Ionis Pharmaceuticals Inc Q3 2024 Earnings Call Summary

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

Question: Gary Nachman - Raymond James - Analyst : So first on olezarsen for FCS, how soon will you be able to launch post approval? And maybe talk about the expected pricing there. Are you in labelling discussions? And anything to call out on those expectations? And just generally, how we should be thinking about that ramp? And then I have one follow-up.

Question: Gary Nachman - Raymond James - Analyst : Okay. Great. And then just on the Phase III Angelman study. So will there be any interim looks over the course of the year since it's a one-year endpoint any monitoring of these patients required on the safety side? And maybe just comment if there was any back and forth in terms of the right primary endpoint for these patients or if there was easy agreement there with the FDA?

Question: Jessica Fye - JPMorgan Chase & Co - Analyst : I have a question about the timing for cardio transform. So I think enrollment completed on July 31, 2023, and 140 weeks after that is April 6, 2026. And in the past, you talked about that readout coming in the first half of '26, then mid-'26. And now it sounds like data in the back half of '26. So did anything change with the approach to that study? For example, I think I recall the follow-up originally being planned is up to 140 weeks for those who entered the trial towards the end having variable but a little bit shorter follow-up than that. So is the plan now to follow all patients out to 140 weeks? Or are you really just budgeting like a full three months or more for data cleanup and analysis?

Question: Mike Ulz - Morgan Stanley & Co LLC. - Analyst : Okay. And then just as a quick follow-up. I guess, how quickly do you expect to be able to enrol patients from the open-label extension as well as the early access program onto paying commercial drug?

Question: Yanan Zhu - Wells Fargo Securities, LLC - Analyst : Great. Congrats on the progress. Maybe first, a couple of very quick follow-ups to earlier questions and answers. On FCS, I was wondering, how do you anticipate the payer dynamic given that this is likely a highly priced drug. Would that reimbursement dynamic to the launch in any way? And for the ATTR cardiomyopathy program, I was just wondering, you just talked about the 140-day full completion. Is there any possibility or desire to expand it further, given that Alnylam study had some of the secondary end points out to 42 months. And I have a follow-up after that.

Question: Yanan Zhu - Wells Fargo Securities, LLC - Analyst : Got it. Very helpful. Then I just have a question about the Phase III design for the Angelman syndrome study. Could you compare and contrast your design with Ultragenyx design Obviously, the primary endpoint is different. And I think that's congrats on getting expressive communication as the primary end point. But in terms of whether it's placebo controlled versus sham control patient age range -- can you talk about the design differences and what the implications might be for enrollment speed and data. 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 06, 2024 / 4:30PM, IONS.OQ - Q3 2024 Ionis Pharmaceuticals Inc Earnings Call

Question: Luca Issi - RBC Capital Markets - Analyst : Maybe if I can circle back on a prior question, Eugene. You're obviously using express communication as a primary endpoint versus obviously your competitors using cognition. Can you just talk about you think the former is the better way to go versus the latter, any call there, I much appreciate it. And then on APOC3, I think ElaLily discontinued their siRNA going after APOC3 wondering what was your reaction to the news and how we should think about implications for your program? 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 06, 2024 / 4:30PM, IONS.OQ - Q3 2024 Ionis Pharmaceuticals Inc Earnings Call

Question: David Lewis - Citi. - Analyst : In terms of your launches for donidalorsen and olezarsen. Even that these are really the first more significant launches that you're doing yourself. Could you describe how the sales operation is coming together? What the sales team will look like for both indications and how you intend to receive, especially with respect to HAE given that it's a very competitive market.

Question: Jason Gerberry - BofA Securities - Analyst : Just a couple on the pelacarsen readout next year. First, with the primary, you're looking at this 90 mg per (technical difficulty) subgroup. If the event reduction looks more compelling in this subgroup, clinically meaningful, how does that alter the addressable market, which I think you guys have framed about [$8] million to [$10] million for secondary prevention, high-risk Lp(a) greater than 70. But how does that change if it's 90 or greater? And then ultimately, the other question is just what underpins your confidence that you'll have the events to read out around middle of next year versus the potential need to run that trial out longer? I think your assumed event rates a couple of percentage points higher than what we've seen in like the typical LDL lowering trials in the past. So just kind of wondering, I assume that there's been some assessment of rates on a binded basis and that sort of underpins and firms up the outlook for timing next year. But I just wanted to confirm on that.

Question: Jay Olson - Oppenheimer & Co. Inc. - Analyst : Congrats on all the progress. Does the Phase III study design for (technical difficulty) support regulatory filings ex-US? And if so, how are you thinking about ex-US partnering opportunities for 582? And then -- and then I had a follow-up if I could.

Question: Jay Olson - Oppenheimer & Co. Inc. - Analyst : Great. Super helpful. And then we had a question on 269, your APP ASO. It's great to see research being done in Down syndrome based on the huge unmet need in that population. Can you talk about the rationale for choosing that indication to start with and maybe elaborate on the development strategy and any plans for other indications and differentiating features for your program versus other RNA therapeutics.

Question: Mani Foroohar - Leerink Partners - Analyst : I know a lot of pipeline questions have been answered. I want to circle back on the growth in TTR polyneuropathy, obviously, accelerating from where your revenue base was prior product. Perhaps a question that is for both you guys and AstraZeneca, that you have some insight. To what extent are we seeing patients switching from the existing oligo therapy, which is two, three months, then may lose some efficacy at the end of that time horizon. -- to your at-home therapy with eplontersen? And what -- so what proportion of your growth is coming from patient switching versus previously untreated patients who are getting up once.

Question: Mani Foroohar - Leerink Partners - Analyst : Great. And a quick follow-up. Your Oligo competitor has talked about in part for the cardiomyopathy market but also in polytropic a belief that those patients who are receiving an in-office therapy the Medicare Part B may have less out-of-pocket or more favorable reimbursement from a facility perspective than an at-home Part D therapy. Can you clarify whether or not you're seeing any headwinds whatsoever from patient auto pocket costs, reimbursement headwinds, et cetera, that would support or refute that competitive argument counter detail.

Question: Andy Chan - Wolfe Research, LLC - Analyst : So regarding the Doni launch in HAE, so a fraction of these patients, my understanding is either on androgen therapy, they're not on (technical difficulty) , there's not (technical difficulty). Do you believe that there's an opportunity there to convert these patients to Doni? Or do you just believe that they're very part of reach? And on the side note, do you think payers are going to begin to play favourites in this market because it's kind of becoming crowded. 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 06, 2024 / 4:30PM, IONS.OQ - Q3 2024 Ionis Pharmaceuticals Inc Earnings Call