Ionis Pharmaceuticals Inc at Bank of America Global Healthcare Conference Transcript

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

Question: Jason Gerberry - BofA Securities - Analyst : Yeah, we did a dinner last night and a lot of focus is on your APOCIII targeted program, which is more near term, but you have a lot going on both this year and next year. So I don't know, one of the challenges with Ionis is you have a lot of programs and you got to hone people in and focus them in. And so where -- as we started our conversation as I imagine. I know where it's going to go, but I figured it's good for the group to hear it.

Question: Jason Gerberry - BofA Securities - Analyst : Got it. Okay. I probably won't spend a ton of time on FCS just because ultimately, it's going to be usurped by SAT. But nonetheless, I think it's important over the next two years before sHTG comes online that investors see Ionis can go out and commercialize the drug and build a market and execute. And so as we think about what the launch looks like, this is a market where a bit of wide range maybe in epidemiology out there. There's questions about how many patients are in the treatment pool versus sort of the heavy lifting to kind of build and pull more patients into the treatment pool. So how much of it is the doctors got these patients that are in there in the office maybe recognize or diagnosed versus patients needing to be more aware of symptoms and coming in and now realizing they have a treatment option available to them.

Question: Jason Gerberry - BofA Securities - Analyst : Sorry, I guess I was more meaning the FCS opportunity and are those patients in the pool -- treatment pool? Or are they needing to being more aware of treatment options to come into the -- those with either genetically or clinically defined FCS.

Question: Jason Gerberry - BofA Securities - Analyst : Yes. And are you surprised at all? I don't know if there's any anecdotes you share in terms of like since you've been out in the market, how many patients did these HCPs tend to see with FCS. I'm just wondering how that process is going. You put up a maybe better-than-expected 1Q. And so I guess I'm wondering how to think through kind of what the next few quarters or two years look like.

Question: Jason Gerberry - BofA Securities - Analyst : And do you see a meaningful difference? I know you're in the processing a European approval of the markets in terms of how the products are going to be viewed by the ultimate payer in terms of how policies will be set around utilization. You've got the ultra-rare pricing point in the US. I imagine when we magnitudes of order different in the OUS setting, but I don't know how you'd sort of maybe frame the two different market opportunities?

Question: Jason Gerberry - BofA Securities - Analyst : Okay. Well, pivot to sHTG, it's an important readout. I believe it's 3Q, where you have your Phase III data readouts for this program. And curious how you'd frame clinically meaningful benefit in this setting, be it either trigger lowering or proportion of study subjects getting to a more normal threshold, which is something that we often hear from physician KOLs that the ability to get patients out of the danger zone is really important to them. So maybe ahead of the data, I'm just kind of curious how you frame a clinically meaningful signal here.

Question: Jason Gerberry - BofA Securities - Analyst : So you have a patient presumably who maybe get to an omega-3 or a fibrate and get 20%, 30% reduction and then you're getting that patient, you're taking them down another 50%.

Question: Jason Gerberry - BofA Securities - Analyst : Yes. So when you hosted a KOL webinar recently on sHTG. And one of the things that I took away as your market segmentation is that like you really see the opportunity here is the patient who's highly afraid of another pancreatitis event. And so likely to adopt therapy as a means to mitigate and prevent that from happening. So you're going to generate data on acute pancreatitis. We all know that the event rates are a little fuzzy, right, to characterize, but you are seeing event rates on a binded basis. So that's encouraging. So I'm just wondering if you can talk about what we'll expect to see at least in 3Q I know that you've talked in the past about pooling the two studies so that you can have a more robust data set around AP or acute pancreatitis. And so I'm wondering if when we get that update, we'll be able to have a sense of if you have a supportive data set around pancreatitis, at least on a numerical trend, which is important, I think, to both US and ex US markets?

Question: Jason Gerberry - BofA Securities - Analyst : So is the challenge to having all that data dedicated by 3Q, the longer 12-month follow-up period and maybe not having all of that data in-house, whereas you have the six month triglyceride lowering data in-house for sure. I'm just trying to maybe follow up on that point around maybe --

Question: Jason Gerberry - BofA Securities - Analyst : Okay. So sounds like 50% placebo adjusted triglyceride-lowering at least the numerical trend on acute pancreatitis and safe, that would be a home run for you. Is that a fair?

Question: Jason Gerberry - BofA Securities - Analyst : And so I think you've framed maybe a familial patients or so that sort of are in that higher risk buckets of either 80 milligrams, the triglyceride level over 500 with a prior event. So that would be maybe the initial kind of low-hanging fruit segment to go after. And then there may be additional broadening of the market opportunity subsequent to that? And I don't know, is that going to be data driven by subsequent kind of Phase IV marketing studies? Or how would you kind of outline the initial opportunity and then how you may broaden that over time?

Question: Jason Gerberry - BofA Securities - Analyst : I think you've mentioned, if you're addressing a market of that size, the ashore rare pricing kind of paradigm really can't exist anymore. And you've talked -- think in the [50] to 20,000 as sort of a tentative preliminary number. Do I have that right in terms of how -- where do you see kind of like the price range ultimately within sHTG if you had to put brackets around it?

Question: Jason Gerberry - BofA Securities - Analyst : Okay. And any US, OUS particulars with this data set that we need to be mindful of in terms of the filings and how -- what the regulators care more about do the studies broadly support kind of a global filing strategy?

Question: Jason Gerberry - BofA Securities - Analyst : Okay. The shift to pelacarsen, the Lp(a) lowering approach as a secondary prevention strategy for cardiovascular disease. You've got a Phase III outcome trial through your partner, Novartis, looking at MACE outcomes. I generally believe like there's a perception that this trial is maybe more enriched than the Amgen OCEAN trial in terms of patients and some of the events that were explicitly outlined to get into the trial, even though there were some differences, I think in the LP(a) levels at baseline. But I think you've published some baseline demographic data. How do you see this trial ultimately enrich for success, and you've been through two interims now you're going to, I guess, a final update next year, how do you think things are tracking generally with this trial?

Question: Jason Gerberry - BofA Securities - Analyst : It was initially published something like 900 to 1,000 events need to do occur. Are you, I imagine, privy to the blinded data that the events are tracking to where they need to be over data by the time you're hoping.

Question: Jason Gerberry - BofA Securities - Analyst : Okay. And maybe talk a little bit about what you see as the clinical support versus the genetic data that supports the role of Lp(a). I think primarily what do you ascribe greater weight to here in terms of confidence that this target, I think we've seen with other genetically defined targets like HCG all the outcome trials didn't work. So as you say, it's a landmark trial. It's the first trial in the space. And I guess that's the main risk that people focus in on.

Question: Jason Gerberry - BofA Securities - Analyst : Can you remind us how you've kind of defined this addressable market and one thing that we hear from some physicians is these Ionis patients if they're just treating to prevent an MI event, oftentimes, patients just don't treat, right? And so I don't know there's how would you kind of segment and define this market given those dynamics that are out there, and we've seen some analogs with other drugs that address risk factors in these different? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. MAY 14, 2025 / 4:20PM, IONS.OQ - Ionis Pharmaceuticals Inc at Bank of America Global Healthcare Conference

Question: Jason Gerberry - BofA Securities - Analyst : Right. Well, we're pretty much out of time. So Brett, thanks so much for joining us then.