Sarepta Therapeutics, Inc. (NASDAQ:SRPT) Q4 2022 Earnings Call Transcript

Colin Bristow: Congrats on all the progress. Another one on the Adcom or lack thereof. You — previously, you had stated that your — one of the focal points of the outcome you’re anticipating would be the surrogacy of truncated dystrophin. Did FDA comment on its comfort around this? Or is this something that’s part of an ongoing dialogue that is just part of the review process? And then maybe just on the manufacturing supply side, if you are approved on the PDUFA, can you just talk about the number of patients you expect to be able to supply at launch and how this will build over time?

Doug Ingram: Sure. First, I would say that entire discussion, that’s kind of the fundamental issue in the review is the use of short and functional dystrophin is reasonably likely to predict a clinical benefit. As it relates to the Adcom comments, we see this — we obviously see the decision to lead Adcom as a positive. We had a very productive mid-cycle review. We’re not going to speculate on what this means from the probability of success. But what we would say is that given that we know that there are no significant safety issues, that there isn’t going to be an Adcom. We can then spend our time really focusing on answering any remaining questions preparing for and executing our preapproval inspections and doing that successfully, making sure that we’re launch-ready and building inventory for launch.

We haven’t given exact patient numbers as it relates to manufacturing but what we’ve said and we stand by it, is that our goal is to launch this therapy and serve this community without back orders with all patients getting the therapy as soon as they are able to get it from an access and reimbursement perspective. And we want to be in a position to do that and we will be.

Operator: Our next question comes from Matthew Harrison of Morgan Stanley.

Matthew Harrison: I guess 2 just follow-ups for me. I know we’ve touched on many of the major issues here. But first, just on manufacturing. Can you just talk, to the extent you can, in a bit more detail? Are these multiple facilities or just lines in the same facility that are being inspected? How comprehensive is this inspection versus maybe what you were expecting or thought you might see? And then just secondly, on the inventory and the supply that you are building now. Is there any chance or any reason that the supply you’ve started to build now could not be used? Could you just talk about supply you have now versus supply you’re continuing to build.

Doug Ingram: Yes. So first of all, on the inspections, there are 3 separate facilities. They are exactly as we anticipated. In fact, I would say none of the questions that have been posed and none of the inspection notices that we’ve received have been at all a surprise. We’ve been very well prepared for them. So we’re in good shape there. From an inventory and supply perspective, we’re on track. And no, we don’t think there’s any significant risk that inventory that we build will be able to be used commercially; so we’re tracking there. A lot of work to do as an organization. Site readiness is a significant issue. Building inventory is a significant issue. Completing this review and satisfying any remaining questions or comments or analysis the FDA is extremely important to us and we need to focus on that as well. But the team is very, very focused on all of this and we’re working overtime to ensure that we have a successful BLA review.

Operator: Our next question comes from the line of Tazeen Ahmad of Bank of America.