Replies to post #80161 on $heff's $tation of $tocks & $olid DD

[$heff]

$SRPT..Q1 2014 Recent Corporate Developments

Duchenne Muscular Dystrophy Program

-- Based on new guidance from the U.S. Food and Drug Administration (FDA), announced plans to submit a New Drug Application (NDA) to the FDA with additional safety and efficacy data and analysis by the end of 2014 for the approval of eteplirsen for the treatment of patients with DMD who have a genotype amenable to skipping of exon 51.

-- Announced plans to initiate several clinical studies of eteplirsen based on the new FDA guidance. Planned studies with eteplirsen in exon-51 amenable genotypes include a historically controlled clinical trial with predefined efficacy endpoints for ambulatory patients between the ages of 7 to 16 years who can walk a minimum distance, and 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 where they cannot walk a minimum distance or have become non-ambulant.

-- Additionally, announced plans to initiate a placebo-controlled study with one or more of the Company’s follow-on DMD exon-skipping drug candidates by the end of the year.

-- Presented clinical data through Week 120 from the Phase IIb open-label extension study of eteplirsen in patients with DMD at the Muscular Dystrophy Association Clinical Conference and the American Academy of Neurology Annual Meeting.

Corporate Updates

-- Announced the company priced an underwritten public offering of an aggregate of 2,650,000 shares of its common stock at a price to the public of $38.00 per share. In addition, Sarepta has granted the underwriters a 30-day option to purchase up to an additional 397,500 shares of common stock on the same terms and conditions as the initial shares sold to the underwriters. The aggregate net proceeds from the offering to the company was approximately $94.5 million, after deducting the underwriting discount and estimated offering expenses payable by Sarepta, but excluding any exercise of the underwriters’ option. The offering closed on April 29, 2014.