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 Fye - J.P. Morgan - Analyst
: Maybe first I'll take a question from the audience here. You talked at the beginning of the presentation about how the strategy of the company
has changed under your leadership. So how are you thinking today about what assets you would still partner out versus keep in-house? And kind
of what are the pros and cons of partnering assets outward?
Question: Jessica Fye - J.P. Morgan - Analyst
: And then you talked about the goal of becoming cash flow positive over the coming years. And based on the slide, it looks like it's going to be very
top line driven. How do you see the company's cost structure evolving now that you do have multiple wholly owned products that you're going
to be launching?
Question: Jessica Fye - J.P. Morgan - Analyst
: And you talked about a desire for new pipeline products to ideally have synergy with the portfolio ahead of them. How much synergy do you see
across the kind of the first wave of commercial launches?
Question: Jessica Fye - J.P. Morgan - Analyst
: There's a lot of kind of going on with the commercial organization this year. But in terms of kind of early or mid-stage pipeline readouts, what
would you focus investors on this year?
Question: Jessica Fye - J.P. Morgan - Analyst
: So you mentioned kind of the first proof of concept for Ionis siRNA. How are you prioritizing target selection and choosing between leveraging an
ASO versus siRNA?
Question: Jessica Fye - J.P. Morgan - Analyst
: So maybe turning to some specific products. Last year, you launched WAINUA. How should we think about growth for that asset in 2025?
Question: Jessica Fye - J.P. Morgan - Analyst
: When we think about cardiomyopathy, following the HELIOS-B results, can you talk about any changes that were made to the Phase 3 cardio
transform trial, for example, on variable follow-up? How should we think about timing for that study? And is it powered for the TAP and non-TAP
subgroups?
Question: Jessica Fye - J.P. Morgan - Analyst
: Maybe turning to another one of your near-term launches for donidalorsen. Where are you envisioning that product kind of initially picking up
patients? Will it be those who are new to HAE prophylaxis? Will it be switchers? And if they're switching, would you expect to get them more for
the existing injectables or oral treatment?
Question: Jessica Fye - J.P. Morgan - Analyst
: Great. Well, with that, I think we're out of time. So thank you.
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