Chris Garabedian, President and CEO, Sarepta Therapeutics (SRPT), Presents at Canaccord Growth Conference

August 18, 2014

Chris Garabedian, President and CEO Sarepta Therapeutics (SRPT), announced that the Phase IIb clinical study on its first product, eteplirsen, will have its results submitted to the FDA by the end of the year, with a launch slated for mid next-year if approved. He was speaking at the Canaccord Genuity 34th Annual Growth Conference at the Intercontinental Hotel in Boston, Massachusetts.

Sarepta Therapeutics targets therapies for the RNA molecule. The company’s lead drug development program is for Duchenne muscular dystrophy (DMD), which affects mostly boys and is 100% fatal, with most passing in their 20s. The Phase II tests for eteplirsen show it slows the decline in walking ability for DMD patients who take a six-minute walk test (6MWT). It also stabilized respiratory muscle function as assessed by pulmonary function tests. It is now in the long-term phase in which patients are continued to be followed for safety and clinical outcomes.

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Cambridge, Massachusetts-based Sarepta has 180 employees, two research facilities and recently purchased a manufacturing plant as it scales up for the potential release of eteplirsen. Garabedian said Sarepta has $284 million in cash and cash equivalents.

The company has produced mid-scale batches of eteplirsen, and will advance that to large scale commercial batches in time for a potential commercial launch next year. It also has started studies with follow-on drugs in its pipeline, which can be modified to adapt to genetic subtypes.

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