Conference Transcript - MIRM 2026-09-15

Speaker 1

Good morning, and thanks for joining us to have a conversation with Chris Peets, CEO of Myram Pharmaceuticals. Myram is a commercial stage biopharmaceutical company focused on transforming treatment of rare liver and debilitating diseases. The company's commercial base is anchored by Liv Marley in cholestatic pruritis in allergels syndrome and PFIC, alongside with bile acid medicines franchise, which together delivered $176.2 million in second quarter revenue and supported raised full-year guidance to $680 million to $700 million. So behind the base also sits a dense set of near-term events. We have a PDUFA date for xylurgisart tip, the oral ALK2 inhibitor for FOP registration enabling Azure 1 and 4 readouts for Brello we took in hepatitis delta in the second half, and also we're going to see data from the X-PAN basket readout. So to talk about the commercial franchise as well as the pipeline, let's get started with Chris. Chris, glad to see you, and thank you for accepting our invitation and talking to our audience today. So to start off, you know, especially for folks who are new to Myram, you know, what is the long-term strategy for the company? And as you grow the commercial base in the cholestatic liver disease, but also you're seeing some looking into that potential FOP and the hepatitis delta. How do you put all these pieces together?

Chris Peetz, CEO

Yeah, thanks. Thanks for hosting. It's a busy year for Miriam. So happy to dive into it and break some of that down. First comment, I will be making forward-looking statements. So refer folks to our SEC filings for full disclosure of risk factors. And to dive into first the the strategy of Miram and who we are, what led to the creation of the company about eight years ago, was really started from a search to look for underappreciated opportunities in rare disease. So our strategy as a company is to pull together these small to mid-sized products that really fly below the radar of most of the larger companies. And there are a lot of opportunities out there like this. And in fact, across rare disease, you know, probably only about 5% of those rare diseases have approved medicines. And there's even opportunity to improve on those that have existing medicine. So there's really a long list of opportunities that we look at and are excited about across the rare disease settings. Today in Miram, as you covered some of the highlights, the way I'd describe it is we have a thriving commercial business based on three approved medicines. Livemarly being the real highlight of the growth profile that we can dive into what we're seeing, really most recently on PFIC, has been a real standout in driving that continued growth and a lot more to come with an estimated 2,000 adult patients that we think are out there that would benefit from genetic testing and a more complete diagnosis. And behind that, across the pipeline, all of these really game-changing potential opportunities for new medicines. The first up is the PDUFA date for Zoologacertib, which would lead to the potential approval of Zoologacertib for FOP. And we can talk about the launch profile there. Really efficient ad for our Rare team. And then data readouts also starting later this month for Relovatug with Azure 1. Azure 4 follows next quarter, expand next quarter. Vantage for PBC first quarter next year. So it's pretty stacked right now. We've got a lot coming on.

Speaker 1

Okay. So on the commercial side of things, the live marley and the bile acid medicines and bile acid biology. So what's the rationale there? And how do you differentiate Miriam's approach versus other folks who are working in the same cholestatic disease therapies?

Chris Peetz, CEO

So looking at Liv Marley's profile and breaking down what we've seen over time. So Liv Marley's first approval was for cholestatic pruritus and allogial syndrome and followed with a label expansion and cholestatic pruritus due to PIFIC. And the profile for response for patients in both Both of these indications can be really quite profound in addressing the puritis that is a hallmark symptom of these diseases. Both Allergeal Syndrome and PFIC can drive highly elevated circulating bile acid levels, and Livmarly interrupts the recirculation of those bile acids to reduce systemic bile acid levels. And in the pivotal studies, you saw quite pronounced reductions in itch because of that. The drug profile, then playing that forward, is one where patients feel better usually on medication because of that improvement in itch. So you see really good persistence, really strong feedback from patients on how they're responding to drug. And the tolerability of it, the mechanism of the drug actually excretes bile acids in stool. So there is some GI side effects to this, but it's typically seen as a favorable tradeoff for the itch benefit that patients see. How it differentiates from others, there is another IVAT inhibitor that's approved in the same indications. And I credit a lot to our clinical profile and our team's performance and the growth profile that Liv Marley's been able to demonstrate, really quarter over quarter continuing to to add both new patient starts, but also supporting strong compliance persistence for patients that are on therapy. That's led to the commercial performance we've seen for Livmarly. We've been spending a lot of time talking about the adult PFIC opportunity this year. It's something that's emerged really over the past year, year and a half, where as we had the PFIC label expansion for Livmarly, and we're more active in some of the adult clinics, really only probably the top 10% is where we've been active historically, we've been seeing that there's historically limited awareness of genetic testing in what's described as the idiopathic cholestatic patients. So those don't really have a precise diagnosis of their cholestatic disease, still have symptomatic burden, still have elevated bile acids, but an incomplete diagnosis. And when a genetic panel for the cholestatic genetic diseases is run, actually many of them will have PFIC genetics. So that's an effort that we've been building on to help support awareness of genetic testing of what is once thought of as a pediatric onset disease actually can have an adult onset profile. And getting the genetic testing out into the adult clinics has helped bring a more complete diagnosis for patients. And that's turned into also with Marley prescriptions. Where we're taking that going forward, you know, to date, you know, really incomplete coverage on the adult prescribing audience. So we're expanding our commercial team now so that as we get into next year, we'll have a more complete call point on the adult liver and GI specialists that treat and diagnose these patients. And that actually has two benefits, not only helping support Livmarly's continued growth, but also having us ready for the Brillova tug and the Lixbat potential approvals that will be down the road. So it's all three products would be with the same Same team.

Speaker 1

A commercial team. So talking about, you know, live morally in adult population, you know, how long does You know, you just talked to us about, you know, diagnosis. So once diagnosed, how long does it take for a genotypically diagnosed patient to start on therapy?

Chris Peetz, CEO

So once a diagnosis is made, then it comes down to, from what we see, a conversation with the physician and the patient about symptomatic burden and treatment options. So with a genetic diagnosis of PFIC, it's on label for Livmarly as an option to treat cholestatic curitis in the PFIC patients. And those that have the symptomatic burden, they're looking for treatment options. And that decision really varies in the amount of time it takes. But those that are seeking more complete diagnosis tend to be doing that because they have symptomatic burden. They're looking for a new treatment option.

Speaker 1

So regarding the revenues from Livmarly by itself, you know, in the second quarter, it was close to $129 million, which was actually a 13% sequential growth. So, as we go forward, you know, just to think about what sub-segment patients come into this, you know, can you break for us, you know, between new patient starts and also the patients who are on, you know, weight-based dose escalation?

Chris Peetz, CEO

So, we haven't parsed out and shared each of those different components. They're all at play, though. So across the different components of the total of Marley revenue, Alvajil syndrome in the U.S. continues to grow quarter over quarter, both through new patient starts, dose adjustments over time for the pediatric patients. PFIC new patient starts, I call out, is the biggest kind of growth driver that we've seen over the past recent quarters. And then international also continues to perform well. And internationally, that is more predominantly Alagil-driven still today, though PFIC has been a bigger contributor of late. So expect continued growth internationally as we roll out both Alagil and PFIC, not only within the current existing markets, but also some additional geographies that we'll address through distributors. Latin America is one that has a good growth potential going forward, Middle East still in early days. Each of these on their own is relatively small, but they all add up to be real contributors.

Speaker 1

And then talking about the commercial footprint and the expansion that you're putting together, I believe right now you're only detailing about, you know, top decile of the adult hepatology and gi prescribers um as you continue to you know strengthen the commercial team um two questions you know how long does it take for these folks you know to be mature in terms of what they're doing you know for the business and when would it actually translate into revenues so the field expansion is work that we're kicking off now uh so efforts underway to to build out that expanded

Chris Peetz, CEO

call point. For it to be really fully deployed, it's probably not until end of this year, early next. And expect that to continue to support the trends that we've been seeing in adult PFIC as we just reach out to broader into that audience. I would say also beyond just the commercial team, which having this increased presence for adult PFIC, those 2,000 potential adult PFIC patients that we've been talking about in the US. There's also kind of broader scientific evidence and recent publications and a lot of the conference material that has been oriented towards genetic holostasis. So it's not just us out there kind of building awareness of these genetic panels. It's also being driven from the KOL community as they're further characterizing, identifying more variants that are drivers of PFIC and understanding how to interpret the genetic testing results. All of these are elements of also being driven by research out of the academic community.

Speaker 1

Okay. So then the next big excitement is about the EXPAND study, which is the data coming out, you know, in the fourth quarter.

Chris Peetz, CEO

So when you, you know, what would you consider as a as a as a clear win on the primary and point itself and then how much of the what is the accessible population for that you know once once you get the label expansion up so the expand studies it's a basket design and was really driven by what we saw for compassionate use requests across a broad profile of different background cholestatic diseases, that each on its own was quite rare to have cholestatic pruritus for any of these underlying conditions. And that interest led to a discussion with FDA about how to design a protocol around it. So fast forward to today, you know, heading into unblinding next quarter, that study has enrolled about half of it in biliary atresia, so patients with puritis due to biliary atresia. These are post-Kasai patients, different profile than what was been studied in some of the earlier studies that were more looking at infants immediately after Kasai. These will be patients that have had a stable response to their Kasai, but develop cholestatic puritis later in life. And the other half, really, I'd describe it as a long tail, like many different causes of cholestasis, each one on its own kind of being a one-off representation in the study.

Speaker 1

And then international business, regarding international business, the CHMP has a positive opinion for Liv Marley, the oral solution for ages up to three months of, I mean, down to three months of age.

Chris Peetz, CEO

How should we think about ex-us contribution you know let's say over the next two to three years if i look at what we see internationally i mean europe is one piece of it but it's broader than that as well so our distributors have been really strong performers across different regions outside of our direct markets in western europe and expect that to continue to grow especially with the the same pfic trends that we've seen in the u.s see that there's similar opportunities across some of the European markets though the the mix between new patient starts and existing markets and new distributors both of those really play in over time okay so

Speaker 1

moving on to Alex about I know you have fielded enough questions on PSE over the last month month and a half but the exciting piece next is the vantage study which we are expecting soon when I say soon in the first quarter of 27 you know, what do you need to see in that study so that, you know, you can take it into a pivotal study as well?

Chris Peetz, CEO

Yeah, kind of put this, the Velixbat program as a whole into a little bit of context. You know, when we started both the PSC and the PBC study, we, through written interaction with FDA, designed and asked for feedback to make these both pivotal studies. So they're both designed as adaptive pivotal studies for cholestatic caritis in PSC and PDC. The key difference between them is the PSC study was a blinded adaptive design throughout, so operationally seamless. We did not unblind interim data, all of this discussed with FDA, to have this be an adaptive pivotal study. So we kind of didn't have interactions with FDA until that data readout. And as we've talked about, we're working towards a planned NDA submission for PSC first half of next year. So no changes to what we've shared about the plans for PSC, and that's all underway. For PBC, the interim structure allowed us to have an unblinded announcement and look at the dose comparison for high and low dose folixabat versus placebo a couple of years So data looked quite strong and was the basis for a breakthrough designation for PBC, and that allowed us to then have further interactions with FDA, clarify what they wanted for the safety database size for PBC, and some of the statistical design elements to support Vantage being the pivotal study for an NDA. game. So feel we're in a strong position heading into the Vantage readout. What we saw at the interim was a strikingly strong result. And so anything close to that, I think, would be a game changer for PBC patients. It's such a pronounced and rapid reduction in curitis that I think it would be a really strong product for the PBC market. The thing to point out is we get a lot of questions about how Velixabat, what role it would play relative to the PPARs that have been recently approved and that are used in those with elevated alkaline phosphatase on UDCA. The thing to note in the Velixabat studies is that it's across the PVC population. So it includes both patients that are well-controlled biochemically on UDCA, as well as those that would be more of a a typical second-line patient where the PPRs are used. We see patients in both settings and consistent puritis response in that interim data set across both profiles of patients.

Speaker 1

So moving on to hepatitis delta, you know, Azure 1 top line is expected this quarter and 4 in the fourth quarter. In the phase 2, you know, the drug showed 100% virologic response across 46 patients, and also an 82% composite response at the 900 milligrams every four weeks. So is that the right benchmark for us to look at when the phase three readout?

Chris Peetz, CEO

The key consideration to keep in mind there is that that's a different time point for those, that specific data point. The phase 2B data that we announced earlier this year, That's the 24-week time point and gives a better sense of where we expect the phase three data points to read out, also 24-week time point, where the 300-weekly arm, which actually has slightly higher exposure overall, so that's overall the dose that we would expect to have a stronger response, saw a really strong virologic response, so that's 100% of patients having that two-log or greater reduction, and also strong ALT normalization. So at the 24-week time point, that phase 2b data that we announced earlier this year is the best benchmark to think about. We do expect response to improve over time. So as you get to the week 48 time point and beyond, we're seeing patients that are on therapy longer continue to have have improved or lower virologic levels and more patients having ALT normalization.

Speaker 1

And then, you know, which is this, the next catalyst, which is immediate, Xyler-Gisterp, which has a fidufidate on 26 September for FOP. So when you look at Regendron's drug, which just got approved, how do you think the competition is going to, you know, look between the two drugs? And what are you watching for from the Regendron's drug?

Chris Peetz, CEO

For us, heading into the Zorgasertid launch, you know, focused on getting a drug out to patients after this potential approval. Key things that we're excited about that got us excited about doing this deal with Insight, who did a great job bringing this through regulatory review to date, is that it's an oral once daily, and the pivotal study included patients down to 12 years of age. And there's a real desire to start treatment younger to prevent the accumulation of these ossifications. So I think that's one of the real strengths and the clinical profile for ZalurgoCertib is that we expect our label would be for patients 12 years and older for the once daily oral regimen.

Speaker 1

So the last question from me is on the on the finance financials with the with the current cash runway that you have you know what what sort of runway should we expect?

Chris Peetz, CEO

So for this year it's a bit of an investment year. So while we had been cashflow positive in the coming quarters, expect to be roughly break even or slightly negative. A lot of that just from the investment for Lovatuck scale up as we are adding a second site for manufacturing. But heading into next year, expect to be operational cashflow positive again. So expect that into next year, there's an opportunity to add to that cash balance and certainly as we get further beyond this CMC investment that we're in now expect the cash flow to increase thank you thank you Chris for being here thanks for asking