PepGen Inc. (NASDAQ:PEPG) Q4 2022 Earnings Call Transcript

Unidentified Analyst: Hey, guys. This is on for Tazeen. We have had a quick clarifying question on CONNECT2. What is the €“ what endpoints are you planning to measure? Is it going to be dystrophin levels? And then how are you thinking about how the potential approval of gene therapy for DMD might affect how the agency is thinking about accelerated approval and maybe enrollment for DMD patients in the U.S.?

James McArthur: So, perhaps I can first take the second part of your question and then I will turn the endpoint part of your question over to our Head of Clinical Development. We think that the potential for approval of a gene therapy is beneficial for patients. It could provide a baseline that could be beneficial, particularly in younger patients. And that said we and many others believe that the treatment of Duchenne muscular dystrophy is going to require a polypharmacy approach with multiple therapies to truly deliver on meaningful levels of dystrophin and clearly, the level of the type of dystrophin being delivered in it with an exon-skip product which is almost full length in nature is obviously quite a bit different than that of a microdystrophin. So we think that there is a great opportunity here to deliver on meaningful change for patients. And with that, I will turn it over to Michelle.

Michelle Mellion: Yes. So in terms of the design of that global randomized controlled clinical trial, what we will be looking at in this clinical trial of course is dystrophin production as well as safety. We will also be including a broad array of clinical assessments to give us an idea of how the increased dystrophin production does affect these assessments and how we can improve performance on these assessments. And we will leave that in further discussions with regulators and the information that we have from them is that is timing along with evaluation of these endpoints maybe important for an accelerated approval.

Unidentified Analyst: Okay. Thank you.

Operator: Thank you. Our next question comes from Laura Chico with Wedbush. You may proceed.

Laura Chico: Hi, good afternoon. Thanks very much for taking the question. I guess I wanted to ask perhaps a different one around the regulatory landscape. So there will be an advisory committee meeting coming up soon for a DMD candidate. I am wondering though do you think there is any discussions that could emerge around dystrophin as a surrogate endpoint? Do you see the landscape changing in terms of FDA’s willingness to accept it? And then I just wanted to verify on CONNECT2, could you reiterate again what was the timing for the assessments that you are going to be performing there specifically on the dystrophin productions? Thanks very much.

James McArthur: So, let me first touch on the regulatory component. And again, I will turn it over to Michelle for the readout from dystrophin their timing. So our understanding is that the upcoming advisory committee meeting is focused on microdystrophin. As I mentioned before, microdystrophin is a very different product than the slightly truncated dystrophin product that is produced by an exon-skipping agent on which there have been four approvals to-date. The product that is produced by exon-skipping, is perhaps 10% to 20% of the native molecule size comprising most of the normal domains present in the normal dystrophin protein and is a molecule that we know is functional in Becker patients, if expressed at high enough levels. And that’s why we believe our approach has the potential to be transformative for patients. With that, I will turn it over to Michelle.

Michelle Mellion: Yes, with that dystrophin production, we will be looking at more performing muscle biopsies at baseline and at week 25. So, this will give us an idea of dystrophin production after approximately 24 weeks of treatment.