Q2 2023 Mirum Pharmaceuticals Inc Earnings Call

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Good afternoon.

And thank you all for joining I would like to welcome Eagle Cheat and Marion from the huge course report second quarter 2020, right financial result, and provide business update.

<unk> and I'll be your moderate careful faithful.

All lines are on mute for the presentation portion of the call today.

Opportunity for questions at the end if you would like to ask a question. Please press Star then one on your telephone keypad.

I would now like cabinet cool AVG.

Angie Mccabe.

VP Investor Relations and finance so Andrew Please go ahead.

Right.

Thanks, Brad and good afternoon, everyone.

Like to welcome you to Miriam Pharmaceuticals second quarter 2023 conference call I'm joined today by our President and CEO , Chris <unk>, Our Chief operating officer, Peter out of edge, and our head of research and development handbag.

Earlier today <unk> issued a news release announcing the company's results for the second 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 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 mirror our pending acquisition.

<unk> bile acid product portfolio and our program is based on management's current expectations, including statements regarding <unk> regarding <unk> current and future business and commercial plans development programs regulatory expectations strategies prospects market opportunities and financial forecasts and guidance.

<unk> 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 <unk> filings with the SEC with that said I'll turn the call over to Chris Chris.

Thank you Andrew and good afternoon to everyone joining us on the call today, we are thrilled to discuss yet another quarter of strong growth in Europe .

We are on track to become a leading multi product franchise and pediatric herpetology through the impact of look Marley for patients globally continued progress of <unk> in the clinic and the pending acquisition of Keane at all and cobalt.

The last quarter saw substantial progress on our five part strategy, which is to grow with Marlin in the U S launch live Marley in international markets established new indications for lip Marlin advancing commercialized Melissa that an adult call static indications.

And acquire additional high impact medicines for rare disease.

We've made great strides on all of these areas of strategic focus so far this year.

First on growing Lamar lease commercial business in the second quarter, we performed well against our guidance of 50% year over year net product sales growth in the U S. In 2023.

As well as growth from international markets, achieving $32 $5 million uplift Marley revenue and $37 $5 million total revenue.

We are also excited about the recent progress in international markets with our approval in Canada, and a growing list of countries selling live Marley and distributor markets.

With Marley indication expansion efforts are also making great progress with our <unk> application under review with the December 13th 2023 Paducah date.

We're looking forward to the potential label expansion based on the compelling March phase III data and bringing with <unk> patients.

In addition, the Embarq biliary Atresia study is fully enrolled heading towards top line data later this year.

<unk> is also progressing nicely towards interim analyses in PSC and PVC later this year positioning those studies to expand into their potentially registrational portions.

And finally on the fifth strategic pillar of product acquisition.

We are advancing towards closing the acquisition of Keane at all in cobalt.

Two commercial products with over an aggregate of $100 million of annual net sales in 2022.

As we've shared recently, we expect this acquisition to deepen our leadership in pediatric herpetology and accelerate our growth across our product line.

We expect the transaction to close in the third quarter and are excited about the split of these bile acid replacement medicines with.

With Lyft Marley and the upcoming phase III readout for Keane at all in CTX.

Financially, we are well positioned to execute on our strategy. We ended the second quarter with a $330 million cash balance in Q2 was another quarter of financial growth.

With relatively steady operating expense levels and a growing top line compared to the first quarter.

The pending addition of Keane at all and KOL bomb offers an opportunity to further accelerate our revenue with a highly synergistic business.

Concurrent to this pending acquisition, we entered a private placement agreement for $210 million supported by several existing and new investors, which we'll use to fund the upfront payment in connection with the acquisition.

This financing allows us to maintain the strength of our balance sheet and positions us for continued growth.

With that I'll pass the call over to Peter to discuss our commercial business in more detail before Pam gives an R&D update.

Thanks, Chris.

We continue to see strong demand for live <unk> with $32 5 million and <unk> net product sales in the second quarter of 2023. This represents 86% growth over the second quarter of 2022.

Our U S business continues to perform well with net product sales of $26 2 million.

This growth was driven by strong underlying new patient demand.

Notably in Q2, we saw an increase in uptake by patients less than 12 months of age consistent with the <unk> label expansion to three months of age and older in March.

Early treatment and allergy Olson is important to look.

To limit the impact of the disease and we're pleased to see with Marley addressing this need in the real world setting.

The U S business performance continues to track very well against our guidance of 50% growth in 2023 net product sales.

Now turning to international markets. We can see we continue to see strong de novo demand in Germany, and France launches in line with what we saw in the U S launches of Marlin.

Pricing and reimbursement discussions are ongoing and major European markets, which we expect will continue throughout 2023 with full launches in most European countries starting in 2024.

We also anticipate use of named patient programs in European and distributor markets to continue to grow throughout 2023.

Overall, we are making great progress and are looking forward to a potential label expansion for <unk> in December in the United States.

And the upcoming readout of the embark study in biliary atresia.

We see the total opportunity for live Marley across Allergist syndrome, <unk> MBA is greater than $1 billion.

And that this opportunity is strengthened by our recently announced agreement to acquire <unk>, and cobalt, which will deepen our presence in pediatric herpetology and established a focused multi product franchise.

There is a substantial amount of top and bottom line synergy to unlock by putting the bile acid portfolio and live Marley together in the hands of one commercial team as the marketing efforts are similar in scope and the approach to market access product distribution and patient services are quite complementary.

We expect the bile acid products to continue to grow at low single digit rates annually with the potential indication expansion to CTX for keynote <unk>, providing an upside opportunity to increase patient diagnosis and treatment upon approval.

Financially the strong second quarter net sales of $32 5 million dilutive Marley and $27 5 million for keynote <unk> and cole bomb reported today by <unk> totaled approximately $60 million for the future combined franchise with growth expected across all three <unk>.

Thanks.

In summary, we are building on the tremendous momentum and potential of our expanded portfolio to deliver these important medicines to patients with rare liver diseases on.

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

Thanks Peter.

We are thrilled to be heading into a data rich second half of the year.

First as Chris mentioned, we are eagerly awaiting the December 13th <unk> date for <unk>.

Secondly, we announced in May the completed enrollment for the Embarq phase III study and Guardiola Tricia.

This study has a six month primary endpoint assessing bilirubin and we're on track for the topline data readout later, this year, which will be the first placebo controlled data with an <unk> inhibitor in this setting.

I'm also pleased to say that the phase <unk> study of elixir that in PSC. Currently has the total number of patients randomized or in screening required for the interim analysis.

This interim will be triggered when 45 randomized patients have been on study for a minimum of three months and is expected around year end.

In addition, the interim analysis for the Phase <unk> study in PBC is also expected around the same time.

As a reminder, after these interim analyses both studies will move into the potentially pivotal portion of the trial.

And finally, turning toward the pending acquisition, we have high confidence in the success of the restore phase III trial evaluating <unk> in patients with CTX.

Look forward to top line data in the fourth quarter.

If positive this study could support a label expansion for CTX with the potential for orphan exclusivity.

Now regarding our ongoing efforts to present, the growing body of data on live Marley. This summer we presented 12 abstracts at both diesel and the European pediatric Hepatology Congresses. These data demonstrated live marley significant ability to reduce bilirubin.

Symptoms associated with allergies sentiment <unk> as well as real world data in which patients with <unk> syndrome came off of the liver transplant waiting list after live monitoring them.

In addition, we published a seminal analysis in herpetology demonstrating that paradis.

And bile acids, and bilirubin or significantly predictive for transplant and event free survival analogy there.

Showing that 93% of live Molly treated patients with pruritus reduction remained transplant free after six years.

So in summary, the upcoming data readouts across indications along with the ongoing robust clinical evidence of love Marley and both allergy landscape.

Cements, our leadership position in pediatric herpetology.

And we remain committed to growing our presence in rare disease.

And with that I'll turn the call back over to Chris Chris.

Thanks Pam.

<unk> is proud to be the leader in pediatric herpetology.

The team continues to deliver high growth with lip Marley and make important advances in treatment options for cholesteric disease across our clinical programs.

At the close of our acquisition of keynote on coal bomb expected in the third quarter. This year, we will have three approved rare disease products and upcoming producer date for label expansion for lip Marley and four additional indications in development in high need orphan settings with upcoming clinical readouts.

We are exceptionally positioned to expand our leadership and impact in rare disease and look forward to bringing these important medicines to patients around the world.

With that 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.

Operator, we have any questions in the queue.

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I apologize.

First question.

Steve <unk> with Raymond James.

Your line is open.

Hey, good afternoon guys.

I wanted to ask a couple questions about.

Embark the first one is just.

Are you like our patient screening consented.

And ultimately enrolled in the study before they've had their kasai procedure.

Or in the days or weeks after and I'm curious if there is a lead in period sort of before treatment, where you track to bilirubin kinetics and see sort of what the.

Losses to the surgery before initiating treatment I have a couple of follow ups as well.

Alright, Thanks for the question I appreciate it.

So for the embark study the patients immediately post the size of a discussion happens sometimes prior to the Kasai procedure.

When they learn of the diagnosis after the after the baby is born and so the consent process can happen either before the kasai surgery or immediately after the BSI surgery, but the goal is to get these patients onto the once again, we are now fully enrolled with to get these patients into the study as quickly as possible.

There are no criteria for bilirubin in the study so we're not looking at any bilirubin effects post the site, we're enrolling all patients and we're really excited to have the study readout towards the end of the year.

Okay great.

Okay. Thanks for that and then just on the six month analysis I'm curious if there is a formal assessment of.

Like transplant.

Or other liver events already in that six month time point.

Or or let's say just through last follow up of all the patients that are on it.

Ruled by the time, you announced the six month bilirubin analysis.

Yeah. Thanks, so for the six month primary endpoint as you know is bilirubin, but we are assessing in other key secondary outcomes of transplant at that six month time point as well as other thresholds of bilirubin that are predictive of transplant.

And then these patients at the end of the double blind treatment period rollover into open label extension. So we will continue to follow those patients long term.

Okay.

Okay and then just the last question I have was just on that open label extension. So.

I think correct me if I'm wrong I think it will go through 24 months total so 18 months post the six month analysis and the question is do you think youll have the ability to leverage natural history control cohort data.

In similar ways that.

Just based on some quality databases and allergy on <unk> for instance.

Here for biliary atresia or if not is that work that you would hope to do at mirror in the coming year. Thanks, So much.

Yeah. That's a great question, we're actually doing that right now we're working with natural history registry looking at.

Kind of aligning those patients with the patients that are enrolled in our study and understanding a little bit more about thresholds and prognostic markers for transplant free survival and we'll continue to work with those registries throughout.

We're looking at six months time points and that will obviously continue to look at long term long term treatment versus natural history. So all of that will be kind of as as much as we can in the time frame.

The package will contain our dataset plus work that we're doing supported by the natural history, and we'll have discussion with the FDA at that time.

Great. Thanks, so much.

Thank you.

Your next question comes from Martin Currie.

With SBB Leerink.

Just Eric now I guess.

Thanks for taking the question guys.

A quick commercial question I know, we had a little bit of discussion last year around <unk>.

Seasonality can you give us a sense of where those trends are now how should we think about modeling.

And sort of what are the sort of key metrics, we should be following as we think about as we think about.

Going through each season this year.

Okay. Thanks for the question Matt Yes.

Yes.

What we've seen in Q2 is really strong demand, but feel really good about how the business plan heading into Q3, but.

Mentioned and we're just now.

Getting a August so we'll have to see how things unfold over the next couple of months.

Overall feel really good about how the businesses going forward.

Great that's helpful.

And can you separately looking forward post presumptive deal close of the accuracy of the acquired assets.

How should we think about.

Margin evolving over time, obviously, you then have a little bit of expense that you place that you bring on but that could be coming with a substantial amount of revenue is there room for further margin expansion going forward.

What time horizon might we see that as we transition from this Q4 first quarter with revenues from all the assets together.

Through next year.

Thanks, Mike for the question.

The overall picture and bringing key at all on coal bomb in Samira, we do see a tremendous amount of synergies. So they will contribute substantially to margin at mirror because of the overlap in our business.

A lot of that behind some of the rationale why we are so excited about bringing these products into our portfolio.

We're not going to be guiding towards margins, but what I can say is that the.

This puts us on a very sound financial footing.

We do expect all three products to continue growing for the foreseeable future. So that will continue to improve financial performance as we go.

Okay.

Great. That's very helpful. Thanks, guys congrats on the quarter.

Yes, Thanks, Mike.

We now have David Nicholas.

With Citibank. Please go ahead with your life.

Hi, Thanks for taking my question. This is <unk> on for David.

So we wanted to ask if you could update us on the timing of the closure on the caviar transaction and also could we have some insight as to how the operating Bang like ship post closure. Thank you.

Yes. Thanks for the question, we're tracking towards closing this quarter. So we do expect a Q3 close.

And then as we move into a post close time periods. We're excited about jumping right into to bring these products over to narrow.

Overall, I know that they'll have a high impact in terms of.

Margin and cash contribution right at the start in Q4.

So see that synergy will play through and having these products in our hands. So I expect that impact to show up with improved.

Performance in.

Q4 from having these products that mirror.

Okay, maybe just a quick follow up on that so how what's your strategy on incorporating this product into your current pipeline.

Thanks for that.

But with the Navy.

Absolutely Yeah, we can ask Peter speak for some of the planning phase.

Fit very well with <unk>.

Morally and the current with Marlin business Paragon into some of the strategies, we have planned thanks, Chris.

Yes, we're really excited about bringing these products in the mirror.

Excited about bringing several really counterparts from severe and congrats as well.

We have a team right now thats been out.

Promoting with violated pediatric herpetologist.

Anticipate that team continues.

Also we will have achieved that focuses on.

Physician Subspecialties that are often involved.

Identifying.

Patients with bile acid synthesis disorders.

I wouldn't expect them to source et cetera.

<unk> on neurology ophthalmology.

Medical genetics.

Ever focus team and come over.

Calling a metabolic focus gene.

And modest expansion of our field footprints pretty pretty.

We're excited about how that plays out going forward.

Okay. That's very helpful. Thank you.

Thanks for the question.

Thank you, we now have AG of H C Wainwright.

Hi, good afternoon, everyone business comments, yet asking a couple questions for Ed. Thank you if that's okay no questions.

Perhaps first question.

Regards to embark data regarding data positive.

Given the <unk> orphan drug designation for our bedroom and Tricia.

Anticipate embark topline data along with natural history data set that you mentioned earlier.

Using collect that lead us.

Enough to support with <unk> approval.

Thanks, Alex for the question overall that is that's our strategy to take that package to FDA.

It's too early to say what the determination will be at this point.

Bilirubin has not yet been fully established as an endpoint for approval.

The work that we're doing with the Embarq data and the natural history work. So looking forward to putting together, what we think will be a.

Really compelling dataset.

Okay got it and then perhaps.

Hey, Pete.

Oh over some major differences, but didnt know of so far between.

FDA and MAA.

The EMA.

What they are looking for.

Who are approvable endpoint, Bulgaria Richard here.

Got it thanks for the question there I mean, there we see a pretty common point of view and what we've had for our conversations and I think it's worth noting that there really is not a lot of clinical data or research that's been done in this space. So the embark study.

Really is a landmark is going to be the largest and first study.

A study of <unk> in the setting.

And one of the few studies that actually has an intervention that early after the kasai, So really important dataset to bring forward to advance care for these patients.

Got it.

Perhaps one final question for <unk>.

Mentioned, the interim analysis for both.

Visa and advantage studies both expected later this year can.

Can you please tell us what type of data.

We can expect to see.

Yes. Thanks for the question so for the PSC. This the study this is a blinded interim.

So we won't be sharing any data.

But it is for dose selection and then the city will upsize if need to or not and then carry on into potentially registrational part two of the study. So wouldn't expect any topline data out there about wed expect to be able to.

Communicate that the study is carrying on and Christian Johan for the PBC Vantage study.

We will be sharing top line data. This is an open interim analysis.

And so we'll be able to have a look at the data and we'll be.

Sharing that with you later.

Okay understood.

Again with the kind of questions.

Data from the <unk> study.

Thanks Thomas.

Yes.

Thank you Peter to ask any further question. Please press Star then one on your telephone keypad.

Our next question comes from Brian <unk> with Baird.

Hi, This is Charlie on for Brian So.

I wanted to ask about the SG&A, we've seen it climbing somewhat steadily over the past several quarters just wanted to know, particularly as you start to integrate.

The bile acid team from <unk>, if we should expect that to keep rising what kind of trajectory that's going to take and along a similar line thinking about <unk> of that as that hopefully gets closer to.

Getting commercialized.

What kind of regulatory strategy are you taking going forward and considering that these are adult liver diseases will this require a different commercial sales force or do you think that you can also leverage the field force that you have currently thank you.

Thanks for the questions I'll comment a bit on SG&A.

So our strategy as I sort of Peter talked about the sales team.

For SG&A, we have what we see is kind of this modest increases over prior quarters really driven by.

Initially the launch in the U S and now some of the international markets that we brought on.

So just for the base business, some incremental SG&A increase over time as what we'd expect and then with adding in Kindle and KOL bomb that will bring incremental SG&A expense, but really modest compared to the top line opportunity.

As Peter was mentioning earlier Theres a lot of synergy in how we approach those markets and the fit with <unk>.

With Marley so we aren't we are bringing some people over who has great experience with those products from severe <unk>.

Excited about bringing those folks into the mirror family.

But overall.

The total contribution will be.

Quite attractive with these products in our hands.

As we look forward to the <unk>, which will be.

We have an opportunity to expand into adult hepatology <unk>.

First the <unk>.

And point in registration to comment on these studies incorporate pruritus is the primary endpoint.

Similar to ours, you'll syndrome, and the ongoing review for <unk> pruritus is the.

Outcome for approval and that's what we're using for the registration approach for the adult indications.

I'll, let Peter comment on the commercialization strategy.

Interesting.

I think we would be able to leverage our commercialization team and infrastructure.

Watch list status for adults. Our current team is primarily pediatric focus.

Maybe it goes to the large higher decile a larger volume of adult other doctors, but we would look to expand to a broader a broader group of adult hepatology suggest urologists I would think of it as kind of an incremental bill for that launch.

Great. Thanks, a ton for the color and congrats on the quarter.

Thanks for the questions.

Thank you.

I'd like to hand call back over to Chris for any final remarks.

Great. Thanks, operator, and thanks again to everyone for joining today's call have a great day Goodbye.

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