Sarepta Therapeutics Inc at Evercore ISI HealthCONx Conference (Virtual) Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Perfect. Clearly, DMD gene therapy is the main area of focus for the company broadly. But before we get more data there, we're going to get an update on your first PPMO program. So I wanted to actually start with that. Maybe you could just give us a quick sense for differentiation there and the first trial that you're running for 5051?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : And so to your point, you said you'll definitely be showing the 20 mg per kg cohort. You recently announced you've gone higher to 30 mg per kg and potentially going even beyond that. How much data should we kind of expect in the upcoming readout later this month?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. I think as we think about this platform, differentiated ASO technology could be quite valuable. I think with your DMD gene therapy and PMO programs, it can be a little nuanced to think about exactly how PPMO grows the top line. Like, obviously, it's a technological improvement over the PMOs. But why was DMD the right place to look at here? And how should we think about -- if you do have a differentiated platform, where you'll go next with the technology?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. Wanted to switch gears to 901, obviously, an extremely important update ahead. There have been a few recent updates on the regulatory and strategy side for this program. So maybe to start, just give us a sense for the recent updates on the manufacturing potency discussions that you had with the FDA? and where you, ultimately, landed on that? And then also on the decision to start Study 103 instead of 301 for now?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : So I guess COVID aside and then the rationale kind of makes sense, but COVID aside, could you hypothetically get Study 301 up and running now? Is there anything else needed from the FDA at all to kind of get that trial in place? And if you had to kind of guess, how much time do you think the 103 approach stays on the regulatory side in the U.S.?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. So just thinking about the up to 10 patients that you're going to be looking at in 103. I know you haven't spoken formally to the FDA about the bridging strategy approach and what they kind of want from those patients. But as we think about expression and variability, how wide can that range be? Like what did you see in Phase I for the 4 people who, obviously, did respond and were seeing expression? And how variable is it based on where you biopsy? And how do you kind of control for some of those elements as well to try to minimize other factors that could affect the variability beyond just the product being given?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. Just based on time lines for 102, and when you kind of completed enrollment, it seems like it's potential -- potentially reasonably likely that you might get those results before you get the 103 results. In that situation, do you go to the FDA after 102, assuming it's positive and talk about the strategy then? Or kind of wait for the 103 results and go to them with the full package?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. And so if the bridging strategy works, with the current data package, you won't have tested 901 yet in the nonambulatory population. Do you think that, that might restrict at all the kind of label you get in terms of age range and ambulatory status until you kind of run some data in that population as well?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. You recently presented the 2-year update from -- on the Phase I study. And I think the data there was pretty compelling. The patients now are all kind of in the 6- to 8-year age range, which, at least, based on natural history, you'd kind of expect some to start declining. What do you think is reasonable to expect for year 3 and beyond for those patients? And how frequently do you think you'll kind of update that data set as well?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. On Study 102, I know you haven't given us all of the assumptions behind powering. But maybe just a quick overview on what you had pulled us for how well the trial is powered? And anything you kind of said on what you're assuming for effect size and (inaudible)?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. So I guess, to your point, we kind of sit here, we see these NSAA plots, and I'm sure you've obviously seen them as well. But they're a little all over the place, but we're behind our desk. So we're looking at a sheet of paper, which is obviously not a reflection of patients in the real world. So maybe you can talk a little about why the extense of variability in the plots that we're looking at may not be an accurate representation of what we should expect from the patients that you've enrolled in 102? If there's enrollment criteria in place? Or anything like that, that really should bring down that new deviation?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Can you remind us what the extent of the blinded data is that you guys get from the trial? Is it safety, efficacy, [dose]? And if it includes some read on efficacy, is there a way to kind of look at the data that you've got so far and have a sense for is this range of variability, obviously, not knowing if it's placebo or treated is in the range of what you would expect?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Sounds good. On the safety side, obviously, you guys seem to, so far, have a differentiated profile compared to the AAV9 approach in. What's cumulates thinking for why rh74 seems to have this better safety profile? And how to know that it's something intrinsic to the vector as well -- as opposed to some difference in manufacturing or other factors to consider?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : So assuming 102 is positive, the 103 biopsies look good with expression in the range that you kind of hoped for, do you think you can take that package and go to other regulatory agencies beyond the FDA like in Europe or elsewhere in the world as well?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Got it. Last question on manufacturing capacity. What's kind of your latest thinking on how many patients you think you'll be able to treat at launch? And then 3 to 5 years out How you're thinking about continuing to build capacity on the manufacturing side?

Question: Maneka Mirchandaney - Evercore ISI Institutional Equities, Research Division - Analyst : Awesome. I know, there's still quite a bit more going on in the pipeline. But I think that's all we've got time for today. This has been really great. Thank you so much, Doug and Ian, for the time and very much looking forward to all of the updates ahead.