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Why All The Internet Buzz For Sarepta Therapeutics’ Orphan Drug Eteplirsen ?

November 12, 2012

       On November 10, 2012, at the Action Duchenne 10th International Conference in London, Dr. Ed Kaye, Sarepta Therapeutics’ Chief Medical Officer (CMO), presents results of the Phase 2B Eteplirsen 48-Week Extension Trial.  Dr. Kaye discusses that Duchenne Muscular Dystrophy (DMD) patients treated with orphan drug Eteplirsen “continue to do well on therapy with no decline in function nor missed infusions or discontinuations”.  

        Per Liisa Bayko, JMP Securities Analyst, who attended the meeting in London : 

–  For Eteplirsen, “ … learned that nNOS, an important marker in muscle cells, was reconstructed, wich further validates the drug candidate …. Intravenous delivery seems to be associated with less severe injection site reactions than drisapersen ….” 

–  Presentation of 62-week Study data may be presented in January 2013 in San Francisco 

–  Eteplirsen’s competitor, GlaxoSmithKline (GSK) and Prosensa’s drug Drisapersen’s for DMD, will not release Phase II placebo controlled study results until 3rd Quarter 2013, at the earliest.  Drisapersen will not likely be approved until 2014 or 2015. “This removes some risk from Sarepta” 

–  Dr. Padraig Wright, from GSK, provides update on ongoing long-term extension (LTE) study of Drisapersen 

–  “ …. Sarepta continues to be blocked from engaging in clinical studies for 51-skipping drugs in the UK given GSK’s patent win, though other European countries may be more flexible ….. Prosensa …. is pursuing legal action in the US …”.   


 ** November 12, 2012 article titled, “Buzz on Sarepta’s Eteplirsen From London Duchenne Meeting”  

** November 12, 2012 article titled, “Sarepta trades higher on Eteplirsen Phase II Update, Analyst Comments” November 12, 2012 article titled, “Sarepta climbs after analyst says competitor delay removes overhang”  

Orphan Druganaut November 9, 2012 Blog Post titled, “Sarepta Therapeutics Announces recent Corporate Developments For Orphan Drug Eteplirsen”  

Orphan Druganaut November 6, 2012 Blog Post titled, “The Power Of Patient Advocacy Groups On Orphan Drug Development: Sarepta Therapeutics’ Orphan Drug Eteplirsen For Cuchenne Muscular Dystrophy”  

Copyright © 2012-2013, Orphan Druganaut Blog.  All rights reserved.

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