Mirum Pharmaceuticals, Inc. (MIRM) May 20, 2026 Conference Transcript

Good afternoon, everyone. I'm Lisa Walter, biotech analyst here at RBC Capital Markets. Thanks for joining us at RBC's 2026 Global Healthcare Conference. This session, we have Miram Pharmaceuticals, and we have the pleasure of hosting Peter Radovich, President and COO, as well as Andrew McKibben, Senior VP, Strategic Finance and Investor Relations. Peter, Andrew, thanks so much for joining us today. How are you both doing?

Doing great, Lisa. Thanks for having us. Great to be here.

Well, it's a pleasure to have you here today. Maybe just to kick things off, can you give us a big-picture overview of where Miriam stands today? There's been two clinical data readouts recently. You have an impressive portfolio, three approved assets in rare liver disease, and multiple pipeline assets as well that are brewing. So can you walk us through the latest with Miriam?

Yeah, happy to do it. First, just wanted to remind the audience we'll be making forward-looking statements here today. And, of course, I would refer you to our SEC filings for full disclosures and risk factors. But, yeah, Miriam's in a great position today. You know, we're a global, about 430-person strong rare disease company. We got our start with Libmarly, which is a really high-impact medicine for primarily pediatric but now a growing adult population of genetic cholestatic liver diseases. And that product has performed very well. As you noted, we now have three approved medicines. The other two came in through BD transactions and have built a really strong, sustainable, high-growth rare disease business. And 2026 is shaping up to be a year where a lot of catalysts are coming together. Just this quarter, we had two really big readouts for the company. the VISTAs study and primary explosin cholangitis pivotal data set read out positive heading towards a pre-NDA meeting and an NDA submission second half of this year really excited about that also had a phase 2b readout from our hepatitis Delta program which was recently came into the company through acquisition of Blue Jay Therapeutics and that data set you know read out you know very much uh in line with our expectations and earlier uh data sets so really excited about that looking forward to the full phase three readouts there from the azure one and four studies uh later this year and then finally we just announced a exciting bd transaction as part of our q1 uh earnings result uh we're happy to get into more detail on that as we go through but another high impact ultra rare disease medicine that we're excited to to put into our infrastructure and bring to patients.

Well, I think that's a great segue to my first question. What is Miriam's approach to BD? You're clearly not shy about doing it.

Yeah, yeah.

What is the runway or the firepower that you have for BD after your recent raise as well?

Yeah, you know, the raise, you know, is really kind of about, you know, addressing existing convertible notes for the 2029 maturity that were on our books and refinancing most of the principal there and taking advantage of a, you know, really issuer-favorable market, getting a 0% coupon instead of a 4% for most of the debt. So really happy about that. But it doesn't really change our fundamental approach to BD. You know, we're very active in BD. We have an active search effort. You know, I think our last several deals that we've done and kind of give you an idea of the phenotypes of deals we like. We've done commercial-stage deals, very late clinical-stage deals like this Zalurga Sertib that's really in an NDA review now. We've done phase three ready deals and phase two ready deals and really looking at assets that address a high medical need where we have a level of conviction on the biology and the clinical data that's there and that we think we feel that we can add value to, that we're the right team to own this asset. And critically, we take a very disciplined approach to PD. We're not shy, but we're also highly disciplined. So we want to make sure that we have assets where we can create value for patients, but also for our shareholders.

Got it. That's really helpful to get your view on that. And I do want to talk about something that affects everybody in biotech and the pharma industry is the recent FDA volatility that we're experiencing. with Commissioner Marty McCary stepping down. Are you worried about any impact to any of your late-stage programs? You just mentioned a pre-NDA meeting coming up, filings later this year. You also have a PDUFA date coming up around the corner in September with your newly acquired asset as well. So are there any worries about this or how are you thinking about it?

That's a great question, Lisa. For me, it's really been business as usual in terms of the review teams we're interacting with. We interact across a variety of divisions. A lot of our programs have been in liver, but also rare disease, and now this new program in endocrinology. The review teams and reviewers we're working with on all of our kind of NDA and pre-NDA interactions are largely the same teams we've been interacting with for many years, the programs we've been leading for many years. So for us, we haven't seen an impact.

Got it, got it. Well, maybe let's dive down into some of your commercial programs. Let's start with Lyft Marley. You already alluded to it. Launch has been going well. Q1, 2026, you had $114 million in net sales. You had 55% growth year over year. And you also raised the full-year guidance to $660 to $680 million. So I guess, can you take us through what's driving this momentum with Lyft Marley? Is it volume, pricing, international expansion, or a combination of all of those?

Yeah, thanks for the question, Lisa. The Q1, we were really happy with the performance. And as you noted, beat and raise. The big driver is demand. And the biggest driver of higher-than-expected demand is the PFIC dynamic, the second indication for Libmarly. It kind of continues to outperform our expectations. we're finding many patients who are later onset PFIC or often adolescents or even adults. It's a kind of newer clinical entity that wasn't something that was considered by the field even a couple of years ago. So that's kind of a market development effort or a new diagnosis effort that's kind of working out really well. Also, our international sales for Lubmarly did better in Q1 than we thought. Usually, we expect kind of a decline, you know, a meaningful decline in Q1 because there's a little bit of a phenomenon in our international Live Marley business where in Q4, we see some distributor name patient sales markets kind of buy in bulk for individual name patients. And then you're kind of working through that demand in Q1, but the underlying demand was strong, so that was kind of better than expected.

Got it. And maybe more importantly, what penetration levels are we at in the ALGS and PFIC indications? And are we getting close to peak penetration, or is there still room to grow?

Yeah, you know, I take those, each indication is a little bit different dynamics. In algeal syndrome, we're about 50% penetrated into the addressable sort of prevalent population. So still, I think a lot more to go. We are kind of the dominant share of IBAD, of the two, kind of us and our competitor, Bilvay. We really dominate the share in Allergeal Syndrome. So the primary kind of competition there is, if you will, activating patients who are eligible but haven't yet come to therapy. And we see those patients, even though FDA approved our product over four years ago, every quarter, every month, it continued kind of steady accumulation of ads in the U.S. and other developed countries in the allergio market. And, you know, kind of it's been on a steady cadence of ads for a couple of years now. So, you know, don't really, you know, see that being a kind of rare disease dynamic where that kind of continues to build throughout the life cycle. And then the PFIC indication, it's probably, if you kind of think about two separate groups, the thing with the pediatric onset PFIC, the kind of classic PFIC, we're probably collectively the class is probably even higher than 50 percent penetrated there but but that second entity I talked about the later onset or the adult PFIC it's very early days there in fact most adult professionals most adult prescribers are just now starting to do genetic testing the majority of them still don't so I think we've made a lot of progress and that's what's driven the growth but I think there's more progress to come with the more adult providers as we can get becoming curious about genetic whole stasis and taking the next step to do the genetic testing and then diagnose PFIC, we think there's a lot of growth to come on that runway.

And you mentioned the competing drug, Bilve. What is the market share dynamic in PFIC between Bilve and Livemarly?

Yeah, so in PFIC in the U.S., I think it's probably 50-50 at this point, roughly, pretty much split in the market, whereas Algio were considerably higher.

Got it. Well, I do want to touch on the EXPAND study. This is a phase three study that's ongoing that could open up a new market in ultra rare cholestatic pruritus. Top line data is around the corner in Q4. How should we think about the addressable market here? What is the opportunity? And how much incremental revenue could expand to drive Liv Marley forward to potentially becoming a billion dollar blockbuster drug?

Yeah, I mean, we think it's a significant contributor. So the readout will be in Q4. And we kind of talk about with Marley being over a billion in peak potential and really see allergy old PFIC and then this kind of expand basket indication as roughly being equal components of that. When we talk about the opportunity, you know, we've traditionally looked at the pediatric opportunity. So that's, you know, about 1,000 patients across the U.S. and Europe. And that's where we have good line of sight to patient populations. I mean, just out there with prescribers, you know, you can see who they have, that's patients that are allergial PFIC, and then also this other bucket. That doesn't really factor in the opportunity on the adult side of things, where we have a bit less visibility. But, you know, as we're out there and, you know, encouraging genetic testing for PFIC, we are seeing more of that. So I think as we get closer and get out there, we'll probably evolve the opportunity.

No, that's helpful. Well, I do want to shift to the looks of that and the VISTA study. You know, you recently announced the Phase 2B met its primary endpoint, and you're expecting to present data, the full data, at EASL later this month. What should we expect to see in your EASL presentation that you haven't already shared?

Yeah, I'm really excited about this data set. And just to recap, statistically significant improvement in paritis, which is the primary endpoint. For the EASL presentation, we've disclosed the material data. So the primary endpoint improvement, safety, some of the key secondaries. We'll have some incremental data on responder rates and be able to show a time course of paritis improvement. But very much what you'd expect from an IVAT. It's very rapid and sustained, and if not improving over time. paritis curve. So, you know, excited to get that out there. It's an oral late-breaker and should close out the late-breaker session on Saturday. But, you know, nice to get this out there soon, too.

Well, we look forward to seeing that data at EASL. And I just want to touch on the opportunity with Velixabat.

You've flagged it that previously that it could reach a billion dollar in revenue. are there any changes to that number since the VISTA study results were announced no not really I mean we've talked about it also being over a billion opportunity the PSC results are in line with what we expected you know we expected to see a benefit on itch just like you see with IBAT and other settings of cholestasis and we've we've shown that here so no no immediate change to the opportunity I mean it's pretty easy when you look at the market opportunities across PSE and PBC to get to some large numbers. But right now, I think where we've kind of set the bar is reasonable, and hopefully there's upside to that.

Got it. Well, let's talk about PBC. One of the risks in these pruritus trials is having a high placebo response. Your VISTA study design incorporated this screening period, these placebo run-in periods to specifically address this issue. I guess, how effective do you think those design elements were in reducing noise in the VISTA study? And more importantly, for the Vantage study in PBC, are you seeing the same level of noise reduction with these screening run-in periods incorporated into that trial as well?

Yeah, I mean, those are elements of, you know, say design. There's also elements of conduct where you're training both the patients and sites around the use of the ITRO tool. So credit to our study team in terms of their execution there. And those do seem to be effective. You saw in the PPC interim, you're always going to see a placebo response, but you want to mitigate that. And now in both of these settings, we've seen these approaches be pretty effective.

Are you seeing roughly the same percentage of screening failures in Vantage versus Vistas?

We don't see, let's see, it's generally comparable, maybe a little bit higher in PBC. Generally where you see screen failures is not for qualifying itch, but more for fluctuating liver labs. I mean, these patients have active liver disease, and that tends to be the main reason. The second reason would just be compliance with the diary, so just not filling it out consistently enough and that's that's important because you you know that that is your the basis for your end point so you want to make sure you have compliance uh with the diary going into the study got it and you know given the the positive results in psc is this making you more confident with the vantage study and the potential there for a positive outcome you know the the interim results in the vantage study made us quite confident i mean this certainly helps reinforce that. But, you know, in a, you know, 30 patient interim analysis, seeing such a clear separation between treatment and placebo across both those groups was very encouraging. And, you know, expect to replicate that in the phase three.

So is the bogey here for Vantage really just to beat GSK's, Alpha Sigma's, Linovoi, which showed 0.72 delta on the pruritus score versus placebo? Is Is that kind of the bar to beat?

Generally, I mean, I think we can do better than that. You know, we were over a two-point improvement from placebo in the interim, and so anything in that zip code is, I think, going to be really differentiated from a commercial perspective.

Got it. And for pricing, do you have any idea why Lenovoi hasn't been priced yet? What's the holdup?

Well, I mean, so GSK sold it to Alpha Sigma. You know, they had to go through the HSR, and so there's probably some just logistics of transfer from one sponsor to another. But we're actively watching for it and would expect them, you know, to launch soon, given that it's approved.

And would you think of pricing it at a premium to Lenovoi? How are you thinking of Elixabet?

You know, that's certainly one of the options. I mean, that's one of the debates that we have a lot, is do you price for PSC, which will be first, or do you price more for PBC? Certainly recognize that pricing for PSC, you have potentially more pricing power, given it's a smaller population, and there's nothing approved in this setting. So that's one bookend. Another bookend would be pricing closer to the PPARs in PBC. Ultimately, we'd want to see where Linovoi price is. we'd like to see the final profile of the looks about in pbc which we'll have before we have to set price in q1 of next year um with the vantage readout so a few more cards to turn over uh but you know suffice to say our goal will be to to you know maximize access and value around looks about and thanks for bringing up the uh the ppar agonist which uh gilead has one ipson has one already approved for for PBC and these are these two drugs have a different mechanism of action versus IBAT.

How should we think about PBC here with these other drugs that are already approved? Is there a possibility for combo treatment? Is it going to be one drug versus the other? How are you thinking about the market opportunity here for PBC?

Yeah it's there's Certainly, room for combo dosing. We already have patients on PPARs in our studies. Typically, it's the fibrates, which are effectively PPARs. And you do see use in these settings. In PBC in general, though, you see similar rates of paritis across lines of therapy. So even patients who are doing just fine on ERSO still experience this moderate to severe paritis. So that's the prime area of focus for us in PBC, where these PPARs would be off-label. But in the second-line setting, PPARs have an effect in some patients but not others, and so those are clearly opportunities where IBAC can play a role.

Got it. Very helpful. Well, I do want to talk about hepatitis D and beloved Tug. The phase TB interim results were quite striking, but they left me with some questions. You know, we saw 100% virologic response in the 300-mig weekly dose, and 45% at that dose achieved the primary endpoint versus zero responders in the placebo arm achieved the primary endpoint of virologic response and ALT normalization. However, the 900-mig, the monthly arm, this didn't quite fare as well as the 300-mig arm, which was dose weekly. So how should we think about the slight disconnect between the two when arguably patients should, in theory, be getting exposed to the same level of drug?

Yeah, I mean, I can take that one, Lisa. Our take there, I think your final point is correct. I mean, the exposure between the two, the pharmacokinetic profile between the two different doses is pretty comparable, maybe slightly higher for 300, but not meaningfully. So probably any differences we're seeing between the arms and efficacy is due to small numbers. In fact, some of those patients in the 900 milligram monthly arm that just missed the two-log viral reduction cutoff, they might have been at 1.5 or 1.7. So they're clearly getting a meaningful response. And likely, if you look at instead of a week 24, if you look at the next time point, maybe week 36, you're going to see them cross that threshold. So I think it's small numbers and just kind of response kinetics is probably what we're seeing here.

And does the Azure One interim increase your confidence for the pivotal readouts in Azure One and Azure Four that are coming later on in the second half?

Yeah, I mean, I'd say so. We already had pretty high conviction based on the Phase 1B data that were presented last year to ASLD. Those were the data we had in hand when we entered into the acquisition of Blue Jay. And then this, it was really nice to see that get replicated here, of course. So I'd say that both data sets kind of have given us the confidence on the full phase three.

Got it. And what about competition here in the hepatitis D space? You have VIR. They have a dual approach with a monoclonal antibody and an siRNA. And you have Gilead in there, in Hepcotex, which is already approved in the EU, but not in the U.S. So how are you thinking about positioning Beloved Tug if there's other competitors in the United States?

Yeah, thanks for the question. I mean, I'd first say it's great news for patients. I mean, especially in the United States, there's been nothing to treat these patients with other than interferon, which most people don't want to give. It doesn't work very well, and there's a lot of toxicity. So it's great that you have, I think, three drugs regimens coming to the market soon that are going to make a big difference for people with the most deadly form of viral hepatitis. So excited to be a part of that. And I think all of them are active regimens. I think with Gilead kind of going chronologically, we expect, as we understand it, a PDUFA date and a potential action this quarter. I think that'll be really helpful to have an option out there that motivates clinicians to do screening. It's right now a very low percentage of hepatitis B patients ever get tested for Delta. You know, I think Gilead has proven they're quite adept at developing viral hepatitis markets over the years. So I think that'll be a positive dynamic for the overall market and growing the number of diagnosed patients, increasing the interest in treatment. I think when we compare, you know, profile to Belovatide versus Belovatide, their epoclunox, we feel there's, you know, kind of advantages probably on efficacy, safety, tolerability, and convenience, more active regimen. And instead of a daily sub-Q with reconstitution, it's a weekly self-administered sub-Q. So feel really good about the profile there. And then, of course, VIR also has a really nice regimen that's, you know, making a big improvement for patients. You know, when we kind of, in this world, be a competitive market, when we look at our profile, we feel really good about how we perform on the composite endpoint that FDA and EMA are focused on, which is both the two-log viral load reduction together with the liver inflammation, ALT normalization, is both of those together are what's most associated with improving outcomes in this setting, whether that's liver cancer, liver decompensation, transplant. So I think that's really strong. We've also enrolled a really broad population. Some of the other programs have excluded more advanced patients with higher ALT or meaningful cirrhosis, and that's about 20% of the population and the most high-need population that will be on-label for us and not for some others. And then finally, just a really simple, single-agent monoclonal antibody that's very well-tolerated. We have a very low rate of flu-like symptoms, which from the feedback we've received from the patients, It's, you know, flu-like symptoms are important to them, and for a chronically administered medicine, it's obviously desirable to have as low of a rate of that as possible.

Well, I do want to touch on one more thing with hepatitis D. You've outlined potential for a $750 million opportunity for a bill of a tug. How do we get there? Do we need both U.S. and ex-U.S. approvals to hit that number?

Well, that is a global number, but, you know, if you look at even just the U.S. and you assume we don't improve the number of diagnosed patients and just stick with the number that are diagnosed today, which is 15,000 patients, if you kind of start there and you assume a competitive split of the market, which is kind of the way we modeled it when we acquired the program and just kind of assume it's competed, there's competitive shares, and you have rare disease pricing, it's pretty easy to get towards those numbers, even just with the U.S. But we do have a development plan and a regulatory plan to make this available broadly around the world.

Got it. Well, I do want to talk about the FOP program. This is the asset that you recently acquired from Insight. What is opportunity here? How do you plan to compete versus Ipsen, Sohonos, which is already approved, and potentially Regeneron's drug as well, which could be launched maybe a month ahead of Zilu?

It's a little assertive. It's a tough one to say. Yeah, we're excited about this program. FOP is just a devastating condition where patients, you know, essentially form a second skeleton every time there's an insult to their soft tissue or connective tissue. So it's really just a horrific, ultra-rare setting. About one in a million is the kind of epidemiology for it. So about 300 patients in the U.S. with this. And, you know, the SOHONOS is only a small proportion of the FOP patients are on SOHONOS. The profile is kind of quite limited, both from an efficacy and safety tolerability perspective. So, you know, we think that both, you know, both this program as well as the Regeneron monoclonal antibody are major advances for patients from an efficacy standpoint. for our program it's an oral once daily profile which for these patients who any little thing including an IV infused monoclonal antibody can actually cause ossifications for them we think that'll that'll give it a nice you know position in the market well maybe last question for me in the two seconds we have left here Peter I guess for for Miriam how are you thinking about balancing profitability versus investing in more bd to build out the pipeline further and potentially the top

line but andrew take that closer our focus is on value creation and i mean that's you know we've taken a really disciplined lens towards that so less about profitability and i think our priority right now is value creation through our pipeline and you know commercial business and you know bd is a big part of that so we'll continue to stay disciplined we will also continue to stay financially independent so profitability is not the goal but you know the business is in a really strong financial position and we aim to keep it that way well peter andrew thank you so much for joining me this afternoon it's been a pleasure to host you both and i hope you have a great day

thanks lisa thank you yeah