Ionis Pharmaceuticals Inc at BMO Biopharma Day: Spotlight on Rare Disease and Ophthalmology (Virtual) Transcript

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

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. Great. So maybe you can start by just giving a brief overview of the company's antisense technology and just the flexibility that you have to modulate all different types of RNA. So how broad that technology could take you and has it taken you with your pipeline?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. That's great. And maybe also explain the LICA technology and how that increases the potency of your drug so dramatically. And looking at some life cycle opportunities with that. Maybe just comment on that. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JUNE 22, 2021 / 4:00PM, IONS.OQ - Ionis Pharmaceuticals Inc at BMO Biopharma Day: Spotlight on Rare Disease and Ophthalmology (Virtual)

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. So are there other therapeutic areas outside of neurology, cardiometabolic-type conditions that would lend itself to the LICA technology and -- as well as your RNA technology, the anti-sense? Or are those the main areas that you're going to be focusing on, you think, for the foreseeable future?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. So on that, maybe just talk about how SPINRAZA compares clinically to the competing products, Evrysdi and Zolgensma? And Biogen recently had some DEVOTE data that evaluated the higher doses of SPINRAZA. So how that compares to the current dosing regimen? And is that going to help differentiate the product further in the market?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. Great. And I just want to remind everyone, if you have a question, please put it in the app, and I'll be happy to ask Eric. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. JUNE 22, 2021 / 4:00PM, IONS.OQ - Ionis Pharmaceuticals Inc at BMO Biopharma Day: Spotlight on Rare Disease and Ophthalmology (Virtual) So I guess maybe going into the cardiovascular disease, maybe highlight your efforts to expand your reach in ATTR and FCS with the next-gen LICAs. So the eplontersen APOCIII-LRx. So how they're differentiated? Maybe just talk about those respective programs and how that's going to follow on from the products that you currently have in the market?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. Excellent. Let's shift over to ALS, and maybe we're expecting the tofersen VALOR pivotal Phase III data in SOD1-ALS. That's going to be in the fall. Just remind us what the Phase I/II data showed? What your expectations are for the Phase III in order to support approval, I guess. Is it going to be just for that subset population? And then also, you have the ATLAS Phase III study, right, in presymptomatic SOD1-ALS patients. So how important is that to the overall program and timing of approval and launch?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. And with the recent tominersen setback in Huntington's, I just want to clarify, is there any potential read through to SOD1-ALS, or more broadly, across other neuro disorders?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. Can you maybe just explain the arrangement with Biogen, how they can exercise options with all the different assets in the neurology pipeline? Do you have any say as to what ultimately is going to be wholly owned or what you think that they're going to pass on? And then how do you manage your wholly owned asset in ALS? So ION363 for FUS-ALS, with tofersen that's partnered with Biogen. So it just feels like it's overlapping a little bit, so how do you manage those different programs?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. Is there anything else in your wholly owned pipeline that you would want to highlight in the last minute or so? You mentioned Alexander. There's Prion, Lafora disease. Pick one that at this stage you're particularly excited about. And what the next data point will be coming out of that?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. And maybe I think we have another 30, 60 seconds. So just your expectations for pelacarsen that's partnered with Novartis. How optimistic are you? There has to be an outcome study, typically challenging when you need outcome studies. Obviously, you have a big pharma company behind it. But where would you rank that in terms of your optimism, I guess?

Question: Gary Jay Nachman - BMO Capital Markets Equity Research - Analyst : Okay. We're going to end on that note. So Eric, thank you for a great discussion. And that's how we're closing out today's conference. Spotlight on rare disease and ophthalmology, had a lot of really good content, a lot of good panelists today. I want to thank my colleague, Matt Luchini, for also co-moderating and running the show. And that's it. I hope everyone who listened enjoyed, and have a great day, everyone.