Mirum Pharmaceuticals Q1 2025 Earnings Report

Mirum Pharmaceuticals EPS Results

• Actual EPS: -$0.30
• Consensus EPS: -$0.35
• Beat/Miss: Beat by +$0.05
• One Year Ago EPS: -$0.54

Mirum Pharmaceuticals Revenue Results

• Actual Revenue: $111.59 million
• Expected Revenue: $98.47 million
• Beat/Miss: Beat by +$13.12 million
• YoY Revenue Growth: +61.20%

Mirum Pharmaceuticals Announcement Details

• Quarter: Q1 2025
• Date: 5/14/2025
• Time: Before Market Opens
• Conference Call Date: Wednesday, May 7, 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 Q1 2025 Earnings Call Transcript

Key Takeaways

• Record-breaking revenues: Q1 total net product sales reached $111.6 million (61% growth YoY) and the company raised full-year guidance to $435 – 450 million driven by robust growth across all three commercial medicines.
• Three regulatory approvals secured: FDA approval of Citexly for CTX in February, FDA approval of a single-tablet formulation of LIVMARLI, and Japanese approval of LIVMARLI for PFIC and Alagille syndrome via the Takeda partnership.
• Advancing high-impact pipeline: Enrollment in the VISTA study of velixibat for PSC is on track to complete in Q3 ’25 with top-line data in Q2 ’26, and updated 28-week VANTAGE PBC data show durable, statistically significant pruritus reductions.
• Expansion of commercial footprint: LIVMARLI global sales grew over 70% to $73.2 million in Q1, bile acid product sales rose 47% to $38.4 million, and the new tablet formulation offers enhanced convenience with accelerating international demand.
• Strong financial position: Cash and investments totaled $298.6 million at quarter end, the company was operating cash-flow positive in Q1 and expects to remain so for the full year, with R&D and SG&A expenses improving by over 10 percentage points as a percent of revenue.

Presentation

[Operator]

I would now like to hand you over to Andrew McKeown, Senior Vice President of Strategic Finance and Investor Relations to begin. Andrew, please go ahead when you're ready.

[Andrew McKibben, Vice President of Investor Relations and Finance at Mirum Pharmaceuticals Inc]

Thanks, Carla, and good afternoon, everyone. I'd like to welcome you to Miriam Pharmaceuticals first quarter twenty twenty five conference call. I'm joined today by our CEO, Chris Pietz our President and Chief Operating Officer, Peter Radovich and Eric Bjerkle, our Chief Financial Officer. Joanne Kwan, our Chief Medical Officer, cannot be with us today as she's at a medical conference in Europe. Earlier today, Meera issued a news release announcing the company's results for the first quarter twenty twenty five.

[Andrew McKibben, Vice President of Investor Relations and Finance at Mirum Pharmaceuticals Inc]

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 Merame'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 the 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 the subsequent SEC filings for more information.

[Andrew McKibben, Vice President of Investor Relations and Finance at Mirum Pharmaceuticals Inc]

With that

[Andrew McKibben, Vice President of Investor Relations and Finance at Mirum Pharmaceuticals Inc]

said, I'd like to turn the call over to Chris. Chris?

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks, Andrew, and good afternoon, everyone. I'd like to start off by highlighting the tremendous progress Merum has made in the first quarter of twenty twenty five. 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 $111,600,000 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 $435,000,000 to $450,000,000 This increase is driven by robust growth from all three of our commercial medicines and highlights the Mirum team's continued strong commercial execution.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

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, Citexly 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.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Our pipeline continues to make great progress as well, starting with the VISTA study of velixibat and 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 twenty twenty six. As a reminder, both the VISTAs and VANTAGE studies successfully passed a 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 mireliksivat through our compassionate use program. Encouragingly, we saw reductions in serum bile acids and all patients had a two point or greater reduction in pruritus.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

PSC remains a condition with no approved therapies and these results build on our conviction for the potential of elixirat to bring life changing results to patients in need. Now looking ahead, this Friday at EASL, we are also excited to present the updated twenty eight week interim data from the VANTAGE study in PBC, where velexibat has been granted breakthrough designation. In this analysis, we show the rapid, deep and statistically significant improvement of pruritus on velexibat we shared last June is durable through the full twenty eight week study. In this updated interim analysis, velexibat showed a 3.8 reduction from baseline and a 2.5 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.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

And finally, for the balance of the pipeline, we remain on track to initiate our Phase II study for MRM-three thousand three hundred seventy nine in fragile X syndrome this year as well as complete enrollment of our Lipmarly EXPAND trial in twenty twenty six. 20 20 five is set to be another year of meaningful growth for Meron 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?

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

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 $111,600,000 Based on the demand we are seeing across our medicines, we are raising our full year net product sales guidance to between $435,000,000 and $450,000,000 For LEMARLEY, total global net product sales grew to $73,200,000 in the first quarter, an increase of over 70% compared to our first quarter twenty twenty four. U. S. LIVMARLI sales were $49,500,000 driven by robust new patient demand across indications.

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

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. Lidmarly is now the only IBAT offering flexible formulations across all ages, an important milestone as we look to expand options for our patients. International Lidmarly sales were $23,700,000 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 mid sized countries. I'll note that our international distributor and partner revenue this quarter included about $6,000,000 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 PPIC indication come online in many international markets this year.

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

In Q1, we also saw strong growth from the bile acid products with $38,400,000 of net product sales, representing 47% growth over the same quarter last year. With Citexly 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 CTS 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 $435,000,000 to $450,000,000 we look forward to continuing our strong execution throughout the year.

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

And now I'll turn it over to Eric. Eric?

[Eric Bjerkholt, Chief Financial Officer at Mirum Pharmaceuticals Inc]

Thank you, Peter. Our financial position is strong and continues to improve. The first quarter twenty twenty five net product revenue of $111,600,000 compared to net product revenues of $69,200,000 the first quarter of last year. Cash, cash equivalents and investments at March 31 were $298,600,000 compared with $292,800,000 at the beginning of the year. Total operating expense for the quarter ended March 31 was $126,800,000 which includes R and D expense of $46,000,000 SG and A expense of $57,700,000 and cost of sales of $23,000,000 R and D expense for the quarter included $7,000,000 in one time milestone payments related to the progress of our pipeline.

[Eric Bjerkholt, Chief Financial Officer at Mirum Pharmaceuticals Inc]

Expense for the quarter also included non cash stock based compensation expense of $15,800,000 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 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 and D and G and 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.

[Eric Bjerkholt, Chief Financial Officer at Mirum Pharmaceuticals Inc]

Now I'll turn the call back over to Chris for final comments.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

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.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

The VISTA study of velexibat and PSC will complete enrollment in the coming months, and we are excited to share a great update for velexibat in PBC this Friday. We're looking forward to starting the Phase II study in fragile X syndrome later this year with MRM-three thousand three hundred 70 nine and completing enrollment of the Libmarli Phase III EXPAND and velixibat 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.

[Operator]

Our first question comes from Kevin Clark, Partner with Evercore.

[Gavin Clark-Gartner, Analyst at Evercore]

Hey guys. Congrats on the great progress. Thanks for taking the questions. First, for the additional velexibat PBC data that's in the EASL abstract, looks like the pruritus benefit deepened a bit at twenty eight weeks. And also, think there were no discontinuations due to diarrhea beyond the one you saw at sixteen weeks.

[Gavin Clark-Gartner, Analyst at Evercore]

Maybe you could just speak to that and anything else from the abstract there?

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

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.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

So I encourage you to stay tuned for an update there. And overall, the profile and benefit that patients are getting, I think that discontinuation statistic you point out, I think, a great testament to what this can mean for patients.

[Gavin Clark-Gartner, Analyst at Evercore]

Great. And then just on the Libmarly 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? Yes.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

So I can touch on the IP and then ask Peter to speak a little bit about what looking forward to on adoption. For IP, I mean, the tablet formulation here did take some work really because of the properties of mirelixibat. 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 02/1943. Pass over to Peter for the second part of the question.

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

Yes. Thanks for the question, Gavin. And we think that capital will be a pretty attractive option for really all the Le Marley patients over twenty five 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 look forward to seeing how that unfolds in the

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

back half of this year.

[Gavin Clark-Gartner, Analyst at Evercore]

Perfect. Thanks, guys.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

And the next question comes from Jessica Farr with JPMorgan.

[Jessica Fye, Managing Director & Equity Research Analyst - Biotechnology at JP Morgan]

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

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks for the question, Jessica.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Yes, mean, again, what we've seen really is just the abstracts that was posted on the Linear Expat program. So I don't want to speak too much to their data. But what it did does do for at least in our view is highlight some of the strengths in the pelixibat 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, by the dose of elixibat. I really believe that we're at a maximally efficacious dose and optimizing activity for the program here.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

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.

[Jessica Fye, Managing Director & Equity Research Analyst - Biotechnology at JP Morgan]

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 II study in the back half?

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Yes. Thanks for the follow-up on the MRM-three thousand three and seventy nine. So we're busy

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

kind

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

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 the 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.

[Jessica Fye, Managing Director & Equity Research Analyst - Biotechnology at JP Morgan]

You.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks for the questions.

[Operator]

Thank you. The next question comes from Michael Ellsworth, Morgan Stanley.

[Selena Zhang, Global capital markets at Morgan Stanley]

Hello. This is Selena on for Mike. Thanks for taking our question.

[Selena Zhang, Global capital markets at Morgan Stanley]

Could you give us an update on the Marley access versus competitors and how that's your policies in Alagille and PFIC are evolving?

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

Yeah. Thanks for the question, Selena. Yeah. The access to the Marley in The U. S.

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

Is very strong. Really don't see that being a barrier for us in either indication. Yeah. I mean, as you know, there some policies that have, know, the Marley in a preferential position analogy on indication from a step through perspective, which is, you know, certainly a beneficial aspect. But I think overall, really see the differentiating factors for LibMARLI being the strong clinical value proposition and the support that Merit provides, and the access is really not a barrier.

[Selena Zhang, Global capital markets at Morgan Stanley]

Thank you.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

Thank you. And the next question comes from Manav Pruhat with Leerink Partners.

[Ryan Mcelroy, Equity Research Associate at Leerink Partners]

Hey, guys. You have Ryan on for Mani. Thanks for taking our question. Was just hoping you can talk about your expectations for the VISTA study in terms of pruritus reduction. Should we look to VANTAGE as a good benchmark for absolute reduction from baseline?

[Ryan Mcelroy, Equity Research Associate at Leerink Partners]

And then just how you guys see that vilixibat being positioned within the PSC landscape?

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Right. Thanks for the question there. First, 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 velexibat's activity level, the VANTAGE interim result really is the most robust data set we have and really and it's consistent with what we see for IVAT that is really fully dosed in a cholestatic pruritus setting. So optimistic that we can be in that range of activity level for the VISTA study.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

I'd refer back actually to that DDW abstract that I mentioned in my prepared remarks where with higher doses of Libmarli 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 it's only a zero to four range. So getting a two point reduction on that scale is really powerful. So overall, 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.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

I'm going turn it over to Peter for market dynamic.

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

Yeah. And in terms of the market dynamic, Brian, 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.

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

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. Really excited for PSC patients with pruritus upon a potential approval here for velixibat. I think it just really has a strong, highly preferred position in the market without meaningful competition.

[Ryan Mcelroy, Equity Research Associate at Leerink Partners]

Great. Thanks, guys.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

And the next question comes from David Lebowitz with Citi.

[David Lebowitz, Analyst at Citigroup]

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 both see this as being steady state right now? Or are you still learning about where ultimately inventory levels will sit? And on the onetimers and whatnot in the operating spend, could you just comment on what we should be

[David Lebowitz, Analyst at Citigroup]

able to what we should

[David Lebowitz, Analyst at Citigroup]

see in that regard later in the year?

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

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

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

Yeah. I mean, in in in

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

terms of the inventory and what we saw in q one, 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 time where they'll 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, 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 truly stock or in a warehouse.

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

And it will pull through to demand sales in 2025. So but it's I think it's important to think about as you trend it out going quarter over quarter that it was it was really a Q1 event.

[Eric Bjerkholt, Chief Financial Officer at Mirum Pharmaceuticals Inc]

Yeah. And as as for your milestone question

[Ryan Mcelroy, Equity Research Associate at Leerink Partners]

go ahead.

[Eric Bjerkholt, Chief Financial Officer at Mirum Pharmaceuticals Inc]

Yeah. We do we do have milestones to 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 we expect and hope there will be additional milestones showing steady progress. For example, they could be That's going to be a question.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks for the questions.

[Operator]

The next question comes from Ryan Deschner with Raymond

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

expectations for the impact of the tablet formulation of Blue Marley 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

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

would you expect us

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

to drive meaningful switch demand? Thanks.

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

Yeah. Thanks, Ryan. I mean,

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

I think it will be a really attractive option for Alagille and PPIC patients that are the label is really for twenty five kilos and over, which is most of the time that's going 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 in other therapies. So pill burden is an issue. So being able to offer one tablet is, I think, pretty attractive.

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

Probably the average patient is still under eight years old. So I think probably in that case, think that liquid will be preferred there. But a substantial minority of them, I would think, probably see the tablet as a pretty attractive option. And I think this could drive we could see patients that are on the solution liquid solution now on the Marley switch to tablet. You know, you could you could imagine clinical scenarios where you have a teenager or or a young adult who's kind of been thinking about an IBAT, but or about Live Marley, and now you now you have a a more convenient option.

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

They want to be able to, you know, go to school, go to travel. But so you can imagine a variety of different new patients and and switch scenarios.

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

Thank you very much.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks for the question.

[Operator]

Our next question comes from Brian Skorney with Baird.

[Luke Herrmann, Senior Research Analyst at Baird]

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

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

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

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

Yes. Yes. And like we said in our prepared comments, we're excited. We've seen a nice uptick from the historical rate. As you know, Kenediol has been available for a while, and we've seen a nice uptick since February.

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

I think some of it is just coming from promotion, 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 with ataxia and motor coordination issues. Ophthalmology is another area where, you know, you can see patients presenting with bilateral cataracts and trying to raise an index of suspicion there to do genetic testing.

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

And those efforts have kind of kind of kind of borne through GI patients with, you know, 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.

[Luke Herrmann, Senior Research Analyst at Baird]

Great. Thank you.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Any question?

[Operator]

And the next question comes from John Walden with Citi.

[Catherine Okoukoni, Analyst at Citizens JMP]

This is Catherine on for John. I just had two quick questions. One about PFIC and the launch in The U. S. As well as ex U.

[Catherine Okoukoni, Analyst at Citizens JMP]

S. How's that comparing to expectations? And then also, can you comment on the sustainability of the profitability into 2026?

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

Thanks, Katherine, the question. I mean, first, I have 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 Merum that's out there spreading the word.

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

And then also just really compelling data coming out of the Phase III program where you see placebo controlled improvements and growth in bilirubin or some of these things that are really impactful. And then maybe Eric can speak to the profitability question.

[Eric Bjerkholt, Chief Financial Officer at Mirum Pharmaceuticals Inc]

Yes. So we're 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 noncash charges, and so we're not expecting to be profitable anytime soon in sort

[Eric Bjerkholt, Chief Financial Officer at Mirum Pharmaceuticals Inc]

of a gap sense. But cash flow

[Eric Bjerkholt, Chief Financial Officer at Mirum Pharmaceuticals Inc]

cash flow positive is what we're focused on.

[Operator]

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

[Chris Peetz, President and Chief Executive Officer at Mirum Pharmaceuticals Inc]

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

[Operator]

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

Participants

Executives

• Andrew McKibben, Vice President of Investor Relations and Finance
• Chris Peetz, President and Chief Executive Officer
• Peter Radovich, COO & President
• Eric Bjerkholt, Chief Financial Officer

Analysts

• Gavin Clark-Gartner, Analyst at Evercore
• Jessica Fye, Managing Director & Equity Research Analyst - Biotechnology at JP Morgan
• Selena Zhang, Global capital markets at Morgan Stanley
• Ryan Mcelroy, Equity Research Associate at Leerink Partners
• David Lebowitz, Analyst at Citigroup
• Ryan Deschner, Vice President - Equity Research at Raymond James Financial
• Luke Herrmann, Senior Research Analyst at Baird
• Catherine Okoukoni, Analyst at Citizens JMP