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: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Great. Thanks, Chris, for that presentation. (Operator Instructions) In the meantime, one of the questions we've gotten since you reported preliminary
revenue for 4Q is what that implies for the number of patients who were treated with LIVMARLI in the fourth quarter. Any way to kind of throw
some color on that?
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JANUARY 11, 2022 / 6:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc at JPMorgan Healthcare Conference
(Virtual)
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: And what about free drug? Were there also patients getting free drug in the quarter who might click over to being paid patients now that you've
got reimbursement, I think the slide said north of 90%?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. Other questions here. Can you say more about the LIVMARLI launch and sales force? And are you comfortable with estimates?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: What about the part -- and I like this question. Now what about the part about being comfortable with analyst estimates?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: With the -- I guess there's a question for this in almost every session. But with the current COVID surge, how is that affecting your launch at all? Is
that something you're kind of seeing interfere with the uptake or so much?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: With the MARCH study in PFIC coming out, I guess, in the back half of the year, what are the efficacy measures that you're going to be focusing on
that could further support the products activity? And how do you plan to release the data?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: A couple more questions coming in here. So thanks, everybody, for asking questions. Can you talk about start forms versus actually filled scripts,
whether they are paid or free?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: We just entered this one is how long it's taking to go from start form to build script? Is that a couple of weeks?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Maybe turning to volixibat. Can you help us understand the market potential for volixibat in ICP, PSC and PBC?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: And can you walk through what you expect to see with the interim analysis of Phase IIB OHANA study in ICP that could provide enough support
for registrational path and talk about how you plan to release that data?
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consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies.
JANUARY 11, 2022 / 6:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc at JPMorgan Healthcare Conference
(Virtual)
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: So I guess maybe 2 follow-ups to that. What's the right framework to think about the communication around each of those interims? And I think
on one of the last slides, it looked like the ICP update was kind of before the PSC update, both kind of second half next to them, but on the slide,
it was first for ICP. So is that the order in which to expect them?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. On the primary endpoint of serum bile acid in OHANA, what's the magnitude of delta you want to see between the treatment arm and the
placebo arm to consider it meaningful? And do you expect a dose response?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. And is there any secondary endpoint that you're measuring in OHANA that the FDA has highlighted as maybe most supportive for the
potential registrational strategy?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Okay. And what do you see as the potential duration of treatment with volixibat in ICP?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. And what about the Phase IIb VANTAGE study in PBC? How is enrollment progressing there?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Great. And then coming back around to VISTAS in PSC when can we expect full data from that study?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Maybe we can pull in for a couple of questions. How should we think about OpEx in 2022 relative to 2021?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: And what's your cash runway and which clinical milestones does it take you through?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Got it. Okay. I probably have time for one more coming in here on the portal. Can you talk about how you envision the cadence of scripts in '22
basically asking like, was there a bolus of patients or I guess not?
Question: Jessica Macomber Fye - JPMorgan Chase & Co, Research Division - Analyst
: Great. So we're out of time. So we'll leave it there. But thanks so much for the presentation and thoughtful Q&A, and thanks, everyone, for listening
in.
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