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 - JPMorgan - Analyst
: Great, thanks for the presentation. So you talked about the business growing in 2024. And then I think there was a stacked bar chart that had a bar
for '24. Should we be using a ruler on the bar chart to kind of estimate the growth you're anticipating or is there any more kind of bookends you
can put around the expected growth this year?
Question: Jess Fye - JPMorgan - Analyst
: Great, and maybe you can talk a little bit about how the competitive dynamics are playing out in the US with Bylvay and Alagille. And I guess,
maybe talk about what the right way to think about the proportion of market share you expect Livmarli to keep is.
Question: Jess Fye - JPMorgan - Analyst
: Okay. What about in PFIC, can you just talk a little bit about how you're thinking about the overall market opportunity for Livmarli in that setting?
I don't know if that's relative to Alagille or kind of from a market share perspective, but what can you tell us there?
Question: Jess Fye - JPMorgan - Analyst
: Great, and last one on Livmarli, can you just share what the kind of learnings were from that Phase 2b EMBARK study that didn't hit the primary
endpoint and the extent to which you think those results do or do not have read-through to IBAT inhibitors in other indications, particularly in the
adult indications with volixibat?
Question: Jess Fye - JPMorgan - Analyst
: Got it. So maybe switching over to volixibat, can you recap what some of those factors were that impacted the enrollment in the PSC and PBC trials
and can you talk about what gives you confidence heading into those updates in the first half?
Question: Jess Fye - JPMorgan - Analyst
: Great. In the slides, I think you flashed up some PSC data for maralixibat. How reliable do you think that data is, or maybe put differently, how much
is the placebo response typically in PSC if we kind of think about a mentally adjusting for the open-label aspect of that data?
Question: Jess Fye - JPMorgan - Analyst
: Great, what about the competitive landscape in PSC and PBC? How are you thinking about that and, it's related to that, where could volixibat fit
into the current treatment paradigm for each of those conditions?
Question: Jess Fye - JPMorgan - Analyst
: And can you also talk about maybe for PBC where we get to see the interim data, what you think of as clinically meaningful and/or research related
to that commercially relevant impact on pruritus?
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JANUARY 10, 2024 / 6:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc at JPMorgan Healthcare Conference
Question: Jess Fye - JPMorgan - Analyst
:
Question: Jess Fye - JPMorgan - Analyst
: Got it. Maybe switching to Chenodal for CTX, can you help us -- maybe give us a framework for how to think about the potential magnitude of the
sales inflection we could see there?
Question: Jess Fye - JPMorgan - Analyst
: Great, and maybe just one last question, how are you thinking about business development now? Is there kind of an integration period where we
shouldn't expect activity from Mirum in the near term on the back of this recent transaction or is there still high uptake? How do we think about
that?
Question: Jess Fye - JPMorgan - Analyst
: Great. I think we're about out of time so we'll stop there, and thank you.
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