Mirum Pharmaceuticals Inc Q2 2024 Earnings Call Transcript

The following is excerpted from the question-and-answer section of the transcript.

Question: Dae Gon Ha - Stifel - Analyst : Hey, good afternoon, guys. Thanks for taking our questions and congrats on the progress. Two from us, one commercial and one clinical, I guess. Starting with commercial, as we look at the approval label across Alagille and PFIC in the US, it's kind of a palindrome between Livmarli and Biliary. So I was hoping if you can maybe comment a little bit on what you've learned from perhaps Biliary's experience in Alagille that you can implement to broaden Livmarli's reach within the PFIC segment before further label expansion can come down the pike. And then in terms of clinical side, Joanne, enrollment progressing favorably sounds great. Is there any initiative for you to perhaps accelerate enrollment into VISTAs and VANTAGE trials? Thanks so much.

Question: Dae Gon Ha - Stifel - Analyst : Great. Thank you very much.

Question: Mani Foroohar - Leerink Partners - Analyst : Hey, guys. Thanks for taking the question. A couple of quick ones. First, when we think about the novel expansion population Cholestatic pruritus, can you give us a sense of how you think about the duration of that study and the time horizon which we might see results, recognizing it's a little bit of a heterogeneous population, and then we have a commercial quick follow-up.

Question: Mani Foroohar - Leerink Partners - Analyst : That's helpful. And then when you think about sort of more on the commercial side, obviously, there's a little bit of a -- there's some element of seasonality in this market, and sort of the pendulum swings one way early in the year and tends to swing the other, certainly around 3Q, as we saw a couple years ago. How do we think about the tempo of new patient ads and any sort of operational details we should think about in terms of seasonality across the next few quarters looking forward?

Question: Mani Foroohar - Leerink Partners - Analyst : Okay. And should we, and on what time horizon should we expect sort of the sort of OUS pricing reference dynamics to play out? That's something we should think about sort of recurring and sort of eroding itself playing out over the course of the next couple quarters. Is that primarily a 2Q, 3Q event? Like how should we think about breaking that into sort of how we model?

Question: Mani Foroohar - Leerink Partners - Analyst : Perfect. Thanks, guys. I know you've got a bunch of people in the queue. I'll hop off.

Question: Jessica Fye - JPMorgan - Analyst : Hey guys, good afternoon. Thanks for taking my questions. I had a few. First, where should we look for presentation of the interim PBC data for Volixibat and what kind of additional details should we expect when you present those results? For example, are there subgroups where we should expect to learn more? Could we see its results broken down by severity of diseases defined by ALP levels or additional data on liver biomarkers or more details on the improvements observed in the fatigue dimension of the PBC40? And then separately on the business development front, can you provide a bit of color about just what type of assets and which disease areas are most interesting to you? And lastly, can you just comment on your IP estate across Livmarli and Volixibat in terms of what IP you currently have and any pending applications? Thank you.

Question: Robert Rafter - Morgan Stanley - Analyst : Hi, this is Robert Rafter from Mike. Thanks for taking our questions. Just in PFIC, do you expect to pursue a label expansion to patients below 12 months? And I can just talk about how many patients are typically diagnosed that are below 12 months and what the opportunity there is. Thanks.

Question: Robert Rafter - Morgan Stanley - Analyst : Thank You.

Question: David Lebowitz - Citi - Analyst : Thank you very much, for taking my question. With respect to the EXPAND trial, given the heterogeneous population, how do you think the FDA would view it from a label expansion perspective?

Question: David Lebowitz - Citi - Analyst : Got it. Thanks for taking my question. Thanks for the question.

Question: Steven Seedhouse - Raymond James - Analyst : Yeah, good afternoon. Thanks for the questions. Just on EXPAND how are you going to be measuring pruritus? Because it seems like this trial would include pediatric and adult patients, depending on the condition. So I'm just curious how you're going to standardize measuring the endpoint across disease types?

Question: Steven Seedhouse - Raymond James - Analyst : okay. And so the adults would be self-reported itch, or would there be a second observer? Yeah, so how does the endpoint look?

Question: Steven Seedhouse - Raymond James - Analyst : Okay. And then the formulation here, is this just going to be the same liquid formulation that's commercially available?

Question: Steven Seedhouse - Raymond James - Analyst : Okay. And then just last question -- and thanks for the multipart question. Are biliary atresia patients that enrolled in EXPAND that were maybe pruritic either on enrollment, and that's for -- excuse me, that enrolled in EMBARK eligible to enroll in EXPAND? Or is anyone who's been treated compassionately eligible to enroll in this study? Or are you excluding anyone with prior exposure to Livmarli?

Question: Steven Seedhouse - Raymond James - Analyst : So just a follow-up on that, Chris. If there are patients that responded by bile acids or bilirubin or some metric in EMBARK, are they eligible to enroll in EXPAND if they're biliary atresia patient?

Question: Steven Seedhouse - Raymond James - Analyst : Okay. Makes sense. Thanks a lot.

Question: Jon Wolleben - Citizens JMP - Analyst : A couple on PFIC for me. Wondering -- without talking about sales here, any metrics on the PFIC launch in the US. Things are going well. Things that you guys could improve upon throughout the rest of the year? And then with the label expansion recently, can you discuss the added risk of the propylene glycol toxicity? Any observation of that in the clinical trials? Or is this just a risk that FDA wanted to include?

Question: Jon Wolleben - Citizens JMP - Analyst : Got it. Thanks.

Question: Thomas Yip - H.C. Wainwright - Analyst : This is Thomas Yip substituting. I got a couple of questions for Ed. Perhaps first, just wondering for Chenodal how do you characterize the additional commercial opportunity in CTX as an on-label indication, I suppose in terms of quantity and also relative to Chenodal existing sales.

Question: Thomas Yip - H.C. Wainwright - Analyst : Understood. And maybe one more from us. Just one for volixibat. Regarding VANTAGE and VISTAS, has the FDA or the EMA reviewed the interim data that you have so far? Any feedback from any regulatory agencies?

Question: Thomas Yip - H.C. Wainwright - Analyst : Thank you so much for taking my questions.