Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Q1 2023 Earnings Call Transcript

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Based on our most recent data, that’s about 13.5% of patients would be currently excluded on the basis of having pre-existing antibodies to binding antibodies to rh74. And then there is a subset of patients who would have some of these earlier mutations in a range, in a region that may have a risk of an innate immune response that’ll be less than 5% of patients. So that remainder is the addressable patient population to launch. Now, with that said, I’m going to go ahead and give you our plans for the future as well, because very soon we’re going to be starting a number of studies in an effort to fully build out the addressable patient population to the extent that science allows us to, we’re pretty confident about that. So the biggest opportunity, obviously, is to get to the non-ambulant patients that is extraordinarily important to us and to them, non-ambulant patients don’t have the luxury of time.

So we’ve got to move as fast as possible. We are starting our Study 303 for the non-ambulant population very soon. The goal is to have sufficient safety and expression data from that study to seek an update to our label early next year to get non-ambulant patients in the label so we can begin to dose them and we’re starting as well. Two additional studies for two alternative approaches to clear preexisting neutralizing antibodies and if we’re capable of doing that, then that would also bring back into frame for the ability to dose patients that are currently excluded because they have something that reacts and is a preexisting neutralizing antibody. That’s an extraordinarily important issue as well. Oftentimes, you’ll – when you talk to patients and their families, parents will say, the worst day of their lives was getting the news that their child had Duchenne muscular dystrophy, and that the second worst day of their lives was finding out that their kids are one of these rare kids, about 13% of kids who have test positive for preexisting neutralizing antibodies.

So we need to move fast to try to solve that issue for them. Thank you for those questions.

Operator: Thank you. Our next question comes from the line of Brian Abrahams with RBC Capital Markets. Your line is now open.

Brian Abrahams: Hi. Good afternoon. Thanks for taking my question. Can you expand on what you’ve been hearing in your preliminary conversations with payers? Is your sense that they would be open to paying for 9001 under accelerated approval, or would prefer to wait for full approval? And should we expect sort of a similar mix as with the exon skippers with regards to the proportion of patients initially receiving access? Thanks.

Doug Ingram: I’ll take broad strokes and Dallan you can follow-up if I’m missing anything. First of all, the conversations have gone very well. We’ve been in dialogue with payers regarding the potential for SRP-9001 for many years now. I mean, I can – I think going back as far as mid-2018, Dallan, myself and others were meeting with payers. Our access and reimbursement team have been meeting with payers significantly. The amount of evidence that we have that supports the conclusion that 9001 is a beneficial therapy for kids and it’s going to do a lot of good is very, very robust. So these discussions have gone very well. With that said, I’m going to be very clear, of course, as is the case with rare disease therapies, right now, access and reimbursement is a complicated challenging thing.

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