REGENXBIO Inc. (NASDAQ:RGNX) Q3 2023 Earnings Call Transcript

So we continue to view that the opportunity to enroll in that age range is something that’s going to be important for our profile clinically and ultimately, commercially. And we do think that there’s strong validation in the 4 to 5 and the emergence of more data in 6 to 7 is also sort of helping inform the benefits there. But we really feel strongly that the C-terminal domain, the functionality associated with it, what we’ve seen from our preclinical data and the early evidence that we’ve reported on and we’ll continue to update on in an older patient. I think, is something that we’re excited about for that age range. Thanks, Lisa.

Operator: Our next question comes from the line of Brian Skorney with Baird.

Brian Skorney: Hey, good afternoon everyone. Thanks for taking my question. It just sounds like you’re getting your decision point on pivotal programs with the superchoroidal administration. So just wondering what’s sort of the rate limiting factor here in terms of AVA and ALTITUDE to kind of make a decision you really need like one-year data from Cohorts 6 and Cohorts 4 and 5 to trigger that point. And you’ve been talking very enthusiastically about Diabetic Retinopathy, do you see sort of the pivotal program moving forward in the DR and wet AMD at the same time? Or will one of these indications potentially move forward earlier than the other?

Ken Mills: Yeah. Thanks for the question, Brian. I think, again, like we have a lot of enthusiasm about the recent data update. And there’s a recency effect there. Of course, the AAO was just a few days ago, and I think the new DR data has now been communicated and has been a focus for us in terms of communication in the last few days. With respect — and then we have AVA update coming only in January. I think our vision has been from the beginning that wet AMD Subretinal, wet AMD suprachoroidal, wet AMD for Diabetic Retinopathy. They are obviously all interrelated because of the same pharmacological agent, but each one of them because of the difference in delivery or in the last case and the difference with respect to the potential market and the sort of evidence that’s needed clinically to think about how to transition in a pivotal, they’re all slightly different path, but they derive from the same consideration in variables that we would look at for any kind of advancement of a program like this, almost independent of modality into a pivotal phase.

I always like to point out that we are the company that is the first to have transitioned into a pivotal phase program with respect to an AAV Gene Therapy in a large market indication in wet AMD. And now we also have the strength and sort of the robust decision-making to sort of bring additional variables into the equation with us with AbbVie. Yeah, the decisions though are going to be different and separate between the suprachoroidal, wet AMD and the suprachoroidal Diabetic Retinopathy. I mean they rely on different data sets and different inputs. So it’s certainly possible that they could happen at the same time, but it’s — it’s as likely that those decisions are independent variables and would come forward. But I think the message today is how strongly we feel about reinforcing our excitement about the value of RGX-314 and the opportunity to continue to realize that value and continue to make that focused investment with an extended runway here, I think, really improves the probability of success of meeting some of these milestones in terms of transitioning the pivotal.

Therefore, realizing some of these monetary milestones that are associated with the partnership in a way that is improved, right, just with that extended runway alone, but at the same time, we’ve also started to become really encouraged about the newer and longer-term data that’s been coming out of the investments that have been made in things like AVA and ALTITUDE. So we excited for things to come here moving into 2024.

Brian Skorney: Thank you.

Ken Mills: Thanks Brain.

Operator: [Operator Instructions] Our next question comes from the line of Annabel Samimy with Stifel.