Q1 2024 Mirum Pharmaceuticals Inc Earnings and Bussiness Update Call

House of Raymond James Please go ahead.

Speaker Change: Hey, good afternoon, thanks for taking the questions two separate questions I'll just ask them.

Raymond James: Both out because they're pretty straightforward first on the PBC readout.

You mentioned price improvement safety in serum bile acids. It would be the focus of of that data release and Im just curious if you'll also.

It'd be sharing liver function tests, all class a L T.

S T. A bilirubin just to get a sense of even from a safety standpoint, what's happening there.

Raymond James: If theres any impact of Av.

Raymond James: De novo bile acid synthesis on on any of those parameters and then the second one.

Just on business development would be curious your comments or thoughts on just the overall.

Do you have that are priorities for near them.

Over the next call. It 12 to 18 months are you thinking about expanding the pipeline or focusing on books about primarily thank you.

Speaker Change: Yeah, I think thanks, Steve for the questions. They all speak to and comments on kind of our business development efforts and let Joanne come back on the PVC Interims.

Joanne: For our strategy and approach to business development remains consistent with what we've done over the course of Merit I mean, it's very much in our DNA. It's how the company came to be and started.

Joanne: And its approach of being.

Joanne: <unk> about making sure anything that we look to bring on as something that we can add value to.

Joanne: That said.

Joanne: At terms and then.

Joanne: It helps grow the company.

Joanne: And it really across a number of different rare disease settings as.

Joanne: That's where we're looking I think we're in a position where.

Joanne: We are quite lucky in that there is a lot of <unk>.

Joanne: Both in the commercial business label expansion opportunities the elixir that.

Joanne: Developments that Theres no urgency to do something so we can remain disciplined in looking at ways to grow the company.

Joanne: And then maybe Joanne can speak to the PBC question, Yeah, Thanks, Chris and thanks for the question.

Joanne: I mentioned this is an interim analysis. So it's pretty limited in terms of scope, we're mainly looking at it to ensure safety and to select a dose moving forward. So with that we'll look at pruritus, we'll look at.

Joanne: Serum bile acids in safety in particular.

Joanne: The data sets can be paying limited. So we think it'll it'll be quite limited in terms of making any conclusion certainly about any other parameters I think we'd look to the final data set for that.

Speaker Change: Thanks, so much.

Speaker Change: Thanks for the questions.

Speaker Change: The next question on the line comes from Brian <unk> of Baird. Please go ahead. Your line is open.

Speaker Change: Hi, This is Luke on for Brian as we set expectations for advantage in particular thinking about a comp to the solid all car response study do you think the subgroup in that study with baseline <unk> greater than four is a reasonable top for pruritus benefit.

Speaker Change: And then are you aware of the 11 point scale. They used in that study is the same as the reported outcomes scale that youre using.

Speaker Change: Thanks for the question, Yes, I mean, the scale used it's similar I mean, there were some very minor differences, but for adults pruritus measurements in Registrational studies, that's kind of this is all in line with FDA guidance uses 10 scale, that's what we're using in our study.

Speaker Change: So there is some definitely some similarity there and the treatment effect in that subset that they looked at.

Speaker Change: It's not too far off with how we looked at our kind of.

Speaker Change: The change from placebo assumptions and powering rate, where we looked at the 175% difference from placebo, so kind of looking at potentially a little bit more effect from.

Speaker Change: From an eye that but in general it's really not too far off if youre thinking about study design assumptions.

Speaker Change: Now of course, we're quite excited about getting this data and seeing what that looks like a particular change from baseline.

Speaker Change: Which is really what what the patient experiences and what youre, what youre doing to address the burden of disease.

Speaker Change: Great. Thank you.

Speaker Change: Thanks for the question.

Speaker Change: The next question on the line comes from David Lebowitz of Citi. Please go ahead. Your line is open.

David Neil Lebowitz: Thank you very much for taking my question.

David Neil Lebowitz: The 175 point difference I'm sure right.

David Neil Lebowitz: Could you just elaborate as to whether you're talking about who the blinded portion or through the actual pivotal question at a subsequent time point.

Speaker Change: And perhaps.

David Neil Lebowitz: Give us some view of what that point and how you will use it to.

David Neil Lebowitz: Consider upsizing if that is needed.

David Neil Lebowitz: What type of thresholds, we can expect.

David Neil Lebowitz: Yeah.

Speaker Change: Yeah, well thanks for the question.

David Neil Lebowitz: We're not going to get into the details of the study design, but I just provided some of the Ah <unk>.

David Neil Lebowitz: Number so that you could get a sense of what kind of treatment effect. We're looking at are the.

David Neil Lebowitz: The 1.75, a treatment difference and the three in terms of center deviation that is just a general number that we're looking for the overall design of the study.

David Neil Lebowitz: So we won't be sharing any specifics in terms of you know kind.

David Neil Lebowitz: Kind of where we are with the income obviously will share the results of Ghansham and central will be blinded.

David Neil Lebowitz: We certainly hope that we'd be continuing the study.

David Neil Lebowitz: So that's what we hope to see in June and wouldn't want to add to that David is that the the measurement is actually looking at over time right. It's the amongst 345, sorry, four five and six.

David Neil Lebowitz: Of the of the treatment effect for the final analysis that does a lot to add power and try to deal with any potential for placebo response, because youre looking at multiple time points and how they roll into the analysis. So that's another factor to the study design applying what we are used from from the <unk> study in the adult settings.

Speaker Change: Got it. Thank you very much for taking my question.

Speaker Change: Yeah. Thanks for the question.

Speaker Change: The next question on the line comes from John Bolton of citizens JMP. Please go ahead. Your line is open.

Unknown Executive: Hey, Thanks for taking the question.

Unknown Executive: Just logistics.

Unknown Executive: One for me on PBC and PSC.

Unknown Executive: When you select the dose the patients on the prior dose get rolled back in it either placebo or the new dose or do you have to re enroll patients and you can can you comment on how long the enrollment could take two.

Unknown Executive: Complete and both of those studies.

Speaker Change: Thanks, John for the question just on timelines.

Speaker Change: I will give a more formal update on what we expect to see for the ticket to the full dataset in June when we had the interim but I'll, let joanne speak to a little bit of the mechanics of how patient flow through the study yeah. So we're going to be continuing we're continuing enrolment in the study at this point.

Joanne: And then will.

Joanne: We will be continuing with one active dose and placebo. So we expect to include all the patients ultimately in the analysis.

Joanne: And ultimately on blind the whole dataset.

Joanne: Thanks for the question John.

Speaker Change: Next question on the line comes from Robert <unk> with Morgan Stanley. Please go ahead. Your line is open.

Robert: Hi, This is robin on for Mike. Thanks for taking our questions can you just provide any color on the ongoing launch prep for <unk> and when do you expect patients on the expanded access program to get on Pedro Thanks.

Speaker Change: Yes, thanks for the question Rohit.

Speaker Change: And U S subs and launches underway.

Speaker Change: You're now seeing a.

Speaker Change: Prescriptions come in for Lamar lead competed patients now.

Speaker Change: We have we have talked about we have about 25 patients in the U S who are on clinical drug most of those are are eligible to roll over at this time and.

Speaker Change: We would expect them to come over to commercial drug in the coming quarters throughout this year.

Speaker Change: And so that's how we see the cadence linerboard.

Speaker Change: Thank you.

Speaker Change: The next question on the line comes from Ed I'll say from H C. Wainwright. Please go ahead. Your line is open.

Speaker Change: Hi, Good afternoon, everyone. This is Thomas Yip asking a couple of questions for Eric. Thank you for the kind of questions.

Wing Cheung Yip: So first following up on.

Wing Cheung Yip: U S performance for.

Wing Cheung Yip: For the smaller P critical.

Wing Cheung Yip: Just wonder of the $42 8 million on net sales in the first quarter, how much was it.

Wing Cheung Yip: Approximately and then also what are some early launch metrics that investors came up here.

Speaker Change: Thanks, Thomas for the question here in Q1, there really there's no P. If a contribution in there yet and we're just.

Speaker Change: The approval came in March and just now kind of rolling over those clinical patients so expect.

Speaker Change: The revenue contribution to be pretty minimal from <unk> over the next quarter or two as we get into the back half of the year, where we expect more of a full reimbursement.

Speaker Change: Got it and then switching gears.

Speaker Change: On the European front.

Speaker Change: And the interaction with EMA <unk> recently given you.

Speaker Change: Your expectation on a recommendation.

Speaker Change: Recommendation and the first half this year and if positive.

Speaker Change: Any ongoing commercial preparations for European market for music.

Speaker Change: Yeah. Thanks for the follow up there yet on the EMA discussion you have over the we have been active in discussing with the MAA and asthma as mentioned and feel confident in our arguments and hope to have an update on that soon so no formal determination yet.

Speaker Change: And then maybe Peter can speak a little bit to the launch prep in Europe for peak, yes, certainly upon a potential approval would be ready to launch with modeling Keybanc in Europe prescribers.

Peter: Prescribers for.

Speaker Change: E P Baker essentially identical to the prescribers with modeling for hours you'll syndromes.

Speaker Change: It will be ready to go with dossier submissions to health technology agencies, etcetera to workers pricing and reimbursement.

Speaker Change: That's a good level as well.

Peter: Okay, and then one last.

Peter: <unk> from us.

Speaker Change: Okay can you give us some.

Peter: The main drivers for you.

Peter: Product sales.

Peter: Slightly if I can move around quarter to quarter could be major factors.

Speaker Change: Okay.

Speaker Change: Yes.

Speaker Change: We mapped in the you know what the change healthcare cyber attack.

Speaker Change: And play for our entire portfolio I think if you look back over time.

Speaker Change: You know in the bile acid product sales. There is there are there's quarter to quarter volatility.

Speaker Change: Given the ultra rare nature of the disease and small number of patients over time.

Speaker Change: But do you see an opportunity to continue to build.

Speaker Change: On those products this year.

Speaker Change: Mid single digit year on year growth consistent with what historically is our expectation going forward really excited about potential approval by FDA next year for.

Speaker Change: Can you at all in CTX and the chance to go out and find more patients there.

Speaker Change: Understood. Thank you again for taking my questions and we look forward to the decision will be a recommendation soon and also your June presentation for Bluelinx about.

Speaker Change: Sounds good thanks Thomas.

Speaker Change: We have no further questions. Therefore, I will hand back the call to Christine <unk> for final remarks.

Christine: Great. Thank you all for joining us today really appreciate the interest in Maryland, and our programs and have a good evening goodbye.

Speaker Change: This concludes today's conference call. Thank you all for joining you may now disconnect your lines.

Speaker Change: [music].

Christine: I have a good evening goodbye.

Speaker Change: This concludes.