Q3 2023 Mirum Pharmaceuticals Inc Earnings Call

November 2, 2023

Good afternoon, I would like to welcome you to the myriad pharmaceutical conference call.

My name is brita and I will be the event specialist run them today's call with you.

All lines are on mute for the presentation portion of the call today with an opportunity for questions and answers yet.

If you would like to ask a question. Please press Star then one on your telephone keypad.

I'd now like to pass the conference over to your host Angie, Kevin Vice President of Investor Relations to begin so Andrew you may begin.

Thanks, Brad and good afternoon, everyone I'd like to welcome you to hear Pharmaceuticals third quarter 2023 conference call I'm joined today by our President and CEO, Chris <unk>.

Our Chief operating officer, Peter outage, our head of research and development and our newest team member Eric <unk>, Our Chief Financial Officer.

Earlier today, we issued a news release announcing the Companys results for the third quarter 2023 copies of this news release and SEC filings can be found in the investors section of our website for details and updates from the quarter can be found in our news release and 10-Q issued today.

Before we begin I would like to remind you that during the course of this conference call, we will be making certain forward looking statements about our programs based on management's current expectations, including statements regarding <unk> current and future business plans development programs and regulatory expectations strategies prospects market opportunities and financial expectations Americans under.

No duty to update these statements and 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 <unk> 10-K for the year ended December 31, 2022, and the subsequent reports filed with the SEC with that said I'd like to turn the call over to Chris.

Chris.

Thanks, Andrew and good afternoon to everyone.

This was another strong quarter of execution at Maryland.

The team has continued to grow our business by focusing on delivering life changing medicines for patients and their families living with rare diseases.

We've achieved multiple goals this quarter, including realizing total revenue of $47 $7 million for the quarter.

We saw continued strong global adoption uplift Marley.

We also completed the acquisition of two high impact and synergistic commercial medicines keen at all and called on them.

We continued our commitment to delivering on our pipeline with the recent positive restore phase III study readout and upcoming clinical and regulatory milestones and.

And all of this has been accomplished while improving our financial position in terms of performance and balance sheet.

Yeah.

These achievements come alongside another special milestone from Europe coming up this weekend, our five year anniversary of launching the company.

I'd like to thank the <unk> teams dedication perseverance and unmatched spirit.

And all of our success possible in a short time with much more to come.

Now moving towards Marley, we see steady demand growth in the U S and internationally. This reflects the positive impact of lift Marley for patients and families and the hours Youll Center community and also the focused execution of the <unk> team to make this important medicines accessible to patients around the world.

I am proud of what we've been able to accomplish with Marley Annapolis Youll syndrome, and what that means for our execution on our expanded portfolio.

This quarter, we grew our business with the acquisition of two commercial medicines keen at all in COBOL and assume sponsorship of a broadly used close basis genetic testing panel vital for diagnosing several rare genetic disorders that is free to patients and providers.

This has created a leading pediatric herpetology franchise and also bring significant and growing revenue contribution was approximately $100 million in annual net product sales from the bile acid portfolio.

Regarding our pipeline we have made good progress here as well in October we announced positive data from the phase III restore study evaluating <unk> in patients with cerebro tenderness xanthomatosis or CTX.

The data showed keen it all has a significant impact across key measures of the disease in this phase III dataset and the potential approval of keynote offer CTX is a critical step forward for CTX patients.

We plan to submit a new drug application for CTX to the FDA in the first half of 2024.

We also look forward to significant upcoming milestones and opportunities for Atlas smartly with the March 13th <unk> date for <unk> and our upcoming phase II data in biliary atresia and for <unk>, we expect to conduct our interim analysis in PSC and PVC in the first half of next year.

Now before I turn the call over to Peter to discuss our commercial business I want to welcome our new CFO, Erik Buerkle, who joined <unk> in September we.

We are thrilled to have Eric and his extensive leadership experience, helping to drive the growth and value creation. We see ahead premiere them, we will hear from him later in the call.

And with that I'll turn the call over to Peter here.

Thanks, Chris.

Total revenue, reaching $47 7 million in Q3 were decided by the tremendous progress that we've made across our U S and international businesses.

Our team continues to bring live mildly to more patients worldwide and we have taken significant steps in establishing a leading pediatric herpetology franchise through the ongoing execution of our commercial strategy with the Marley and the addition of keynote on cobalt.

Starting with <unk> in the third quarter, our revenues grew to $39 million in total net product sales, which represents over 100% growth before the addition of the bile acid portfolio.

This reflects the life changing impact of them early as having an allergy or patients that pruritus and the excellent execution of our commercial team.

The us business, which saw $28 6 million. This quarter continues to grow internationally, we see similar strength in patient uptake in Germany, and France, as well as distributor markets.

Going forward, we continue to anticipate quarter to quarter variability in international revenue.

Overall, we are looking forward to our continued expansion in both the U S and globally and are excited for the opportunity to broaden access to live Marley to patients with <unk> syndrome and future indications.

Moving to <unk>. The addition of these medicines presents a significant opportunity to both augment lydmar lease growth and significantly enhance our relationships with the patient communities and prescribers and pediatric hepatology.

There are striking similarities between the commercial models for these medicines. So at the hands of one team, we expect top and bottom line synergies.

In terms of our commercial strategy, our pediatric herpetology team has been extremely effective. So we don't plan to change a model that is working well, but we do see some opportunity to augment our efforts, but the expertise that we have brought in contributor.

Pediatric Herpetologist are the main call point privilege Marley and COBOL and we will continue to deepen our relationships with these prescribers through our expanded offering of high impact medicines, and our branded cholestasis genetic testing panels.

Which is an important diagnostic tool that positions <unk> as a partner from the start of the patient journey.

While <unk> seen it all have grown steadily over the last several years, we see opportunity to improve diagnosis and increased treatment across these indications.

And CTX for example, the consequences of the disease Furbished manifests in Oregon other than the liver such as juvenile bilateral cataracts and may not be linked to Cts until much later in adulthood, usually after a irreversible neurological damage has occurred.

To address this gap in diagnosis and treatment, we've increased investment in disease awareness and diagnosis amongst specialties, where this disease, often first presents such as ophthalmology neurology and medical Ninja medical genetics.

This expanded team will allow us to reach more patients earlier in their journey and is a key step as we prepare for a potential approval and launch in CTX.

In summary, we are thrilled by the addition of the new products and team members and look to build on the tremendous momentum we've achieved delivering important medicines to even more patients with rare liver diseases.

On that note I will turn the call over to Pam Okay. Thanks.

Thanks Peter.

Last month, our team announced positive data from the phase III restore study evaluating <unk> in CTX.

The study objective was to evaluate the safety and efficacy of 10 adult by measurement of yearend bile alcohols, and other biomarker metrics, including cholesterol.

The study showed a market and statistically significant improvements across the primary and all key secondary endpoints, demonstrating a broad and consistent effects of <unk> in patients with Etfs.

As a reminder, disease progression is Cts is due to the accumulation of cholesterol in the kitchen and thin and we were thrilled to see the depth of improvement across all measures.

In particular, the cholesterol results are a critical finding prior to this it was not certain that that will begin at all could impact cholesterol levels quickly.

Also there was start trials highlight the importance to diagnose and treat CTX patients as early as possible.

Additionally, a greater proportion of patients receiving placebo required rescue therapy.

Demonstrating the immediate impact of keen adult and Cts.

And while Canada is currently the standard of care in CTX and recognized as a medical necessity by the FDA. We are very pleased that the results confirm the life changing impact in adult can have for these patients.

We're grateful to the work that you are team has put into the development and execution of the study.

Including selection of the endpoint, which were developed in close collaboration with the FDA and.

And with the strong results. We believe we are well positioned for our NDA filing, which we expect in the first half of next year.

Moving onto clinical milestones, we're looking forward to reporting top line data later this year from our phase two be embark study at Lyft Marley for pediatric patients with biliary atresia, which will be the first placebo controlled data with an <unk> inhibitor in this setting.

Some of it thinks about in PSC in PVC, we expect to report on the interim analysis in the first half of next year, we continue to push on screening activities and are looking forward to sharing you have there.

Lastly, I am proud of the growing body of important clinical data and real world evidence validating.

For patients with Alger syndrome empty thick.

The body of research was presented at the Nash again meeting recently, including data from our March Piecyk Phase III study and real world experience and knowledge they'll send them spaced in a tremendous effects across the clinical and real world things.

In summary, <unk> leadership position in pediatric Hepatology continues to build and we remain committed to growing our presence across these indications.

We also look forward to the multiple upcoming data readouts across settings, where we believe <unk> inhibition can play a key role in changing the treatment paradigm for patients living with biliary atresia PSC and PVC.

And with that I'll now turn the call over to Eric to discuss our financial results Eric.

Thanks, Pam before I begin I'd like to mention how pleased I am to have joined <unk> team at such an exciting time for the company and I look forward to helping advance new medicines for patients with rare diseases.

Now for the third quarter financial results.

Earlier today, we issued a press release that included financial results for the quarter, which I'll briefly summarize full details can be found in our Form 10-Q also filed today.

As highlighted by Chris and Peter we recognized $47 7 million of net product sales in the third quarter at 47% increase over net sales in the previous quarter.

This reflects product sales of 38 7 million for the modeling as well as $9 million 14, a dollar in call button I'd like to point out that for the bile acid products, we only recognize product sales since the date of the transaction closed on August 31st, but we'll see the full quarterly.

<unk> of these medicines in the fourth quarter of this year.

Total operating expenses for the quarter were $72 9 million, which includes R&D expenses of $26 1 million SG&A expenses of $36 5 million and cost of sales of $10 2 million.

For the quarter ended September 30, net loss was $23 6 million or <unk> 57 per share.

Net loss for the quarter included non cash stock based compensation expense of $8 4 million and intangible amortization of $2 6 million.

At the close of the third quarter ended September 32023, we had cash cash equivalents and investments of $306 million.

In summary, we're well funded with a growing revenue base, which places us in an exceptional position to execute on our business plan, including execution of our clinical trials 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.

Thanks, Eric as you can tell Miriam has continued to execute on all fronts.

We've built the leading franchise in pediatric hepatology <unk> with three standard of care medicines, and a leading genetic testing tour.

We are seeing strong sales growth across all of our medicines.

We've generated important phase III data to support the filing for approval of keynote all in CTX.

We have several clinical and regulatory catalysts ahead, as we strive to make continued advances for rare disease patients around the world.

And we've accomplished all of this while putting Miriam and a strong financial position.

We look forward to keeping you updated on our exciting progress as we head into 2024.

Operator, please open the call for questions.

Thank you.

If you would like to ask a question. Please press Star then one on your telephone keypad.

We have the first question from.

Joseph <unk> with Cantor Your line is now open.

Thanks, very much for taking the question and congrats on another exceptional quarter just wanted to ask because ipsen on their call indicated that they had seen some switching of patients from <unk>.

Merely to Bill way for <unk> syndrome curious as to what Youre actually seeing.

From your lens and whether you expect their lunch too.

To impede the strong growth curve, you've you've been on for Lyft squarely. Thank you.

Yeah.

Thanks, Josh for the question I'll hand over to Peter to talk about the competitive dynamic yes. Thanks, Josh.

Indeed laboratory has been approved and marketed in the U S for over two years now in Q3 as the first quarter, where there was another product available in <unk> syndrome, and I think that what we really point you to is the headline tape.

The net revenue that we saw in Q3 was a really strong step up over Q2, but even a little stronger than what we saw from Q1 to Q2 in terms of the viral use sales.

So we think the story is there the <unk> growth trajectory is very strong and that's not tongue.

<unk> not been impacted.

Can you comment on the split between U S and rest of world as well can you comment on whether you have seen any.

Attrition of patients to two buildings and if so how many and why do you think they might have switched.

Well I think on the Peter commented on the U S dynamic internationally.

The regulatory situation is a bit different where we've not seen in Europe. For example on approval of build the analogy of the syndrome.

So we are.

We are continuing to grow and add patients. There is a really strong situation for international markets really anchored around Europe at this point.

Great. Thank you.

Thanks for the questions. Thank you.

Your next question comes from Kevin Clark gotten at Evercore ISI.

Hey, congrats on the quarter and thanks for taking my questions.

For the Embarq readout, what is the baseline bilirubin value taken in relation to the Kasai procedure.

Yeah. Thanks for the question.

So the baseline bilirubin that we've seen the embark study is very similar to what's observed in the natural history.

Pre kasai, it's about close to about 10, which has been shown in a few extra patients at about nine five to 10.

And post aside we see it around seven and so that really makes a lot of room in the six months for improvement.

A treatment.

So we can start sharing that data later.

For this year.

And just to clarify is the baseline value from the trial after the kasai limits around the $7.

Yes, it's after the cost side.

Okay got it.

Thanks for taking the question.

Thanks, Kevin.

We now have money for <unk> with Leerink partners. Your line is open.

Hey, congrats on the strong quarter and thanks for taking the question Ed.

I guess the broader question around.

Looking high approval.

The NBA and value, which at all.

Basically part of the data in hand.

How should we think about the avenues that you have available to you to accelerate growth.

Whether through.

Pricing promotional opportunities et cetera, other perhaps more available than it was prior.

Prior to having that data and FDA approval and how should we think about avenues to accelerate growth in that asset potentially expand margins as a consequence.

Yes, thanks for the question Manny.

<unk>.

We anticipate FDA approval <unk> Cts potential approval in 2025 and thinking.

Thinking thinking from that point.

The opportunity here is to increase the diagnosis rate once you have a diagnosed patient in a clinic, we see substantially all patients are offered a T. Bill already it's really seen as a necessity.

So, but we do know from Kols and literature diagnosis rates are quite low we think only 10% of patients approximately are diagnosed. So we've spent a lot of time thinking about what can we do to increase that 10% obviously.

Don't have to do a lot.

To make that make a difference.

In terms of the growth of the products, So where we have a team and efforts around.

Promote disease state awareness.

Testing and neurology ophthalmology medical genetics to try to to try to find more patients essentially to bring more patients into a diagnosis and under care.

Great and then I guess secondarily on the pipeline.

Obviously, a lot of focus on biliary atresia, given the unmet need there.

Presuming that you see are positive.

Our positive results on bilirubin some perhaps.

Directional benefit on an.

To vet transplant, obviously studies are powered for that.

Can you walk us through what the time horizon would be.

Now what the steps are for you to engage with regulators and then be able to perhaps provide us and investors on in a public setting up an update on what the file ability or pivot or.

Probability or a path to approval, but it would be that indication.

Yes, thanks for that question Mike.

Biliary atresia timelines have laid them out in total.

The topline data from from the embark study.

At the end of the year.

And we'll then take that data the way you described having a positive bilirubin analysis and supportive trends on outcomes would be a really strong outcome here that we'd be excited to take forward to FDA. We look to have that meeting in the first half of the year.

And sometime towards midyear be able to update publicly on what our plans are on taking it forward.

Based on the FDA input.

Great that's helpful.

Is it reasonable to assume that that might be something we'd hear from you guys like in the first half of next year or is it just too early to kind of make those assumptions around like our.

Narrow that timeline might be.

We take.

Types of interactions are dependent on scheduling with the FDA. So as we get closer through getting to a meeting with FDA, we would be able to provide some clarity for now the best guidance. We have is having an update by midyear next year.

Great that's helpful.

Other analysts waiting I'll hop off blood.

Excellent thanks for the question.

Thank you. Your next question comes from the line of Steve seed House with Raymond James.

Okay.

Thanks. Good afternoon, everyone wanted to ask about embark first just do you have a sense based on the demographics, you've enrolled the number of clinical events you'd be able to handle.

Analyzed by the time of the topline data and also the registry work you guys are doing in parallel.

Will that be available and part of the topline analysis before year end secondly.

Secondly, I just wanted to ask about the PBC and PSC data.

I guess delayed.

Maybe you would refute that but the.

And widened in terms of just the expectation on when the timing would be of those in terms can you just talk about.

Some of the inputs.

There as well thanks, so much.

Yes, thanks for the question.

So with regard to your first question on the events I'm familiar with it and we are seeing events coming in.

And as you can expect these patients are highly progressive disease, but this is blinded data and we will be able to share topline more information with you towards the end of the year.

With regard to the registry and we're working very closely with the natural history registry as you related to and what we're really looking at is aligning our patient population and putting patients in that registry to really understand in this particular embark population what the.

The levels are very prognostic for transplant free survival and that will hopefully will help to.

Bolster our support of information as part of our discussion with the FDA.

With regard to PSC PVC, yeah. So we've seen some screen failures come in since our last earnings call. A lot of these are coming from the E diary compliance so patients have to complete pruritus score daily. So we're working closely with the sites to minimize that theres been a few patients that have <unk>.

Greenfield for liver labs screening with the labs, and then a couple for us.

Thanks credit scores.

But we remain actively screening I'm really excited to cause tissue data next year.

Alright, thanks, so much.

Thanks for the question.

We now have David Lebowitz.

Thank you very much for taking my question considering the.

DNA recently.

A firm decision to not kids orphan disease status to build a for lgs.

How do you.

Believe that leads through or just not read through too.

Molly relative to <unk>.

Thanks for question, David Yeah, It's a really interesting situation on watching this unfold and I think just for a little bit of background on the key factor is may.

To maintain orphan designation is to have some significant benefit over available therapies.

Our understanding of what the recent outcome was with EMA on the recent calls Youll syndrome decision and we're.

Yes.

Waiting to hear back for final feedback from EMA.

Optimistic about the dataset.

March P. Fixed study provides and that it's a broader genetic.

Profile of patients.

It starts at a younger age and it's a stronger response rate those are all things that we included in our submissions and some of the briefing materials on this point and we should have an update on feedback from the agency by the end of the year.

Thanks for taking my question.

Yes, thanks for the question.

We now have Brian Tony does that.

Hey, good afternoon, guys. Thanks for taking the question. My question is also on <unk> can you just.

Give us a little bit of an idea how you sort of characterize the primary the change in bilirubin and what that means versus the secondary endpoint.

Getting patients below two per deciliter and interesting time in terms of expectations from the placebo arm on that secondary endpoint I think the children liver disease Research network.

Is that about 50% of intense.

At three months I believe last week or so.

Is that a reasonable assumption for the placebo rate or for that endpoint and embarking on is there anything to consider in the design that might make that more or less.

Thanks.

Yeah. Thanks. Thanks for the question. So maybe I'll start with your last question first of all in the natural history that shows that somewhere between seven five to 10 from the literature.

Post assai in less than half of those patients will clear Jonathan So the majority of those patients will remain elevated to varying degrees as you alluded to those patients that are that are doing really well and have good established wildfire lesson two milligrams per deciliter, they will do pretty well, but the majority of them are really between two.

And greater than six and so we'll also be looking at.

If we shift patients from a high risk being greater than six to moderate risk between two and six and it may be industrial moderate risk to low risk. So we will be looking at bilirubin and all different ways and our primary endpoint to answer. Your first question is looking at the proportion of the percent change difference between.

Active and placebo at month, six using MMR and analysis.

And I think that was it was there.

Another question.

Thank you.

We now have.

Okay.

Zero.

Hi, Good afternoon, everyone. This is Thomas Yip asking a couple of questions for Ed. Thank you for taking my questions.

So first congratulations on the positive results David said earlier this month earlier last.

This month actually.

So can you talk about a little bit about Kindle.

The CTX first half next year.

In addition to restore data.

What we anticipate will be cost that data package would that be a historical data or.

Both sports post marketing usage data.

Thomas Thanks for the question.

Debbie.

The NDA approach, we see as relatively straightforward here for Keane at all there's been extensive back and forth with FDA on what they are expecting for the NDA and the discussion of the restore phase III study design.

So that's something that we did work on and got comfortable that.

This was there was getting alignment with FDA that this would support an NDA for the CTX indication.

And the NDA will include kind of a typical battery of additional data analyses that are that you would see for.

Our small molecule application, so nothing particularly unique about the NDA overall.

Okay got it.

And then perhaps just one more question from us.

All of them already and outages in Europe.

S market.

Get close to the end of the year, what's your anticipated.

Growth.

<unk>.

R J.

<unk> income for 2024.

We have not provided any guidance on 24 at this point and were still discussing internally, whether we will.

If so what then witness so stay tuned on that.

Okay understood.

Okay. Thank you again for taking my questions.

Looking forward to.

Embark data readout.

Thanks Thomas.

Thank you.

We now have a final question on the line from.

John.

Of JMP Securities.

Yeah.

Hey, Thanks for taking the question.

Just on Canada on cobalt I'm wondering if you could talk a little bit about the early experience with those products and then also the genetic testing.

What kind of levers do you think that will give you for with barley down the road.

But how how should we think about CTX approval and its potential impact on sales.

Yeah. Thanks for the question Doug.

It's been it's been.

Really exciting to bring these products in the genetic testing into Mira.

Yes, Ken commented on some of the dynamics in the script or our liver team now is focused on promoting both live Marley and cobalt.

Pediatric herpetologist as well as.

Kind of having a leadership role with the with the cholestasis snack testing, which really kind of enhances the value proposition that the Marin team can bring to our customers the prescribers and other health care professionals, certainly improves access and ability to kind of <unk>.

Having reasons to be in front of their customers. So really excited about what this is doing premiers profile in supporting <unk> growth.

So I think thats.

One point and then yes, we as we move forward into 2020 for 2025 continue to expect the bile acid products to deliver low to mid single digit growth.

We think we can.

We're excited about the investments we can make in Cts and trying to increase the diagnosis rate. They are buying more patients find them earlier, when we could probably have a larger impact.

Helpful color. Thank you.

Thanks, John.

Thank you as a reminder, its star one to ask any further questions.

Our chemical family of Mesa.

Q3 2023 Mirum Pharmaceuticals Inc Earnings Call

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