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: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Okay. And on that note, I mean, you've guided to cash flow breakeven in the fourth quarter. How are you thinking about balancing R&D investments
as well as potential profitability going forward?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Absolutely. So what are the main contributors to LIVMARLI growth that you've been seeing? And what do you expect to see from the growth
trajectory going forward?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Maybe just on Alagille and PFIC, can you talk to the competitive landscape and how you see that impacting your trajectory, if at all?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Okay. Maybe turning to biliary atresia. Can you tell us sort of the rationale here and what we should be looking for, upcoming data?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Absolutely. And thinking about the development strategy, what would be the theoretical next steps after data? Is there a potential for accelerated
approval and opening those conversations? And you mentioned bilirubin, how should we think about what would be clinically meaningful there
in terms of the change?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Okay. Great. And since you mentioned PBC, what should we be looking for in the timing of getting sort of the interim data in both PBC and PSC?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: All right. Great. And since, I guess, we'll be getting the data in -- or at least the more detailed data from PBC, how are you thinking about what's
clinically meaningful and commercially relevant in the context of the landscape and what you're hoping to see there?
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NOVEMBER 08, 2023 / 7:00PM, MIRM.OQ - Mirum Pharmaceuticals Inc at UBS BioPharma Conference
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Great. And I guess, maybe in the context of PPARs, how do you think the IBAT inhibitors will compare in terms of the magnitude of impact on itch?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: And how should we think about the time lines for potential data from the confirmatory portions of each of these studies?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Got it. And I guess from a regulatory perspective, help us understand what might be required in order to get a label for pruritus.
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Okay. Maybe turning back to the commercial strategy. Can you talk a little bit more about the rationale behind the deal with Travere and the value
proposition of CHENODAL and CHOLBAM?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: And you recently reported data in CTX. Can you tell us a little bit more about the commercial opportunity there? And I know it's available now but
medical necessity and just how filing will shape the opportunity.
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Okay. And maybe taking a step back, strategically, how are you thinking about potential for further business development or collaboration from
here?
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NOVEMBER 08, 2023 / 7:00PM, MIRM.OQ - Mirum Pharmaceuticals Inc at UBS BioPharma Conference
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Makes sense. Maybe just going back to biliary atresia with the few minutes left. Can you tell us a little bit more about the rationale for IBAT in biliary
atresia and your confidence that this could work?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Absolutely. And I guess, how should we think about the time line for when you might engage with the FDA and when we could learn more about
the strategy there?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Absolutely. Well, seeing how it's November now, any more granularity you can give us on when? Before the end of the year or no? It's okay...
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Thank you. Maybe just to close things out, can you remind us of your key milestones over the next 12 months?
Question: Eliana Rachel Merle - UBS Investment Bank, Research Division - Analyst
: Great. Well, with that, thank you, guys. Thanks for joining us, and thanks, everyone in the room for being here and joining us in Miami.
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