Mirum Pharmaceuticals, Inc.

Hello and welcome to the Mirim Pharmaceutical third quarter 2025 financial results and business update. My name is Harry and I'll be your operator today. All lines are currently in listen-only mode and there'll be an opportunity for Q&A after management's prepared remarks. To enter the queue for questions, please press star followed by one on your telephone keypad. I would now like to hand the conference over to Andrew McKibbin, SVP of Strategic Finance and Investor Relations. Please go ahead.

Thanks, Harry, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceuticals' third quarter 2025 conference call. I'm joined today by our CEO, Chris Peets, our President and Chief Operating Officer, Peter Radovich, our Chief Medical Officer, Joanne Kwan, and Eric Bierkle, our Chief Financial Officer. Earlier today, Miriam issued a news release announcing the company's results for the third quarter 2025. Copies of this news release and SEC filings can be found in the Investors section of our website. Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements based on management's current expectations, including statements regarding Miriam's programs and market opportunities for its approved medicines and product candidates. These statements represent our judgment and knowledge of events as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed. We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10-Q and subsequent SEC filings for more information. With that said, I'd like to turn the call over to Chris. Chris?

Thanks, Andrew, and good afternoon, everyone. 2025 continues to be an outstanding year for MIRA. We've created a leading rare disease company, purpose-built to create and deliver life-changing medicines to patients. Our success comes from that foundation, a team deeply connected to patients and families, turning their insights into meaningful therapies and measurable performance. In the third quarter, we delivered strong commercial results, advanced our clinical pipeline, and strengthened our financial foundation. I'm proud of the way our team continues to execute with focus and consistency. First, on commercial performance, we reported third quarter revenue of $133 million, representing a nearly 50% year-over-year increase over the same period last year. This performance reflects the strength and breadth of our commercial portfolio, including continued momentum from the US PFIC launch and expanding demand from our international markets. Turning to R&D, we remain on track for three potentially pivotal readouts over the next 18 months. First up is the VISTA's Phase IIb study in PFC. With enrollment complete, we expect to announce top-line data in the second quarter of 2026. With a successful interim analysis last year and a consistent body of supporting data with IVAT inhibitors across multiple cholestatic diseases, We're optimistic about Elixabat's potential to become the first approved treatment in this setting. We're also progressing well with our Vantage study of Elixabat and PDC, the Expand study of Lithmarly and ultra-rare cholestatic conditions, and our newly initiated Phase II study of MRM3379 and Fragile X syndrome. We've also taken meaningful steps to further strengthen our financial performance. This quarter, our cash balance grew significantly, and we recognized positive net income for the first time. This is an important milestone that highlights the operating leverage in our commercial model. It's been another solid quarter of execution for Merum. I want to thank the entire Merum team for their continued dedication to patients. We built a high-growth, cash flow positive rare disease company with a broad pipeline and global footprint, and we're just getting started. And with that, I'll hand the call over to Peter.

Peter? Thanks, Chris. Q3 was another excellent quarter for Merit, with total net product sales of $133 million. This was driven by continued robust performance of Livmarlie in both the U.S. and international markets, as well as steady contribution from our bile asset portfolio. Livmarlie net product sales totaled $92 million for the quarter. In the U.S., Livmarlie demand remains healthy in both Algea syndrome and PFIC, with $64 million in net product sales. Allergy syndrome growth remains durable, and PPIC continues to contribute meaningfully, reflecting the real-world benefit of expanded diagnosis and increased genetic screening. As we begin reaching into broader segments of the medical community, particularly adult-focused providers, we're finding that genetic testing is still less embedded in practice and often requires more education and dialogue. So we view this as an area where sustained engagement can continue to drive incremental gains. Internationally, the Marley demand continues to grow, with 28 million in net product sales this quarter. Demand across our direct and partner markets remains robust, supported by expanding reimbursement and launches in new geographies. Q3 was the first full quarter of commercialization for our partner Takeda in Japan, with in-market adoption dynamics generally consistent with Liv Marley's US launch. Our bioacid medicines, Colbom and Cetexly, generated 41 million in net product sales this quarter, supported by increased CTX patient finding following Cetexly's FDA approval earlier this year. And I'm happy to say that we now expect to land in the upper end of our prior full-year 2025 guidance range with $500 to $510 million in revenues. This reflects the continued strength of our U.S. business in both Allergy Syndrome and PFIC, steady contributions from our bile acid portfolio, along with the typical quarter-to-quarter variability in international partner and distributor ordering patterns. Looking ahead, we continue to see substantial growth potential across our portfolio, with peak revenue potential for Livmarlie, Philipsabat, and MRM-3379, each exceeding 1 billion. And then with that, I'll turn it over to Joanne for an update on the pipeline. Joanne?

Thanks, Peter. I'm pleased to provide an update on the continued progress across our clinical pipeline, where we're seeing continued collaboration and momentum with physicians and patients across all of our ongoing studies. Starting with Velixibat, we completed enrollment in the Phase IIb VISTA study in primary sclerosing cholangitis, or PSC, and expect to announce top line data in the second quarter of 2026. PSC represents a significant area of unmet need with no approved therapies and limited treatment options. We're deeply grateful to the investigators and the PSC patient community for their partnership in advancing this important study. As a reminder, the outcome of the interim analysis of the VISTA study last year was what we'd hoped for. The recommendation was to keep the current sample size which we believe reflects a strong signal for the final analysis. It's worth noting that the study was powered using conservative assumptions, a placebo-adjusted treatment effect of 1.75 points, and a standard deviation of three. A case series is being presented at AASLD of eight PSC patients treated with Meralixibet under our compassionate use program, a continuation of a case series presented earlier this year at DDW. All of these patients had meaningful reductions in pruritus and four of the eight had complete resolution. This data supports a role for IVAD inhibition as treatment for PSC. Turning to PBC, the Vantage study continues to progress well, and we expect to complete enrollment next year. Interim data presented last year demonstrated statistically significant improvement in pruritus, meaningful reductions in serum bile acids, and encouraging improvements in fatigue. We're excited to advance this study through the informatory stage. Additional analyses from the Vantage interim will be presented at AASLD, which highlight the decreases in fatigue and improvement in sleep in the LexVat patients, as well as showing a decrease in IL-31 in treated patients. Our EXPAND study evaluating Livmarly and additional settings of cholestatic pruritus is also enrolling well. This study is designed to broaden access to patients across multiple rare cholestatic diseases who currently have few or no treatment options. It represents a meaningful label expansion opportunity, and we're targeting enrollment completion in 2026. Finally, I'm excited to share that we've initiated our phase two study of MRM3379, our brain penetrant PDE4D inhibitor for Fragile X syndrome. The preclinical data we recently presented from a mouse FMR1 knockout model of Fragile X showed that MRM3379 reversed the disease phenotype across multiple behavioral assessments, and increases our confidence in the importance of this pathway in Fragile X. Overall, we're very encouraged by the progress across our development programs and look forward to upcoming milestones in 2026. With that, I'll turn the call over to Eric to discuss our financial results. Eric?

Thanks, Joanne, and good afternoon, everyone. We delivered another solid quarter of financial performance, highlighted by total net product revenue of $133 million representing a 47% increase over the prior year and reflecting growth across all our commercial medicines. This quarter included approximately 5 million in sales to our partner Takeda in Japan. We do not expect additional sales to Takeda in Q4 of this year. Total operating expense for the quarter ended September 30 was 130 million, which includes R&D expense of 43 million, SG&A expense of $62 million and cost of sales of $26 million. Expenses for the quarter included non-cash stock-based compensation expense of $18 million and intangible amortization and other non-cash items of $6 million. The intangible amortization and other non-cash items expense are largely reflected in our cost of sales. Our cash operating margins continue to improve, and we delivered gas profitability in the third quarter, generating approximately $3 million in net income. While this reflects the strength and scalability of our business model, we view quarterly gas profitability as a milestone, not yet a consistent expectation as we continue to invest in growth. Cash, cash equivalents and investments were $378 million at September 30, an $85 million increase from the beginning of the year. We continue to be well-funded and financially independent, providing us the resources required to expand our patient impact and grow our business. With that, I'll turn the call back to Chris. Thanks, Eric.

Before we open the call for questions, I want to close by reflecting on what's been an incredibly productive quarter. Across every dimension of our business, commercial, clinical, and operational, we continue to execute with purpose and discipline, anchored by the same patient-centric approach that's driven our success from the start. That's what's enabled us to become a high-growth, cashflow-positive, leading rare disease company. Thanks again to the Merum team and to the patients and families who inspire our work every day. With that, operator, please open the call for questions.

Thank you. Now opening the call. If you would like to ask a question, please press star one on your telephone keypad. If you change your mind and would like to exit the queue, please press star followed by two. And finally, when preparing to ask your question, please ensure that your phone is unmuted locally. Our first question will be from the line of Jessica Phi with JP Morgan. Please go ahead. Your line is open.

Hey, this is Abdul for Jess. We just had two questions. What are going to be the key drivers of Liv Marley's performance as we look at 2026? And can you talk about why the midpoint of the new guidance range now implies 4Q REFs flat sequentially from 3Q? I don't think we saw that dynamic last year. Thanks.

Hi, Abdul. Thanks for the question. On key drivers into 2026, I mean, we see a lot of basically what we have today rolling forward into next year. You know, we expect it will probably give guidance early in the year next year on what that year looks like. But we are in early innings of the PFIC launch. both in the U.S. and internationally. So expect that to continue to build in over time. And I think for, you know, the guidance this year for Q4, maybe I'll ask Peter to speak to what we see from kind of the quarter-to-quarter dynamics.

Yeah, and then the guidance. Oh, thanks, Chris. Yeah, I appreciate the question, Abdul. You know, the main dynamic is tried to highlight in our prepared remarks. We see growth for live marley U.S. We see the bile acid growth. portfolio continuing to do what it does. It's really the Liv Marley International line where we expect variability as we move quarter to quarter. As we've talked about before, that business has periodic large orders from distributors, and we saw those come in in Q3. We also mentioned that we had Decatur revenue in Q3, which we also had Q1 and Q2 that we don't expect in Q4. So there's a fair bit of an inventory build there. So really the dynamic is in the Liv Marley International line. Thank you.

Thanks for the question.

The next question will be from the line of Josh Schimmer with Cantor. Please go ahead. Your line is open.

Thanks for taking the questions. Maybe I have two quick ones. First, what trends are you seeing in terms of adoption of the solid tablet formulation of livmerli and what percent of cells are for that versus the liquid? And then for velixibat, What are you thinking in terms of the appropriate price analogs, especially after we've seen a significant increase in rare orphan disease prices perhaps over the last year, particularly for conditions that perhaps are less prevalent than PBC and more aligned with PSE? Thank you.

Thanks for the questions, Josh. Yeah, so in terms of the solid tablet just, you know, launched in the US in mid-June, so this is really our first full quarter with it. And, you know, we've seen a very encouraging kind of uptake and really switches from the liquid. So, if you look at the prescribing information, patients are eligible to switch if they're at least 25 kilos. I think what I could say is that, you know, substantial proportion of those who are eligible based on their weight are switching. So, certainly excited about what that can mean long term. in terms of persistence and adherence and an easier single tablet per dose format that would be preferred by these adolescents and adults. So excited about that dynamic. And then, yeah, if it looks about pricing, obviously haven't made a final decision there. I've monitored those dynamics that you were talking about. We've kind of, you know, base case thinking. You can look at the other, you know, PPARs and the other products that are kind of approved in PBC at 130 to 150, but we're still analyzing. I think it's kind of too early to say what the right pricing strategy is for .

Thank you. Thanks for the questions.

The next question will be from the line of Gavin Clark-Gartner with Evercore. Please go ahead. Your line is open.

Hey, guys. Thanks for taking the question. Just to add one, what's your expectation for paragraph four filers? Maybe just helpful to lay out your confidence in your whole IP portfolio, especially around the method patents and including BlixPath. Thanks.

Hey, Gavin. Thanks for the question. Overall, I mean, we're in the window where we could potentially see that and kind of all routine for this point in the lifecycle for Lidmarly. and really quite confident in our overall IP position. In particular, you mentioned the method patents that are specific to dosing of Lipmarly and these indications. We've seen this has been really the key fundamental observation that's made all of MIR impossible, and the IP behind it we see is quite strong and in a great position and prepared to defend it. So more to come if and when we do see any filers, but nothing today. Great.

Thanks. Thanks for the question.

The next question will be from the line of James Condoulas with Stifel. Please go ahead. Your line is open.

Hey. Thanks for taking our question. This is Mark on for James. So, you know, recently on earnings, Shinogi, seem to suggest it's still an open question around sort of what exactly the best endpoint is for their Fragile X study. We wanted to see if you guys had any perspectives on that and sort of the implications for your program that you initiated this year. And then we had a second question on PSC and these patients typically kind of have inflammatory disease, sort of like comorbidities and We know that iBAT inhibitors by nature sort of have some of these GI side effects. So I'm curious your thoughts on the safety risks there. And if you can see sort of anything in the blinded data on, like, GI side effects and whether those look any materially different than, say, PBC or allogels. Thank you.

Thanks for the question, Mark. Can't really speculate too much on Shionobi's. update and what's going on underneath that, but I'm going to turn it to Joanne to talk a little bit about our endpoint strategy and what we're, our approach on our programs.

Yeah, thanks for the question. You know, we feel that we're in a good spot at this point. You know, the preclinical data in terms of this pathway, the importance of this pathway in Fragile X is quite strong. We recently presented some preclinical data with our compound in a mouse model, mouse knockout model, which, you know, supports efficacy in us moving forward. And then we've also, you know, had very good engagement with the community, with patients and with physicians. We also had a very successful and engaging pre-ND meeting with the FDA earlier this year, and they're entirely aware of the range of endpoints that we're looking at, and we're, you know, well aware of the types of validation that are needed for these types of outcomes. So I think we're actually in a pretty good spot. You know, a lot of interest from the community, and we're looking forward to conducting the study and seeing what we see.

And then on the PSC safety sample, I actually looked at Joanne for that.

Yeah. And so with regards to that, you know, for the PSC study, you know, we've had a data monitoring committee following with us. And so no issues have been raised, no suggested modifications to the protocol. So we feel pretty comfortable there's no big safety issues here. Feel pretty comfortable with, you know, moving forward with the way the protocol was initially designed. So it's not, no issues have emerged there.

Profile overall is consistent with what we know about iPads at this point.

Thanks. Thanks for the questions.

The next question will be from the line of Joseph Foam with TD Cowan. Please go ahead. Your line is open.

Hi there. Good afternoon. Thank you for taking my questions and congrats on the progress. Maybe on the PSC study now that that one is fully enrolled, are you able to talk a little bit about the baseline criteria of the patients that were enrolled, especially as it relates to the population that was studied in the interim analysis population? And maybe second, can you also discuss a little bit the importance of hitting on quality of life measures or bile acid in the distant itch, and will those secondary endpoints be provided in the top-line release in the second quarter? Thank you.

Thanks, Joseph, for the question. I think overall we've not, you know, planned to present or analyze some of the baseline criteria at this point. What we know from, and we can take it more generally from the enrollment criteria and what was in the interim, is the patients are selected for itch, so you do have quite elevated baseline pruritus scores. And from what we're seeing, it's quite representative of the PSC population in terms of background disease, background medications, things like that. So overall, kind of in line with what we expected for the population. And shifting to the question about endpoints, the focus from a regulatory standpoint is 100% on that pruritus endpoint being the outcome that we've discussed with FDA. We do expect to, are excited about and expect to see, based on other settings, expect to see movement on things like fatigue and the bile acids. Bile acids obviously being a key mechanistic marker, fatigue being a really important measure for patients. But again, those are secondary for a reason. The regulatory path is entirely through that pruritus endpoint.

Thank you. Thanks for the questions.

The next question will be from the line of Ryan Deshner with Raymond James. Please go ahead. Your line is open.

Thanks, and congrats on the quarter. Can you remind us what went into the decision to offer BID dosing for the EXPAND study, and how would you expect the dosing instructions to look on an expanded label in colostatic pruritus patients? And then I have a follow-up. Thanks.

Thanks, Ryan, for the question. Simple answer is empirical, right? So this is based on observations we've had in compassionate use settings at dose levels that have explored across a range in this kind of all in the bracket of these elevated dose levels from the allogeal label up to the PFIC label. And empirically, this is where we've seen really great response stories from compassionate use examples.

And Brian, you said you had a follow-up as well? Yeah. Real quick, how big of an impact has the government shutdown been so far for things like genetic screening programs and other programs related to AllerGeo and PTEC? Thanks.

Today, no impact that we've seen across kind of all of our interactions with customers and really across the business.

Thank you. Good question. The next question will be from the line of . Please go ahead. Your line is open.

Hey, guys. We have Ryan on for Mani. Thanks for taking our question, and congrats on the quarter. Can you just talk a little bit about the pace of new PFIC ads that you guys saw in the third quarter compared to the second quarter? I know you talked a lot about genetic testing and new patient diagnoses. And then, you know, maybe more broadly, as you guys start to see, you know, consistent positive cash flow and you have several launches on the horizon, maybe just talk through your BD strategy about adding more products to the pipeline. Thanks. Yeah. Thanks for the question.

I can turn it over to Peter to jump into those.

Yeah. You know, in terms of the pace of PPIC ads, it continues to be healthy. It continues to come from a broad, you know, patient population, everything from infants to adults that we've kind of commented on that is a dynamic where the paradigm is really being changed with adult providers to think about genetic cholestasis as kind of a clinical entity to be suspicious about. So, that's kind of an educational effort. And, you know, some of the major medical centers are on board with that. They're looking into genetic causes of cholestatic liver diseases and the patients they can't explain with other diseases, but most aren't, right? So that's just kind of a gradual effort, but it's continuing to bear fruit in Q3. Then, oh, BD, yeah, do you want to?

Sure, I mean, the thing we'd say on BD is, you know, since the beginning of the company, that's really been at our core is, looking for underappreciated programs. So we continue to do that and expect to always be active doing that. But we're in just a fantastic position where there's no urgency and no need. So we have a very high bar. And as you can see from the programs we've brought in since the start of the company, we look for good value creation opportunities. So that will continue to be the standard we take going forward. in the company to grow and build and, you know, optimistic about adding more down the road.

Appreciate it.

Thanks again.

Thanks for the question.

Next question will be from the line of Mike Holtz with Morgan Stanley. Please go ahead. Your line is open.

Hi. This is Rohit on for Mike. Thanks for taking our questions. Just with the recent literates about PDUFA announced for GSK, how do you see the competitive dynamics playing out in PBC? Thanks.

Thanks for the question. I think two overarching things to think about for the competitive landscape in PBC. One is just kind of a reminder on lines of therapy and where the program plays. And in the Vantage study, there is no baseline alkaline phosphatase criteria. So our program incorporates both first and second line PBC settings. So those that have stable alkaline phosphatase on UDCA, that likely wouldn't be a treatment candidate for some of the PPARs that are recently launched. but still have itch, that's a candidate for Glixibed study, and we expect ultimately Glixibed marketed treatment. And then with respect to Lenarixibed as a competitor, we're very excited about the interim data that we saw from the Vantage study and what it means for the dose level that was selected. The placebo-adjusted difference that we saw on itch in that data set It was striking. It led to breakthrough designation. It's really everything that we'd hoped to see from all that we've learned about dosing of this mechanism in these settings. So quite excited about the competitive profile of Elixabed, given that highly active dose level. Thank you. Thanks.

Questions?

The next question will be from the line of John Wallaban with Citizens. Please go ahead. Your line is open.

Hey, thanks for taking the question. Wondering if you guys are anticipating seeing similar disease-modifying effects over time with the Lixabet, as you saw with Livmarly and PSC and PBC, and if so, you know, what would be the timeframe, and do you think that would be an important consideration for adoption and use over time?

Hey, John, thanks for the question. I mean, the overarching first thought there is the first readouts here we think are probably too soon to be looking at that and focused on the itch endpoint and really see that as the launch profile. But I'll turn to Joanne to talk through some of what we'll be looking at and what we'll be able to see over time from the program.

Yeah, thanks for the question. As Chris alluded to, the whole discussion, especially with the regulators, has been around How do we get something in PSC approved? And clearly, that's with a paritis endpoint. At this point, in the field of PSC, that's really the only approval endpoint. Obviously, we'll look at other things, look longer term. We do expect those types of endpoints may take quite a long time to evolve. We'll continue to follow these patients. And obviously, we'll continue to engage with the agency in terms of appropriate endpoints. But we do think a concrete path forward is with paritis. And we're pretty confident in terms of, you know, the ability to affect that in a positive way for patients.

Thanks, guys. Thank you. And with no further questions on the line at this time, I would like to hand the call back to Chris Peets for some closing remarks.

Great. Thanks again, everyone, for joining us today and for your continued support. We look forward to updating you next quarter. Good afternoon.

This will conclude the Murham Pharmaceuticals third quarter 2025 financial results and business update. Thank you to everyone who was able to join us today. You may now disconnect your lines.