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February 2014

Sarepta Therapeutics Announces Eteplirsen Demonstrates Stability on Pulmonary Function Tests through 120 Weeks in Phase IIb Study in Duchenne Muscular Dystrophy

Sarepta Therapeutics

Sarepta Therapeutics, Inc. (NASDAQ: SRPT), a developer of innovative RNA-based therapeutics, announced on February 5 new pulmonary function data through Week 120 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results through more than two years of treatment showed stable pulmonary function in the Intent-to-Treat (ITT) study population (N=12). These data are consistent with previously reported 120-week clinical data showing a general stabilization of walking ability in eteplirsen-treated patients evaluable on the 6-minute walk test (6MWT).

Respiratory muscle function from baseline through Week 120, as measured by maximum inspiratory and expiratory pressure (MIP and MEP), showed a 14.6 percent mean increase in MIP and a 15.0 percent mean increase in MEP. Analyses of MIP percent predicted (MIP adjusted for weight) and MEP percent predicted (MEP adjusted for age) demonstrated a mean increase from 90.2 percent at baseline to 95.2 percent at Week 120 in MIP percent predicted, and a slight mean increase from 79.3 percent at baseline to 79.6 percent at Week 120 in MEP percent predicted. In addition, there was a mean increase in forced vital capacity (FVC), a measure of lung volume, of 8.7 percent from baseline to Week 120, and FVC percent predicted (FVC adjusted for age and height) was maintained above a mean of 90 percent through Week 120, with 101 percent at baseline and 93 percent at Week 120.

“We now have pulmonary function clinical outcome measures demonstrating general stability over a time period and in an age group where we would expect to see decline,” said Edward Kaye, M.D., senior vice president and chief medical officer of Sarepta Therapeutics. “While we will continue to monitor these patients on pulmonary function tests, the 120-week data potentially provide early evidence towards achieving a key treatment goal, the delay or avoidance of pulmonary ventilation assistance in DMD patients.”

“The first sign of respiratory muscle weakness in DMD is often deterioration in MIP and MEP as patients approach and enter their early teen years,” said Jerry Mendell, M.D., director of the Centers for Gene Therapy and Muscular Dystrophy at Nationwide Children's Hospital and principal investigator of the Phase IIb study. “As the muscles continue to weaken over the course of the disease, respiratory dysfunction requiring ventilation support can severely impact quality of life and respiratory failure is often a significant factor in patient mortality in the later stages of disease progression.”

To read the full press release, click here.

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