Sarepta Therapeutics,
Inc. SRPT, a developer of innovative RNA-based
therapeutics, today announced updated data from Study 202, a Phase
IIb open-label extension study of eteplirsen in patients with
Duchenne muscular dystrophy (DMD). Results at 74 weeks showed a
continued stabilization of walking ability in eteplirsen-treated
patients evaluable on the 6-minute walk test (6MWT). As previously
reported, Study 202 met its primary endpoint of increased novel
dystrophin as assessed by muscle biopsy at week 48 and is now in the
long-term extension phase in which patients continue to be followed
for safety and clinical outcomes. Eteplirsen is Sarepta's lead exon-skipping compound in development for the treatment of patients
with DMD who have a genotype amenable to skipping of exon 51.
After 74 weeks, patients in the 30 mg/kg and 50 mg/kg dose cohorts
who were able to perform the 6MWT (modified Intent-to-Treat or mITT
population; n=6) showed a statistically significant treatment benefit
of 65.2 meters (p d 0.004) when compared to the
placebo/delayed-treatment cohort (n=4). The eteplirsen-treated
patients in the mITT population demonstrated less than a 5 percent
decline (13.4 meters) from baseline in walking ability. After
experiencing a substantial decline earlier in the study, the
placebo/delayed-treatment cohort also demonstrated stabilization in
walking ability from week 36 through 74, the period in which
meaningful levels of dystrophin were likely produced, with a less
than 10 meter decline over this timeframe.
"We are encouraged to see a continued stabilization of walking
ability in patients treated with eteplirsen for nearly one and a half years," said Chris Garabedian, president and chief executive officer
of Sarepta Therapeutics. "These data are particularly compelling when
viewed in the context of published natural history studies, which
showed substantial declines on the 6-minute walk test over this
timeframe in a similar ambulatory DMD population. These results
con
tinue to support the potential of eteplirsen to be a major advance
in the treatment of DMD in altering the course of this progressive
and irreversible disease."
Through 74 weeks, eteplirsen was well tolerated and there were no
clinically significant treatment-related adverse events, serious
adverse events, hospitalizations or discontinuations. As previously
reported at 62 weeks, one patient had a transient elevation of urine
protein on a laboratory urine dipstick test, which resolved and
resulted in no clinical symptoms. The patient continued treatment
without interruption and remained free of proteinuria through week
74.
Across both the eteplirsen (mITT) and placebo/delayed-treatment
cohorts, there is evidence of continued stabilization on clinical
laboratory tests, echocardiogram, pulmonary function tests and muscle
strength.
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