GlaxoSmithKline plc (NYSE:GSK)’s muscular dystrophy drug gains muscle with initial studies – SRPT

Northern, WI 04/11/2013 (usastockreport) – Drisapersen, GlaxoSmithKline plc (NYSE:GSK) (Closed: $47.75, Up by 0.91%)’ Duchenne muscular dystrophy which is in its mid-stage study achieved its primary objective. This is a significant step as the competition between Sarepta Therapeutics Inc (NASDAQ:SRPT) (Closed: $38.85, Down by 2.51%) and Glaxo Smith Kline will automatically be heating-up. This drug is being developed to help reverse the highly debilitating effects of the muscular disease. According to the study results that had been posted on Glaxo’s investor CureDuchenne’s sites the results of the drug study had been very positive.

Long over-due drug

After 48 weeks of treatment, the patients who had been administered drisapersen showed a marked difference from the ones who were on a placebo. This data will be presented at the genetics conference that is to be held in ColdSpringHarbor in New York, by Prosensa Therapeutics BV Glaxo the Dutch biotechnology company. Cure Duchenne is a non-profit organization based in Newport Beach, California and mentioned on its website that the drug approval has been pending for ten years and that they are optimistic about phase three, the data for which will be available later in this year.

The elusive cure

There is no known cure for Duchenne muscular dystrophy and this disease affects one out of 3,500 new-born boys. This genetic disease is the result of gene mutations that cause a break in the gene sequence. This sequence is the one that is responsible for the instructing the production of dystrophin which protects muscles. Since the bodies of the children are unable to produce it, the muscles weaken at a very rapid pace and eventually the boys need wheelchairs by the time they are 12 years of age. Glaxo’s drisapersen therapy is an RNA-based one. It skips the exon that lies next to the one that is defective and unblocks dystrophin production.

Mutual benefit

This drug has been designated as an orphan drug by U.S and U.K regulators. If it proves to be effective and safe and receives the nod from authorities and is eventually marketed, it has the potential to reach $766 million (500 million pounds) in sales. Since the Sarepta and Prosensa technologies are very similar, investors are concerned that a patent issue may arise. In 2011, Sarepta had challenged the Prosensa patent in the EU but had lost the case. The result has been that Prosensa blocked the entry of its rival into the European market. In the event that the Sarepta U.S patent is challenged by Prosensa the two rival-companies may eventually arrive at a mutual royalty-based agreement.

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