Good afternoon, and thanks for joining us at the 4th Annual NASDAQ Bioconnective. My name is Swaim Papala-Ramakant, or RRK, Senior Analyst at the Policy Reinhardt Health Care Team. Thank you for joining us to have a conversation with Peter Radavich, President and CEO, and Andrew McClellan, of Myron Pharmaceuticals, the ticker MIRM, a $6 billion commercial company that's focused on commercializing novel therapies for male debilitating diseases of the liver. Their commercial portfolio is made up of liver marley for cholestatic pruritis and bile acid replacement therapies. However, the pipeline is quite exciting with, you know, best-in-class therapy, Rolavita, Lixabat for primary sclerosis, cholangitis, and primary biliary cholangitis. They also have Xylargist therapy for FOP genetics. And to discuss their commercial portfolio and also a developmental pipeline. I welcome Peter and Andrew to this subject. So, Peter, just to get started off, give us a little bit of a background of your corporate strategy. And also, as we think about how this company has, you know, grown, you know, the general net sales that were five years ago, we were at some $10 million, and, you know, your recent guidance for 2026 is the range of $660 to $680 million, you know, the target for cash flow positive end of next year, sorry, which is a tremendous goal the company has achieved. So if you can give us a little bit of a highlight as well as what the strategy is as the company continues to grow.

Thanks for the question, Arke, and thanks for the invite. It's great to be here. I appreciate you and the organizers, including us. And before jumping into it, I just want to say we will be making forward-looking statements. So, of course, refer you to the risk factors and our disclosures on file with the SEC. Yeah. And so back up on Mira, Mirim is a rare disease company focused on developing and commercializing high impact rare disease medicines for children and adults living with a variety of rare diseases, which you have named in the outset there. And in terms of your question about capital allocation and kind of how we think about that as we go forward, we think about capital allocation at Mirim through the lens of how do we build a high growth sustainable, durable, rare disease company that can serve patients' interests and return value to shareholders. And so that's kind of the way we think about it. You know, really nice line of sight, as you mentioned, to top line growth with another beat and raise, an increase in guidance in Q1. We were actually cashflow positive last year, and we're cashflow positive in Q1, although our guidance is this year, we expect to probably not be cashflow positive, returning to cashflow positivity next year as we work through investments in our phase three pipeline, especially the recent addition of Brilovatug for hepatitis delta virus. So that's the kind of high level lay of the land and how we think through capital.

Fantastic. So, you know, in the next 12 months, you know, I have potentially four registrational top line data, starting from Vistis, as you for the expand and also advantage. Plus, we're going to have Bidufa of September 26th on Zyler and Strip. So, how does management think about all these various value drivers and what is your internal investment being sequenced so that, you know, you can de-lis some of these events.

Yeah, I can take that. I mean, you know, ultimately, we are really excited about the setup. You know, all of these programs have some measure of de-risking associated with them. You know, for HTV, we just announced the Phase 2B top line a couple weeks ago with a very clear effect. PBC, you've seen the interim data, so we feel really good about this. And, you know, from a kind of investment staging perspective, we're moving forward, you know, with all of these in parallel, right? There's very clear synergies on the liver side of the business between build for Belixabat and the build for HDV. And then with ZollerConsertiv, an FOP, that fits right in the bag of our team who's on kind of rare genetic diseases. so that they're currently marketing Goalbaum and Textly. So not a lot of incremental investment there. So from an investment perspective, it's all kind of moving together, which is great. And then finally, rounding out with expand, I mean, that's with Marley's extension. So we're already kind of doing that.

So now your portfolio spans across various categories you can call it, from rare cholestatic liver disease, HDV, Fragilic syndrome, I mean Fragilic neurology, and also FOP genetics.

So, you know, with this broad, you know, how do you, you know, how do you move that, you know, from liver morally and move on the scale uh what scale of revenue do you think you know create formal franchises between these um structures thanks for the question okay yeah i think even today we're already uh moving towards organizing our business in two different units i think as andrew alluded to you look at our business today live marley it's a rare liver disease product most of the uh business is coming from pediatric liver doctors, but growing in the adult side with adult PFIC. And that platform will have Felixabat added on top of it for PSC, PBC, as well as for Lovatov, for Hepatitis Delta to have kind of a fully functional, you know, liver unit that can reach both pediatric and adult liver prescribers. And then the rare genetic side of the business today is comprised of Cetexli and Colbomb. Those are important products for us that are growing nicely. When we announced the in-license of Zalurga Certib for FOP, one of the things we noticed there as we did the diligence is that a very high proportion of the prescribers are at the same exact accounts as our Cetexli and Colbomb team. So you get a lot of synergy there.

It's some different prescribers, maybe some different specialties, some of it's medical genetics, but you also pull an endocrinologist with FOP and bone diseases but it's the same account so we get a lot of leverage that product becomes kind of immediately accretive to us and so that's kind of the way we we think we can serve the products and patients in our portfolio now as we come around those two different things so looking into the future i've been you know let's say three to five years down the line, currently no longer is the bulk of the national district, that all of these can be certainly a big revenue .

So when we look across these, how should we think about what sort of revenue concerns contribution will we get from all these drugs and is there a way for us to kind of rank order these things yeah good question then we all we always get asked which of our children we like the best so it's hard to say because we like them all um but i think in terms if i was trying to you know rank order i mean you might one way to focus on that might be where we have clearest line of sight uh to both the clinical and regulatory profile as well as the commercial potential so with that with that kind of framing i think you'd start with live marley obviously it's been approved by fda for about five years and uh two indications clinical profile label all very well described several quarters of sales trends that you can look at that are available to give you kind of line of sight to what we see is at least a billion in peak sales uh for live marley uh so that's that's kind of obviously the bedrock of the company and you know uh you know quite a bit of visibility there to the profile. I think if you move from there, you know, Belixabat, we just reported this quarter our pivotal PSC data. So, you know, now we have kind of line of sight to, you know, an NDA and what that profile kind of looks like as we disclosed in the top line readout, you know, between PSC and PBC, a lot of ways to see that opportunity for Belixabat as being a billion dollar peak opportunity with the much larger TAM. you're talking about in the adult settings there. And then as you move from there, I think Perlova-Tug is really interesting. Obviously, we don't have pivotal data yet in hand there, but two different phase two data sets, a phase one, two data set that was reported last year at ASLD, and then just this quarter, as I think Andrew alluded to, randomized phase two B, Azure One data. I think in virology, we see pretty good replication between phase two and phase three. So we have pretty high conviction there that that'll be at least a $750 million opportunity, potentially more, depending on how successful we can be at developing the market, which we're pretty optimistic about. And then finally, is Allergy Assertive, really excited about that. I mean, we pegged that as a 200, at least $200 million opportunity. Recognize that it's kind of hard for the investment community to assess that because there's no clinical data in the public domain yet, but hopefully that will come very soon. Suffice to say, we were excited enough to enter into the license agreement based on what we saw. And of course, FDA has accepted that priority review on a period of the date in September. So maybe that tells you a little bit about the profile, but I understand you'll get more visibility, hopefully very soon when that data are released.

So in terms of the clinical updates that, you know, I'm thinking about visitors, you know, that delivered at 1.64% for just a, you know, each row reduction with a 55.6 percent in the moderate to severe primary cohort. Yeah and you're you know looking out for the two-year open expansion data as they've become. You know in thinking through the durability and also you know what sort of depth and consistency of maintenance data, do you think the FDA would require in terms of approval? And what sort of a label are you expecting?

Yeah, the easy part to start is, from a label perspective, would be treatment of Colossac crinus due to PSE, similar to what we've seen for ALSGL and PFIC. In terms of the response and durability, we expect it to be quite durable throughout the extension. If you look at other IVAT settings, it is a very durable response that tends to incrementally improve over time and expect to see that here and are kind of seeing that in the long-term data. So, you know, we're obviously excited to share that and when we can, I think you'll see what we're talking about. But from an FDA perspective, I mean, what they're really looking at is, you know, the stat sig improvement on paritis. I think we've got a very clear data set and very significant effect on that. on Pritis in this setting, which if you look at other approvals, this is very much clearing that bar.

The other major update that you recently provided is that you might face to be interim So, can you discuss a little bit about the data itself and, you know, you're looking at the 300 milligram dose and also looking at once and four weeks dose um how i'm not asking what you favor but what do you think is generally um uh commercially relevant um dose and where do you think the science steps yeah yeah i can address that one uh so yeah

this reminder that phase 2b azura one study has the delayed treatment arm the 300 milligram subcutaneous self-administered and then the 900 milligram monthly but healthcare professional administered and you know the feedback we get universally from physicians from patients level providers pairs is that the self-administered option is preferred people you know want to be able to administer their medicine without the burden of interacting with the health care professional so that would be kind of the preferred uh approach of course the phase two and three program have optionality built into it this is the kind of first of three real looks we'll get at that we'll also when the azura one and azura four study readout second half of this year we'll have another look at it but you know i think what you saw and this readout was you know comparable maybe numerically a little bit better for the 300 if it looks like that in the end we'd probably end up proposing the 300 uh self-administered approach for labeling but we'll have to see how the and then um thinking about and as you have two studies with the same group um you know you know what sort of um implementable efficacy uh our convenience because you said it's a few um you think it's meaningful yeah so yeah these these studies are designed with the european market in mind and you know the feedback we've received uh you know primarily for the reason you mentioned the difference in convenience and maybe even tolerability already you know uh quite different a daily subview that requires 30 to 45 minutes of reconstitution versus a uh you know weekly potential weekly self-administered uh ready to inject uh liquid and file kind of approach that you wouldn't you wouldn't really need to see large differences in that seat for you know the preference to be uh towards towards for lower tuck uh from payer and physician interactions and you know you can certainly look at the published phase two data that we have and we're pretty confident about and speed-outs we think should compare favorably.

And regarding the HDV opportunity, HDV, there's a precondition for it that the patient has to be co-infected with HBV. So how do you envision therapy itself, you know, do they have to be combined with HBV and they have become like are there certain preferred type pieces obviously there are multiple workers have been approved and you know depending on that combination does that change the market size i don't see it impacting the market size too much i mean we expect them to be treated for their their happy uh generally speaking though you know you're seeing some emerging data around functional cures and hepatitis b which is the question that we get a lot That's a very different patient profile than the co-infected patient.

Typically, you're seeing some achievement of functional cure in FB patients with low viral loads. So in the 1 to 3,000 range, the baseline surface antigen levels in a co-infected patient are around 10,000 plus. So very different setting. So, you know, in short, expect these patients to be treated for their hep B, but don't see the functional cures really impacting the market opportunity where they contact the patients.

What's your data, what should we expect to see there, and you know, how should we how should we think about clinically meaningful data from that representation?

Yeah, I mean, we've, so we've disclosed the material update. So you'll have incremental data at EASL, you know, thinking about like responder rates and things like that.

But I think, you know, you know, the headline and that is the clinic and that is a clinically meaningful headline uh this 1.67 zero adjusted response so we think that that sets us up well from a you know sort of positioning this with the fda and risk benefit perspective and then um you know this is um looking at proveritis um especially in the psc population um where there's no food therapy and today um what's the expectation for having an advisory can link in on this and also um you know in terms of uh getting to the commercial launch which

potentially could be you know um it could be uh next year probably from an advisory committee perspective you know this will now we have five i've had indications approved right live marley has two bill bay has two lino boy has one for all five of those reviews we haven't seen an advisory committee yet so that's probably the best thing we could point to in terms of guiding expectations and in terms of timeline for launch the uh nda submission we've got a second half of this year we'll request priority review and of course we know at the time of filing if that's granted or not it would seem to us like psc for the reasons you already said rk would would likely you know meet the criteria for a priority review and if that were to be the case that would point

towards a launch mid next year okay um the advantage is that there's the next exciting study where, you know, the interim data certainly showed 2.4 and 2.6 placebo-adjusted reductions on the each broken scale for the 20 milligrams and the 80 milligrams. How do you position, you know, Olexabad against Lynxabad, which is already a proof, and, you know, what sort of a, you know, what is the advantage that Olexabad has against insulin Ixbat, you know, whether it is clinical or, you know, or on the commercial front. So, I mean.

I mean, definitely the efficacy profile is differentiated. I mean, we're working off of our interim data set. But, you know, when you look at that placebo-adjusted benefit on itch, it's significantly higher than what a linear Ixbat showed. And some of that, you know, we believe comes down to dosing. We are dosing at more effective levels of I-BAT inhibition with the Luxabat. which is where you see deeper and broader responses. Same story is played out with Liv Marley to analogy LMPPIC. So the clinical efficacy perspective, I think is where we differentiate the most.

Okay. So, the recent licensing of services with a $16 million upfront and a $15 million offset at the pool, you know, I'm just trying to understand the logic and the commercial strategy to do that and you know how how should we think about your general business development plan from here on um you know keeping that um as a base case yeah we're we're really excited about that as a profile or phenotype of bd deal you know we look

at it's a late stage program with the fidupe date in september we see a 200 million dollar at least peak sales opportunity and the opportunity to license that for 16 million up front you know for us you know really attractive and not to mention uh immediately accreted program that kind of fits in the bag of our existing infrastructure so we we like that profile uh but you know zooming out broader bd i mean we we look for um you know high impact medicines that can make a big difference on you know patients with high unmet needs of course we're looking at probability of success i think the deals we've done probably give you a sense of the the you know the kinds of things we look at we looked at late stage things like this we you know with we got involved right as the uh phase three studies were kind of getting up and going we've done the fragile x program which is uh what's much smaller up front but a phase two ready program where there's some strong rationale, but yet to establish a proof of concept, so more high-risk, high-reward kind of phenotype. So that probably gives you a sense of what we look at, but ultimately we need to build a durable, high-growth rare disease company.

As we're getting past the time, so as we're really close, Andrew, you're entering a very But what are the major catalysts that should be on the platform?

Well, you know, as Peter kind of mentioned, it's hard to pick your favorite children. But just from a sequence perspective, you know, while the HDB readouts expand and then and then PBC, I think those are those are those are big ones for us. we're excited about all of those for different reasons but that kind of fits into the the growth of our our liver business um but that's kind of foundationally with marley right now but you know expand is expanding that into a broader setting full aesthetic products that we're very excited about i think it's kind of missed sometimes by investors just because it's a little bit harder to do the epi on this kind of long tail of other diseases and causes of cholestasis um and then with the looks of that and uh prolovitug i mean that's expanding from a really pediatrics to adults and frankly it's synergistic with what we're doing with pfick uh so you're really excited about that setup um so it's hard to pick a favorite but it's you know as you said we're going to be busy thank you thanks for your comments okay thanks appreciate it great to be here