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: Gena Wang - Barclays - Analyst
: Okay, great. So maybe we will talk about DMD data. Recently, you presented a very comprehensive and impressive data, and I think it generated
a lot of excitement. So maybe walk us through like what -- I think one part is it's also very consistent.
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MARCH 13, 2025 / 1:30PM, SLDB.OQ - Solid Biosciences Inc at Barclays Global Healthcare Conference
I know it's only three-patient data, but it was very consistent, high level expression. And you did have a lot of measurements showing the consistent
potential clinical benefit.
So maybe tell us maybe the next step and what's the status there in terms of a patient enrollment And with current data, I'm pretty sure you're also
communicating with the patient-doctor community. How would that help you expedite your enrollment process?
Question: Gena Wang - Barclays - Analyst
: Okay, super helpful. So maybe talk about the additional 10 to 12 patient -- sorry, total 10 to 12 patient enrollment. You already enrolled seven as
of February 17, which are like three to five patients, I think previously you said, in mid-April. We're only one month away. How's the status there?
Question: Gena Wang - Barclays - Analyst
: So the extra three to five patients, are you on track to complete this enrollment mid-April?
Question: Gena Wang - Barclays - Analyst
: That's good. Yeah, since you talk about ex-US, I think you, total, have six sites, five US, one ex-US, right, Canada? Yeah, Canada.
So what is the plan? And then also, I just want to make sure. Like, right now, you don't have any waiting period. You can enroll as fast as you want,
right, as long as -- yeah.
Question: Gena Wang - Barclays - Analyst
: So then for the -- okay, so for the 7 to 12, you're just emerging sentinel dosing. So you can dose as fast as you can. And you are not planning to
dose higher than 12, right, for the pivotal study?
Question: Gena Wang - Barclays - Analyst
: So for the 30 patient, mainly it's 4 to 12 and you would not dose beyond -- outside of this age range, right?
Question: Gena Wang - Barclays - Analyst
: Right. So that would be a Phase 3 study.
Question: Gena Wang - Barclays - Analyst
: Okay, sorry. Phase 3 study, separate cohort, right. So the Phase 3 study -- sorry. So the Phase 3 study, Bo, you mentioned 0 to 18 age. So maybe the
exact age (inaudible) Phase 3, what is the age?
Question: Gena Wang - Barclays - Analyst
: Right. So going back to Bo's comments, separate cohort, 0 to 18 ages. What size will be that cohort?
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MARCH 13, 2025 / 1:30PM, SLDB.OQ - Solid Biosciences Inc at Barclays Global Healthcare Conference
Question: Gena Wang - Barclays - Analyst
: So just like a rough idea, like 10 patients, 20 patients for that separate cohort.
Question: Gena Wang - Barclays - Analyst
: So like the 0 to 18 patient cohort, how many patients you are thinking? And then the purpose, I'm pretty sure you wanted to have a broad label,
potentially. And how many you think that will be sufficient to serve that purpose?
Question: Gena Wang - Barclays - Analyst
: Okay, that's fair. Okay. And then -- oh, so I think we're talking about the sites, active sites. So one part, do you think also -- like, any plans on how
many sites you wanted to activate for the pivotal study? Do you need additional sites in order to expedite?
Question: Gena Wang - Barclays - Analyst
: Yeah, pivotal. And then Phase 3 will be the next step, right?
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MARCH 13, 2025 / 1:30PM, SLDB.OQ - Solid Biosciences Inc at Barclays Global Healthcare Conference
Question: Gena Wang - Barclays - Analyst
: I see. Okay. So likely existing sites.
Question: Gena Wang - Barclays - Analyst
: Are these sites -- yeah. So like, we talked to some doctors. They have a logistic capacity. They can only treat one to two patients per month, but
those for the commercial. So here, maybe --
Question: Gena Wang - Barclays - Analyst
: I think for them, maybe it's because all the logistic, the insurance, would take like several months. So in your case, like all the sites you are active
now, do they -- like how big is the patient pool? Are they also treating the -- providing the commercial drug to the machine?
Question: Gena Wang - Barclays - Analyst
: Okay, good. Next step -- okay, the time. Yeah, okay. You already mentioned that. We have a few more minutes.
You do have tons of other programs, so maybe talk about the CPVT, the R&D first half of '25, the steps to submit the final steps to take to submit
the R&D where you are. And then maybe if you can share any initial thoughts on the clinical trial design.
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MARCH 13, 2025 / 1:30PM, SLDB.OQ - Solid Biosciences Inc at Barclays Global Healthcare Conference
Question: Gena Wang - Barclays - Analyst
: Right. Going back to FA, like, any initial doctor-patient feedback regarding the administration part?
Question: Gena Wang - Barclays - Analyst
: Good. I think we are on time. Maybe last quick question on the manufacturing. I know part of the great data is also contribute to the high-quality
manufacturing process. Maybe give us a little bit color on the scalability of the commercial supply. I think you kind of alluded to it at the beginning,
but --
Question: Gena Wang - Barclays - Analyst
: Okay. Great. Well, thank you very much, and we look forward to the data update.
Question: Gena Wang - Barclays - Analyst
: Thank you.
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