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Reports US

US stock market daily report (April 21, 2014, Monday)

April 22, 2014, Tuesday, 05:49 GMT | 00:49 EST | 10:19 IST | 12:49 SGT
Contributed by Millennium Traders

Sarepta Therapeutics, Inc. (SRPT-Nasdaq) shares soared higher on Monday after announcing plans to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) before the end of 2014, for the approval of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). Sarepta, developer of innovative RNA-based therapeutics, shares ended the trading session higher by over 39%.

Eteplirsen is the lead exon-skipping drug candidate in development by Sarepta and is for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51.

Chris Garabedian, president and chief executive officer of Sarepta Therapeutics said, “As we announce our plan to submit an eteplirsen NDA by the end of 2014, we are very pleased with the detailed guidance that the FDA has provided us on a potential eteplirsen approval pathway and their support of a historically controlled eteplirsen confirmatory study. We also appreciate that the FDA shares our urgency in dosing a broader base of eteplirsen patients and has encouraged us to begin the clinical program with our follow-on exon-skipping drugs as soon as possible.”

A guidance letter from the FDA, regarding the plan to submit an NDA for eteplirsen by the end of 2014, is based on a strategy regarding the submission of an NDA for eteplirsen under a potential Accelerated Approval pathway and served as the final meeting minutes for four meetings that took place between November 2013 and March 2014. Reports from the FDA included that “with additional data to support the efficacy and safety of eteplirsen for the treatment of DMD, an NDA should be fileable.” Additionally, outlined examples of additional data and analysis that - if positive - will be important to enhance the acceptability of an NDA filing by addressing areas of ongoing concern in the existing dataset. The FDA provided clear guidance on an open-label, historically controlled confirmatory study of eteplirsen, as well as initial guidance on a placebo-controlled study of one or more follow-on DMD drug candidates, which, like the open-label study, could also be considered an acceptable confirmatory study to verify the clinical benefit of eteplirsen in the event of an accelerated approval, was provided by the FDA.

Edward Kaye, M.D., senior vice president and chief medical officer of Sarepta Therapeutics said, “We are excited to have guidance from the FDA that allows us to move quickly into additional clinical trials with eteplirsen to confirm our current understanding of eteplirsen’s safety profile, its effect on dystrophin production, and its impact on clinical outcomes in DMD patients. We are particularly pleased that the FDA shares our interest in accelerating the clinical development of our follow-on exon-skipping drugs and we expect to initiate enrollment in this trial later this year.”

Sarepta plans to initiate numerous additional clinical studies with eteplirsen later this year in exon-51 amenable genotype, based on the FDA’s guidance. Sarepta plans to initiate a placebo-controlled study with one or more of its follow-on DMD exon-skipping drug candidates by the end of 2014. Studies in the pipeline include a clinical trial with predefined efficacy endpoints for ambulatory patients between the ages of 7 to 16 years who can walk a minimum distance; two additional clinical trials that will evaluate safety and biomarkers in DMD patients younger than 7 years and DMD patients who have advanced in their disease progression to a point they cannot walk a minimum distance or have become non-ambulant.

Sarepta plans to immediately take steps to initiate the additional eteplirsen clinical studies with the goal of beginning dosing in the confirmatory study in the third quarter, with dosing in the additional trials (i.e., younger and more advanced DMD patients) to begin later this year. Once available, detailed study eligibility criteria and clinical site information will be posted on as well as - an online resource center from Sarepta for the DMD community.