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 Fye - J.P. Morgan - Analyst
: Great, good morning, thanks for taking my questions. On the baseline ItchRO scores, curious if you think the modest differences across the arms
factored into the in the efficacy, your how you interpret these efficacy results in PBC?
And second question is, curious if you saw any improvements in alkaline phosphatase or any other liver markers apart from serum bile acids?
Question: Jessica Fye - J.P. Morgan - Analyst
: And just as a follow-up, can you speak to or both the OEM background meds looked like more broadly, for example, on what proportion of patients
were on background fibrates?
Question: Jessica Fye - J.P. Morgan - Analyst
: Thank you.
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JUNE 17, 2024 / 12:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc Interim Analysis Results
Question: Josh Schimmer - Cantor Fitzgerald - Analyst
: Congrats on the results and thanks for taking the questions. Maybe first, quick follow-up to that. Just because question, would you expect to see
improvement in those patients who did have ALT elevated and baseline? And was there any indication you were seeing that?
Question: Josh Schimmer - Cantor Fitzgerald - Analyst
: You also indicated for patients experienced SAEs, including one in the placebo arm. Can you provide additional details on what those were?
Question: Josh Schimmer - Cantor Fitzgerald - Analyst
: Got it, and then there were high rates of diarrhea as noted do you have split in terms of what percent or roughly what percent was mild versus
moderate and how long did it persist? Or what percent of patients did it not resolved?
Question: Josh Schimmer - Cantor Fitzgerald - Analyst
: And then the last question for me. What do you attribute the improvement in continuing that you are seeing in these patients?
Question: Josh Schimmer - Cantor Fitzgerald - Analyst
: Thank you. Congrats again.
Question: Gavin Clark-Gartner - Evercore ISI - Analyst
: Hi, guys. Let me congrats on the data also, and thanks for taking my questions. Just first, based on your market research, roughly what percent of
PBC patients have lower ALP scores and also moderate to severe pruritus?
Question: Gavin Clark-Gartner - Evercore ISI - Analyst
: It makes sense. Maybe you could just compare contrast how this compares to what we've seen volixibat previously? Thanks.
Question: Gavin Clark-Gartner - Evercore ISI - Analyst
: Great. Congrats again.
Question: Steven Seedhouse - Raymond James - Analyst
: Good morning. Congratulations, thanks for taking the questions. Chris, in your opening comments, you mentioned 60% of PSC patients having
pruritus. I assume that includes mild pruritus, but I just wanted to double check. Is that moderate to severe or is it (Inaudible)?
Question: Steven Seedhouse - Raymond James - Analyst
: Okay. It makes sense. Two clarifications on the data, just on the first on the fatigue result at week 16, was that persistent at six months? And then
on the SAEs just wanted to clarify if any of those were deemed treatment related, particularly the liver injury, SAEs you noted earlier? Thanks.
Question: Steven Seedhouse - Raymond James - Analyst
: Great. Thank you so much.
Question: David Lebowitz - Citi - Analyst
: Thank you very much for taking my question. First one on PBC, could you guess, elaborate on how you see and this looks like that might fit in the
treatment paradigm in comparison to mineral about [seladelpar] given the data datasets we've seen to this point.
Question: David Lebowitz - Citi - Analyst
: Thank you for that. In terms of the fatigue, is there any correlation between introduction and a suite that might play a role in the fatigue.
Question: David Lebowitz - Citi - Analyst
: Good to hear. And just lastly, on PSC, given no approved drugs and curate a certain what seemed like a reasonable endpoint? I guess what extent
have the discussions been with the agency on that as an endpoint? And would you say that they're very likely to accept it?
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JUNE 17, 2024 / 12:30PM, MIRM.OQ - Mirum Pharmaceuticals Inc Interim Analysis Results
Question: David Lebowitz - Citi - Analyst
: Excellent. Thank you for taking my questions and congratulations.
Question: Brian Skorney - Robert W. Baird & Co. - Analyst
: Hey, guys, thanks for taking the question. Maybe as a follow-up speed question on different and burden between PSC and PBC. Was the baseline
ItchRO requirement the same for VANTAGE and VISTAS? And can you talk at all about the relative screen fail rates for each indication based on that
score?
Question: Brian Skorney - Robert W. Baird & Co. - Analyst
: Got it. Thanks. Appreciate it.
Question: Mike Ulz - Morgan Stanley - Analyst
: Hi, guys, thanks for taking the question. And congrats as well. Maybe just one quick one on PSC. Can you just remind us of what the threshold was
on the interim pruritus analysis? And then maybe secondly, just PBC you talked about the potential for a much broader market opportunity than
some of the competitors, both in the front and second line setting. So maybe if you could give us just a breakdown between front-line and second-line
patients there? Thanks.
Question: Mike Ulz - Morgan Stanley - Analyst
: Got it. Thank you and congrats again.
Question: Ed Arce - H.C. Wainwright - Analyst
: Good morning, everyone. Congrats on the impressive data, and this is time for asking a couple of questions for us. So first question, perhaps can
you provide a breakdown of the PBC patients who experienced diarrhea between the 20 milligram and 80 milligram cohort?
Question: Ed Arce - H.C. Wainwright - Analyst
: Okay. Okay. Got it. And then perhaps if you could discuss what are some potential mechanism for these have taken improvement in PBC patients
in very short from content?
Question: Ed Arce - H.C. Wainwright - Analyst
: Got it. And that's good. And perhaps one final question for the VISTAS or PSC study. So regarding the efficacy threshold, is this based on a specific
figure or rather a trend of improvement and as mentioned?
Question: Ed Arce - H.C. Wainwright - Analyst
: Okay. And congratulations again on the impressive data.
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