Mirum Pharmaceuticals Q2 2025 Earnings Report

Mirum Pharmaceuticals EPS Results

• Actual EPS: -$0.12
• Consensus EPS: -$0.31
• Beat/Miss: Beat by +$0.19
• One Year Ago EPS: -$0.52

Mirum Pharmaceuticals Revenue Results

• Actual Revenue: $127.79 million
• Expected Revenue: $107.91 million
• Beat/Miss: Beat by +$19.87 million
• YoY Revenue Growth: +64.10%

Mirum Pharmaceuticals Announcement Details

• Quarter: Q2 2025
• Date: 8/6/2025
• Time: After Market Closes
• Conference Call Date: Wednesday, August 6, 2025
• Conference Call Time: 4:30PM ET

Mirum Pharmaceuticals (NASDAQ:MIRM), a biopharmaceutical company, focuses on the development and commercialization of novel therapies for debilitating rare and orphan diseases. Its lead product candidate is LIVMARLI (maralixibat), an orally administered and minimally absorbed ileal bile acid transporter (IBAT) inhibitor that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the United States and internationally. The company is also involved in the commercialization of Cholbam, a cholic acid capsule, which is approved as treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder and Smith-Lemli-Opitz syndrome; and Chenodal, a tablet, which is approved for the treatment of radiolucent stones in the gallbladder, and under Phase 3 development for the treatment cerebrotendinous xanthomatosis. In addition, it develops Volixibat, an oral and minimally absorbed agent designed to inhibit IBAT, currently under Phase 2b clinical trial for the treatment of adult patients with cholestatic liver diseases. The company was incorporated in 2018 and is headquartered in Foster City, California.

Mirum Pharmaceuticals Q2 2025 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: Total revenues reached $128 million in Q2 (up 64% YoY), and 2025 guidance was raised to $490–$510 million, signaling strong commercial momentum.
• Positive Sentiment: Pivotal pipeline milestones are on track, including completion of VISTA Phase 2b PSC enrollment this quarter (data expected Q2 2026) and initiation of the Phase II Fragile X study by year-end.
• Positive Sentiment: LIVMARLI uptake in PFIC has exceeded expectations due to expanding genetic testing and disease awareness, underpinning its potential as a >$1 billion franchise.
• Positive Sentiment: Cash flow positive performance continued, with operating cash flow positive in Q2 and a strong cash, equivalents and investments balance of $322 million as of June 30.
• Neutral Sentiment: International sales showed quarter-to-quarter variability driven by large periodic Takeda orders in Japan amid ongoing reimbursement expansions.

Presentation

[Operator]

Thank you for your patience everyone. The Mehran Pharmaceuticals reports second quarter twenty twenty five financial results will begin in two minutes' time. Hello, everyone, and welcome to the Miron Pharmaceuticals Report Second Quarter twenty twenty five Financial Results and Provide Business Update. My name is Carla, and I will be coordinating your call today. I would now like to hand over to your host, Andrew McKeown, SVP of Strategic Finance and Investor Relations to begin. Please go ahead when you're ready.

[Andrew McKibben, SVP - Strategic Finance & IR at Mirum Pharmaceuticals Inc]

Thanks, Carla, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceuticals' second quarter twenty twenty five conference call. I'm joined today by our CEO, Chris Pete our President and Chief Operating Officer, Peter Radovich our Chief Medical Officer, Jo Anne Kwan and Eric Bjerkholt, our Chief Financial Officer. Earlier today, Mirum issued a news release announcing the company's results for the 2025. Copies of this news release and SEC filings can be found in the Investors section of our website.

[Andrew McKibben, SVP - Strategic Finance & IR at Mirum Pharmaceuticals Inc]

Before we start, I'd like to remind you that during the course of this conference call, we'll be making certain forward looking statements based on management's current expectations, including statements regarding Merame'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?

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks, Andrew, and good afternoon, everyone. 2025 is shaping up to be another outstanding year for Merit, the second quarter that underscores the momentum behind both our commercial medicines and our pipeline. Merit was founded in 2018 with a vision of bringing life changing medicines to patients with rare disease around the world. Today, we have three approved medicines with reimbursed patients in over 30 countries, a pipeline that is rapidly advancing opportunities in still larger settings, highlighted by three potentially pivotal studies reading out over the next twenty four months.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Our strategy is rooted in commercial execution, scientific innovation and financial discipline, and we're proud of the continued progress on all three fronts that we will cover today. On that note, on second quarter results, we're excited to share another strong update for Miriam with total revenues reaching $128,000,000 or 64% growth over the second quarter last year. Litmarley is a key driver of these results and is continuing to bring substantial benefit to patients and to build a differentiated position with physicians. As the top line suggests, we are reaching more patients than we initially anticipated. Given the growth we are seeing across our medicines, we are raising our full year guidance for 2025 to be $490,000,000 to $510,000,000 positioning us for another year of close to 50% top line growth.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Turning to the pipeline. The progress we are making is setting the stage for an exciting 2026, where we have a clear path to three late stage milestones. In particular, the VISTA's Phase 2b study in primary sclerosing cholangitis, PSC, is on track to complete enrollment this quarter, with top line data expected in the second quarter next year. Now this program passed its interim analysis last year, and a consistent precedent data for IDAT inhibition across other cholestatic settings gives us confidence in the potential of elixibat in PSC. Exciting steps lie ahead to potentially bring this much needed treatment to PSC patients.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

We are also seeing excellent momentum in our VANTAGE PVC and EXPAND studies and are looking forward to starting our Phase II study of MRN-three thousand three and seventy nine in Fragile X Syndrome now that we have FDA feedback on the program, which Joanne will tell you more about shortly. I would also like to say thank you to the Meron team, whose dedication to bringing high impact medicines to patients has made all this progress possible. I'm proud of how this group has come together to create a high growth, cash flow positive, rare disease leader and exciting pipeline. And with that, I'll hand the call over to Peter to walk through the commercial performance in more detail.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Peter? Thanks, Chris. Q2 was another strong quarter for Merum, with total net product sales approximately $128,000,000 driven by continued momentum across Lidmarley in both The United States and international markets, as well as solid performance from our bile acid portfolio. In The U. S, Lidmarly demand remains strong with in Alagille syndrome and PBIT with approximately $57,000,000 in net product sales for the quarter.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Notably, we are seeing more PBIT patients than we had originally anticipated, which we believe is due in part to increased disease awareness and broader use of genetic testing, leading to more PFIC diagnoses in patients with later onset cholestasis. While PFIC is often associated with clinical presentation in infants, we're increasingly seeing PFIC patients presenting later in childhood, adolescence or even adulthood. And expanding recognition of this variability and highlighting the importance of genetic testing across age groups has been a core focus of our launch strategy. We're also seeing real synergy between the approved Alagille syndrome and PFIC indications with providers increasingly viewing Libmarly as a preferred treatment across these settings of pediatric cholestasis. The combination of these factors is translating into a meaningful uptick in volume growth.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Importantly, our recent U. S. Launch of the single tablet per dose formulation in June adds meaningful convenience for patients, though I'll note that Q2 results reflect the performance of our oral solution. Internationally, we are seeing durable LIVMARLI growth across both direct and partner markets with $31,000,000 net product sales. This was driven by expanding reimbursement and growing demand as well as strong performance in our partner markets.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

In Q2, our partner Takeda secured reimbursement in Japan and launched Libmarly in June with promising demand observed in the initial days of commercialization. Under our license agreement with Takeda, we received large periodic orders for Libmarli, creating quarter to quarter variation in international product sales. We also saw strong performance from our bile acid portfolio, with Cetexly and Cobalt contributing approximately $40,000,000 in revenue. These medicines continue to benefit from steady demand and increased engagement following the Citexly approval earlier this year. Given the momentum across our medicines, we are raising full year revenue guidance to $490,000,000 to $510,000,000 driven largely by Libmarly's strong performance, particularly growth in our international business, steady increase in Alagille demand and our PFIT launch in The U.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

S. It's an exciting time for realizing Libmarly's potential. Looking long term, with the current trends in Alagille syndrome and PFIC and the label expansion opportunity in ultrarare cholestasis we aim to unlock through the EXPAND study, we believe LIVMARLI ultimately has the potential to be a $1,000,000,000 plus revenue brand. We're excited about continuing to execute to realize that potential and prepare for potential launches ahead of our clinical pipeline. And for an update on the pipeline, I'll turn it over to Joanne.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

Thanks, Peter. I'm pleased to share updates on the continued progress of our clinical pipeline, where we're seeing strong interest and engagement across all studies. Starting with velexibat, we're very encouraged by the momentum in our VISTA study for patients with pruritus due to PSC. The last patients are in screening now, keeping us on track to complete enrollment this quarter and on track for expected top line data in the 2026. With regards to PBC, the VANTAGE study is proceeding nicely and we expect to complete enrollment next year.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

The EXPAND study evaluating LIVMARLI in additional settings of cholestatic pruritus is also progressing well, and we expect to complete enrollment in 2026. Finally, I'm excited to share more on MRM-three thousand three and seventy nine, our brain penetrant PDE4D inhibitor for fragile X syndrome. We had the opportunity to discuss the program and endpoints with the FDA in a pre IND meeting earlier this year and our IND has recently cleared. We are on track to initiate the Phase II study by the end of the year. Our study will enroll approximately 52 male participants aged 16 to 45 with Fragile X syndrome.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

We will enroll males who are confirmed genetically what is called full mutation as these patients are known to be most severely affected from a cognitive standpoint and therefore have the greatest unmet need for new therapy. This is a randomized, double blind, placebo controlled study evaluating three active doses in order to identify the optimal dose. An additional open label cohort will include approximately eight younger patients, males aged 13 to less than 16 at the lowest dose to evaluate PK and allow us to move into younger populations in subsequent trials. The primary endpoint is safety and tolerability and the key secondary efficacy endpoint is a change from baseline at week twelve on the NIH Toolbox crystallized cognition composite, a well recognized cognitive measure also used by other programs in this space. Based on FDA feedback, we do anticipate that this ultimately will be the primary endpoint of Phase III.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

We're excited by the pace and engagement across our pipeline and look forward to sharing further updates in the coming quarters. I'll now hand the call over to Eric to discuss our financial results for the quarter. Eric?

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals Inc]

Thanks, John, and good afternoon, everyone. We delivered another early quarter of financial performance highlighted by total net product revenue of $128,000,000 representing a 64% increase over the prior year and reflecting growth across all our commercial medicine. Total operating expense for the quarter ended June 30 was $133,000,000 which includes R and D expense of $46,000,000 SG and A expense of 63,000,000 and cost of sales of $23,000,000 Expenses for the quarter included non cash stock based compensation expense of $18,000,000 and intangible amortization and other non cash items of 6,000,000 The intangible amortization and other non cash items expense are largely reflected in our cost of sales. We were operating cash flow positive for the quarter and expect to continue to be cash flow positive for the full year. Our cash operating margins continue to improve. For example, our cash contribution margin from our commercial business exceeded 50% in the second quarter. Cash, cash equivalents and investments were $322,000,000 at June 30, a $29,000,000 increase from the end of last 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, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks, Eric. I want to take a moment again to acknowledge the incredible efforts of the Merit team, The progress we've made so far this year, both commercially and across the pipeline, reflects our continued commitment to delivering life changing medicines for patients with rare disease. We are operating from a position of strength, and the opportunities ahead make this an exciting time for Merum. We have a clear strategy, the right team in place, and a growing impact on the lives of patients and families around the world. With that, operator, please open the call for questions.

[Operator]

Sure. We will begin now with a question and answer session. And our first question comes from Gavin Clark Gartner with Evercore.

[Gavin Clark-Gartner, MD - Biotechnology Equity Research at Evercore ISI]

Hey, guys. Congrats on another great quarter. First, I just wanted to ask on LIVMARLI. What are you seeing for overall therapy persistence rates? And has that changed at all over the last couple of years?

[Gavin Clark-Gartner, MD - Biotechnology Equity Research at Evercore ISI]

And then on the pipeline, for the ongoing VISTAs PSC trial, is there anything you're seeing on a blinded basis that gives you increased confidence? Maybe blended pruritus variability tracking within expectations, baseline characteristics continuing to come in as expected? Or anything else you can give us there would be really helpful. Thanks.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Yes. Thanks, Gavin, for the question. I'll let Peter speak to the persistence question and then let Joanne comment a little bit about how Vista's conduct is going.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Yeah. Thanks for the question, Gavin. In terms of persistence, our information is most stable from the Alagille indication where we've got patients about several several years or some approaching a decade. And, you know, if you think about persistence, probably seventy to seventy five percent are on after one year. So that's the kind of attrition in year one. And then in subsequent years, the attrition is much less than that.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

So that's probably the way to think about it in LGO. And then PPIC is just probably a bit too early to comment.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

Hey, Gavin. This is Joanne. Thanks for the question. With regards to your questions about VISTAs, you know, we're feeling pretty confident, and part of it is that the standard deviation that we powered the study on, was pretty conservative. So, you know, our best, estimate is that the standard deviation should come in less than that.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

So given that we powered the study, assuming a treatment difference of 1.75 with a standard deviation of three, probably it's closer to two. So I think that gives us, added confidence. And I can also share with you that the baseline characteristics in general reflect the PSC population. So I think I think these are all points that give us, you know, some good confidence kind of proceeding forward as we're getting the last patients in the screening.

[Gavin Clark-Gartner, MD - Biotechnology Equity Research at Evercore ISI]

Great. Thank you.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

Thank you. The next question comes from Jessica Farr with JPMorgan.

[Analyst]

This is Abdul on for Jess. Congrats on the quarter. Can you provide details on the expected revenue distribution between Live Marley and the bile acid business for the remainder of the year? Thank you.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks for the question. We're not breaking down guidance by specific products. But one thing I would say is that some of the trends that we've seen year to date, we expect those to kind of, in general, continue moving forward. So that's kind of the best color I can give on how it breaks down in that $490,000,000 to $510,000,000 range.

[Analyst]

Thank you.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

The next question comes from Ryan Dashner with Raymond James.

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

Congrats on the quarter. I wanted to ask, what main drivers are you attributing to the growth that we're seeing in Litmarley sales? And then also how meaningful an impact on script volumes are you seeing specifically due to the availability of the tablet format in Litmarley? Thank you.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks, Ryan, for the question.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

I'll give a first comment and let Peter add on to it. I think one of the several dynamics going on here. I think one, in general, that we've noticed is really just as PFIC has been added in The US, building awareness of availability of genetic testing and the concept of later onset PFIC diagnoses, which, to be honest, when we, you know, got the label expansion and we're initially starting out in PFIC, we thought it was pretty minimal. What we're finding is that it is it just it's more there are more of them out there than we originally expected. So that's that's one of the the the drivers that we've actually deployed against over the past twelve months.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Yeah. I think that's a that's that that that is one of the main drivers, really growing the pie in PFIC, if you will, the the total, you know, market for the class in that setting. Also happy with the continued growth in Alagille syndrome, and certainly, the international business has performed well. In terms of your question about the tablet, that was introduced in the month of June. So obviously, part of our comments there, the outside is not didn't have an effect this quarter, but certainly encouraged.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

I've had a lot of positive feedback from, patients and and providers who have chosen chosen to go to the the tablet since then.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks for the question, Ryan.

[Operator]

Thank you. So the next question comes from Brian Skorney with Baird.

[Luke Herrmann, Research Analyst at Baird]

Hi. This is Luke on for Brian. Thanks for taking the question. Two on Lodmarley. Can you discuss any inventory impacts in the second quarter?

[Luke Herrmann, Research Analyst at Baird]

And also, could you provide a little more insight on the Takeda order cadence? Do you expect it to be more of a seasonal trend? Or would it be more regular than that?

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Yeah. Thanks for the question, Luke. As far as inventory, it's really only relevant in so far as Japan and Takeda. No no inventory in the business in The US or or Europe anywhere else. With Takeda, it is kind of large periodic orders that happen.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

And from time know, from you know, we expect there'll probably be another one this year, but we don't have perfect line of sight into it. There's variable consideration placed on it when the order comes, so that's why, you know, in subsequent quarters, the estimate can can change. But that's kind of the best color we'd give to you. We'd expect quarter to quarter variability there.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Yeah. Specifically, in in q two, it's 11,000,000, was the the number out of the q two number.

[Luke Herrmann, Research Analyst at Baird]

Great. Thank you.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

The next question comes from Mike Oates with Morgan Stanley.

[Michael Ulz, Executive Director - Biotechnology Equity Research at Morgan Stanley]

Good afternoon. Thanks for taking the question and congratulations on the quarter as well. Maybe just a question on Fragile X. Sounds like you made some nice progress interacting with the FDA on the trial design. Just curious if there's anything else you need feedback on from the FDA or any next steps before you start that study in the fourth quarter? Thanks.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

Yeah. Thanks for the question. So, you know, we're actually good to go. We have a the the clearance from the IND, so we have a study may proceed letter. You know, we've engaged a lot with the patient community and also the physician community to make sure they we incorporate their comments into the design of this study.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

So we feel pretty good in terms of where we are and certainly on track to enroll the first patient by the end of the year.

[Michael Ulz, Executive Director - Biotechnology Equity Research at Morgan Stanley]

Great. Thank you.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

The next question comes from Fulhar with Leerink Partners.

[Ryan Mcelroy, Equity Research Associate at Leerink Partners]

Hey guys. You have Ryan on for Mani. Congrats on the quarter. Just curious how well penetrated do you guys think you are in the Alagille and PFIC markets? Kind of looking at your commentary that LIVMARLI can be a $1,000,000,000 product.

[Ryan Mcelroy, Equity Research Associate at Leerink Partners]

Wondering how you see that broken out by Alagille and PFIC. And then just one on the pipeline. I know Fragile X design is pretty ironed out, but are there any specific elements you're particularly interested in from Shia Nogi's update later this year? Thank you.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Yes. Thanks for the question. I can touch on the last point first and then pass over to Peter to talk about some of the sizing for the various components for Lipmarley. In terms of the Phase two precedent data from the Shinobi program and the upcoming Phase three, a couple of thoughts that we have on it. First, the Phase two is great proof of concepts out there and kind of what got us excited about 03/1979 and the ability to have potentially wider therapeutic index, get more of the drug into the CNS.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

And in terms of kind of what we are looking for out of the Phase III, if that differentiated profile that we have, think, kind of makes us really interested to run our proof of concept kind of regardless of the outcome. Expect we're not going to learn a lot from any top line release, but we'll be looking closely and see if there's anything to incorporate into the future studies in the program.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

And in terms of your question, Ryan, about penetration and opportunity, if you think The U. S, in Alagille syndrome, we think we're approaching fifty percent penetration. But, you know, still every quarter, q two, you know, adding adding patients and and both infants as well as kind of older older patients who are more prevalent in in that prevalent addressable pool. So still see the potential to keep growing Alagille further and further in subsequent quarters as we have and grow that business. PPIC has been really interesting.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

As Chris mentioned in his comments, we're growing the pie right now as we speak. I think traditionally, the field and others in the area thought about, know, PFIC is really an infant onset disease. And if it's not that scenario, then it's not PFIC. And I think what we've shown through our education and genetic testing efforts is that there's a lot more there than was previously thought. So we have kind of less visibility to kinda how big that could be.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

I think, you know, historically, we thought about PFIC as being about a third of Alagille. Quite frankly, that's probably probably an underestimate at this point. So excited to define that as we move forward.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

And then I'd add on, the one other component of when you think about the total LeMarley potential, the $1,000,000,000 plus number that we're looking at, the EXPAND study also is a big contributor there, where the EXPAND patient population is really at least the size of PFIC and it's kind of the conservative view of it. And all of these dynamics kind of continuing to build over time the patients on therapy and ultimately the size of the brand.

[Ryan Mcelroy, Equity Research Associate at Leerink Partners]

Awesome. Appreciate the color.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Yes. Thanks for the question.

[Operator]

The next question comes from Josh Schumer with Cantor Fitzgerald.

[Josh Schimmer, Managing Director at Cantor Fitzgerald]

Thanks very much. One from me on the EXPAND study. I was wondering if you could share if any of those eligible patients are already on this MALI through compassionate use or other exceptions?

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Yeah. Thanks for the question. I'll let Joanne speak a little bit to the background of how we thought and came upon designing the Xtamp study. So it really kinda speaks to that that question.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

Yeah. Thanks for the question. So, you know, really, the the EXPAND study came around because we had a lot of requests for compassionate use in these patients with cholestatic pruritus, you know, from from these other settings. And so, you know, we're taking patients who have not previously been treated in order to assess their response in the setting. So, you know, so far, we're encouraged by the engagement we've heard from sites and also, you know, just the interest from patient populations as well.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

The next question comes from David Lebowitz with Citi.

[David Lebowitz, Senior Research Analyst - Biotechnology at Citi]

Thank you for taking my questions. I know a few years ago, $500,000,000 was due to kind of the peak for LIVMIARLI. This is obviously a dramatic shift. How much of it comes from potential from the new indications versus PFIC and ALGS? New potential indications.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Yes.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Thanks for the question. The $500,000,000 I think you're referencing is we used to kind of give a size for the indication of Alagille in The U. S, just kind of the market sizing. So this is the time with these indications that we put out guidance on where we think LipMarley can ultimately get to. So slightly different lens than kind of indication sizing.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

And really, a lot of the confidence in doing that is just how much we're seeing come together across all these different settings. So the three indications, know, Alagille, PFIC, and EXPAND in The US, quite sizable. What we're seeing on the international side, also kind of running ahead of where our expectations were. So a lot of this is kind of a change in what we've seen so far this year in terms of uptake and and things that we're doing directly as well as distributors and partners and the success that they're seeing.

[David Lebowitz, Senior Research Analyst - Biotechnology at Citi]

Got it. Thanks for taking my question.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Yeah. Thanks for the question.

[Operator]

The next question comes from Jonathan Bullevin with Citizens.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Hey, thanks for taking the question.

[Jon Wolleben, Managing Director at Citizen JMP]

Two for me. One on PSC, you guys estimate that there's about sixty five percent of the population with active pruritus. I'm wondering if there's any evidence that that's due specifically due to excess bile acids or if there could be any other drivers of pruritus in that group? And then second one, just wondering on CTX, if you guys have seen any inflection now that you can promote and what you expect for that long term? Thanks.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Yeah. Thanks for the question. I'll pass this over to Peter and Joanne to speak to the two components and let Peter lead

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

off with CTX. Yeah. We're, you know, we're certainly, you know, excited about the FDA approval now, first full quarter, since that year. You know, a lot of our efforts are on patient finding, across different, specialties where, you know, patients present. Not not expecting you know, I think it's a gradual steady build in CTS.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Patient finding is laborious, but excited about the potential there to to grow this longer term. Joanne?

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

Yeah. Yeah. Thanks for the question. Regards to PSC, you know, we do think that, bile acids do have a role here.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

However, the pathophysiology is a bit different than some of the other diseases like allergy and the PFIC that we studied in the past. You know, in reality, what we're treating is cholestasis, and that and therefore, you know, there's an intrahepatic component to it that we don't measure. What we think is that the what we're measuring in serum bile acids is really kind of spillover. And so we think that the serum bile acid level is probably less important when we're thinking about a disease like PSC. Probably more important when you're thinking of, you know, allergy and PFIC, for instance.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

So I think, you know, slightly different disease pathophysiology, but still kind of the important central feature here is cholestasis.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Interesting. Okay. Alright. Thank you.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

So the next question comes from Swayampakula with H. C. Wainwright.

[Swayampakula Ramakanth, Managing Director & Senior Equity Analyst at H.C. Wainwright & Co.]

Thank you. Thanks for taking my questions. I have I have two of them. The first one being on Limarley. So, you know, you made some remarks regarding how Takeda is managing the the Japanese part of the the collaboration.

[Swayampakula Ramakanth, Managing Director & Senior Equity Analyst at H.C. Wainwright & Co.]

Any remarks on on, how how you're going about in Europe? And, also, in within Europe, what sort of efforts are you making to increase your penetration? So that's question number one. And the question number two is on on on the based on the EXPAND study, what sort of, additional population patient population can you bring to LiveMarly? Thanks.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

It's great. Thanks for the question. And maybe I can make a couple comments on kind of end market, Marly, and then have Peter speak to the opportunity and and expand. And, you know, so commentary on Europe and kind of where we're at, what I'd say is, you know, European performance to date is largely Alagille syndrome. So that kind of what's to come and and most exciting in some of our direct European markets is now bringing forward the PFIC indication and adding that in.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

It's it's right now kind of coming through. It's the reimbursement steps for adding that indication. So that's what's kind of on the horizon in Europe. And maybe speak to expand?

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Yeah. And in terms of expanding, you know, a lot of lot of different ultra rare patient populations individually when you look at the under eye underlying kind of etiology of cause of the cold saccharides that that could that could pull in. Certainly, biliary atresia patients with cold saccharitis will be a portion of that, but there are also several others. And we hear about these, as Joanne mentioned earlier, through compassionate access requests from sites who have these patients in their clinic, they don't have anything to offer them. So excited about the potential to study them and potentially have an option.

[Swayampakula Ramakanth, Managing Director & Senior Equity Analyst at H.C. Wainwright & Co.]

Thank you. Thanks for taking my questions.

[Peter Radovich, COO & President at Mirum Pharmaceuticals Inc]

Thank you.

[Operator]

And that was the final question. Well, that does conclude the Q and A portion of today's call. So we'll hand back over to Chris Pitts for any final comments.

[Chris Peetz, Co-Founder & CEO at Mirum Pharmaceuticals Inc]

Great. Thanks again for joining us today and for your continued support. We look forward to updating you in the quarter's end. Have a great afternoon.

[Operator]

This concludes today's call. Thank you, everyone, for joining. You may not disconnect. Have a great day.

Participants

Executives

• Andrew McKibben, SVP - Strategic Finance & IR
• Chris Peetz, Co-Founder & CEO
• Peter Radovich, COO & President
• Joanne Quan, Chief Medical Officer
• Eric Bjerkholt, CFO

Analysts

• Gavin Clark-Gartner, MD - Biotechnology Equity Research at Evercore ISI
• Analyst
• Ryan Deschner, Vice President - Equity Research at Raymond James Financial
• Luke Herrmann, Research Analyst at Baird
• Michael Ulz, Executive Director - Biotechnology Equity Research at Morgan Stanley
• Ryan Mcelroy, Equity Research Associate at Leerink Partners
• Josh Schimmer, Managing Director at Cantor Fitzgerald
• David Lebowitz, Senior Research Analyst - Biotechnology at Citi
• Jon Wolleben, Managing Director at Citizen JMP
• Swayampakula Ramakanth, Managing Director & Senior Equity Analyst at H.C. Wainwright & Co.