Needham & Company analyst Chad Messer reiterated a Buy rating and $52 price target on Sarepta Therapeutic (NASDAQ: SRPT) following Q1 results and more details on the path to the eteplirsen filing.

Messer commented, "As we learned last month, Sarepta plans to file the NDA for eteplirsen in Duchenne's muscular dystrophy (DMD) by YE:14 based on recent discussion with the FDA. The company is moving forward on this groundbreaking event and is also preparing to start 3 additional eteplirsen studies as well as a controlled study for 2 additional exon skipping drug for DMD this year. The company is well capitalized to execute on these planned studies and the launch of eteplirsen having ended 1Q:14 with $233.1 million and raised an additional $94.5 million subsequent to the end of the quarter. We maintain our Buy rating as we anticipate a 2015 approval and blockbuster sales for eteplirsen."

Elaborating on the path to the eteplirsen NDA Filing, Messer notes: "Based on FDA guidance, Sarepta is working in several areas to provide additional evidence of eteplirsen's efficacy and safety to be included in the NDA filing by YE:14. The company is continuing to provide information about the measurement of dystrophin and is assessing the feasibility of collecting a 4th biopsy from patient in the Phase IIb study, which may lead to additional data that could be included in the filing. 144 week efficacy and safety data will be presented in October and will be included. The FDA has indicated that efficacy on either dystrophin or the 6-minute walk test could serve as basis for conditional approval. Additonally, Sarepta will intiate by year-end 3 historically controlled confirmatory studies including one with predefined endpoints in ambulatory patients between the ages of 7 to 16 years, a study to evaluate safety and biomarkers in DMD patients < 7 years, and a study in patients who no longer meet ambulation requirements. Any available data from these studies will be included in the filing as well as provided to FDA during the time of review. Assuming the NDA filing is expected, we expect the FDA to convene an advisory committee around mid-2015 to review all available data at that time. The company also plans to initiate a placebo controlled study with 2 additional DMD exon-skipping drug for exons 53 and 34. The FDA has indicated that either demonstrating efficacy from a drug similar to eteplirsen or data from the open label confirmatory study could serve as part of the necessary evidence for full eteplirsen approval. We look for a potential 2H:15 approval for eteplirsen and project peak sales in the U.S. and Europe of >$1.5 billion. The approval of additional exon skipping drugs would substantially expand this opportunity."

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Shares of Sarepta Therapeutic closed at $34.28 yesterday.