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 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 and financial guidance. 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?

Thanks, Andrew, and good afternoon, everyone. We have a number of important updates to cover today, but 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, and today, we are a broader rare disease company with three approved medicines and a pipeline positioned to deliver multiple new therapies over the next two years.

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.

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 to $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.

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 2b portion of the AZUR-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 AZUR at EASL later this month. Now, 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 zolurgisertib, recently licensed from Incyte.

Zolurgisertib 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. The 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 zolurgisertib'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 with 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. With that, I'll turn the call over to Peter to walk through the commercial portfolio and preparation for these three upcoming potential launches.

Peter?

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.

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 to $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 in PSC and the AZUR-1 study of brelovitug in hepatitis delta represent important steps in extending our presence into larger, primarily adult liver settings, where patients have limited or no approved treatment options.

These programs build directly on the global commercialization platform we have established for Livmarli, Chenodal, 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 GI 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 liver 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.

Turning to our rare genetic disease business, we are very excited by the addition of zolurgisertib 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 1 per million, which translates to approximately 300 patients in the United States and around 900 patients globally. Patients with FOP are largely managed by specialized tertiary centers, with most of these centers also seeing patients for Chenodal and Cholbam, aligning to our rare genetic commercial footprint.

We've evaluated data from the PROGRESS study [unclear] on the basis of [adult?] patients.

Look forward to the broader data from the PROGRESS upcoming medical [unclear]. Zolurgisertib has priority review by the FDA September 26, 2026.

If approved, we expect to launch zolurgisertib by the [unclear] with expand to [all?] countries [in?]

2027. We are excited about the addition of zolurgisertib, which we estimate peak sales of $200 million.

This business fits our medical and commercial capabilities, passion for support offered for folks who live with FOP. With that, Eric will review our results.

Thank you, Peter. Q1 was another strong quarter with continued momentum in our commercial [unclear] and

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 impacts of the Bluejay acquisition and the zolurgisertib 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 percent, 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.

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 non-GAAP results.

As Chris and Peter mentioned, we recently entered into an exclusive license agreement with Incyte. In return for worldwide rights to zolurgisertib, 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 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. 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.

Thanks, Eric. Eric is in a strong position after a very busy start to 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.

Hi, this is Yesha 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, and then how many of those patients will immediately be treatable at launch? Then I have one follow-up on Livmarli.

Thanks for the questions. We point towards the approximately 300 identified patients in the U.S.

coming from the patient advocacy group IFOPA. In terms of addressable patients, probably the main feature there is the NDA application Incyte filed is for age 12 and over, so that'd be the main piece to consider, which would be the majority of the prevalent patients.

Then

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, and within PFIC, is the contribution still skewing towards those older patients?

Thanks for the question. Certainly Livmarli U.S.

PFIC was the biggest driver, and continue to see both pediatric and adult patients come to treatment. I think 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 that potentially benefit from Livmarli.

Just on the international piece, 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, somewhat also in part of not only additional countries performing, but also PFIC starting to show up in that international number.

Thank you.

Thanks for the questions.

Great. Thanks so much for taking the questions.

Also on zolurgisertib, how are you thinking about its differentiation versus maybe some of the other programs in development, garetosmab, if I'm pronouncing that right, and Sohonos? That's number one.

Number two, are you planning to explore the program in other ossification disorders? Then, number three, I think I heard you say peak sales for the asset of $200 million.

Is that global or U.S.? Thank you.

Thanks, Josh, for the questions. Just to clarify, that 200-plus is a global number for us.

In terms of kind of positioning here, the programs that you covered there, those are the ones that we're tracking: Sohonos being approved, and the other program being in registration phase. For Sohonos, the data coming out of the PROGRESS study here for zolurgisertib is a real step forward in terms of the overall activity profile, tolerability, and safety profile.

We see the clinical data here being quite meaningful advance on what's currently available in the market, which has quite a few limitations to it. Then, compared to the pipeline, this is an oral, which we see as a big advantage, particularly in this 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.

Got it. Then plans for other ossification disorders?

We're still early days in thinking about it. At this point, want to stay very focused on getting this launched for FOP, but it's certainly something we'll consider as we get further down the road.

Thanks very much.

Thanks for the questions.

Hey, thanks for taking the question. A little unusual having something under review where we 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, what would be the forum for it to make sense to get this out into the public domain?

Thanks for the question, Jon. Fully appreciate the uniqueness of the situation.

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 zolurgisertib 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, fitting better in a rare disease company like Mirum, the work they've done on it is quite strong.

They want to have the data presented first at a medical meeting. 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.

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

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 the product.

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

Got it. Okay.

Thanks, Chris.

Yep, thanks for the questions.

Hi, this is Rohan 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? Then also, can you just talk about how you're thinking about pricing in FOP?

Thanks.

I can start, then I'll hand it over to Peter. I think as you've seen now through 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 kind of touch on anything more on that, or the second part of the question.

Nothing more on the the BD strategy. On the zolurgisertib pricing, obviously, we'll make a a decision and communicate that closer to approval.

Just, I think we're thinking about a U.S., you can look at the Niemann-Pick C products, other ultra-rare settings like that where you have a strong value proposition and similar [RP?] is probably get you in the ballpark.

Thank you.

Thanks for the questions.

Hey, thanks for taking my question, and congrats on the quarter. Maybe one follow-up on HDV.

I think where we've heard a couple questions is maybe in that 900 mg monthly arm, specifically as it relates to TND virologic response is maybe a little bit of an outlier relative to some of your prior data and the rest of your sort of data set. Just curious your perspective here, and 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 evaluating efficacy for these different drugs?

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

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 AZUR-1 Phase 2b portion.

I think in terms of what we're focused on, 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. Maybe I'll ask Joanne to touch on the data we've seen to date.

Yeah. Chris was talking about the composite.

All very true. When we look at the curves in terms of the virologic response, we do see declines in everyone.

When you structure the endpoint, if you don't meet a certain point by week 24, then you're either on one side of line or the other. We do see decreases in all of the patients.

Certainly no evidence of lack of response or resistance or anything like that. Really, partly it's an artifact of time.

We do see deepening response with continued treatment. Again, this is a fairly small, numerically fairly small group.

We'll have a lot more information with the full AZUR-1 and AZUR-4 Phase 3 datasets to make a final call on that.

Makes sense. Thank you.

Thanks for the questions, James.

Good afternoon, guys. Thanks for taking the 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 Ipsen your corporate nemesis.

I'm just wondering if you could kind of give your broad thoughts on where you think Sohonos' profile leaves an opportunity for another entrant, and compare and contrast how zolurgisertib 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?

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 comments on a little bit more on positioning.

I think, certainly the feedback we've heard from stakeholders, patients, caregivers, physicians, others, with the available therapy in the market today, there's just a lot to be desired in terms of both efficacy and safety. We'll love to be able to get into more details once we have the progress data presented at upcoming medical conference.

From what we've seen in our review, the zolurgisertib profile really exciting about what it could mean for these patients, both efficacy-wise as well as a convenient, oral, well-tolerated regimen.

Great. Thank you.

Thanks for the questions.

Oh, great. Thanks so much for taking our question.

Maybe just some more detail if you can share on the opportunity for FOP. Are there any overlap with your current call points, and did you disclose the deal terms with Incyte?

Maybe just one more, given the recent positive results in hep D and PSC, wondering if this has impacted your thinking on when Mirum could become a profitable company? Thanks so much for taking our questions.

Maybe I'll ask Peter to kind of speak to the FOP points, then have Eric kind of give some financial forward-looking views.

Great overlap with our existing team, our rare genetics team that's focused on Chenodal, Cholbam. We mentioned that the significant majority of the FOP patients are cared for in centers that also prescribe Chenodal and Cholbam.

Different prescribers most of the time, some overlap in biochemical genetics. The biggest prescriber there will be endocrinologists, so that's a new kind of physician target, but the center overlap is really high with our existing rare genetics business.

Excited about adding this product to that team.

Then on the financials, we did disclose the upfront license fee was $60 million, and then the next milestone would be $25 million upon FDA approval. There's some other commercial milestones, but also a royalty in 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, and so profitability will be pushed out probably till '28 on a GAAP basis.

Would reiterate that we expect to be operating cash flow positive next year.

Yes.

Hey guys, good afternoon. Thanks for taking my question.

Can you estimate the contribution to first quarter Livmarli sales from Alagille versus PFIC? Then, another one on FOP, just thinking about that market, what do you see as the penetration for palovarotene, and would you envision the ALK being used in combination with that drug?

Thanks.

Thanks for the questions. Just briefly on Livmarli, we're typically aren't breaking out by indication, but what we can say is that they're both growing, but PFIC is the bigger growth driver.

Maybe pass the FOP question over to Peter.

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

Thank you.

Thanks for the questions.

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 ex-U.S. 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 in the upcoming, and how should we think about upcoming data for the younger age groups that are being tested? Thanks.

Thanks for the questions, Ryan. On ex-U.S.

strategy, actually, a European filing is upcoming, we could actually still have that in this quarter. Incyte is still driving those activities, and, again, their team's doing a great job.

In terms of the overall 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, 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 and enrolling now, they're not too far out.

Awesome. Thanks.

Great. Thanks for the questions.

Thanks for the question. A couple for me.

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, and I have a follow-up. Thanks.

Thanks for the question, Ryan. I think that FOP patients often have a longer diagnostic odyssey than they should.

There are patients get diagnosed at birth, but often I think the literature says average age of diagnosis is 7 years, and it's obviously people that wait longer than that. I think that's improved with availability of genetic testing, but 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, pretty substantial majority of the patients are identified with FOP, probably different story in middle- and lower-income countries. What's your second question?

Hey, do you have a follow-up, Ryan?

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

Incyte does have this PROGRESS study. It's been rolling well, and I think we'll be able to disclose more about what they've seen from that at the upcoming medical conference, but have certainly seen a lot of physician and patient interest in the program.

Thanks very much. Great.

Thanks for the questions.

go ahead. 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, do you think that could be enhanced by garetosmab, Regeneron's activin A drug, or are these largely just going to be competitive therapeutics in the landscape?

Then, second, when we think about the potential expansion opportunity for Livmarli in the basket trial that's going to 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.

I can make a quick comment on the garetosmab positioning. It probably best to get into more detail on this after our data is presented so we can give a more complete picture.

We think the profile for zolurgisertib in its clinical positioning is really strong as a convenient oral single agent and excited about bringing that forward. Maybe have Peter take the follow-up.

On expand, we've kind of talked about that indication being about a third of the at least $1 billion peak sales opportunity for Livmarli, and still reiterate that. Thank you.

Thanks for the questions.

Hi, this is Charles 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 could come on after launch, and do you expect to have some sort of bridging program?

Thanks for the question, Charles. Given the kind of the nature of the relationship here, we're going to 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, but just don't want to get into specifics ahead of having that data presented.

Fair. Then, I guess another question, if I may, on the sales force expansion.

You're growing it to 60 in the field, and I just wanted to maybe get a little more color on when you expect the team to be fully on board, and also when do you expect them to be fully functional? Thank you.

Yeah. Kind of prepared comment was starting this later this year, and so I kind of think, early next year, we'd be kind of fully on board.

That team would cover both pediatric and adult settings where, not just Alagille, in adult, PFIC can be found, but also 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.

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

Thanks. Thanks for the questions.

Great. Thank you all for joining us today and for all the support in a great start to 2026.

Have a great afternoon.