All right. Welcome, everyone. We'll get started. I'm Josh Schimmer from the Cantor Biotech Equity Research Team, and very pleased to introduce from Mirum Pharma. We have Peter Radovich, President and Chief Operating Officer, and Eric Bjerkholt, Chief Financial Officer, coming off of a very hot second quarter for both LIVMARLI and CTEXLI. Maybe set the stage and share everything that's going right at Mirum. Right. Thank you, Josh, and thanks to Cantor for inviting us to present here. I'll start off by saying we will be making forward-looking statements, and there's a long list of risk factors in our SEC filings, so please consult those. Mirum, we're a rare disease company with three approved medicines. We've increased our guidance every quarter this year and currently stand at $490 million- $510 million in revenue. We are cash flow positive, and I think we say we are financially independent because of that, which I think is a big benefit. In addition to our commercial business, we have a very full pipeline with four clinical trials from three different product candidates, and three of those trials are, we believe, registration trials. A lot of good news to come over the next sort of 6- 18 months or so. Maybe we can start on CTEXLI just because it's kind of easy to bang out. You've actually done a great job inflecting the product since taking it over and getting approval. Maybe share a little bit of the playbook that's been able to drive new patient identification and confidence that that can continue to identify unmet need situations. Yeah, thanks for the question, Josh. We've kind of built a dedicated team to focus on CTEXLI and the bile acid products outside of hepatology. We have some of the, about half the prescribers are hepatologists, but the other half are medical genetics, neurology, and have followed leads, supported screening efforts, and kind of gone far and wide to try to find these patients. The literature would suggest about 10% of them are diagnosed. I don't think it's going to be the kind of dynamic where you see a sudden logarithmic inflection. I think it's kind of a gradual, steady, scrappy effort to go out there and find patients, but certainly have been successful in doing that and decided that there's a lot of headroom to go. I think you've mentioned you've got a small, like, 10-person sales force scouring ICD codes, looking for telltale signs. What are those telltale signs that might clue them in to having a patient in need? Yeah, you know, it's a CTX. It's a progressive disease, and the clinical manifestations and organ damage accumulate over time. Some of the manifestations, and they're multi-system in nature, so some of the ones we focus on, some of these patients without the diagnosis yet may be in neurology with kind of ataxia and movement disorder clinics. Many of the patients from the literature will have bilateral cataracts before they're adults. That's ophthalmology, pedophthalmology can be an intervention point to deliver education of, hey, if you see a patient with bilateral cataracts, maybe be suspicious about genetic testing. A chronic diarrhea, actually, without other explanation is another one where we found patients in GI, liver. Those are some of the ones we focus on. Those are still somewhat broad. They are. Criteria. How do you get the focus on a more enriched population to screen? Yeah, I mean, they are, and that's why we kind of follow ICD codes and HITs, and it's also just not something that's going to see a sort of a logarithmic change overnight. Each of those occurrences, I mean, they're broad, but there's not a lot of patients that get bilateral cataracts, and the bigger barrier there is, you know, many ophthalmologists don't want to do genetic testing, so it's trying to kind of facilitate access and comfort there. Movement disorder clinics, the nice thing in neurology is that field is moving more towards precision in general, so it's kind of making sure that the gene for cerebrotendinous xanthomatosis (CTX) is on the broad panels, because for them, it's not really useful to send just for genetic testing for CTX. It's, well, can I test for everything that could be a potential genetic cause of a movement disorder? It's making sure you're on those so there's more utility for the clinician. It certainly seems like a job for newborn screening. Where are we in that implementation? Yeah, you know, the field has been active. The CTX community has been active for a while on trying to get CTX on the newborn screen. I think that would be the ideal place to find patients and start them on CTEXLI before they accrue any organ damage and could, I think, have the biggest clinical impact there. There was a pilot project. The biggest one actually happened, it's still ongoing, but had a nice readout here in New York where they, and this is kind of one of the requirements for HRSA and RUSP, the group in the federal government that decides on newborn screening. They want to see a real-world kind of implementation. Is it practical to do this? Actually, I think they found kind of about one in 18,000 patients, which is a higher rate than the literature suggests here in New York. I think the CTX community more broadly, the physicians and advocates that are kind of pushing for this, were excited by that. There are obviously changes happening within HRSA and newborn screening at the federal level right now that are kind of unclear how that's going to play out affecting the whole rare disease community. Optimistic that with the success in New York, other states will pilot this, and maybe at some point in the future, this could be something that comes through at the federal level. Is like a year timeline overly aggressive? Probably, right? Because this is, it's just hard to, with all the changes that are happening within RUSP and the committee that advises RUSP that got dismantled by the health secretary, it's difficult to know then, like, that really is the piece that the historical paradigm for getting things on the newborn screen. Right now, it's just not clear how that will unfold. It's tough to put a timeline on it. How did Zellweger then make it on for CHOLBAM for newborn screening? You know, it's not explicitly on the newborn screen. What is, is XALD. If you ever saw the Lorenzo's Oil movie, you know, that's on there, but you're looking at very long-chain fatty acids, which also kind of coincidentally pick up Zellweger. That happened before the latest changes at the federal level, the HHS, whatnot. Turning to LIVMARLI, great execution and ongoing adoption. Maybe you can characterize where you think you are within Alagille in terms of current penetration and your ultimate target penetration in the population? Yeah, you know, right now, we in the U.S. and more mature kind of European markets where we're directed, we think we're about 50% roughly penetrated into the prevalent pool. We are adding prevalent patients every quarter. It's a steady cadence of adds that we're seeing. That's been steady for several quarters now. We haven't quantified what we think the ceiling could be, we just quite frankly don't know. We definitely think there's plenty of headroom to grow. That's kind of 50% of addressable, not necessarily, not 50% of prevalent. So it's really patients that we think, you know, have cholestatic pruritus that could benefit. That's really a lot of what our marketing is focused on. If you're thinking about market quantification, that kind of ignores the incident patients because there's newborns every year. That's an area where LIVMARLI does very well, and those patients kind of naturally come to therapy, you know, in a pretty straightforward fashion. Is there newborn screening for Alagille, or does there need to be? There's not. I don't think there needs to be because, you know, most of those patients, the literature is, you know, at least 90% present with neonatal jaundice and symptoms in the first year of life. One of the hepatologists that works at Mirum says people don't miss yellow babies. You get a jaundice kid, and they're immediately working to rule out biliary atresia because if you have biliary atresia, you need to get a surgery very rapidly for it to work. As part of that workup, they kind of quickly order genetic testing. That diagnosis rate for Alagille is pretty high. Roughly how many patients now with Alagille on LIVMARLI? We don't provide kind of patient-level information, but the nice thing about our revenue recognition model is we recognize revenue when the family actually signs for LIVMARLI at their house. It really is a kind of a demand sales number, a pretty pure look into demand sales. One can do arithmetic with the price and kind of back into it a little bit that way. Your estimate for the total number of patients in the U.S. with Alagille? We think prevalent 2,000- 2,500. The majority, probably at least 75%, have pruritus. Not all of them have their native liver, and some of them have decompensated, so those probably wouldn't be candidates. What is the incidence? It's about one in 50,000 births have Alagille, so a little under 4 million births per year in the U.S. Okay. Is that about 100 in the U.S.? Did I get that right, Eric? Eric's better at math than me. It's probably a little higher than 100. A little higher. Okay. In terms of the incident population, against adjusted 75% with pruritus, you can tell they're pruritic when they're very young? Usually, it can be, you know, it's interesting. That's one thing we educate on, fussiness, fidgety, that is a sign. It's really, do they have liver involvement is the first thing that they look at. If there's jaundice and the liver labs are high, then they have liver involvement. Usually, the clinicians are very much like, how do we establish pruritus here? You might look at fussiness and, are they rubbing? It's hard when they're an infant, but it is something that people do to support the indication. As a growth driver for revenue, those are the patients I imagine are starting lowest, but also growing fastest to provide maybe some degree of visibility of franchise growth in the coming years, even absent of additional patient identification. I think that's well said. In any given quarter, the amount of consumption from infants is not very high. You can go add a bunch of infants and not see it on an in-quarter number very, very well, but they do increase their consumption quite quickly. Also executing very well now on the PFIC launch and even finding adult patients. Is that now kind of where the patient identification efforts are for new patients as opposed to the, I'm guessing the young patients are mostly treated, incident patients very small. Your target demographic here now, likely adults. Yeah, older children, adolescents, and adults. Absolutely, yeah. Maybe you can talk a little bit about the effort to go and find them and how many adult PFIC patients you think might be out there to discover. Yeah, I mean, that one compared to the conversation we were having about CTX, which I think you correctly said is a broader strategy. This one is kind of nice because it's focused, which is, you know, approaching gastroenterologists, hepatologists. They have patients in their clinic who have cholestasis. They have high liver labs. They have the symptoms. They've gone through the kind of diagnostic workup and the differential checking for, you know, PSC, PBC, things that you would, could be a potential cause of that, but they don't have autoantibodies. They don't meet any of those criteria. They're kind of getting labeled as unknown or idiopathic cholestasis. Those patients are there. They're in the clinic. Sometimes they're getting old off-label drugs anyway. The education kind of focuses on them and driving an index of suspicion about genetic testing. We're able to provide the genetic testing so that that kind of facilitates managed care access issues to it. There's a decent kind of diagnostic yield off of that. That's kind of where we found. Are you able to kind of ballpark the number that you found so far and the number you think might still be out there? You know, it's early, it's still early days. It's hard because it's a little bit of an unknown, unknown. We just don't know how big that denominator is. Those idiopathic patients don't have like an ICD code that we can quantify. There are some papers out there that have been presented or posters that have been presented at medical conferences saying that maybe 15%- 20% of those patients might have a PFIC mutation. We still don't know how big that denominator is. We're kind of going out hand to hand doing the education right now. It sounds like you're having early traction with the oral tablet formulation. I'm guessing both treatment naive and potentially switching patients. Maybe you can talk a little bit about that switching dynamic. Is there a patient profile who may be on Bylvay who would be a particular likely candidate to prefer the LIVMARLI tablet? Yeah, I mean, the switching, we launched the tablet in June, so kind of commented, it's really not at all reflected in our Q2 numbers, but have seen a lot of interest and switches from the liquid of LIVMARLI. I think it's kind of any patient that's old enough to swallow a pill. The nice thing here is it's a single, pretty small tablet per dose. One a day for Alagille, two a day for PFIC. Added convenience, especially for teenagers. Some might already be on, they're often already on urso, so there's other pills. The other IBAT inhibitor is a larger pill burden for patients. It is kind of, if there's adolescents and adults that want to simplify pill burden, it's a nice option. Have you started efforts to identify adult PFIC patients in Europe as well? Yeah, we have. I mean, we haven't, the pricing and reimbursement process for the PFIC indication expansion is still kind of playing out in Europe. Really, when you think about our international sales that we've reported to date, the vast majority of that is Alagille, but the same kind of clinical dynamic is there. In fact, some of those papers I referenced do come from Europe. I think the adult providers around the world are getting more and more interested in genetics and could some of these unknown patients have a genetic reason for their cholestasis? Maybe turning to the Takeda relationship, which has generated some fairly robust revenue over the last couple of quarters. How do we think about this line item going forward, recognizing that still early in the launch in Japan by Takeda, but you also have this scaling down royalty, right? There is a tailwind and a headwind. How do you think about these forces playing out over the next few years? I'll start with the end. The scaling down will take place over time and, you know, not very quickly. We think we can generate robust revenue from that. I think Japan is a significant market opportunity. That's the first country where they got approved for both Alagille and PFIC at the same time. Reimbursement is attractive from our standpoint and our economics are very good. The thing to watch out for is that the way the agreement works today, when they order from us and we ship, we book revenue, but we have to estimate what portion of that shipment is actually going to result in revenue and over what timeframe. We need to model their demand curve and, you know, we're trying to be conservative in that regard so that, you know, when there's a true up, it's, if anything, hopefully a positive true up. You shipped in the first quarter, you recognized $6 million. You didn't ship in the second quarter, and you true-upped $11 million. Yes. Okay, that kind of gives us maybe a sense of that curve over the first two quarters. When do you expect the next order to come through? We expect another order this year, so there will be additional revenue in the second half. Presumably, the timing of the order is going to be important, right? Because we've got $16 million of revenue booked for the first half. I guess now that's recognizing product shipped to Takeda that you expect to be delivered to patients. Exactly. What happens in the third quarter? We haven't said which quarter the shipment will take place in, but it's a smaller shipment than the first one. We will estimate what portion of that will ultimately be sold and recognize that portion of that as revenue. How did you come up with the estimates of $6 million and $11 million so that then in third quarter we dropped out, right? Because it's not necessarily this linear dynamic and you go. No, I mean, we have a model, right? It's a launch curve for Japan, which is our model, not necessarily their model. That's where I'm saying we're trying to be conservative. Okay, maybe we can talk about the expanded basket study. By the way, you sound like our auditors now. Exactly. As I'm thinking through this, I'm very confused. Right. Yeah, so are they. Yes, exactly. Just like an auditor. Maybe we can talk about the expanded basket study of LIVMARLI and the various diseases that you're looking to capture there, and how you've come up with the estimate of 500 or more patients with these conditions ultimately to treat. Yeah, so, you know, I'm going to take a step back and just share for those that don't know. I mean, the genesis of the expanded study actually started with a lot of, you know, it was really, I would say, physician-led. A lot of inbound that Mirum received, over 100 compassionate access requests that we fulfilled. I think much more demand than that has been communicated to us over the years of these patients who aren't Alagille, aren't PFIC, but have cholestatic pruritus. They have high bile acids. They have really bad pruritus. I'm thinking about the physiology. Why wouldn't your drug work here? FDA actually said, hey, this is a lot of paperwork, all these single-patient INDs. Can we wrap it into a protocol? We did. I think probably the largest on the list to your first question is probably biliary atresia patients. There's a whole kind of long list of additional patients who have rare, really ultra-rare conditions. Secondary sclerosing cholangitis can be there. Scoliopathies. It's really kind of a diagnosis of exclusion. The idea is this would be a place to study those ultra-rare patients who wouldn't be sort of studiable in a practical sense in a standalone protocol. What do you think about a similar study in the adult patient population? Because as you identified with your PFIC patients, there's 80% of patients with an identified cholestatic pruritus who don't have PFIC who might be good candidates. Yeah, you know, this study actually does the primary cohort and analysis in pediatrics and the Ichiro observer. It's really kind of focused on the pediatric population. There is an additional cohort, a supplemental cohort that enrolls adults. We will get some experience from that group and learn more about that side of it as well. Is the idea that that cohort may suffice for a label expansion on its own? If not, inform you of what you would need to do to capture them. Yeah, I think it'll give us a lot of information. It's kind of hard to speculate at this stage. You know, would or wouldn't they be suitable for labeling? That's probably a review issue and ultimately something that has to be discussed once the data is there. The other scenario you described, inform us on what else we'd have to do, I think is right. Have you had meaningful compassionate use requests from these adult patients? Yeah, there's been interest there as well. A variety of patients post-transplant, cholestatic pruritus. There's a long list of indications that come up. Because the rationale from the FDA in theory would be the same, right? If you're getting a lot of compassionate use requests for adults or kids, and they want all that to be on label, then one might surmise that they'd be permissive with this study to have a broad. Yeah, it's going to come down to substantial evidence of effectiveness and how we think about all these different entities under one umbrella. Because that could be, I mean, these are adults, big, right? The higher weight, higher dose, higher price per patient. As we think about the contribution they could deliver, and as you address that unmet need, it could be quite substantial. Yeah, I mean, our thinking is probably, you know, that's kind of upside for us. This at least 500 is kind of a line of sight number on the pediatric side where we feel confident we can stand behind that based on how we see the study screening going, how our kind of field, medical, and other interactions with the sites are, the volume of interest we've seen come in. It's not like an ambitious number. It really is what we have kind of line of sight to on the pediatric side. Where's your caution about starting to factor those adult patients into these estimates? I think it's probably just a little early, both on the regulatory side as well as kind of study enrollment, who actually ultimately comes into the study, what constitutes substantial evidence of effectiveness. I think to the docs in practice, it makes a lot of sense, high bile acids, cholestatic pruritus, let's get the bile out of the body, but we just don't want to get out over our skis. Have you been seeing similar efficacy in the adults and peds in the compassionate use setting? It's small numbers in compassionate use. I think it'll, it's, we probably have to see how things go in the expanded study. I was just going to make a general comment that as a team and as a company, we try hard to underpromise. We don't promise away. I'm with you there. Well said. All right, why don't we go to the liquid bed and PSC then? Second quarter of 2026 is the projected timing. You've got positive interim and as well as the disclosed interim from the PBC study. Assuming that nothing weird happens from here till the end of the trial, you'll likely be launching the product in 2027. Maybe talk a little bit about the patient identification efforts that you have underway and what kind of commercial footprint expansion you would need to target PSC. Yeah, we recently, at the beginning of this year, actually expanded our Medical Science Liaison, our MSL team. I've doubled that. Now I have a team of 12, many of them coming from long track records in adult liver, some of them clinicians, others who have worked in other adult liver companies that you know. They've kind of brought, they've kind of focused on educating around the burden of pruritus and unmet need, hopefully supporting enrollment in Vista's advantage, as well as have helped educate on genetic cholestasis, the conversation we were having earlier about PFIC. I think that education's been an important part of the overall effort. As we look towards data Q2 next year and filing beyond that, we would look to kind of expand the commercial team, the field sales team. Right now, it's about 17 in the U.S. sales representatives who are focused mostly in pediatric and then the high decile adults, PSC and then PBC as well. We think there's a fair bit, maybe even the majority of patients being managed in private practice GI. I would envision that going up to probably on the order of 50. The good news on the, just, that's on the sort of field sales number of humans perspective. From an OpEx perspective, we think we get a lot of leverage off of what we have. We've already built up all the infrastructure, market access, distribution, supply chain, compliance, quality, etc. Other than that field sales expansion, we think it's a pretty modest incremental investment. We already built up our medical team, for example, which has been very helpful in terms of helping enroll the trials, but also starting to educate physicians on the disease. All right, it's a modest increase in commercial costs. Yeah. On the R&D side, as you pass through the phase II-B trials of PSC and PBC, you may be ramping up Fragile X as a partial offset. Do you see meaningful increase in R&D spend going forward unless you find something that you fall in love with and you're like, we need to spend a lot. We also have open label extension studies, so it doesn't go to zero. No, we do not expect major increases in R&D. You kind of started by noting you're cash flow positive now, and we've talked about a whole litany of revenue drivers going forward. I mean, we should now be on the cusp of Mirum turning into a very profitable company over the coming years. Is that reasonable? Assuming everything goes according to plan and the opportunities in PSC and PBC are what we think they are, once we get through the investments we need to make to have successful launches, which we've talked about, then, yeah, I mean, as CFO, I like the way it looks a few years out. It creates a new set of strategic decisions, though, to make with the cash flow that you're going to have. How do you think about Mirum over the long term, right? How you're going to deploy this cash flow? Do you prioritize, should we purchase? Do you prioritize hoarding cash for a bigger acquisition, or is it another strategy that you're contemplating? I can't say we've had deep discussions on the topic internally at this point because it's a few years out. We will need to have those discussions. I will say we do, we would like to continue to build the company, and that requires probably bringing in additional programs over time. I don't think we have a gun to our head in any way, shape, or form. Over time, it's something we'd like to do. We do have a fairly robust BDE effort, always looking at things that could be appropriate, that we can add value to, and that are appropriately priced. How do you envision your risk appetite evolving again as you become increasingly profitable? Does it go up? Does it go down? Maybe you can frame it relative to Fragile X. Is that the kind of program that you think you'd look to accumulate additional assets or pursue higher or lower risk targets, or all of it, I guess? In theory, you'll have enough cash flow to consider all of it. I think the size of what we could consider obviously changes as the company gets bigger and we have more cash. I would also say that I'm Norwegian, and so I am risk-averse by nature. Oh, that's great to hear. That's well said. The Fragile X program probably represents the early end of the bookends of what we would look at, some level of validation for the mechanism already from another program. More biology risk than that probably is not our sweet spot. As you think about strategic planning forward, what do you see Mirum 's core competencies to leverage being? I'm going to start. Yeah. I think we have a great late-stage, you know, mid-late-stage clinical development team and regulatory team that's shown they can register our products all over the world. Obviously, really proud of the commercial capabilities and medical affairs capabilities we built, partnering with physicians and patients, patient advocates to get our products out around the world. I think that's really the core of the company. Late-stage appetite. Do you see yourself more as innovators now as commercializers with expertise in rare hepatobiliary or rare diseases? Is that kind of the signal of how you view yourselves and where you're at? Rare. Yeah, rare diseases. Yeah, I think we're good at finding patients and building. On the regulatory side, figuring out path to market with not conventional evidence, right? The LIVMARLI Alagille application had that feature, CTEXLI. Yeah. Excellent. Peter and Eric, thank you so much for joining. Great discussion. Thanks, everyone, for tuning in. Thanks, John.