Sarepta Therapeutics,
Inc. SRPT, a developer of innovative RNA-based
therapeutics, today announced data through Week 96 from Study 202, a
Phase IIb open-label extension study of eteplirsen in patients with
Duchenne muscular dystrophy (DMD). Results through nearly two years
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.
After 96 weeks, patients in the 30 mg/kg and 50 mg/kg eteplirsen
cohorts who were able to perform the 6MWT (modified Intent-to-Treat
or mITT population; n=6) experienced less than a 5 percent decline
(17.5 meters) from baseline in walking ability. A statistically
significant treatment benefit of 70.8 meters (p d0.001) was
observed for the mITT population compared with the
placebo/delayed-treatment cohort (n=4), which initiated treatment at
Week 25 following 24 weeks of placebo. After experiencing a
substantial decline earlier in the study, the
placebo/delayed-treatment cohort also demonstrated stabilization in
walking ability from Week 36 through 96, the period from which
meaningful levels of dystrophin were likely produced, with a decline
of 18.5 meters over this timeframe. These analyses were based on the
maximum 6MWT score when the test was performed on two consecutive
days.
"We are very encouraged the study has demonstrated walking stability
in patients for more than a year since confirming that eteplirsen
treatment produced dystrophin in their muscles," said Chris
Garabedian, president and chief executive officer of Sarepta
Therapeutics. "We look forward to sharing these updated data with the
FDA as part of our New Drug Application for eteplirsen, which we plan
to submit in the first half of 2014."
As previously reported, a boy in the placebo/delayed-treatment cohort
was not able to perform the 6MWT at the Week 84 clinic visit due to a
broken ankle assessed by the investigator as a treatment-unrelated
adverse event. Although this boy received rehabilitation and was able
to perform the 6MWT, his walking ability at the time of the test had
not returned to the level observed prior to the injury, and this
lower 6MWT distance contributed to the overall decline in the
placebo/delayed-treatment cohort. The decline in walking distance
observed in this cohort from Week 36 improves from a decline of 18.5
meters to a decline of 4.7 meters when this patient's 96-week test
score is excluded from the analysis.
Through 96 weeks, eteplirsen was well tolerated and there were no
reported clinically significant treatment-related adverse events, no
treatment-related serious adverse events, hospitalizations or
discontinuations.
Across patients in the eteplirsen and placebo/delayed-treatment
cohorts (Intent-to-Treat or ITT population), there is evidence of
continued stabilization on clinical laboratory tests,
echocardiograms, pulmonary function tests and measures of muscle
strength.
Summary of Additional 6MWT Analyses
Patients performed two 6MWT evaluations on consecutive days at time
points coinciding with a muscle biopsy procedure at baseline and
Weeks 12, 24 and 48. All other evaluations were a single 6MWT. The
pre-specified primary analysis included the maximum distance walked
at those clinic visits where repeated tests were taken. Other
analyses of the repeated 6MWT results assessed mean, minimum, and Day
1 (first measure) scores. Results from these additional 6MWT analyses
confirm the robust treatment effect observed in the primary analysis.
Summary of 6MWT: Week 96 Treatment Results*
-----------------------------------------------------------------
-----------
Estimated
Treatment
Analysis of Baseline Adjusted Mean Benefit
Repeated 6MWT 6MWT 6MWT Change (Eteplirsen P-Value
Values** (meters) from Baseline Minus
(meters) Placebo/delaye
d-Tx)
----------------------------------------------------------------------------
Maximum Score
Eteplirsen (n=6) 399.7 -17.5 70.8*** d0.001
----------------------------------------------
Maximum Score
Placebo/delayed- 394.5 -88.3
Tx (n=4)
----------------------------------------------------------------------------
Mean Score
Eteplirsen (n=6) 388.6 -6.3 67.7 d0.004
----------------------------------------------
Mean Score
Placebo/delayed- 380.3 -74.1
Tx (n=4)
----------------------------------------------------------------------------
Minimum Score
Eteplirsen (n=6) 377.5 4.7 64.5 d0.009
----------------------------------------------
Minimum Score
Placebo/delayed- 366.0 -59.8
Tx (n=4)
----------------------------------------------------------------------------
Day 1 Score
Eteplirsen (n=6) 379.7 2.3 67.2 d0.008
----------------------------------------------
Day 1 Score
Placebo/delayed- 371.5 -64.9
Tx (n=4)
----------------------------------------------------------------------------
* All 6MWT analyses are based on a Mixed Model Repeated Measures
test.*
** All 6MWT analyses include the mITT population
*** The
pre-specified primary analysis of the 6MWT results was based on the
maximum score.
Edward M. Kaye, M.D., chief medical officer and senior vice president
of Sarepta Therapeutics, will present these data on October 3 at the
18th International World Muscle Society (WMS) Congress at 2:30 p.m.
PDT at the Asilomar Conference Grounds in Pacific Grove, California.
The presentation will be archived in the investor relations section
of the Sarepta Therapeutics website at www.sarepta.com for 90 d
ays.
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