Ionis Pharmaceuticals Inc Q4 2024 Earnings Call Transcript

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

Question: Mike Ulz - Morgan Stanley & Co LLC - Analyst : Maybe just one on Olezaresen and SHTG, just given the ESSENCE study is supposed to read out midyear ahead of the core studies. And I realize it's more of a safety study and you're enrolling a different population. But what kind of read-through could we make from ESSENCE to your core studies? Thanks.

Question: Jessica Fye - JPMorgan Chase & Co - Analyst : I was hoping if you could talk a little bit about what you're seeing in the TTR polyneuropathy market? For example, what portion of new starts do you think you're capturing there?

Question: Akash Tewari - Jefferies - Analyst : Can you talk a little more about your design for SHTG, the Phase 3? I mean you've mentioned previously the enrollment rate in that trial is 10x that of FCS. What does that imply for the effect size you've powered for, particularly on acute pancreatitis and severe abdominal pain? And really, how does your event rate assumption maybe differ versus FCS, but also that ESSENCE study that you mentioned isn't necessarily representative of your other trials? And then any feedback from KOLs on what would be a clinically meaningful reduction would be very helpful as well. Thank you.

Question: Yanan Zhu - Wells Fargo Securities - Analyst : First, I wanted to have a quick follow-up to a prior question on the WAINUA polyneuropathy 84% quarter-over-quarter growth. How much of that growth is driven by switching because this year, they're both naive patients and switching patients, but wondering how much of a force is the switch from siRNA. And my main question: I think Biogen announced a couple of updates to some neurology collaboration programs. Could you provide a little more color and what's the plan for those programs? Thank you. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. FEBRUARY 19, 2025 / 4:30PM, IONS.OQ - Q4 2024 Ionis Pharmaceuticals Inc Earnings Call

Question: Jay Olson - Oppenheimer & Co. Inc - Analyst : Congrats on all the progress. TRYNGOLZA, I think you mentioned 3,000 patients in the US. Can you just talk about how many of those patients are currently diagnosed and available for treatment? And if it's not too early, how many of those patients are on treatment or any other important metrics you could share to help track the launch of TRYNGOLZA?

Question: Chi Meng Fong - BofA Securities - Analyst : This is Chi on for Jason. I guess I would like to follow up TRYNGOLZA launch in FCS. Thanks for all the commentary on early launch experience so far. I'm curious, based on early launch experience, what insurance company you think are requiring in terms of documentation for reimbursement consideration? With (technical difficulty) and one thing that we have heard is that genetic confirmation is the most straight-forward documentation to get insurance company on board, somewhat a bit of a gray area when it comes to clinical diagnosis, part of it is the lack of consensus that knows this criteria. Some of it is what insurance company, except as clinical diagnosis criteria. Can you talk about that? And when you talk about several hundred patients currently identify and become formally diagnosed, how many of those are getting genetically confirmed? and How many of those are getting clinically diagnosed. And if you can talk about that mix within that 3,000 patients US prevalence that you have estimated, that will be great as well.

Question: Gary Nachman - Raymond James - Analyst : Hi. Thanks, and my congrats as well on the progress. So as you prepare for new launches for Doni in HAE and Olezaresen in SHTG, just talk about how you're scaling the commercial organization following the FCS launch. What's in place versus what you need to add, specifically in terms of reps for those other programs? And then just for the Angelman program, just what's your expectations for enrollment timing given the competitive dynamics there? Thank you.

Question: Yaron Werber - TD Cowen - Analyst : I have a quick question also as you think about -- if you think about how do you power the Angelman Phase 3 study, I know you haven't announced before full [product] design yet, but on expressive communications. And then maybe just for Beth, as you think about revenue, as you mentioned, it's more second half weighted. Are there particular milestones we need to keep in mind that are driving that? Thank you.

Question: Yaron Werber - TD Cowen - Analyst : Are both doses powered against placebo? Thank you.