Mirum Pharmaceuticals Inc

NASDAQ:MIRM

Earnings Call Transcript 2021-Q1

[Operator]

Welcome to the Mirum Pharmaceuticals Q1 Earnings Call. My name is Rees, and I will be your operator for today's call. [Operator Instructions] I will now turn the call over to Ian Clements. Ian, you may begin.

Please standby as we are experiencing technical difficulties. Again, please stand by one moment, please.

[Ian Clements, executive]

Hello, Rees, can you hear us now?

[Operator]

Hello. Yes, I can. My apologies for the technical error.

[Ian Clements, executive]

Thanks, Rees. I'll take you from the top. Apologies, everyone, for the technical snap through there. So thanks for joining us. As I mentioned, we -- I'm joined today by our President and CEO, Chris Peetz, our Chief Operating Officer, Peter Radovich.

Earlier this afternoon, Mirum issued a news release announcing company's results for the quarter ended March 31, 2021. 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 conference call, we will be making certain forward-looking statements about Mirum and our programs based on management's current expectations, including statements regarding Mirum's business plans, development programs, strategies, prospects, market opportunities and financial forecasts and guidance. These statements are subject to numerous risks and uncertainties, and actual results could differ materially from the results anticipated by these statements.

Investors should read any of the risks -- should read risk factors set forth in Mirum's 10-Q for the quarter ended March 31, 2021, and any subsequent reports filed with the SEC.

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

[Christopher Peetz, executive]

Great. Thanks, Ian. Mirum is focused on advancing life-changing medicines for rare liver diseases. Today, we'll provide updates and context around some of the exciting recent steps we've taken in Building Mirum as the leading rare liver disease company. It's an important time in realizing our vision.

I'll cover our regulatory and pipeline updates, followed by Peter on global commercialization and then Ian with the financial update.

First, recapping recent announcements. Our first potential launch is around the corner for maralixibat for Alagille syndrome in the U.S. with the PDUFA date in the third quarter. We strengthened our IP position with the patent allowance in the U.S. that extends exclusivity for maralixibat to 2040.

We've broadened our pipeline to include late-stage programs in 6 indications in the gene therapy collaboration for PFIC. And entered into a partnership for maralixibat in Greater China to accelerate the global launch of maralixibat.

Today, we'll talk about these updates and the value we continue to build within Mirum's programs. With that in, maralixibat has great momentum and is on the cusp of launch, if approved by the FDA. In March, our NDA was accepted for priority review by the FDA with the PDUFA date of September 29. We are planning to be launch-ready by late summer. The FDA also indicated that at this time, there are no plans for an advisory committee.

From our standpoint, our regulatory review progress is on track, and we've been able to accommodate all inspections to date. If maralixibat is approved in the U.S., it would serve as the first treatment approved for patients with Alagille syndrome.

Peter will share more with you about our launch readiness and the unveiling of our patient services program, Mirum Access Plus. We are optimistic about the benefit maralixibat can have for patients with Alagille syndrome.

In the ICONIC study, which serves as our pivotal study, highly significant improvements in pruritus were observed, with more than 80% of patients achieving a clinically meaningful reduction in its score. We have 6 years of follow-up data and are seeing durable responses across all 3 randomized studies of maralixibat and Alagille syndrome, a program that included 86 patients in total.

Now we are analyzing the impact of maralixibat on event-free survival across these studies, looking at time to biliary diversion surgery, liver transplant, hepatic decompensation of [indiscernible]. These data and natural history comparisons are planned to be part of our European filing for Allagille syndrome, anticipated next year as an indication expansion to the PFIC2 application. Our PFIC2 application in Europe has been validated. We are working through the review process and remain on track with our plans to launch maralixibat for PFIC2 in Europe in early 2022, should it be approved.

As a reminder, this filing is based on key findings from the Indigo Phase II study. First, serum bile acid response leading to improvement in itch, growth and other liver parameters. That is followed by improvements in transplant-free survival in those serum bile acid responders. And third, our filing includes comparisons to a natural history cohort on event-free survival.

Turning to our Phase III March PFIC study. The study is advancing well with approximately 50 patients with PFIC subtypes 1, 2, 3 and 4 randomized across 2 cohorts. Some countries continue to be impacted by COVID-19 and competitive enrollment, so we now plan to extend enrollment in order to achieve the target number of patients per cohort. This study is projected to be the largest randomized study ever conducted in PFIC, and includes a broad range of genetic subtypes.

As a reminder, March PFIC evaluates a higher dose of maralixibat that has been shown to drive greater bile acid clearing activity. And importantly, we remain on track to report top line results in early 2022. And further strengthening our position in PFIC, we recently announced an exciting new gene therapy collaboration with the vet therapeutics, a leader in liver-directed gene therapy.

While achieving incredible clinical results with maralixibat, including improvements in 5-year transplant-free survival, we recognize that there will be PFIC patients with genetic subtypes who will not respond to ASBT inhibition. This was the impetus for entering into the optional license agreement with Vivet for their 2 PFIC gene therapy programs.

Our vision is that maralixibat will be first-line treatment in PFIC. And VTX-803 and VTX-802 will be second-line treatment options for PFIC3 and PFIC2 and those patients who do not respond to ASBT inhibition. These programs work by addressing the root cause of the disease through correction of the defective genes that cause each condition. This is cutting-edge technology. And while early, it's our hope that these gene therapies could someday offer a cure for patients living with PFIC3 and PFIC2.

In collaboration, Vivet's experience gene therapy team will lead work until IND submission and Mirum will lead the clinical development and commercialization globally. We look forward to sharing more with you as these studies progress.

Of note, Vivet has 2 abstracts at the upcoming American Society for gene and cell therapy on additional VTX-803 studies showing further preclinical proof of concept.

Now shifting back to our clinical development programs underway. We were pleased to expand the maralixibat program with the initiation of the Phase IIb EMBARK study in Biliary Atresia. And we also significantly expanded our clinical programs for volixibat. We believe this is another potentially transformative treatment for several cholestatic liver diseases. Earlier this year, we launched our Phase IIb VISTAS study for adults with primary sclerosing cholangitis. And more recently, we launched the Phase IIb OHANA study that will evaluate volixibat in intrahepatic cholestasis of pregnancy. And finally, a Phase IIb study in primary biliary cholangitis is targeted for later this year.

We've incorporated FDA feedback into the design of all of these studies, all of which we believe are potentially registrational. We expect interim analysis for the VISTAS and OHANA studies to occur in 2022.

So the growing body of evidence of maralixibat, volixibat strengthens the least and their potential transformative impact on the lives of patients with cholestatic liver diseases and their families.

On that note, I will now turn it over to Peter, who will provide an overview of our launch readiness activities and partnership update. Peter?

[Peter Radovich, executive]

Thanks, Chris. With our PDUFA date of September 29, just over a quarter away, we are working diligently to ensure we are ready to deliver maralixibat to the Allagille syndrome community, should it be approved by the FDA.

Our goal is to establish maralixibat as the standard of care in Allagille syndrome. We believe in its opportunity to transform the management of this disease, offering an early treatment option that could potentially delay or prevent the need for liver transplant, which, as Chris said, is based on more than 6 years of clinical data in this patient population.

If approved, maralixibat would serve as the first approval in Allagille syndrome and will represent the only pharmacologic therapy with clinical data to support its use. We have made great progress toward being launch-ready by late summer ahead of our PDUFA date.

I'd like to spend a few moments highlighting some of these key activities. An important component of our launch strategy is ensuring thorough disease awareness of Allagille syndrome by health care professionals. To that end, we launched our Allagille syndrome disease awareness campaign, a program developed for health care providers to increase awareness of the disease, educate on pathophysiology, including the link between bile asset and clinical manifestation seen in patients and to improve the time to diagnosis. Our goal is also to elevate awareness around the burden of pruritus, which is often the most troubling symptom for patients with Allagille syndrome and their families.

To further ensure launch-readiness, we have achieved several critical milestones that prime Mirum and maralixibat for success. For instance, hiring the right team is up utmost importance. Our colleagues and medical affairs were first to be fully on board and engaging in scientific discussions with our key pediatric liver centers.

On the commercial side, we recently completed hiring of our U.S.-based team, including our field sales representatives. I'm particularly pleased with the highly scaled group we were able to bring together, which consists of broadly experienced professionals who have a track record of success in rare and pediatric disease markets.

We also hired our payer account team, and we have filled other crucial commercial roles in market access, marketing, trade distribution and patient services. Together, this team of high-caliber, experienced professionals have worked to build the capabilities to launch maralixibat in Allagille syndrome and beyond.

One hallmark of our launch readiness is the development of our patient support program, which we are calling Mirum Access Plus. The goal of this program is to provide a seamless and caring experience for patients and families, addressing key educational gaps for patients who are prescribed maralixibat through the development -- through the deployment of a fully dedicated staff.

We work closely with patients, families, advocates and health care professionals to understand the need and concerns that are of utmost importance as they think about starting a trial on a prescription medication for Allagille syndrome. This input has been invaluable to us. And help to establish what we hope will be a program that provides exceptional customer service and one that addresses and provide support around the issues that are really critical to families that they navigate a new diagnosis and treatments.

In Allagille syndrome, many patients exhibit a range of symptoms and are no strangers to prescription medications and the associated issues that often follow. Through our payer team, our goal is to ensure broad quality payer coverage. Our bill payer team has already begun discussions with payers as a top prelaunch priority and to date, have engaged with payers covering approximately 95% of United States lives.

Through these conversations, we will introduce [indiscernible] and our focus on cholestatic liver disease, helping to underscore the disease burden as well as to provide key clinical detail around maralixibat to accelerate formulary reviews.

Post launch, the team's goal is to gain formulary acceptance and align any payer of clinical management criteria to evidence-based medicine. Turning to our plans for commercializing maralixibat in Europe. Earlier this year, the EMA validated our marketing authorization application for maralixibat for patients with PFIC2, which puts us on track for a launch in early 2022, if approved.

To support the launch in Europe and globally, we are building out our team in Europe and have begun to fill key roles. We are also actively preparing for payer dossier submissions in key markets. Our strategy is to have a targeted approach, focused on larger European markets first, while leveraging early access programs.

More broadly, our goal is to extend maralixibat's availability worldwide through partnerships with distributors or out-licensing opportunities. In the regions outside of Mirum's poor operations in the United States and Europe. We've engaged in conversations with top rare disease companies with local expertise who have a proven track record of success in commercializing treatments and high unmet need rare disease settings.

With this strategy in mind, we announced last week our first out-licensing agreement with CANbridge Pharmaceuticals, a leading rare disease company based in China. We believe Cambridge should be a strong strategic partner who is poised to accelerate the global launch of maralixibat in China, Taiwan, Hong Kong and Macau based on their experience developing and commercializing medications for patients in these highly populous regions. They will also support the development of maralixibat ability of attrition.

We look forward to sharing more about additional geographic expansion efforts in the coming months. I came to Mirum last year because of my enthusiasm and confidence in what maralixibat has proven in the clinic and I'm thrilled to be at the forefront of delivering the first medication for the Allagille syndrome community. I also look forward to keeping you appraised of our great progress as we near launch.

I will now turn the call over to Ian to share an update on our financial health. Ian?

[Ian Clements, executive]

Thanks, Peter. I'm not going to do a deep dive into our quarterly finance source. As those are available in the press release, which we distributed earlier this afternoon, and in that Form 10-Q, both of which you can view in the Investors & Media section of our corporate website at mirumpharma.com. I will, however, provide an overview of what we believe to be the highlights that demonstrate our financial strength and position us well to support the launch of maralixibat in 2 geographies and approved, as well as advance our development pipeline for both maralixibat and volixibat.

From a balance sheet perspective, at the close of the first quarter ended March 31, 2021, we had cash, cash equivalents and investments of $213.1 million. Subsequent to the quarter end in April, we received $65 million from Oberland capital Related to the acceptance of our NDA for maralixibat in Allagille Syndrome.

As a reminder, we also have access to up to a further $85 million from Oberland Capital. This figure comprises of: one, $35 million upon approval of our NDA of maralixibat and Allagille syndrome; and two, a further $50 million from mutually agreed upon in-licensing or acquisition opportunities. Additionally, we anticipate receiving the rare disease priority review voucher in connection with our expected FDA approval of maralixibat later this year that may provide a source of funds.

With respect to operating expenses, first, G&A expense. For the quarter ended March 31, 2021, our G&A expenses were $9.5 million, and this reflects increased activity around launch readiness, including market research, disease state awareness activities and increased personnel costs to support our planned launches, if approved.

Second, R&D expenses were $38.1 million, which included a $15 million milestone to Takeda, consisting of $5 million, which would have been due, had we initiated a Phase III clinical trial in Allagille syndrome, and $10 million associated with the acceptance of the company's NDA filing for maralixibat for the treatment of cholestatic pruritus in patients with Allagille syndrome.

Now turning to our recent business development activities. First, regarding our option in licensing agreement with Vivet Therapeutics, we paid an upfront fee of EUR 3.5 million and additional cost-sharing expense for initial development work of EUR 5.3 million.

We'll also contribute on an ongoing cost-sharing payments on development work with potential progress payments and opt-in payments at option exercise. We'll also grow mid- to high single-digit royalties on sales of VTX-803 and VTX-802, if approved.

Finally, turning to our recent announced licensing agreement with Cambridge Pharmaceuticals. As Peter outlined, last week, we entered into an agreement with Cambridge. And under the terms of this licensing agreement, Cambridge has obtained the exclusive rights to develop and commercialize maralixibat within the Greater China regions, for Allagille syndrome, PFIC and Biliary Atresia.

In exchange, we're entitled to receive an $11 million upfront payment, R&D funding and up to $109 million for the achievement of future regulatory and commercial maralixibat milestones. With significant double-digit tiered royalties based on product net sales. Our strong and healthy balance sheet is a testament to strategic investments, judicial spend and planning surrounding our upcoming anticipated launches and continued pipeline development.

With access to approximately $400 million in capital, we expect our cash position to sustain our operations for the next 3 years.

With that, I'll turn the call over to Chris for final comments. Chris?

[Christopher Peetz, executive]

Thanks, Ian, and thanks, again, to everyone who joined the call today. Before we go to questions, I wanted to share a few final thoughts. We are paving the way to being the leader in the treatment of rare liver diseases with clinical programs across a wide range of indications. We take pride in the strong value of scientific evidence that we have built, including more than 6 years of outcomes in safety data in both Allagille syndrome and PFIC.

We had numerous clinical and commercial milestones ahead in both the U.S. and Europe. We are confident in our ability to achieve these milestones and create a remarkable impact from the lives of patients and their families.

A quick recap of these milestones. First, we plan to launch maralixibat for the treatment of pruritus in patients with Allagille syndrome in the U.S. in Q3. Our European application primarily with PFIC2 was validated, and we are planning to launch in Europe in early 2022, if approved. We expect top line data from our Phase III PFIC study in early 2022. And we expect interim analyses from our 2 volixibat Phase IIb programs for ICP and PSC next year as well.

And finally, in the second half of this year, we will be initiating a Phase IIb study evaluating volixibat in patients with primary biliary cholangitis. We have built a strong team and a pipeline with tremendous potential. We are positioned for success and meaningful outcomes for our patients and families and also in creating significant value for our shareholders.

Thanks again for joining us. Ian, Peter and I are happy to take your questions. Operator, would you please remind everyone of the prompt.

[Operator]

[Operator Instructions] We have our first question from Mani Foroohar from SVB Leerink.

[Mani Foroohar, analyst]

So really obvious and boring questions because someone has to. In anticipation of the launch, obviously, there's an element of warehouses of patient volume in the EAP, clinically beneficial for the patients, obviously, got a little bit of uncertainty around timing of those patients transitioning to commercial pay and flowing through the income statement. How should we expect you to disclose metrics around EAP, patient numbers, new start, start forms, give a sense of what the -- what metrics we should expect? And on what time horizon would you expect to transition to just giving us a revenue number?

[Christopher Peetz, executive]

Great. Thanks, Mani, for the question. Just from a high level standpoint, at this point, we don't have kind of clear metrics that we're going to be communicating. But what I can tell you is that there is really great interest across the U.S. and ex-U.S. countries and Mirum Access Program. So we have multiple sites open, multiple patients at a number of those sites.

So that effect is happening out there. That's not something that we'll give updates on ahead of the approval. Keep in mind that we also have -- some of the patients from the original maralixibat studies in Alagille syndrome, those patients all still kind of -- many of them are still on therapy long term.

And kind of with that as kind of setting expectations a bit, I'll turn to Peter to comment a little bit on how we think about managing that transition to really convert some of these patients to commercial supply upon approval.

[Peter Radovich, executive]

Thanks, Chris. And thanks for the question, Mani. Yes, our... the Mirum Access Plus program will be in touch with those patients. I think the first and most important point is we'll ensure uninterrupted supply through the approval process. And they'll be in touch with them around the time of the approval.

I think in terms of being able to comment on kind of precise time lines, there's a heterogeneity of payers in the U.S. and the what -- I think what we'd expect to see is variability on formulary coverage decisions and access decisions on the commercial side as well as variability on the state Medicaid side.

So I think as we get into the launch, we'll be able to give you more details on how that's going, which I think will ultimately be what drives the timing for those patients coming over to reimbursement of the product.

[Mani Foroohar, analyst]

Great. That's really helpful. And I guess the other question. Now that we're entering into a world where we've got very uneven sort of pandemic status, hospital volume status, et cetera. Just looking at how do you -- as you continue to have multiple phases, Phase IIb studies, et cetera? How should we think about the geographic split of enrollment in your ongoing late stage studies, especially ITP, et cetera?

[Christopher Peetz, executive]

A couple of comments on what we're seeing out there. As you know, there is some clearly a different impact by country. As we look at the volixibat program and the Biliary Atresia study where we've opened those up first in the U.S. We're not really seeing any COVID impact on sites in the U.S. in a meaningful way at this point.

On some of the upcoming country openings that we have in those programs, a lot of those are in countries that the early starts, frankly, aren't as impacted. So we're not projecting any headwinds from a pandemic standpoint as we look at volixibat and Biliary Atresia rollout. U.K., for example, being one of the key countries ahead for us, where there's just not as much in terms of restrictions currently.

[Operator]

Our next question is from Yasmeen Rahimi from Piper Sandler.

[Unknown Analyst]

This is Jesse on for Yas. Can you guys hear me okay?

[Christopher Peetz, executive]

Yes. We can hear you.

[Unknown Analyst]

Cool. First, congrats on the progress this quarter. I had three. Did you want to go one at a time or me roll them off at once.

[Christopher Peetz, executive]

I think one at a time sounds, high-risk to try to remember all 3.

[Unknown Analyst]

Okay. So our first -- my first question is for the MARCH-PFIC study, you guided that there's expanded enrollment due to COVID. Can you provide us some color on the enrollment updates, how many sites are going -- or with the progress of that one is expected to be fully enrolled?

[Christopher Peetz, executive]

Yes. Thanks for the question, and I think helpful and important to get into some of the color here. From a starting point, we have approximately 50 patients randomized currently. So we've made tremendous progress in terms of tracking towards the largest randomized study ever conducted in this setting.

We're also seeing a very broad mix of genetic subtypes. So in that enrollment to date, that includes PFIC types 1, 2, 3 and 4. And in terms of impact by site over time, it was kind of evolved over the course of the pandemic so far. And continues to be that way depending on how each country has been managing factors locally.

At this point, we have a broad spread in terms of geography, for site and we're getting close to where we want in terms of a mix of patient subtypes as well as the number of PFIC2 patients in the study.

[Unknown Analyst]

Cool. And then my next question is what information is needed to kick off the Phase IIb and PBC in the second half of the year?

[Christopher Peetz, executive]

Yes. So for the volixibat PBC study, we recently went through kind of follow-up steps with FDA to finalize our protocol in IND. So at this point, we are just completing some of the operational steps for start ups. So that's just around the corner, frankly.

And for all of the volixibat programs, we felt important to go meet with regulators, discuss the endpoints so that we could incorporate everything they wanted to see for an NDA. So that we took a full shot on the goal of these studies as potentially registrational.

[Unknown Analyst]

Cool. And then my last question is referring to EASL. What data should we expect to see for maralixibat in the next -- in, I guess, 2 months from now?

[Christopher Peetz, executive]

Thanks for the question there. The -- for conference abstracts, we'll have an announcement just ahead of the conference in terms of what we're presenting. We do have abstract in covering the maralixibat program in a number of analysis. I think probably what the real exciting analysis that I think everybody is interested in to some of the long-term event-free survival analysis that we've been conducting for Alagille syndrome. We're in the midst of finalizing some of that work now. So it is not submitted for EASL, and you can look for that later this year.

[Operator]

[Operator Instructions] Our next question comes from Etzer Darout from Guggenheim Partners.

[Paul Jeng, analyst]

This is Paul on for Etzer. I have, I guess, a follow-up on the EAP comments earlier and sort of interest there for maralixibat in a number of countries across the globe. Can you speak on any feedback you've received on the drug from physicians and patients from that program? And then maybe how that could influence your focus for efforts beyond U.S. and major EU countries?

[Christopher Peetz, executive]

Thanks for the question. A couple of comments to make there. First, the feedback has been positive. So we have heard back from -- from different channels that's overall good experience. More from a kind of a concrete next step standpoint, we are incorporating the first cohort of these patients in the update as part of our NDA process, so safety data from the first set of these patients will also be submitted to FDA. So further expanding the amount of maralixibat data that gets worked in -- ultimately worked into the filing.

[Paul Jeng, analyst]

Great. And just a quick follow-up then, so -- on the gene therapy programs from your partnership. So kind of where are these assets currently in preclinical development? And sort of how far away might they be from the clinic?

[Christopher Peetz, executive]

Great. And so just to clarify, I heard the question on time line for the Vivet collaboration to get into clinic. And I'll let Peter comment a little bit on some of the work that's going on there and what you can expect ahead.

[Peter Radovich, executive]

Thanks, Chris. Thanks for the question. You Vivet, we have a platform, AV platform, and they're working on optimizing both of these vectors right now. The 803 program in PFIC3 is further along. And we think, while it's important to note that the research is difficult to put time lines around where we think that there's an IND in a 1 to 2-year window for the 803 program with 802 program falling behind that.

[Operator]

Our next question comes from Brian Skorney from Baird.

[Jack Allen, analyst]

This is Jack Allen on for Brian. You mentioned your disease awareness campaign. I was wondering if you could elaborate a little bit on the color -- or provide a little bit more color with regard to that program? And are you contributing to any testing programs to help identify patients and develop a kind of patient database?

[Christopher Peetz, executive]

Yes. Sure. No, thanks for the question. So with regards to the disease awareness campaign, I mean just -- I can provide some color on the strategy. And I think one of the things we see in rare diseases almost universally is one of the most important things is to promote the disease to the medical community itself, not even a product because awareness outside of -- we talked about the 125 key accounts and really the top half or 1/4 of those are the kind of experts you see on the stage of EASL and Nasdaq, et cetera.

But outside of that, there's not a lot of awareness of the symptoms, how to make the diagnosis, the journey that these patients go through with Allagille, not on like other rare diseases. So we really focused with our disease awareness campaign on trying to reach the broader community of health care professionals beyond just the top centers, community-based pediatric GI docs, neonatologists and even some region to primary care. Of course, this is nonpersonal and so this is -- the idea here is really to increase awareness of the disease, shorten times diagnosis and educate on a link between the bile acids themselves and the adverse clinical outcomes. So this is really kind of an opportunity for market-shaping that we took ahead of the anticipated approval.

And now in the second half of the question, I think there was...

[Jack Allen, analyst]

Testing.

[Christopher Peetz, executive]

Testing. Yes, I knew I was missing something. Yes, we are -- we have a collaboration that's really led by trever. And that we do provide support to them as industry consortium that provides support to allow for a testing program that can run, I think now they're almost up to 70 genes in putting the gene because Allagille for children with cholestasis. So we do contribute to that and have a data exchange agreement with [indiscernible] around that. So -- and that we have seen that help improve diagnosis over the years, we've been involved and very much lines up with the epidemiology. We talked about with Allagille syndrome being about 2,000 to 2,500 patients in the USA.

[Operator]

Our next question comes from Steve Seedhouse from Raymond James.

[Ryan Deschner, analyst]

This is Ryan Deschner, on for Steve Seedhouse. I wanted to ask a little bit more about the Vivet potential assets. How different would primary and secondary endpoints and serum biomarkers look for a clinical VTX-803 program in PFIC3 versus maralixibat in PFIC2 and is expansion into other PFIC subtype of possibility?

[Christopher Peetz, executive]

Thanks, Ryan. Appreciate the question. So just kind of as a taking a step back, as we think about gene therapy in PFIC3 and PFIC2, the approach here is to completely restore functionality for the transporter in these patients that have mutations that lead to a complete or almost complete lack of function in that transporter. So in effect, what we're trying to do is similar to the maralixibat treatment except for in the settings where maralixibat can be effective, there's some working transporters.

So what that means is for the patients that are the most severe, that's where these gene therapies will be most impactful because ASBT inhibition is just not going to feel to be effective if you have no transborder function. So that said, kind of back to your question on what do you measure. We're trying to do the same thing here. So when you look at the levels of serum bile acids, other liver parameters, this thing that we saw improve in Indigo in the PFIC2 patients and that we expect to see in the March study across all PFIC sub types. You'd be looking at some of those same measures.

So serum bile acid as kind of a primary marker of cholestasis and bile transport function and then the other liver parameters that show -- if they improve, then your impacting the overall liver health.

[Ryan Deschner, analyst]

Okay. And did I hear it correctly that the target time line or the targeted regulatory, I guess, positioning for exercising the licensing option would theoretically be around the time of IND for these assets?

[Christopher Peetz, executive]

That's correct. Yes. And the it's addressed on a product-by-product basis. So it is specific to VTX-803 for PFIC3, VTX-802 for PFIC2. And we can make a decision all the way up through IND acceptance.

[Operator]

There are currently no further questions in the queue. [Operator Instructions]

[Christopher Peetz, executive]

Thanks, operator. We can go ahead and wrap and just make a couple of closing comments. Just first of all, thank you to all those who joined us today. As we share, this is a big year for Mirum, we're advancing potentially life-changing therapies for rare liver disease. Our approval and launch of maralixibat for Allagille syndrome is expected next quarter.

Our pipeline has expanded its 6 late-stage indications and a gene therapy collaboration for PFIC and we project our balance sheet and future access to non-dilutive capital provides 3-plus years of runway. We look forward to sharing updates in the coming months. Thanks so much.

[Operator]

Thank you, ladies and gentlemen. This concludes today's conference. Thank you for participating. You may now disconnect.