Ionis Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference Summary

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

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Yes. Great. Thanks for that introduction. And maybe we can start with WAINUA. As you mentioned, recently launched with your partner AstraZeneca. Maybe you can just talk about your responsibilities in the launch and maybe expand a little bit on some of those early favorable launch trends? 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 05, 2024 / 4:20PM, IONS.OQ - Ionis Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Yes. Great. Maybe just sticking with WAINUA. You mentioned the ongoing CARDIO-TTRansform study in ATTR cardiomyopathy. Maybe you can just give us some update there on how that progress is going, and then any updated thoughts on potentially stopping that study early?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Maybe can you talk a little bit about the process you're going through in order to make that decision, and how long could that take? And I guess, what -- if you disclose anything, is no news means you continue to completion? Or do you make some announcement that you won't start?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Makes sense. Maybe just based on some of the data you've seen so far internally for your program or even externally with the HELIOS-B data, just where do you think the silencers fit in relative to stabilizers? Are they front line, are they combo, both? Any thoughts there?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Yes. Makes sense. And how do you view that market sort of changing when tafamidis potentially goes generic?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : What do you think the bar is for a combination use before it goes generic? Is it -- do you need to show some statistical significance or are strong trends enough? Or just any thoughts there?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Makes sense. Maybe we can shift gears to olezarsen, FCS. You're currently under FDA review. You've got a PDUFA date coming up later this year. Maybe just remind us what was so exciting about the data you showed and kind of where you are in preparing for the launch.

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : How important is educating patients and identifying the patients, since you'll kind of be the first to market here?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : For the sHTG studies, can you talk about the potential of showing a benefit on pancreatitis and how important that is or isn't?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Makes sense. Maybe just last question on olezarsen before we move on, is just competitive positioning. You could potentially have a competitor in the market maybe sometime next year or the year after. Just how's your -- what's the differentiation?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Makes sense. Maybe we can shift gears to donidalorsen, HAE. You presented some promising data earlier this year. Maybe just highlight some of the key takeaways there. 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 05, 2024 / 4:20PM, IONS.OQ - Ionis Pharmaceuticals Inc at Morgan Stanley Global Healthcare Conference

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Should we be anticipating standard review or a priority review there?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Makes sense. A lot to talk about here, maybe we can switch to another pipeline asset. You recently shared some Angelman data earlier this year and decided to move that forward. Maybe just touch on that.

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Maybe I'll just ask a broad question. Anything else in the pipeline that you think you want to highlight?

Question: Michael Ulz - Morgan Stanley & Co LLC - Analyst : Yes. So good, it looks like we're just about out of time, so why don't we end it there? Thanks so much, Brett. Appreciate your time.