Ionis Pharmaceuticals Inc at UBS Global Healthcare Conference Summary

The following is excerpted from the question-and-answer section of the transcript.

Question: Elia Merle - UBS Equities - Analyst : <_ALACRA_META_ABSTRACT>Maybe before we jump into the specific programs, you guys have probably one of the largest pipelines of any of the companies I cover. Can you give us kind of a high-level overview of maybe what you view as the most important milestones for the company in the next 12 months? Elizabeth Hougen - Ionis Pharmaceuticals Inc - Chief Financial Officer, Executive Vice President - Finance Sure, absolutely. This is, as you know, a really exciting time to be at the company. We have a very rich late-stage pipeline, and we've been embarking, over the last nearly five years, on shifting our business model so that we're not only discovering and developing our medicines, but we're going to be delivering them to patients ourselves. So our focus in the next, say, 12 to 18 months is going to be very heavily on regulatory actions and new product launches, much as it's been this year as well. So we've got coming up here very shortly in a few weeks, we hope, approval for Olezarsen, one of our most meaningful opportunities in our late-stage pipeline. So we anticipate approval for that drug here on December 19, and we're ready to launch it. We're excited about those opportunities, and we can talk a little bit about that. Following behind that, we hope to have European approval for WAINUA, our drug for ATTR polyneuropathy that's approved in the US and other countries around the world, but we're looking forward to the European approval here shortly for that drug. And then as we get into next year, we have the approval, we hope, for Donidalorsen for HAE in the US possibly in Europe as well and the launch of that drug. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 3:15PM, IONS.OQ - Ionis Pharmaceuticals Inc at UBS Global Healthcare Conference And then we have multiple Phase 3 readouts, two in particular from our wholly owned pipeline. That would be severe high triglycerides with Olezarsen, and that's in the second half of the year. And then Pelacarsen, which is not wholly owned, that's with Novartis. That's a drug in Phase 3 development for Lp(a)-driven cardiovascular disease. And then we have our late-stage neurology asset that's wholly owned, Zilganersen for Alexander disease, we expect Phase 3 data for that. And then a host of mid-stage pipeline readouts. So the next 12 to 18 months, and then well beyond that even, it's a really rich, catalyst-driven event period for us.

Question: Elia Merle - UBS Equities - Analyst : Absolutely. And I know you mentioned there's an internal countdown clock to the PDUFA. After approval, how should we think about sort of how quickly you can distribute the drug? And more specifically -- I know we've talked about how this is an ultra ultra-rare REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 3:15PM, IONS.OQ - Ionis Pharmaceuticals Inc at UBS Global Healthcare Conference condition. In terms of those patients, sort of where are you -- and the prescribers as well, where are you in identifying who the patients are and what proportion maybe you've identified and figured out where they are? So my question is basically, how quickly can this launch?

Question: Elia Merle - UBS Equities - Analyst : Absolutely. And you also have a competitor on your heels in FCS, although you certainly have a lead from a timing perspective. Maybe how do you see Olezarsen differentiated and how you see the competitive landscape playing out?

Question: Elia Merle - UBS Equities - Analyst : And feel free to just skip this question. I'm sorry to ask, but in terms of the label, do you expect to potentially have the non-genetically confirmed FCS patients in there or even just commercial usage there?

Question: Elia Merle - UBS Equities - Analyst : Interesting. I mean that certainly would widen the potential population. But I'll leave it there. Turning to SHTG and the Phase 3 reading out in the second half of next year, but certainly an important catalyst for you guys. What should we be looking to see? I mean we know there's potent lowering of triglycerides. But I guess, what should we be looking to see in terms of maybe other end points?

Question: Elia Merle - UBS Equities - Analyst : Yes, certainly transitioning into a commercial stage company, which is exciting. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 3:15PM, IONS.OQ - Ionis Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Elia Merle - UBS Equities - Analyst : I did want to ask about TTR, but maybe just quickly, I'll ask about expenses. I mean, launching -- I mean HAE is competitive, but also on the smaller side. But SHTG, I mean now we're starting to talk about larger like populations. How should we think about the scale of the sales force buildup and some of the sort of costs associated with this?

Question: Elia Merle - UBS Equities - Analyst : Absolutely. So turning to TTR. Can you just remind us how the CARDIO-TTRansform study differs from a design perspective versus HELIOS-B? And sort of how you think that could position you longer term in the market?

Question: Elia Merle - UBS Equities - Analyst : Absolutely. And how should we think about the latest timing for your base case around when you will get the Phase 3 data?

Question: Elia Merle - UBS Equities - Analyst : Absolutely. And what we saw from Alnylam prior to the readout of HELIOS-B was kind of a tweak to the stats plan, particularly something that enabled them to look at the open-label extension after sort of the primary treatment period. Is this something that you would be considering?

Question: Elia Merle - UBS Equities - Analyst : Great. And something that's been pretty topical lately is the discussion of Part B versus D and the different reimbursement dynamics. I guess maybe first, just before I ask about some of these different dynamics, how much do you think reimbursement will matter? From a prescribing perspective, are physicians going to prescribe the drug that they think is best? Or are there going to be meaningful differences in how the drugs are covered that could affect prescribing? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 13, 2024 / 3:15PM, IONS.OQ - Ionis Pharmaceuticals Inc at UBS Global Healthcare Conference

Question: Elia Merle - UBS Equities - Analyst : Yes. It will be interesting to see this play out, certainly unprecedented in a lot of ways.

Question: Elia Merle - UBS Equities - Analyst : Maybe we can talk about polyneuropathy and early days still, but some of the trends that you're seeing with WAINUA.

Question: Elia Merle - UBS Equities - Analyst : Add-ons, that's interesting. Can you tell us a little bit more about that?

Question: Elia Merle - UBS Equities - Analyst : Interesting. Are you seeing this in the commercial and Medicare segment? Or is it skewing more towards commercial?

Question: Elia Merle - UBS Equities - Analyst : Okay. Sorry, I did put you on the spot on that. Great. Well, turning to HAE, which you are also launching soon, it's a competitive market. Maybe can you walk us through sort of where you see your competitive edge in this space and where you see yourself sitting?

Question: Elia Merle - UBS Equities - Analyst : Yes, absolutely. And maybe just I'll open it up since I know there's a lot of pipeline programs that we could touch on. But maybe what are you most excited about?

Question: Elia Merle - UBS Equities - Analyst : Yes, absolutely. And speaking of the neurology franchise, you have a lot of other interesting programs across tau, Alexander, maybe just with one minute left, a quick overview of those and specifically the time lines there.

Question: Elia Merle - UBS Equities - Analyst : And one last quick one, if I may. How are you thinking about what proportion of these assets going forward do you want to keep to yourself versus potential partnerships?

Question: Elia Merle - UBS Equities - Analyst : Great. Well, exciting time, and thank you so much for joining us.