Sarepta Therapeutics
SRPT, a developer of innovative RNA-based therapeutics,
today 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 pat
ients.
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