Mirum Pharmaceuticals Q1 2026 Earnings Report

Mirum Pharmaceuticals EPS Results

• Actual EPS: -$13.43
• Consensus EPS: -$0.75
• Beat/Miss: Missed by -$12.68
• One Year Ago EPS: -$0.30

Mirum Pharmaceuticals Revenue Results

• Actual Revenue: $159.88 million
• Expected Revenue: $148.21 million
• Beat/Miss: Beat by +$11.68 million
• YoY Revenue Growth: +43.30%

Mirum Pharmaceuticals Announcement Details

• Quarter: Q1 2026
• Date: 5/6/2026
• Time: Before Market Opens
• Conference Call Date: Wednesday, May 6, 2026
• Conference Call Time: 4:30PM ET

Mirum Pharmaceuticals (NASDAQ:MIRM) is a late-stage biopharmaceutical company dedicated to the development and commercialization of innovative therapies for rare cholestatic liver diseases. The company’s primary focus lies in addressing the unmet medical needs of patients suffering from genetic and progressive forms of pediatric liver disorders, where limited treatment options currently exist.

Mirum’s lead product candidate, maralixibat (Livmarli), is an ileal bile acid transporter inhibitor designed to reduce systemic bile acid accumulation and alleviate associated pruritus and liver damage. Maralixibat has achieved regulatory approval in the United States for the treatment of cholestatic pruritus in patients with Alagille syndrome and is under review or in clinical development for other rare cholestatic conditions, including progressive familial intrahepatic cholestasis (PFIC). In addition to its lead compound, Mirum maintains a pipeline of product candidates aimed at expanding the therapeutic options available to patients with orphan liver diseases.

Founded in 2016 and headquartered in Brisbane, California, Mirum Pharmaceuticals operates with a patient-centric approach that emphasizes collaboration with advocacy groups and key opinion leaders in hepatology. Under the leadership of President and Chief Executive Officer Derek Chalmers, the company is actively pursuing regulatory approvals and commercial partnerships to bring its therapies to patients in the United States and international markets.

Mirum Pharmaceuticals Q1 2026 Earnings Call Transcript

Key Takeaways

• Positive Sentiment: Raised full-year revenue guidance to $660M–$680M, driven by strong LIVMARLI uptake (notably PFIC in adults) and stronger-than-expected international sales.
• Positive Sentiment: Clinical progress is expanding Mirum beyond pediatrics — the VISTAS study (volixibat in PSC) showed significant pruritus improvement and the AZURE‑1 Phase IIb (brelovitug in hepatitis delta) supported activity, with late‑breaking presentations expected at EASL.
• Positive Sentiment: Mirum licensed zolegorsertib (ALK2 inhibitor) from Incyte for FOP; an NDA is in priority review with a PDUFA on Sept 26 and Mirum expects a potential launch by year‑end if approved, targeting a strategic fit in its rare genetic franchise and a >$200M peak sales opportunity.
• Neutral Sentiment: Q1 shows revenue growth (net product sales ≈ $160M) and cash/investments of ≈ $421M, but operating expense was elevated ($949M) due mainly to one‑time Bluejay acquisition costs; management says R&D investment is funded, expects operating cash flow positive next year and GAAP profitability by 2028.
• Positive Sentiment: Mirum plans to expand its U.S. liver commercial team from ~20 to ~60 field reps to reach >4,000 liver healthcare providers, supporting launches and commercialization in PSC, hepatitis delta and adult liver settings.

Presentation

[Operator]

Good afternoon, and welcome to Mirum Pharmaceuticals' First Quarter 2026 Earnings Conference Call. My name is Tracy, and I will be your operator today. All lines are currently in a listen-only mode, and there will be an opportunity for Q&A after management's prepared remarks. If you would like to ask a question during that time, press star one to raise your hand. I would now like to hand the conference over to Andrew McKibben, SVP of Strategic Finance and Investor Relations. Please go ahead.

[Andrew McKibben, SVP of Strategic Finance and Investor Relations at Mirum Pharmaceuticals]

Thank you, Tracy, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' first quarter 2026 earnings conference call. For our prepared remarks, I'm joined today by our Chief Executive Officer, Chris Peetz, our President and Chief Operating Officer, Peter Radovich, and Eric Bjerkholt, our Chief Financial Officer. Our Chief Medical Officer, Joanne Quan, will be joining us for Q&A. Earlier this afternoon, Mirum issued a press release reporting our first quarter 2026 financial results. Copies of the press release and our SEC filings are available 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 and financial guidance.

[Andrew McKibben, SVP of Strategic Finance and Investor Relations at Mirum Pharmaceuticals]

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 about these risks and uncertainties. With that said, I'd like to turn the call over to Chris. Chris?

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks, Andrew. Good afternoon, everyone. We have a number of important updates to cover today. I'd like to start by grounding in the vision we set when we founded Mirum in 2018, building a company focused on bringing forward medicines for overlooked rare diseases. This quarter reflects the progress we've made in turning that vision into a durable, growing business. Our start was based on LIVMARLI. Today we are a broader rare disease company with three approved medicines and a pipeline positioned to deliver multiple new therapies over the next twoyears. These high-impact programs are grouped across two focus areas: rare liver disease, where we have built clear leadership, and rare genetic disease, where we are establishing a second growth platform, each with distinct commercial capabilities. Across both, we've built a financially self-sustaining business that can support continued investment in the portfolio.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Our strategy is driving compelling results. Starting with rare liver disease, uptake of LIVMARLI remains strong, driven in part by performance in PFIC, which continues to exceed expectations. Based on that demand and continued performance across all brands, we are raising our full-year revenue guidance to $660 million-$680 million. More importantly, we are now seeing the next phase of our rare liver disease business take shape. Our recent clinical readouts in PSC and hepatitis delta represent important potential expansions for this business, extending beyond our pediatric foundation into larger patient populations with significant unmet need. In PSC, the VISTAS study of volixibat showed a significant improvement in pruritus, reinforcing the potential for volixibat to play an important role for these patients who currently have no approved medicines.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

This is a major advance in PSC research and positions volixibat as a potential first approved medicine for patients in the U.S. In hepatitis delta, results from the phase IIb portion of the AZURE-1 study further support the potential for brelovitug in a patient population where treatment options are extremely limited. We look forward to the upcoming late-breaking presentations for both VISTAS and AZURE at EASL later this month. In parallel to this expansion of our rare liver disease business, today we are announcing another step in building out our rare genetic disease business with the addition of zilurgisertib, recently licensed from Incyte. Zilurgisertib is a once-daily oral ALK2 inhibitor in development for fibrodysplasia ossificans progressiva, or FOP, an ultra-rare progressive condition where patients develop bone in soft tissues.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

This accumulation of excess bone leads to profound physical immobilization, with most FOP patients becoming wheelchair dependent by early adulthood and severely impacts life expectancy. Based on the strength of zilurgisertib's PROGRESS study conducted by Incyte, an NDA has been accepted with priority review with a PDUFA date of September 26th this year. If approved, we expect to launch by year-end. This is a strong strategic fit aligning with our capabilities in rare genetic disease, where care is concentrated in a small number of specialized centers and requires deep engagement with patients, caregivers, and physicians. Stepping back, we've built a company with multiple commercial growth drivers, a pipeline of meaningful upcoming catalysts, and the financial strength to advance our portfolio independently. This foundation is translating directly into high-impact medicines for patients and into value creation as we deliver on our strategy.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

With that, I'll turn the call over to Peter to walk through the commercial portfolio in preparation for these three upcoming potential launches. Peter?

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Thank you, Chris. The first quarter was another period of strong commercial execution, with total net product sales of approximately $160 million. This included LIVMARLI net product sales of $84 million in the U.S. and $30 million internationally, with the bile acid medicines contributing $46 million. Robust adoption in PFIC, particularly in adult patients, continues to be a strong point for LIVMARLI, as education to increase awareness and recognition of genetic cholestasis among adult liver providers continues to be successful. Additionally, we saw stronger than expected performance in Q1 international LIVMARLI sales, as well as continued new patient adds in Alagille worldwide.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

The bile acid medicines grew in a manner consistent with their cadence over the last several quarters, highlighted by our rare genetics team continuing to identify undiagnosed patients with CTX. Overall, we expect these dynamics to continue and as a result, are raising our full year 2026 net product sales guidance to $660 million-$680 million. As Chris mentioned, we are also beginning to see the next phase of growth in our rare liver disease business take shape. The recent results from the VISTAS study of volixibat and PSC and the AZURE-1 study of brelovitug and hepatitis delta represent important steps in extending our presence into larger, primarily adult liver settings where patients have limited or no approved treatment options.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

These programs build directly on the global commercialization platform we have established for LIVMARLI, CTEXLI, and CHOLBAM, heavily leveraging our existing technologies, people, and infrastructure. We plan to expand our U.S. and international teams starting later this year to reach liver healthcare providers in adult settings, including GIs, liver providers who manage PSC patients and hepatitis delta, as well as other care settings like infectious disease and selected primary care providers where we believe we can increase the number of diagnosed hepatitis delta patients. In the U.S., our current 20-person liver field commercial team reaches about 1,500 healthcare providers with currently focus on pediatric liver providers and some higher volume adult pro-providers. After our planned expansion to approximately 60 U.S. field commercial personnel, we anticipate being able to reach over 4,000 liver healthcare professionals, representing the vast majority of potential prescribers for our rare liver business.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Turning to our rare genetic disease business, we are very excited by the addition of zilurgisertib for the treatment of FOP, where there remains a desperate need for additional treatment options. FOP is a devastating, relentlessly progressive condition in which soft tissues such as muscles, tendons, and ligaments gradually turn into a second skeleton, leading to cumulative loss of mobility, severe disability, and early mortality. FOP is a highly concentrated ultra-rare disease with an estimated prevalence of about one per million, which translates to approximately 300 patients in the U.S. and around 900 patients globally. Patients with FOP are largely managed by specialized tertiary centers, with most of these centers also in the patients currently CTEXLI allowing our rare gene commercial support. We've got data from the real study, secondary endpoint on the basis of validation.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

We look forward to the presentation of the progress at upcoming medical. The zilurgisertib had it for priority by the FDA September 2026. Approved, we use zilurgisertib with several countries in 2027. We had it about the initiative, which we a potential system for our rating business. Peak sales of $200 million. This has been this for medical and capabilities action for support operating from[audio distortion]. With that Eric to review results. [audio distortion]

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals]

Disciplined investment behind our pipeline, which remains on track across all programs. Today, I'll walk you through the financials for the quarter, including an overview of the impact of the Bluejay acquisition and the zilurgisertib transaction. The net product sales for the first quarter were $160 million compared to net product sales of $112 million in the first quarter of last year. Cash, cash equivalents, and investments as of March 31st were $421 million, compared with $391 million at the beginning of the year. In the first quarter, the cash contribution margin from our commercial business was in the mid-50s%, and cash flow from operations was about $2 million. First quarter financials were significantly impacted by one-time expenses related to the acquisition of Bluejay Therapeutics, which closed in January of this year.

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals]

The total net cash outflow related to this acquisition was $253 million, which was offset through net financing proceeds of approximately $260 million. Total operating expense for the quarter ended March 31st was $949 million, which includes $761 million in expense associated with the acquisition of Bluejay, R&D expense of $98 million, SG&A expense of $96 million, and cost of sales of $29 million. Expenses for the quarter included stock-based compensation, intangible amortization, and other non-cash expenses of $64 million, including $35 million of stock-based compensation expense associated with the acquisition of Bluejay. The intangible amortization and other non-cash item expenses are largely reflected in our [audio distortion]. Chris and Peter mentioned we recently entered into an exclusive license agreement with Incyte.

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals]

In return for worldwide rights to the zilurgisertib, Incyte received an upfront payment of $60 million and is eligible to receive additional development and regulatory milestone payments, including $25 million upon U.S. FDA approval for FOP, ownership of a rare pediatric disease priority review voucher, if awarded, as well as sales-based milestones and tiered royalties on worldwide net sales in the mid to high single-digit percent range. As we've discussed previously, we expect R&D expense to step up in 2026 as we invest behind brelovitug ahead of the anticipated BLA submission next year. For example, R&D expense in the first quarter included $21 million related to the development of brelovitug. Importantly, this expense expected increase is fully funded. We are continuing to scale the business with discipline, balancing investment in growth with a strong balance sheet and financial independence.

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals]

This approach positions us to advance our pipeline and execute on upcoming milestones without compromising our long-term financial strength. I'll now turn the call back to Chris for closing remarks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks, Eric. Mirum's in a strong position after a very busy first of the year. What is most encouraging about the quarter is not just the number of positive updates, but how clearly they fit together. We continue to grow our commercial medicines. We're expanding our rare liver business into larger indications. We've added what we believe is a transformational medicine to our rare genetic business. Importantly, this is all coming together within a high-impact scalable business model. We're excited about the progress ahead as we approach multiple pivotal readouts, potential regulatory submissions, and potential new product launches. I'd also like to thank the entire Mirum team for all the hard work in getting us where we are today. Your dedication brings new treatment options to patients around the world. With that, operator, please open the call for questions.

[Operator]

We will now begin the question-and-answer session. If you would like to ask a question, please press star one to raise your hand. To withdraw your question, press star one again. We ask that you pick up your handset when asking a question to allow for optimum sound quality. If you are muted locally, please remember to unmute your device. Please stand by now while we compile the Q&A roster. Your first question comes from the line of Gavin Clark-Gartner with Evercore ISI. Your line is open. Please go ahead.

[Yesha Patel, Analyst at Evercore ISI]

Hi, this is Yesha Patel on for Gavin. I just had one on FOP. Wondering your current view on the number of diagnosed FOP patients in the U.S. based on claims, patient advocacy, and provider research. How many of those patients will immediately be treatable at launch? I have one follow-up on LIVMARLI.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah, thanks for the questions. You know, we point towards the approximately 300 identified patients in the U.S., coming from a patient advocacy group, IFOPA. In terms of addressable patients, you know, probably the main feature there is the NDA application Incyte filed is for age 12 and over. That'd be the main piece to consider, which would be the majority of the relevant patients.

[Yesha Patel, Analyst at Evercore ISI]

In terms of LIVMARLI, specifically on the guidance raise, wondering what's kind of driving the bulk of that increase. Is it due to the ex-U.S. expansion or kind of the continued PFIC ramp? Within PFIC, is the contribution still skewing towards those older patients?

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. Thanks for the question. I mean, certainly LIVMARLI U.S. PFIC was the biggest driver, and continue to see both pediatric and adult patients come to treatment. I think the, you know, the older adolescents and adults really is the major driver, although we're still, we think, still in early innings. We've made good progress educating adult providers on genetic testing, but probably still the minority of them are actually doing that. I think there's probably more adult patients to find out there could potentially benefit from LIVMARLI. Just on the international piece, you know, in Q1, historically, we've seen a bit more seasonality and a little bit of a softer number in Q1, and that just wasn't as much of a factor this year.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Somewhat also in part of not only additional countries performing, but also PFIC starting to show up in that international number.

[Yesha Patel, Analyst at Evercore ISI]

Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

Your next call comes from the line of Josh Schimmer with Cantor. Your line is open.

[Josh Schimmer, Analyst at Cantor Fitzgerald]

Great.

[Operator]

Please go ahead.

[Josh Schimmer, Analyst at Cantor Fitzgerald]

Great. Thanks so much for taking the questions. Also on the zilurgisertib, how are you thinking about its differentiation versus maybe some of the other programs in development, garetosmab, if I'm pronouncing that right? SOHONOS, that's number one. Number two, are you planning to explore the program in other ossification indications, disorders? Number three, I think I heard you say peak sales for the asset of $200 million. Is that global or U.S.? Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks, Josh, for the questions. Yeah, just to clarify the, that peak 200+ is a global number for us. In, in terms of, kind of positioning here, you know, the programs that you covered there, those are the ones that we're tracking. SOHONOS being approved.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

The other program being in registration phase. For SOHONOS, you know, the data coming out of the PROGRESS study here for zilurgisertib is a real step forward in terms of the overall activity profile and tolerability and safety profile. We see the clinical data here being a quite meaningful advance on what's currently available in the market, which has quite a few limitations to it. Compared to the pipeline, this is an oral, which we see as a big advantage, and particularly in the setting where you can potentially drive ossifications from injections and some of these other interventions. Having an oral we see as a nice differentiator for the program.

[Josh Schimmer, Analyst at Cantor Fitzgerald]

Got it. Plans for other ossification disorders?

[Chris Peetz, CEO at Mirum Pharmaceuticals]

You know, It's still early days in thinking about it. I, you know, at this point wanna stay very focused on getting this launched for FOP. It's certainly something we'll consider as we get further down the road.

[Josh Schimmer, Analyst at Cantor Fitzgerald]

Okay. Thanks very much.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

Your next call comes from the line of Jon Wolleben with Citizens. Your line is open. Please go ahead.

[Jon Wolleben, Analyst at Citizens]

Hey. Hey, thanks for taking the question. You know, little unusual having something under review where I haven't seen any of the data for, just wondering what you guys have been privy to to make you comfortable with this acquisition, and then, you know, what would be the form for it to make sense to get this out into the public domain?

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah, thanks for the question, Jon, and fully appreciate the uniqueness of the situation. You know, in our review, This is a conversation that actually has been going on for quite some time, as is typical for a license transaction like this. We've had full access to clinical data, to the regulatory correspondence, and the NDA. We feel quite confident in the profile for zilurgisertib and where they're at in the regulatory process. From the Incyte side, where they've done a fantastic job putting together this program, and saw it, you know, fitting better in a rare disease company like Mirum. You know, the work they've done on it is quite strong. They want to have the data presented first at a medical meeting.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

We're hopeful that's happening relatively soon. Once we get that presented, we'll be able to share more on the pivotal data and overall product profile.

[Jon Wolleben, Analyst at Citizens]

Will you guys be eligible for a pediatric review voucher if approved?

[Chris Peetz, CEO at Mirum Pharmaceuticals]

We do expect this to be eligible for a voucher. Under the terms of the agreement, Incyte will keep that voucher, and we will launch.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

the product. The way operationally this works is Incyte is kind of, given they're mid-stride with the filing and review, they'll complete the primary role through approval, then we'll take over sponsorship at the point of U.S. approval.

[Jon Wolleben, Analyst at Citizens]

Got it. Okay. Thanks, Chris.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. Thanks for the questions.

[Operator]

Your next question comes from the line of Mike Ulz with Morgan Stanley. Your line is open. Please go ahead.

[Analyst at Morgan Stanley]

Hi, this is Rohit on for Mike. Thanks for taking our questions. With the recent pipeline acquisitions, can you talk about how you're thinking about BD moving forward? Also, can you just talk about how you're thinking about pricing in FOP? Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. I can start and I'll hand it over to Peter. You know, I think, as you've seen now from Mirum, kind of over the history of the company, we see a priority in staying active on the BD front. That's how you find unique opportunities that fit and add value to the company. We will continue to work to find good programs to bring into the team. Maybe have Peter kinda touch on any more on that or the second part of the question.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Yeah, I mean, nothing more on the BD strategy on the zilurgisertib. Pricing, obviously we'll make a decision and communicate that, you know, closer to approval. Just, you know, thinking about a U.S., I mean, you can look at the Niemann-Pick C products and other ultra-rare settings like that where you have a strong value proposition and similar ASP is probably get you in the ballpark.

[Analyst at Morgan Stanley]

Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the question.

[Operator]

Your next question comes from the line of James Condulis with Stifel. Your line is open. Please go ahead.

[James Condulis, Analyst at Stifel]

Hey, thanks for taking my question, and congrats on the order. You know, maybe one follow-up on HDV. I think, you know, where I've we've heard a couple questions is maybe in the 900 mg monthly arm, specifically as it relates to, you know, the TND virologic response is maybe a little bit of an outlier relative to, like, some of your prior data and other, you know, the rest of your sort of dataset. Just, like, curious your perspectives here and, you know, as you think about the commercial opportunity here. For docs in the real world, what do you think is kind of the most important measure as it relates to, you know, evaluating efficacy for these different drugs?

[James Condulis, Analyst at Stifel]

Is it that, you know, TND virologic response, other measures of virologic response, the composite? You know, just curious your perspective there. Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. Thanks for the question. I'll maybe make a couple comments then have Joanne speak to some of the data that we're seeing out of the AZURE-1 phase IIb portion. I think in terms of what we're, you know, focused on and what we think is most relevant for ultimate use and driving adoption here is that composite of virologic response and ALT normalization. Those two factors are really what's pointed to in the FDA guidance and shows that you're not only addressing the viral load, but you're also addressing the liver inflammation that is part of the disease. Seeing both of those move means you're kind of going after both components of both of the considerations for both the infection and the liver.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Maybe I'll ask Joanne to touch on the data we've seen to date.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals]

Yeah. You know, Chris was talking about the composites, you know, all very true. When we look at the, you know, curves in terms of the virologic response, we do see declines in everyone. You know, when you structure the endpoint, if you don't meet a certain point by week 24, then you're either on one side of the line or the other. We do see decreases in all of the patients. There's certainly no evidence of lack of response or resistance or anything like that. Really, you know, partly it's an artifact of time. We do see deepening response with continued treatment. You know, and again, this is a fairly small, numerically fairly small group.

[Joanne Quan, Chief Medical Officer at Mirum Pharmaceuticals]

We'll have a lot more information with the full AZURE-1 and AZURE-4 phase III datasets to make, you know, to make a final call on that.

[James Condulis, Analyst at Stifel]

Makes sense. Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the question, James.

[Operator]

Your next question comes from the line of Brian Skorney with Baird. Your line is open. Please go ahead.

[Brian Skorney, Analyst at Baird]

Hey, good afternoon, guys. Thanks for taking my question, and great quarter. I guess I'd love to also ask a question on FOP too. It seems like you're doubling down on making hits in your corporate nemesis. I'm just wondering if you could kind of give your broad thoughts on where you think SOHONOS's profile leaves an opportunity for another entrant and compare and contrast how zilurgisertib might address these. The timeline would put us right around mid-cycle review with the FDA right now. I'm just wondering if you could say if that has already happened or is still pending.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. Thanks for the question. On the review, yes, that has happened, and I'd just say things are tracking as expected. Maybe I'll ask Peter to comment on a little bit more on positioning.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Yeah. I think, certainly the feedback we've heard from, you know, stakeholders, patients, caregivers, physicians, others, available therapy in the market today, you know, there's a lot to be desired, you know, in terms of both efficacy and safety. Yeah, you know, we'll obviously be able to get into more details once we have the PROGRESS data presented at an upcoming medical conference. You know, from what we've seen in our review, there's a more conservative profile, really exciting about, you know, what it can mean for these patients, both efficacy-wise as well as a convenient oral and well-tolerated regimen.

[Brian Skorney, Analyst at Baird]

Great. Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

Your next question comes from the line of Lisa Walter with RBC Capital. Your line is open. Please go ahead.

[Lisa Walter, Analyst at RBC Capital Markets]

Oh, great. Thanks so much for taking our question. Maybe just some more details you can share on the opportunity for FOP. Are there any overlap with your current call points? Did you disclose the deal terms with Incyte? Maybe just one more. You know, given the recent positive results in HDV and PSC, wondering if this has impacted your thinking on when Mirum could become a profitable company. Thanks so much for taking our questions.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. Maybe I'll ask Peter to kinda speak to the FOP points and then have Eric kinda give some financial forward-looking views.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Yeah. Great over-overlap with our existing team, our rare genetics team that's focused on CTEXLI, CHOLBAM. We mentioned that, you know, the majority, the significant majority of the FOP, patients are cared for in settings, centers that also prescribe CTEXLI and CHOLBAM. Different prescribers most of the time. Some overlap in medical genetics. FOP has, you know, the biggest prescriber there will be endocrinologists. That's a new kind of position target. The center overlap for the is really high with our existing, rare genetics business. Excited about adding this product to that team.

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals]

Yeah. Then on the financials, we did disclose the upfront license fee was $16 million, then the next milestone would be $25 million upon FDA approval. There's some other commercial milestones, also a royalty in a low to mid-to-high single-digit range. We expect after launch that this product will be accretive very, very quickly. Your question was also about path to profitability, and I think that's much more driven by brelovitug and volixibat, as well as our current commercial business. As we've said, we're spending a lot on R&D this year for both of those products. Profitability will be, you know, pushed out probably till 2028 on the GAAP basis.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Would reiterate that, you know, expect to be operating cash flow positive next year.

[Eric Bjerkholt, CFO at Mirum Pharmaceuticals]

Yes.

[Operator]

Your next question comes from the line of Jessica Fye with JPMorgan. Your line is open. Please go ahead.

[Jessica Fye, Analyst at JPMorgan]

Hey, guys. Good afternoon. Thanks for taking my question. Can you estimate the contribution to 1st quarter LIVMARLI sales from Alagille syndrome versus PFIC? Another one, FOP, just thinking about that market, what do you see as the penetration for palovarotene? Would you envision the ALK2 being used in combination with that drug? Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions. I mean, just briefly on LIVMARLI, you know, we typically aren't breaking out by indication, but what we can say is that PFIC is They're both growing, but PFIC is the bigger growth driver. Maybe pass the FOP question over to Peter.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Yeah. When we look at the, you know, the U.S., the market where this medicine's available, CHOLBAM, it's probably a minority of diagnosed patients that are currently receiving it. We can see from pharmacy claims data, and that kinda, you know, matches what we've heard in physician and caregiver interviews. It, you know, it can be tried, but it can often be difficult to tolerate and stay on.

[Jessica Fye, Analyst at JPMorgan]

Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the question.

[Operator]

Your next question comes from the line of Mani Foroohar with Leerink Partners. Your line is open. Please go ahead.

[Analyst at Leerink Partners]

Hey, guys. You have Ryan on for Mani. Thanks for taking our question, and congrats on the quarter. Maybe just circling back to FOP, what's the latest thinking here on an OUS filing and when you guys would expect to launch there? Then just going back to the peak sales of $200 million, we're just wondering, is that in the 12-plus age group that you guys would get approved in the upcoming? How should we think about upcoming data for the younger age groups that are being tested? Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions, Ryan. On OUS strategy, actually, a European filing is upcoming, so we could actually still have that in this quarter. Incyte is still driving those activities. You know, again, their team's doing a great job. In terms of the overall, you know, peak estimate, that $200 million-plus, that's the full brand in FOP over life cycles, the way we look at it. The younger age patients, you know, we do expect that the label would launch with the 12 and older. There are two other cohorts in the study that are ongoing that would support potentially taking that age lower over the near term, frankly. Those are ongoing enrolling now, they're not too far out.

[Analyst at Leerink Partners]

Awesome. Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Great. Thanks for the questions.

[Operator]

Your next question comes from the line of Ryan Deschner with Raymond James. Your line is open. Please go ahead.

[Ryan Deschner, Analyst at Raymond James]

Thanks, thanks for the question. A couple from me. You know, what's your strategy for identifying FOP patients in the U.S. and abroad and addressing the relatively high misdiagnosis rate for FOP? Do you anticipate any early line of sight into a substantial group of patients from Incyte's prior clinical studies or maybe a compassionate use program or something like that in FOP? I have a follow-up. Thanks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah. Thanks for the question, Ryan. Yeah. The, you know, I think the FOP patients often have a longer diagnostic odyssey than they should. You know, there are patients who get diagnosed at birth, but often the I think the literature says the average age of diagnosis is seven years, and so obviously people have waited longer than that. I think that's improved with availability of genetic testing. You know, that's, I think, an opportunity to continue to raise awareness, just like all of our rare genetic diseases, to try to shorten that diagnostic odyssey as much as we can. We do think in the U.S., though, most of the, you know, a pretty substantial majority of the patients are identified with FOP.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Probably a different story, in, you know, middle and lower income countries. Was there a second question? Hey, did you have a follow-up, Ryan?

[Ryan Deschner, Analyst at Raymond James]

Just was also wondering if there was anything notable so far in the VISTAS extension in terms of rollover discontinuation rates, pruritus or other patient metrics that might take a little longer to modulate over time.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Yeah, I mean, Incyte does have a, you know, this PROGRESS study. You know, it's, you know, it's enrolling well, and I think we'll be able to disclose more about what they've seen from that at the upcoming medical conference. Have certainly seen a lot of physician and patient interest in the program.

[Ryan Deschner, Analyst at Raymond James]

Okay. Thanks very much.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

Your next question comes from the line of Joseph Thome with TD Cowen. Your line is open.

[Joseph Thome, Analyst at TD Cowen]

Hi there.

[Operator]

Please-

[Joseph Thome, Analyst at TD Cowen]

Hi. Hi there. Good afternoon. Thank you for taking my questions. Maybe one on FOP. I guess the level of ALK2 inhibition you're seeing with the therapy, I guess, do you think that could be enhanced by garetosmab, Regeneron's Activin A drug, or are these largely just gonna be competitive therapeutics in the landscape? Second, when we think about the potential expansion opportunity for LIVMARLI in the basket trial that's gonna be reading out later this year, I guess how should we think about that in your overall projection for LIVMARLI? How much is this basket population? Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

If I can make a quick comment on the garetosmab positioning. You know, it's probably best to get into more detail on this after our data is presented, so we can give a better, a more complete picture. We think the profile for zilurgisertib and its clinical positioning is really strong as a convenient oral single agent, and excited about bringing that forward. Maybe if Peter could take the follow-up.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Yeah. No, you know, we've kind of talked about that indication, being about a third of the at least $1 billion peak sales, opportunity for LIVMARLI. You know, still reiterate that. Thank you.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the questions.

[Operator]

Your next question comes from the line of Charles Wallace with HCW. Your line is open. Please go ahead.

[Charles Wallace, Analyst at H.C. Wainwright & Co.]

Hi. This is Charles. I'm on for RK. Thanks for taking my question. For FOP, another question on that. How many patients from the PROGRESS study, I think there was 63 in that study, do you expect will could come on after launch? Do you expect to have some sort of bridging program?

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks for the question, Charles. You know, given the kind of the nature of the relationship here, we're gonna wait until we have that data presented to give some of the detail on it. Overall, I think it's a really compelling profile, and the feedback has been positive. Just don't wanna get into specifics ahead of having that data presented.

[Charles Wallace, Analyst at H.C. Wainwright & Co.]

Okay. That's fair. I guess another question on the salesforce expansion. You're growing it to 60 in the field. I just wanted to maybe get a little more color on when you expect these the team to be fully on board and also when do you expect them to be fully functional? Thank you.

[Peter Radovich, President and COO at Mirum Pharmaceuticals]

Yeah. you know, I kind of prepared a comment with starting this year, later this year. I kind of think, you know, early next year we'd be kind of fully on board and that team would, you know, cover both pediatric and adult settings where, you know, not just Alagille, and adult PFIC can be found, but also, you know, PSC and Delta. I think by early next year they'd be active in all those areas. Of course, with the pipeline products, the activity would really start upon potential FDA approval.

[Charles Wallace, Analyst at H.C. Wainwright & Co.]

Great. Thanks for taking my questions, and congrats on a great quarter.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

Thanks. Yeah, thanks for the questions.

[Operator]

There are no further questions at this time. I would now like to turn the call back to Chris Peetz for closing remarks.

[Chris Peetz, CEO at Mirum Pharmaceuticals]

All right. Well, thank you all for joining us today and for all the support and a great start to 2026. Have a great afternoon.

[Operator]

This concludes today's call. Thank you for attending. You may now disconnect.

Participants

Executives

• Andrew McKibben, SVP of Strategic Finance and Investor Relations
• Chris Peetz, CEO
• Eric Bjerkholt, CFO
• Joanne Quan, Chief Medical Officer
• Peter Radovich, President and COO

Analysts

• Brian Skorney, Analyst at Baird
• Charles Wallace, Analyst at H.C. Wainwright & Co.
• James Condulis, Analyst at Stifel
• Jessica Fye, Analyst at JPMorgan
• Jon Wolleben, Analyst at Citizens
• Joseph Thome, Analyst at TD Cowen
• Josh Schimmer, Analyst at Cantor Fitzgerald
• Lisa Walter, Analyst at RBC Capital Markets
• Ryan Deschner, Analyst at Raymond James
• Yesha Patel, Analyst at Evercore ISI
• Analyst at Leerink Partners
• Analyst at Morgan Stanley