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: Jess Fye - J.P.Morgan - Analyst
: Great. Thanks for the presentation. Maybe starting with LIVMARLI, you gave us guidance for the year. What's driving the growth in 2025.
Question: Jess Fye - J.P.Morgan - Analyst
: When you talked about LIVMARLI being about 40% penetrated in Alagille, was that just LIVMARLI? And then I guess how penetrated is the overall
Alagille market?
Question: Jess Fye - J.P.Morgan - Analyst
: You talked about PFIC as an opportunity as well. What are the strategies you're using to increase LIVMARLI's market penetration there?
Question: Jess Fye - J.P.Morgan - Analyst
: What are the latest competitive dynamics between LIVMARLI and Bylvay in PFIC? And where does the market share stand there?
Question: Jess Fye - J.P.Morgan - Analyst
: Okay. I think you put on one of the slides that volixibat could be $1 billion opportunity. Your FXS asset could be another $1 billion asset. How does
LIVMARLI and the CHENODAL and CHOLBAM franchise stack up relative to those?
Question: Jess Fye - J.P.Morgan - Analyst
: You also highlighted the EXPAND trial to potentially further broaden the LIVMARLI label. How is enrollment going there? How should we think
about enrollment timelines? And how does that kind of opportunity factor into the product's peak potential?
Question: Jess Fye - J.P.Morgan - Analyst
: Maybe switch to volixibat. What can you tell us about enrollment of VISTAS and VANTAGE?
Question: Jess Fye - J.P.Morgan - Analyst
: And how are each of those studies powered? And how confident are you in the final result based on the interims?
Question: Jess Fye - J.P.Morgan - Analyst
: Okay. Another question we sometimes get about volixibat is just how to think about it relative to Glaxo's linerixibat. So how do you envision
competitive dynamics with that asset in PBC assuming that you're also on the market there?
Question: Jess Fye - J.P.Morgan - Analyst
: Got it. And when you talk about $1 billion peak potential for volixibat in PSC and PBC, how should we split that out between the indications?
Question: Jess Fye - J.P.Morgan - Analyst
: Got it. You mentioned the upcoming label extent expansion, supporting incremental growth for the CHENODAL franchise. Can you put any more
color around what we should expect for that business this year?
Question: Jess Fye - J.P.Morgan - Analyst
: Maybe we can talk about your newest asset, Mirum-3379. Can you just first, maybe talk about the rationale for bringing that product into the
company and what drew you to the asset?
Question: Jess Fye - J.P.Morgan - Analyst
: And maybe sticking with kind of business development, bringing in new assets. Can you talk more about your priorities there, therapeutic areas
of interest, maybe size of potential deals?
Question: Jess Fye - J.P.Morgan - Analyst
: When you say bookends, are you talking about sort of like degree of derisking or something else?
Question: Jess Fye - J.P.Morgan - Analyst
: And do you want to see what you get from Fragile X before you go on and do the next deal?
Question: Jess Fye - J.P.Morgan - Analyst
: Any last questions? Okay. We'll wrap it up. Thank you.
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