Mirum Pharmaceuticals Q3 2025 Earnings Report

Mirum Pharmaceuticals EPS Results

• Actual EPS: $0.05
• Consensus EPS: -$0.10
• Beat/Miss: Beat by +$0.15
• One Year Ago EPS: -$0.30

Mirum Pharmaceuticals Revenue Results

• Actual Revenue: $133.01 million
• Expected Revenue: $130.11 million
• Beat/Miss: Beat by +$2.90 million
• YoY Revenue Growth: +47.10%

Mirum Pharmaceuticals Announcement Details

• Quarter: Q3 2025
• Date: 11/4/2025
• Time: After Market Closes
• Conference Call Date: Tuesday, November 4, 2025
• Conference Call Time: 4:30PM ET

Mirum Pharmaceuticals (NASDAQ:MIRM) is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapies for rare cholestatic liver diseases. The company’s primary focus lies in addressing the unmet medical needs of patients suffering from genetic and progressive forms of pediatric liver disorders, where limited treatment options currently exist.

Mirum’s lead product candidate, maralixibat (Livmarli), is an ileal bile acid transporter inhibitor designed to reduce systemic bile acid accumulation and alleviate associated pruritus and liver damage. Maralixibat has achieved regulatory approval in the United States for the treatment of cholestatic pruritus in patients with Alagille syndrome and is under review or in clinical development for other rare cholestatic conditions, including progressive familial intrahepatic cholestasis (PFIC). In addition to its lead compound, Mirum maintains a pipeline of product candidates aimed at expanding the therapeutic options available to patients with orphan liver diseases.

Founded in 2016 and headquartered in Brisbane, California, Mirum Pharmaceuticals operates with a patient-centric approach that emphasizes collaboration with advocacy groups and key opinion leaders in hepatology. Under the leadership of President and Chief Executive Officer Derek Chalmers, the company is actively pursuing regulatory approvals and commercial partnerships to bring its therapies to patients in the United States and international markets.

Mirum Pharmaceuticals Q3 2025 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: Mirum reported strong Q3 results with $133 million in net product revenue (≈47% YoY growth), achieved GAAP profitability (~$3 million net income) and ended Q3 with $378 million in cash and investments, and now expects to land at the upper end of its 2025 guidance ($500–$510 million).
• Positive Sentiment: LIVMARLI continues to drive growth with $92 million in Q3 sales (US $64M, international $28M), growing adoption of the new solid tablet formulation, and ongoing PFIC patient-finding that the company expects to further expand over time.
• Positive Sentiment: The clinical pipeline is advancing with potentially pivotal readouts ahead — VISTAS (Volixibat in PSC) enrollment complete and top-line data expected in Q2 2026, ongoing VANTAGE (PBC) and EXPAND studies, and initiation of a phase 2 study of MRM-3379 in Fragile X.
• Negative Sentiment: Management flagged near-term revenue variability (noting large, lumpy international distributor orders and no expected Takeda Japan sales in Q4), and said Q3 GAAP profitability is a milestone rather than a consistent expectation as they continue to invest in growth.
• Neutral Sentiment: The company reports no emerging safety signals in PSC (DMC review) and strong confidence in its IP position and regulatory path (pruritus as the primary approval endpoint for Volixibat), while pricing for Volixibat and competitive dynamics in PBC remain under evaluation.

Presentation

[Operator]

Hello and welcome the Mirum Pharmaceuticals 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 McKibben, SVP of Strategic Finance and Investor Relations. Please go ahead.

[Andrew McKibben, SVP of Strategic Finance and Investor Relations at Mirum Pharmaceuticals]

Thanks, Harry, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals Third Quarter 2025 Conference Call. I'm joined today by our CEO, Chris Peetz, our President and Chief Operating Officer, Peter Radovich, our Chief Medical Officer, Joanne Quan, and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Mirum 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 Mirum's programs and market opportunities for its approved medicines and product candidates.

[Andrew McKibben, SVP of Strategic Finance and Investor Relations at Mirum Pharmaceuticals]

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.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks, Andrew, and good afternoon, everyone. 2025 continues to be an outstanding year for Mirum. 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.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Turning to R&D, we remain on track for three potentially pivotal readouts over the next 18 months. First up is the VISTAS phase 2b study in PFIC. 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 IBAT inhibitors across multiple cholestatic diseases, we're optimistic about Volixibat's potential to become the first approved treatment in this setting. We're also progressing well with our VANTAGE study of Volixibat in PBC, the EXPAND study of LIVMARLI in ultra-rare cholestatic conditions, and our newly initiated phase 2 study of MRM-3379 in Fragile X syndrome. We've also taken meaningful steps to further strengthen our financial performance. This quarter, our cash balance grew significantly, and we recognize positive net income for the first time.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

This is an important milestone that highlights the operating leverage in our commercial model. It's been another solid quarter of execution for Mirum. I want to thank the entire Mirum team for their continued dedication to patients. We've 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.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Thanks, Chris. Q3 was another excellent quarter for Mirum, with total net product sales of $133 million. This was driven by continued robust performance of LIVMARLI in both the U.S. and international markets, as well as steady contribution from our bioacquired portfolio. LIVMARLI net product sales totaled $92 million for the quarter. In the U.S., LIVMARLI demand remains healthy in both Alagille syndrome and PFIC, with $64 million in net product sales. Alagille syndrome growth remains durable, and PFIC 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.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Internationally, LIVMARLI 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 LIVMARLI's U.S. launch. Our bioacquired medicines, CHOLBAM and CHENODAL, generated $41 million in net product sales this quarter, supported by increased CHENODAL patient finding following CHENODAL'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-$510 million in revenues. This reflects the continued strength of our U.S. business in both Alagille syndrome and PFIC, steady contributions from our bioacquired portfolio, along with the typical quarter-to-quarter variability in international partner and distributor ordering patterns.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Looking ahead, we continue to see substantial growth potential across our portfolio, with peak revenue potential for LIVMARLI, Volixibat, and MRM-3379 each exceeding $1 billion. And with that, I'll turn it over to Joanne for an update on the pipeline. Joanne.

[Joanne Quan, CMO at Mirum Pharmaceuticals]

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 Volixibat, we completed enrollment in the phase 2b VISTAS 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 VISTAS 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.

[Joanne Quan, CMO at Mirum Pharmaceuticals]

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 Volixibat 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 the role for IBAT 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 confirmatory stage.

[Joanne Quan, CMO at Mirum Pharmaceuticals]

Additional analyses from the VANTAGE interim will be presented at AASLD, which highlights the decreases in fatigue and improvement in sleep in Volixibat patients, as well as showing a decrease in IL-31 in treated patients. Our EXPAND study, evaluating LIVMARLI in 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 2 study of MRM-3379, 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 MRM-3379 reversed the disease phenotype across multiple behavioral assessments and increases our confidence in the importance of this pathway in Fragile X.

[Joanne Quan, CMO at Mirum Pharmaceuticals]

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.

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals]

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.

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals]

Our cash operating margins continued to improve, and we delivered GAAP 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 GAAP 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.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

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, cash flow positive, leading rare disease company. Thanks again to the Mirum team and to the patients and families who inspire our work every day. With that, operator, please open the call for questions.

[Operator]

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 Fye with J.P. Morgan. Please go ahead. Your line is open.

[Operator]

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

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Hey, Abdullah. Thanks for the question. On key drivers in the 2026, I mean, we see a lot of basically what we have today rolling forward into next year. We expect that we'll 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 the guidance this year for Q4, maybe ask Peter to speak to what we see from kind of the quarter-to-quarter dynamics. Yeah, and. Oh,

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Thanks, Chris. Yeah, I appreciate the question, Abdullah. The main dynamic is tried to highlight in our prepared remarks. We see growth for LIVMARLI U.S. We see the bioacquired portfolio continuing to do what it does.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

It's really the LIVMARLI 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 Takeda 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 that's really the dynamic that's in the LIVMARLI international line.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Thank you.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Thanks for the question.

[Operator]

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

[Joshua Schimmer, Managing Director at Cantor Fitzgerald & Co.]

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 LIVMARLI and what % of sales are for that versus the liquid? And then for Volixibat, 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 PSC? Thank you.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Thanks for the questions, Josh. Yeah, so in terms of the solid tablet, just launched in the U.S. in mid-June, so this is really our first full quarter with it. And 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 a 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, Volixibat pricing, obviously haven't made a final decision there. I've monitored those dynamics that you were talking about. We've kind of base case thinking.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

You can look at the other PPARs and the other products that are kind of approved in PBC at the 130-150, but we're still analyzing. I think it's kind of too early to say what the right pricing strategy is for Volixibat.

[Joshua Schimmer, Managing Director at Cantor Fitzgerald & Co.]

Thank you.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

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

[Gavin Clark-Gardner, Managing Director and Biotechnology Equity Research at Evercore]

Hey, guys. Thanks for taking the question. Just had one. What's your expectation for Paragraph IV filers? Maybe just helpful to lay out your confidence in your whole IP portfolio, especially around the method patents and including Volixibat. Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

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 life cycle for LIVMARLI, and really quite confident in our overall IP position. In particular, you mentioned the method patents that are specific to dosing of LIVMARLI and these indications. We've seen this has been really the key fundamental observation that's made all of Mirum possible, 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 to date.

[Gavin Clark-Gardner, Managing Director and Biotechnology Equity Research at Evercore]

Great. Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the question.

[Operator]

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

[Operator]

Hey, thanks for taking our question. This is Mark on for James. So. Recently on earnings, Shionogi seemed 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 curious your thoughts on the safety risks there. And if you can see sort of anything in the blinded data on GI side effects and whether those look any materially different than, say, PBC or Alagille. Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah, thanks for the question, Mark.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Can't really speculate too much on Shionogi's update and what's going on underneath that, but turn it to Joanne to talk a little bit about our endpoint strategy and what our approach on our programs.

[Joanne Quan, CMO at Mirum Pharmaceuticals]

Yeah, thanks for the question. We feel that we're in a good spot at this point. 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 supports efficacy in our moving forward. And then we've also had very good engagement with the community, with patients and with physicians. We also had a very successful and engaging pre-IND 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 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. A lot of interest from the community, and we're looking forward to conducting the study and seeing what we see.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

On the PSC safety standpoint, I actually looked to Joanne for that.

[Joanne Quan, CMO at Mirum Pharmaceuticals]

Yeah, and so with regards to that, for the PSC study, 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 moving forward with the way the protocol was initially designed. So no issues have emerged there.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

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

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

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

[Joseph Thome, TD Cowen]

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.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks, Joseph, for the question. I think overall, we've not planned to present or analyze some of the baseline criteria at this point. What we know from and what we can say kind of more generally from the enrollment criteria and what was in the interim is the patients are selected for itch. So we 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.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

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.

[Joseph Thome, TD Cowen]

Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

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

[Ryan Deschner, Vice President and Equity Research- Biotech at Raymond James]

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 cholestatic pruritus patients? And then I have a follow-up. Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks, Ryan, for the question. I mean, the 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 Alagille 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.

[Ryan Deschner, Vice President and Equity Research- Biotech at Raymond James]

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 Alagille and PFIC? Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

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

[Ryan Deschner, Vice President and Equity Research- Biotech at Raymond James]

Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Good questions.

[Operator]

The next question will be from the line of Manny Murray with Leerink Partners. Please go ahead. Your line is open.

[Ryan Deschner, Vice President and Equity Research- Biotech at Raymond James]

Hey, guys. We have Ryan on from Cantor. Thanks for taking our questions. Congrats on the quarter. Can you just talk a little bit about the pace of new PFIC adds 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 maybe more broadly, as you guys start to see 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.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. Thanks for the question. Actually, I'll turn it over to Peter to jump into those.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Yeah. In terms of the pace of PFIC adds, it continues to be healthy. It continues to come from a broad 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. Some of the major academic medical centers are on board with that, and 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 obesity. Yeah. Do you want to? Sure.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

I mean, the thing we'd say on BD is 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, look for good value creation opportunity. So that will continue to be the standard we take going forward. And plenty in the company to grow and build and optimistic about adding more down the road.

[Ryan Deschner, Vice President and Equity Research- Biotech at Raymond James]

Appreciate it. Thanks again.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

The next question will be from the line of Mike Olds with Morgan Stanley. Please go ahead. Your line is open.

[Operator]

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

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. Rohit, 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 Volixibat 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 Volixibat study and what we expect ultimately of Volixibat marketed treatment. And then with respect to Linerixibat 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.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

The placebo-adjusted difference that we saw on itch in that dataset is striking, led to breakthrough designation. And 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 Volixibat given that highly active dose level.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

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

[Jon Wolleben, Managing Director at Citizens Financial Group, Inc.]

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

[Chris Peetz, CEO at Mirum Pharmaceuticals]

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.

[Joanne Quan, CMO at Mirum Pharmaceuticals]

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 pruritus 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 pruritus, and we're pretty confident in terms of the ability of Volixibat to affect that in a positive way for patients.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Jon Wolleben, Managing Director at Citizens Financial Group, Inc.]

Thanks, guys.

[Operator]

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

[Chris Peetz, CEO at Mirum Pharmaceuticals]

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

[Operator]

This will conclude the Mirum 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.

Participants

Executives

• Andrew McKibben, SVP of Strategic Finance and Investor Relations
• Peter Radovich, President and COO
• Chris Peetz, CEO
• Joanne Quan, CMO
• Eric Bjerkholt, CFO

Analysts

• Analyst 2 at STIFEL FINANCIAL CORP.
• Analyst 3 at Morgan Stanley
• Jon Wolleben, Managing Director at Citizens Financial Group, Inc.
• Joshua Schimmer, Managing Director at Cantor Fitzgerald & Co.
• Ryan Deschner, Vice President and Equity Research- Biotech at Raymond James
• Analyst 1 at JPMorgan Chase & Co.
• Joseph Thome, TD Cowen
• Gavin Clark-Gardner, Managing Director and Biotechnology Equity Research at Evercore