapersen
Action
mechanism: This antisense oligonucleotide induces exon skipping of exon 51 in the DMD gene.
Disease: Duchenne muscular dystrophy
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country:
Trial
details: The study included 51 boys with DMD who were at least five years old, still able to walk and stand up from the floor without help in less than 15 seconds. This study compared 6mg/kg/week with 3mg/kg/week and placebo and was not statistically powered to show a significant difference between the arms.
Latest
news: * On March 17, 2014, Prosensa has reported encouraging 48-week data from its U.S.-based, Phase II placebo-controlled study (DMD114876 or DEMAND V) of its lead compound, drisapersen, for the treatment of Duchenne Muscular Dystrophy (DMD). The results of this study indicate that, compared to placebo, boys in the higher-dose drisapersen group (6 mg/kg once weekly) experienced stabilization and even improvements in their muscle function and physical activity as measured by the six-minute walk test (6MWT) for the 24-week treatment phase and maintained this improvement during the 24-week follow-up period. Additionally, when evaluating the percent-predicted six-minute walk distance (6MWD), a clinically meaningful treatment difference of 5.2% was observed at week 24 and 4.8% at week 48.
The study included 51 boys with DMD who were at least five years old, still able to walk and stand up from the floor without help in less than 15 seconds. As previously reported, boys in the group who received a 6mg/kg dose of drisapersen each week for the 24-week treatment period show a 27.1 meter improvement in the 6MWT (including a 16.1 m increase from baseline) over the boys in the placebo group at the end of the treatment period (p=0.069), indicating a clinically meaningful outcome for the primary endpoint.
Drisapersen at weekly doses of 3 and 6 mg/kg/week was generally well tolerated, although the majority of subjects treated with drisapersen reported injection-site reactions (none severe or serious). Renal abnormalities were common and occurred both in the placebo and drisapersen groups.
Principal investigator, Craig M. McDonald, M.D., Professor and Chair of Physical Medicine & Rehabilitation and Professor of Pediatrics at the University of California, Davis School of Medicine, will report the 48 week results in a poster session (Abstract #50) at the Muscular Dystrophy Association 2014 Clinical Conference in Chicago, Illinois (March 16-19).
