Hello and welcome to the Miran Pharmaceutics Report's first quarter 2025 financial results. My name is Carla and I will be coordinating your call today. During the presentation you can register to ask questions by pressing star followed by one on your telephone keypad. If you change your mind please press star followed by two. I would now like to hand you over to Andrew McCubbin, Senior Vice President of Strategic Finance and Investor Relations to begin. Andrew, please go ahead when you're ready.

Thanks Carla, and good afternoon everyone. I'd like to welcome you to Miriam Pharmaceuticals' first quarter 2025 conference call. I'm joined today by our CEO Chris Peets, our President and Chief Operating Officer Peter Radovich, and Eric Bierkel, our Chief Financial Officer. Joanne Kwan, our Chief Medical Officer, could not be with us today as she's at a medical conference in Europe. Earlier today, Miriam issued a news release announcing the company's results for the first quarter 2025. Copies of this news release and SEC filings can be found in the Investors section of our website. Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements based on management's current expectations, including statements regarding Merrim's programs and market opportunities for its approved medicines and product candidates. These statements represent our judgment as of today and inherently involve risks and uncertainties that may cause the actual results to differ materially from the results discussed. We're under no duty to update these statements. Please refer to the risk factors in our latest Form 10Q and the 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. I'd like to start off by highlighting the tremendous progress Miriam has made in the first quarter of 2025. We continue to deliver across our key strategic objectives, furthering the growth of our commercial medicines and advancing our high-impact pipeline. We are excited to share the details of another record-breaking quarter for Miriam, with total revenues reaching $111.6 million, or 61% growth over the first quarter last year. Further, given how strong the year has started across the commercial business, we are updating our full year revenue guidance to be $435 to $450 million. This increase is driven by robust growth from all three of our commercial netizens and highlights the Mirim team's continued strong commercial execution. In addition, I'm happy to report we've received three important regulatory approvals since the start of the year, adding growth drivers to the business. First, Citexley was FDA-approved for the treatment of CTX in February, and we've begun promotional efforts to reach this underdiagnosed community. Second, Livmarly was approved in Japan for Pfeffek and Allergil syndrome through our partner, Takeda, and most recently, a convenient single-tablet form of Livmarly was approved by the FDA. These milestones highlight our commitment to reach more patients globally. Our pipeline continues to make great progress as well, starting with the VISTA study of Elixabat and PSC. We are getting close to completing enrollment and now expect to achieve this in the third quarter of this year, with top-line data expected in the second quarter of 2026. As a reminder, both the VISTAs and Vantage studies successfully passed a dose selection interim analysis last year and are now in their confirmatory portions of the studies. As an example of what impact IDOT inhibitors can have in PSC, I'd like to highlight a recent presentation earlier this week at DDW of a case series of PSC patients receiving Merilixabat through our Compassionate Use program. Encouragingly, we saw reductions in serum bile acids, and all patients had a two-point or greater reduction in pruritus. PSC remains a condition with no approved therapies, and these results build on our conviction for the potential of Velixabat to bring life-changing results to patients in need. Now, looking ahead this Friday at EASL, we are also excited to present the updated 28-week interim data from the Vantage study in PBC, where Velixabat has been granted breakthrough designation. In this analysis, we show the rapid, deep, and statistically significant improvement of pruritus on Velixabat we shared last June as durable through the full 28-week study. In this updated interim analysis, the looks of that showed a 3.8-point reduction from baseline and a 2.5-point placebo-adjusted reduction in psoriasis. We're excited to advance this program through the confirmatory portion of the study, and we continue to expect enrollment completion next year. And finally, for the balance of the pipeline, we remain on track to initiate our Phase II study for MRM3379 in fragile X syndrome this year, as well as complete enrollment of our Livmarly expand trial in 2026. 2025 is set to be another year of meaningful growth for Miram as we continue to advance our commercial portfolio and pipeline. With strong execution and financial discipline, we are well positioned to continue our leadership in rare disease. And with that, I'll turn it over to Peter to give a brief update on the commercial business. Peter.

Thanks, Chris. I'm pleased with the continued strong growth we are seeing across our three medicines and our commercial team delivered another great quarter with total net product sales of 111.6 million dollars based on the demand we are seeing across our medicines we are raising our full year net product sales guidance to between 435 450 million for the marley total global net product sales grew to 73.2 million dollars in the first quarter an increase of over 70 compared to our first quarter in 2024. U.S. Livmarly sales were $49.5 million, driven by robust new patient demand across indications. For the remainder of the year, we expect to see continued growth in both Algeo syndrome and PFIC, and the approval of the tablet formulation adds to these positive dynamics, with a single tablet per dose providing a distinct convenience advantage. Livmarly is now the only IVAT offering flexible formulations across all ages, an important milestone as we look to expand options for our patients. International Livmarly sales were $23.7 million. We saw strong demand growth in our direct European markets, driven by both continued penetration in algeal syndrome in established markets and new launches in mid-sized countries. I'll note that our international distributor and partner revenue this quarter, including about six million dollars of inventory which is a new dynamic that has not been seen in prior quarters sales numbers overall the continued underlying demand growth across our international territories is the driving trend for this business and we're excited to see the pfick indication come online in many international markets this year in q1 we also saw strong growth from the bile acid products, with $38.4 million of net product sales, representing 47% growth over the same quarter last year. With Cetextly now approved in CTX, our efforts are focused on engaging healthcare professionals across several specialties to find patients in this underdiagnosed condition. And I'm pleased to say we're starting to see progress, as we have seen an increase in new CTX patients since the FDA approval in February. Overall, it's been a tremendous start to the year for the commercial business. With the increased full-year guidance of $435 to $450 million, we look forward to continuing our strong execution throughout the year. And now I'll turn it over to Eric.

Thank you, Peter. Our financial position is strong and continues to improve. The first quarter 2025 net product revenue of $111.6 million compared to net product revenues of $69.2 million the first quarter of last year. Cash, cash equivalents, and investments at March 31st was $298.6 million compared with $292.8 million at the beginning of the year. Total operating expense for the quarter ended March 31st was $126.8 million, which includes R&D expense of $46 million, SG&A expense of $57.7 million, and cost of sales of $23 million. R&D expense for the quarter included $7 million in one-time milestone payments related to the progress of our pipeline expense for the quarter also included non-cash stock-based compensation expense of 15.8 million and intangible amortization and other non-cash items of 6 million the intangible amortization and other non-cash items expense are largely reflected in our cost of goods sold we were operating cash flow positive for the quarter and we expect to be cash flow positive for the full year. The cash contribution margin from our commercial business improved from approximately 47% in the first quarter of last year to approximately 53% for the first quarter this year. In addition, year-over-year, R&D and G&A expense improved as a percent of revenue by over 10 percentage points.

We continue to be well-funded and financially independent providing us the resources required to execute on a business plan now i'll turn the call back over to chris for final comments thanks eric as a quick recap we've had a great start to the year our three commercial medicines are growing ahead of initial expectations and we're raising our full year guidance in the last few months we saw three important regulatory approvals that support the long-term growth potential of our commercial medicines and our growing pipeline is making excellent progress. The VISTA study of Velixabat and PSC will complete enrollment in the coming months, and we are excited to share a great update for Velixabat and PDC this Friday. We're looking forward to starting the Phase II study in Fragile X syndrome later this year with MRM-3379 and completing enrollment of the Livmarly Phase III Expand and Velixabat Vantage studies next year.

Overall, we are in excellent financial position, and I look forward to continued progress in the quarters ahead and with that operator please open the call for questions of course we will now begin the question and answer session if you would like to ask a question please press star followed by one on your telephone keypad if you change your mind please press star followed by two when preparing to ask your question please ensure your device is unmuted locally our first question comes from kevin clark cartner with evercore hey guys Congrats on the great progress.

Thanks for taking the questions. First, for the additional Velixabat PBC data that's in the easel abstract, it looks like the pruritus benefit deepened a bit at 28 weeks, and also I think there were no discontinuations due to diarrhea beyond the one you saw at 16 weeks. Maybe you could just speak to that and anything else from the abstract there.

Yeah, thanks for the question, Gavin. Yeah, I'm really excited with how the data matured in the interim analysis. So as you point out, the response overall deepens over time with the separation curves looking really strong. We'll have a few more data points that will be included in the presentation as well, so I encourage you to stay tuned for an update there. And overall, the profile and benefit that patients are getting, I think that discontinuation statistic you point out, I think it's a great testament to what this can mean for patients. Great.

And then just on the Livmarly tablet formulation, can you just frame the additional IP that could come around with that and also commercially where you see adoption of the tablet formulation?

Yeah, so I can touch on the IP and then ask Peter to speak a little bit about what we're looking forward to on adoption. For IP, I mean, the tablet formulation here did take some work really because of the properties of Merillixabat. So, it did result in some novel IP. We have an allowed patent that we expect to grant soon that covers the formulation that would extend coverage out to 2043.

Yeah, thanks for the question, Gavin, and, you know, we think the tablet will be a pretty attractive option for, you know, really all the Livmarly patients over 25 kilos, or, you know, you can think of older children and adolescents up to adults, I think that, you know, it'd be a pretty attractive option for many of them. So, I look forward to seeing how that unfolds in the back half of this year. Thanks, guys.

And the next question comes from Jessica Farr with J.P. Morgan.

Hey, guys. Good afternoon. Thanks for taking your questions. Curious how you think your interim Phase 2 PBC data compares to the pruritus data for one or exabat.

And also curious what you make of the placebo response observed on paritis in that trial then i have a second question thanks for the question jessica yeah i mean the um again what we've seen really is just the uh abstracts that was posted on the linerex bat program so i don't want to uh you know speak too much to uh to their data uh but what it did does do for uh at least in our view is highlight some of the strengths in the Felixabat program, where not only in change from baseline, but in the placebo-adjusted difference, having really strong outcome there. But a lot of it, we think, driven by the dose of Felixabat. I really believe that we're at a maximally efficacious dose and optimizing activity for the program here. So that's what stands out for us, is that driving that 3.8 reduction from baseline, which then results in the 2.5 placebo-adjusted difference.

Great. And then can you, maybe switching to Fragile X, can you just remind us what needs to happen between now and when you start the Phase II study in the back half?

Yeah, thanks for the follow-up on the MRM3379. So we're busy kind of putting together the IND now. In the background, what we've been working on is some study planning, having a dialogue with FDA on that, and this is an IED with a new division, so that's really what we've been working on to date. And as we get close to the first patient in, we'll provide a more detailed update on what that study design looks like. No change overall from kind of how we thought about from the high-level summary thoughts, but we'll provide further detail later this year.

Thank you.

Thanks for the questions.

Thank you. The next question comes from Michael Osbrod, Morgan Stanley.

This is Selena on to Mike. Thanks for taking our question. Could you give us an update on the Marley access versus competitors and how step-through policies in Algeo and PFIC are evolving?

Yeah, thanks for the question, Selena. Yeah, the access to Livmarly in the U.S. is, you know, very strong. You know, really don't see that being a barrier for us in either indication. Yeah, I mean, as you know, there are some policies that have, you know, Livmarly in a preferential position in the Algeo indication from a step-through perspective, which is you know it's certainly a beneficial uh aspect but i think overall um you know really see the differentiating factors for libmarly being the strong clinical value proposition and the support the mayor provides and the access is uh really not not a barrier thank you thanks for the question thank you and the next question comes from mani with leering partners hey guys you have

ryan on from monty thanks for taking our question um we're just hoping you can talk about your expectations for the vista study in terms of pruritus reduction you know should we look to vantage as a good benchmark for you know absolute reduction from baseline and then just how you guys see that um looks about being positioned within the psc landscape right thanks for the question there first you're giving some background on uh data expectations and then i'll actually ask peter to speak through the competitive positioning kind of on the commercial side for looking forward.

In terms of our expectations, how we think about the Lixabat's activity level, the Vantage interim result really is the most robust data set we have. And really, it's consistent with what we see for IBAT that is really fully dosed in a cholestatic paritis setting. So optimistic that we can be in that range of activity level for the VISTA study. I'd refer back actually to that DDW abstract that I mentioned in my prepared remarks, where with the higher doses of Livmarly in those compassionate use patients, you're driving pretty substantial pyritis reductions. One thing to note is that scale actually is the clinician scratch scale. So it's only a zero to four range. So getting a two-point reduction on that scale is really powerful. So, you know, overall, I feel good about how we've dosed and set up the study design for VISTAs to use Vantage as an example of what we could be seeing. I may turn it over to Peter for market dynamic.

Yeah, and in terms of the market dynamic, Brian, the work we've done in PSC, probably not surprising to you. I mean, obviously, no FDA-approved therapies. I'd characterize it as a very unsatisfied market. It's just not satisfactory therapy from the perspective of prescribers or patients. So, you do have some of the off-label therapies that we see get used in our other indications, pediatric as well as adult. But I'd say the satisfaction with those is quite low. So, you know, really excited for PSC patients with puritis, you know, upon a potential approval here for Volixabed. I think, you know, just really have a strong, you know, highly preferred position in the market without meaningful competition.

And the next question comes from David Lovowitz with Citi.

Thank you very much for taking my question. A couple of questions on the numbers regarding the inventory, which you said, I believe, was the first time this quarter. So is this – should we see this as being steady-state right now, or are you still learning about where ultimately inventory levels will sit? And on the one-timers and whatnot in the operating spend, could you just comment on what we should be able to – what we should see in that regard later in the year?

Yeah, I'll turn it over to maybe Peter to comment on the inventory and have Eric talk through some of the other lineups.

Yeah, I mean, you know, in terms of the inventory and what we saw in Q1, you know, really as we've expanded our international partner markets, you can kind of think of, you know, there's really two kinds of orders that we've seen over the years. And up until now, it's really been orders, you know, the first kind where there's a patient identified, there's a prescriber, there's a payer, and there's an order. oftentimes they're ordering three or six or even nine months of a product but those you know there's a there's consumption happening right after that order that's why those you know our international number has been a little bit lumpy over the years this is a this is a this this is a new one here what we saw in q1 which is as we you know kind of added partners we have you know new partners coming online where they're they're ordering and they're kind of holding it's truly uh stock or in a warehouse and it will pull through to demand sales uh in 2025 uh so you know but it's i think it's important to think about as you you know trend it out going quarter over

quarter that it was you know it was really really q1 event yeah and as for your milestone question yeah we do we do have our business both tied to development and regulatory progress as well as the commercial progress this one happen to be tied to development progress. We don't give specific guidance on what we can expect for the rest of the year, but as we continue to progress our business, we expect and hope there will be additional milestones showing that progress. Thanks for your questions.

The next question comes from Ryan Deschner with Raymond James.

I'm curious what your expectations for the impact of the tablet formulation of Publum Marley on demand and competitive positioning is, as well as what proportion of allegeal patients you think are you to expect to opt for the tablet at a more steady state, and would you expect this to drive meaningful switch demand? Thanks.

Yeah. I mean, I think it'll be a really attractive option for allergial NPVIC patients that are, you know, the label is really for 25 kilos and over, which is, you know, most of the time that's going to be when you're between 8 and 10 years old. So I think that having a single tablet per dose is quite attractive. Most of these patients are on background therapies or so and other therapies. So pill burden is an issue. so being able to offer one tablet is, I think, pretty attractive. You know, probably the average patient is still under eight years old. So, you know, I think probably in that case, you know, I think that liquid will be preferred there. But, you know, a substantial minority of them, I would think, would, you know, would probably see the tablet as a pretty attractive option. And yeah, I think with this could drive, you know, we could see patients that are on the solution, liquid solution, now on the Marley switch to tablet, you know, you could imagine clinical scenarios where you have a teenager or a young adult who's kind of been thinking about an IBAT, but, or about live Marley, and now you, now you have a more convenient option. They want to be able to, you know, go to school, go travel. So, you can imagine a variety of different new patients and switch scenarios.

Thank you very much.

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 comes from Brian Scorney with Baird.

Hi, this is Luke on for Brian. Thanks for taking the question and congrats on the quarter. On to Texley, could you provide a bit of insight on the rate of patient identification since approval as well as your go-forward expectations?

Thanks, Luke, for the question. I'd actually ask Peter to maybe talk about a couple of our efforts on that front.

Yeah, yeah. And like we said in our prepared comments, we're excited. We've seen a nice uptick from the historical rate. As you know, kinadiol has been available for a while, and we've seen a nice uptick since February. I think some of it is just coming from promotion, you know, kind of being out there and having an FDA-approved product for the indication matters, I think, to clinicians and patients. So, you know, we've seen that. We've also, you know, invested in disease awareness because a lot of the challenge here is making the diagnosis. So, kind of work towards raising awareness of presenting symptoms in neurology where often this can present as ataxia, motor coordination issues. Ophthalmology is another area where, you know, you can see patients presenting with bilateral cataracts and trying to raise an index of suspicion there to do genetic testing. And those efforts have kind of kind of borne through GI patients with, you know, chronic diarrhea without other explanation, so kind of focused in some of these different areas, and that's been kind of where we've made progress so far.

Great, thank you. Good question.

And the next question comes from John Walden with Citizens.

Hi, this is Catherine on for John. I just had two quick questions.

What about PFIC and the launch in the u.s as well as x us also comparing to expectations and then also a comment on the sustainability of um the profitability into 2026. thanks catherine for the question i mean first i mean a quick comment on uh pfic launch and i'll pass it over to eric on uh the uh kind of profitability question i mean the pfic launch uh actually has been going i'd describe it as quite a bit better than expectations, but it's still, that means, you know, relatively small numbers because PFIC is a smaller indication overall compared to Allergyle, but we're excited about what we're seeing and, you know, really attribute that to a great team here at Mirum that's out there spreading the word, and then also just really compelling data coming out of the Phase III program where, you know, you see placebo-controlled improvements and growth in some of these things that are really impactful. And then maybe Eric can speak to the profitability question.

Yeah, so to be clear, we were cash flow positive in the first quarter, and we're expecting to be cash flow positive for the full year. Profitability is a different issue because we have a number of non-cash charges, and so we're not expecting to be profitable anytime soon in sort of the gap sense. but cashflow positive is what we're focused on.

Thank you very much. So that was our final question. So I will hand back over to the CEO, Chris Bates, for any final remarks.

Great, well, thank you all for joining us today and hope you all have a great day, bye.

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