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: Gary Nachman - Raymond James - Analyst
: Excellent. Very good overview. Thank you. Understanding you're the CFO and not the Head of R&D, but maybe just for people not
as familiar just what sets you apart in terms of your RNAi technology. There are a few companies out there that have similar types
of technology and capabilities?
What do you see as your main strengths? And then maybe just talk briefly about you've been targeting the liver mainly over the
years and now moving more into CNS and muscle and the importance of that thinking farther down the road.
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MARCH 04, 2025 / 1:40PM, IONS.OQ - Ionis Pharmaceuticals Inc at Raymond James Institutional Investors
Conference
Question: Gary Nachman - Raymond James - Analyst
: That's great. A lot of exciting opportunities there in neurology. So then let's shift gears. So describe what the process has been like
transitioning to a commercial company, how you guys prep for it over the last year, leading up to the Tryngolza launch? And how
you feel you've been executing thus far with it and some of the early read on key metrics.
Now it's a small indication, but getting you started.
Question: Gary Nachman - Raymond James - Analyst
: Excellent. Maybe just touch on the partnership with AstraZeneca for WAINUA and how that's helped in terms of your learnings and
what your level of involvement has been with that launch, and that's being up quite nicely based on the -- that they're reporting in
the competitive space. Initially, when you announced that partnership, people were saying, well, up a lot of the economics, an
important asset, but now you have the partnership, helping you out commercially. So maybe talk to that a little bit.
Question: Gary Nachman - Raymond James - Analyst
: Great. We'll get more into the cardiomyopathy a little bit after. But next upcoming catalysts, just sticking with the commercial train
of thought here. How are you practicing -- for doni in HAE. That's a the -- for that to be a good commercial product?
And how much bigger of is it compared to FCS?
Question: Gary Nachman - Raymond James - Analyst
: Phase III.
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MARCH 04, 2025 / 1:40PM, IONS.OQ - Ionis Pharmaceuticals Inc at Raymond James Institutional Investors
Conference
Question: Gary Nachman - Raymond James - Analyst
: A little more reps you think you would need just...
Question: Gary Nachman - Raymond James - Analyst
: And you think that's greater than $1 billion opportunity, market opportunity?
Question: Gary Nachman - Raymond James - Analyst
: Okay. We're a little short on time. There's definitely a few other is great, but a few other things I want to get to. So for next year, you
have 2 very important day catalysts for partnered programs. Pelacarsen for LP(a) lowering with Novartis in the first half of the year,
if everything goes well.
And then the ATTR cardiomyopathy with AstraZeneca in the second half of the year. So without getting too much into the details,
just talk about the importance of those data readouts why you're confident that if they read out positively, these are going to be
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MARCH 04, 2025 / 1:40PM, IONS.OQ - Ionis Pharmaceuticals Inc at Raymond James Institutional Investors
Conference
very big opportunities for you? And then just touch on the economics with the partners so people appreciate the value that you're
going to get from those?
Question: Gary Nachman - Raymond James - Analyst
: And just to be clear, that's just with WAINUA alone. That's not the overall market of that?
Question: Gary Nachman - Raymond James - Analyst
: Okay. I'm going to ask you to keep your CFO hat on. And how well capitalized is the company currently? What are you spending
requirements are for the next couple of years? And how do you see the path to profitability especially now that you're transitioning
to a commercial company and they have all these other programs ongoing?
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MARCH 04, 2025 / 1:40PM, IONS.OQ - Ionis Pharmaceuticals Inc at Raymond James Institutional Investors
Conference
Question: Gary Nachman - Raymond James - Analyst
: That's without raising any more money.
Question: Gary Nachman - Raymond James - Analyst
: With all the milestones
Question: Gary Nachman - Raymond James - Analyst
: Okay. And also just very quickly just touch on the royalties that you get from SPINRAZA with Biogen and the importance of that and
your confidence that those are to continue at roughly the same level, if not higher, for the foreseeable future. I mean that's an
important source of funding for the company.
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MARCH 04, 2025 / 1:40PM, IONS.OQ - Ionis Pharmaceuticals Inc at Raymond James Institutional Investors
Conference
Question: Gary Nachman - Raymond James - Analyst
: Okay. Excellent. We're up on time. Thank you so much, Beth. That was great, and good luck with all the upcoming catalysts.
Hopefully, the stock will follow. And everyone, have a good day.
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