Q1 2022 Mirum Pharmaceuticals Inc Earnings Call
In the first quarter, namely net product revenue of $10 9 million.
Continuing to show the progress of our commercial efforts.
Our first quarter performance gives us confidence in continued growth throughout the year.
And I'll, let Marty opportunity for Clos static drivers and <unk> syndrome is only the beginning of what's possible for Miriam.
The foundation of our pipeline and Miriam success is innovative research for rare disease to bring new medicines to patients as quickly as possible.
As we continue to execute against the greater than $500 million U S market opportunity analysis Youll syndrome, we are progressing our five other late stage with <unk> and <unk> clinical programs, which will generate multiple data readouts over the next two years.
Well in parallel we are progressing expansion in global markets with Marley for hours Youll syndrome.
Now with that I'll turn it over to Peter who will provide an update on our <unk> launch progress Peter.
Thanks, Chris.
We're proud of with mileage launch success and its role as the first and only FDA approved medication for pruritus analysis.
Dave I'll share further color about our $10 9 million net slip mildly relevant.
What we're seeing in the commercial business and why we're so excited about the growth trajectory.
Before I get into the details of the quarter I think it is important to note that there was no inventory in our reported product sales. Thus the revenue number here is a true representation of demand.
Now taking a closer look at the first quarter revenue was driven by strong demand dynamics consistent rebuilt cadence in both broader and earlier than expected support compares.
On this point in the first quarter approximately 75% of the expenses were reimbursed.
And we expect the rate, we expect that rate in Q2 and beyond to be at 90% or higher.
Regarding payer mix, we continue to see approximately 50% Medicaid and 50% commercial at this stage of the launch.
And we've heard very positive feedback on treatment benefits from clinicians and families observing impressive treatment compliance in the commercial side.
In fact, we are seeing similar compliance from what we saw in our clinical trials in the real world setting a testament to the importance of this medicine for patients and parents.
On adoption dynamics, we're pleased that the majority of our targeted to key accounts have prescribed <unk> tomorrow.
Further we have seen that some patients residing in outlining areas often don't travel through a major center in a metropolitan area and instead receive care from the community based pediatric Gi of liver tox.
Accordingly, we've added a few sales territories to ensure our teams can adequately reach patients.
Turning to our plans for <unk> International launches the mirror International team and our partners are preparing for launch following anticipated approval in Europe later this year.
Our team is initiating early access programs now while ensuring the readiness for launch in key European countries.
Further our seven commercialization partners outside of Western Europe are matching our dedication to introduce the <unk> to patients around the world with potential approvals starting early next year in these geographies.
And now I'll hand, it over to Pam to provide an update on our pipeline.
Thanks Pierre.
In the first quarter of 2022, our team has been focused on laying the groundwork prior important upcoming milestones for this year and beyond.
Most notably we expect top line data from our March <unk> phase III clinical trial in the fourth quarter.
As a reminder, the March peak study now fully enrolled includes multiple piecyk subtypes of higher doses from the phase III Indigo study, which was the basis for breakthrough therapy designation.
And the March study, if the largest randomized phase III clinical trial ever conducted in <unk> with more than 90 patients enrolled.
Moving to the rest of our pipeline, we have four additional indications under evaluation with cholestasis.
By year end 2022, we're expecting two interim analyses from our elixir that programs.
First interim analysis will be from our phase <unk> study evaluating <unk> in patients with primary sclerosing cholangitis and the second will be open label data from our phase <unk> study for patients with intrahepatic cholestasis of pregnancy.
And in 2023, and we're targeting interim data from our phase <unk> study evaluating <unk> in patients with primary biliary cholangitis.
In addition, we're expecting primary data from our phase <unk> study evaluating <unk> for children with Tricia.
And then prides itself on its innovative research and for being recognized and well respected peer reviewed journals and with that I am very excited to share that our <unk> indigo phase III data have been published in herpetology communication just yesterday.
The authors found that <unk> that led to rapid and sustained reductions in serum bile acid levels in patients with non truncating piece of Q, leading to five year of transplant free survival.
As well as reductions in pruritus and meaningful improvements in growth and quality of life.
They further concluded that <unk> appears to be a realistic and effective treatment strategy, which benefited the lives of patients and caregivers.
We're leaving disease symptoms, increasing both survival and providing a well tolerated non surgical alternative.
We're excited about the potential to confirm and hopefully improve upon these results and our March peak of Phase III readout later this year.
In addition to the European Medical Journal also published our recent presentations from <unk> and natural gas.
So all in all we are very excited for what's to come as we build upon the incredible success of of Maui.
Static paradis impatient with I'll just end on one interventional there and we will keep you all updated on our progress across the various additional programs and data readouts and on that note I will turn the call over to Ian.
Thanks Pat.
A press release filed earlier today and the 10-Q to be filed later today provide a full financial update I'll call out a few of the highlights here.
Total revenue for the quarter is $12 9 million, including $10 9 million of net product revenue from this Marty trials and $2 million in licensing revenue from apartment coverage in China.
Our total operating expenses for the quarter were $45 6 million, which includes research and development expenses of $24 1 million and SG&A expenses of $19 1 million.
Cost of sales of $2 4 million.
Maryland remains well funded and at the close of the first quarter ended March 31, 2022, we had cash cash equivalents and investments of $239 9 million.
With that I'll turn the call back over to Chris for any final comments Chris.
Thanks, Tim and thanks, everyone for joining today.
To close mirror continues to make remarkable progress and nearly $13 million in revenue. We achieved this quarter speaks to not only our team's dedication to ensure this medicine is in the hands of patients.
But also to live marlies impressive safety and efficacy profile and ability to fill an urgent unmet medical need.
We look forward to continuing on this trajectory in the months and years ahead as we both build on this initial launch with Marley.
Thats the balance of our pipeline to develop meaningful therapies for communities living with rare disease.
Operator, please open the line for questions.
Thank you that just as a reminder, if you'd like to ask a question. Please your staff from about one on your telephone to keep at now.
And our first question is from Jessica Fye of Jpmorgan. Your line is now open. Please proceed.
Hey, guys. Thanks for taking our questions. This is Nick on for Jeff. So first question is from us.
With revenue of $10 9 million seems strong and well below the $8 million or that you gave last quarter can you just talk a little bit about what drove that the dynamics behind it and how you're thinking about the remaining quarters, leading up to be at least $50 million guide for the full year 2022, and maybe how those how that revenue should be distributed.
Yes, thanks for the question.
I can speak to kind of the full year.
Kind of a guidance question and I'll pass over to Peter talked about the dynamics underneath that.
And just to speak to the full year with at least $50 million number that we spoke about previously.
Was.
Provided a floor and not necessarily a guidance points.
What we see today looking forward for the full year is that we're comfortably above that number is where we expect the year to land. So I wouldn't look at that as a guidance number and feeling like we're tracking.
To be certainly above it and I'll, let Peter speak to kind of what drove the demand in the quarter and some of the dynamics out in the field for the.
In terms of the Q1 performance.
And I think we saw really strong demand dynamics as well as regional dynamics that we've been.
So I'm really pleased there with the cadence of refills and as I mentioned in the prepared comments also from the payer perspective.
He was kind of executed really well and.
Now we are at now are expecting going forward that 90% of our prescriptions will be reimbursed in that kind of came in a little faster than others.
Other rare launches that we've looked at which is informed by our expectations for the for the prior thoughts.
Okay.
Great and maybe just one quick follow up on that 90% drug reimbursed by year 'twenty two.
Currently stand now and should we expect you to achieve that 90%.
In Q4 could we potentially see that maybe before that in Q3.
We think Q2 and going forward.
The expectation for the 90%.
Great. Thanks, so much for taking my questions.
Thanks.
Thank you Nick and our next question is from Manny Suraj from SPP Manny. Your line is now open if you'd like to proceed with your question.
Yes.
Hey, Good morning. This is Rick on the call from Mani Congrats on the quarter and thanks for taking our questions.
So we're just looking for some additional color on the <unk> launch.
I guess first as far as the expanded access patients in the U S goes.
Of those patients where we have been transitioned to commercial product at this point or is that still an ongoing process.
Yes, thanks for the question.
So the expanded access and clinical study patients.
The conversion was pretty rapid actually so we saw them convert over to commercial drug in the fourth quarter.
So that first launch quarter.
Okay got it.
I guess I'll sort of thinking about later this year with the potential launch in Europe .
Could you just maybe discuss a little bit higher.
How you see the timeline to reimbursement across the EU and maybe some of the target countries for the initial launch there.
Sure.
At a high level following EMA approval.
The first country will launch in is Germany, where shortly after launch we kind of have the.
So sorry, shortly after the central approval, we'd have the opportunity to launch and then I think variable timelines our focus has been.
Primarily western Europe European countries, but.
Thanks.
A typical orphan launch timeline, we kind of expect over the next.
Over the course of 2023.
To be launching into.
Other western European countries of France, Italy, UK as well.
Yeah, and I'd add on that in addition to those to kind of mirror territories, we're going to be.
Folks on the ground actually launching the products through some of our partner and distributor markets will see expect to see approvals.
Starting early next year, leading to launches in some of those partner markets.
Okay fantastic.
A question from US I guess this.
Also thinking through potential company updates for the rest of the year.
We potentially see any guidance revisions as you get more color on either what Marty we launch across the year or do you think youre currently happy with for guidance and don't see that being updated for the rest of the year.
Yes, we're at this point, we're not providing continuing to not provide guidance, but just to reiterate.
Very comfortable that we are above that number so.
So I would not look at that $50 million guidance number at all frankly.
And so we're still early in the launch and things continue to track well, we do expect quarter over quarter growth throughout the year.
And.
As we get closer to year end, we will evaluate whether we provide our formal guidance when we get closer.
Great. Thank you so much for taking our questions.
Thank you.
Thank you and our next question is from the line of Josh <unk> Evercore.
Your line is now makes me. Please proceed.
Great. Thanks for taking the question questions. So you emphasized growth throughout the year to what extent is that do you expect that to be driven by addition of patients in the U S compared to contribution from from other territories Youre on a fairly torrid growth trajectory, thus far in terms of addressing the eligible unmet medical need how long do you.
I think you're going to be able to sustain this type of growth.
Thanks for the question Josh.
Youre looking at the dynamics and as we look at the growth for this year, we're seeing that largely driven by the U S. So actually those contributions will be very modest in the calendar year. We do expect some of it to come online, but as we talk about our growth expectations and the full year.
Number we're looking primarily at the U S.
And maybe I'll, let Peter chime in a little bit on kind of what the.
Dynamics in that over the year will be yes, yes, I think within the U S.
We're really pleased with where we're at today in terms of adoption.
<unk> ahead.
Comparable rare launches that we look at.
But still it's still see.
More addressable <unk> patients.
Haven't received with milder than those who have and.
Kind of a typical pattern, we see as our accounts, maybe prescribed with modeling for a more severe effect severely affected yields in Ah patients kind of game experience gained comfort and then broad.
So we expect to see kind of those dynamics continue to play out throughout 2022.
Great and then R&D spend has ticked down nicely over the past couple of quarters, how should we think about that line going forward.
Okay.
Yes.
R&D spend perspective, I think that one would anticipate 30 kind of 35 I think is the way that I look at it we're not giving kind of formal guidance on our opex for the year, but clearly as we look at some of the studies that are kind of coming off of the study just started to ramp up. So you see some of the for example, the <unk> <unk> study kind of as we come.
Towards the end of that wind down a little bit.
Inversely, obviously, we look at the ramp up on the Bluelinx about studies to replace the expenses there.
Characterize it as kind of a drop of ethanol.
Yes.
Great. Thanks for the color.
Next question.
Yes.
Thank you. Our next question is from <unk> Rahimi Sandler stimuli.
Tim Your line is now open. Please proceed.
Good morning team and congrats on a great quarter two questions for you maybe the first one would be.
There's a competitor product that's going to read out in phase three in LNG and I would love to in the fourth quarter.
Our activities are going to be put in place sort of into the second half of the year to really.
And sure that position.
Physicians continue to build strong brand awareness with Marley.
And and continue to really keep their patients on this product.
Not be enticed to switchover, that's one and then the second question.
Is on the advantage study that's going to readout.
So the interim.
Analysis is sorry, Mr Studies, that's what I meant to ask this the study can you give us a little bit of color of like.
What the intra quarter.
Cut off.
That would qualify the phase II b to that transition.
And to like a phase III.
And what type of information you would be sharing with us and thank you again for taking my questions.
Great. Thanks, Thanks, Jess speak to the first one then I'll pass it over to Pam to speak to that.
And I was looking at the impact of with Marley analogy Wilson I mean, the data we have there that's really the focus of what we're doing this year is making sure. We rollout then get access for as many patients as possible as urgently risk as we can and get some of the data that we've generated from liberum.
Ineligible syndrome published Pam mentioned.
<unk>.
Journal article that highlighted some of the long term event free survival data.
Building awareness of that and showing what.
It's been so exciting from the clinical program.
More broadly.
All plays into how we see the year playing out.
Let <unk> speak to our business.
Yeah. Thanks, Scott for the question. So just at a high level thoughts about programs altogether, we're really excited about the studies.
The opportunity that it presents across the Cola static indications and particularly for this study to answer your question.
Enrolling on expecting to complete a blinded interim analysis here.
And this interim is blinded so we won't be reporting out any data.
And we need to keep it blinded so that we can retain those patients to use them in the part two registrational question of the study.
And what we plan to learn from that interim and we'll be making a dose selection. So that data will inform our dose selection as well as the turbine determine sample size for the second question of the studies to determine if we need to upsize the trial or not.
Then we will see that into the potentially registrational questionable study. So we will keep you updated.
We talked about that portion of the study.
And I think just to kind of reiterate in brief recap the point on the business study design and we discussed it with FDA and incorporate all of their feedback for this to be the pivotal study once it moves into the second part to part two.
Tim just to clarify so let's say the announcement comes and says you don't need to increase the sample size.
And youre going to go with let's say the highest dose.
<unk>.
And does that mean that the blinded committee that looked at the data.
Really quite remarkable improvements in pruritus that gave them the confidence that it has a high statistical powering.
To hit in that by transitioning into a pivotal study like I guess, what I'm trying to get out is like.
How strongly confident can we be on that announcement.
But the efficacy was really astonishingly stronger than that.
And predictability of the phase III.
Yes, yes, yes, youre thinking about the right way, so theres, a prespecified threshold that.
It does mean that yes.
The results remain blinded.
There is an effect in there and so that's exactly how we design the study.
And if that's not the case, we will have an open analysis and share the findings.
We can.
Got it thank you so much.
Thank you and our next question is from the line of <unk> of H C. Wainwright. Your line is now open. Please proceed.
Great. Thanks for taking my questions.
Congratulations on an impressive quarter.
First I wanted to ask about.
What youre seeing I know you've spoken about in terms of the commercial progress early in this launch.
On several aspects, including on the payer side, but I'm wondering if you could speak about.
The dynamic between physicians and patients in sort of a push and pull there.
That is also underline the strong demand that youre seeing.
And I guess in a related question.
Some companies have been reporting.
Impact from the <unk> wave.
This quarter.
Actually in January February .
Any thoughts or commentary around that as well and I have a follow up thanks.
Yes, Thanks Ed.
Peter.
Thanks for taking the questions.
So, yes, I think the.
The dynamic between physicians and patients the periodic analogy Olson was really debilitating and I think it's recognized as one of the major unmet needs in the management of these patients. So I think as I mentioned kind of what we see is.
Particularly positions that arent part of the clinical program.
They want to get familiar with the Marley So they'll trial out on a couple of bit more severely affected patients I'm just kind of gained comfort.
With this new medicine, and then kind of broadened over time.
The other con way or is it really good good client.
We I think now that we're past that we are kind of kind of back in person and most of the country.
I think COVID-19.
And especially the staffing issues and a lot of these clinics that have kind of come up around the pandemic had been a pretty pretty big challenge for a lot of new product launches.
A lot of patients haven't seem to herpetologist in a couple of years, and we're scheduling and logistical backlogs to get them in.
The clinic.
And NBC, which is generally when clinicians would look to start a new product medication.
So I think there have been challenges there.
For us obviously, despite those challenges.
The performance has been.
Very very strong and I think thats another thing as those as those issues kind of workout work themselves out and we come out of the pandemic I think there is no reason that we could see continued growth.
As those dynamics.
That's great. Thank you.
And then I.
Just wanted to.
Ask.
Sort of.
We continue to build on this.
This commercial platform.
That is growing quite rapidly.
And.
With the additional lift Marley indications obviously.
Strongly overlapping in terms of call points in.
And just.
Thinking about how you could leverage what you're building now for future indications and thinking about.
How you can.
How would you communicate the.
The efficiencies there that you could gain.
And future indications thanks.
Thanks, Eric a couple of points to make on that firstly within with Marley spoon.
Not on that one.
100% call point overlap between indications particular eligible center probably has the broader call point of the three just given the dynamic of the indication and number of patients out there.
So that sets us up well to have a highly leveraged.
Our organization here as we launch the additional with Marley indications eventually and then expand into adult herpetology I'd say already.
<unk> business is highly performing in the first quarter. If you just look at the net revenue and commercial expenses were already breakeven as a commercial organization.
So see this as a really attractive business model.
We go and push further into the <unk> launch and add the additional indications.
Great. Thanks, so much and congrats again.
Thanks, Ed.
Thank you before we take our next question just as a reminder, if you'd like to ask a question. Please press star followed by one on your telephone keypad.
Our next question is from Brian <unk> of Baird. Brian . Your line is now open. Please proceed.
Hey, good morning, everyone.
Really impressive launch, but would really be great to get any sort of qualitative insight into sort of the penetration of the identified lgs markets. So far in the U S.
Obviously off to a quick start, but I guess from the field can you give any sort of thoughts on what the penetration is looking like into sort of a low hanging fruit or patients who are sort of warehouse or have prior experience with merrell looks about.
And under care versus kind of when would we expect to get to a point.
We're looking at pulling in patients, who maybe are not under active care identifying patients not in the system yet thanks.
Okay.
Thanks, Brian .
Just from a broadly speaking.
It's been a great fast start, but we do see opportunity in.
More patients to go deeper here. So we are still early in the launch maybe I'll ask Peter to speak to some of the things we're doing to kind of expand and kind of go further into into the launch yes sure and then.
One comment also.
I think as Youll syndrome is a very high diagnosis rate.
So I think.
Vast majority of patients out there that the disease are identified and in the system and we have noticed that some patients for example chose not to travel to a major metropolitan area.
They prefer to get management from their local pediatric Gi docs, that's a very attractive target for US is we are just not they are still not that many pediatric Gi docs in the country.
So that's kind of one area we've been able.
To expand but I think the take home point is as Chris mentioned, which there is still.
More addressable patients of how to proceed with <unk>. So we do.
For <unk> continued growth.
Okay, and then maybe if I could ask another question on March <unk> study.
At 570 micrograms per kilogram tid versus the live Morley label, which is at 380, QD, but titrated up from 190, just so just wanted to see could you give US a reminder of the clinical experience that you have at this dose to date and any thoughts on sort of this higher.
A higher level of dosing on expectations for how much this could further.
Further impact bile acid reductions on pruritus here and given the love Marley pricing if.
If it is not the same milligram basis, it would seem like a pretty substantial step up would you be able to offer to brands with differential pricing.
Okay.
Thanks, Brent on the pricing front, we're still.
It's too early to talk about our pricing strategy.
Kind of finalize that as we get to the.
Potential label expansion bus pan to speak to some of the clinical questions.
Yes. Thanks, Thanks for the question.
We're really excited about that.
Well first of all very excited about our recent publication yesterday on the Indigo study and at that Dennis as you mentioned, which is which we started with a lower dose.
280 micrograms per kilogram per day than when we double that dose in that phase II study, we saw a greater proportion of patients having a response driving more a bile acids out of the body with higher doses. So we think that by going even higher than that which is doubling that.
All of the dose in the phase III study that we are hopeful that we can even drive greater bile acids out of hepatocytes in the systemic circulation.
Im hoping that translates to greater response rates in the non consenting to population and we're looking forward to see how that translates into all of the other subtypes and the 90 patients that we enrolled in the study.
Great. Thank you for taking my questions.
Thank you.
Thank you and final question is from the line of Steve seat House of Raymond James Steve. Your line is now open please.
Good morning, This is Ryan Deschner on for Steve.
Im just wondering if you guys are still actively seeking collaborators to potentially look at.
This alternative mechanisms like your patch relationship with David.
To broadly address Patrick.
Patrick genetic subtypes.
Thanks, Brian overall, we do have what.
I would say has been active.
Corporate development effort.
The overall objective here is to continue to grow Miriam and really grow in a way that it was back to the roots of how the company started finding underappreciated programs that need the attention to bring them to patients.
If we look at opportunities kind of broadly across pediatric rare disease, and orphan liver that fit with the team here the expertise that we have in house from <unk>.
Development regulatory standpoint.
And then also the commercial team that has broad experience across rare and liver disease.
So it's still it's still active.
Looking broader than the current indications that we have.
Excellent. Thank you very much.
Thank you.
And we have no further questions at this time, so my pleasure to hand back to Chris peaks for any closing remarks.
Great. Thank you operator, and thanks, everyone for joining us.
Thanks for your continued support of <unk>, we look forward to sharing our progress with you next quarter Goodbye.
Okay.
Thank you to everyone who joined US today. This concludes the call and you may now disconnect.
Yeah.