Q2 2022 Mirum Pharmaceuticals Inc Earnings Call
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Good afternoon, My name is Chris and I'll be your conference operator today at this time I'd like to welcome everyone to the mirror in Q2 2022 business update.
All lines have been placed on mute to prevent any background noise.
After the Speakers' remarks, there'll be a question and answer session.
I'd like to ask a question. During this time simply press Star then the number one on your telephone keypad.
To withdraw your question. Please press star one again.
Thank you Ian Clements Chief Financial Officer, you May begin.
Thanks, Chris and good afternoon, everyone.
I'd like to welcome each of many pharmaceuticals second quarter 2022 conference calls I'm joined today by our President and CEO , Chris Tietz, Chief operating officer, Peter out of it.
The Palm Bay.
What are you. This afternoon <unk> issued a news release announcing the company's results for the second quarter 2022.
This news release and SEC filings can be found in the <unk>.
This section of our website.
Before we begin we'd like to remind you that during the course of this conference call, we will be making certain forward looking statements about that.
On that program.
Based on management's current expectations and.
Including statements regarding their business plans development programs strategies prospects market opportunities and financial forecasts and guidance.
My name 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.
You should read the risk factors set forth in their 10-K for the year ended December 31st 2021, and any subsequent reports filed with the SEC.
With that said I'd like to turn the call its Chris.
Thank you Ian and good afternoon to everyone.
It's been another great quarter from here in the second quarter, we achieved $17 5 million.
Barley that says which reflects our team's commitment and expertise.
And the life changing impact of what barley for eligible patients with post doctorate.
This quarter's progress furthers our goal of an accounting a leader in rare disease.
We are proud of our launch success and look forward to continued growth in the United States, while it becomes standard of care in this setting.
The success and impact for patients with ALS Youll centre in the U S are why we are so excited about what's to come across all of our programs.
First U S launch shows what is possible for the upcoming potential European and broader international approvals of love Marley and Al Jolson.
This disease has severe burden around the globe and we look forward to realizing the promise of love Marley for patients more broadly outside the U S. Starting with the anticipated fourth quarter European approval.
Second.
Clinical impact we've seen in ours, you'll syndrome, and our pivotal data as well as recent presentations showing improvement and long term outcomes provides evidence of the potential full of Marley and horlicks about to improve lives across a number of calls that acceptance.
We are progressing our five late stage <unk> clinical programs, which will generate multiple data readouts over the next 18 months.
First of which will be data from our landmark <unk> phase III study and important label expansion opportunity filled with Marley later this year.
We have a lot to look forward to as we head into a busy end to 2022, the progress of Marlin commercialization and in our promising pipeline is made possible by both the unwavering dedication of our team and our collective mission to bring important new treatments to patients around the world.
With that I'll turn it over to Peter who will provide a commercial update.
Thanks, Chris we are delighted with ongoing launches as we get the first and only FDA approved medication for this disease.
Hands of more patients.
Today, I'll share further color or outlook or at least $17 5 million net product sales.
And what we're seeing in the commercial business.
As a reminder, there is no inventory our reported product sales, which are therefore very accurate representation patient demand.
Now taking a closer look second quarter revenue was driven by a steady addition of new allergy all syndrome patients as well as consistent we felt with limited treatment discontinuation with.
Which continue to mirror, what we saw in the clinical trials.
On the payer side, we have achieved our goal of over 90% of lift Marley dispenses being reimbursed, which has been a real strong point of the launch.
Yes.
We are thrilled about the rapid acceptance by.
By physicians payers and patients in the first three quarters on the market.
But it's clear we're still in the early days of launch with significant room to grow in the United States.
Our market research suggests that physicians intend to substantially increase utilization of lip margins beyond the group that has received a prescription.
So looking forward, we expect continued quarter over quarter growth in the United States.
Now turning to our plans for Lyft <unk> International launches, we're excited about the potential European approval of <unk>, barley, and the fourth quarter.
The Mira team in Western Europe has been active this year and early engagement with physicians payers and other key stakeholders.
As is typical with launching a rare disease medicine in Europe , we expect reimbursement to progressive country by country over the year or two following approval.
We expect to launch first in Germany. Shortly after EMEA approval, followed by other major markets in Western Europe .
Beyond Western Europe , our eighth commercialization partners are also making great progress towards introducing live Barra Leach new allergy all patient populations worldwide with potential approvals starting as early as this year in these geographies.
Yes.
In total outside the United States. We currently have over 100 allergy all syndrome patients on the lip Marley clinical program that we expect to roll over to commercial therapy upon approval in local commercial availability.
And on top of this and the <unk> clinical program. We currently have nearly 100 patients ongoing worldwide, who also will be eligible to roll over to commercial therapy upon regulatory approvals and local commercial availability.
And now I'll hand, the call over to Pam to provide an update on our pipeline.
Thanks Peter.
In the second quarter of 2022, our team has continued to build the foundation for our important upcoming milestones for 2022 and 2023.
Sessions, as well to deliver life changing medicines to rare disease patients.
First off we expect top line data from our March P 58, three clinical trial in the fourth quarter of this year.
This landmark study builds on the silos and the Indigo phase II study of <unk> and.
And whats dramatic clinical response metric breakthrough designation for <unk>.
Patients with sustained serum bile acid responsive indigo also demonstrated five year transplant free survival with a smile a remarkable finding in this disease.
Also needs respond as we saw significant improvement in pruritus growth and liver function.
And all of our learnings from the long term indigo experienced have been applied to our March pizza Phase III study.
Now as a reminder, this is the largest randomized clinical study conducted to date with more than 90 patients randomized.
The study includes all pizza subtypes at higher doses than was previously tested in our phase II clinical study and we look forward to sharing topline results next quarter.
We also have four additional late stage studies that we expect to read out in 2023.
First the phase <unk> study of Alex about in primary sclerosing cholangitis.
This is Alex about to be a major advancement in the treatment of this progressive disease with no approved therapies.
We now expect to conduct the interim analysis midyear 2023.
This revised timeline is largely driven by the current environment for running clinical studies with impacts on site openings outside the U S and the related supply chain.
We're optimistic about the direction of the study and we've recently had several international site opening and patient screening.
Steady conduct as otherwise gone well with some patients now on the open label extension portion of the study and we continue to get positive feedback from sites.
Okay.
Regarding the Ohio study in patients with intermediate Cholestasis pregnancy, We recently launched a streamlined protocol amendment.
Enrolling patients for the open label Phase of the study and are planning for an interim readout in the first half of next year.
Finally in the second half of next year, we remain on track for data from both the elixir that phase two the vantage study in adults with primary biliary cholangitis as well as primary data from our <unk> phase <unk> embark study in biliary atresia.
And across our pipeline and publications, we remain dedicated to driving academic and collaborative research in rare disease.
We are proud of the significant data presentations, we delivered this quarter above S began and equal on all of these noteworthy presentations on long term outcomes and responsibly, Mali and <unk> syndrome can be found in the publications and presentations section of our corporate website.
And on that note I will turn the call over to Ian Ian.
Pam.
Earlier today, we issued a press release that included financial results for the quarter, which I will briefly summarize.
Additionally, full details can be found in the Form 10-Q also filed today.
As highlighted by Chris and Pizza, we recognized $17 $5 million of net product sales in the second quarter, a 61% sequential increase over the first quarter of 2022.
We expect continued growth of <unk> revenues over the coming quarters.
I'll turn to operating expenses for the quarter were $48 9 million, which includes research and development expenses of $25 $4 million SG&A expenses of $21 million and cost of sales of $2 5 million.
I should also note that.
These numbers include expenses related to noncash stock based compensation of $6 $6 million.
For the quarter ended June 32022, net loss was $26 9 million.
Or <unk> 84 per share.
<unk> remains well funded and at the close of the second quarter ended June 32020, we had cash cash equivalents and investments of $225 million.
To further strengthen our position, we recently reduced our royalty and milestone obligations for live Molly <unk> with the acquisition is detergent, which was a previously a licensing partner and is now a wholly owned subsidiary of mirrors.
In summary, with a growing top line contribution and efficient business model, we have a strong position to achieve critical milestones and expand our global commercial presence over the coming years.
Now I'll turn the call back over to Chris for any final comments, Chris. Thanks.
Thanks, Steve.
Thanks, everyone for joining today.
To close Miram continues to execute on all fronts in 2022, as we deliver on our commitment to make meaningful change for rare diseases with Marley and our development pipeline.
The $17 5 million in net product sales, we achieved this quarter brings us to $28 $4 million year to date.
And we intend to build on this trajectory in the months and years ahead through continued growth of laboratory in the U S potential additional approvals and launches in Europe and beyond.
And through pipeline opportunities as we look to announce topline data from our March <unk> Phase III study later this year. The first of several key datasets over the coming 18 months.
And with that operator, please open the line for questions.
Thank you as a reminder, if you'd like to ask a question. Please press Star then one on your telephone keypad.
Our first question is from Jessica Fye with Jpmorgan. Your line is open.
Hey, guys. Thanks for taking my question nice results this quarter.
Where do you expect to patient adds on with Marley to come going to come from going forward is it more from broadening to new physicians or from existing prescribers going deeper within their patients maybe into more moderate patients that they initially started with marley and less severe.
And then thinking about the Europe launch how should we think about the average price you might achieve in Europe relative to the U S.
And lastly, when the phase III March pubic data reads out what in particular are you going to be looking for in that dataset in comparison to all brands built a data and feedback.
Thanks for the question, Jeff I'll turn it over to Peter to touch on those two commercial topics and then we'll circle back on the different data. Thanks for the question Jeff.
Where we expect to see growth in the United States with regards to new patient adds I think primarily it's the latter phenomenon. You described a lot of our target physicians have prescribed initially as you note oftentimes to maybe the more severely affected patients have gained comfort with the Marley and we see them kind of growing their prescriptions over.
Time and to their patient populations, there maybe to a lesser extent some newer accounts to come on line as well, but really predominantly the former phenomenon.
In terms of European pricing is probably a little bit early to get into specifics as we look towards approval in Q4.
Fundamentally feel really confident in <unk> value proposition and the data that underpins the submission and the Marley can achieve a strong price and the European countries kind of on par with other rare disease medicines.
And circling back to the piece of question.
Touch the opening Tom in the past the Pam to talk through some of the <unk>.
Analysis points, but overall, we're looking to.
<unk> or b, the really tremendous effects that we saw in indigo phase two study that led to a breakthrough designation already with FDA for piece of type two.
And while that's built into the analysis plan.
And I just would add to that I think we're just really excited about this study because as mentioned it's the largest.
PCI population that's ever been studied with over 90 patients enrolled and I think that's a pretty significant number when you look at the rarity of the disease.
In this study.
The primary endpoint is in the primary cohort of <unk> and so what we're looking at as a primary end point is on pruritus key.
Key secondary endpoints include some bile acid and then there is a step down approach using hierarchical methodology in which we'll then look at.
All of the patients, which includes <unk> as well as the other subtypes. So the totality of the <unk> population and then again looking at the primary endpoint key secondary endpoints. So a lot of data to come and really looking forward to share that with you next quarter.
Yeah.
Our next question is from many.
With SBB your line is open.
Hi, Good afternoon. This is learning the songs on behalf of money I just had a follow up question on the piece of data.
Yes.
Would you be able to provide a little more granularity on timing of presentation should we expect that to be.
I would say that at an upcoming medical meeting.
Currently in terms of the data that we may see where would you anticipate differentiation from competitive assets.
Thanks for the question Lily on timing.
Nothing more specific in Q4.
We will plan to share topline findings when we conduct the unwinding and look to get the details presented at a conference.
The next one that would make sense.
And I think too early to say, which one that will be.
In terms of differentiation I think the key points that we think about it is we designed the study was from the findings on dose ranging across all the prior clinical work with Lyft Marley suggest that at these higher doses that we're using in the March <unk> Phase III study you can expect more bile acid clearer.
So that should then translate into better clinical response, so we'll be looking for response rates on pruritus and serum bile acids.
To see how those compare competitively.
Yes.
The next question is from Yasmin Rahimi with Piper Sandler Your line is open.
Hi, guys. Thank you so much for taking my question. This is Lauren on for yes, just two questions. One could you shed some light onto why timelines for <unk> moved from Q2 two to 2023.
And then the second question about the <unk> study as well.
What do you expect will be an outstanding dataset and then how soon could you get that data package to the label and then have you started to warehouse these patients ready to put on with Marley. Thank you so much.
Great Yeah. Thanks for the question I'll start with the <unk> follow up and then let Pam speak to some of the other.
Another pipeline questions.
And in terms of the data timing when we unwind in Q4.
We will look to turnaround and submit that for label expansion relatively quickly so we'd expect to get it in.
Early next year in the first half of next year.
And worked through reviews.
Subsequent approvals for pubic based on that data.
And in terms of patients available as Peter mentioned.
We already have.
Nearly 100 cubic patients currently on drug.
It is around the globe with.
With an expanded access program also open four geographies.
Around the globe as well that will eventually be eligible for commercial rollover once we get through those approvals and reimbursements.
So yes, we do have a very.
Active clinical program in <unk>.
We will be eligible for rollover down the road.
I can't speak to the board.
Yes. So thanks for the question so for the <unk> study, which is the PSC study.
We're enrolling and what we're seeing is that we have patients on the long term extension.
The label Phase of the study and so we remain really positive that that that's a great sign patients are remaining on track.
With regard to the timelines I think that there is pretty typical of what <unk> been seen across the industry frankly with some slower startup some staffing shortages.
Hangover from Covid and sell existing as well as.
Supply chain issues.
All of that with lab kits and things like that so all of that is I think been mitigated most of these issues and sites are now up and running we've got new centers that are now being.
Opening outside of the U S and patients are in screening. So we're feeling pretty good about where we are today and looking forward to that data readout next year.
Thank you so much.
Thank you.
The next question is from Ed Arce with H C. Wainwright Your line is open.
Great. Thanks for taking my questions.
Congrats on another strong quarter.
Two questions for me firstly on the $17 5 million in <unk> sales.
Just wanted to dig in a little deeper if you could.
Describe what.
Really are some of the key drivers to that rapid growth.
In your view is really working.
So far during the launch and then secondly.
How many patients are on commercial drug as opposed to.
Other pre commercial programs.
And then of that what is the split between those sort of naive.
Those that have come onto commercial drug as a rollover from from trials. Thank you so much.
Great. Thanks, Ed for the question I'll take it.
A little bit on.
That last part of the question I'll, let Peter speak to a little bit of what we're seeing.
Being in the field, what's being effective dynamic on an uptake in the first point to make.
Excellent current patient numbers on commercial drug given that there is no inventory in our model. It is pretty clear so that $17 five with you.
By the by what the approximate net prices per patient per quarter, you can see that roughly translates to about 200 patients on drug.
Last quarter, when we give you that direction. So it's pretty simple the track from the net sales, which is really what we're using to gauge our performance.
In terms of the U S rollover.
It really is very much a minority of the patients on drug the vast majority of patients in the U S were prescribed.
After the approval.
And.
One thing Thats kind of gives us a lot of optimism about ex U S growth is that.
100 patients that we mentioned for the eligible syndrome expanded access and clinical program.
Is much larger than what we had in the U S comparatively.
But with that said I'll, let Peter talk a little bit about.
What we're seeing out there in the field.
Yes, thanks for asking what's really been.
Steady addition of new patients over time since approval.
Really high compliance persistence rates, which is consistent with what we expected from our clinical trial.
A product like <unk>, which has a rapid symptomatic benefit the patients can feel the.
The product very quickly after they start taking it and likewise, if you forget to take it for a few days youll notice that as well. So we see very high compliance persistence and very low discontinuation rate. So those those phenomenon together with steady new patient adds is really what's driving the growth maybe the final point I'd add as we mentioned that over 90%.
Our dispenses, where reimbursed this last quarter.
So really happy with that I've seen the payers come online.
Now with the vast majority of our expenses are paid.
Okay.
Great. Thanks Thats helpful.
Thanks for the question.
Question is from Brian <unk> with Baird. Your line is open.
Yes.
Hey, guys Charlie on for Brian today, Thanks for taking the question congrats on the quarter.
Just wanted to get a little more color on how youre thinking about the magnitude of commercial potential in <unk> compared to ally Gilson drome, especially given kind of what we've seen in this space, it's been lagging behind in terms of sales.
Compared to how <unk> been doing in lgs. So I'd just be curious how youre thinking about those especially given we've heard from our kols that they should be similar.
Prevalence and incidence rates, so just be great to get your <unk>.
Your thoughts on that.
Okay. Thanks, Charlie for your question, that's actually a good one to dig into a couple of dynamics there.
While overall from what we see this as both literature and what we're seeing in conversations with some of the prescribers.
Pediatric hematologist.
I apologize that seeing these patients.
Is that <unk> syndrome.
Much more common.
And we we kind of see it as likely in the three to four times the size of feedback overall.
For the 2000 2500 kids in the U S that we talk about for hours Youll syndrome.
One of the.
Interesting dynamics that makes some of the literature and epidemiology, a little bit harder to three precise for pizza.
Is that it is a recessive disorder.
So it does vary by geography, much more than our GL syndrome does.
So kind of the dynamic you mentioned, there where you may have one farewell.
<unk>, a similar number of <unk> patients as our GL patients that often is very much city or state dependence.
There are several centers, where the opposite is what you find when you had these conversations for eligible sentiments very common and they may not see a cancer patient hardly at all.
It does vary by geography mix at <unk>.
There is a little bit harder to to nail down.
Great. Thanks.
Thanks for the question.
Again, Thats star one if you'd like to ask a question. The next question is from Steve <unk> with Raymond James Your line is open.
Hi, This is Ryan Deschner on for Steve.
I was just wondering if you think it's possible to demonstrate.
A reduction in preterm birth, given not only the clinical outcome importance, but also the formal economic.
Importance.
In the ICT programs.
Thanks, Brian for the question I'll, let Tim jump in on this one yeah. So.
It's a great question. Thanks for the question. So I think when you look at the literature on elevated serum bile acids. I think what you are alluding to is that any increase in serum bile acids is an increased risk to the fetus.
And so obviously, what we are also looking for as the in one of our secondary endpoints in the potentially registrational portion of the Ohio. ICP study is that we are looking at those outcomes as well and so therefore any reduction that you would see in serum bile acid.
Hopefully translate to those on <unk> pedal outcomes. So credit's looking is obviously the primary that's the most.
Near term outcome FDA likes that endpoint looking at serum bile acids, and then also looking at other events such as in towards digital outcomes.
But we will hopefully be the that'll be in that part two portion of the study.
Got it thank you very much.
Thanks, Brian .
We have no further questions at this time I'll turn it over to Chris <unk> for any closing remarks.
Great interest and thank you for everyone for joining us today have a great evening.
But.
Ladies and gentlemen, this concludes today's conference call. Thank you for participating you may now disconnect.
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