Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Q3 2023 Earnings Call Transcript

Douglas Ingram: Yes. Let me answer those questions quickly. First, I don’t think there will be any shift in the desire for this therapy. I think except for patients that are not in the parents of patients that are not in the 4 to 5 year-old range I think are probably even more compelled to want this therapy and not going to wait impatiently as they should be in patient to have this label broadened. We don’t share patient numbers. We’re going to use as our metric for success and the measure of our success on net product revenue. As we said, on the issue of sort of bolus or warehousing of patients, there were certainly some number of patients that physicians were getting together and gathering to ensure that they could preferentially get dosed before they aged out because the label, obviously, as you know, restricts the dosing to 6 and below 6% and 5%.

But we’ve had a steady stream of new start forms since the approval. And so looking forward, we have a steady stream of start forms that are working through the process. And one other thing people have been asked, but I’ll say it done the team, Dallan and others have done a brilliant job on site activation. I think our goal, as you know, our goal and aspirationally been to have 50 sites ready to infuse at launch. And then very aspirationally, we thought perhaps by the end of the year, sometime next year, we can get all the way to 70 sites, but we’re at 70 today. So the team has just done a brilliant jump making — getting sites ready and up and running and there is a steady stream of start forms to work through.

Uy Ear: Thank you.

Operator: Please standby for the next question. The next question comes from Danielle Brill with Raymond James. Your line is open.

Danielle Brill: Hi guys, good afternoon. Thanks so much for the question. Doug, so we spent a lot of time discussing EMBARK efficacy at data I’d like to switch over to safety. I recall myocardis events being discussed at the AdCom, including one event that had occurred in EMBARK at the time. Just curious if there are any additional safety events of this nature that occurred in the study or any other SAEs leading to hospitalization? Thank you.

Douglas Ingram: Thank you for your question, Danielle. I will turn the call over to Louise to respond.

Louise Rodino-Klapac: Yes, we did not see any differences in the types of SAEs or the frequency of the SAEs that was one of the most reassuring things EMBARK was the continued safety profile and now taken together all of the previous trials, we have a large safety data base that’s consistent on those trials.

Operator: Please standby for the next question. The next question comes from Ritu Baral with TD Cowen. Your line is open.

Ritu Baral: Good afternoon everyone. Thanks for taking the question. Doug, could you — and maybe, Dallan, walk us through how you see patient numbers for the different scenarios that are regulatory experts suggest could are in play essentially for label expansion. You mentioned 10% of your PMO sales are for 45 — those are covered — but how would you segment it by maybe 6 to 7 year-olds if FDA wants to go down that route again? And how that number would change you do got the full ambulatory population, which per my calls now extends to like 12 years of age.

Douglas Ingram: For ambulatory?

Ritu Baral: Yes. Like that’s the average age of loss of ambulation now 12 13.

Douglas Ingram: That may be correct. I’ll have Dallan confirm that in a second. Broadly speaking, I mean, look, first of all, I want to be very clear, we’re not looking for a label expansion to go from 4.5 to 4 to 7, and we don’t think there’s any reason scientifically one would be limited to 4 to 7, given the data. And we’ve never seen that before. Once — you’ve never seen in any other label for Duchenne therapy, these sorts of age-related restrictions. But answering a broadest question, the all ambulatory versus all ambulatory and non-ambulatory is about 50%. So there’s — so the ambulatory population is about half of all patients and the non-ambulatory the other half, of course, — and so that, to our mind, is the big cut. But Dallan, is there anything else you’d like to say on this topic.

Dallan Murray: No, I think you covered it, Doug. Our goal is to target all the patients that we can get in the entire population, read you to your question about average age of loss of ambulation. I think you’re in the right range. But the KOLs have been talking a lot about the definition of loss of ambulation too because, as you know, there’s heterogeneity. So you have some patients walking at much older ages, and we have a whole cohort of patients who’ve been treated with the PMOs for years now that are going to, we believe, have older ages of loss of ambulation as well. So it won’t be — we believe the ambulatory population won’t be defined by age. But as Doug said, our aspiration is a broad label and targeting all of the patients who will be eligible.

Ritu Baral: And is that 50% of your PMO sales too?

Douglas Ingram: Yes. The ambulatory versus non-ambulatory split, I think it’s in that range of 50%. I think the access is more difficult in the older patients. So on average, we’ve got a higher penetration in the younger given population.

Ritu Baral: Great. Thank you.

Operator: Please standby for the next question. The next question comes from Mike Ulz with Morgan Stanley. Your line is open.

Mike Ulz: Good afternoon and thanks for taking the question. You mentioned now that you have about 70 sites are active. Can you give us a sense of what percentage of those sites have actually prescribed ELEVIDYS so far? Thanks.

Douglas Ingram: We are going to — apologies I’m going to frustrate you. We’re going to resist that level of detail either on numbers of sites are infused or probably more specifically number of patients infused at any one time. As we have done for the last seven years, and I think we’ve done generally over time to good success with folks. We use net product revenue is the marker for success and uptake and the like. So apologies for that, Mike.

Operator: Please standby for the next question. The next question comes from Tim Lugo with William Blair. Your line is open.