Mirum Pharmaceuticals at RBC Conference: Strategic Growth Highlights

On Wednesday, 21 May 2025, Mirum Pharmaceuticals (NASDAQ:MIRM) presented at the RBC Capital Markets Global Healthcare Conference 2025. The company showcased its strategic advancements, highlighting record growth in LIVMAARLI sales and significant pipeline developments. While geographic expansion and new formulations offer promising prospects, challenges remain in market penetration and competition.

Key Takeaways

• Mirum Pharmaceuticals reported a record first quarter for LIVMAARLI, with $73 million in revenues, driven by growth in Alagille syndrome and PFIC indications.
• The EXPAND study is progressing, with enrollment expected to be completed next year, potentially broadening LIVMAARLI’s indications.
• The bile acid portfolio, including Cetexly and Colbam, showed significant growth, with a 50% increase in revenues.
• The vilixibat program for PSC and PBC is advancing, with promising interim data on pruritus improvement.
• Mirum is developing a Fragile X program, maintaining strong intellectual property protection.

Financial Results

• LIVMAARLI revenues reached $73 million in the first quarter, with two-thirds from the U.S. and one-third from international markets.
• The bile acid portfolio, comprising Cetexly and Colbam, generated $37 million, marking a 50% increase from when these assets were under Trever’s ownership.
• Growth in Colbam usage is largely attributed to its application in Smith-Lemli-Opitz syndrome.

Operational Updates

• LIVMAARLI has nearly 50% market penetration in the U.S. for Alagille syndrome, targeting 1,000 to 1,200 patients.
• The new tablet formulation of LIVMAARLI, approved by the FDA, offers a competitive edge with IP protection extending to 2043.
• Takeda, Mirum’s partner, has gained approval for LIVMAARLI in Japan, with a launch expected soon.
• The VISTA study for vilixibat in PSC is on track, with enrollment expected to complete in Q3 this year.

Future Outlook

• Mirum anticipates continued growth for LIVMAARLI in Alagille syndrome and PFIC, along with further geographic expansion.
• Completion of the EXPAND study enrollment is expected next year, with data readout following six months later.
• The company aims to increase patient numbers for Cetexly through patient finding and reimbursement initiatives.
• Data from the VISTA study in PSC is anticipated in Q2 next year, with a focus on pruritus as an approval outcome.
• A Phase 2 study for the Fragile X program is planned for later this year, with ongoing FDA engagement.

Q&A Highlights

• Pruritus remains a significant concern for PSC and PBC patients, with 60% experiencing active scratching.
• Payers are supportive of funding treatments that improve pruritus, recognizing its impact on daily life.

For a detailed understanding, readers are encouraged to refer to the full transcript.

Full transcript - RBC Capital Markets Global Healthcare Conference 2025:

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: I’m Brian Ebers, one of the senior biotech analysts at RBC Capital Markets. We’re really pleased to have our next company here, Miriam Pharmaceuticals, represented by their CFO, Eric Viarholt, and their SVP of Finance and IR, Andrew McKibbin. Eric and Andrew, thank you guys so much for being here.

Eric Viarholt, CFO, Miriam Pharmaceuticals: Thanks for having us, Brian, and, thank you to RBC for inviting us to this conference.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Great. So maybe we can kick things off on the commercial side. And, know, you recently reported, LivMarley sales. Can you talk a little bit more about the catalyst behind the continued strong commercial performance that you’re seeing for this drug and maybe how far along you are now with market penetration in the, ALGS and PFIC indications?

Eric Viarholt, CFO, Miriam Pharmaceuticals: Sure. Let me start out by saying that, both Andrew and I will be making forward looking statements today, so please refer to the risk factors in our SEC filings. So, first quarter was a record quarter for us. LIVMAARLI, we recorded about $73,000,000 in revenues, about twothree of that is in The U. S, onethree ex U.

S. And in The U. S, we’re approved for both Alagille syndrome and PFIC. We expect to see continued growth for many, many years, for both Alagille and PFIC in The U. S.

And also continued geographic expansion internationally. So specifically, to answer your question on the, Alagille syndrome, we think we’re we think there’s about a thousand to twelve hundred prevalent population, of Alagille patients patients that are addressable with LIVMALI in The US, and, we’re approaching fifty percent penetration of that population. In addition, the annual incidence is about a hundred patients, maybe a 20, and, we’re capturing, really a major share of those new newly diagnosed patients with pruritus. For and so, you know, given that we’re only less than fifty percent penetrated, we see significant growth opportunities continuing throughout the patent life, which reaches out to 02/1940. For PFIC, we’re just scratching the surface, just starting.

We’re capturing newly diagnosed patients. We’re capturing finding actually already diagnosed patients that are have not yet seen an IBAT inhibitor. But, you know, we’re clearly behind, Belyvy, who was launched first in PFIC and and still has the majority share, but we’re very pleased with, our launch so far and expect to see significant growth in PFIC as well. And then internationally, we’re now in over 25 countries. We’re commercializing ourselves in the major Western European countries and now starting also in the in the mid sized Western European countries, and then we use distributors and partners in other geographies.

We expect continued geographic expansion this year. Our partner Takeda in Japan just recently was approved for both Aladil and PFIC, and we expect them to launch in Japan very soon and have high hopes for that market where we have really good economics.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Great. And then I know you guys are looking in this EXPAND study, the basket study, for additional indications, cholestatic pruritus indications. Can you talk a little bit more about that study? How many additional patients you think you can bring on to LiveMarley? How enrollment’s going?

And just maybe remind us of the process for those data to translate to the potential, commercial expansion to some of

Eric Viarholt, CFO, Miriam Pharmaceuticals: these smaller subcategories. It’s important to take a step back and understand how that trial came about, the EXPAND study. We had a number of compassionate use requests, in fact, so many that the FDA complained and said too many respects requests. Please run a study. And so in collaboration with the FDA, we designed this basket study, which are consists of a number of, you know, much smaller, cholestasis indications.

Primarily, the biggest one of those is probably biliary atresia. And we think in the aggregate, it’s, at least 500 addressable patients, which means, you know, put it in context, it’s probably at least a PFIC indication. And, we’re planning to enroll that study and complete enrollment, next year. And, it’s about a six month study, and so we’ll we’ll we’ll get data, you know, sometime after full enrollment.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Got it. Good. And then you’ve talked recently about the new tablet formulation for the drug. Can you tell us a little bit more about sort of how import how different that is from the prior formulation and how important that can be both to driving additional market uptake and sort of capturing the pace that the space that’s still kind of remaining that you talked about in earlier, and then also how it impacts your competitive positioning overall?

Eric Viarholt, CFO, Miriam Pharmaceuticals: Yes. Thank you. Yeah. So the tablet formulation was, approved very recently by the FDA. The, prior formulation is a solution, which is, appropriate for, small children.

But as kids get older and thrive on Live Marley, there was some demand for a solid formulation, which is more convenient as you’re a teenager or an adult. Think about you know, you’re not taking a liquid kid’s Tylenol anymore. You probably prefer the tablet, and it’s similar with kids as they transition to adulthood. And so we now have approval where for four different solid doses in addition to the liquid. And so for each patient, they would only take one pill per dose, which puts us in a very good competitive position versus Bilvey, which, requires multiple large pills per dose.

It could be as many as 18, could be as low as six depending on what weight you’re on and what dose you’re on. And so previously, you know, we we were at a competitive disadvantage with older patients because we didn’t have a solid formulation, but now we think we’re at a competitive advantage because of the lower pill burden.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: And what proportion of sales or the market are these older patients?

Eric Viarholt, CFO, Miriam Pharmaceuticals: It’s a little hard to tease out, but, we’re and it’s gonna be an increasing share, I think, as the kids that are on LivMarley age into their teenage years and adulthood. But, you know, I don’t know if you wanna make sure I get it.

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Generally, the average age is around, you know, six to seven. So it it’s it’s skewed lower.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: K. But over like you said, over time

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Over time, that will Yeah.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: These patients grow. More prominent. Okay. Let’s talk about CTX. I guess I’m curious on, you know, the recently approved, Cetexly, what you guys are seeing on the ground in terms of new patient starts, just how broadly this disease is is diagnosed, and how patient finding efforts are are going?

Like, what’s the ultimate opportunity you see there?

Eric Viarholt, CFO, Miriam Pharmaceuticals: Yeah. Let me broaden my answer a little bit. So the bile acid portfolio, which consists of Citexly and Colban, generated about $37,000,000 in revenues in the first quarter, and it’s important to know that we acquired this asset from Trever a couple of years ago, and that’s a 50% increase over the last quarter in Traverse hands. So we have been able to move the curve a little bit. They had previously been growing at low single digits annually, and we think we’re able to to move that.

For, Colbam, much of the growth is coming from, Smith lemliopid syndrome. And then for Catexley or Chenodal, it’s all CTX, and it’s standard of care for CTX. And, right now, you know, it’s only a little over a hundred patients diagnosed and on on Cetexly or on Chenodal, But we think that’s only ten percent diagnosed, ten percent of the potential, which doesn’t mean it’s gonna be easy to increase the diagnosis. But now that we have approval, we have the ability to, one, promote and try to actively find those patients, whereas previously we couldn’t. And secondly, we can, do a copay reimbursement now, which we previously could not either.

And so for both of those reasons, we are hopeful that we will be able to find new patients and, put them on Cetexly, for for the long term. Great. Let’s shift gears to PSC.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: I know you guys are looking at vilixibat there. Maybe talk about the overall market opportunity for for PSC, just, you know, how big of an issue pruritus is for those patients? And what are we continuing to learn about the impact IBAT inhibitors can have on pruritus in PSC?

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Yeah. I mean, overall, you know, we see about thirty thousand, maybe more patients in The US. And so, I mean, net net, see it as a very substantial opportunity. These are much larger PSC, PBC, much larger indications than the ultra rare algal and PFIC settings, where pruritus is a major issue for these patients. If you kind of look at the the patient community and, you know, what they talk about, it’s pruritus and fatigue.

You know, and generally in the range of sixty percent of patients from the work that we’ve done and kind of triangulating with some of the patient advocacy groups, about sixty percent of patients have active scratching. And, you know, frankly, this is these kind of the patients who would be on label for for aveloxabat, directed treatment. They have nothing approved, nothing approved to treat the disease or to treat pruritus. So they currently cycle through off label therapies. There’s some experimentation that physicians do, but lots of debate and controversy over the therapies that are currently used for the disease.

And for pruritus, it’s the same, types of things that we see in the pediatric community. So, cholestyramine, which is not well not well tolerated from a taste perspective, so rarely do patients stay on that. Rifampin in in some cases, antihistamines, which can just knock you out. None of that stuff is that effective. And, you know, what we see with IBAT inhibition in, you know, all of these cholestatic settings where you have something that’s impairing the bile flow, driving symptomatic pruritus, and, really a toxic level of bile acids in the in the liver and body, IBAT inhibition flushes that out.

So you see a very, rapid and dramatic reduction in pruritus. Importantly, if you optimize your dosing. At low doses, you see it kind of work. At maximal doses, you see a much different effect. And we’ve seen that across our development program, and I think we’re there with the right dose for velexibat.

I would say as it relates to PSC specifically, we have two data sets with, LivMarley in PSC, one run by the previous sponsor, several years ago, and then recently at DDW, a set of patients, on compassionate use LivMarley for PSC. And what you see is over half, over half resolved their itching. So very consistent effect, you know, to us, not a surprise, but, you know, very excited about the VISTA study past its interim, on track for data in Q2 of next year.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Yeah. And speaking of the interim, can you talk a little bit more, about that? I guess, how the overall study is progressing, you know, what this interim read told you, and what you’re viewing is, what could be a clinically meaningful HRO score reduction, at at twenty eight weeks.

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Yeah. Study’s progressing very well. You know, very excited about that. We we kind of clarified, or narrowed our enrollment completion time into q three of this year. So very happy with the progress and momentum of that study.

Typically, you know, what what you want to see from a clinical meaningful change from baseline is about a two point change. So two point improvement on a zero to 10 numerical rating scale of worst itch. A very simple scale, two point improvement from baseline is what your target is. And then, you know, generally at least a one point improvement over placebo, so the placebo adjusted score. The p s the PSC interim was a blinded interim, we did not see the data.

It’s a it’s a smaller population than PBC, and we really wanted the study to keep going if we were seeing a positive effect that was predictive of a final positive dataset. So we didn’t want to slow it down. In conjunction, we also unblinded, the PBC interim analysis. Since that requires a larger safety database, that gave us some room to take a look at the data. And just to put that in context, we saw almost a four point improvement from baseline and a 2.5 placebo adjusted improvement.

So very clear signal, which is why we had confidence in taking this approach in PSC that if the study is on track, keep it going. So now we have those patients from that interim count as part of the final dataset, and we know we’re seeing something positive there.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Okay. Great. And as we sort of get closer to the to that dataset, can you talk a little more about the regulatory path in PSC? I know for disease modification, there’s been challenges, but for pruritus, there seems to be a a tried and true path that you guys can can follow from from your other indications. But talk a little bit more about that, And, you know, what’s your latest expectation?

Any chain does that set changed at all with some of the changes to to the FDA, and how you’re thinking about what potential labeling might look like ultimately in PSC?

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Yeah. And we haven’t seen any changes. And I think that this kind of touches on one of the really unique aspects of our program is that pruritus in and of itself is an outcome. So field function survives, that’s what the FDA looks at, and outcomes are what they want in PSC. So given that price is an outcome, you know, this is an endpoint that confers full approval.

So not surrogate approval, but full approval, which, you we are very excited about. That’s something you can study in a short period of time, and that’s how we’ve designed this study, you know, in conjunction with the FDA reviewing the endpoint and approval pathway and Cisco analysis plan. Unlike the disease modification endpoints, which, you know, as it stands, there’s no surrogate that’s approved. And so you’re looking at some aspect of an outcome study, which is, you know, long and expensive and, you know, carries some degree of risk. So really unique and positions us to be the first approved therapy for PSC, which is very exciting.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Excellent. And then you touched a little bit on the interim on PBC, but can you talk a little bit more? I know you recently had kind of some longer term data at EASL, and just maybe what that longer term follow-up tells you about the durability of effects, with with the Lexabat.

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Yeah. I mean, the durability was very clear. So effectively, at EASL, a few weeks ago, we presented the full twenty eight week worth of data for all the interim patients, so about 31 patients out to week 28. And what you see is a very consistent durable response that if anything, you know, improves a bit at the margin. You also see improvements in fatigue, which is, very important.

I mean, something we’ll be looking at closely in PSC as well. Pritis and fatigue are two of the main symptoms and, you know, not necessarily, linked to just improvement in pruritus if you’re seeing improvements in fatigue, as well as some changes in markers of, inflammation. So IL 31, for example, and some interesting signals with the pro

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: c three. Excellent. And can you also help us contextualize the benefits that you guys are seeing versus some of the emerging, competitive data from from others? And I I guess curious how similar the designs and or different the designs and populations are to really enable a true comparison.

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Yeah. Mean, we we see these data sets as very comparable. We’re using the same scale for each measurement, zero to 10 NRS scale. Ours is branded Itro, but it’s effectively the same thing. And the duration of about six months of treatment is also a very fair comparison to to look at from a timing perspective.

You know, ultimately, think this comes back to dosing. You know, we put a lot of work into making sure we were optimizing the level of IBAT inhibition with the doses selected. We know that GSK because this I think this is the main dataset, to reference phase three in PBC, presented at EASL a couple weeks ago. They went with a lower dose than had been evaluated in, their dose ranging in phase two. And, you know, conversely, they you you see an effect.

The study was stat sig, but the placebo adjusted benefit was not particularly strong. It was about point seven, improvement over placebo versus a 2.5 improvement in the valixabat study. So I think the dosing piece matters, and we’ve seen that very clearly in PFIC with Libmarli, where early studies, you see an effect, but when you and we effectively doubled the dose and did twice daily, and there you saw a much more pronounced, benefit. Got it.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: What’s your sense as the payer landscape in PBC and PSC?

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Fairly receptive. You know, it’s I I think there’s some, on on the payer side, some hesitancy to pay for, biomarker improvements. You know, it’s harder to correlate that with an outcome, and that takes some time to play out in, in PBC particularly. Psoriasis is, you know, very material to a patient’s daily life. So if you’re improving that, that’s very tangible benefit.

And payers like tangible benefits. So we’ve we’ve done quite well from a pricing and reimbursement perspective with with Marley, and we expect to do well here too.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Great. And then maybe just in the last few minutes, can you tell us a little bit more, give us the latest on the Fragile X program, what you see as the potential advantages of your agent, and what you’d be looking for, to learn out of Shinobi’s upcoming phase three?

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Yeah. I’m, you know, very excited about this program. This is with MRM three thousand three seventy nine. It’s a PD-four d inhibitor, and we we are planning to start a phase two study, later this year, you know, actively very active engagement with the FDA, which which has been great. So I have seen no disruptions on that front.

So that program’s on track. And why we’re excited about it is this is the, you know, one mechanism that has actually been shown to work in in fragile X, particularly on the cognitive aspect of the disease. So we reference, the Shinobi program as part of our rationale for moving forward with this deal to bring this asset in. They have a they have a PD-four d inhibitor, positive phase two, showed clear benefit on an endpoint called the NIH toolbox and assessments of cognitive impairment. At a three month endpoint, they’re taking that into phase three.

That’s in phase three now and should should read out actually towards the end of the year. Our compound is a bit different, from theirs in that it’s got higher brain penetration. And so the one advantage of that is that you may be able to deliver more PD four d where you need it. So improve on the efficacy profile potentially while also improving on the tolerability profile. Systemic exposure of PD-four d at some point leads to, dose limiting, tolerability.

So emesis is particularly what, this can trigger at some levels, and you see that with the pan PD fours, out there. So a higher brain penetration can basically enable more effective dosing, and that’s what we’ll

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: be exploring in the phase two. Good. Makes sense. And then maybe just in the last two minutes, can you remind us your IP position across your portfolio and then maybe also work in how the tablet formulation might impact, Marley?

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Yeah. You know, as Eric mentioned, for Lipmarly, we have IP to twenty forty, and this is, orange book listed, dosing patents, which, you know, we, you know, we’re we’re very excited about. The tablet formulation that was, just approved, that also had IP grants, I believe last week, to 2043. And that was, not a trivial exercise to turn it from a liquid into a tablet. So, you know, quite happy with the portfolio that we’re building and we’ll continue to build.

Velixibat will, likely fall under that same 2,040 dosing family. This kind of goes back to the comments I made earlier about the dosing finding that higher is better. That’s actually a novel finding and quite important to establish effective dosing ranges for IBAT. And then for Citexly, there, with this approval, we’ve been granted seven years of orphan exclusivity. So, kind of think about that as seven years from, February.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Great. Well, we’re out of time. But, Eric and Andrew, thank you guys, so much. Really appreciate it.

Andrew McKibbin, SVP of Finance and IR, Miriam Pharmaceuticals: Thank you for having us.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Thanks again.

Eric Viarholt, CFO, Miriam Pharmaceuticals: Alright. Thank you.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: You guys being here.

Eric Viarholt, CFO, Miriam Pharmaceuticals: You too.

Brian Ebers, Senior Biotech Analyst, RBC Capital Markets: Yeah. Thanks for helping.

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