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Good morning, and welcome to the Merham Pharmaceuticals Business Update Call. My name is Kelsey, 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. I would now like to hand the conference over to Andrew McKibben, SVP of Strategic Finance and Investor Relations. Please go ahead. Thank you, Kelsey, and good morning, everyone. I'd like to welcome you to Miriam's conference call to discuss top-line results from the Phase III EXPAND study of Liv Marley, or Maralexabab, in ultra-rare settings of cholestatic pruritus. For prepared remarks, I'm joined today by our Chief Executive Officer, Chris Peets, and our Chief Medical Officer, Rob Myers. Also joining us for the Q&A portion of the call are Peter Radovich, our President and Chief Operating Officer, and Eric Bjerkel, our Chief Financial Officer. Earlier today, Miriam issued a press release announcing these results. A copy of that release and our SEC filing, along with a presentation summarizing the data, are available on 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 Merrim's programs and market opportunities for its approved medicines and project candidates and financial guidance. These statements represent our judgment and knowledge 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 10Q and subsequent SEC filings for more information about these risks and uncertainties. With that, I'd like to turn the call over to Chris and Rob for some brief remarks before we open the call for Q&A. Chris? Thanks, Andrew, and welcome, everyone, for another great update from the Miriam Pipeline. Today marks another important milestone in our effort to bring Live Marley to more patients living with ultra-rare cholestatic liver diseases. We are excited to announce that the Phase III EXPAND study demonstrated a statistically significant and clinically meaningful improvement in pruritus. In addition to meeting the primary endpoint, the study met all secondary endpoints, including showing a highly significant reduction in pruria and biliary atresia patients. Results in biliary are notable as there are no approved medicines in this setting, and we estimate about 2,000 biliary patients across the U.S. and Europe suffer from cure itis our next step is a planned supplemental nda submission in the first half of next year stepping back expand is a great example of how we think about building mirim we start with patient need pair it with scientific potential and then apply efficient execution to bring meaningful medicines to those patients that approach has now translated into our fourth positive study readout this year. And we see continued opportunity for the Mirim business model ahead. Today, fewer than 5% of rare diseases have an approved treatment. We believe focused companies like Mirim can play an important role in changing that. And finally, before I turn it over to Rob, I'd like to thank the patients, investigators, and study teams around the world who made EXPAND possible. Rob? Thanks, Chris. My first few weeks at Mirim have been exciting, and I'm thrilled to be here. From the outset, the rationale behind EXPAND was straightforward. Many patients suffer from polystatic pruritus due to rare liver diseases that are simply too uncommon to support traditional disease-specific development programs. As a result, many of these patients have historically had little or no opportunity to participate in registrational clinical trials. EXPand was designed in alignment with and at the request of the FDA to address this challenge. Rather than studying a single disease, the trial enrolled patients with different rare cholestatic liver diseases that share a common underlying biology and a symptom that can profoundly impact quality of life, cholestetic pruritus driven by impaired bowel flow. This innovative approach allowed us to generate meaningful data in patient populations that might otherwise never be studied individually. Importantly, the results strongly support that strategy. The primary efficacy population in EXPAND included 46 pediatric patients with moderate to severe pruritus across a range of ultra-rare cholestatic liver diseases. Approximately one-half of enrolled patients had biliary atresia, and patients were randomized to receive maralixibab or placebo during a 20-week placebo-controlled treatment period. Now, let me walk you through the results in more detail, starting with the primary endpoint. EXPAND demonstrated a statistically significant and clinically meaningful improvement in pruritus with Mariluxivac. Pruritus was assessed using a caregiver-reported scale ranging from 0 to 4, corresponding to no itch and very severe itch, respectively. Across weeks 13 through 20, patients treated with Marilluxivat experienced a mean reduction in pruritis of 1.81 points from baseline. Putting the magnitude of this reduction in context of the 2.65 mean baseline value makes it particularly impressive. This reduction with Meralixibat compares to 0.83 points for placebo, representing a placebo-adjusted treatment difference of 0.98 points and a p-value of 0,0004. The secondary endpoints further reinforce the primary efficacy findings and the biologic rationale underlying Meriluxibat's mechanism of action. Serum bile acids were reduced by 70% with Merilixibat compared with 3.5% with placebo, corresponding to a treatment difference of 69% and a p-value of less than 0.0001. Taken together, these findings demonstrate meaningful improvement in a symptom that matters greatly to patients and their caregivers, supported by substantial reductions in the bile acids believed to drive cholestatic heredity. I want to spend a little more time on biliary atresia, where there are currently no approved treatment options. This was the largest patient subgroup in EXPAND, and the results closely mirrored the positive results observed in the overall study population. In the pre-specified biliary-atresia subgroup, Maralixabat achieved a mean reduction in pruritus from baseline of 2.15 points compared with 1 point for placebo, corresponding to a placebo-adjusted treatment difference of 1.15 points and a p-value of 0.0002. Importantly, the treatment effects observed in patients with biliary atresia were highly consistent with those with other cholestatic liver diseases enrolled in EXPAND. Across both groups, we observed meaningful improvements in pruritus accompanied by substantial reductions in serum biolapse. Now turning to safety. Maralixabat demonstrated a safety profile in EXPAND that was consistent with our prior clinical experience, and no new safety signals were identified. Treatment emergent adverse events were reported in 96% of patients in both the Maralizabat and placebo groups. Serious treatment emergent events occurred in 20% of Maralitzabat-treated patients and 18.5% of placebo-treated patients. There was one discontinuation from the study in the Maralexabat arm and none in the placebo arm. Consistent with the mechanism of IBAT inhibition in our prior clinical experience with Merilixibat, the most reported adverse events were diarrhea and abdominal pain, occurring in 64% and 20% of Merilxibat-treated patients, respectively. Importantly, no patient discontinued treatment due to diarrhea. Overall we're very encouraged by these results. EXPAND met its primary endpoint and all pre-specified secondary endpoints, while Meriluxibat demonstrated safety profile consistent with our prior clinical experience. More broadly, expand as through a growing and remarkably consistent body of evidence supporting IVAD inhibition as a therapeutic approach for cholestatic pruritus. Across five positive clinical studies and multiple cholestetic liver diseases, we continue to see meaningful reductions in pruritis accompanied by improvements in bile acid biology. Taken together, these findings reinforce our belief that targeting Using bile acid biology can deliver clinically meaningful benefit to patients suffering from polystatic psoriasis, regardless of the underlying disease. Now I'll turn it back to Chris. Thanks, Rob. And we're very pleased with these results and excited about the potential to bring Live Marley to these additional settings. From here, our focus turns to the planned supplemental NDA submission in the first half of next year. This label expansion opportunity comes on top of the continued growth we're seeing in Liv Marley and patients with allogel syndrome and PFIC. And today's update adds to an already remarkable year for Merim's pipeline. The Etebrios launch and FOP is on track to kick off this month, and we're excited about early feedback on the approval. This is an exciting growth opportunity for Merrim's rare genetic disease business. And Berlovitide's recent positive Azure One readout gives us confidence in the upcoming Azure Four study readout later this quarter. Those two studies will be the basis of the plan BLA in the first half of next year. And Velixibat provides further opportunity for the rare liver business. The VISTA's results earlier this year in PFC were granted breakthrough designation. We're moving forward with our strategy to interact with FDA ahead of an NDA submission planned in the third half of the year. Further, the Vantage study in PBC is on track for top-line data next quarter. We're closing out the year with a lot of momentum and very excited about what lies ahead for Miriam. And with that, operator, please open the call for questions. We will now begin the question and answer session. Please limit yourself to one question and one follow-up. If you would like to ask a question, please press star 1 to raise your hand. To withdraw your question, press star 2. We ask that you pick up your handset when asking a question to allow for optimum sound quality. If you are muted locally, please remember to unmute your device. Please stand by while we compile the Q&A roster. Your first question comes from the line of Egal Nachamuvitz with Citigroup. Your line is open, Egal. Please go ahead. We will take another question instead from Ryan Deschner with Raymond James. Your line is open, Ryan. Please go ahead. Hi. Good morning, and congrats on the readout today. On the roughly 70% reduction in serum bile acids, specifically regarding the biliary atresia patients. How clinically meaningful do you interpret this result to be and have a follow-up? Thanks. No, thanks for the question. I would interpret that roughly 70% reduction as being very clinically meaningful. We've observed across multiple cholestatic liver diseases now that this degree of reduction is associated with improvements in pruritus that are also clinically meaningful, and we've observed the same thing here in the overall population and in these biliary atresia patients as well. Thank you. Appreciate it. And where do you expect pricing on a per patient basis to shake out relative to Livmarly and Aligil and PFIC? And could this be another situation where revenue could grow over time, specifically driven by the actual growth of the patients? Thanks for the follow-up, Ryan. First comment is no change in the pricing strategy for Livmarley, but pass over to Peter talk about launch expectations yeah ryan the price for live marley is already established so the unit pricing is kind of set therefore the indication pricing is a function of dose and consumption so this would be about a 50 percent more dose than the algeo indication which is about uh four to five hundred net annualized per year so that that would be the kind of expand dosing for the protocol. And yeah, you would expect to see weight-based dosing and dose changes over time with weight gain. Thank you very much. Thanks for the question. Your next question comes from the line of Josh Shimmer with Cantor Fitzgerald. Your line is open, Josh. Please go ahead. We will take a question instead from Kalpeet Patel with Wolf Research. Your line is open, Kalpeeth. Please go ahead. Yeah. Hey, good morning, and many congrats on clearing this study. A couple from us, one for the label, what's the minimum and maximum ages that you're planning to seek a label for? And then second, have you looked at the percentage of pediatric patients who achieved a sustained one-point or greater reduction from baseline over weeks 13 to 20? And if you have done that analysis, can you break that out for the VA and non-VA patients? Thank you. Thanks for the question, Kalpit. First on label, expect from an age perspective this to be pretty similar to what we see for algeol and PFIC, where there's a lower end defined in basically infancy to young childhood with no expectation on an upper end as how we expect this to be the case here. And just as a reminder, you know, the inclusion criteria is our best indicator of how that indication statement plays out, which is effectively a definition of exclusion. So, cholestatic pruritus excluding some of the larger indications. And then we'll pass it over to Rob maybe to talk about what we've seen on some of the response rates on itch. No, thanks, Chris. Yeah, we saw remarkably consistent results in terms of itch response. But in answer to your specific question, in the biliary atresia subgroup, roughly 90% of patients had at least a one-point improvement in pruritus. And in the overall population, this was about an 80% response rate. So, quite a clinically meaningful improvement with maralexibat treatment. Okay, great. Thanks for taking the question. Yeah, thanks for the question, sir. Your next question comes from the line of Jessica Fye with JP Morgan. Your line is open, Jessica. Please go ahead. Hello. This is Adam on for Jess. Thank you for taking our question. Just could you elaborate what are the remaining steps between now and the S&DA submission, please? Thank you. Thanks for the question. You know, pretty typical post-readout where we'll be compiling this data, putting it into a write-up with all the follow-up analyses to a supplemental NDA filing. Really straightforward overall in getting to that in the first half of next year. Thanks for the question. Your next question comes from the line of Brian Scorney with Baird. Your line is open, Brian. Please go ahead. Hey, good morning, team. Thanks for taking the questions. Congrats on the data. I wanted to to get your thoughts on the non-biliary fuchsia opportunity here. You enrolled about 50% of the study in non-BA, and I think BA should be a pretty well-diagnosed population. So, I guess, was there any stratification in the study to think about, or do you think that there would be sort of 50-50 in terms of patient flow in the commercial setting of addressable BA patients and non-B.A. patients? And do you have any thoughts on sort of the penetration of diagnosis of the non-'B.I. altered orphan cholestatic patients? Do you think it could be, you know, greater than the 2,000 PA patients when all is said and done once more diagnosis is pushed? Yeah, thanks for the question, Brian. I mean, it's a really good question. I mean, the way I would think about it here, I mean this study had, you know, 24 sites from U.S. and around the world enrolled for a little over a year. And as you note, about half the patients were biliary atresia, half the patients were other. These were pediatric investigators, so I think that's important to know. It's kind of representative of kind of the practice dynamics and setting there. So, you know, in terms of how large that, you know, other bucket could be, the non-biliary atresia bucket could be, I think, that the actual study enrollment here is probably the best guide. As far as diagnosis, yeah, biliary atresias are very well diagnosed. Substantially, all patients or diagnosed as infants. The basket of other, I think it's pretty variable depending on the indication, but, you know, certainly would be hopeful that, as you see often in, you know, new product introductions, as awareness of a new product, you know, grows and, you know, there's something to do about, you know, pruritus and high bile acids, then, you know, hopefully recognition and diagnosis would increase in those settings. Right. Thank you. Your next question comes from the line of Joseph Tomey with TD Cohen. Your line is open, Joseph. Please go ahead. Hi there. Good morning. Congrats on the data, and thank you for taking my question. Maybe just now that we have the data out there, and you did mention the updated PFIC patient metrics in the last quarterly call, how are you seeing the breakdown in contribution between LGL-PFIC and this sort of other cold, static disease bucket in your $1 billion plus low barley expectation from a revenue perspective? And then second, if you could just talk about your expectations for how quickly this could come to market. Would this be a priority review situation standard, or kind of what's the base expectation given your interactions with the FDA? Thank you. Thanks for the question, Joe. On the latter part of it first, you know, we would see this as a candidate for priority review, just given the unmet need and the setting. And I'll pass it over to Peter to talk about the breakdown of the 1 billion-plus peak. Yeah, I think the first message overall of this, you know, readout really gives us confidence in the plus part of that 1 billion plus peak guidance that we have here, you see a lot of different ways to get there from these components. You know, the update we're providing in the materials today, if you look just at the biliary atresia component of the EXPAND study, about 1,000 addressable patients in the U.S., 2,000 U.s. plus Europe. That's comparable to algeol. Actually, when you think about the addressable population of algeo, you know, maybe the two differences from algeal, the dose here is different. It's about 50% higher, so that does play through the price. And then, of course, no competition in biliary atresa, although we've had pretty limited effects of competition in Allergy O syndrome. So I think if you think about peak sales, you know, the biliary atresia opportunity would be at least as large as the AllerGel. Great. Thank you. Thanks for the question. Your next question comes from the line of James Condalus with Stifle. Your line is open, James. Please go ahead. Hey, thanks for taking my question and congrats on the data. Maybe one on the epi here. I I think historically, you may be appointed some more like 1,000 across the U.S. and EU. And, you know, is that right? And can you talk about sort of, you know, what drove that sort of revision upwards to this 2,000? And then a second question here, just sort of on this other non-biliary atresia subgroup. In the real world, do you expect those patients to be adults or pediatrics? Obviously, you notice the study you mentioned enrolled pediatric patients. But just like curious in the real world. Is that sort of representative of what that other cholestatic bucket may look like? Thanks so much. Yeah, thanks for the question. One kind of initial comment, then I'll let Peter speak a little bit more to it. The way we're talking about the epi here really is just a little bit of a shift in framing it because with the results in biliary atresia being so strong, you can look at this from a more traditional kind of epi standpoint on looking at a specific disease, just being the biggest component of it versus the others, which is really a long tail of less common presentation. Peter, maybe speak to the adult versus pediatric and some of the thoughts on the dynamic and the other. Sure. Yeah, I think this, you know, in terms of the other patients in this study, and is that representative of pediatric or adult? I think it's well representative of pediatric, right? These are 24 sites. they were all pediatric investigators. So I think, you know, if you're looking at the list of diseases on the corporate deck, I think it's a pretty good representation of what you'd expect in the pediatric setting. The adult dynamic is probably a little bit different. That's, you know—we're encountering some of that as we go search for adult PFIP patients, and, you Know, as we've talked about before, there's a Pretty Good group of, you other idiopathic colostatic pruritus patients in adult practices a decent proportion of them have a p15 but probably more don't um and so i think that that but that's a sort of a different population that was involved in the study thanks man thanks thanks for the question your next question comes from the line of lisa walter with rbc your line is open lisa please go ahead Oh, great. Thanks for taking our question, and thanks for sharing the presentation just this morning. Just a quick question on the open-label extension study. I guess, when can we expect to see that data, and do you plan to share the results broken down by other patient subgroups, like the other disease indications, or perhaps by adults and pediatric subgroup in the OLE results? Any caller here would be helpful. Thanks so much. Yeah, thanks, Lisa, for the question. Really pretty rich publication plan opportunity here. And we'll let Rob speak on some of the different cuts we can expect to see from the OLE and the primary. Yeah, no, thanks Chris. You know, the OLe part of the study is ongoing and we will have another data cut there after 20 weeks of additional treatment. What I can tell you is that patients who have rolled over into the open-label extension are having similar responses to what we observed in the double-blind period with Mariluxivab treatment. In terms of disclosing results for the individual diseases, in general, these are small ends in each group of anywhere from one to two patients for most of these categories so will be somewhat difficult to you know present data for for every subgroup but you know we feel very confident about the data in the biliary atresia and overall non-biliar atresa subgroups and they look quite similar Thanks for the question. Our next question will be from the line of Joe Schwartz with LeeRinc Partners. Your line is open, Joe. Please go ahead. Thanks. Congrats on the strong data. First on the patients with biliary atresia, can you talk about them in a little bit more detail, given liver transplant is pretty common, but the patients here average about six years old with near normal bilis. So they look like successful Kazai patients who still itch. Is that the population behind the label in the 2,000 patient figure? And how long do you expect patients to take Livmarli before transplant? And then outside of BA, I think there were four diseases you said? Has the FDA said whether the label would name those conditions or be a broad label that covers other diseases that weren't in the trial? Thanks for the questions, Joe. On the dynamic in biliary atresia, the way you're thinking about it is kind of what we've seen in the study and how we expect kind of the overall natural history to play out for those patients that have cholestatic pruritus. They're post the successful CASI. They've gotten through the very young age where you tend to see a lot of transplants happening quite young. And throughout childhood can suffer from that cholestic pruritis. So expect similar treatment duration and profile to what you see in algeal syndrome, frankly. And then on the label, it's premature to really know what the precise label language would be. A lot of back and forth with FDA on the design of the protocol, but we don't really speculate on the label or indication statement at that point. So expect that to be part of the SNDA review. Thank you. Thanks for the question. Your next question comes from the line of Swayampakula Romakunth with H.C. Wainwright. Your line is open, Swayapakula. Please go ahead. Thank you. This is R.K. from H.C. Wainwright. One quick question in terms of trying to identify patients and how to think about launch economics. How concentrated are potential patients within the existing Livmarly prescribers? And in terms expenses, what incremental patient-finding commercial X investment do you need to outlay as you get this expand label you know over the next year or so thanks to the question RK you have highly concentrated in the same prescribers that currently prescribed with Marley and LGL syndrome and fee fit and then your second question no incremental economics So it's the team we have out there now. Thanks. Thank you for taking the question. Your next question comes from the line of John Wollobin with Citizens. Your line is open, John. Please go ahead. This is Catherine on for John. I just have another follow-up question on the opportunity. So you're seeing a thousand pediatric patients with VA. I'm wondering how many of those patients do you have the option to identify that have itch or does that number include patients that have ish? And also when you say the opportunity is similar to algea syndrome, do you mean in size or in like the opportunity given the increase in price in the patients? Thanks for the question, Katherine. I appreciate the opportunity to clarify that. Yeah, it's really addressable patients. So the 1000 patients in the US for biliary atresia, Those are biliary atresia patients, post-CASI, with their native liver, and that have puritis. So those are truly, you know, the profile of patients that you saw in this study that we'd expect to have on a label, you know, versus an overall sort of prevalence number. And likewise for allogeal. That is 1,000 addressable patients, alloegeal patients with their Native liver who have puritus, you know, that would kind of fit in the current allogee syndrome label. So that's what we mean. that we're really comparing truly addressable patient numbers for both. Perfect. Thank you for the clarification. Thanks for the question. Your next question comes from the line of Egal Nuchamuvitz with Citigroup. Your line is open, Egal. Please go ahead. Hi, Greta. Hopefully you can all hear me now. Thank you. So maybe you could address one of these. I'm not sure exactly what was already asked. But could you comment on how you're going to handle the six adult patients, if you haven't discussed that already, how that's going to work its way into the SNDA? If that was addressed, if he could comment briefly on the diarrhea, was there any prophylaxis like lopiramide or anything like that done, or was that not necessary? And then if that was already discussed, then if he can comment briefly just on this expanded population? How much are they concentrated already within the liver centers where Livmarly is prescribed, or if it's going to require some additional market diligence to gather those patients? Thank you so much. Yeah, you got it. Thanks for the question. I'll touch on the last part first and pass it over to Rob. In terms of identifying the patient population and where they're at. It was actually the interest and demand from investigators and prescribers initially that drove this study design and the interest to do this study. So it was from compassionate use and off-label interest that kind of led to the expanded design. So just an example to say that these patients are with the same prescriber for allogeal and in particular in the pediatric setting, and so it's very efficient in terms of being able to get to them and identifying those treatment candidates. I would maybe talk about some of the diarrhea treatment and the adult patient dynamic. Pass it over, Rob. Thanks, Chris. In general, I'd say the diarrhea profile is consistent with what we observed in the past with Mariluxibat and other IVAT inhibitors. Roughly 60% of patients did have diarrhea. this typically occurred early on in treatment and was short-lived. All of these cases were mild to moderate and no patients actually discontinued due to diarrhea. And in answer to your specific question, we did not use prophylaxis with loperamide for this. Thanks for the question. There are no further questions at this time. I will now turn the call back to Chris Pete's CEO for closing remarks. Great. Well, thanks everyone for joining today. And again, a big thank you to the Miriam team for making moments like this possible for the company and importantly for patients living with rare disease. This concludes today's call. Thank you for attending. You may now disconnect.