Mirum Pharmaceuticals Inc

NASDAQ:MIRM

Earnings Call Analysis

Financial Performance and Revenue Growth

In the last quarter of 2023, the company reported a notable surge in revenue, clocking in at $69.6 million, which showcased significant growth from $27.9 million in the same quarter of the previous year. For the full year ended December 31, 2023, the amassed revenue stands impressively at $186.4 million compared to $77.1 million in the year prior, reflecting a sizeable increment. Mirum's flagship product, LIVMARLI, contributed a solid $41.4 million globally in the quarter with $31.4 million from the U.S. and $10 million internationally. Meanwhile, the bile acid products added $28.1 million to the quarter's earnings.

Product Portfolio and Growth Outlook

The company does not share detailed guidance at the product level, but the management has expectations of mid-single-digit growth for its bile acid product portfolio based on historical trends. They express confidence in the continued strong market performance of LIVMARLI in the U.S. and anticipate variability in its international sales. Amidst the competitive landscape, LIVMARLI's growth trajectory has not been significantly impacted, and the company expects to maintain its momentum across its product lineup.

Dose Adjustments and Patient Growth

An interesting dynamic observed is the dose adjustment for pediatric patients receiving LIVMARLI as they grow. The company has reported that this has been a consistent pattern, indicative of the evolving needs of their patient demographic as they mature.

Cost Management and Margins

Gross margin expectations take into account the ongoing intangible amortization from the bile acid acquisition, spread over 50 quarters. The company anticipates this to continue at a similar percentage of sales. A significant inventory reserve noted in the fourth quarter is expected to be an exception rather than the norm in subsequent quarters.

Net Loss and Operational Health

Despite the upsurge in revenue, the company incurred a net loss of $160.9 million, or $3.94 per share, in 2023. This includes non-cash expenses like $35 million in stock-based compensation and $10.4 million in intangible amortization largely reflected in cost of goods sold. However, the cost management appears more efficient when considering the decreased cash used in operating activities, down to $70.9 million from $120.1 million in the year before. With a cash reserve of $286.3 million, the company remains in a robust financial position, advocating for its self-sustaining business model and capacity for growth through clinical development and expansion of its global commercial presence.

Clinical Development and Regulatory Prospects

Mirum continues to innovate, with several clinical and regulatory catalysts underway. For example, the company is working towards broadening the CHENODAL label to cover CTX, opening up more opportunities in international markets through increased disease state awareness and diagnosis rates.

Sales Guidance and Prospects

Looking ahead, the company projects net product sales in the realm of $310 million to $320 million for the year 2024. This forward-looking statement underpins the company's business confidence and the anticipated growth trajectory of its products, even as it refrains from sharing granular product-level forecasts.

Earnings Call Transcript 2023-Q4

[Operator]

Good afternoon, ladies and gentlemen. Thank you for joining today's Mirum Pharmaceuticals Reports Q4 [ Year-End ] and 2023 Financial Results and provides business update. My name is [ Tia ], and I will be your moderator for today's call. [Operator Instructions]

It is my pleasure to pass the call over to Andrew McKibbin, Vice President of Investor Relations and Finance. Please proceed.

[Andrew McKibben, executive]

Thanks, [ Tia ], and good afternoon, everyone. I'd like to welcome you to Mirum Pharmaceuticals Fourth Quarter 2023 Conference Call.

I'm joined today by our CEO, Chris Peetz, our President and Chief Operating Officer, Peter Radovich, our Chief Scientific Officer and Head of Research, Pam Vig, our Chief Medical Officer, [indiscernible]; and Eric Bjerkholt, our Chief Financial Officer.

Earlier today, Mirum issued a news release announcing the company's results for the fourth quarter and full year 2023. Copies of this news release and SEC filings can be found in the Investors section of our website.

Before we begin, I'd like to remind you that during the course of this call, we will be making certain forward-looking statements about Mirum and our programs based on management's current expectations including statements regarding current and future business plans, development programs and regulatory expectations, strategies, prospects, market opportunities and financial expectations. Mirum's under no duty to update these statements, and they are subject to numerous risks and uncertainties and actual results could differ materially from those results anticipated by these statements. Investors should read the risk factors set forth in Mirum's 10-Q for the quarter ended September 30, 2023, and the subsequent reports filed with the SEC.

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

[Christopher Peetz, executive]

Thanks, Andrew, and good afternoon, everyone. I'm excited to kick off an update on the many achievements for Mirum in 2023, another year of significant growth for us and cover highlights for what lies ahead for the company.

Our continued progress in 2023, an opportunity ahead reflects the dedication of the Mirum team to making a difference in the lives of patients and their families around the world. Over the year, we transformed our business with a broadened reach to patients in the U.S. and internationally, achieving $179 million in net product sales and 142% year-over-year growth in total revenue. This was driven by continued strong growth for LIVMARLI and the expansion of our commercial portfolio with the acquisition and integration of CHENODAL and CHOLBAM.

We have now built a growing self-sustaining leading rare disease business that is positioned for a great 2024 and beyond. The last year has shown the value creation potential of our strategy to drive growth in our commercial medicines, unlock the potential of upcoming label expansion opportunities, leverage our expertise in cholestasis in adult settings and continue to grow the pipeline.

Looking at the year ahead for execution on our strategy. Overall, from the current commercial business, we expect to achieve $310 million to $320 million of net product revenue in 2024. This is expected to be driven by growth across all 3 commercially available medicines. We also have multiple regulatory and clinical catalysts this year to advance our pipeline. For LIVMARLI and PFIC, we are on track for our PDUFA date on March 13. We're excited for the opportunity to bring the strong results of the [ March ] PFIC Phase III study to patients with a potential label expansion.

We're also preparing for the submission of the positive [indiscernible] Phase III results of CHENODAL and CTX in the first half of the year, an important label enabling opportunity. And we also made great progress with the VISTAS and VANTAGE studies for volixibat in PSC and PBC. Patients for the interim analysis have been enrolled, and we expect to conduct the dose selection interims of these adaptive studies in the first half of the year. We see the PSC program as an opportunity to bring the first ever therapy to market for this progressive burdensome disease.

I'm proud of all of our progress in 2023 and the potential ahead for Mirum. We look forward to continuing to grow the impact of our programs, providing life-changing medicines to patients with rare disease. Now before I turn the call over to Peter to discuss our commercial business, I wanted to welcome our new Chief Medical Officer, Dr. Joanne Quan, who joined Mirum in January. We are thrilled to have Joanne and her extensive leadership and scientific expertise, helping to drive the value creation we see ahead from Mirum. We'll hear from her later on this call.

And for now, I'll turn the call over to Peter to discuss our commercial business. Peter?

[Peter Radovich, executive]

Thanks, Chris. We are excited by the tremendous progress that we have made across our U.S. and international businesses. Coming off of 2023, where we saw 138% growth in total net product sales compared to 2022. We are already seeing the benefits of our newly expanded pediatric hepatology franchise and enhanced offering amongst the patient and prescriber community. I'm proud of the Mirum team's focus on patients while simultaneously driving the seamless integration of [indiscernible] and [ Pinedale ] And this is evident in the $69.6 million of total net product sales we achieved in the fourth quarter of 2023, which reflected growth across all 3 commercial medicines.

LIVMARLI, total global net product sales grew to $41.4 million in the fourth quarter. Our U.S. business saw $31.4 million and $111 million for the quarter and year, respectively, representing approximately 63% year-over-year growth. And internationally, we are seeing consistent strong uptake as well, but we continue to anticipate quarter-to-quarter variability in international revenue.

Moving on to CHENODAL and CHOLBAM. The fourth quarter was the first full quarter with these 2 medicines under Mirum's leadership, and I'm very happy with how quickly we have been able to transition these medicines into our business. In the fourth quarter, we recognized net product sales of $28.1 million, the highest quarter to-date for the bile acid products. And looking ahead, we expect steady growth for these 2 medicines in line with historical trends.

Taking all the strong dynamics in the business together, we expect net product sales to be $310 million to $320 million for 2024. This represents over 70% growth from 2023 with increased contribution from all commercially available medicines.

In summary, 2023 was a fantastic year for our team as we expanded our portfolio to 3 commercial products and grew our presence both in the U.S. and worldwide. We continue to see the tremendous impact of all 3 products across their patient communities, and we look forward to further expansion in 2024 as we stay committed to our mission of delivering life-changing medicines to patients worldwide.

And with that, I'll turn it over to Joanne. Joanne?

[Unknown Executive]

Thanks, Peter. Before I begin, I'd like to say that I'm thrilled to join Mirum. This experienced team has accomplished so much in such a short period of time. and I'm excited to continue the momentum and progress in the treatment of rare diseases.

In 2024, we are looking forward to multiple regulatory and development milestones. From a regulatory perspective, we are tracking well towards our PFIC PDUFA date of March 13 and expect a decision for the EMA in the first half of the year. Regarding CHENODAL and CTX, we're making good progress here as well. We've had positive interactions with the FDA and no additional clinical studies are required to support our NDA submission, which is planned in the first half of 2024.

Turning to volixibat. We're also looking forward to our upcoming interim analysis in our VISTAS PSC study and our VANTAGE study in PBC patients. We are on track for the interim analysis to read out in the first half of this year. Note that the studies are now continuing to enroll patients with the goal of supporting registration. As a reminder, the VISTAS PSC study will have a blinded interim analysis just to part dose selection. Based upon the data, this will allow patients from the selected dose and placebo arms to be included in the pivotal data set.

For the larger indication of PBC, the study is designed so that we [ unblind ] and show the top line results from the interim analysis, which will include data on pruritus improvement, change in serum bile acids and safety. Both of these studies represent an important step towards addressing the accumulation of bile acids in broader patient groups with adult cholestasis where a significant portion of patients are without adequate treatment options for the cholestasis and its severe symptomatic [indiscernible]. I'm excited to be part of this team and look forward to sharing our progress with [indiscernible].

With that, I'll now turn the call over to Eric.to discuss our financial results. Eric?

[Eric Bjerkholt, executive]

Thanks, Joanne. Earlier today, we issued a press release that included financial results for the quarter and full year, which I'll briefly summarize. Note, our 10-K filing is planned for next week as our auditors finalize the first 404(b) and other audit procedures for Mirum.

Total revenue in the fourth quarter 2023 was $69.6 million and for the year ended December 31, total revenue was $186.4 million, including total net product sales of $178.9 million. This is compared to total revenues of $27.9 million and $77.1 million for the fourth quarter and full year 2022, respectively.

For operating expenses for the year ended December 31 were $293 million, which includes research and development expenses of $102.6 million, SG&A expenses of $145.9 million, and cost of sales of $44.5 million. For the year ended December 31, 2023 net loss was $160.9 million or $3.94 per share. Net loss for the year included noncash stock-based compensation expense of $35 million and intangible amortization of $10.4 million. This intangible amortization is largely reflected in our cost of goods sold.

In the fourth quarter, COGS also reflected a reserve in excess of $5 million for inventory primarily related to the bile acid acquisition. For the year ended December 31, '23, our cash used in operating activities was $70.9 million, down from $120.1 million the year before. We had cash, cash equivalents and investments of $286.3 million as of December 31, 2023. We remain well funded with a strong and self-sustaining business. Our increasing revenue base places us in an exceptional position to grow the business through clinical development and continued expansion of our global commercial presence over the coming years.

Now I'll turn the call back over to Chris for final comments.

[Christopher Peetz, executive]

Thanks, Eric. It has been a strong year for Mirum, finishing with the biggest ever revenue quarter for LIVMARLI and the [ Biloxi ] portfolio. We are pleased for another great year in 2024 with 3 growing commercial products and with several clinical and regulatory catalysts as we strive to make continued advances for rare disease patients around the world. We look forward to keeping you updated on our exciting progress throughout the year.

And with that, operator, please open the call for questions.

[Operator]

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

[Unknown Analyst]

This is Nick on for Jess. Maybe looking at the 2024 net product sales guidance of $310 million to $320 million, can you maybe discuss a bit more about how much LIVMARLI growth is baked in there versus contributions from CHENODAL, CHOLBAM. And also comment a little bit on LIVMARLI Alagille maybe provide an update on what you're seeing on the competitive dynamics for [indiscernible], if any?

[Christopher Peetz, executive]

Yes. Thanks for the question. And first, just from a starting point, we're not giving product level guidance at this point, but really great dynamics across the products, and I'll let Peter kind of give a little color on what we're seeing across the business.

[Peter Radovich, executive]

Yes. And I think as you think about the component in some of the color commentary there, the bile acid product portfolio, we expect it to grow on historical trends, which have been kind of on the order of mid-single-digit year-on-year growth rates. And then looking into LIVMARLI, growth case in the U.S. has just been strong over the last overall quarters of its marketing. Expected to see it continue to move forward with that team. And then on the international side, we do expect quarter to afford variability there for LIVMARLI international sales.

And then your other question about competitive dynamics now as [indiscernible] I think as you can see from the numbers spirits, really seen a measurable impact. from those at all the LIVMARLI growth trajectory has been impacted by that.

[Unknown Analyst]

Great. And maybe just one more. Thinking about the VANTAGE study and how do Alagille patients [indiscernible] patients about the first-line setting second-line setting post [indiscernible], beyond the rate of [indiscernible] observed across these 2 settings, can you maybe talk a little bit more about the differences in how it presents and/or maybe the severity of itch that is observed in these 2 patient populations?

[Christopher Peetz, executive]

Joanne and I can speak to the difference between the line therapy.

[Unknown Executive]

Yes. So as you know, Pruritus is a big issue in both of these diseases. We have a broad patient population that we're accessing in terms of the [indiscernible] studies. And actually, there's -- this is broader than, for instance, some of the other entrants in this space who are looking at biochemical [indiscernible]. I'll remind you that we have agreement from FDA that provide us with the registrational end point and both these studies are actually positioned to be registration after the interim analysis. So we feel pretty confident in terms of both the [indiscernible] design the drug itself, our dose selection and moving forward at this point.

[Operator]

The next question comes from the line of Gavin Clark-Gartner with Evercore ISI.

[Unknown Analyst]

This is [indiscernible] on for Gavin. Just two questions for us. For the first one. For LIVMARLI, especially with the start of the year, just wondering if you have seen patients being reweighted and so on higher doses as they grow?

[Peter Radovich, executive]

We have and we published some of that information last year, not surprisingly, is a rate-based product. And the growth that we've seen published with LIVMARLI to see weight-based's dose adjustments.

[Unknown Analyst]

Right. And then one more for the EU business. Do you have any commentary on potential implications of [indiscernible] refiling [indiscernible] EU under a different brand name, not with [indiscernible]? And then I also want to confirm that there would be no reimbursement or access impact on LIVMARLI in the EU?

[Christopher Peetz, executive]

Good question. At this point, we're focused on getting access broadly across all international markets with LIVMARLI and [indiscernible] Alagille and having great progress as we see demand continuing to grow across Europe and other international markets. Can't really speak too much to the [indiscernible] procedure other than that we're making great progress getting with LIVMARLI, really rolled out a standard of care and [indiscernible] across all markets.

[Operator]

The next question comes from the line of Mani Foroohar with Leerink Partners.

[Lili Nsongo, analyst]

This is Lili Nsongo on for Mani. I'm sorry, I couldn't hear -- and I apologize if it was asked earlier, I heard a question, but we couldn't hear the answer. This was regarding the growth expected for [indiscernible] of [indiscernible] based increases as the patients get older and to add to that, if you could comment in terms of how should we look -- or should we think about the way and then the dosage for new patients versus existing ones?

[Christopher Peetz, executive]

Yes. So the question -- and just to make sure that I recap it here and ask for any color from Peter. Basically, the question -- the original question was around do we see adjustments to dose as patients are on therapy over time per the label. And we do see that over time. I think we mentioned that there's been a recent poster presentation on it. So that is a dynamic that we see out with the treating physicians. And so we're able to -- the microphone pick us up this time.

[Lili Nsongo, analyst]

Yes. Yes, we could hear you very clearly. And as a follow-up regarding the valuation on assets. Could you maybe comment in terms of what the international growth opportunities are for those assets?

[Christopher Peetz, executive]

Yes. So a question about CHENODAL, CHOLBAM growth opportunities. And I'll let Peter speak a little bit to the dynamic of what we have today and then also a little bit about the CHENODAL label expectations of expansion opportunity for CTX.

[Peter Radovich, executive]

Yes. I think with all products, we continue to see growth in line with historical trends, certainly with CHENODAL recently announced Phase III data for CTS and potential approval next year for CTS. I think there's an opportunity CHENODAL never been promoted for CTS before. So I think there's an opportunity to find more patients through disease state awareness and hopefully increasing the diagnosis rate. One thing we know about CTS is maybe only about 10% of the patients are diagnosed and under management. So the opportunity there is to try to identify more patients and on therapy.

[Operator]

The next question comes from the line of Mike Ulz with Morgan Stanley.

[Unknown Analyst]

This is [indiscernible] on for Mike. Can you just talk about your expectations for the upcoming LIVMARLI PDUFA in PFIC? And any launch preface associated? And do you think that any patients are currently using it off-label?

[Christopher Peetz, executive]

Thanks for the question, [ Rohit ]. On the regulatory front, we're where we expect to be so excited about the PDUFA date coming up. And I'll let Peter speak a little bit to how we're preparing for that and some of the first opportunities we see there.

[Peter Radovich, executive]

Yes. We're really excited about the potential approval for LIVMARLI and PFIC. I think the [ March ] data reinforced the strong value proposition that LIVMARLI offers to our various stakeholder groups, and I think that will certainly help us. The physician prescribing universe is essentially identical to the [indiscernible] audience. So it's really no need for meaningful operating expense increase to access it. And we do have a number of patients in the mid-20s who are receiving clinical rollover or expanded access to LIVMARLI who would be eligible to roll the commercial on approval.

[Operator]

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

[Unknown Analyst]

This is [indiscernible] for Steve Seedhouse. So our first question is related to the gross margin. I think you mentioned there was an inventory charge related to the acquisition in 4Q. Just what are your expectations for gross margin in 2024? And perhaps also the OpEx trajectory in 2024?

[Unknown Executive]

As for us, gross margin, we do expect that the intangible amortization will continue. I mean it's largely related to the bio acid acquisition intangibles, which we're amortizing over 50 quarters. As far as sort of traditional cost of goods, it consists of the actual cost of product and royalties. So that will continue at approximately the same sort of percentage of sales.

And then we did have a larger than we expect going forward amortization -- or reserve for the inventory that came with the acquisition. So we might have some reserve in some quarters, but not to the extent we had in the fourth quarter.

In terms of overall operating expenses, R&D and SG&A. Fourth quarter is probably pretty representative of what we expect the next few quarters.

[Unknown Analyst]

Okay. Okay. And then just a question on your overall enrollment dynamics in this disadvantage. I think in VISTAS, your interim is only on 45 patients. So can you comment about the overall enrollment target date? And do you see any differences between the studies in terms of the enrollment dynamics?

[Christopher Peetz, executive]

Thanks for the question. I mean the simple answer here is we have patients in for the interim, we're continuing to enroll for Part 2 now. And we'll be able to give better guidance for the full study enrollment at that interim when it comes up.

[Operator]

The next question comes from the line of David Labels with Citi.

[Unknown Analyst]

This is [indiscernible] on for David. We wanted to ask about the blinded PSC interim analysis. So besides the dose selection, would you share any other data points at a blended basis?

[Unknown Executive]

So we will be blinded in this interim and the design so that if it passes the threshold. It is designed to be predictive of a [indiscernible] and statistically significant positive pivotal analysis in the end. So that's all I can really share with you at this point. And it's really the special is developed based on what we know about the drug, this class of drugs, PSC and general and [ pruritus ]. So we feel pretty confident in terms of the design being robust and this being kind of well set up to a productive registration study for us.

[Christopher Peetz, executive]

In terms of communication at the point of interim for the VISTA study, assuming a positive interim, we would communicate that the interim has occurred and study continues as planned, which would be the extent of the information that we received here on the team as well.

[Unknown Analyst]

Okay. So as a follow-up to that, could you tell us a bit more about -- I mean, while unlikely, is there any other scenario like that would play out somehow like if you see that like the efficacy is not crossing the threshold on a blinded basis that was [indiscernible].

[Christopher Peetz, executive]

So the study design in the event where that threshold is not met, basically converts to an open interim analysis, we'd be able to look at the look at the data, share top line signings of it and what the next steps are for the study in that scenario.

[Operator]

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

[Brian Skorney, analyst]

On PBC, beyond pruritus, how are you thinking about the impact on outcomes and other markers in VANTAGE? And how can this help you make the case to physicians and patients?

[Christopher Peetz, executive]

Yes. Thanks for the question. I'll let Joanne speak a little bit to kind of the registrational plan for PBC.

[Unknown Executive]

Yes. So in terms of the interim, we're just going to be looking at top line. So pruritus [indiscernible] and [ safety ] as top line. And as you know, it is a big issue in this particular patient population for which there's really no particular therapies. So we're pretty confident that this study, the way it's designed, actually addresses an important unmet medical need at this point.

[Christopher Peetz, executive]

Yes. And just to recap, one of the points made earlier, the eligibility criteria for the VANTAGE study does not have an alkaline phosphatase criteria. So it includes patients that are traditionally thought of as first line that has really only UDCA as on label and still experience the same rates and severity of [indiscernible] as later lines of treatment. So a lot of unmet need across the PBC patient population.

[Operator]

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

[Antonio Arce, analyst]

Congrats on another strong quarter. I have 3, if I may. Firstly, on the VANTAGE study in PBC. As you said, it's a blinded data readout. Obviously, this is, as you mentioned, basically pruritus [indiscernible] versus placebo and whether it's [indiscernible]. But I'm also wondering if there are specific thresholds of activity on pruritus that you're looking at, not just so much to proceed, but also as you think about the competitive landscape and where you'd like to see that come in?

Secondly, if you could speak a bit about the PFIC opportunity in terms of sort of the sales, the speed of the sales ramp, kind of what you're expecting the trajectory to be through the remaining quarters of this year.

And then lastly, I'm sorry, I didn't hear when Peter was reviewing -- I think it was Peter, the numbers for LIVMARLI, both U.S. and international as well as the bile acid products for the fourth quarter. Thanks so much.

[Christopher Peetz, executive]

Maybe I'll just hit on recap one of the points on VANTAGE and then pass it over to Peter to talk about PFIC and recap some of those sales numbers. And the way we think about VANTAGE and volixibat in PBC, it really comes down to this -- the highlight here on lines of therapy and where the various agents are labeled and expected to be labeled. The majority of patients are first-line patients, and the majority of them have pruritus. So that's really the primary unmet need that we're going after. So showing a significant impact on that pruritus, we think, will be very clinically meaningful. And that's the lens that we're going to be taking towards that interim data when we look at it and make a decision on next steps. and continuing to roll into the pivotal portion of the study.

With that, I'll pass it over to Peter on the next 2 points.

[Peter Radovich, executive]

Sorry, I'll try to speak that a little bit here. So on the PFIC 2024 revenue expectation piece, I think we'll be upon a potential approval here in March we'll be spending a fair bit of time working closely with payers to get LIVMARLI positioned in their new-to-market policies. So I think you think about 2024, we'd expect a lot of free drug or path with PFIC really coming in more in 2025. Somewhat similar to what we saw in the [indiscernible] initial launch where the early quarters were high pop drug.

And then in terms of the numbers, from Q4, LIVMARLI totaled $41.4 million worldwide, $31.4 million, $10 million international. And then bile acid products were $28.1 million for Q4.

[Antonio Arce, analyst]

Great. And then maybe just a further clarification on PFIC. I appreciate the comments. Just wondering, given that you mentioned earlier, there's about 20-ish patents patients right now on expanded access. How soon would you expect those to be able to roll over on to commercial drug?

[Peter Radovich, executive]

Yes. What we saw in the [indiscernible] launches that occurred in a quarter or two, generally, is what we saw. And so I think that's probably consistent with our expectation for those '25 or so.

[Operator]

The next question comes from the line of Jonathan Wolleben with Citizens JMP.

[Unknown Analyst]

This is [ Catherine ] on for John. I had two kind of quick questions. [indiscernible] kind of follow-up on volixibat and just kind of how you guys see it fitting into the PBC paradigm in the setting of other proof agents that might potentially be effective? Also on pruritus, whether you see kind of the combo therapy sort of a monotherapy for earlier patients? And then also just as far as preparing for the PFIC launch, I know you said that there's not going to be too much of an investment as far as SG&A growth, like what else is kind of being done to sort of get the word out there about a [indiscernible] know that it's coming.

[Christopher Peetz, executive]

Thanks for the questions. And just to kind of recap the PBC positioning one more time here. The VANTAGE study includes first-line patients that really are -- when you look at the eligibility criteria for the PPAR programs, for example, and how [indiscernible] gets used outside the U.S., we are upstream of that setting where when a patient is biochemically controlled, they can still have substantial pruritus, right? So that's the area where [indiscernible] can play a very unique role in being in front of the other second-line therapies, PPARs and OCA, for example. So pretty unique positioning that we see for PBC. I'd add on in PSC, really exciting opportunity here where volixibat is positioned to be the first and only therapy for PSC by using pruritus as an endpoint, really gives an outcome to use for bringing a new therapy to these patients. So quite excited about the positioning there. I'm going to pass it over to Peter for part 2 here.

[Peter Radovich, executive]

Yes. In regards to PFIC, obviously, there's been -- there's no promotion until an approval. But I mean, I just kind of emphasize that it's a really small number of physicians that take care of these patients. in the U.S., I mean, you're probably talking about on the order of 100 positions in the entire country that take care of PFIC patients. There have been presentations at the major medical conferences on the data Phase III data as well as the Phase II data going back for years. So I think there's a kind of a relatively high awareness of the data and certainly, from [indiscernible] approval and commercialization, a lot of comfort with LIVMARLI's clinical profile, kind of familiarity with using it, prescribing it. working with our patient support hub, the [ Miramax ] Plus program, [indiscernible], et cetera. So I think that's probably a fair summary of where things stand today.

[Operator]

There are no additional questions at this time. I will pass it back to Chris Peetz for any closing remarks.

[Christopher Peetz, executive]

Great. Thanks again for everyone for joining us for today. [indiscernible] on the call, I do want to put a plug-in for Rare Disease Day tomorrow, today to build awareness for the more than 7,000 rare diseases impacting patients around the world. and celebrate some of the meaningful advances in research that we've seen in recent years.

Tomorrow, as part of Rare Disease Day, we're excited to be able to share a manuscript published in hepatology featuring long-term data in patients with Alagille syndrome treated with LIVMARLI. So please, [indiscernible], supporting the many advocacy groups that passionately lead efforts to support patients in research and rare disease tomorrow, and have a great evening. Goodbye.

[Operator]

That concludes today's conference call. Thank you. You may now disconnect your lines.