Sarepta (SRPT) Updates on Eteplirsen Study 202; Significant Data Recorded in mITT Population

December 7, 2012 8:35 AM EST Send to a Friend
Sarepta Therapeutics (NASDAQ: SRPT) announced updated data from Study 202, its open-label, Phase IIb extension study of eteplirsen for the treatment of Duchenne muscular dystrophy (DMD). Patients treated with eteplirsen for 62 weeks and evaluable on ambulatory measures (modified Intent-to-Treat population) maintained a statistically significant clinical benefit on the primary clinical outcome measure, the 6-minute walk test (6MWT), compared to patients who received placebo for 24 weeks followed by 38 weeks of eteplirsen treatment. As reported previously, Study 202 met its primary endpoint of increased novel dystrophin as assessed in muscle biopsies at week 48 and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes.

In the modified Intent-to-Treat (mITT) population, which includes evaluable patients from both the 30mg/kg and 50mg/kg dose cohorts, patients treated with eteplirsen for 62 weeks demonstrated a statistically significant benefit of 62 meters over the placebo/delayed-treatment cohort using a mixed-model repeated measure statistical test. The mITT consisted of 10 of the enrolled 12 patients (4 eteplirsen-treated patients receiving 50 mg/kg weekly, 2 eteplirsen-treated patients receiving 30 mg/kg weekly, and 4 placebo/delayed-treatment patients), and excludes two patients who showed signs of rapid disease progression and lost ambulation by week 24. The eteplirsen treatment cohort (n=6) continued to show disease stabilization and the cohort has shown less than a 5% decline in walking distance on the 6-minute walk test from baseline. The placebo/delayed-treatment cohort (n=4) also demonstrated stability in walking distance from week 36 through week 62 with a less than 10 meter change over this timeframe, the period in which dystrophin was likely produced, with confirmation of significant dystrophin levels at week 48 through analysis of muscle biopsies in these patients.

The safety profile of eteplirsen was evaluated across all patients through week 62 and there were no clinically significant treatment-related adverse events, no serious adverse events, and no discontinuations. One patient had a laboratory treatment-related adverse event, a transient elevation of urine protein on a urine dipstick test, however this elevation was not observed on a 24-hour urine protein measurement and resulted in no clinical symptoms or interruption of treatment. This patient did not show elevations of the specific renal markers of cystatin C or KIM-1. Across both the treatment and placebo/delayed treatment cohorts there is evidence of continued stabilization on pulmonary function tests, echocardiogram, muscle strength and clinical laboratory tests over the 62 weeks.

Results from the mITT population, which combines the evaluable eteplirsen-treated patients across the 30mg/kg and 50mg/kg cohorts, have previously been reported and will be used as the primary assessment of ambulatory clinical measures for the remainder of Study 202. Given there was no significant difference between the 30 mg/kg and 50 mg/kg arms on the production of dystrophin through 48 weeks, this mITT population is the most appropriate to assess dystrophin production and its potential predictive benefits on ambulatory clinical outcomes, such as the 6MWT.


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