Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q2 2023 Earnings Call Transcript

We were able to get their Lp(a) levels down below the threshold associated with CBD. So really, we want to maintain that efficacy. We want to reproduce that efficacy and a potential follow molecule, but what we’re really focused on is pelacarsen. We’re very confident in our ability to deliver a molecule with semiannual dosing to be annual dosing, utilizing a mixture of our technology capabilities, and that is the focus with Novartis. And we’re going to be working hand in hand with them to bring the best molecules forward to follow on pelacarsen.

Kostas Biliouris: Sorry. No, I was going to ask approximately how many behind is the follow-on molecule compared to pelacarsen?

Brett Monia : Yes. We haven’t stated — discussed that publicly Kostas about what the timelines are. And obviously we’re still in research phase. So it would be a little risky to actually speculate on that right now.

Operator: The next question comes from Mike Ulz with Morgan Stanley.

Mike Ulz : Just on eplontersen in cardiomyopathy, maybe you can comment on the recent Bridge data and sort of how that impacts your thinking in terms of the CARDIO-TTRansform study? And then maybe secondly to that, is there a potential for you to maybe stop the study earlier? Or is the thinking still to go through sort of the full results there?

Brett Monia : Sure, Mike. So BridgeBio data, obviously, good patience to have another potential option for TTR cardiomyopathy. To state the obvious, we need a lot more data. We need to see more data. We’re looking forward to seeing more detailed data and I think they’re planning to present later this year. The demographics of the studies supports our trial design, reinforces our trial design, gives us even greater confidence in our expanded trial design, which we’re going to — which we’re going to have — we have now as we have completed enrollment, a very good balance between tafamidis patients [indiscernible] as well as naive patients. So it supports that. CV mortality — overall mortality is very important in this population. And maybe I’ll ask Onaiza to just really expand as on the importance of this from a physician standpoint, from a payer perspective.

Onaiza Cadoret: Yes. So as you know, eplontersen Michael, that we love the breadth of our trial. We believe we’re going to — it’s a landmark trial. It’s going to generate the significant amount of data to really go in and arm the physicians with the data that they need to actually treat these patients. This means anything from EHA Class I, II and III, so mild to severe hereditary and wild-type naive to silence or therapy naive to cardiomyopathy therapies and TTR as well as on top of standard of care tafamidis as well. We really love our endpoints. We have done a fair amount of work in recent times to really understand the difference in terms of how physicians view mortality versus cardiovascular death. And that is actually a more meaningful cardiovascular endpoint, along with the richness of cardiovascular hospitalizations and then some of our secondary endpoints, obviously, in 6-minute walk and other measures that are going to be at play.

But the endpoint is just actually really meaningful. So all in all, we are really excited about our data set, our trial design and looking forward to seeing the output of that in the coming times.