Ionis Pharmaceuticals Inc at Piper Sandler Healthcare Conference Summary

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

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : <_ALACRA_META_ABSTRACT>So Brett, maybe just kind of an intro question. We all know Ionis is a long history as a leading antisense in ASO company. You're in the process of becoming a fully integrated biopharma company and are kind of at a very important point right now in the company's history. So just walk us through that, your late-stage programs, your commercial programs and just how you are positioned for value creation.

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : Excellent. So maybe starting out on eplontersen. I think we're about a year into the renewal launch in ATTR polyneuropathy. Can you just talk to learnings or takeaways you have from that experience over the last year, specifically on the self-administration profile? Anything you can extrapolate to the cardiomyopathy indication?

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : So along that line of thinking, there's been a lot of interest debate the ATTR cardiomyopathy market, how it's going to evolve as new stabilizer in silence or therapies come to market. So the question is, how do you expect the treatment paradigm to look when eplontersen is launching in cardiomyopathy and how does this affect your positioning of the drug? What -- help us understand the thinking around launch environment?

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : Excellent. So now shifting to olezarsen. This is obviously a -- it's going to be a (inaudible) first question since we're awaiting the olezarsen PDUFA in FCS and the sHTG data next year. But can you just talk or help us understand the FCS and then the sHTG launch, progression and strategy and what that might look like?

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : Excellent. Another cardiovascular asset with data next year, pelacarsen. Can you just talk to you your confidence with partner Novartis on the -- on your Phase 2 readouts next year and really how you envision Lp(a) competitive landscape evolving. It would be great to get some color there.

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : Great. I guess we'll -- I know we want to keep an eye on time and get to donidalorsen in HAE, where you mentioned you have a PDUFA date coming up next August 21. So talking to investors, there's a lot of focus or concern maybe about the crowdedness of the treatment landscape here in HAE for approved and developmental therapies. Can you just talk to that, where do you see donidalorsen fitting in and just learning good analogs from the HAE space or other spaces that you think might apply here?

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : And given that this is a switching market, I guess, how mission critical is it for your switching data to make it onto the label?

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : Great. Well, I want to leave a little time at least for the [poly owned neuro franchise], specifically 582 and Angelman. So yeah, I guess our understanding that partner Biogen kind of ran out of time to exercise their opt-in right for this program and so you were able to regain full rates. So as we sit here today now that you have regulatory clarity in hand. This program is in a good spot, I think, for the company. So can you just walk us through the regulatory interactions you've had, what gives you confidence that you've identified the right Phase 3 trial design and endpoints. And just like overall confidence that you can replicate the HALOS data in Phase 3. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. DECEMBER 04, 2024 / 2:00PM, IONS.OQ - Ionis Pharmaceuticals Inc at Piper Sandler Healthcare Conference

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : Great. And then I think we all know there's another asset in late-stage development for [ultragenics]. So I guess, how do you envision 582 being differentiated based on your Phase 3 and your Phase 2 experience. And is this -- or isn't it a so called winner take all market?

Question: Allison Bratzel - Piper Sandler & Co. - Analyst : Excellent. I think with that, we're at time. But thank you for stopping by the Piper conference. Great discussion.