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

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A €“Brett Monia: Thank you, Yaron. So for Factor B, and Eugene, you can chime in. There has been those studies that have been terminated that didn’t conclude to their natural €“ comes to their natural conclusion, the design conclusion of the study. We did a Phase I study €“ maybe that’s what you’re referring to, that can Phase I. We have a single dose and the multiple 2 Phase I is a single dose and multiple dose as your standard to measure safety as well as pharmacodynamic activity reductions in Factor B, that’s what set us up for the current geographic atrophy Phase II study, which, as Eugene said earlier, nrolment has really picked up and is going very well. I’m not sure I can say much about it. There have been no study terminations that were not planned for the Factor B programs either in GA or IgA nephropathy. The program is going really well. Onaiza, do you want to talk a little bit about IRA?

Onaiza Cadoret: Yes. So as you know, we’ve kind of been looking at the implications of the IRA for our portfolio. For eplontersen specifically, as you know, there’s an orphan direct designation exclusion for single disease orphan drugs. And for eplontersen, both indications of PN and CM qualify as a single disease. And that is because of the way we got the ODD exemptions. Our orphan drug designation is for ATTR, not necessarily for one indication and the other. So you should think that through as you’re thinking about that, and we would then be excluded from that maximum fair price negotiation as well. The other thing to keep in mind is this is a really good thing for patients. In a way, we took away the disadvantage for patients for out-of-pocket costs under Medicare Part D €“ so the so-called donut hole catastrophic care really kind of goes away, and we do believe that really allows patients to kind of make the right choice for their product, and not leave it up to kind of cost between a Medicare Part D or a Medicare Part B benefit as well.

So we think both of those are playing really well for eplontersen as we look into the IRA implications.

Operator: The next question comes from Gena Wang with Barclays.

Gena Wang: Just a few very quick ones. First, I wanted to confirm that the voters milestone will be reported as part of the R&D revenue. Second, regarding the manufacturing facility construction, what is the asking cost facility? And then lastly, quickly regarding the CARDIO-TTRansform. I remember you mentioned in the past that you’ll go in patient teams. Can you remind us a percentage of patient hereditary patients? What is the goal regarding the percentage of patients in the final?

Brett Monia: So Gena, you were breaking up a little bit, but I think I got your questions. Thanks for the questions. Beth, do you want to take the punters milestone and the manufacturing facility?

Beth Hougen: Sure. Absolutely. Yes. Thanks for the question, Gena. So eplontersen approval milestone will be reported as an R&D revenue item. And on the manufacturing facility cost we’re anticipating that will be about $350 million. We are already well along in the construction process with design, programming, engineering, architectural efforts underway. And so you should anticipate that cost to be spread out over the next several years. We expect to be manufacturing API in that facility and up and running in late ’25, mid-’26 time frame.

Brett Monia: And then for CARDIO-TTRansform, I’ll ask Eugene to comment on what our targets are goals for hereditary, but for the tafamidis, again, you broke up a little bit, but our goal, as I think I stated earlier, is to get a relatively equal balance between naive patients and tafamidis patients. And right now, the upsizing of the study is delivering exactly that. We’re well on our way. Hereditary?

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