Q1 2026 Mirum Pharmaceuticals Inc Earnings Call

May 6, 2026

Operator 3: Welcome to Mirum Pharmaceuticals' Q1 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.

Operator: Welcome to Mirum Pharmaceuticals' Q1 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.

Speaker #3: 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 call and the conference over to Andrew McKibben, SVP of Strategic Finance and Investor Relations. Please go ahead.

Speaker #2: 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.

Andrew McKibben: Thank you, Tracy, and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals' Q1 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 Q1 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.

Speaker #2: 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.

Speaker #2: 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 improved medicines and product candidates and financial guidance.

Speaker #2: 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.

Andrew McKibben: 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.

Speaker #2: We are under no duty to update these statements. Please refer to the risk factors in our latest form, 10Q, and subsequent SEC filings for more information about these risks and uncertainties.

Speaker #2: With that said, I'd like to turn the call over to Chris. Chris?

Speaker #3: 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.

Chris Peetz: 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 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.

Speaker #3: 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.

Speaker #3: Our start was based on Liv Marley, and today we are a broader rare disease company with three approved medicines and a pipeline position to deliver multiple new therapies over the next two years.

Speaker #3: 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.

Speaker #3: 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.

Chris Peetz: 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.

Speaker #3: Starting with rare liver disease, uptake of Liv Marley 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.

Speaker #3: 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.

Speaker #3: Extending beyond our pediatric foundation into larger patient populations with significant unmet need. In PSC, the VISTA study of elixivat showed a significant improvement in pruritus, reinforcing the potential for elixivat to play an important role for these patients who currently have no approved medicines.

Speaker #3: This is a major advance in PSC research and positions elixivat as a potential first approved medicine for patients in the US. And in hepatitis delta, results from the phase 2b portion of the ARE-1 study further support the potential for Brilovite in a patient population where treatment options are extremely limited.

Chris Peetz: This is a major advance in PSC research and positions volixibat as a potential first approved medicine for patients in the US. In hepatitis delta, results from the Phase 2B 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. 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 Zulugacertib, recently licensed from Incyte. Zulugacertib 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.

Speaker #3: We look forward to the upcoming late-breaking presentations for both VISTAs and Azure at ESIL 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 Zelirgecerted, recently licensed from Insight.

Speaker #3: Zelirgecerted is a once-daily oral ALK2 inhibitor in development for fibrodysplasia ossificans progressiva, or FOP. An ultra-rare progressive condition where patients develop bone and soft tissues.

Speaker #3: 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.

Chris Peetz: This accumulation of excess bone leads to profound physical immobilization, with most FOP patients becoming wheelchair-dependent by early adulthood and severely impact life expectancy. Based on the strength of Zulugacertib'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.

Speaker #3: Based on the strength of Zelirgecerted's progress study conducted by Insight, an NDA has been accepted with priority review, with a PDUFA date of September 26th this year.

Speaker #3: 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.

Speaker #3: Stepping back, we've built the company with multiple commercial growth drivers, a pipeline of meaningful upcoming catalysts, and the financial strength to advance our portfolio independently.

Speaker #3: This foundation is translating directly into high-impact medicines for patients and into value creation as we deliver on our strategy. And with that, I'll turn the call over to Peter to walk through the commercial portfolio and preparation for the three upcoming potential launches.

Chris Peetz: With that, I'll turn the call over to Peter to walk through the commercial portfolio in preparation for these 3 upcoming potential launches. Peter?

Speaker #3: Peter?

Speaker #2: Thank you, Chris. The first quarter was another period of strong commercial execution with total net product sales of approximately $160 million. This included Liv Marley net product sales of $84 million in the US and $30 million internationally, with the bioasset medicines contributing 46 million.

Peter Radovich: Thank you, Chris. The Q1 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 US 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.

Speaker #2: 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.

Speaker #2: Additionally, we saw a stronger-than-expected performance in Q1 international Liv Marley sales as well as continued new patient ads and allogene worldwide. The bioasset 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.

Speaker #2: 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.

Peter Radovich: 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 VISTA 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. 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.

Speaker #2: And 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 VISTA study of elixivat and PSC in the Azure One study of Brilovite 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.

Speaker #2: These programs build directly on a global commercialization platform we have established for Liv Marley, Citexli, and Colvam, heavily leveraging our existing technologies, people, and infrastructure.

Speaker #2: We plan to expand our US 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.

Peter Radovich: We plan to expand our US and international teams starting later this year to reach liver health care 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 US, 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 providers. After our planned expansion to approximately 60 US 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.

Speaker #2: In the US, our current 20-person liver field commercial team reaches about 1,500 healthcare providers with currently focused on pediatric liver providers and some higher volume adult providers.

Speaker #2: After our planned expansion to approximately 60 US 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.

Speaker #2: Turning to our rare genetic disease business, we are very excited by the addition of Zelirgecerted for the treatment of FOP, where there remains a desperate need for additional treatment options.

Peter Radovich: Turning to our rare genetic disease business, we are very excited by the addition of Zulugacertib 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 US and around 900 patients globally. Patients with FOP are largely managed by specialized tertiary centers, with most of these centers also on the patients currently on CTEXLI and Alagille are rare gene commercial We've got data from the MIDAS study backing Gary and want the basis of the application.

Speaker #2: 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.

Speaker #2: 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 United States and around 900 patients globally.

Speaker #2: Patients with FOP are largely managed by specialized tertiary centers with most of these centers also in the patients' current Citexli allowing our rare genetic commercial circle.

Speaker #2: We've got the data from the Mirum study impacting dairy endpoints of basis of application. We look forward to the president from the progress upcoming medical.

Peter Radovich: We look forward to the precedent from the PROGRESS upcoming medical. The Zulugacertib had it for priority by the FDA 20 September 2026 date. Approved, we expect Zulugacertib to be ready with expanded countries in 2027. We are excited about the addition of Zulugacertib, which we believe has shown first step for our rare business, piece of $200 million. This business

Speaker #2: The Zelirgecerted added for priority by the FDA September 2026. Approved, we use Zelirgecerted by the. With expanding countries in 2027. We added about the addition to which we have potential system for raising business P step 200 million business in this medical and capability attaching for support offering from Ultra like FOP.

Chris Peetz: capability, passion, and personal outpouring from all of you has led to that vote. With that, Eric, your view of results.

Peter Radovich: capability, passion, and personal outpouring from all of you has led to that vote. With that, Eric, your view of results.

Speaker #2: And with that, Eric, to review results.

Eric Bjerkholt: Yeah, an idea that one. Strong year with continued growth in the commercial, our commercial model, 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 impact of the Blue Jay acquisition and the Delirgy Therapeutic transaction. The net product sales for Q1 were $160 million compared to net product sales of $112 million in Q1 of last year. Cash, cash equivalents, and investments as of 31 March were $421 million, compared with $391 million at the beginning of the year. In Q1, the cash contribution margin from our commercial business was in the mid-50s%, and cash flow from operations was about $2 million.

Speaker #3: Yeah, Eric. Strong year with continuing in the commercial 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 impact of the BlueJ acquisition and the Zelirgecerted transaction.

Speaker #3: 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.

Speaker #3: 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-50 percent range, and cash flow from operations was about $2 million.

Speaker #3: First quarter financials were significantly impacted by one-time expenses related to the acquisition of BlueJ 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.

Eric Bjerkholt: Q1 financials were significantly impacted by one-time expenses related to the acquisition of Blue Jay Therapeutics, which closed in January 2024. 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 31 March was $949 million, which includes $761 million in expense associated with the acquisition of Blue Jay, 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 Blue Jay. The intangible amortization and other non-cash item expenses are largely reflected in our comprehensive results.

Speaker #3: Total operating expense for the quarter ended March 31st was $949 million, which includes $761 million in expense associated with the acquisition of BlueJ, R&D expense of $98 million, SG&A expense of $96 million, and cost of sales of $29 million.

Speaker #3: 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 BlueJ.

Speaker #3: The intangible amortization and other non-cash item expenses are largely reflected in our compensation total. As Kristen Peter mentioned, we recently entered into an exclusive license agreement with Insight.

Eric Bjerkholt: As Chris and Peter mentioned, we recently entered into an exclusive license agreement with Incyte. In return for worldwide rights to Zulugacertib, Incyte receives an upfront payment of $60 million and is eligible to receive additional development and regulatory milestone payments, including $25 million upon US 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 digits 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 Q1 included $21 million related to the development of brelovitug. Importantly, this expense, expected increase is fully funded.

Speaker #3: In return for worldwide rights to Zelirgecerted, Insight received an upfront payment of $16 million and is eligible to receive additional development and regulatory milestone payments including $25 million upon US FDA approval for FOP ownership of a rare pediatric disease priority review voucher if awarded, as well as sales-based milestones and shared royalties on worldwide net sales in the mid to high single-digit percent range.

Speaker #3: As we've discussed previously, we expect R&D expense to step up in 2026 as we invest behind Berlowitz ahead of the anticipated BLA submission next year.

Speaker #3: For example, R&D expense in the first quarter included $21 million related to the development of Berlowitz. Importantly, this expense, expected to increase, is fully funded.

Speaker #3: 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.

Eric Bjerkholt: 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.

Speaker #3: I'll now turn the call back to Chris for closing remarks.

Speaker #2: Thanks, Eric. Mirum's been 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.

Chris Peetz: Thanks, Eric. Mirum's in a strong position after a very busy 1st 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.

Speaker #2: We continue to grow our commercial medicines. We're expanding our rare business into rare liver business, into larger indications. We've added what we believe is a transformational medicine to our rare genetic business.

Speaker #2: And 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.

Speaker #2: 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.

Speaker #2: And with that, operator, please open the call for questions.

Speaker #1: 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.

Operator 3: We will now begin the question-and-answer session. Your first question comes from the line of Gavin Clark-Gartner with Evercore ISI. Your line is open. Please go ahead.

Operator: We will now begin the question-and-answer session. Your first question comes from the line of Gavin Clark-Gartner with Evercore ISI. Your line is open. Please go ahead.

Speaker #1: 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.

Speaker #1: 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.

Speaker #1: Your line is open. Please go ahead.

Speaker #4: Hi, this is Yasha on for Gavin. I just had one on FOP. Wondering your current view on the number of diagnosed FOP patients in the US based on claims to patient advocacy and provider research, and then how many of those patients will immediately be treatable at launch?

Yesha Patel: 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 US 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 with LIVMARLI.

Speaker #4: And then I have one follow-up. I'm with Marley.

Speaker #2: Yeah. Thanks for the questions. Yeah, we point towards the approximately 300 identified patients in the US coming from patient advocacy group IFOPA. In terms of addressable patients, probably the main feature there is the NDA application inside filed as for age 12 and over.

Chris Peetz: Yeah. Thanks for the questions. Yeah, you know, we point towards the approximately 300 identified patients in the US, coming from patient advocacy group, FOPA. In terms of addressable patients, you know, probably the main feature there is the NDA application Insight filed is for age 12 and over. That'd be the main piece to consider, which would be the majority of the relevant patients.

Speaker #2: So that'd be the main piece to consider, which would be the majority of the prevalent patients.

Speaker #4: And then in terms of those Marley specifically on the guidance series, wondering what's kind of driving the bulk of the increase. Is it due to the XUS expansion or kind of the continued PFIC ramp?

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

Speaker #4: And within PFIC, is the contribution still skewing towards those older patients?

Speaker #2: Yeah. Thanks for the question. I mean, certainly, Liv Marley US PFIC was the biggest driver. And continue to see both pediatric and adult patients come to treatment.

Chris Peetz: Yeah, thanks for the question. I mean, certainly LIVMARLI US 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. Yes, 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. That just wasn't as much of a factor this year.

Speaker #2: 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.

Speaker #2: So I think there's probably more adult patients to find out there potentially benefit from Marley.

Speaker #3: And yes, 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.

Speaker #3: Somewhat in part, not only are additional countries performing, but also PFIC is starting to show up in that international number.

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

Speaker #4: Thank you.

Yesha Patel: Thank you.

Speaker #2: Thanks for the questions.

Chris Peetz: Thanks for the questions.

Speaker #1: Your next call comes from the line of Josh Shimmer, with Cantor. Your line is open. Please go ahead.

Operator 3: Your next call comes from the line of Josh Schimmer with Cantor. Your line is open. Go ahead.

Operator: Your next call comes from the line of Josh Schimmer with Cantor. Your line is open. Go ahead.

Speaker #5: Great. Thanks so much for taking the questions. Also on Zelirgecerted, how are you thinking about its differentiation versus maybe some of the other programs and development, if I'm pronouncing that right, and so who knows, that's number one.

Josh Schimmer: Great. Thanks so much for taking the questions. Also on Zulugacertib, 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 US? Thank you.

Speaker #5: Number two, are you planning to explore the program in other classification indications? So disorders. And then number three, I think I heard you say peak sales for the asset of 200 million.

Speaker #5: Is that global or US? Thank you.

Speaker #2: Thanks, Josh, for the questions. Yeah, just to clarify the peak, the 200 plus is global number for us. And in terms of kind of positioning here, the programs that you covered there, those are the ones that we're tracking in Soho as being approved.

Chris Peetz: Thanks, Josh, for the questions. Yeah, just to clarify that peak, that 200 plus is a global number for us. 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, and the other program being in registration phase. For Sohonos, you know, the data coming out of the PROGRESS study here for Zulugacertib 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.

Speaker #2: And the other program being in registration phase. And for Soho's, the data coming out of the progress study here for Zelirgecerted is a real step forward in terms of the overall activity profile and tolerability and safety profile.

Speaker #2: So 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.

Chris Peetz: 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.

Speaker #2: And then, compared to the pipeline, this is an oral, which we see as a big advantage, particularly in the setting where you can potentially drive ossifications from injections and some of these other interventions.

Speaker #2: So having an oral we see as a nice differentiator for the program.

Speaker #5: Got it. And then plans for other ossification disorders?

Josh Schimmer: Got it. Plans for other ossification disorders?

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

Speaker #3: We're still in the early days of thinking about it. At this point, we 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.

Speaker #5: Okay. Thanks very much.

Josh Schimmer: Okay. Thanks very much.

Speaker #2: Thanks for the questions.

Chris Peetz: Thanks for the question.

Speaker #1: Your next call comes from the line of John Wollenben, with Citizens. Your line is open. Please go ahead.

Operator 3: Your next call comes from the line of Jon Wolleben with Citizens. Your line is open. Please go ahead.

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

Speaker #6: Hey, thanks for taking the question. Little unusual having something under review or haven't seen any of the data for. Just wondering, what do you guys have been privy to to make you comfortable with this acquisition?

Jon Wolleben: Hey, thanks for taking the question. You know, a little unusual having something under review or 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?

Speaker #6: And then what would be the form for it to make sense to get this out into the public domain?

Speaker #3: Yeah, thanks for the question, John. I 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 licensed transaction like this.

Chris Peetz: Yeah. Thanks for the question, John, 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 Zulugacertib 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.

Speaker #3: So we've had full access to clinical data, to the regulatory correspondence, and the NDA. So we feel quite confident in the profile for Zelirgecerted.

Speaker #3: And where they're at in the regulatory process. From the insight side, where they've done a program and saw it fitting better in a rare disease company like Mirum, the work they've done on it is quite strong.

Speaker #3: They want to have the data presented first at a medical meeting, so 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.

Chris Peetz: 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.

Speaker #6: And will you guys be eligible for a pediatric review voucher if approved?

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

Speaker #3: So we do expect this to be eligible for a voucher under the terms of the agreement. Insight will keep that voucher and we will launch the product.

Chris Peetz: We do expect this to be eligible for a voucher. Under the terms of the agreement, Insight will keep that voucher, and we will launch the product. The way operationally this works is Insight is kind of given they're mid-stride with the filing and review. They'll complete the primary role through approval, and then we'll take over sponsorship at the point of US approval.

Speaker #3: So the way operationally this works is, Insight is kind of given they're mid-stride with the filing and review—they'll complete the primary role through approval.

Speaker #3: And then we'll take over sponsorship at the point of US approval.

Speaker #6: Got it. Okay. Thanks, Chris.

Jon Wolleben: Got it. Okay. Thanks, Chris.

Speaker #3: Yep. Thanks for the questions.

Chris Peetz: Yeah. Thanks for the questions.

Speaker #1: Your next question comes from the line of Mike Olds, with Morgan Stanley. Your line is open. Please go ahead.

Operator 3: Your next question comes from the line of Michael Yee with Morgan Stanley. Your line is open. Please go ahead.

Operator: Your next question comes from the line of Michael Yee with Morgan Stanley. Your line is open. Please go ahead.

Speaker #7: 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?

[Analyst] (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? Can you just talk about how you're thinking about pricing in FOP? Thanks.

Speaker #7: And then also, can you just talk about how you're thinking about pricing in FOP? Thanks.

Speaker #3: Yeah, I can start and I'll hand it over to Peter, but I think as you've seen now for Mirum kind of over the history of the company, we see a priority in staying active on the BD front.

Chris Peetz: 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 kind of touch on any more on that or the second part of the question.

Speaker #3: That's how you find unique opportunities that fit and add value to the company. So we will continue to work to find good programs to bring into the team.

Speaker #3: Maybe have Peter kind of touch on that a bit more, or the second part of the question?

Speaker #2: Yeah, I mean, nothing more on the BD strategy on the Zelirgecerted pricing. Obviously, we'll make a decision and communicate that closer to approval. But just I think for thinking about a US, I mean, you can look at the Neiman Pixie products and other ultra-rare settings like that where you have a strong value proposition and similar FD is probably get you in the ballpark.

Peter Radovich: Yeah, I mean, just nothing more on the BD strategy. On the Zulugacertib pricing, obviously we'll make a decision and communicate that, you know, closer to approval. Just, you know, I think if we're thinking about a US, 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 IP is probably get you in the ballpark.

Speaker #7: Thank you.

Ryan McElroy: Thank you.

[Analyst] (Morgan Stanley): Thank you.

Speaker #2: Thanks for the questions.

Chris Peetz: Thanks for the question.

Speaker #1: Your next question comes from the line of James Condulis, with Stifel. Your line is open. Please go ahead.

Operator 3: Your next question comes from the line of James Condulis with Stifel. Your line is open. Please go ahead.

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

Speaker #8: Hey, thanks for taking my question and congrats on the quarter. Maybe one follow-up on HDV. I think where I've we've heard a couple of questions is maybe in the 900-meg monthly arm, specifically as it relates to the TND virologic response, is maybe a little bit of an outlier relative to some of your prior data and other the rest of your sort of data set.

James Condulis: Hey, thanks for taking my question and congrats on the order. You know, maybe one follow-up on HDV. I think, you know, where we've heard a couple questions is maybe in the 900 mig 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 data set. Just, like, curious your perspectives here and, you know, as you think about the, you know, 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?

Speaker #8: Just curious your perspectives 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?

Speaker #8: Is it that TND virologic response? Other measures of virologic response? The composite? Just curious your perspectives there. Thanks.

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

Speaker #2: Yeah, thanks for the question. I'll maybe make a couple of comments and then have Joanne speak to some of the data that we're seeing out of the 01 phase 2B portion.

Chris Peetz: 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 2B 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 a part of the disease. Seeing both of those move means you're kind of going after both components of the both of the considerations for both the infection and the liver.

Speaker #2: And I think in terms of what we're 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.

Speaker #2: 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 a part of the disease.

Speaker #2: So seeing both of those move means you're kind of going after both components of the both of the considerations for both the infection and the liver.

Speaker #2: Maybe I'll ask Joanne to touch on the data we've seen today.

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

Speaker #8: Yeah. And so Chris is talking about the composites. All very true. When we look at the curves in terms of the virologic response, we do see declines in everyone.

Joanne Quan: Yeah. You know, Chris talking about the composites, you know, are all very true. When we look at the, you know, curves in terms of the virologic response, we do see declines in every one. 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, again, this is a fairly small, numerically fairly small group.

Speaker #8: So 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.

Speaker #8: But we do see decreases in all of the patients certainly no evidence of lack of response, or resistance, or anything like that. So really partly it's an artifact of time.

Speaker #8: We do see deepening response with continued treatment. And again, this is a fairly small—numerically fairly small—group. We'll have a lot more information with the full AZURE-1 and AZURE-4 Phase 3 data sets to make a final call on that.

Joanne Quan: We'll have a lot more information with the full AZURE-1 and AZURE-4 phase 3 data sets to make, you know, to make a final call on that.

Speaker #7: Makes sense. Thank you.

James Condulis: Makes sense. Thank you.

Speaker #2: Thanks for the questions, James.

Chris Peetz: Thanks for the questions, James.

Speaker #1: Your next question comes from the line of Brian Scorney, with Baird. Your line is open. Please go ahead.

Operator 3: Your next question comes from the line of Brian Skorney with Baird. Your line is open. Please go ahead.

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

Speaker #7: Hey, good afternoon, guys. Thanks for making 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 it in your corporate nemesis.

Brian Skorney: 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' profile leaves an opportunity for another entrant and compare and contrast how Zulugacertib 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.

Speaker #7: So I'm just wondering if you could kind of give your broad thoughts on where you think Sohonis's profile leaves an opportunity for another entrant and compare and contrast how Ziller might address these.

Speaker #7: And the timeline would put us right around mid-cycle review with the FDA right now. So I'm just wondering if you could say if that is already happened or is still pending.

Speaker #2: Yeah, thanks for the question. On the review, yes, that has happened and I'd just say things are tracking as expected. But maybe I'll ask Peter to comment on a little bit more on positioning.

Chris Peetz: 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.

Speaker #9: Yeah, I think certainly the feedback we've heard from stakeholders, patients, caregivers, physicians, others, available therapy in the market today, there's a lot to be desired in terms of both efficacy and safety.

Peter Radovich: Yeah. I think, you know, 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 of the Zulugacertib profile, really exciting about, you know, what it could mean for these patients, both efficacy-wise as well as a convenient oral and well-tolerated regimen.

Speaker #9: And we'll have to be able to get into more details once we have the progress data presented in an upcoming medical conference, but from what we've seen in our review, the Zelirgecerted profile is really exciting about what it can mean for these patients, both efficacy-wise as well as being a convenient, oral, and well-tolerated regimen.

Speaker #7: Okay, thank you.

Brian Skorney: Great. Thank you.

Speaker #2: Thanks for the questions.

Chris Peetz: Thanks for the questions.

Speaker #1: Your next question comes from the line of Lisa Walter. With RBC Capital, your line is open. Please go ahead.

Operator 3: Your next question comes from the line of Tessa Romero with RBC Capital. Your line is open. Please go ahead.

Operator: Your next question comes from the line of Tessa Romero with RBC Capital. Your line is open. Please go ahead.

Speaker #10: 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 Insight?

Tessa Romero: 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? 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.

Lisa Walter: 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? 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.

Speaker #10: And maybe just one more given the recent positive results in Hep D and PSC, wondering if this is impacted your thinking on when Murum could become a profitable company.

Speaker #10: Thanks so much for taking our questions.

Speaker #2: Yeah, maybe I'll ask Peter to kind of speak to the FOP points and then have Eric kind of give some financial forward-looking views.

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

Speaker #9: Yeah, great. Overlap with our existing team, our rare genetics team that's focused on Texley COLBOM. We mentioned that the majority the significant majority of the FOP patients or FOP patients are cared for in settings centers that also prescribe Texley and COLBOM.

Peter Radovich: Great overlap with our existing team, our rare genetics team that's focused on CTEXLI, CHOLBAM. We mentioned that, you know, 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 physician target, the center overlap is really high with our existing rare genetics business. Excited about adding this product to that team.

Speaker #9: Different prescribers most of the time. Some overlap in medical genetics. FOP has 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.

Speaker #9: So excited about adding this product to that team.

Speaker #8: Yeah, and then on the financials, we did disclose the upfront license fee was 16 million, and then the next milestone would be 25 million upon FDA approval.

Eric Bjerkholt: Yeah. Then on the financials, we did disclose the upfront license fee was $16 million, and the next milestone would be $25 million upon FDA approval. There's some other commercial milestones, 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 said, we're spending a lot on R&D this year for both of those products. So profitability will be, you know, pushed out probably till 2028 on the GAAP basis.

Speaker #8: There are some other commercial milestones, but also a royalty in the low to, well, mid- to high-single-digit range. And we expect, after launch, that this product will be accretive very, very quickly.

Speaker #8: But your question was also about path to profitability, and I think that's much more driven by Brilovitec and Valixibat, as well as our current commercial business.

Speaker #8: As we 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 the gap basis.

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

Speaker #2: But would reiterate that expect to be operating cash flow positives next year.

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

Speaker #8: Yes.

Eric Bjerkholt: Yes.

Speaker #1: Your next question comes from the line of Jessica Fai. With JPMorgan. Your line is open. Please go ahead.

Operator 3: Your next question comes from the line of Jessica Fye with JP Morgan. Your line is open. Please go ahead.

Operator: Your next question comes from the line of Jessica Fye with JP Morgan. Your line is open. Please go ahead.

Speaker #11: Hey, guys. Good afternoon. Thanks for taking my question. Can you estimate the contribution to first-quarter Liv Marley sales from Alagil versus PFIC? And then another one, FOP, just thinking about that market.

Jessica Fye [Managing Director, Equity Research Analyst: Hey, guys. Good afternoon. Thanks for taking my question. Can you estimate the contribution to Q1 LIVMARLI sales from Alagille versus PFIC? Another one, FOP, just thinking about that market, what do you see as the penetration for palovarotene, and would you envision the ALK2 being used in combination with that drug? Thanks.

Jessica Fye: Hey, guys. Good afternoon. Thanks for taking my question. Can you estimate the contribution to Q1 LIVMARLI sales from Alagille versus PFIC? Another one, FOP, just thinking about that market, what do you see as the penetration for palovarotene, and would you envision the ALK2 being used in combination with that drug? Thanks.

Speaker #11: What do you see as the penetration for Palveritine? And would you envision the ALK being used in combination with that drug? Thanks.

Speaker #2: Jess, for the questions— I mean, just briefly on Livmarli, we typically aren’t breaking out by indication, but what we could say is that PFIC, they’re both growing, but PFIC is the bigger growth driver.

Chris Peetz: Jessica, the questions, I mean, just briefly on LIVMARLI, you know, we typically aren't breaking out by indication. 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.

Speaker #2: Maybe past the FOP question over to Peter.

Speaker #9: Yeah, and when we look at the US, the market where this medicine is available—Palveritine—it’s probably a minority of diagnosed patients that are currently receiving it.

Peter Radovich: Yeah. When we look at the, you know, the US, the market where this, medicine's available, palovarotene, you know, 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 that, you know, it, you know, can be tried, but it can often be difficult to tolerate and stay on.

Speaker #9: You can see from pharmacy claims data and that kind of matches what we've heard in physician and caregiver interviews that can be tried, but it can often be difficult to tolerate and stay on.

Jessica Fye [Managing Director, Equity Research Analyst: Thank you.

Jessica Fye: Thank you.

Speaker #11: Thank you.

Speaker #2: Thanks for the questions.

Peter Radovich: Thanks for the question.

Speaker #1: Your next question comes from the line of MABI Forahar with Lyrnic Partners. Your line is open. Please go ahead.

Operator 3: Your next question comes from the line of Mani Foroohar with Leerink Partners. Your line is open. Please go ahead.

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

Speaker #7: Hey, guys. You have Brian on for Moni. 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?

Ryan McElroy: Hey, guys. You have Ryan on for Manny. 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.

[Analyst] (Leerink Partners): Hey, guys. You have Ryan on for Manny. 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.

Speaker #7: And 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?

Speaker #7: And how should we think about upcoming data for the younger age groups that are being tested? Thanks.

Speaker #2: Thanks for the questions, Ryan. Our next US strategy actually a European filing is upcoming, so we could actually still have that in this quarter.

Peter Radovich: Thanks for the questions, Ryan. On OUS strategy, actually a European filing is upcoming, we could actually still have that in this quarter. Insight 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, and they're rolling now, so they're not too far out.

Chris Peetz: Thanks for the questions, Ryan. On OUS strategy, actually a European filing is upcoming, we could actually still have that in this quarter. Insight 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, and they're rolling now, so they're not too far out.

Speaker #2: Insight is still driving those activities. And again, their teams did 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.

Speaker #2: 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.

Speaker #2: Over the near term, frankly. So those are ongoing and rolling now, so they're not too far out.

Speaker #7: Awesome. Thanks.

Ryan McElroy: Awesome. Thanks.

[Analyst] (Leerink Partners): Awesome. Thanks.

Speaker #2: Great. Thanks for the questions.

Peter Radovich: Great. Thanks for the questions.

Chris Peetz: Great. Thanks for the questions.

Speaker #1: Your next question comes from the line of Ryan Deshner with Raymond James. Your line is open. Please go ahead.

Operator 3: Your next question comes from the line of Ryan Deschner with Raymond James. Your line is open. Please go ahead.

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

Speaker #12: Thanks for the question. A couple for me. What's your strategy for identifying FOP patients in the US and abroad and addressing the relatively high misdiagnosis rate for FOP?

Ryan Deschner: Thanks. Thanks for the question. A couple for me. You know, what's your strategy for identifying FOP patients in the US 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 Insight's prior clinical studies or maybe a compassionate use program or something like that in FOP? I have a follow-up. Thanks.

Speaker #12: And do you anticipate any early line of sight into a substantial group of patients from Insight's prior clinical studies or maybe a combination of use program or something like that in FOP?

Speaker #12: And I'm a follow-up. Thanks.

Speaker #2: Yeah, thanks for the question, Ryan. Yeah, I think that FOP patients often have a longer diagnostic odyssey than they should. There are patients that get diagnosed at birth, but often I think the literature says average age of diagnosis is seven years and obviously people that wait longer than that.

Peter Radovich: Yeah. Thanks for the question, Ryan. Yeah. The, you know, I think that FOP patients often have a longer diagnostic odyssey than they should. You know, there are patients that get diagnosed at birth, but often, I think the literature says the average age of diagnosis is 7 years, and there's obviously people that wait 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 US, though, most of the, you know, a pretty substantial majority of the patients are identified with FOP.

Speaker #2: 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.

Speaker #2: We do think in the US, though, most of the pretty substantial majority of the patients are identified with FOP, probably a different story in middle and lower-income countries.

Peter Radovich: Probably a different story, you know, in middle and lower income countries. There's the second question.

Speaker #2: And what's your second question?

Speaker #9: And did you have a follow-up, Brian?

Peter Radovich: Hey, did you have a follow-up, Ryan?

Speaker #12: Yeah, 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.

Ryan Deschner: Yeah, 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: Yeah, I mean, Insight 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.

Speaker #9: Yeah, I mean, Insight does have this progress study that'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.

Speaker #9: But I've certainly seen a lot of physician and patient interest in the program.

Speaker #12: Okay, thanks very much.

Ryan Deschner: Okay. Thanks very much.

Speaker #2: Thanks for the questions.

Chris Peetz: Thanks for the question.

Speaker #1: Your next question comes from the line of Joseph Tome with TD Cohen. Your line is open.

Operator 3: Your next question comes from the line of Joseph Thome with TD Cowen. Your line is open.

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

Speaker #13: Hi there. Hi there. Good afternoon. Thank you for taking my questions. Maybe one on FOP, I guess. The level of. Out to inhibition you're seeing with the therapy?

Joseph Thome: Hi there.

Operator 3: Please-

Operator: Please-

Joseph Thome: 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 and 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.

Speaker #13: I guess, do you think that could be enhanced by Garitos, MAB, Regeneron's active in a drug, or are these largely just going to be competitive therapeutics in the landscape?

Speaker #13: And then second, when we think about the potential expansion opportunity for Liv Marley in the basket trial that's going to be reading out later this year, I guess, how should we think about that?

Speaker #13: much is this basket population? Thank you.

Speaker #2: Thank you. Make a quick comment on the Garitos, MAB positioning. And probably best to get into more detail on this after our data is presented so it can give a better, a more complete picture.

Chris Peetz: If I can make a quick comment on the garetosmab positioning and, 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 Zulugacertib and its clinical positioning is really strong as a convenient oral single agent, and excited about bringing that forward. I'll let Peter take the follow-up.

Speaker #2: But what do you think the profile for Zoligacertib and its clinical positioning is really strong as a convenient oral single agent? And excited about bringing that forward.

Speaker #2: Maybe if Peter takes the follow-up.

Speaker #13: Yeah, and expand we've kind of talked about that indication being about a third of the at least 1 billion peak sales opportunity for Liv Marley.

Peter Radovich: Yeah. On expand, 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.

Speaker #13: And still reiterate that.

Speaker #12: Thank you.

Joseph Thome: Thank you.

Speaker #2: Thanks for the questions.

Chris Peetz: Thanks for the question.

Speaker #1: Your next question comes from the line of Charles Wallace with HCW. Your line is open. Please go ahead.

Operator 3: Your next question comes from the line of Charles Wallace with HCW. Your line is open. Please go ahead.

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

Speaker #14: Hi, this is Charles. I'm for RK. Thanks for taking my question. So for FOP, another question on that. How many patients from the progress study of, I think there was 63 in that study, do you expect will could come on after launch?

Charles Wallace: Hi, this is Charles. I'm from 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?

Speaker #14: And do you expect to have some sort of bridging program?

Speaker #2: Thanks for the question, Charles. And given the kind of the nature of the relationship here, we're going to wait until we have the data presented to give some of the detail on it.

Chris Peetz: 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.

Speaker #2: 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.

Speaker #14: Okay. That's fair. And then I guess another question that may on the Salesforce expansion. So you're growing it to 60 in the field. And 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?

Charles Wallace: Okay. That's fair. I guess another question for Nate on the sales force 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.

Speaker #14: Thank you.

Speaker #2: Yeah, I could kind of prepare a comment with starting this later this year, and so I kind of think early next year we'd be kind of fully on board.

Peter Radovich: 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.

Speaker #2: And that team would cover both pediatric and adult settings where not just alageal and adult people can be found, but also PSE and Delta.

Speaker #2: So I think early next year they'd be active in all those areas. And of course, with the pipeline products, the activity would really start upon potential FDA approval.

Speaker #14: Great. Thanks for taking my questions and congrats on the recorder.

Charles Wallace: Great. Thanks for taking my questions and congrats on a great quarter.

Speaker #2: Thanks. Yeah, thanks for the questions.

Chris Peetz: Thanks. Yeah, thanks for the questions.

Speaker #1: There are no further questions at this time. I would now like to turn the call back to Chris Peetz for closing remarks.

Operator 3: There are no further questions at this time. I would now like to turn the call back to Chris Peetz for closing remarks.

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

Speaker #2: 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.

Chris Peetz: 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.

Speaker #1: This concludes today's call. Thank you for attending. You may now disconnect.

Operator 3: This concludes today's call. Thank you for attending. You may now disconnect. This event has now concluded. Thank you for joining Mirum Pharmaceuticals Reports Q1 2026 financial results and provides business update. The line will disconnect automatically.

Operator: This concludes today's call. Thank you for attending. You may now disconnect. This event has now concluded. Thank you for joining Mirum Pharmaceuticals Reports Q1 2026 financial results and provides business update. The line will disconnect automatically.

Speaker #15: This event has now concluded. Thank you for joining Mirum Pharmaceuticals' reports first quarter 2026 financial results and provides business update. The line will disconnect automatically.