Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) Q4 2022 Earnings Call Transcript

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So it’s very exciting. There’s a really nice team that works on early to mid-stage neurology here. So you’ll hear more about that in the second half of the year. And then we also have some things that can move into our specialty franchise, just like we have done in donidalorsen in specialty. We do have a couple of other specialty products such as sealers for polycythemia vera. So we’re looking forward to seeing some of the — the data there, which is encouraging at this point in time. So that’s kind of our way to kind of market in the near term and in the midterm. And then we work very closely with R&D, as I said, integrating kind of our early-stage pipeline as well to understand kind of what indication strategy look like and how we want to think about going to market with that for ourselves and where we think there’s more benefit for partners to actually again, realize the full potential of the product.

Brett Monia: Thanks, Onaiza.

Operator: The next question comes from Yale Jen with Laidlaw & Co.

Yale Jen : In terms of the Factor B, I know Roche is advancing the study into a Phase III study this half. And I’m curious whether there’s also geographic atrophy indication, whether — how would the company think giving that there is one product is recently approved and the other one is a late clinical stage right now.

Brett Monia: Both programs — thank you, Yale, for the question. Both programs are going very well. Maybe Eugene will want to add to this. But the IgA nephropathy, there’s really nothing new to report there, except that the news we reported late last year and early this year that Roche still plans on initiating the Phase I study Igneproperty is on track. That’s a rare indication. And then for geographic atrophy, it’s going really well, right?

Eugene Schneider: Yes, the enrollment certainly has picked up those pandemic, and we’re very pleased with how the study is progressing. So it is a large study, 1 of the largest Phase II studies we’ve conducted recently. So we’re quite optimistic and also Roche obviously is very interested and keen to make a decision on the next steps for that particular program.

Brett Monia: The Phase II GA study is designed to set up a potential Phase III study based on the almost — the acute study. And if your question was asking about how Roche is going to handle a rare indication versus a broad indication? That’s a question for Roche. I can’t answer that one for you, sorry.

Yale Jen : And maybe one more question, come from investors. In terms of eplontersen in ATTR-CM, you guys already upside the trial study. It seems like that if you have the data later on, you may not do that, but just curious whether there’s still options to change upsize the study if you feel that could be needed in the future?

Brett Monia: We don’t expect to upsize the study. We think we’re right where we need to be, Yale. the study is on — we have the CARDIO-TTRansform label extension study is well underway. We have patients now they’ve mentioned the OLE. All of this is why the timing for the decision we made last year to upsize the study when we did was so important. And as I said in my opening — I think my first question I had — everything is going according to plan with respect to blinded event rates, balance of tafamidis versus naive and enhancing the number of patients with hereditary TTR cardiomyopathy. So there are no plans to further upsize the study. We’re approaching the finish line.

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