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: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Yes. Great, thanks for that introduction. And Doug, as you mentioned, it's been quite a successful year for you with the broader label
for ELEVIDYS. So maybe just talk about, since that label expansion, what you're sort of seeing in trends?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: In the past, you've mentioned there's a couple of key factors that might influence the launch. You've touched on one or a few already,
just in terms of infusion centers. Maybe talk about the status of that, your thoughts about further expanding that or not?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: And another factor you mentioned is just payer reimbursement. So maybe just walk us through what you're seeing there in the time
frame to sort of getting payers on board?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: And just given the much broader patient population now, do you think that could change in the future and you might see more
pushback? Or is that unlikely?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Makes sense. Another factor you talked about is just the supply and manufacturing. So maybe talk about the current status there
and your plans for the future?
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SEPTEMBER 06, 2024 / 1:15PM, SRPT.OQ - Sarepta Therapeutics Inc at Morgan Stanley Global Healthcare
Conference
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: And I wanted to follow up on one of your earlier comments just about the patient journey somewhere between three to six months
from start form to infusion over time, are there opportunities to sort of shorten that more to the three months' time frame as opposed
to the six months?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: And just given the patient journey. Talk about how that impacts for the trajectory of revenues going forward and sort of your level
of confidence in hitting your 2025 guidance?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Yes. And then just given your label has both ambulatory and non-ambulatory patients and I think the non-ambulatory was sort of
a bit of a surprise for some people. How do you expect utilization, at least earlier in the launch? Are you seeing some of those patients
on therapy?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Can you also talk about the ages of the patients you're seeing come on to therapy? Is it a broad range?
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SEPTEMBER 06, 2024 / 1:15PM, SRPT.OQ - Sarepta Therapeutics Inc at Morgan Stanley Global Healthcare
Conference
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Yes. Makes sense. And can you talk about just the ENVISION study in terms of the current status, when that data readout and it's
important?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Okay. Great. And you're also doing some work to further expand or the addressable patient population. Maybe you can talk about
that a little bit?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: You have a big opportunity in front of you in Duchenne, but there's obviously also some competitors looking at other gene therapies
as well. Maybe just share your thoughts there and any potential impacts.
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Got it. And if you could touch just quickly on Europe and the progress you're making there with your partner?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Okay. Great. Maybe we can shift to some of your work you're doing in the pipeline and maybe 5051, your PPMO, maybe remind us
next steps for that program?
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: You mentioned some competitors. Maybe just talk a little bit about how you feel your data stacks up?
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SEPTEMBER 06, 2024 / 1:15PM, SRPT.OQ - Sarepta Therapeutics Inc at Morgan Stanley Global Healthcare
Conference
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Makes sense. And maybe now since you've got the broader label, you started to talk about business development. So maybe just
share your initial thinking there.
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: You mentioned your -- obviously, your experience in gene therapy, is that drive the decision making? Like do you have a preference
for those types of assets or not necessarily?
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SEPTEMBER 06, 2024 / 1:15PM, SRPT.OQ - Sarepta Therapeutics Inc at Morgan Stanley Global Healthcare
Conference
Question: Michael Ulz - Morgan Stanley Co. LLC - Analyst
: Okay. Great. It looks like, unfortunately, we're out of time. But Doug, thanks so much for spending time with us today. We really
appreciate it.
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