Welcome to the Mirum Pharmaceuticals Q1 Earnings Call. My name is Rees, and I will be your operator for today's call. At this time all participants are in a listen-only mode. Later we will conduct a question-and-answer session. [Operator Instructions] I will now turn the call over to Ian Clements. Ian, you may begin.

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 the 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 would like to turn the call over to Chris Peetz. Chris?

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 six 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…

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 six 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…

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 two 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 equivalence and investments of $230.1 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 $15 million milestones [indiscernible] (0:17:26), consisting $5 million which would have been due, had we initiated a Phase…

Thanks, Ian. And thanks again to everyone joined the call today. Before go to questions, I wanted to share a few final taught. We are paving the way 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 six 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 on 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 Alagille syndrome in the U.S. in Q3. Our European application for maralixibat and 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 MARCH-PFIC study in early 2022. And we expect interim analyses from our two 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, will you please remind everyone of the prompt?

Absolutely. Thank you. We will now begin the question-and-answer session. [Operator Instructions] And we have our first question from Mani Foroohar from SVB Leerink. Please go ahead with your question.

Thanks for taking my question. It’s Mani Foroohar. So some 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 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 us 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?

Great. Thanks, Mani, for the question. 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 U.S. and ex-U.S. countries and the expanded access program. So we have multiple sites open, multiple patients at a number of those sites. So that effect is happening out there, but not something that we'll give updates on head of the approval. Keep in mind that we also have some of the patients from the original maralixibat studies in Alagille syndrome, those patients also kind of – many of them still on therapy long-term. And kind of with that as a kind of setting expectations a bit, maybe 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.

Thanks, Chris, and thanks for the question, Mani. Yes, 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 timelines, there is 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.

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.

Thanks for the follow-up. And a couple of comments on what we're seeing out there. As you 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. UK, for example, being one of the key countries ahead for us, where there's just not as much in terms of restrictions currently.

Great. That's really helpful. I'll hop back in the queue.

Great, thanks, Mani.

Our next question is from Yasmeen Rahimi from Piper Sandler.

Hi, team. This is Steffi on for Yas. Can you guys hear me okay?

Yes, we hear you.

Cool. First, thank you for taking our questions and congrats on the progress this quarter. I had three. Did you want to go one at a time or me roll them off at one?

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

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

Yes, thanks for the question. And I think helpful 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 today 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.

Cool. Thank you. 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?

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 startups. 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.

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

Thanks for the questionnaire. 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 analyses. 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.

Great. Thank you so much.

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

Hey, this is Paul on for Etzer. Thanks for taking our question. 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?

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 will also be submitted to FDA. So further expanding the amount of maralixibat data that gets worked in – ultimately worked into the filing.

Great. 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?

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.

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 one to two-year window for the 803 program with 802 program falling behind that

Thank you.

Our next question comes from Brian Skorney from Baird.

Hi. Thank you, guys for taking the question. 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? Thank you so much.

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 non-personal 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…

Testing.

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.

Great. Thank you.

Thanks for the question.

Our next question comes from Steve Seedhouse from Raymond James.

Good afternoon. 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?

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 are 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.

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?

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.

Okay. Thank you very much.

Thanks for the question.

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

Thanks operator, we can go ahead and wrap and 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 six late-stage indications and a gene therapy collaboration for PFIC and we project our balance sheet and future access to non-dilutive capital provides three-plus years of runway. We look forward to sharing updates in the coming months. Thanks so much.

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