Mirum Pharmaceuticals Q2 2025 Earnings Call Transcript

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Provided by Quartr
August 6, 2025

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.
AI Generated. May Contain Errors.
Earnings Conference Call
Mirum Pharmaceuticals Q2 2025
00:00 / 00:00

There are 12 speakers on the call.

Speaker 10

Thank you for your patience, everyone. The Mirum Pharmaceuticals reports second quarter 2025 financial results will begin in two minutes' time. In the meantime, you can register questions at the question star, followed by one on your telephone key. Hello, everyone, and welcome to the Mirum Pharmaceuticals reports second quarter 2025 financial results and provide business update. My name is Carla, and I will be coordinating your call today. During the presentation, you can register to ask questions by pressing star, followed by one on your telephone keypad. If you change your mind, please press star, followed by two. I would now like to hand you over to your host, Andrew McKibben, SVP of Strategic Finance and Investor Relations, to begin. Please go ahead when you're ready.

Speaker 4

Thanks, Carla, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' second 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 Kwan, and Eric Bjerkel, our Chief Financial Officer. Earlier today, Mirum issued a news release announcing the company's results for the second quarter of 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'll 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.

Speaker 4

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?

Speaker 3

Thanks, Andrew, and good afternoon, everyone. 2025 is shaping up to be another outstanding year for Mirum Pharmaceuticals. The second quarter underscores the momentum behind both our commercial medicines and our pipeline. Mirum Pharmaceuticals was founded in 2018 with the 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, and a pipeline that is rapidly advancing opportunities in still larger setups, highlighted by three potentially pivotal studies reading out over the next 24 months. 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.

Speaker 3

On that note, on second quarter results, we are excited to share another strong update for Mirum Pharmaceuticals, with total revenues reaching $128 million, or 64% growth over the second quarter last year. LIVMARLI 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 to $510 million, positioning us for another year of close to 50% top-line growth. 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.

Speaker 3

In particular, the VISTAS Phase IIb 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. This program passed its interim analysis last year, and the consistent precedent data for IVAB inhibition across other cholestatic settings gives us confidence in the potential of volixibat in PSC. Exciting steps lie ahead to potentially bring this much-needed treatment to PSC patients. We are also seeing excellent momentum in our VANTAGE PBC and EXPAND study. We are looking forward to starting our Phase II study of MRM-3379 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 Mirum Pharmaceuticals team, whose dedication to bringing high-impact medicines to patients has made all this progress possible.

Speaker 3

I'm proud of how this group has come together to create a high-growth, cash-flow-positive, rare disease leader with an exciting pipeline. With that, I'll hand the call over to Peter to walk through the commercial performance in more detail. Peter?

Speaker 4

Thanks, Chris. Q2 was another strong quarter for Mirum Pharmaceuticals, with total net product sales of approximately $128 million, driven by the continued momentum across LIVMARLI in both the United States and international markets, as well as solid performance from our bile acid portfolio. In the U.S., LIVMARLI demand remains strong in Alagille syndrome and PFIC, with approximately $57 million in net product sales for the quarter. Notably, we are seeing more PFIC 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.

Speaker 4

An 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 LIVMARLI as a preferred treatment across these settings of pediatric cholestasis. The combination of these factors is translating into a meaningful uptick in volume growth. Importantly, our recent U.S. launch of the single-tablet 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 million net product sales. This was driven by expanding reimbursement and growing demand, as well as strong performance in our partner markets.

Speaker 4

In Q2, our partner Takeda secured reimbursement in Japan and launched LIVMARLI in June, with promising demand observed in the initial days of commercialization. Under our license agreement with Takeda, we received large periodic orders for LIVMARLI, creating quarter-to-quarter variation in international product sales. We also saw strong performance from our bile acid portfolio, with CHENODAL and CHOLBAM contributing approximately $40 million in revenue. These medicines continue to benefit from steady demand and increased engagement following the CHENODAL approval earlier this year. Given the momentum across our medicines, we are raising full-year revenue guidance to $490 to $510 million, driven largely by LIVMARLI's strong performance, particularly growth in our international business, steady increase in Alagille syndrome demand, and our PFIC launch in the U.S. It's an exciting time for realizing LIVMARLI's potential.

Speaker 4

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

Speaker 8

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 volixibat, we're very encouraged by the momentum in our VISTAS 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 second quarter of 2026. With regards to PBC, the VANTAGE study is proceeding nicely, and we expect to complete enrollment next year. 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-3379, our brain-penetrant PDE4D inhibitor for Fragile X syndrome.

Speaker 8

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, age 16 to 45, with Fragile X syndrome. 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.

Speaker 8

The primary endpoint is safety and tolerability, and the key secondary efficacy endpoint is a change from baseline at week 12 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, Phase III. We're excited about the pace and engagement across our pipeline and look forward to sharing further updates in the coming quarters. I will now hand the call over to Eric to discuss our financial results for the quarter. Eric?

Speaker 11

Thanks, Joanne, and good afternoon, everyone. Delivered another early quarter of financial performance, highlighted by a total net product revenue of $128 million, representing a 64% increase over the prior year and reflecting growth across all our commercial medicines. Total operating expense for the quarter ended June 30 was $133 million, which includes R&D expense of $46 million, SG&A expense of $63 million, and cost of sales of $23 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. 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.

Speaker 11

For example, our cash contributions margin from our commercial business exceeded 50% in the second quarter. Cash, cash equivalents, and investments were $323 million at June 30, a $29 million 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.

Speaker 3

Thanks, Eric. I want to take a moment again to acknowledge the incredible efforts of the Mirum team. The progress we've made so far this year, both commercially and across the pipeline, reflects our continued commitment to delivering life-changing medicine for patients with rare disease. We are operating from a position of strength, and the opportunities ahead make this an exciting time for Mirum. 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.

Speaker 10

Sure. We will begin now with the question and answer session. If you'd like to ask a question, please press star, followed by one on your telephone keypad. If you change your mind, please press star, followed by two. When preparing to ask your question, please ensure your device is unmuted locally. Our first question comes from Gavin Clark-Gartner with Evercore.

Speaker 10

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

Speaker 3

Yeah, thanks, Gavin, for the question. I'll let Peter speak to the persistence question, and then let Joanne comment a little bit about how VISTAS conduct is going.

Speaker 4

Yeah, thanks for the question, Gavin. In terms of persistence, our information is most stable from the Alagille syndrome indication, where we've got patients about several years or some of them approaching a decade. If you think about persistence, probably 70% to 75% are on after one year. That's the kind of attrition in year one. In subsequent years, the attrition is much less than that. That's probably the way to think about it in Alagille syndrome. PFIC is just probably a bit too early to comment.

Speaker 8

Hey, Gavin. This is Joanne. Thanks for the question. With regards to your questions about VISTAS, we're feeling pretty confident. Part of it is that the standard deviation that we powered the study on was pretty conservative. Our best estimate is that the standard deviation should come in less than that. Given that we powered the study, assuming a treatment difference of 1.75 with a standard deviation of 3, probably is closer to 2. I think that gives us added confidence. I can also share with you that the baseline characteristics in general reflect the PSC population. I think these are all points that give us some good confidence proceeding forward as we're getting the last patients into screening and completing.

Speaker 3

Great. Thank you.

Speaker 10

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

Speaker 10

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

Speaker 3

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

Speaker 3

Thank you.

Speaker 3

Yeah, thanks, everybody.

Speaker 10

Thank you. The next question comes from Ryan Dashner with Raymond James.

Speaker 10

Thanks, and congrats on the quarter. I wanted to ask, what main drivers are you attributing to the growth that we're seeing in LIVMARLI sales? Also, how meaningful of an impact on script volume are you seeing specifically due to the availability of the tablet format in LIVMARLI? Thank you.

Speaker 3

Thanks, Ryan, for the question. I'll give a first comment and let Peter add on to it. I think one of the, and there's several dynamics going on here. I think one, in general, that we've noticed is really just as PFIC has been added in the U.S., building awareness of availability of genetic testing and the concept of later-onset PFIC diagnoses, which, to be honest, when we got the label expansion and were initially starting out in PFIC, we thought was pretty minimal. What we're finding is that it's just, it's more, there are more of them out there than we originally expected. That's one of the drivers that we've actually deployed against over the past 12 months.

Speaker 4

Yeah, I think that is one of the main drivers, really growing the pie in PFIC, if you will, the total market for the class in that setting. Also, happy with the continued growth in Alagille syndrome. Certainly, the international business has performed well. In terms of your question about the tablet, that was introduced in the month of June. Obviously, it's part of our comments there that outside of that, it didn't have an effect this quarter, but certainly encouraged. I've had a lot of positive feedback from patients and providers who have chosen to go to the tablet since then.

Speaker 3

Thanks for the question, Ryan.

Speaker 10

Thank you. The next question comes from Brian Skorney with Baird.

Speaker 10

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

Speaker 4

Yeah, thanks for the question, Luke. As far as inventory, it's really only relevant insofar as Japan and Takeda goes. No inventory in the business in the U.S. or Europe anywhere else. With Takeda, it is kind of large periodic orders that happen. From time to time, we expect there'll probably be another one this year, but we don't have a perfect line of sight into it. There's variable consideration placed on it when the order comes. That's why, in subsequent quarters, the estimate can change. That's kind of the best color we'd give to you if we'd expect quarter-to-quarter variability there.

Speaker 3

Specifically in Q2, it was $11 million out of the Q2 number.

Speaker 4

Great. Thank you.

Speaker 3

Thanks for the question.

Speaker 10

Thank you. The next question comes from Mike Oath with Morgan Stanley.

Speaker 10

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.

Speaker 8

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

Speaker 8

Great. Thank you. Thanks for the question.

Speaker 10

The next question comes from Manny Sohar with Leerink Partners.

Speaker 10

Hey, guys. You have Ryan on for Manny. Congrats on the quarter. Just curious, how well penetrated do you guys think you are in the Alagille syndrome and PFIC markets? Kind of looking at your commentary that LIVMARLI can be a billion-dollar product, wondering how you see that broken out by Alagille syndrome and PFIC. 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 Shionogi's update later this year? Thank you.

Speaker 3

Yeah, thanks for the question. I can touch on the last point first and then pass it over to Peter to talk about some of the sizing for the various components for LIVMARLI. In terms of the Phase II precedent data from the Shionogi program and the upcoming Phase III, a couple of thoughts that we have on it. First, the Phase II is great proof of concept out there and kind of what got us excited about MRM-3379 and the ability to have a potentially wider therapeutic index, get more of the drug into the CNS. In terms of what we are looking for out of the Phase III, that differentiated profile that we have, I think, kind of makes us really interested to run our proof of concept kind of regardless of the outcome.

Speaker 3

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.

Speaker 4

Yeah. In terms of your question, Ryan, about penetration and opportunity, if you think about the U.S., in Alagille syndrome, we think we're approaching 50% penetration, but still every quarter, including Q2, adding patients in both infants as well as kind of older patients who are more prevalent in that prevalent addressable pool. Still see the potential to keep growing Alagille syndrome further and further in subsequent quarters as we have and grow that business. PFIC has been really interesting, 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 PFIC as really an infant-onset disease. If it's not that scenario, then it's not PFIC. What we've shown through our education and genetic testing efforts is that there's a lot more there than was previously thought.

Speaker 4

We have kind of less visibility to how big that could be. Historically, we thought about PFIC as being about a third of Alagille syndrome. Quite frankly, that's probably an underestimate at this point. Excited to find that as things move forward.

Speaker 3

To add on, the one other component of when you think about the total LIVMARLI potential, the billion-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. It's that kind of the conservative view of it. All of these dynamics continue to build over time with patients on therapy and ultimately the size of the brand.

Speaker 3

Awesome. Appreciate the caller.

Speaker 3

Yep. Thanks for the question.

Speaker 10

The next question comes from Josh Schumer with Cantor Fitzgerald.

Speaker 10

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 LIVMARLI through compassionate use or other exceptions.

Speaker 3

Yeah, thanks for the question. I'll let Joanne speak a little bit to the background of how we came upon designing the EXPAND study. It really kind of speaks to that question.

Speaker 8

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

Speaker 10

The next question comes from David Lobowitz with Citi.

Speaker 10

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

Speaker 3

Yeah, thanks for the question. The $500 million I think you're referencing is we used to kind of give a size for the indication of Alagille syndrome in the U.S., just kind of the market sizing. This is kind of the first time with these indications that we've put out guidance on where we think LIVMARLI can ultimately get to. It's a slightly different lens than kind of indication sizing. Really, a lot of the confidence in doing that is just how much we're seeing come together across all these different settings. The three indications, Alagille syndrome, PFIC, and EXPAND in the U.S. are quite sizable. What we're seeing on the international side is also kind of running ahead of where our expectations were.

Speaker 3

A lot of this is kind of a change in what we've seen so far this year in terms of uptake and things that we're doing directly, as well as distributors and partners and the success that they're seeing.

Speaker 3

Got it. Thanks for taking that question.

Speaker 3

Yeah, thanks for the question.

Speaker 10

As a reminder, if you'd like to ask a question, it is star one on your telephone keypad. The next question comes from Jonathan Bullivan with Citizens.

Speaker 3

Hey, thanks for taking the question. Two for me. One on PSC. You guys estimate that there's, you know, about 65% of the population with active pruritus. I'm wondering if there's any evidence that that's specifically due to excess bile acids or if there could be any other drivers of pruritus in that group. The 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.

Speaker 3

Yeah, thanks for the question. I'll pass this over to Peter and Joanne to speak to the two components, and maybe let Peter lead off with the CTX.

Speaker 4

Yeah, we're certainly excited about the FDA approval now. First full quarter since that year. A lot of our efforts are on patient finding across different specialties where patients present. Not expecting, I think it's a gradual, steady build in CTX. Patient finding is laborious, but excited about the potential there to grow in the longer term.

Speaker 8

Yeah, thanks for the question. Regards to PSC, we do think that bile acids do have a role here. However, the pathophysiology is a bit different than some of the other diseases like Alagille syndrome and PFIC that we studied in the past. In reality, what we're treating is cholestasis, and therefore, there's an intrahepatic component to it that we don't measure. What we think is that what we're measuring in serum bile acids is really kind of spillover. 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, Alagille syndrome and PFIC, for instance. I think, you know, slightly different disease pathophysiology, but still kind of the important central feature here is cholestasis.

Speaker 3

Interesting. Okay. All right. Thank you.

Speaker 4

Thanks for the question.

Speaker 10

The next question comes from Swayam Pakula with H.C. Wainwright.

Speaker 10

Thank you, Gavin. Thanks for taking the question. I have two of them. The first one being on LIVMARLI. You made some remarks regarding how Takeda is managing the Japanese part of the collaboration. Any remarks on how you're going about in Europe? Also, within Europe, what sort of efforts are you making to increase your penetration? That's question number one. The question number two is based on the EXPAND study, what sort of additional patient population can you bring to LIVMARLI? Thanks.

Speaker 4

That's great. Thanks for the question. Maybe I can make a couple of comments on kind of in-market LIVMARLI and then as Peter speaks of the opportunity in EXPAND. Commentary on Europe and kind of where we're at, what I'd say is, European performance to date is largely Alagille syndrome. What’s to come and most exciting is some of our direct European markets is now bringing forward the PFIC indication and adding that in. It's right now kind of coming through. It's the reimbursement steps for adding that indication. That's what's kind of on the horizon in Europe. Maybe speak to EXPAND.

Speaker 3

Yeah, and in terms of EXPAND, a lot of different ultra-rare patient populations individually, when you look at the underlying kind of etiology or cause of the cholestatic pruritus, that could pull in. Certainly, biliary atresia patients with cholestatic pruritus will be a portion of that. There are also several others. We hear about these, as Joanne mentioned earlier, through compassionate access requests and from sites who have these patients in their clinic, and they don't have anything to offer them. Excited about the potential to study them and potentially have an option.

Speaker 3

Thank you. Thanks for taking my questions.

Speaker 3

Thank you.

Speaker 10

That was the final question. This does conclude the Q&A portion of today's call. We'll hand back over to Chris Peetz for any final comments.

Speaker 3

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

Speaker 10

This concludes today's call. Thank you, everyone, for joining. Have a great day.

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