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Shares of Prosensa (RNA - Snapshot Report) plummeted more than 70% to $7.14 on Sep 20, 2013, following the announcement of negative data from a phase III study on drisapersen. Prosensa is developing drisapersen in collaboration with GlaxoSmithKline (GSK - Analyst Report) for the treatment of patients suffering from Duchenne muscular dystrophy (DMD).

We note that Prosensa granted Glaxo an exclusive worldwide license to develop and commercialize drisapersen in 2009.

The randomized, double-blind and placebo-controlled phase III study compared the use of drisapersen versus placebo in DMD patients. The study failed to meet the primary endpoint as drisapersen did not show statistically significant improvement in the 6 Minute Walking Distance (6MWD) test versus placebo. No difference in the key secondary assessments of motor function was observed.

Full evaluation of the benefit-to-risk profile of drisapersen is expected to be completed by the end of 2013.

Drisapersen enjoys orphan drug status in the EU, Japan and the U.S. In Jun 2013, the U.S. Food and Drug Administration (FDA) granted breakthrough therapy designation to drisapersen. We note that the breakthrough therapy designation was granted to drisapersen on the basis of data from a phase II study which evaluated the use of the candidate in patients suffering from DMD.

We note that several other companies including Sarepta Therapeutics, Inc. (SRPT - Snapshot Report) are developing therapies targeting the DMD market. Sarepta shares climbed 18% on the drisapersen setback news.

Prosensa carries a Zacks Rank #4 (Sell) while Glaxo carries a Zacks Rank #3 (Buy). Currently, companies like Actelion Ltd. (ALIOF) look more attractive with a Zacks Rank #1 (Strong Buy).

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