Mirum Pharmaceuticals Inc Q3 2024 Earnings Call Transcript

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

Question: Gavin Clark-Gartner - Evercore ISI Institutional Equities - Analyst : Congrats on all the progress and thanks for taking the questions. So I just wanted to focus in on Fragile X. So first question here, maybe just for those who are less familiar, what's the current registrational endpoint in Fragile X? Is it on behavioral or cognitive clinical endpoints? And is there any opportunity to pursue an accelerated path?

Question: Gavin Clark-Gartner - Evercore ISI Institutional Equities - Analyst : Got it. That's helpful. And are you planning to have any engagement with regulators prior to initiating the Phase II or will next engagement likely be end of Phase II meeting?

Question: Gavin Clark-Gartner - Evercore ISI Institutional Equities - Analyst : Got it, thanks.

Question: Jessica Fye - J.P. Morgan Securities LLC - Analyst : Hey, guys, good morning. Thanks for taking my question, I had a couple. First, on volixibat, can you share a little more detail on the data we should expect from the late-breaking presentation at AASLD from the VANTAGE interim? How much of an update will this be? What would you focus the street on? And what subgroups, if any, will you present? That's one. Second, LIVMARLI, it looks like growth was really solid quarter over quarter, year over year. I think last year, 3Q, you talked about seasonal headwinds. Did you see any of that this year? And then lastly, on business development in the context of this latest in-licensing, how do you see the company evolving with this asset kind of going beyond the kind of rare liver disease kind of legacy focus? And do you expect to kind of remain active on the business front on the back of this in-licensing? Thank you.

Question: Jessica Fye - J.P. Morgan Securities LLC - Analyst : Thank you.

Question: Dae Gon Ha - Stifel, Nicolaus & Company, Inc. - Analyst : Hey, great, good morning, Congrats on the quarter, looking pretty bright ahead, I guess a couple of questions from me as well, just thinking about the OpEx growth or I guess, not OpEx. But the revenue guidance increase, just curious if it is primarily driven by Alagille growth ahead in Q4 or is it really going to be about PFIC as we think about modeling purposes? When we think about EXPAND, Joanne, can you expand a little bit more? You said sites are opening. But in terms of outlook on cadence of patient enrollment and -- any kind of timeline that you envision in terms of enrollment completion, that would be great. And then lastly, on the MRM-3379, just curious about your overall strategy on the neurology side. I mean, Fragile X is the starting point here. But are you thinking about expanding on the PDE4 inhibition for other indications? Is there anything that's lower-hanging fruit after Fragile X? Thanks so much.

Question: Dae Gon Ha - Stifel, Nicolaus & Company, Inc. - Analyst : And then, on the sales trends?

Question: Dae Gon Ha - Stifel, Nicolaus & Company, Inc. - Analyst : Makes sense. Thanks for taking our questions, guys.

Question: Brian Skorney - Robert W. Baird & Co., Inc. - Analyst : Hey, good morning, guys. Thank you for taking my question. On the PDE4D, I know Shionogi has run a Phase III with zatolmilast in Fragile X and so, I'm just kind of wondering, is the Phase II data with that sort of the basis of the licensing deal here? And then can you maybe compare and contrast to your molecule with the Shionogi one? And would you anticipate initiating Phase II before the readout of that study next year or would you maybe wait on those results to decide on potential endpoints and power? Thanks.

Question: David Lebowitz - Citi Investment Research (US) - Analyst : Thank you very much for taking my question. Can you compare the decisions to in-license the Travere assets with 3379 and what you are thinking in general about your strategy relative to business development going forward?

Question: Timur Ivannikov - Raymond James & Associates, Inc. - Analyst : Hi, this is Timur Ivannikov on for Steve Seedhouse. Congrats on the quarter. We have a couple of questions about Fragile X. So first one is, do you have plans to enroll patients younger than 18 years old? And at what point could you enroll those patients? And then in terms of the molecule specifics, do you know how much more CM activity you can achieve in the brain? That is how much is the xanthomas leaving on the table versus normal?

Question: Michael Ulz - Morgan Stanley & Co. LLC - Analyst : Maybe just one on chenodiol and CTX now that you sort of have a PDUFA date late December. Maybe just remind us what the potential market opportunity is there and what impact getting that in the label might have on sales?

Question: Michael Ulz - Morgan Stanley & Co. LLC - Analyst : Great, thank you.

Question: Thomas Yip - H.C. Wainwright & Co., LLC - Analyst : Hi, good morning, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking our questions. So first question, perhaps following on the previous question. Can you give us more details on payer discussions for PFIC in the US and EU? I suppose specifically, what percentage of target prescribers have you reached so far?

Question: Thomas Yip - H.C. Wainwright & Co., LLC - Analyst : Got it. And then perhaps another question for LIVMARLI, just wondering if you can discuss enrollment progress for the EXPAND study or perhaps any initial investigator feedback that you have so far?

Question: Thomas Yip - H.C. Wainwright & Co., LLC - Analyst : Understood. Perhaps one more question from us. So with in-licensing of the 3379, does that mean lucatinib disorder is now a target for Mirum or just rare genetic diseases in general?

Question: Thomas Yip - H.C. Wainwright & Co., LLC - Analyst : Understood. Thank you again for taking my questions. Looking forward to hearing more about 3379 in the coming months. REFINITIV STREETEVENTS | www.refinitiv.com | Contact Us consent of Refinitiv. 'Refinitiv' and the Refinitiv logo are registered trademarks of Refinitiv and its affiliated companies. NOVEMBER 12, 2024 / 1:30PM, MIRM.OQ - Q3 2024 Mirum Pharmaceuticals Inc Earnings Call