Ionis Pharmaceuticals Inc at Wells Fargo Healthcare Conference Transcript

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

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Great. So I think for people who are familiar with the Ionis story, they appreciate the company is at a very important stage today. You're looking to commercialize your first wholly-owned product in just a few months. And next year, we'll be hearing about data from a couple of indications affecting millions of people. So those are all big events. I mean at this juncture, could you talk about the setup of the Ionis story? And what's your vision for the company in one to two years from now?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Well, that's a lot on company's plate, very exciting. Maybe let's just start with ATTR cardiomyopathy. I guess it's a topic on people's mind given competitor, Alnylam's presentation of data, Helios-B data at the ESC meeting last Friday. Could you share your overall thoughts on that data?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Got it, got it. Could you maybe talk about the -- based on Helios-B data, those initial insights on monotherapy activity versus combination versus tafamidis alone. And then what's the implication for commercialization? And how do you see the silencer class entering the market? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 12:45PM, IONS.OQ - Ionis Pharmaceuticals Inc at Wells Fargo Healthcare Conference

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Great. That's super helpful. And then maybe to follow up on the last point about the trend in combo dataset. That's admittedly the probably the weakest data from the whole dataset in combination patients. You have a larger study. But I was wondering what is the benefit are you shooting for statistical significance in that subgroup, or it doesn't have to be like with a stats data to support combo use?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Great, great. Yeah, that's super helpful again. So any thoughts on whether you might read out cardio transform earlier?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Great, great. And lastly, when we anticipate that data readout in the data, you have articulated all the benefits of having a larger dataset and also the differentiated point about imaging studies and what role that might play. But on the efficacy, is your assumption or should our default assumption be that all silencer should be the same, or could there be differentiation?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Great, great. Thanks. I was going to ask about the at-home administration, that convenience factor, how had that borne out in WAINUA launch. So I think you just mentioned it resonated very well.

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Got it. Great. So let's move on to olezarsen for FCS and also for SHTG. So Arrowhead just presented also at the ESC meeting their Phase 3 data for FCS. Could you share your thoughts on the data? How should we compare their triglyceride reduction and acute pancreatitis benefit with olezarsen?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : That's great. So I guess you also have the SHTG data next year and then launch after that. But of course, I think the competitor product also has the data obviously, are positive and will be also launched into FCS. How do we think about commercial launches of all those indications?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Maybe switching gears again to donidalorsen for HAE. Mainly the question is how close are you to following that that NDA to FDA? REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. SEPTEMBER 04, 2024 / 12:45PM, IONS.OQ - Ionis Pharmaceuticals Inc at Wells Fargo Healthcare Conference

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Got it, got it. If we may touch on pelacarsen. This is your partner program with Novartis, targeting Lp(a). I think there's more and more interest as we're getting close to that data. So mainly people wanted to know what does success look like for this first-in-class study and what's the threshold for clinically meaningful change on the primary endpoint of CV event rate?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Got it. Great. Thanks for that insight. And then perhaps on Angelman syndrome. I think this is a very interesting topic. You reported positive data in July. But then before that, partner Biogen decided not to opt in. There has been a lot of questions around that. Could you talk about your -- what's the -- what's surrounding that Biogen decision and your excitement level for this program?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : That's great. That's great. So we didn't have time to really touch on any of the earlier-stage programs, I think you have MsPA backbone for longer duration in your neurology program, you have siRNA maybe for heart failure indication. Just in general, as you mentioned, the CNS, muscle, ortho, those are kind of new areas ASOs. Maybe just give us an overview -- a preview of what's to come in new technology front?

Question: Yanan Zhu - Wells Fargo Securities - Analyst : Great, great. With that, I think we're out of time. Thank you, Brett, for your time and insights.