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

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Gary Nachman : First, a few more on eplontersen. So what do you plan to do with the 66-week data when that’s available, talk about the timing of filings ex U.S.? And does that impact the PN filing in the U.S. at all? Or is that completely separate? And then if you have the PDUFA late this year for PN, how soon would you and AZ be able to launch the product? And then on cardiomyopathy, what’s the likelihood that you might have early readouts for that data? And then I have a follow-up for Beth.

Brett Monia: Okay. So maybe I’ll ask Onaiza after I touch on 2 of your other questions to talk a little bit about launch planning for planters in PN. The week 66 data we’re planning to present in the first half of this year at a medical meeting. Of course, we’ll put out top line results ahead of that as soon as we have it. We’re very much looking forward to that. That data will be very important for ex U.S. filings for potential approval for eplontersen, and that is the plan is to file — I have additional filings for approvals outside the U.S. with week 66 data as a key component of those filings. We don’t see any meaningful impact of the week 66 data for the NDA that’s currently under review by the FDA based on the week 35 data.

We don’t plan on submitting a supplemental NDA with that data at this time. The data that — the week 35 data, we believe, is very more than sufficient to get the outcome that we desire by the FDA for eplontersen in polyneuropathy, doesn’t rule out the possibility of a future supplemental NDA after approval. That will be data driven. Regarding the cardiomyopathy early readout, we will continue to monitor any changes in the landscape — the competitive landscape that could influence a decision by Ionis, AstraZeneca to read the study out earlier. We also will be focused on blinded event rates and patient demographics that will — all that will be driven — will be factored in to a decision to potentially read the study out early. We do have several — we do have the option to read the study out early based on a review of all of that.

Onaiza, maybe you could talk a little bit about the late year PDUFA date that we’re anticipating and then plans for launching eplontersen?

Onaiza Cadoret: Yes. Gary, we’re preparing for success for the PN launch. So there are a lot of pre-commercial investments that are already being made for the U.S. We have, as you know, a field medical team that’s been in place over 2 years, really ensuring that there’s enough disease education out there over the last 2 years. We plan and have already actually hired out our field directors for the nurse educator team. And then as we look to the second half of the year and we hear back on the 74-day filing, Astra will start building out the sales capabilities. So basically, we are going to be very ready to go upon approval and not have a lot of time between approval and launch as well.

Gary Nachman : Okay. Great. Very helpful. And then for Beth, just the greater than $575 million revenue guidance. You talked about some of the key factors directionally. So will the overall mix and quarterly cadence be similar to 2022? And then how will you be reporting the Royalty Pharma agreement? How is that factored in the guidance? And then can you still hit the guidance if tofersen or eplontersen are delayed into next year at all.

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