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: Gavin Clark-Gartner - Evercore ISI - Analyst
: Congrats on the great progress. So I just had one more strategic question. I know that you aren't able to call for redemption of convertible notes
until next year. But just bigger picture, as your stock has traded well above the conversion price, the back of really strong earnings performance,
pipeline advancement, does this all change how you think about BD and capital allocation in general?
Question: Jessica Fye - JPMorgan - Analyst
: I was hoping you could talk about the underlying assumptions like market penetration or share, potential competition, et cetera, that are behind
the $1 billion revenue potential for MRM-3379 in Fragile X. And I guess while we're talking about that asset, are there any other indications with
the mechanistic rationale that could make sense to pursue?
Question: David Lebowitz - Citi - Analyst
: In terms of the recent approval for CTX, congratulations on that. Could you, I guess, elaborate on how you expect this could affect the sales trajectory
on this point going forward, given there's already use in that particular population?
Question: Rohit Bhasin - Morgan Stanley - Analyst
: This is Rohit on for Mike. Can you just talk about the patient mix between Alagille syndrome and PFIC for LIVMARLI in fourth quarter of 2024 and
how you expect this to play out in 2025? And then additionally, how many of the patients on the PFIC expanded access program are now on paid
drug?
Question: Ryan Deschner - Raymond James - Analyst
: Congrats on the quarter. My question is, how much commercial team expansion would you anticipate doing for expanded pruritus in ultra-rare
cholestatic patients, pending the result of the Phase 3 EXPAND study? And then I have a quick follow-up.
Question: Ryan Deschner - Raymond James - Analyst
: Yes. Do you have a feel at this point for how many -- specifically, how much of an impact the growing average patient weights are in impacting
sales growth in Alagille? How big actually is this growth component now compared to some of the other tailwinds here? And how do you expect
it to grow moving forward?
Question: Luke Herrmann - Robert W. Baird & Co., Inc. - Analyst
: This is Luke on for Brian. We were hoping you could just talk a little bit about how the VANTAGE study takes into account prior OCA use and
enrollment. And do you think patients recently discontinuing OCA could see an impact on pruritus measurements as compared to a patient not
discontinuing?
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FEBRUARY 26, 2025 / 9:30PM, MIRM.OQ - Q4 2024 Mirum Pharmaceuticals Inc Earnings Call
Question: Jonathan Wolleben - Citizens - Analyst
: Just wondering, piggy backing on a prior EXPAND question. What do you envision the label looks like if you have success in that Phase 3 trial? And
then how do you wrap your heads around that 1,000 patient US and EU prevalence, given it's a collection of a bunch of small indications?
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