Q2 2025 Mirum Pharmaceuticals Inc Earnings Call and Business Update
Hello everyone and welcome to the mirin Pharmaceuticals report. Second quarter 2025 Financial results and provide business update. My name is Carla and I will be coursing your call today during the presentation. You can register to ask questions by pressing star. Followed by 1 on your telephone keypad. If you change your mind, please press star followed by 2. I would now like to hand over to your host, Andre mackean sdp of stretched, strategic finance, and investor relations to begin. Please go ahead. When you're ready.
Thanks Carla and good afternoon, everyone.
I'd like to welcome you to mirror and pharmaceutical second quarter of 2025 conference call. I'm joined today by our CEO. Chris Pete, our president and Chief Operating Officer Peter radovich, our chief medical officer joined Juan and Eric Burrell, our Chief Financial Officer earlier today. M issued a news release announcing the company's results for the second quarter of 2025 copies of this news release in September can be found in the investor section of our website. Before we start, I'd like to remind you that during the course of this conference call, we'll be making certain forward looking statements, based on Management's, current expectations, including statements regarding Mears programs and Market opportunities for us approved medicines and product candidates. These statements represent our judgment and events as of today and inquiry and uncertainties, that may cause actual results to differ materially from the results discussed
We under no duty to update these statements, please refer to the risk factors in our latest form, 10 q and subsequent SEC filings. For more information. With that said, I'd like to turn the call over to Chris Chris.
Thanks Andrew and good afternoon everyone.
2025 is shaping up to be another outstanding year Premier. The second quarter, that underscores the momentum behind both our commercial medicines and our pipeline.
Merit was founded in 2018 with a vision of bringing life-changing medicines to patients with rare disease around the world.
Today we have 3 approved medicines with reimburse patients and over 30 countries, and the pipeline that is rapidly advancing opportunities and still larger settings, highlighted by 3 potentially pivotal studies, reading out over the next 24 months.
Our strategy is rooted in commercial execution, scientific innovation and financial discipline.
And we're proud of the continued progress on all 3 fronts that we will cover today.
On that note on second quarter results, we are excited to share another strong update from mum with total revenues reaching 128 million or 64% growth over the second quarter last year.
With Marley as a key driver of these results, we are continuing to bring substantial benefits to patients and to build a differentiated position with physicians.
At the Top Line suggests, we are reaching more patients than we initially anticipated.
Given the growth we are seeing across our medicines. We are raising our full year guidance for 2025, to be 490 to 510 million positioning us. For another year of close to 50% Topline growth.
Turning to the pipeline. The progress we are making is setting the stage for an exciting 2026 where we have a clear path to 3 Lakes stage milestones.
At this program pass, its interim analysis, last year, and a consistent precedent, date of inhibition across other tossed static settings, gives us confidence in the potential of Elixir that npsc.
Exciting steps. Why I had potentially bring this much-needed treatment to PSC? Patients.
We are also seeing excellent momentum in our Vantage PVC and expand studies are looking forward to starting our Phase 2 study of MRN 3379 and practical X syndrome. Now that we have FDA feedback on the program which Joanne will tell you more about shortly.
I would also like to say thank you to the Mirum team, whose dedication to bringing high-impact medicines to patients has made all this progress possible. I'm proud of how this group has come together to create a high-growth, cash flow-positive rare disease leader with an exciting pipeline. And with that, I'll hand the call over to Peter to walk through the commercial performance in more detail. Peter.
Thanks Chris Q2 was another strong quarter for mam with total net product sales of approximately 128 million driven by continued momentum of the Marley in both the United States and international markets as well as solid performance from our bio asset portfolio.
In the US with Marley demand remains strong with in the allergy syndrome and pit with approximately 57 million in net, product sales for the quarter.
notably we are seeing more people peep patients than
Your onset cold stages.
While pick is often associated with clinical presentation and infants were increasingly seeing P fit patients presenting later in childhood adolescence or even adulthood
An expanding recognition of this variability and highlighting the importance of genetic testing across age groups has been a core focus of our launch strategy.
We're also seeing real Synergy between the approved allergy syndrome and prefix indications for the providers. Increasingly viewing live Marley as a preferred treatment across these settings of pediatric Co stasis.
The combination of these factors is translating into a meaningful uptick in volume growth.
Importantly, our recent us launch of the single tablet per dose, formulation in June as meaningful convenience for patients, though. I'll note that Q2 results reflect the performance of our oral solution.
Internationally. We are seeing durable with Marley growth across both direct and partner markets with 31 million net product sales.
This was driven by expanding reimbursement and growing demand as well as strong performance in our partner markets.
In Q2 our partner ticada secured reimbursement in Japan and launched live Marley in June with promising demand observed. In the initial days of commercialization.
under our license agreement with ticada, we received large periodic orders for live Marley creating quarter-to-quarter variation in international product sales,
We also saw strong performance from our bio asset portfolio was the texi and Cobalt contributing approximately 40 million in Revenue.
These medicines continue to benefit from steady demand and increased engagement following this effectively approval earlier this year.
Given the momentum across our medicines. We are. Raising full year. Revenue guidance to 490 to 510 million driven largely by live, Marley, strong performance, particularly growth in our international business, steady increase in algal demand, and our pit launched in the US.
It's an exciting time for realizing live, Marley's potential.
Looking long term with the current trends in algae, Health syndrome and pick and the label of expansion opportunity in ultra rare coal, stasis, we aim to unlock through the expand studies, We believe We believe that Marley ultimately has the potential to be a 1 billion plus Revenue, Grant
We're excited about continuing to execute to realize that potential to prepare for potential launches ahead of our clinical pipeline.
And for an update on the pipeline, I'll turn it over to Joanne.
Thanks Peter. I'm pleased to share updates on the continued progress of our clinical pipeline where we're seeing strong interest in engagement across all studies.
Starting with Melissa Bat, we're very encouraged by the momentum in our Vista study for patients with kuraitis due to PSC.
The last patients are in screening now keeping us on track to complete enrollment this quarter and on track for expected Topline data.
In the second quarter of 2026.
With regard to the PBC. The Vantage study is proceeding nicely and we expect to complete enrollment next year.
The expand study evaluating with Marley in additional settings of kilos atic Paris is also progressing well and we expect to complete enrollment in 2026.
Finally, I'm excited to share more on mrm, 3379 our brain, penetrant pte 4D inhibitor for fragile X syndrome.
we had the opportunity to discuss the program and endpoints with the FDA in a pre-ind meeting earlier this year and our IND has recently cleared
We are on track to initiate the phase 2 study by the end of the year.
Our study will enroll approximately 52. Male participants aged 16 to 45 with fragile X syndrome.
We will enroll males who are confirmed genetically. What is called Full mutation?
As these patients are known to be most severely affected from a cognitive standpoint, and therefore, have the greatest unmet need for a new therapy.
This is a randomized, double blind Placebo control study, evaluating 3 active doses in order to identify the optimal symptoms.
In additional open label, cohort will include approximately 8. Younger patients.
Uh males aged 13 to less than 16 at the lowest dose to evaluate PK and allow us to move into younger populations in subsequent trials.
The primary endpoint is safety and tolerability, and the key. Secondary efficacy endpoint is a change from Baseline at week 12.
On the NIH. Toolbox crystallized cognition composite a well-recognized cognitive measure also, used by other programs in this space.
That this ultimately will be the primary endpoint phase 3.
We're excited about the pace and engagement of our pipeline look forward to sharing further updates in the coming quarters.
I'll now hand the call over to Eric to discuss our financial results for the quarter.
Eric.
Thanks John and good afternoon everyone. We delivered another solid quarter of financial performance. Highlighted by total, net product revenue of 128 million. Representing a 64%, increase over the prior year and reflecting growth across all our commercial medicine.
Total operating expense for the quarter and the June 30th was 133 million, which includes R&D expense of a 46 million sg&a expense of 63 million and cost of sales of 23 million.
Expenses for the quarter included, non-cash, stock based compensation, expense of 18 million and intangible amortization. And other inbound cash items for 6 million
The intangible amortization and other non-cash items expense are largely reflected in our cost of sales.
We were operating cash flow, positive for the quarter and expect to continue to be cash for positive for the full year.
Our cash.
Offer, we need to improve.
For example, our cash contribution margin from our Commercial Business, exceeded 50% in the second quarter.
Cash, cash equivalents, and investments were worth $322 million at June 30, an increase of $29 million from the end of last year.
We continue to be well-funded and financially independent providing us. The resources required, to expand, our patient impact, and grow our business with that. I'll turn the call back to Chris,
Thanks Eric.
Want to take a moment again to acknowledge the incredible efforts of the mere. The progress we've made so far this year, both commercially and across the pipeline. Reflects our continued commitment to delivering life-changing medicines for patients with rare disease.
We are operating from a position of strength and the opportunities ahead to make this an exciting time for me. Now, we have a clear strategy, the right team in place and a growing impact on the lives of patients and families around the world.
Make that operator, please open the call for questions.
Sure, we will begin. Now with the question and answer session. If you'd like to ask a question please press star, followed by 1 on your telephone keypad. If you change your mind, please press star, followed by 2. When preparing to ask your question, please. Ensure your device is a muted locally.
And our first question comes from Gavin, Clark Gartner with evercore.
Hey guys, congrats on another great quarter. Um, first, I just wanted to ask on live Marley, what are you seeing for overall therapy, persistence rates and has that changed at all over the last couple years and then on the pipeline for the ongoing Vistas PSC trial. Is there anything you're seeing on a blinded basis? Like gives you increased confidence maybe it's, you know, Blended for right is variability tracking, within expectations, uh, Baseline characteristics continuing to come in and expected or anything else you can give us there would be really helpful. Thanks.
Yeah. Thanks for having for the question. What the computer speak to the persistence question and let you and comments a little bit about how this is conduct is going.
Yeah, thank thanks for the question. Gavin, in terms of persistence are information is most stable from the allergy indication where we've got patients about several uh, several years or some of them approaching the decade. Uh, and you know, if you think about persistence, probably 70 to 75% are on after 1 year. So that's the kind of attrition in year 1 and then so
Subsequent years, the attrition is, is much less than that. Uh, so that's by the way, I think about an LG and then pick is just probably a bit too early to comment
Um, hey Gavin. This is Joanne. Thanks for the question. Um, with regards to your questions about Vistas, you know, we're feeling pretty confident. And part of it is that the standard deviation that we powered the study on, uh, was pretty conservative. So, you know, our best, uh, estimate is the standard deviation should come in less than that. So given that we powered the study uh, assuming a treatment difference in 1.75 with a standard deviation 3 probably is closer to 2. So I think that gives us um at a confidence and I can also share with you that the Baseline characteristics in general, reflect the PSD population
so, I think I think these are all points that give us, um, you know, some good confidence to kind of proceeding forward as we're getting the last patients in the screen and
Great. Thank you.
Thank you, question.
5 with JP Morgan.
Hi, this is Abdul on for Jess. Um, congrats on the quarter. Um, can you provide details on the expected Revenue distribution between live Marley and the bio ass business for the remainder of the Year? Thank you.
Yeah, thanks for the question. And we we're not breaking down guidance by specific products, but, you know, 1 thing I would say is, I think some of the trends that we've seen here today, we expect those to kind of, in general, continue, moving forward. Um, so that's kind of the best color I can give on on how it breaks down and the 490 to 510 range
Thank you.
Thanks for the question.
Thank you. The next question comes from. Ryan Dashner with Raymond James.
Thanks and congrats on the quarter um wanted to ask what main drivers, are you attributing to the growth that we're seeing in with with Marley sales? Um and then also how meaningful and impact on script volume are you seeing specifically due to the availability of the tablet format to lift, Marley. Thank you.
Thanks, Ryan, for the question. I'll give a first comment on what Peter added to it. You know, I think one of the several dynamics going on here is that, in general, we've noticed that, really, just as pick has been added in the U.S., building awareness of the availability of genetic testing.
And you have a concept of later onset pick diagnosis which to be honest when we you know got the label expansion and were initially starting out in pick we thought was pretty minimal. Uh what we're finding is that it is it just it's more they're more of them out there than we originally expected. So that's that's 1 of the the the drivers that we've actually deployed against over the past uh, 12 months.
Yeah, I think that's a, that's that, that, that is 1 of the main drivers. Really growing. The p and pick, if you will the the total, uh, you know, market for the class in that setting also happy with the continued, uh, growth in LG syndrome and certainly, um, you know, the international businesses performed. Well, in terms of your question about the tablet, uh, you know, that was that was introduced in, in, in the month of June. So obviously, uh, it's part of our comments there. The outset is not did didn't have an effect at this quarter but certainly encouraged uh had a lot of positive feedback from uh patience and and providers who have uh chosen chosen to go to the the tablets since then.
Thanks for the question around.
Thank you. So the next question comes from Rise coordinate with
Hi. This is Luke on for Brian. Thanks for taking the question. Uh, 2 on live Marley, can you discuss any inventory impacts in the second quarter? And also could you provide a little more insight on the Tada order? Cadence, do you expect it to be more of a seasonal Trend? Or would it be more irregular than that?
Thanks for the question. Yeah, as far as inventory. It's really, uh, only relevant, uh, you know, in so far as Japan and cicada because, no, no inventory in the business in the US or Europe, anywhere else with teada, it is kind of large periodic orders uh, that happen and uh, from time you know, from, you know, expect there'll probably be another 1 this year but we don't have perfect line of sight into it. Uh, there's variable consideration placed on it when the order comes. So that's why you know, sub supporters to estimate can can change, but that's kind of the best color we give to you. We'd expect quarter to quarter variability there.
Specifically in in Q2 okay, 11 million.
Uh, what's the the number of the Q2 number?
Great. Thank you.
Thanks for the question.
Okay.
The next question comes from Mike oath with Morgan Stanley.
Uh, good afternoon. Thanks for taking the question and congratulations on the quarter as well. Maybe, just a question on fragile X. Uh, sounds like you made some nice progress interacting with the FDA on the trial design, just curious. If there's any, anything else, you need feedback on or, or from the FDA or or any next steps. Before you start that study in the fourth quarter, thanks.
Yeah, thanks for the question. Um, so you know, we're actually good to go. We have the clearance on the IND, so we have a study made proceed letter. Um, you know, we've engaged a lot with the patient community and also the physician community to make sure we incorporate the comments into the design of this study. So, we feel pretty good in terms of where we are, and certainly on track to enroll the first patient by the end of the year.
Thanks for the question.
The next question comes from our new start with luring partners.
Hey guys, you have Ryan on formani, congrats on the quarter. Um, just curious how well penetrated do you guys think you are in the allergy Olympic markets, kind of looking at your commentary that live Marley can be a billion dollar product wondering how you see that broken out by allergy and pick and then just 1 on the pipeline. I know, fragile X design is pretty ironed out, but are there any specific elements to your particularly interested in from Shia noises update later this year? Thank you.
Yeah, thanks. Thanks for the question. I can touch on the the last Point first, and then pass over to Peter, to, to talk about, you know, some of the sizing for the, various components for live Marley. Um, in terms of, you know, the uh, the phase 2 precedent data from the the Shinobi program and the upcoming phase 3. Um, if a couple of thoughts that we have on a, you know, first to phase 2 is great proof of Concepts out there and kind of what got us excited about 3379 and the ability to have uh, kind of potentially wider therapeutic index, get more of the the drug uh, into the CMS
Um, and in terms of, you know, kind of what we are looking for out of the phase 3, um, if that differentiated profile that we have, I think kind of makes us, you know, really interested to run our proof of Concepts, kind of, regardless of the outcome. Um, expect we're not going to learn a lot from any Topline release, but, um, you know, we'll kind of be looking closely and, uh, see if there's anything to incorporate into the future studies in the program.
Yeah, and in terms of your question, right? About, uh, penetration and opportunity. Uh, if you think about think about the US, you know, analogy L syndrome, we think we're approaching 50%, uh, penetration. But, you know, still every quarter including Q2, uh, you know, adding adding patients. And, and, you know, both infants, as well, as kind of older older patients, who are more prevalent in, in that, uh, prevalent addressable pool. So, you know, still see the the potential to keep going out as you go further and further, you know, exceptional quarters as as we have and, and, and grow that that business pick is, is, you know, been really interesting and has spent Chris mentioned. His comments were growing the pie right now, as we speak, I think traditionally the field, and, and others in the area thought about, you know, pick is really an infant onset disease. And if, if it's not that scenario, then it's not pick. And I think what we've shown through our education and genetic testing, uh, efforts is that there's a lot more there than was previously thought. So, uh, we have kind of less visibility to kind of how big that could be. I think, you know, his
Historically, we thought about pick as being about a third of algal, quite frankly, that's probably probably an underestimate at this point. Um so excited to to find that as we move forward.
And and add on, you know, the 1 other component of when you think about the total of Mount Marley potential, the billion plus number that we're looking at it. Expands study also is a big contributor there where they expand patient population is, you know, really at least the size of pic and, you know, that kind of the conservative view of it. Um, and all of these Dynamics kind of continuing to build over time, the patients on therapy and ultimately the size of the brand.
Awesome. Appreciate the caller.
Yep, thanks for your question.
The next question comes from Josh Schumer with Country Fish Gerald.
Thanks very much. Um 1 from me on the expand study. I was wondering if you could share if um any of those eligible patients are already on this Marley. Um through compassionate, Esau um other exceptions.
Yeah, thanks for the question. What? Joanne speak a little bit to the background of, you know, how we thought, you know, Came Upon designing the uh expanse studies. That really kind of speaks to that that question.
Yeah, um, thanks for the question. Um, so you know, really the, the expense that he came around, because we had a lot of requests for compassionate use in these patients with policy, um, you know, from from these other settings. And so, you know, we're taking patients, who have not previously been treated in order to assess their treatment response in this setting. Um, so you know, so far we're encouraged by the engagement, we've heard from sites and also uh, you know, just the interest from patient populations as well.
The next question comes from David Lebovitz of B City.
For the Marley on the SBC, dramatic shift.
How much of it comes from Potential from the new indications, uh, versus, uh, pick a and algs.
no potential indication, I think
Yeah, thanks for the question. The the 500 million I think you're referencing is we used to kind of give a size for the indication of allergy in the US just kind of a market sizing
Um, so this is kind of the first uh first time with these indications that we um put out guidance on where we think with Marley can ultimately get to so slightly different lens than kind of in the indication sizing.
Um, and really a lot of the confidence in doing that is just how much we're seeing come together across all these different settings. So the three indicators, uh, you know, the pick and expand in the U.S.
Uh, quite sizable, what? We're seeing on the international side, also kind of running ahead of where our expectations were. So uh a lot of this is kind of a change in what we've seen so far this year, in terms of uptake and and things that we're doing directly, as well as Distributors and partners. And the success that they're seeing
Got it. Thanks for taking my question.
Yeah, thanks for the question.
So, just as a reminder, that if you'd like to ask a question, is there 1 on your telephone keypad? The next question comes from Jonathan Boulevard, with citizens,
Hey, thanks for taking the question, 2 for me. Um, 1 on PSC, you guys estimate that there's, you know, about 65% of the population with active paridis and wondering if there's any evidence that that's due specifically, due to excess bile acids, or if there could be any other drivers of pus in that group. And then, um, second 1, just wondering on CTX, if you guys have seen any inflection, now that you can promote, and, um, what you expect for that long term, thanks.
Yeah, thanks for the question. I'll pass this over to, uh, Peter and Joanne to, to speak to the the 2 components and Peter lead off with the CTS. Yeah, we're, you know, we're certainly, uh, you know, sided about the FDA approval. Now, uh, First full quarter, uh, since that here. Um, you know, a lot of our efforts are on patient finding, uh, across different Specialties where, you know, patients present, not not expecting, you know, I think it's a gradual study build, uh, and CTX patient finding is, uh, laborious but excited about the potential there, uh, to to grow as long as we can.
Yeah, um yeah. Thanks for the question. Um, regards to PSC. Uh, you know, we do think that, uh, Val assets do have a role here. However, the path of physiology is a bit different than some of the other diseases, like algae on the pick that we studied in the past. You know, in reality, what we're treating is called stasis. Uh, and and therefore, you know, there's an interest padic component to it that we don't measure. Uh, what we think is that the what we're measuring in serum, bile acid is really kind of spill over. Um, and so we think that the serum by acid level is probably less important when we're thinking about disease like PSC. Um, probably more important when you're thinking of, you know, allergy and PC.
Um, so I think you know, uh, slightly different disease pathophysiology but still kind of being an important central feature here is co-aces.
Interesting. Okay. All right. Thank you.
Thanks for the question.
So the next question comes from sakula with HC way, right?
Thank you, and thanks for taking my questions. I have, I have 2 of them, uh, the first 1 being on, um,
Live Marley. Um, so you know, you made some remarks regarding how, um, tuxedo is managing the the Japanese part of the the collaboration. Um, any remarks on on, um, how how you're going about in Europe. Uh, and uh, also within Europe, what sort of efforts are you making to increase your penetration? So that's question. Number 1 on the question number 2 is on, um, on on the based on the expense study, what sort of um additional population, a patient population? Can you bring to live Marley? Thanks.
See if indication and adding that in its right now kind of coming through, it's uh, the reimbursement steps for adding that indication. Um, so that's what's kind of on the horizon in Europe and maybe speak to expand. Yeah. And in terms of expanding, um, you know, a lot of lot of different ultra rare, uh, patient populations individually, when you look at the under underlying kind of ideology of cause of the cosine crisis, that that could that could pull in certainly failure. Patients with, uh, cosine status will be a, a portion of that. But there are also several others and, you know, we hear about these as as Joey mentioned earlier, through compassionate access requests. And and, and from from sites who have these patients in their clinic and they don't they don't have anything to to offer them. So excited about um, you know, the potential to to study them and potentially, uh, have an option for them.
Thank you, thanks for taking my questions.
Thank you.
And that was the final question for that. This concludes the Q&A portion of today's call. So I will hand back over to Chris Pitt for any final comments.
Great. Thanks again for joining us today and for your continued support. We look forward to updating you in 10 quarters ahead. Have a great afternoon.
Be concludes today's call. Thank you everyone for joining. You may not disconnect have a great day.