New muscular dystrophy drug trials fail to meet goals

GlaxoSmithKline and Prosensa reported this week that trials for a drug to treat Duchenne muscular dystrophy have failed to provide statistically significant improvement in muscle strength.

The trial assessed if patients were able to walk faster over a period of six minutes after taking a course of the new drug, drisapersen. However, the results showed no improvement when compared to a placebo.

The results are from the Phase III test, a late stage clinical trial. This major setback comes very late into the research and development. GlaxoSmithKline and Prosensa were leading the medical industry with a treatment for muscular dystrophy, however the news of their failure has caused Prosensa’s investors to start looking at alternative medical companies – Prosensa’s stock market price fell by 75% before trading opened.

Duchenne muscular dystrophy affects 1 in 3600 boys and causes muscle degeneration and eventually death. It is caused by a gene mutation and currently there is no effective treatment or cure and the condition results in paralysis, Children with the condition have a life expectancy of 25 years.


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GSK suffered another set-back earlier this month after its drug to combat melanoma also failed to produce any meaningful results.

GlaxoSmithKline’s Carlo Russo said “We appreciate that these results will be disappointing for boys with DMD and their families”.

Hans Schikan, CEO for Prosensa said “While we are disappointed that this study did not meet its primary endpoint, we remain committed to the overall programme and will continue to work closely with GSK”.

Research is continuing in this area and hopefully future trials will be more successful.

Source: http://uk.reuters.com/article/2013/09/20/us-gsk-prosensa-idUKBRE98J0FD20130920

Reference: http://www.gsk.com/media/press-releases/2013/gsk-and-prosensa-announce-primary-endpoint-not-met-in-phase-iii-.html


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