Q4 2021 Mirum Pharmaceuticals Inc Earnings Call

Thank you for your patience. Today's call will begin momentarily. Thank you for your patience. Today's call will begin momentarily.

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Good afternoon, and thank you for attending today's Mirum Pharmaceuticals fourth quarter business update call. My name is Amber and I will be your moderator for today's call. All lines will be muted during the presentation portion of the call with an opportunity.

for questions and answers at the end. If you would like to ask a question. Please press star one on your telephone keypad at any time. It is now my pleasure to hand, the conference over to our host Ian Clements, CFO of Mirum Pharmaceuticals. Ian, please proceed.

Thanks. Good afternoon, everyone.

Good afternoon, everyone.

I'd like to welcome you to Mirum Pharmaceuticals, fourth quarter and year-end 2021 conference call.

I'm joined today by our President and CEO, Chris Peetz.

Our chief operating officer, Peter Radovich.

And our Head of R&D, Pam Vig.

Earlier this afternoon, Mirum issued a news release announcing the company's results for fourth quarter and full year ended December 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 minimum and that programs based on management's current expectations.

Any statements regarding Mirum's business plans, development programs strategies prospects market opportunities and financial forecasts and guidance.

Mirum is under no duty to update these statements and they are subject to numerous risks and uncertainties and actual results could differ materially from the results anticipated by these statements.

Investors should read the risk factors set forth in Mirum's 10-K for the year ended December 31st 2021.

Subsequent reports filed with the SEC.

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

Thank you Ian and good afternoon to everyone joining us on the call today.

2021 was a banner year for Mirum and marked a significant turning point for both the company and the eligible syndrome community.

[inaudible] marley launches off to a very strong start as we deliver on our mission to provide life changing medicines for rare diseases.

Today, we will cover our accomplishments to date and our upcoming milestones with some brief prepared remarks, and then take questions.

Peter will share some launch details and then Pam will discuss our pipeline and finally, Ian will touch on a few financial highlights.

To start I would like to remind people of the devastating need for treatment of pruritus [Alagille] syndrome.

This is the most burdensome effective cholestasis in Alagille syndrome.

It impacts daily life and sleep for patients and families and is a leading indication for liver transplant.

The launch of LIVMARLI has introduced a crucial new treatment for colystatic pruritus in these patients.

Our label and our clinical data showed the tremendous potential of LIVMARLI.

And the impact of the long term dataset with six years of treatment experience was recognized last year in the lancet and with late breaking presentations at [inaudible] looking at event free survival and transplant free survival.

The strength of the LIVMARLI clinical data and the hard work of our dedicated team is leading to great launched success.

We are pleased to report fourth-quarter 2021, net product revenue of $3.1 million.

And we anticipate the strength to continue throughout the year.

Based on what we're seeing so far in 2022, we expect first quarter net product revenue to be at least $8 million and the full year to be at least $50 million.

As a reminder, we estimate the overall total opportunity in Alagille syndrome to be more than $500 million in the US alone.

And the early experience in the market confirms our view of this.

We have built Mirum to be a compelling business case, not only on the US opportunity, but with global reach.

As the US continues to grow, we expect to see approvals in Europe later this year and then partner markets into 2023.

Also protecting the value we've built in the business today, we announced the patent grants and US [Orange] book listing form of LIVMARLI with coverage into 2040.

Building further beyond LIVMARLI commercialization, we have an exciting calendar of clinical readouts with data expected across all five of our additional programs over the next two years.

And in short, we expect 2022 to be a truly remarkable year for Mirum and the patients we serve.

Now with that, I'll turn it over to Peter who will provide an update on our LIVMARLI launch progress. Peter.

Thanks, Chris.

We are proud of the initial results from our launch of LIVMARLI, the first and only FDA approved medication for this disease.

Today I'll share further color about around $3.1 million net LIVMARLI revenue.

$1 million net slip Marley revenue.

What we are seeing in the commercial business and why we're so excited about the growth trajectory.

First, it's important to note that there is no inventory in our reported product sales. Thus the revenue number is a true representation of demand revenue is recognized when the patient receives drop.

The revenue number is a true representation of demand revenue is recognized when the patient receives drop.

Also approximately 60% of dispensers in the fourth quarter were reimbursed, which exceeded our expectations and is indicative of strong performance by our market access team.

We believe the proportion of reimbursed expenses will increase throughout 2022.

And in fact, we're seeing this happen already in the first quarter and we expect to track towards approximately 90% of dispensers being reimbursed by year-end.

We are very pleased with this result, and attribute our success both to LIVMARLI's strong product profile as well as stellar execution by the entire Marin team, who brought a tremendous amount of passion ingenuity and experience to this launch.

Looking ahead, we believe that LIVMARLI has significant growth potential in the United States. We're encouraged by the high level of awareness of the treatment benefits of LIVMARLI as evidenced by the breadth of physicians, who have prescribed LIVMARLI to date.

Encouragingly, we're seeing very high refill rates and treatment compliance in the commercial setting.

We expected this based on the feedback we are hearing both from physicians and caregivers of LIVMARLI rapid and robust symptomatic improvement in this highly symptomatic disease.

This is further enhanced by our minimum access plus program, which is designed to maximize patient and family support to enable long term treatment adherence.

So putting all of the dynamics together, we are seeing in the business. We expect net revenue of LIVMARLI to be at least $8 million in the first quarter of this year.

And looking at the full year. This strong start gives us confidence we will achieve at least $50 million for 2022 LIVMARLI net product sales.

Turning to our plans for LIVMARLI's international launches, we remain on track for European approval in the fourth quarter of this year and we have begun initiating commercial early access programs in Europe and other international markets.

In markets outside of Western Europe our seven commercialization partners are making excellent progress towards bringing LIVMARLI to patients suffering from Alagille syndrome around the world.

In fact, we're excited to share that our partner GC pharma recently filed in South Korea.

And much like what we've seen in the United States. Prelaunch engagements with healthcare professionals, and Payors and international markets reveal both a high unmet medical need in Alagille syndrome, as well as receptivity to LIVMARLI's strong value proposition.

Plus, in addition to the 2022 revenue growth driven by launch success in the United States. We believe we are also well positioned for LIVMARLI revenue growth in 2023 and beyond as international markets come online.

And now I'll hand it over to Pam to provide an update on our pipeline.

Thanks, Peter.

It has been an outstanding finish to 2021, when we have presented groundbreaking data first of all the publication in the lancet from a pivotal study in Alagille syndrome, highlighting the rapid and sustained symptomatic relief provided by LIVMARLIf followed by two late-breaking presentations at AAFOB showing that LIVMARLI improved six year.

Event free and transplant free survival as well as identifying predictive markers of event free survival.

And these data have reinforced our belief that LIVMARLI is a huge step forward for patients with Alagille syndrome, and we continue our efforts on research and analysis and you remain committed to expanding our scientific knowledge and worldwide academic collaborations.

Now on the people front, we're excited to have recently announced that we have completed enrollment in our March phase III study.

And we expect topline data in the fourth quarter of this year.

As a reminder, the March PFIC study enrolled all PFIC subtypes and higher doses than previously tested and is the largest randomized phase III clinical trial ever conducted with more than 90 patients enrolled in the study.

This is a remarkable achievement given the size of the PFIC population.

Lastly, we're pleased to share that we have opened an international extended access program for eligible patients with PK.

Now in their pipeline, we have for other indications addressing cholestasis in both children and adults with several milestones.

In 2022, we're expecting two interim analyses for Maralixibat programme.

One from our phase <unk> study evaluating [Maralixibat] in patients with primary sclerosing cholangitis and a second from our phase [inaudible] study for patients with Interpublic cholestasis of pregnancy.

In 2023, we're targeting interim data from our phase two b vantage study evaluating [Maralixibat] in adults with primary biliary cholangitis as well as primary data from our phase two the embark study.

Evaluating LIVMARLI for children with biliary atresia.

Now all of these upcoming milestones as well as our robust R&D pipeline and research efforts puts us in a great position for continued growth.

And on that note, I will turn the call over to Ian.

Thanks, Pam.

[inaudible] the press release, and 10-K issued and filed today provide a full financial update.

I will call out a few of the highlights here.

In addition to the $3.1 million of net product revenues discussed we recorded $16 million in licensing revenue from our partners in South Korea, and China, bringing total revenue number to $19.1 million for the year.

Our total operating expenses for the year were $192.6 million, which includes research and development expenses of $131.4 million SG&A expenses of $59.2 million.

Cost of sales of $1.9 million.

Of note. These costs include $18.9 million of expense that option agreement that will not continue into 2022.

Also $23.2 million of expense from regulatory and clinical milestones related to the multiple progress points of course the programs last year.

Mainly remains well funded and at the close of the fourth quarter ended December 31, 2021, we had cash, cash equivalents and investments of $261.5 million.

Recall that in November of last year, we announced the sale of our priority review voucher granted by the US FDA in September of 2021, with the approval of live Molly $310 million.

This without growing topline contribution puts us in a strong position to achieve critical milestones and expand our commercial presence over the next few years.

So with that, I'll turn the call back over to Chris for any final comments. Chris.

Thanks, Ian.

And thank you to everyone for joining today.

To close, Mirum has made remarkable progress this past year as we continue to advance treatments for devastating rare diseases.

Our launch is off to a great start and the Mirum team is energized by the stories, we're hearing from physicians and caregivers on the impact that LIVMARLI is having on the Alagille syndrome patients.

Again based on what we're seeing in these early days of commercialization, we have confidence in achieving at least $50 million in net product revenue this year.

This will provide a tremendous base for our business as our pipeline of five late-stage clinical programs matures.

Thank you again for joining us. Operator, please open the line for questions.

Of course, thank you.

If you would like to ask a question. Please press star followed by one on your telephone keypad. If for any reason you would like to remove that question. Please press star followed by two. Again to ask a question, that's star one.

As a reminder, if you are using a speaker phone. Please remember to pick up your handset before asking your question. We'll pause here briefly ask questions are registered.

Our first question comes from Jessica Fye with JPMorgan. Jessica. Your line is now open.

Jessica Fye with J P Morgan Jessica.

Jessica Your line is now open.

Hey, guys. Good afternoon, thanks for taking my questions.

Just a couple.

First, can you walk through the patient and reimbursement dynamics that underpin your expectation for at least $8 million of sales in the first quarter of 2022?

Yeah.

Yes, thanks for the question.

Quite excited about what we're seeing in the early days and I'll let Peter speak to some of the dynamics underneath that number.

Thanks for the question.

So on the patient side, we're seeing really strong adoption as noted by our net revenue number I think it's important to note that the net revenue number we are providing here doesn't have inventory, it's really direct and transparent.

Into what's happening at the patient level.

To be counted as recognized as net revenue and our distribution model.

The family actually is signing for LIVMARLI at their house. So it's giving you a direct view of what's happening there.

On the reimbursement side.

We've been really pleased with where we've gotten to with payers. I think this is a direct reflection of us kind of getting out there early last year.

The burden of Alagille syndrome, having those clinical discussions and what we expect by the end of the year to have 90% of our dispensers reimbursed and as we've kind of come into the year. We've seen coverage policies come down from several COVID-19 plans that are really in line with the.

LIVMARLI labeling, I'm just kind of allowing physicians to use this wavelength, which is very broad.

Mike.

That wavelength, which is very broad.

Okay, a couple of thoughts. How many current LIVMARLI patients were previously on expanded access?

Can you talk a little bit more about the decision to launch the expanded access program for PFIC patients and how much do you expect to get there in the context of Bill day already approved in that setting?

Yes, Thanks, Jess for the question. I'll take the expanded access point briefly here and then pass it over to Peter and on the PFIC expanded access program.

Access point briefly here and then pass it over to Peter and on the beef expanded access.

Program.

It's important to keep in mind that PFIC is rarer than Alagille syndrome. So we don't expect to see as many patients coming into that in total.

But there are many countries where it's not available and also as we've seen responses are not universal to build it. So there is still unmet need.

We're seeing demand for.

Yeah. Thanks. On the Alagille side, we have ruled over all of the Alagille expanded access patients in the US to commercial drug.

Important to note, though that in Q4, the $3.1 million net revenue number, the majority of that was actually driven by de Novo prescriptions.

So the really the clinical and EAP rollovers really the minority of that and the majority of that is just driven by new prescriptions and really encouraged by the broad prescriber base, we're seeing we've seen broad participation prescribing from across our 120 or so.

Key accounts as we launch, which we think is a real indicator of health.

Great and just a last one. Can you elaborate on what the new 647 patent covers?

Yeah. So, briefly it covers the use of LIVMARLI and Alagille syndrome at the labeled dose.

It covers the use of live Marley analogy Olson drum at the labeled dose.

So quite excited to see that grants and it listed in the Orange book.

Thank you.

Thank you.

Thank you, Jessica. Our next question comes from Mani Foroohar with SVB Securities. Your line is now open.

Our next question comes from Mani for Ah Ha with S. The be secure. Securities. Bonnie Your line is now open.

Securities.

Bonnie Your line is now open.

Hey. Thanks, guys and congratulations on all the progress.

So if I'm hearing you correctly, the 8 million for this quarter and 50 for the year is that exclusively US sales or is there any probability adjusted European Japanese et cetera sales in there.

And then secondarily, how should we think about modeling licensing revenue coming in from your partners will be?

The right way to think about it as a proportional relative to say net product sales. Do you expect it will be lumpy or like how should we be modeling that?

I can start with the first the first question, Mani.

The first the first question Manny.

While we are launching some of these commercial early access programs in international markets as I mentioned, we expect that to be very small this year in really 2022, you should think about as substantially all of the revenue.

Will be coming from the US, but really excited about.

In 2023 and beyond what the international launches can be improved with revenue growth sustained over time.

Yeah, and just to round it out on the licensing front, there will be some contribution from them.

But relatively modest compared to the US business, so the 8 and 50 that we talk about that's from the US business exclusively.

Okay, and apropos of that.

So what I'm hearing suggests that number is kind of a florida for the quarter, which would suggest to me that it represents the revenue one would expect from the patients that you already have prescriptions for in hand.

Is there some proportion of some assumption around new patient adds between now and the end of the quarter also baked into that $8 million or is all of that incremental?

So the way we look at we're looking at those numbers as.

I'll borrow some of your language. It is in a sense a floor in what we're seeing in the business. That does assume some modest growth in prescriptions as well, but we're trying to give some of that transparency to what we're seeing in the business, which is really strong here in these early days, so kind of trying to communicate here from where we stand today really.

Early in the year.

Pretty comfortable on hitting those numbers.

Great. That's helpful. Thanks, guys. Congrats again.

Thanks.

Thank you, Mani. Our next question comes from Steven Seedhouse with  Raymond James. Steve, your line is now open.

Our next question comes from.

Steve <unk> with.

Raymond James Steve Your line is now open.

Hi, This is Ryan Deschner on for Steve.

I wanted to ask you guys should give us a little more detail on the prescription volume split between large and small accounts for Marley and if you're seeing any super prescriber accounts so far.

Thanks, Ryan. I'll pass it over to Peter to answer that one yes. Thanks, Ryan. We've actually seen prescriptions from across the board, it's been really encouraging youre seeing now.

Some of the biggest programs in the country being early adopters and we've also seen regional children's 

Community hospitals [inaudible] prescribers. So it's been a broad participation from across our prescriber base.

Thank you very much and then just real quick can you also comment on the duration of treatment that you are seeing so far.

Comment on the duration of treatment that you are seeing so far.

Yes, I think one of the I'm trying to get to in our remarks that one of the real strengths here, what we're seeing.

Real strength here, what we're seeing.

It is refills are really happening on a very kind of predictable cadence every month and the feedback we're hearing from physicians and caregivers is just really really positive.

They're able to see a rapid and robust symptomatic improvement with LIVMARLI very quickly after initiating which is we think one of the major drivers of the.

This improvement was look mildly very quickly after initiating which is we think one of the major drivers of the.

Retail's kind of occurring in a very predictable cadence.

Got it, thank you very much.

Thanks.

Thank you, Ryan. Our next question comes from Ed [inaudible] with [HC] Wainwright. Ed, your line is now open.

Our next question comes from Ed Arce with H C Wainwright.

Ed Your line is now open.

Thanks for taking my questions and congrats on the early progress here.

Thanks for taking my questions and congrats on the early progress here.

Three questions for me firstly on the reimbursement.

Noted 60%. of drug dispensed is already being reimbursed and you're targeting.

A drug dispensed is already being reimbursed then youre targeting.

90% reimbursement rate by the end of this year.

Firstly, so just kind of walkthrough the process as that improves throughout the year. What particular things need to happen and kind of walk us through that process.

The process as that.

Proves throughout the year.

Now what.

What particular things need to happen and kind of walk us through that process.

Secondly is just a commentary around the penetration across your targeted physicians. I think you said 120 key accounts.

Just a commentary around the penetration across your targeted physicians I think you said 120 <unk>.

Key accounts.

Just some more detail around where that stands now and how you see it expanding throughout this year and then lastly, if you have this metric.

More detail around where that stands now and how are you.

We see it.

Expanding throughout this year and then lastly, if you have this.

It would be helpful. Just thinking about the time it takes between.

First writing the script to drug in the hands of patients. Thanks so much.

Two drug in the hands of patients. Thanks, so much.

Great. Thanks. Those are all in Peter's shop. So I'll pass it over to Peter walked through. Sure . Thanks for the question. So yeah on the reimbursement and kind of what it's going to take to get.

Two to exiting the year with 90% of defense is being reimbursed. So the first step is to ensure we have reimbursable pathways in place kind of a way to get reimbursed.

Generally, the two categories of those either plan will write a policy or they'll handle a rare disease drug like this through medical exception. By the end of Q1, we expect 100% of [Alagille] utilized in the US to have a pathway to get reimbursed and then the next step after that is ensuring high quality of coverage and that's kind of.

That's a policy we've seen so far from the bigger plans I've just had been really very favorable and very much aligned with the evidence in the literature for LIVMARLI in the labeling by the FDA. So that's really those are really kind of ensuring the quality of access is there now that we have a pathway for all eligible patients in the US is really the next step.

Next step in.

Our hub working to pull those through to pay dispensers.

That's really what needs to happen on the reimbursement side. I think your second question was about kind of penetration. We have seen a significant number of the 120 accounts are prescribed kind of the pattern. You mean can I ask what is it going it hasn't wasn't hit to sort of expand that. The pattern we're observing in the early days is usually a new position that hasn't prescribed

often opt into maybe one or two of their most phenotypically severe patients gain comfort with the medicine based on that initial trial and then we see kind of expansion is that that experience kind of unfolds in those accounts.

That's kind of how we see those dynamics playing out and then finally, you asked about prescription to fill. In there we're seeing the average time from a prescription received a bill being a couple of weeks on average.

In some cases, it's as fast as days, we've seen some go out really fast in other cases, obviously longer than that.

That's something that we hope to continue to be able to work on as we go through the year, but really really happy with where we are out there as well.

Great. That's very helpful. Thanks so much, Peter.

Yep. Thanks, Ed.

Thank you, Ed.

Our next question comes from Josh Schimmer with Evercore. Josh, your line is now open.

Josh Your line is now open.

Thanks so much for taking my question just one on the Maralixibat for ICP, maybe you can give us a sense of what we should be looking for in the near term phase III analysis later this year, what do you think approvable endpoints for that indication are likely to be.

Thanks, Joshua. Pam walk through that. Thanks, Josh for the question so for ICP.

We know the FDA approvable endpoint is pruritus and so.

We are targeting that but it's a little bit of a different disease setting.

These women not only have pruritus and as well as elevated bile acids but theres also harm to the fetus with very elevated serum bile acids and so on that front, we know that every increase in serum bile acid.

Harm to the fetus with very elevated serum bile acids and so on that front, we know that every increase in serum bile acid.

Micro mill per leader is the increased risk to the fetus nacelle frankly any reduction in serum bile acids that we see in this setting is going to be meaningful.

For the fetus and obviously, those reductions in [serum bile] acids will be meaningful for <unk>. So we expect open-label interim data later this year and looking forward to reporting that out.

In serum bile acids will be meaningful for <unk>. So we expect a open label interim data later this year and looking forward to reporting that out.

Got it, thanks very much.

Thank you.

Thank you, Josh.

Our next question comes from Brian Skorney with Baird. Brian, your line is now open. Hi, this is Luke.

Brian Your line is now open hi, this is Luke.

Hi. This is Luke on for Brian. Thanks for taking the question.

So the March PFIC readout can you remind us of the powering assumptions.

On the observed [inaudible] primary endpoint.

And then is there a level of improvement on pruritus and serum bile acid reduction that you believe could provide a meaningful commercial advantage as [inaudible] in the space?

We are in the space.

Great. Thanks for the question.

Pam set some expectations on what we're thinking about for data empowering yeah. So with regard to the study.

March peak of Phase III study, really excited about that.

As mentioned, we've got 90 patients enrolled in this study across all PFIC subtypes, and I think a really important factor In this study is that we are going to much higher doses than what we've done previously in our phase III Indigo study. And in that study, if you recall when the dose was doubled.

We had an increase rate of response, and so what we're hoping for is that even with higher doses. We will see an increased response rate across PFIC subtypes and that'll be reported out top-line data later this year.

Rate of response, and so what we're hoping for is that even with higher doses. We will see an increased response rate across <unk> subtypes and that'll be.

Reported out top line data later this year and then.

So I don't know anything else to add there, but that's where I'm really excited about that data and looking forward to reading out.

Does that cover the question for you?

Yes. Thanks.

Great. Thanks.

Thank you, Brian. Our next question comes from Yasmeen Rahimi with Piper Sandler, your line is now open.

Our next question comes from yes.

Yes mean rahimi.

With Piper Sandler, Yes mean your line is now open.

Hi. Thanks for taking my question. I have a few for you. Maybe the first one to start off can you comment on if you're seeing any. That's being built off label for pizza or other called static diseases. Two can you actually quantify sort of the compliance rate that youre seeing in the real world.

That's being built off label for pizza or other called static diseases. Two can you actually quantify sort of the compliance rate that youre seeing in the real world.

Other than maybe describing it to the extent you can.

The third one is in regards to the boiler back to back readout, especially on desktop and all that are both two in the fourth quarter.

As well as I guess March PFIC in the fourth quarter.

How do we really get comfortable about the data is really coming in the fourth quarter and that we're not notified you know that it could push into first half of 2023.

Just kind of provide some color or some matrix that gives us really comfort that this is the timeline in which topline data is going to be expected and thank you again for taking my questions.

Yes, thanks for the questions.

I'll touch on the first couple and then ask Pam to speak to the Maralixibat and March timing. I think first on off label, it's really insignificant. So we're not not saying any notable off label prescriptions.

March timing I think first on off label, it's really insignificant. So we're not not saying any notable off label prescriptions.

And compliance rate and it's put an actual number on it. This early in the launch we think it's just it's premature but I'd say, it's strikingly supportive it's really highlight of the launch and what we're seeing so far as we get further in and you have more of that to longer-term treatment.

<unk>.

More of that to longer term treatment.

Similar to what we see in our six years of clinical follow up.

That's when I think it'll be more relevant to get a view of that.

Relevant.

<unk> of that so.

Throughout the year, we expect that compliance and persistence to be a real highlight and supporting the early success and the full year numbers.

And what Pam speak on pipeline timing. Yes, thanks for the question.

So really excited about our Maralixibat programs, I'll start there.

First data will be on the PFIC study in an interim analysis expected later this year and what we've seen so far is that patients are rolling into the long term extension portion of the study and they are staying on drug. So we view that as a really strong signal.

Of the study effect and excited to to share that data later this year.

For ICP, we continue to push forward in this setting there are some enrollment challenges on the backdrop as you can imagine with COVID-19 and pregnancy, but we're working to address those by making adjustments to the protocol is necessary and operational adjustments as needed. And for PBC interim data is expected in 2020.

And on the March PFIC phase III side, we have completed enrollment for.

This study so it's a six month primary endpoint enrolment is done so you can wind the clock forward and certainly top line data will be shared this year.

Okay. Thanks.

Thanks, Jeff.

Yeah.

Thank you, Yasmeen.

There are currently no additional questions waiting at this time, so I'll pass the conference back over to Chirs Peetz for any additional remarks.

Great. Thank you, operator.

And thanks, everyone for joining. Your support makes the advancement of our important medicines possible.

We'll talk with you next quarter. Goodbye.

Yeah.

That concludes the Mirum Pharmaceuticals, Q4 business update call. Thank you for your patience. You may now disconnect your lines.

Miriam Pharmaceuticals, Q4 business update call. Thank you for your patients you may now disconnect your lines.

Uh huh.