Mirum Pharmaceuticals, Inc. (MIRM) Earnings Call Transcript & Summary

Welcome to Mirum Pharmaceuticals conference call to discuss the FDA approval of LIVMARLI. [Operator Instructions] I will now turn the call over to Mirum's CFO, Ian Clements. You may proceed.

Thank you, operator. Good afternoon, everybody. Thank you for joining us to discuss the exciting news we announced earlier today regarding the FDA approval of LIVMARLI or maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome. With me on the call is our President and CEO, Chris Peetz; Chief Operating Officer, Peter Radovich; and Chief Strategic Officer, Pam Vig. Before we begin, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements about Mirum and management's current expectations, including statements regarding Mirum's business plans, development programs, strategies, prospects, commercialization and market opportunities, financial forecasts and guidance, anticipated FDA interactions or announcements and certain other business matters. Forward-looking statements are subject to numerous and substantial risks and uncertainties that could cause actual results to differ materially from currently expected results. Please review Mirum's annual and quarterly reports filed with the SEC for information on these risks and uncertainties. The forward-looking statements are added on today's date and Mirum assumes no obligation to publicly update these statements except as may be required by law. With all of that said, I would like to turn the call over to Chris. Chris?

Thanks, Ian, and good afternoon, everyone, who's joining us today. Today is a landmark day for patients and families living with Alagille syndrome who have been waiting for new treatment options and a remarkable inflection point for Mirum with our first product approval. Alagille syndrome remains the leading genetic cause of pediatric liver transplants, with many of these transplants being carried out to relieve the severe pruritus and systemic burden of disease that Alagille syndrome patients endure. Earlier today, we announced the FDA approval of LIVMARLI oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome of 1 year of age and older. LIVMARLI is the first-ever FDA approval in this indication and the first FDA-approved product for Mirum. We'd like to thank the team at the FDA for their hard work and collaboration throughout their priority review of the LIVMARLI application. The need for new therapies in Alagille syndrome is stark, with 6 and 10 children progressing to liver transplant or death by adulthood. Severe pruritus in Alagille syndrome is tremendously debilitating and can be unrelenting and is a leading indication for liver transplant. The burden of this disease is hard to overstate. We believe this community deserves better. Our mission is to deliver life-changing new medicines for the treatment of rare liver diseases. We are humbled to now be able to deliver LIVMARLI to patients. And we believe this is just the start as we work to expand access for LIVMARLI worldwide and advance the rest of our pipeline. We are incredibly grateful to the patients, their families and their caregivers who participated in our clinical trials and inspired us continually as we work to advance LIVMARLI. I would also like to thank the health care professionals, academic partners and all others who made LIVMARLI clinical studies possible. Finally, thank you to the entire Mirum team for your unwavering commitment to patients and meaningful scientific discovery and working with purpose and urgency for the approval of LIVMARLI. It's now our top priority to ensure that every patient in the U.S. with Alagille syndrome who could benefit from LIVMARLI gains access and receives treatment. Our commitment is that no patient goes without treatment due to financial reasons. To that end, we've established Mirum Access Plus, or MAP, a patient access program designed to provide end-to-end support for Alagille syndrome patients and care workers. Now during today's call, Pam Vig, our Chief Scientific Officer, will discuss our FDA-approved label and the data supporting today's approval; and Peter Radovich, our Chief Operating Officer, will walk through our launch plans, the cost of therapy and our patient support programs. With that, I will now turn the call over to Pam. Pam?

Thank you, Chris. To reiterate Chris' comments, we are absolutely thrilled to be able to offer a new medicine to children suffering from cholestatic pruritus in Alagille syndrome. Pruritus in this setting is devastating and debilitating, with the itch often being described as fire ants under the skin, an unreachable itch and patients often wind up with bloody sheets having scarring of the skin and sleep deprivation, all of which cause a significant impact to the patient, the family and an extensive caregiver burden. Pruritus and Alagille syndrome can be so significant that it is a leading indication of a liver transplantation. And until today, there have been no approved therapies for Alagille syndrome patients. LIVMARLI is now available as a new treatment option in this setting, and we're very pleased with the FDA-approved label for LIVMARLI. LIVMARLI is a minimally absorbed, orally administered inhibiter of the ileal bile acid transporter, also known as IBAT or ASBT. Elevations of serum bile acids is one of the hallmark features of cholestasis in Alagille syndrome and the blocking of IBAT results in lower levels of bile acid systemically and in the liver, thereby reducing bile acid-mediated effects and complications in cholestasis. Now I'm really excited to share some of the key highlights today regarding efficacy and safety of the filing. The key outcome measure from the pivotal ICONIC study, which formed the basis for FDA approval, was pruritus as measured by ItchRO(Obs), which captures itch severity reported by the caregiver. Patients entered the study with a mean baseline score of 3.1 on a 0 to 4 ItchRO scale. Meaning these children have severe pruritus at baseline despite ongoing background anti-pruritus therapy. Pruritus was reduced by a robust 1.7 points at week 18 in the overall group with a difference of 1.4 points between LIVMARLI and placebo at week 22, with a p-value of less than 0.0001. These reductions are dramatic and clinically meaningful, with greater than 80% of patients having a pruritus response. Additionally, these results were strongly correlated across several different pruritus measures, including caregiver reported, patient reported and clinician reported, demonstrating the consistent treatment effect of LIVMARLI. And in addition, the pruritus effect seen in the ICONIC study continue to be durable through more than 5 years of follow-up. Serum bile acids were significantly reduced in the majority of patients as early as week 12 and the reductions are maintained. Other clinically relevant results from the study included improvements in clinician xanthoma severity scale, pediatric quality of life measurements, which includes the multidimensional fatigue score as well as improvement in high disease score. All of these critical measures are significantly improved with LIVMARLI further demonstrating the clear clinical benefit in patients with Alagille syndrome. Now on the safety side, LIVMARLI has been studied extensively for nearly a decade, and its safety database represents the largest cohort studied in Alagille syndrome with an IBAT inhibitor. LIVMARLI safety data is based on 5 years of follow-up across the cohort of 86 Alagille patients who enrolled in the LIVMARLI clinical program. The most common adverse events across this extended follow were GI events, liver transaminase elevations, fat-soluble vitamin deficiency, all of which are also part of the background of Alagille syndrome. Safety events are also written into the label as a number of events per 100 person-years and this view of the safety data really puts into perspective the incidence rates over time. Characterization of long-term safety in a chronic disease setting is particularly important, and we are very pleased with how the final labeling presents these observations from 5 years of treatment exposure. So taking all of this data together, LIVMARLI is the first medication shown to deliver durable and clinically needful improvements in cholestatic pruritus and Alagille syndrome and introduces a new treatment paradigm for this setting. These exciting data suggests the potential of LIVMARLI to extend transplant-free survival due to significant improvement of pruritus. And with that, I will now turn the call over to Peter to discuss our plans for the commercial launch of LIVMARLI in the U.S. Peter?

Thank you, Pam. First, I want to join the team in expressing my excitement about the significance of today's approval for the Alagille syndrome community. I'm proud of our team here at Mirum, this dedication to uncovering the potential of LIVMARLI for the rare liver disease community never wavered. As we have shared previously, our commercial team is uniquely designed for rare pediatric liver disease. And our field teams have been deployed since June, engaging with health care professionals who manage patients with Alagille syndrome as well as payers. In fact, at this point, our field commercial and medical teams have had extensive interactions with nearly all the experts who manage Alagille syndrome patients. Our profiling efforts give us confidence in the estimated addressable population of 2,000 to 2,500 children with Alagille syndrome in the United States. Crucially, we are encouraged by the receptivity to LIVMARLI's strong value proposition in Alagille syndrome from both health care professionals and payers. With the FDA approval of LIVMARLI, the team will now begin active promotion to health care professionals in the 120 key accounts we're managing the majority of patients with Alagille syndrome. We'll also be working with regional and national payers to accelerate the time to a reimbursable pathway for LIVMARLI. I'm excited to say that LIVMARLI is now available for prescribing, and we're ready to receive LIVMARLI enrollment forms immediately. Turning to access and pricing. Given the strength of the data in Alagille syndrome, we believe that LIVMARLI has a robust value proposition as the first and only medicine ever approved for use in this disease. There are many factors that contributed to determining the price of this therapy, including the strong clinical benefit to patients and families, the severity of the disease with 6 and 10 children progressing to transplant or death by adulthood. Keeping in mind that pruritus is a leading indication of liver transplants, the size of the patient population with an estimated 2,000 to 2,500 pediatric patients in the U.S. and the lack of any other FDA-approved treatment option. LIVMARLI is formulated as a convenient, non-refrigerated, grape-flavored liquid in a multi-dose bottle. The dosing table in the prescribing information describes the once-daily dose in milliliters that the physician prescribes based on the patient's weight. As is standard for liquid specialty medicines, the milliliter will be the billable unit to payers for LIVMARLI. The cost per unit will be $1,550. For a 17-kilogram child, which is in line with the average weight of patients in our studies, we expect the gross annual cost of therapy to be approximately $391,000. We believe that this approach is comparable to other products serving similarly sized patient populations and diseases of similar severity. Most importantly, Mirum is fully committed to ensuring LIVMARLI is accessible to patients who could benefit from this therapy. To realize this vision, we have established Mirum Access Plus, or MAP. This patient access program is designed to provide end-to-end support and the central point of contact for Alagille syndrome patients and caregivers as well as health care professionals to help provide treatment support, understand the insurance process and financial assistance options. One of our goals with MAP is to ensure that patients who are uninsured, lack coverage or need assistance with their out-of-pocket costs are able to receive treatment. In fact, patients will have very low out-of-pocket costs for LIVMARLI, with none expected to exceed $10 per fill. Further, for eligible patients without insurance coverage, we are committed to providing LIVMARLI free of charge. The dedicated team behind MAP has been staffed, trained and fully operational since August to support patients and health care professionals initiating treatment with LIVMARLI. We are focused on product availability and patient support during this important launch period. So looking forward, we expect a gradual and steady build for LIVMARLI revenue in the initial quarters of launch as we navigate payer and physician dynamics in an undeveloped Alagille syndrome market where we're introducing the first FDA-approved medicine and establishing a new treatment paradigm. In some circumstances, the time to reimbursement could take up to 12 months as commercial and Medicaid payers establish new-to-market coverage policies. We will continue to work with payers to accelerate the reimbursable pathway for LIVMARLI. And most importantly, we will ensure patients have access to LIVMARLI immediately through our MAP program. Having said this, we believe the value of the LIVMARLI to patients is high and that over time, the uptake will reflect the true value of this therapy. We believe that the market opportunity in Alagille syndrome in the United States is in excess of $500 million. So as we've outlined today, the approval of LIVMARLI is an important advancement for Alagille syndrome patients and an exciting milestone here at Mirum. We're thrilled that have the opportunity to deliver this therapy to patients with Alagille syndrome and we're very eager to get started. I will now turn the call back over to Chris for closing remarks. Chris?

Thanks, Peter. In summary, as the first and only FDA-approved treatment for cholestatic pruritus in patients with Alagille syndrome, LIVMARLI represents a significant advancement for the Alagille syndrome community. Mirum's mission is to develop transformative treatments for rare liver diseases, and we take inspiration from our name, Mirum, which in Latin means remarkable, as we develop remarkable therapies for these deserving communities. To that end, we are proud to say that LIVMARLI is available immediately for prescribing in the U.S. And looking beyond the U.S., as a reminder, we recently submitted our application in Europe for the treatment of cholestatic liver disease in patients with Alagille syndrome, which was validated by the European Medicines Agency this week. This application includes data from a new analysis showing LIVMARLI significantly improved event-free survival compared to a natural history cohort, which is planned to be presented at an upcoming medical congress. Alagille syndrome is just the start for Mirum with continued development of our pipeline of late-stage programs and 5 additional rare liver diseases, an emerging gene therapy program and the trajectory of growth ahead. Finally, I'd like to close my remarks by again extending my most sincere appreciation to the patients, their families and their caregivers, clinical investigators, the Alagille syndrome Alliance, Mirum employees and support staff and our investors, without whom, this momentous day could not have occurred. And with that, I'll ask the operator to open the call for Q&A. Operator?

[Operator Instructions] The first question will come from the line of Jessica Fye with JPMorgan.

This is Daniel for Jessica Fye. Congratulations. In the prepared remarks, you highlighted a gradual build as you launch LIVMARLI. Maybe can you help us elaborate at that point and maybe help us that expectations for the cadence over the next couple of quarters? And then my next question is...

Sorry, go ahead. Yes, go ahead, with the second question.

I guess my second question is, how soon do you expect patients to start transitioning from the expanded access program to the prescription of LIVMARLI? And do you expect there to be an initial bolus because of that?

Great. Thanks for the question. I'll just -- I'll start with the second one first and -- on the extended access patients. They will, essentially, with their next fill, move over to commercial supply. So we do expect to see that in the early months of launch. But it's kind of the underlying dynamic through the launch of payers is worth some comments, and I'll ask Peter to speak a little bit about how we see the launch rolling out in the quarters ahead.

Sure. Yes. Happy to provide a little bit more color commentary behind adoption and our thought process to progress to a gradual build. There's probably 2 comments there. One point is that Alagille syndrome is an undeveloped market. This is -- we're really transforming the treatment paradigm here with the first ever FDA-approved medicine. So that the big of -- that quite a bit distinct to a market where maybe you're launching a fifth in class, where #5 is a little bit different than one through 4. Those kind of products can get to their peak or get to where they're going pretty quickly as compared to a setting where you're really building a market from scratch. The second point is around on the payer side. And what we're expecting on the payer side is not unique to LIVMARLI . If you just look at rare disease launches, when you see it, it takes time for payers to establish new-to-market coverage policies. So on the commercial side, often you'll see NDC blocks in the early quarters. And as I mentioned, in some instances, it can take as long as 12 months. On the Medicaid side, this is Mirum's first product. So we have to get a rebate agreement in place with CMS, which we expect to occur in early '22 and in some cases, -- some of the 50 states, we'll be able to go right then and others will take time from there. So we'll be updating you as we go through the launch process on what the picture looks like in terms of pathway to reimbursement across the different Alagille patient payers.

The next question will come from the line of Mani Foroohar with SVB Leerink.

Two quick ones. I think, first of all, as you have been engaging with the treating physician and getting a better sense of where the diagnosed patient population currently lands, is there any gap between that 17 kilogram median patient in the pivotal study and what we should think about as a reasonable sort of weighted average weight for patients in the diagnosed patient population versus the undiagnosed patient population? And then I have a quick follow-up.

Thanks for the question, Mani. So we -- as we've looked at different data sources, both from our clinical trial program as well as others, we do think that the 17-kilo average is representative of what we're likely to see in the real world, and that was the average at baseline on entering the clinical trials. We have observed growth on our clinical trials. I think that's important to be aware of. And so that -- I think that's our expectation. Most of the patients are less than 30 kilos. So the distribution, that's -- 17 is the average. There's a distribution certainly below that. And then in the pediatric population, really, it kind of ends around 30 kilos.

Great. That's helpful. And I guess, secondarily, when you think about modeling timing of rollout of other -- of rollout and economics in other geographies, Europe, Japan, et cetera, can you walk through how we should think about that? And given that you've got a couple of different partnerships you announced recently, can you give us some sense of where the economics would fall versus the U.S. in Japan, Europe, Brazil, some of these other markets, some of which you're sharing [ with a ] partner?

Yes. Thanks for the follow-up there. The short answer is that will start to show up next year. And we'll continue to have the various partner markets pick up access approvals or reimbursement in the years that follow. And the first -- it's very different by geography. The earlier ones that we would expect to see is initial access in countries like Israel or countries that have reimbursed early access programs, of which there are a couple in Europe, for example, that we're working on. On the Asia side, we do expect China and Korea to be relatively soon, end of 2022 or early 2023. Japan will -- our partner there, Takeda, is planning to run an additional study. So that one will be sequenced after China and South Korea.

The next question comes from the line of Josh Schimmer with Evercore.

And more importantly, thank you for bringing forward this important treatment option for this incredibly tremendous unmet medical need. I have 2 questions. First, for the 2,000-plus patients that you estimate in the U.S., how many of those patients have you actually identified and how many are in the expanded access program? And then second, the label for dosing says up to 380 micrograms per kilogram per day. Some of the extended Alagille trial evaluation, the dosing went up to nearly 2x that level. The PFIC study is valuating nearly 3x that level on a daily basis. So how are you thinking about ensuring all patients are able to get access to the right amount of drug for them? What do you think that's going to be for Alagille and for PFIC? And do you expect payers are going to allow patients to go higher than what's on the label?

Thanks for the question and thanks for the thoughts. On the -- starting on the patient finding side, we found -- we're -- first of all, we're not going to -- we're not disclosing the exact numbers on this, but I will say that we are -- we finally identified through our field team and the genetic testing program, a substantial number of those 2,000 to 2,500 that we're targeting. So do have our hands around where our first calls were this afternoon already as the team has been reaching out to a lot of the physicians that are engaged in the program. There's a little bit of a head start in just how experienced the KOL community is with this program. We have nearly 30 sites that have been involved in some way with the maralixibat studies for expanded access program. So that helps with kind of knowing where these patients are and who's going to be some of the first to treat. And then last point on the expanded access program that you asked about. Those patients will rotate in. We haven't disclosed an exact number on how many patients are coming from the expanded access program. [ We ] have a sense of the scale of it from looking at the clinicaltrials.gov posting still and see a lot of this initial outreach in the launch period where we have sites with patients identified ready to prescribe as kind of unfocused in the coming months. So I'll pass it over to Pam actually to speak to the dosing question.

Yes. Thanks very much for the question. So regarding dose, following 5 years of evaluation in the study, we determined that 380 micrograms per kilogram per day is an effective dose, in which we see over 80% of patients responding on that dose. Now you're right, in the long-term extension, the dose would double for some patients, but the dynamic of that is that the ItchRO score on aggregate in that group was already below 1, meaning those kids had little to no itch already. So doubling of the dose, really, there wasn't much further to go on their response as they're already responding [ to TD ]. And the dynamic with PFIC is that we know that dose matters, and we know this from experience from our INDIGO study. All PFIC studies to date have about a 30% serum bile acid response and about a 50% pruritus response. And that's what we've kind of seen across the board in the PFIC studies. And we know that with higher doses from our experience in INDIGO that when we double the dose, we had additional responders. And so for our March PFIC Phase III study, we're going even higher than that, and hopefully, we'll be able to have more data to share with you next year.

The next question will come from the line of Yasmeen Rahimi with Piper Sandler.

First of all, congratulations for getting the drug approved and a tremendous amount of dedication came through all of you and everybody at Mirum. So many, many congrats I'm sending you. I have a number of questions for you. Maybe a good place to start as a follow-up to Josh's question. Thank you, Chris, for noting that a substantial number of the 2,000 to 2,500 patients have been already identified. Can you comment if you think substantial -- your wording of substantial is at least more than 60%? So just giving us a range of what you mean with substantial could be helpful. And then the second question is, for you, is like how much of the clinical data or what type of the clinical data will make it into the actual label? Will there be the bile acids in the label? Will there be that drive to your follow-up data in the label that could be helpful? And then I have a follow-up.

Great. Thanks for the question. Yes, first, starting with the patient finding, and I wanted just to kind of be very open about it. One of the reasons why we don't share those numbers is, we're looking at different sources, and it's hard to see where the overlap is. And at this point, we don't have that kind of precision. We feel very comfortable that there are 2,000 to 2,500 addressable patients out there. And we're very active in making sure that their care providers know about LIVMARLI as a treatment option and enabling access for them. And -- could you remind me of your second question, actually?

Yes. The second question is what type of the data will make it into the clinical section of the label? Will it just have the pruritus improvement?

So the label will focus on pruritus. It is posted and available now. I'll let Pam highlight a couple of the other features on the efficacy side and pharmacodynamics that are presented in there.

Yes. Thanks. So one of the things that is in the label right now as Chris mentioned, we -- the indications for cholestatic pruritus and Alagille syndrome. So the label reflects that and the pruritus response seen there, which is very significant with the majority of patients responding is at 80% responding at the 380-microgram per kilogram per day dose. Serum bile acids is also in the label under the pharmacodynamics section. So we were able to show the serum bile acid reductions are seen very early, as early as week 12 and are maintained and now we know through 5 years. And one of the things that we are really excited about right now is we have new data demonstrating improved long-term event-free survival and transplant-free survival, with maralixibat treating patients when we compare it to a natural history cohort. This is a 6-year natural history comparison. The p-value is less than 0.0001, and we're really looking forward to presenting that in the upcoming medical conference.

And how soon do we -- would that publication be in print so that the medical liaisons could actually utilize that data as they go out to speak with physicians?

I think that -- so there's a couple of data points there. One is we have our primary manuscript on the ICONIC, which shows 5 years of data. That is hopefully coming out relatively soon, imminently. And for the 5-year event-free survival, transplant-free survival data, we're hoping that, that will be presented at the Congress very soon as well. And as soon as that's out, we'll be posting that on our website, you'll be able to see the data then. And the sales will be able to talk about as soon as it's reflected.

Great. And then just one follow-up question, if I may ask, and this is in relation to the GI tolerability that you guys really nicely quantified over a long term. Is there utility on being able to present this too? And I don't know if that made it into the label, just making the point that the GI tolerability occurs early and then subsides. So just kind of comment on how that data could be used in discussions with physicians?

Yes. Thanks for the question. We've actually already presented that data twice this year at 2 different congresses, one at ESPGHAN and one at EASL in 2021. And that's on our website. But what we see is that diarrhea is early on treatment onset within 4 weeks. It's transient. And it's mostly mild in severity, and the majority resolved within 2 days. There have been no discontinuation for diarrhea across 5 years in all patients. And I think perhaps most importantly, when we reviewed this data with our advisers, the transient and mild diarrhea really at the onset of treatment is not viewed as an issue, nor will it limit the use of LIVMARLI. But -- yes, go ahead.

I'd just add one comment on that, too, and how this shows up in the label and how it was managing the discussions with FDA. It's a really unique data set to have 5 years of safety follow-up in a rare disease approval. And the way that it's been presented here with the 100 person-years exposure really shines a light on how well tolerated and tangible the AE profile is. Kind of state it in another way, when you -- if you were to have 100 patients followed for 365 days, you would expect 41 events, which from the background of the disease really is not that noteworthy.

The next question comes from the line of Brian Skorney with Baird.

This is Luke Herrmann on for Brian. First, congratulations on the approval. So first, I guess, could you remind us maybe of the dispersion of the patients across the 120 target accounts? And then could you just compare and contrast the EU and U.S. markets a little more, maybe similarities and differences and how the U.S. efforts will carry over to EU?

Yes. Thanks for the question. Just starting from the second one. Alagille syndrome, it really is -- shows up proportionate to population. Given that it's frequently a dominant mutation, you can really just look at differences in population by geography and expect a very similar rate of incidence and prevalence. And I'll ask Peter, actually, to comment on some of the account profiling and where these patients sit across the various types of accounts.

Sure. Yes. So we talk about 120 accounts deliver the vast majority of the care for Alagille syndrome, probably north of 80% of the care delivery in the United States based on claims data. And certainly, within that 120, I don't know if I can give you a precise quantification at this moment, but the top 60 are even more than half, right? So it's really -- it's very concentrated at 120. And then within that, it gets increasingly concentrated as you move up in terms of volume.

Great. And then maybe just one more quick one on safety, if you don't mind. Just on the less frequent AEs of bone fracture, GI bleeding, do you think these are more indication-specific? Or is there anything else that you can share on those?

Yes. Thanks for the question. A couple of points to think. First, one important point is that the ADR table represents all events greater than 5% of patients regardless of related event. And so when you follow patients for 5 years, you would expect to see the background of the disease showing up for sure. With regards specifically to your question on bone fractures, this is again, 5 years of data. And the background is that about 28% to 38% of fracture rates are reported in Alagille patients, most of these occurring before the age of 13. And so with LIVMARLI, we see 3.3 events per 100 person-years. And I think that really helps put this into perspective. So nothing in these rates indicate that this is outside of the background of the disease.

The next question will come from the line of Ed Arce with H.C. Wainwright.

Great. And let me add my congrats on the approval of LIVMARLI . A lot of work I know went behind us, both at Mirum and before. So it's -- kudos to the whole team. First question is helping us to understand how we should think about these first patients and then more of a steady state, how should we think about delineating the patient journey through the Mirum Access Plus program? In particular, given payer coverage is obviously a gating factor early on. How should we think about the process that goes on there? And while some payers may take up to 6 months -- or excuse me, up to 12 months, how should we think about the cadence early on proportionately number of payers that could come in within the first few months? And then I have a couple of follow-ups.

Thanks, Ed, for the question. First comment on that, as we move into tomorrow and start taking prescriptions, our focus is on getting patients started. So the kind of payer access and payer element is somewhat separated from getting to patient starts, which is the core focus of the coming months. And I'll let Peter speak to some of the specifics of what that patient experience is like and somewhat of a difference between covered and uncovered route to access for drug as we pick up that reimbursement over the months ahead.

Thanks, Chris. And thanks for the question, Ed. And I'll provide some overview of comments here, but we actually have a page on our corporate deck that gives you a little bit more detail on the journey that you can go look at. But essentially, it starts with the patient enrollment form or a prescription from the physician. That's actually now available on livmarlihcp.com, and that can be submitted into the MAP program. And then the program looks at verifying patients' insurance status, submit the claims of the insurance. In some cases, there'll be an appeal to support with that and then there'll be a dispense from there. And as Chris noted, in some cases, and you noted in your question, indeed, there isn't coverage, especially in the early months post approval. This is a common phenomenon in rare disease. And so for those patients, they can qualify to receive free drug if they don't have insurance coverage. And that will enable us to get patients started on therapy and give them an opportunity to benefit from this therapy immediately as we work through getting a reimbursable pathway in place with payers. And I think the other point to note is really MAP, I'm commenting a lot on the upfront piece, the prescription to fill piece, but this is a chronic disease. And we saw over 80% of patients had a pruritus response in our clinical trial -- clinical trial [ extensive ] patients on more than 5 years. So MAP is also set up to provide long-term support for long-term adherence in the real world so that we can transfer these outcomes to the real world that we saw in the clinical trial.

Great. Okay. That's helpful. So a couple more for me, and this first one sort of dovetails into what you were just saying. And that is, could you help us with our expectations going forward for the next 2, 3 quarters on what we can expect for early launch metrics that you could be providing on regular quarterly earnings calls, third quarter, fourth quarter, first quarter of next year as you begin to get patients not only on drug, but then also eventually reimbursed and covered as well? I would imagine that the patient enrollment forms in particular, that you mentioned would be key, but if you could help us understand that? And then the other question is around Table 1 in the label, the individual dose volume. Just wondering if you could comment, maybe Pam could, how physicians view this algorithm in terms of convenience, especially relative to other volume liquid drugs? And that can lean, especially given there's 13 rows of different volumes here. So any thoughts there would be helpful.

I'm happy to start on the question about metrics. So we will be providing adoption metrics in the early quarters of launch. And the goal there will be to really help you characterize the launch performance and get a picture of adoption at the physician and the patient level as well as on the payer side, as you're noting. So we'll certainly be providing more detail on that as we go through the launch process. And I think maybe I'll make a comment on the dosing table and ask Pam to chime in. We've done a tremendous amount of Advisory Boards and interactions with physicians at these 120 accounts. And the feedback we've received from pediatric hepatologists and nurses and others, is this is actually a very simple dosing regimen. The grape-flavored liquid is preferred. They're pretty accustomed to doing weight-based dosing for pediatric patients. When they do the quarterly visits, it's part of the standard orders to relay the patient and redose. So the feedback we've received is that, that will be a pretty straightforward approach to adopting this product.

Yes. And I would just echo that, that it's pretty simple and straightforward. It's given like a children's TYLENOL. The weight is checked and the volume is distributed. It's pretty basic.

And sorry, just to chime in with an additional comment. Compared to the long list of medications, complex weight-based dosing that is already present in this setting. And that this, as we've seen in patients on study and in the expanded access program, this can start to replace some of those other therapies. They will come off with some of the more complex dosing regimens. This is a huge improvement for what these families work with now.

The next question comes from the line of Steven Seedhouse with Raymond James.

Congratulations on the approval on Alagille. Ryan Deschner on for Steve. Just wanted to ask, what's the difference between the FDA and MA labels you might be expecting in Alagille? And then as a follow-up, I wanted to check in to any further update on the [ pediatric voucher ] or expected proceeds?

Thanks, Ryan. And we lost you on the second part of the question, but I'll go ahead and answer the first and then see if we can get you to repeat it. And on the differences between the U.S. and European filing packages, really comes down to this new data set on long-term event-free survival driving slightly different verbiage on the European label. FDA has, from the beginning, been very consistent on using pruritus as the outcome and the basis for the efficacy statement that you see in the package insert ties to what these families are dealing with day to day and one of the most common contributing factors to transplant. That's why you see the indication statement of treatment of cholestatic pruritus in the FDA label. The proposed European label will be for treatment of cholestatic liver disease and Alagille syndrome. And that difference in wording is driven by the inclusion of long-term outcomes on liver-related events. So the data that we announced a couple of weeks ago showing that LIVMARLI showed an improvement in event-free survival compared to a natural history cohort on time to surgery, hepatic decompensation transplant or death. Could you repeat the second portion of your question?

Sure. Just wanted to check in on -- see if there were any further updates on the pediatric voucher or the priority review voucher sale in terms of time line or expected proceeds?

Yes, sure. Thanks, Ryan. Yes, as part of the FDA communication, we did indeed receive the priority review voucher and we are intending to monetize said voucher. At this point, we're evaluating the options and with respect to the timing of the [ VAT ] monetization. But I think as we look across [ a bit ] here, as we look across the balance sheet, with just shy of $240 million over the last -- kind of the last quarter, headline is that we have in excess of 3 years of runway.

There are no additional questions waiting at this time. So I'll pass the call back to Chris Peetz to provide closing comments.

Great. Thank you, operator, and thanks to everyone for joining today's call. It is a big day for the Alagille syndrome community, and we are so excited about the availability of LIVMARLI for prescribing. Thanks to all involved in making this important step forward possible.

This concludes the conference call. Thank you for your participation, and enjoy the rest of your day.