Sarepta Therapeutics,, Inc. Common Stock Morgan Stanley 24th Annual Global Healthcare Conference

All right. Well, thank you, Mike. It is a pleasure to be here with you all today. James and I are happy to tell you a bit about what is going on at Sarepta. I have just recently joined Sarepta. I have been here going on 2 months now, but it has been a very exciting and very rewarding 2 months. I think Sarepta has a story that is very unusual, and I think we can actually say unique, even though that word is often overused in our industry, because it is a company that has a cadre of marketed products that are serving patients today and have been doing so for many years, that offer us a number of benefits.

First of all, they offer patient benefits, which is what is most important in our industry, but they also generate revenue that gives us a very strong and stable financial position and a very strong balance sheet. We can use that strong financial position to drive a very innovative and very promising pipeline that has the potential to expand our therapeutic footprint to drive growth, we believe, in the near term and long term. I am sure we will talk about that pipeline and everything that we are doing, for example, in the siRNA space. As I said, this is a setup that you do not find in our industry.

As someone who has spent more than 25 years discovering, developing, and commercializing therapeutics, when I looked at this opportunity, I was compelled by the interplay between those marketed products and a pipeline that I think holds tremendous scientific progress and can make a real difference for patients. I look forward to speaking to you all a little bit more about that as our session continues today.

Yeah. So near term, there are a number of important areas of focus. First of all, we want to continue to support and drive the business for our marketed products. As I mentioned, we have a cadre of marketed products, ELEVIDYS, people obviously are very familiar with, and our PMO franchise, both very important to the company. We recognized that there was some disruption, particularly on the ELEVIDYS side in 2025. Whenever you have a disruption caused by safety events like those that were disclosed in 2025, attention shifts very considerably towards safety, towards risk.

Yeah. It's important in our industry and our business and for patients to focus on benefit risk.

ELEVIDYS clearly demonstrates a strong benefit for patients. You see that in the initial registrational trials. You see it in the long-term studies, and we released 3-year data earlier this year that I think strongly makes that case for long-term benefit. So getting that conversation shifted back towards benefit risk- is absolutely essential because we need to keep in mind that unfortunately, the condition that we're treating with ELEVIDYS is progressive, debilitating, and unfortunately life-shortening.

Yeah. And so one needs to look at benefit risk and the benefit that is delivered.

We focused on recentering that conversation. We have expanded our commercial footprint. We have expanded our medical support for the product, and I think we are in that process of recentering, stabilizing, and continuing to support that business. Driving that business as well as driving our PMO franchise is important to that longer-term strategy that I described. But equally importantly, continuing to drive the pipeline is going to be absolutely essential. We have been serving DMD patients for a number of years, and we want to continue to serve DMD patients in the near and long term. But we want to expand our footprint, as I have said, and we have a very promising siRNA pipeline. I dug in deeply to the data behind our siRNA pipeline before I decided to join.

I think it is truly differentiated. I think the delivery technology that we have available to us drives very high muscle concentrations for programs where that is relevant and also gives us the opportunity to penetrate deep brain nuclei in the CNS, for example, in our Huntington's program. We have extremely potent constructs that can knock down genes effectively. In our space, where target validation is not an issue, in other words, we know what causes these diseases, the ability to deliver a therapeutic to the cell type of interest, to do it with high efficiency, to do it with good safety profile, and to efficiently, in the case of the conditions we are talking about, knock down the gene very efficiently, that is what is crucial to driving long-term success.

Those sorts of data translate into the clinic and translate into late-stage development with a much higher probability than most areas of drug discovery and development. So continuing to focus on that pipeline, driving it forward, driving through the near-term readouts, which we have coming later on this year and beyond as we move those programs we believe into registrational studies, is going to be a core focus. If you look at those two elements of the strategy, supporting the ongoing business and driving the pipeline, we believe those can create a significant amount of value.

Yep. Everybody believes that they are undervalued, but I think we can make a strong case based on our cash flows, based on our pipeline, that there is tremendous untapped potential in this company.

Those two areas of focus are what will unlock that in the near term and then give us the license in the longer term to continue to think about how we grow and expand our business.