MIRM - NASDAQ

Hello and welcome to Mirum Pharmaceuticals Reports First Quarter 2025 Financial Results. My name is Carla, and I'll be coordinating your call today. [Operator Instructions] 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; Eric Bjerkholt, our Chief Financial Officer. Joanne Quan, our Chief Medical Officer could not be with us today as she's at an 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.

Of course. We will now begin the question-and-answer session. [Operator Instructions] 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.

Perfect. Thanks guys.

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. Yes, I mean the -- again, what we've seen really is just the abstracts that was posted on the linerixibat program. So, I don't want to speak too much to their data. But what it does do for -- at least in our view, is highlight some of the strengths in the Volixibat program where not only in change from baseline, but in the placebo-adjusted difference, having really strong outcome there. But a lot of it, we think, driven by the dose of Volixibat. I really believe that we're at the maximally efficacious dose and optimizing activity for the program here. So, that's what stands out for us is that driving that 3.8 reduction from baseline, which then results in the 2.5 placebo-adjusted difference.

Great. And then can you -- maybe switching to Fragile X. Can you just remind us what needs to happen between now and when you start the Phase 2 study in the back half?

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?

Ryan, thanks for the question there. First, you're giving some background on kind of data expectations, and then I'll actually ask Peter to speak through the competitive positioning kind of on the commercial side for looking forward. In terms of our expectations, how we think about Volixibat's activity level, the VANTAGE interim result really is the most robust data set we have and really -- it's consistent with what we see for IBAT that is really fully dosed in a cholestatic pruritus setting. So, optimistic that we can be in that range of activity level for the VISTAS study. I'd refer back actually to that DDW abstract that I mentioned in my prepared remarks, where with the higher doses of LIVMARLI in those compassionate use patients, you're driving pretty substantial pruritus reductions. One thing to note is that scale actually is the Clinician Scratch Scale. So, only a 0 to 4 range. So, getting a 2-point reduction on that scale is really powerful. So overall, we feel good about how we've dosed and set up the study design for VISTAS to use Vantage as an example of what we could be seeing. I maybe turn it over to Peter for the market dynamics.

Yes. And in terms of the market dynamic, Ryan, the work we've done in PSC, probably not surprising to you. I mean obviously, no FDA-approved therapies I'd characterize it as a very unsatisfied market. It's just not satisfactory therapy from the perspective of prescribers or patients. So, you do have some of the off-label therapies that we see get used in our other indications, pediatric as well as adult. But I'd say the satisfaction with those is quite low. So, really excited for PSC patients with pruritus -- upon a potential approval here for Volixibat, I think it just really have a strong, highly preferred position in the market without meaningful competition.

Great. Thanks guys.

Thanks for the question.

And the next question comes from David Lebowitz with Citi.

Thank you very much for taking my question. A couple of questions on the numbers. Regarding the inventory, which you said, I believe, was the first time this quarter. Is this -- should we see this as being steady state right now? Or are you still learning about where ultimately inventory levels will sit? And on the one-timers and whatnot in the operating spend, could you just comment on what we should -- what we should see in that regard later in the year?

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

Yes, I mean in terms of the inventory and what we saw in Q1, really, as we've expanded our international partner markets, you can kind of think of -- there's really two kinds of orders that we've seen over the years. And up until now, it's really been orders, the first kind where there's a patient identified, there's a prescriber, there's a payer and there's an order. Oftentimes, they're ordering three or six or even nine months of product, but those -- there's consumption happening right after that order. That's why those -- our international number has been a little bit lumpy over the years. this is a new one here of what we saw in Q1, which is as we kind of added partners, we have new partners coming online where they're ordering and they're kind of holding it's truly stock in a warehouse. And it will pull through to demand sales in 2025. So -- but it's -- I think it's important to think about as you trended out going quarter-over-quarter that it was really a Q1 event.

Yes. And as for your milestone question. Yes, we do have to milestone in our business, both tied to development and regulatory progress as well as commercial progress. This one happened to be tied to development progress. We don't give specific guidance on what we can expect for the rest of the year. But as we continue to progress our business, we expect and hope there will be additional milestones showing steady progress. For example, there could be--

Perfect. [indiscernible].

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.

Yes. Thanks, Ryan. I mean I think it will be a really attractive option for Alagille and PFIC patients that are -- the label is really -- is for 25 kilos and over, which is most of the time, that's going to be when you're between eight and 10 years old. So, I think having a single tablet per dose is quite attractive. Most of these patients are on background therapies or so and other therapies. So pill burden is an issue. So being able to offer one tablet is, I think, pretty attractive. Probably the average patient is still under eight years old. So, I think probably in that case, I think the liquid will be preferred there. But a substantial minority of them, I would think, would probably see the tablet as a pretty attractive option. And yes, I think this could drive -- we could see patients that are on the solution -- liquid solution now on LIVMARLI switch to tablet. You could imagine clinical scenarios where you have a teenager or a young adult who's kind of been thinking about an IBAT, but about LIVMARLI and now you have a more convenient option. They want to be able to go to school, go travel. So, you can imagine a variety of different new patients and switch scenarios.

Thank you very much.

Thanks for the question.

[Operator Instructions] 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. I mean, first, a quick comment on PFIC launch, and I'll pass it over to Eric on the kind of profitability question. I mean the PFIC launch actually has been going -- I'd describe it as quite a bit better than expectations. But it's still -- that means relatively small numbers because PFIC is a smaller indication overall compared to Alagille. But we're excited about what we're seeing and really attribute that to a great team here at Mirum that's out there spreading the word. And then also just really compelling data coming out of the Phase 3 program where you see placebo-controlled improvements in growth in bilirubin or some of these things that are really impactful. And then maybe Eric can speak to the profitability question.

Yes. So, to be clear, we are -- we were cash flow positive in the first quarter, and we're expecting to be cash flow positive for the full year. profitability is a different issue because we have a number of non-cash charges. And so we're not expecting to be profitable anytime soon in sort of a GAAP sense, but cash flow positive is what we're focused on.

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.