The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: I want to start with that on the crossover data for 9001 coming up early next year. Just give us a sense for what we should be looking for in that
data set. Like, realize there's the natural history comparator. These patients also have a well-documented trajectory from the prior year that they
were on placebo. So just give us a sense for how we should be thinking about this data set.
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NOVEMBER 30, 2021 / 6:25PM, SRPT.OQ - Sarepta Therapeutics Inc at Evercore ISI HealthCONx Virtual
Conference
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: How did you select the natural history cohort? Like what variables were you trying to match for? And maybe like what would you kind of expect
to see in this group of natural history patients?
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: And on the patients who are crossing over, they've got the year on placebo before being dosed. How should we think about the trajectory changes?
Are you hoping to see something very clear that you get the gene therapy, and there's a market change in trajectory? Or are there more nuances
than that just because we're also talking about an older patient population and patients are aging? Just curious like how we should be contextualizing
the trajectory on endpoints as well.
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: You talked about some of these other endpoints that you'll be measuring as well beyond NSAA. Are we going to get that some of those with the
update early next year? And any sense for which ones we should be focused on?
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: Got it. That makes sense. And I guess if the crossover data looks good, we've seen that the 103 data. What are your latest thoughts on whether or
not you might try to file in the younger patients? And I guess what's the downside for trying as long as you're setting expectations that should be
a base case scenario?
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: Got it. Makes total sense. I want to switch gears to limb-girdle since that's another large opportunity. You guys have shown some strong data in
the 2E population, but now kind of focused on CMC. Maybe just give us a sense for what's kind of left on the CMC side? What you think our timelines
for getting that ironed out? And also just the latest thoughts on what might be needed for registration? Sorry that's the -- [products and customs]
in one.
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: Perfect. I want to spend -- and this is the -- last couple of minutes on the PPMO side. You've got the registrational cohort up and running for 5051.
I guess as we think about this landscape, there are a number of next-gen antisense programs being developed. How do you kind of think about
these programs and positioning of your PPMOs versus some of the other technologies that are coming up on the horizon?
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: Last question, just we obviously know the timelines for the 51 amenable population on -- how are you thinking about the other opportunities in
DMD that run we might hear more about those on [TALENs].
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consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
NOVEMBER 30, 2021 / 6:25PM, SRPT.OQ - Sarepta Therapeutics Inc at Evercore ISI HealthCONx Virtual
Conference
Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst
: Awesome. I think we're already a couple of minutes over. But thank you so much, Doug, for taking the time and looking forward to follow these
updates next year and beyond.
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