Mirum Pharmaceuticals, Inc. Q1 FY2025 Earnings Call

Hello and welcome to Mirum Pharmaceuticals Reports First Quarter 2025 Financial Results. My name is Carla, and I'll be coordinating your call today. I would now like to hand you over to Andrew McKibben, Senior Vice President of Strategic Finance and Investor Relations to begin. Andrew, please go ahead when you're ready.

Thanks Carla, and good afternoon everyone. I'd like to welcome you to Mirum Pharmaceuticals' first quarter 2025 conference call. I'm joined today by our CEO, Chris Peetz; our President and Chief Operating Officer, Peter Radovich; and Eric Bjerkholt, our Chief Financial Officer. Joanne Quan, our Chief Medical Officer could not be with us today as she's at a medical conference in Europe. Earlier today, Mirum issued a news release announcing the company's results for the first 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. These statements represent our judgment as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed. We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10-Q and subsequent SEC filings for more information. With that said, I'd like to turn the call over to Chris. Chris?

Thanks Andrew and good afternoon everyone. I'd like to start off by highlighting the tremendous progress Mirum has made in the first quarter of 2025. We continue to deliver across our key strategic objectives, furthering the growth of our commercial medicines and advancing our high-impact pipeline. We are excited to share the details of another record-breaking quarter for Mirum with total revenues reaching $111.6 million or 61% growth over the first quarter last year. Further, given how strong the year has started across the commercial business, we are updating our full year revenue guidance to be $435 million to $450 million. This increase is driven by robust growth from all three of our commercial medicines and highlights the Mirum team's continued strong commercial execution. In addition, I'm happy to report we've received three important regulatory approvals since the start of the year, adding growth drivers to the business. First, CTEXLI was FDA approved for the treatment of CTX in February, and we've begun promotional efforts to reach this underdiagnosed community. Second, LIVMARLI was approved in Japan for PFIC and Alagille syndrome through our partner, Takeda. And most recently, a convenient single tablet form of LIVMARLI was approved by the FDA. These milestones highlight our commitment to reach more patients globally. Our pipeline continues to make great progress as well. Starting with the VISTAS study of Volixibat in PSC, we are getting close to completing enrollment and now expect to achieve this in the third quarter of this year with top line data expected in the second quarter of 2026. As a reminder, both the VISTAS and VANTAGE studies successfully passed the dose selection interim analysis last year and are now in the confirmatory portions of the studies. As an example of what impact IBAT inhibitors can have in PSC, I'd like to highlight a recent presentation earlier this week at DDW of a case series of PSC patients receiving maralixibat through our compassionate use program. Encouragingly, we saw reductions in serum bile acids and all patients had a 2-point or greater reduction in pruritus. PSC remains a condition with no approved therapies, and these results build on our conviction for the potential of Volixibat to bring life-changing results to patients in need. Now, looking ahead, this Friday at EASL, we are also excited to present the updated 28-week interim data from the VANTAGE study in PBC, where Volixibat has been granted breakthrough designation. In this analysis, we showed the rapid, deep, and statistically significant improvement of pruritus on Volixibat we shared last June is durable through the full 28-week study. In this updated interim analysis, Volixibat showed a 3.8 point reduction from baseline and a 2.5 point placebo-adjusted reduction in pruritus. We're excited to advance this program through the confirmatory portion of the study and we continue to expect enrollment completion next year. And finally, for the balance of the pipeline, we remain on track to initiate our Phase 2 study for MRM-3379 in Fragile X syndrome this year as well as complete enrollment of our LIVMARLI EXPAND trial in 2026. 2025 is set to be another year of meaningful growth for Mirum as we continue to advance our commercial portfolio and pipeline. With strong execution and financial discipline, we are well-positioned to continue our leadership in rare disease. And with that, I'll turn it over to Peter to give a brief update on the commercial business. Peter?

Thanks, Chris. I'm pleased with the continued strong growth we are seeing across our three medicines, and our commercial team delivered another great quarter with total net product sales of $111.6 million. Based on the demand we are seeing across our medicines, we are raising our full year net product sales guidance to between $435 million and $450 million. For LIVMARLI, total global net product sales grew to $73.2 million in the first quarter, an increase of over 70% compared to our first quarter in 2024. U.S. LIVMARLI sales were $49.5 million, driven by robust new patient demand across indications. For the remainder of the year, we expect to see continued growth in both Alagille syndrome and PFIC and the approval of the tablet formulation adds to these positive dynamics with a single tablet per dose providing a distinct convenience advantage. LIVMARLI is now the only IBAT offering flexible formulations across all ages, an important milestone as we look to expand options for our patients. International LIVMARLI sales were $23.7 million. We saw strong demand growth in our direct European markets, driven by both continued penetration in Alagille syndrome in established markets and new launches in midsized countries. I'll note that our international distributor and partner revenue this quarter included about $6 million of inventory, which is a new dynamic that has not been seen in prior quarter sales numbers. Overall, the continued underlying demand growth across our international territories is the driving trend for this business, and we're excited to see the PFIC indication come online in many international markets this year. In Q1, we also saw strong growth from the bile acid products with $38.4 million of net product sales, representing 47% growth over the same quarter last year. With CTEXLI now approved in CTX, our efforts are focused on engaging healthcare professionals across several specialties to find patients in this underdiagnosed condition. And I'm pleased to say we're starting to see progress as we have seen an increase in new CTX patients since the FDA approval in February. Overall, it's been a tremendous start to the year for the commercial business. With the increased full year guidance of $435 million to $450 million, we look forward to continuing our strong execution throughout the year. And now I'll turn it over to Eric. Eric?

Thank you, Peter. Our financial position is strong and continues to improve. The first quarter 2025 net product revenue of $111.6 million compared to net product revenues of $69.2 million in the first quarter of last year. Cash, cash equivalents, and investments at March 31st was $298.6 million compared with $292.8 million at the beginning of the year. Total operating expense for the quarter ended March 31st was $126.8 million, which includes R&D expense of $46 million, SG&A expense of $57.7 million, and cost of sales of $23 million. R&D expense for the quarter included $7 million in one-time milestone payments related to the progress of our pipeline. Expense for the quarter also included non-cash stock-based compensation expense of $15.8 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 goods sold. We were operating cash flow positive for the quarter, and we expect to be cash flow positive for the full year. The cash contribution margin from our commercial business improved from approximately 47% in the first quarter of last year to approximately 53% for the first quarter this year. In addition, year-over-year R&D and G&A expense improved as a percent of revenue by over 10 percentage points. We continue to be well funded and financially independent, providing us the resources required to execute on our business plan. Now, I'll turn the call back over to Chris for final comments.

Thanks Eric. As a quick recap, we've had a great start to the year. Our three commercial medicines are growing ahead of initial expectations, and we're raising our full year guidance. In the last few months, we saw three important regulatory approvals that support the long-term growth potential of our commercial medicines and our growing pipeline is making excellent progress. The VISTAS study of Volixibat in PSC will complete enrollment in the coming months, and we are excited to share a great update for Volixibat in PBC this Friday. We're looking forward to starting the Phase 2 study in Fragile X syndrome later this year with MRM-3379 and completing enrollment of the LIVMARLI Phase 3 EXPAND and Volixibat VANTAGE studies next year. Overall, we are in excellent financial position, and I look forward to continued progress in the quarters ahead. And with that, operator, please open the call for questions.

We will now begin the question-and-answer session. Our first question comes from Gavin Clark-Gartner with Evercore.

Hey, guys. Congrats on the great progress. Thanks for taking the questions. First, for the additional Volixibat PBC data that's in the EASL abstract, it looks like the pruritus benefit deepened a bit at 28 weeks. And also, I think there were no discontinuations due to diarrhea beyond the one you saw at 16 weeks. Maybe you could just speak to that and anything else from the abstract there?

Yes. Thanks for the question, Gavin. Yes, we're really excited with how the data matured in the interim analysis. So, as you point out, the response overall deepens over time with the separation curves looking really strong. We'll have a few more data points that will be included in the presentation as well. So, I encourage you to stay tuned for an update there. And overall, the profile and benefit that patients are getting, I think the discontinuation statistic you point out, I think it's a great testament to what this can mean for patients.

Great. And then just on the LIVMARLI tablet formulation, can you just frame the additional IP that could come around with that? And also commercially, where you see adoption of the tablet formulation? Thank you.

Yes. So, I can touch on the IP and then I'll ask Peter to speak a little bit about kind of what we're looking forward to on adoption. For IP, I mean, the tablet formulation here did take some work really because of the properties of maralixibat. So, it did result in some novel IP. We have an allowed patent that we expect to grant soon that covers the formulation that would extend coverage out to 2043. I'll pass it over to Peter for the second part of the question.

Yes. Thanks for the question, Gavin. And we think that tablet will be a pretty attractive option for really all the LIVMARLI patients over 25 kilos or you can think of older children and adolescents up to adults. I think that would be a pretty attractive option for many of them. So, I look forward to seeing how that unfolds in the back half of this year.

Thanks for the questions.

And the next question comes from Jessica Fye with JPMorgan.

Hey guys, good afternoon. Thanks for taking our questions. Curious how you think your interim Phase 2 PBC data compares to the pruritus data for linerixibat? And also curious what you make of the placebo response observed on pruritus in that trial? Then I have a second question.

Thanks for the question, Jessica. What we've observed is primarily related to the abstracts posted on the linerixibat program, so I don't want to comment too much on their data. However, in our view, it highlights some of the strengths of the Volixibat program. We've seen significant improvements not only in change from baseline but also in the placebo-adjusted difference, which we attribute largely to the dosage of Volixibat. I truly believe we are at the maximum effective dose, optimizing our program's activity. What stands out for us is the 3.8 reduction from baseline, leading to a 2.5 placebo-adjusted difference.

Great. Can you remind us what needs to happen between now and when you start the Phase 2 study later this year?

Yes. Thanks for the follow-up on the MRM-3379. So, we're busy kind of putting together the IND now. In the background, what we've been working on is some study planning, having a dialogue with FDA on that. And this is an IND with a new division, so, that's really what we've been working on to date. And as we get close to kind of first patient in, we'll provide a more detailed update on what that study design looks like. No change overall from kind of how we thought about from the high-level summary thoughts, but we'll provide further detail later this year.

Thank you.

Thanks for the questions.

Thank you. The next question comes from Michael Ulz with Morgan Stanley.

Hello. This is Selena on for Mike. Thanks for taking our question. Could you give us an update on LIVMARLI access versus competitors and how step through policies in Alagille and PFIC are evolving?

Yes. Thanks for the question, Selena. Yes, the access to LIVMARLI in the U.S. is very strong. Really don't see that being a barrier for us in either indication. Yes, I mean, as you know, there are some policies that have LIVMARLI in a preferential position in the Alagille indication from a step-through perspective, which is it's certainly a beneficial aspect. But I think overall, really see the differentiating factors for LIVMARLI being the strong clinical value proposition and the support that Mirum provides and the access is really not a barrier.

Thank you.

Thanks for the question.

And the next question comes from Mani Foroohar with Leerink Partners.

Hey guys, You have Ryan on for Mani. Thanks for taking our question. I was just hoping you can talk about your expectations for the VISTAS study in terms of pruritus reduction. Should we look to VANTAGE as a good benchmark for absolute reduction from baseline? And then just how you guys see that Volixibat being positioned within the PSC landscape?

Thank you for the question, Ryan. To start, I appreciate your background on data expectations, and I’ll ask Peter to discuss our competitive positioning on the commercial side moving forward. Regarding our expectations for Volixibat’s activity level, the interim results from VANTAGE provide us with the most comprehensive data set we have, and they align well with what we observe for IBAT when fully dosed in a cholestatic pruritus context. We are optimistic that we can achieve that same level of activity for the VISTAS study. I’d like to reference the DDW abstract I mentioned in my prepared remarks, which highlights that higher doses of LIVMARLI in compassionate use patients result in significant reductions in pruritus. It’s important to note that the scale we are using is the Clinician Scratch Scale, which ranges from 0 to 4. Achieving a 2-point reduction on that scale is substantial. Overall, we are confident in the dosing and study design of VISTAS, using VANTAGE as a benchmark for what we might expect. Now, I’ll turn it over to Peter to discuss the market dynamics.

Yes. In terms of the market dynamic, the work we've done in PSC aligns with what you would expect. There are currently no FDA-approved therapies, making this a very unsatisfied market. The existing therapies do not meet the needs of prescribers or patients. Some off-label therapies are utilized in other indications, both pediatric and adult, but satisfaction with those options is quite low. We are really excited for PSC patients with pruritus, especially with the potential approval of Volixibat, as it could have a strong, highly preferred position in the market without significant competition.

Great. Thanks guys.

Thanks for the question.

And the next question comes from David Lebowitz with Citi.

Thank you for allowing me to ask my question. I have a couple of queries concerning the figures. About the inventory, which you mentioned is for the first time this quarter, should we consider this to be a stable position right now? Or are you still figuring out where inventory levels will eventually be? Additionally, could you provide some insights into what we might expect regarding one-time expenses in the operating budget later this year?

I'll turn it over to Peter to comment on the inventory and have Eric talk through some of the other line items.

In terms of inventory and what we observed in Q1, as we have expanded our international partner markets, there are really two types of orders we've noticed over the years. Until now, most orders have been characterized by having a patient, a prescriber, a payer, and then an order. Typically, these orders involve three to nine months of product, and consumption usually follows shortly after the order. This is why our international sales have fluctuated over the years. What we experienced in Q1 was different; with the addition of new partners, we've seen orders where they're essentially stocking products in a warehouse. This will translate to sales demand in 2025. It’s crucial to keep in mind that this was primarily a Q1 occurrence as you analyze trends from quarter to quarter.

Yes, regarding your milestone question, we do have milestones in our business related to both development and regulatory progress as well as commercial progress. This particular milestone was linked to development progress. While we do not provide specific guidance on what to expect for the remainder of the year, we anticipate that as our business continues to advance, there will be additional milestones indicating steady progress. For instance, there could be more developments.

Perfect.

Thanks for the question.

The next question comes from Ryan Deschner with Raymond James.

Hi there. Curious what your expectations for the impact of the tablet formulation of LIVMARLI on demand and competitive positioning is as well as what proportion of Alagille patients you think you'd expect to opt for the tablet at a more steady state? And would you expect this to drive meaningful switch demand? Thanks.

I believe this will be an appealing option for Alagille and PFIC patients. The label is for those weighing 25 kilos and up, which typically includes children aged between eight and ten years. Having a single tablet per dose is quite beneficial, as many of these patients are already on multiple background therapies, making pill burden a concern. Thus, offering one tablet is advantageous. However, most patients are likely still under eight years old, so the liquid formulation may be preferred in those cases. Nevertheless, a significant number might find the tablet option appealing. I think we could see patients who are currently using the liquid form switch to the tablet. Additionally, there are scenarios where a teenager or young adult, who has been contemplating transitioning from LIVMARLI to an IBAT, will find the more convenient tablet option attractive for their school and travel needs. This could result in various new patient acquisitions and switching situations.

Thank you very much.

Thanks for the question.

Our next question comes from Brian Skorney with Baird.

Hi, this is Luke on for Brian. Thanks for taking the question. Congrats on the quarter. On CTEXLI, could you provide a bit of insight on the rate of patient identification since approval as well as your go-forward expectations?

Thanks Luke for the question. I'll actually ask Peter to maybe talk about a couple of our efforts on that front.

Yes. Yes. I mean like we said in our prepared comments, we're excited. We've seen a nice uptick from the historical rate. As you know, chenodiol has been available for a while, and we've seen a nice uptick since February. I think some of it is just coming from promotion and kind of being out there and having an FDA-approved product for the indication matters, I think, to clinicians and patients. So we've seen that. We've also invested in disease awareness because a lot of the challenge here is making the diagnosis. So, kind of work towards raising awareness of presenting symptoms in neurology where often this can present as ataxia and motor coordination issues. Ophthalmology is another area where you can see patients presenting with bilateral cataracts and trying to raise an index of suspicion there to do genetic testing. And those efforts have kind of borne through GI patients with chronic diarrhea without other explanation. So, kind of focused on some of these different areas, and that's been kind of where we've made progress so far.

Great. Thank you.

Thanks for the question.

And the next question comes from Jon Wolleben with Citizens.

Hi, this is Catherine on for Jon. I just had two quick questions. One about PFIC and the launch in the U.S. as well as ex-U.S. How is that comparing to expectations? And then also, can you comment on the sustainability of the profitability into 2026?

Thanks, Catherine, for the question. First, I want to make a quick comment about the PFIC launch before handing it over to Eric to address the profitability question. The PFIC launch has been going significantly better than we anticipated. However, the numbers are still relatively small since PFIC is a smaller indication compared to Alagille. We're really enthusiastic about the progress we're seeing, which we attribute to our dedicated team at Mirum who are actively promoting the product. Additionally, the compelling data from the Phase 3 program showing placebo-controlled improvements in growth and bilirubin levels has been quite impactful. Now, I'll let Eric address the profitability question.

Yes. To be clear, we were cash flow positive in the first quarter, and we expect to remain cash flow positive for the full year. Profitability is a different issue due to several non-cash charges, so we do not expect to be profitable in the GAAP sense anytime soon, but our focus is on being cash flow positive.

Thanks for the question.

Thank you very much. So, that was our final question. So, I will hand back over to the CEO, Chris Peetz, for any final remarks.

Great. Well, thank you all for joining us today, and hope you all have a great day. Bye.

Thank you, everyone, for joining today's call. Have a great day. You may now disconnect.