The following is excerpted from the question-and-answer section of the transcript.
(Questions from industry analysts are provided in full, but answers are omitted - download the transcript to see the full question-and-answer session)
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Great. Brett, we'll just give it a second for your colleagues to pop up here. And while that's happening, I'd remind folks that you can send a question
by hitting that Ask a Question button online. That will populate a portal, and then I can ask those questions to management.
But I wanted to start with one that has been coming up a lot recently, which is, in your presentation at the Investor Day in December, you highlighted
your expectation of having 12 or more medicines on the market in 2026. Which of those programs are you most excited about? And can you talk
to us about the market opportunities?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. Great. There's a number of questions coming in about ATTR in particular. So I'm just going to pick the one that sort of seems to sum them
up the most, which is: how does the Ionis approach compare to the Alnylam approach? And what is the potential advantage? Is it dosing, depth
of response, breadth of response? How do they compete coming later to market? Presumably that's referring to the LICA program.
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. With the update from the Huntington's natural history study as well as the open-label data for tominersen coming in the first half of this
year, how do you expect that to inform expectations around the pivotal study in Huntington's?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. Got it. Maybe switching to cardiology. Have you had any feedback from the FDA on your plans to move IONIS-AGT-LRx into the Phase IIb
study in uncontrolled hypertension and Phase II in heart failure with reduced ejection fraction? And when could you initiate those trials?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. And for the next oral formulation of ION449, what's the latest timeline there?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. Got it. You started out the presentation talking about the strategy to keep more of your products wholly owned and commercialize them
independently. And related to that, you've also talked about kind of within that, approaching indications that have a rapid path to market and then
expanding out from there once you kind of establish a commercial foothold in different disease areas. So just thinking about that a little more
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JANUARY 12, 2021 / 8:40PM, IONS.OQ - Ionis Pharmaceuticals Inc at JPMorgan Healthcare Conference (Virtual)
specifically, what are the factors that you're hoping to characterize in these markets as you enter them before launching commercial efforts in
larger indications?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. Great. We're just about out of time here. So I think we'll leave it there. But thanks, everyone, for tuning in, and thanks so much to the Ionis
team as well.
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