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: Michael Ulz - Morgan Stanley - Analyst
: Well, great. Thanks for that introduction, and a lot going on. Congratulations on getting your study fully enrolled. And I thought though maybe
we could start just with LIVMARLI. Obviously, that's your revenue driver. You touched on this in your prepared remarks that you're seeing some
really nice growth there. And a big part of that is PFIC. So maybe just dig into that a little bit more and what's happening there?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 08, 2025 / 12:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc at Morgan Stanley Global Healthcare
Conference
Question: Michael Ulz - Morgan Stanley - Analyst
: Just for PFIC in terms of identifying the patients? Are you doing anything unique than what you've done sort of in the past with Alagille, for example?
Or is it just maybe you underestimated what the initial patient numbers might look?
Question: Michael Ulz - Morgan Stanley - Analyst
: Makes sense. You also mentioned sort of the strength in your international markets. So maybe talk about some of those markets you recently
launched? And then sort of looking ahead, what are the additional markets you'll be looking out for?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 08, 2025 / 12:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc at Morgan Stanley Global Healthcare
Conference
Question: Michael Ulz - Morgan Stanley - Analyst
: Great. maybe we can just flip back to Alagille syndrome for a minute in the US and kind of that's been sort of your initial launch there. So it's been
several years. You keep growing that. Sort of where are you in that sort of process and how much room is left, I guess?
Question: Michael Ulz - Morgan Stanley - Analyst
: Makes sense. And in your sort of introductory comments, you sort of mentioned you recently increased your sort of peak sales expectations for
LIVMARLI to $1 billion plus. Maybe you can just talk about, maybe a different components of that and I don't know if you're willing to go there, but
the breakdown of those components?
Question: Michael Ulz - Morgan Stanley - Analyst
: Maybe in terms of the EXPAND study, if you can just sort of remind us, the rationale there and how you started to head in that direction for those
rare conditions?
Question: Michael Ulz - Morgan Stanley - Analyst
: Okay. And how, I guess, enrollment is tracking sort of as you expected? Or is it faster maybe or --
Question: Michael Ulz - Morgan Stanley - Analyst
: Got you. And maybe just last question on LIVMARLI before we move on, maybe just touch on current IP? Or is there more IP coming that we should
be looking out for? Or what's the status there?
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 08, 2025 / 12:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc at Morgan Stanley Global Healthcare
Conference
Question: Michael Ulz - Morgan Stanley - Analyst
: Great. Maybe we can move on to just the bile acid portfolio. You had two programs there. You added them a few years ago, and you're seeing
some nice growth. So maybe just give us a little background and kind of what's driving the growth there?
Question: Michael Ulz - Morgan Stanley - Analyst
: Yes, definitely making nice progress there. And maybe now we just shift to Volixibat. And maybe just start with PSC since that's sort of more
advanced in development, maybe talk about the market opportunity there and the unmet need?
Question: Michael Ulz - Morgan Stanley - Analyst
: You mentioned coming up with an endpoint for this disease has been a bit challenging. You sort of focused more on the symptoms and you've
seen some nice impact there. But I guess from a regulatory perspective, are there any issues or hurdles you need to sort of clear to sort of make
that an approvable endpoint? Or is that -- are we there already?
Question: Michael Ulz - Morgan Stanley - Analyst
: Makes sense. And I think it was last year, you gave some sort of interim analysis. So maybe walk us through that and kind of how to think about
that.
Question: Michael Ulz - Morgan Stanley - Analyst
: Have you mentioned what the threshold was in VISTAS for the interim on pruritus or --
REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us
consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
SEPTEMBER 08, 2025 / 12:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc at Morgan Stanley Global Healthcare
Conference
Question: Michael Ulz - Morgan Stanley - Analyst
: And then maybe just on pruritus in general, like what's considered a meaningful difference? And then remind us what you showed in the VANTAGE?
Question: Michael Ulz - Morgan Stanley - Analyst
: Great. And maybe just sticking with VISTAS and again, you complete enrollment that you announced today, and now you're heading towards data
in the second quarter of next year. So maybe just talk a little bit about what to expect from that readout and kind of what you're looking for and
what's sort of a positive readout in your view?
Question: Michael Ulz - Morgan Stanley - Analyst
: Okay. And then maybe just shifting to PBC and maybe just a similar questioning market opportunity there, unmet need?
Question: Michael Ulz - Morgan Stanley - Analyst
: And just for the VANTAGE study, can you just remind us the current status there in terms of where you are in enrollment and kind of timelines
around that?
Question: Michael Ulz - Morgan Stanley - Analyst
: And maybe you can touch on the competitor in the space or the recent data we've seen and kind of how you're positioned there?
Question: Michael Ulz - Morgan Stanley - Analyst
: Makes sense. Maybe we can just shift quickly to your FXS program. Maybe just talk about that a little bit, how it's differentiated and kind of next
steps there?
Question: Michael Ulz - Morgan Stanley - Analyst
: And maybe just quickly remind us where you are in the Phase II. Is it started yet? Or when will you start it?
Question: Michael Ulz - Morgan Stanley - Analyst
: Maybe in the last few minutes, we can talk about some of these macro issues. We're asking a couple of questions to all our companies at the
conference. So maybe if we just start at the top with the first question, China's rise in biotech innovation, how are you thinking about your competitive
position here? And will this influence your R&D or BD strategy?
Question: Michael Ulz - Morgan Stanley - Analyst
: Maybe next question, how are you currently leveraging artificial intelligence or thinking about AI's future disruption potential in your business?
Question: Michael Ulz - Morgan Stanley - Analyst
: Got you. And then maybe last question, just what's been most impactful for Mirum on the regulatory side in terms of would it be FDA? Would it
be MFN, if you have any impacts there or tariffs?
Question: Michael Ulz - Morgan Stanley - Analyst
: Great. Looks like we're just about out of time. So why don't we end it there. Chris, thanks so much. Appreciate it.
|