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On Monday, 08 September 2025, Mirum Pharmaceuticals (NASDAQ:MIRM) presented its strategic outlook at the Morgan Stanley 23rd Annual Global Healthcare Conference. The company, focused on rare diseases, shared both achievements and challenges. With revenue projections between $490 million and $510 million for the year, Mirum highlighted its expanding international presence and ongoing product development, while also acknowledging areas for improvement in patient identification.
Key Takeaways
- Mirum Pharmaceuticals is on track to achieve $490 to $510 million in revenue this year.
- LIVMARLI’s peak sales expectations have risen to over $1 billion, driven by label expansion.
- Volixibat’s VISTAS Phase 2b study in PSC has completed enrollment, with results expected in Q2 next year.
- International markets, notably Japan, are significantly contributing to growth.
- The company is leveraging AI for operational efficiency and focusing on expanding its genetic testing support.
Financial Results
- Revenue Projections:
- Anticipated revenue for the year is between $490 million and $510 million.
- LIVMARLI:
- Peak sales expectations have increased to over $1 billion.
- Approximately 40% of peak sales are expected from Alagille syndrome, with the remainder from PFIC and XPAND indications.
- CHOLBAM and CHENODAL:
- Q2 sales reached $40 million, an increase from $25 million per quarter at the time of acquisition.
Operational Updates
- LIVMARLI:
- Growth driven by recent label expansion to include PFIC, with enhanced patient identification.
- Flexible formulations, such as liquid solutions for younger patients, have boosted performance.
- International growth is robust, particularly in Japan through partner Takeda.
- Volixibat:
- VISTAS Phase 2b study in PSC has completed enrollment; results expected in Q2 next year.
- VANTAGE study for PBC to complete enrollment next year, with results anticipated in the first half of 2027.
- MRM-3379:
- Phase 2 study for Fragile X syndrome is underway, focusing on a CNS-penetrant profile.
- Global Reach:
- Products are reimbursed in over 30 countries through various distributors and partners.
Future Outlook
- Volixibat:
- Anticipated data from the VISTAS study in PSC in Q2 next year.
- VANTAGE study results for PBC expected in early 2027.
- XPAND Study:
- Enrollment is on schedule to finish next year.
- MRM-3379:
- Phase 2 study has commenced, targeting Fragile X syndrome.
- CHOLBAM and CHENODAL:
- Continued growth is projected for these bile acid replacement drugs.
Q&A Highlights
- Patient Identification:
- Mirum is enhancing support for diagnosis through genetic testing awareness.
- XPAND Study:
- The study is inclusive of both children and adults with various causes of cholestasis.
- Intellectual Property:
- Key patents, particularly the 2040 family, are crucial for LIVMARLI’s indications, with potential extensions to 2043.
- Regulatory Landscape:
- Interactions with the FDA remain constructive and direct.
- Artificial Intelligence:
- AI is being utilized for initial drafting of documents to improve team efficiency.
Readers are invited to refer to the full transcript for a more detailed account of Mirum Pharmaceuticals’ strategic insights and future plans.
Full transcript - Morgan Stanley 23rd Annual Global Healthcare Conference:
Mike Olds, Biotech Analyst, Morgan Stanley: All right. Good morning, everyone, and thanks for joining us at the Morgan Stanley Global Healthcare Conference. I’m Mike Olds, one of the biotech analysts here, and it’s my pleasure to introduce Chris Peetz, CEO from Mirum Pharmaceuticals. Before we get started, I just need to read a quick disclaimer for important disclosures. Please see the Morgan Stanley Research Disclosure website at www.morganstanley.com/researchdisclosures. If you have any questions, please reach out to your Morgan Stanley sales representative. With that, I’ll just turn it over to Chris to give us some introductory comments for people that might not be familiar with the story.
Chris Peetz, CEO, Mirum Pharmaceuticals: Hey, Mike, and thanks for having us. I’m going to kick off with, you know, also forward-looking statements to be made here by me. I’ll refer you to our SEC filings for full risk factors disclosure. A quick highlight of Mirum Pharmaceuticals. We are a rare disease-focused company. We have three approved medicines, on track for $490 to $510 million in revenue this year. Really strong growing commercial business and a very active pipeline in the near term here. Just this morning, announced completion of enrollment of the VISTAS Phase 2b study of volixibat in PSC and positioned that as one of three kind of potentially pivotal studies that we have coming up over the next 18 months, with the VISTAS PSC being the first one. Expect top-line data 2Q next year.
Behind that, a follow-up indication for volixibat in primary biliary cholangitis and label expansion opportunity for LIVMARLI with the XPAND study, and a MRM-3379 Fragile X program behind that. A lot going on in the company overall. LIVMARLI is really the highlight of the commercial performance, recently. With a recent label expansion from PFIC to second indication for LIVMARLI, we’ve seen a really nice step up in growth overall, driven by new patient finding, new patient diagnosis, and a really strong profile for LIVMARLI. Positioning it now, we see line of sight to a billion-dollar plus for the brand. Exciting moment for the company and really happy with how these high-impact medicines have been rolling out.
Mike Olds, Biotech Analyst, Morgan Stanley: Great, thanks for that introduction. There’s a lot going on. Congratulations on getting your study fully enrolled. I thought maybe we could start just with LIVMARLI. Obviously, that’s your revenue driver. You touched on this in your prepared remarks that you’re seeing some really nice growth there. A big part of that’s PFIC. Maybe just dig into that a little bit more and what’s happening there.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah. For LIVMARLI, PFIC is what’s new. I’d start with actually Alagille syndrome because this is a great base to the LIVMARLI product profile. What we’ve seen for LIVMARLI in Alagille syndrome since its approval is now a pretty steady pattern of new patient accumulation, really great performance in the U.S., and that continues. There’s a good baseline support from the Alagille syndrome business. Internationally, that’s really what’s driving most of the performance—still Alagille syndrome, with PFIC in its early days internationally. In the U.S., the PFIC dynamic, since we had that label expansion last year, we’ve actually been pleasantly surprised about how well we’ve done from a patient finding standpoint. Unlike Alagille syndrome, there’s probably a bigger underdiagnosed pool of patients with PFIC.
We put a lot of focus on getting conversations going about cholestatic patients that maybe haven’t had genetic testing, trying to get some of those diagnoses confirmed. It’s also worth noting that with our LIVMARLI’s flexible formulation, we have a liquid solution for younger patients and have recently approved a tablet that’s one tablet per dose for older patients. That’s played into some of the performance as well, where for a newly diagnosed patient, that liquid formulation is a little easier, and then also a simpler administration for older patients that might want a tablet.
Mike Olds, Biotech Analyst, Morgan Stanley: Yeah. Just for PFIC, in terms of identifying the patients, are you doing anything unique than what you’ve done sort of in the past with Alagille, for example, or is it just maybe you underestimated what the initial patient numbers might look like?
Chris Peetz, CEO, Mirum Pharmaceuticals: There’s a little bit of each of that, right? I do think we absolutely underestimated what was the actual patient population out there. For Alagille syndrome, it’s worth highlighting a difference in presentation where Alagille syndrome is typically, we think, better diagnosed and earlier diagnosed because of the multi-systemic nature of it. There are facial characteristics. They tend to be more jaundiced in younger age, and we do think they get picked up more regularly. The Alagille syndrome population is pretty well defined from what we’re seeing. For PFIC, which really is a simplifying term for a number of distinct genetic mutations in bile acid-related transporters, to say PFIC kind of oversimplifies what’s going on there. For some of the specific subtypes, there can be a later-onset presentation.
There are a lot more patients out there that maybe weren’t picked up in childhood and start to present with elevated bile acids, elevated liver labs, and itch later on in childhood or even in adulthood. That was kind of what we underestimated. What we’ve been doing proactively is much more supporting the diagnosis. We have a lot of this through a field medical team, have supported, I think, broader awareness of the availability of genetic testing, starting to get, in particular, some of the older patients, the why behind testing for the adult hepatologists. We’re seeing some of the impact of that.
Mike Olds, Biotech Analyst, Morgan Stanley: Makes sense. You also mentioned the strength in your international markets. Maybe talk about some of those markets you’ve recently launched, and then looking ahead, what additional markets you’ll be looking out for.
Chris Peetz, CEO, Mirum Pharmaceuticals: International performance has played out nicely. I mean, it’s still, as mentioned, early days because what we’re seeing to date is largely Alagille syndrome-driven. A note on how we approach the international commercialization. You know, we’re direct in a shorter list of countries, so Canada and Western Europe. That’s where it’s Mirum people on the ground, so we call those our direct markets. Beyond that, we get closer to global reach using distributors and partners, so kind of the partner markets. What we see in the direct markets really echoes a lot of what the U.S. is seeing for Alagille syndrome. Nice steady growth over time and new patient starts. In distributor markets, we continue to get access in new countries. We’re now over 30 countries with reimbursed product. A real highlight from the second quarter, first and second quarter, frankly, is Japan.
Our partner Takeda is off to a great start with their recent approval. That was a big factor in our Q2 numbers, seeing some of their first orders get recognized. More to come from the international rollout as we get more distributors up and rolling. Ahead, we’ll have a PFIC expansion as well for the international markets.
Mike Olds, Biotech Analyst, Morgan Stanley: Great. Maybe we can just flip back to Alagille syndrome for a minute in the U.S. and kind of that’s been sort of your initial launch there. It’s been several years. You keep growing that. Where are you in that process and, you know, how much room is left, I guess?
Chris Peetz, CEO, Mirum Pharmaceuticals: When we’re thinking about the patient population here that we think LIVMARLI can be applicable for, there’s both the prevalent pool of patients that, you know, as mentioned, we do think are pretty well diagnosed. We think we’re probably about half penetrated into that prevalent pool of patients. On top of that, new diagnoses every year, call it about 100 is what is typical new Alagille syndrome cases diagnosed. There’s about 100 infants that present any given year. We think we do even better than that 50% because the treatment decisions are just a lot more straightforward, frankly, at that new diagnosis time point. The question that always follows is like, where is this going? How high does that 50% go? We certainly see it moving higher. We do continue to see new starts in the prevalent population, even this far into the launch.
What’s behind that that we’ve talked about before is, you know, the physician-patient conversation and decision on who’s a patient eligible for treatment and who’s a LIVMARLI treatment candidate. A lot of that’s a somewhat subjective conversation about what is the burden of my pruritus, what is my symptomatic burden that I’m dealing with. There’s a lot to unpack to get those conversations to happen, from both the physician side and from the patient or family side. That’s how we see that 50% continuing to go higher over time.
Mike Olds, Biotech Analyst, Morgan Stanley: Makes sense. In your sort of introductory comments, you sort of mentioned you recently increased your sort of peak sales expectations for LIVMARLI to $1 billion plus. Maybe you can just talk about, you know, maybe the different components of that. I don’t know if you’re willing to go there, but the breakdown of those components.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah, happy to give a very high-level picture of it.
Mike Olds, Biotech Analyst, Morgan Stanley: Sure.
Chris Peetz, CEO, Mirum Pharmaceuticals: Not something that we’ve really kind of detailed out by indication. The three big pieces here, Alagille syndrome, which we now have clear line of sight, steady trends, kind of has a good view of where that’s going. That’s certainly the biggest component from how we look at it. Call that roughly 40% or so of where we see the peak getting to. The other two pieces probably somewhat equivalent as well, so between PFIC and the XPAND indication. Though, we’ll note that we’re still learning as we go on those two pieces. PFIC, as noted, there’s been more out there than we initially thought. For XPAND, we’re starting trying to be pretty conservative in terms of what that population is. It’s a long tail of different causes of cholestasis.
Initial kind of performance from the clinical study enrolling and conversations with investigators and physicians, it’s at least the size of PFIC in terms of patient population.
Mike Olds, Biotech Analyst, Morgan Stanley: In terms of the XPAND basket study, if you can just sort of remind us, you know, the rationale there and how you sort of had in that direction for those rare conditions.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah, it’s an interesting program. The XPAND study is a basket study, and there are several different causes of cholestasis. Different diseases or post-surgical complications or implications cause cholestasis, and with that comes elevated systemic bile acids, the pruritus that shows up in Alagille syndrome and PFIC. It’s quite similar in how it develops in these other causes of cholestasis. We were initially seeing a lot of just compassionate use demand for patients that kind of fit in the long tail. These could be older biliary atresia patients, and that’s a big portion of it. Secondary sclerosing cholangitis, a number of kind of more rare occurrences of cholestasis that are secondary to other diseases. Given the volume of compassionate use that we saw, the FDA actually requested us to put a protocol together around it.
That kicked off a conversation to develop this protocol and strategy to gather these otherwise hard-to-study indications because each one on its own is just too rare to run a standalone study. That allowed us to put them into a pooled population. Call it at least 500 patients in the U.S. that fit the criteria that we have here and on track to complete enrollment next year.
Mike Olds, Biotech Analyst, Morgan Stanley: Okay. Is enrollment tracking sort of as you expected, or is it faster maybe?
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah, as expected. I mean, in terms of what we’re seeing out there, the patients are there, and it’s really about getting sites open and the families engaged with the clinical trial. It is a study that’s open to children and adults. Worth noting that the focus analysis and the real primary endpoint is in the pediatric patients, though we do see adult interest in it as well. Our screening and enrolling adult patients into it. That’s the target 45 patients that we talk about is in the pediatric population.
Mike Olds, Biotech Analyst, Morgan Stanley: Gotcha. Maybe just last question on LIVMARLI before we move on. Maybe just touch on, you know, current IP, or is there more IP coming that we should be looking out for? What’s the status there?
Chris Peetz, CEO, Mirum Pharmaceuticals: For IP, we point to a key family of patents, the 2040 family, that are directed towards LIVMARLI indications and dosing. There is more around that, including, you know, formulation and some other applications that extend a bit beyond out to 2043. As we look at it, we plan on 2040 as really the key patent family.
Mike Olds, Biotech Analyst, Morgan Stanley: Okay. Great. Maybe we can move on to just the bile acid portfolio. You had two programs there. You added them a few years ago, and you’re seeing some nice growth. Maybe just give us a little background and kind of what’s driving the growth there.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah. So CHOLBAM and CHENODAL are the two bile acid replacement drugs that we acquired a couple of years ago now. We’ve been able to do quite a bit with them, really across both brands on identifying patients, as well as the real highlight of the work we’ve done has been getting to the approval of CHENODAL. A lot of great work started from Travere, the company that we acquired these from. We were able to go through the final NDA to get CHENODAL a formal label for the treatment of CTX. What we’ve been doing to grow these products has been execution on patient finding for CTX in particular, pretty substantially underdiagnosed. We’ve been iterating on different programs on supporting genetic testing in different specialties. These patients do present in a number of different settings. Generally, it can be quite advanced into adulthood before they’re found.
That’s where we’ve been seeing early traction. Expect it to be a continued build over time as we iterate through some of these different programs. Excuse me. We’ve done really well. When we had our first quarter with these, they were about $25 million a quarter, and recently, the Q2 was at $40 million. A lot of good impact over time and see it growing and continuing to grow into the future.
Mike Olds, Biotech Analyst, Morgan Stanley: Yeah. Definitely making nice progress there. Maybe now we just shift to volixibat and maybe just start with PFIC since that’s sort of more advanced in development. Maybe talk about the market opportunity there and the unmet need.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah. So volixibat for primary sclerosing cholangitis, PSC, really exciting program. PSC is one of the larger adult cholestatic indications. Historically, it’s been really challenging from a drug development standpoint where there’s not really been an actionable surrogate endpoint. It’s a lot of work on trying to find different alternatives. Unlike some of the other liver settings, there isn’t as predictable of serum measures that you can use for drug development. What we’ve done with the approach for volixibat is to target symptoms, and that actually gives us an actionable near-term outcome that can be used for registrational purposes. The VISTAS study for volixibat in PSC has a primary endpoint of pruritus in patients with PSC and is designed to use that as the registration pathway, similar to what we’ve done in the pediatric setting.
How that maps on to the patient population and the impact we can have there, probably about 30,000 PSC patients in the U.S. Those that have it, it’s probably about two-thirds of that. When you start to have conversations to understand what the impact of this means for patients, it’s a potential game changer for them. The day-to-day burden of itch and fatigue as well is another one of the symptomatic burdens that’s a real highlight in this setting, the day-to-day burden of this disease that these patients are facing. Feedback we’ve gotten from response stories from volixibat and LIVMARLI as well, I should note, has some case studies. Feedback has been very positive on what it means for the patients day-to-day.
Mike Olds, Biotech Analyst, Morgan Stanley: You mentioned coming up with an endpoint for this disease has been a bit challenging. You’ve focused more on the symptoms, and you’ve seen some nice impact there. From a regulatory perspective, are there any issues or hurdles you need to clear to make that an approvable endpoint, or are we there already?
Chris Peetz, CEO, Mirum Pharmaceuticals: It’s a pretty straightforward approach. Using an itch endpoint with the 0 to 10 NRS scale, that’s what the itch-row scale is in this study. It’s a pretty typical approach. There’s the standard validation work that goes into any PRO endpoint, and that’s all baked into our program. While it’s novel for PFIC, it is otherwise a pretty standard approach to using itch as an outcome.
Mike Olds, Biotech Analyst, Morgan Stanley: Got it. Makes sense. I think it was last year, you gave some sort of interim analysis. Maybe walk us through that and kind of how to think about that.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah. The interim analysis design for VISTAS is part of what makes us so excited about where we’re at now. I’ll tie in a little bit of the VANTAGE study here because these were designed really to help inform each other. Last year, we conducted interim dose-selecting analyses for both of these studies. For the VISTAS study in PSC, it was designed to help the independent monitoring committee follow an algorithm to make a dose selection decision and keep the study blinded, assuming all was within the target effect size. For the VISTAS study, interim analysis stayed blinded. There was a potential outcome there where the effect size was close to the boundary that we had designed. There could have been a recommendation to upsize. That didn’t happen, which makes us feel confident on where we’re at.
That was designed to be predictive of a positive outcome for the overall study. It is really kind of an interim analysis designed to give us confidence in the final primary data that we’re going to see next year. For VANTAGE, however, because PBC is a more common indication and we’re targeting overall a larger safety database, we could unblind that data. The VANTAGE study had an unblinded interim analysis that was announced and subsequently presented. That gives us confidence in the dose selection as well. Both of these selected the lower dose to move forward. The unblinded VANTAGE analysis, we think, helps us understand what was potentially happening in the VISTAS study.
Mike Olds, Biotech Analyst, Morgan Stanley: Have you mentioned what the threshold was in VISTAS for the interim on pruritus?
Chris Peetz, CEO, Mirum Pharmaceuticals: It’s designed as a threshold based on the statistical effect size. Think of it more as a curve. We haven’t disclosed what the exact numbers are, but it’s basically a curve looking at the placebo-adjusted difference and the variability within the dataset. With that curve, there was a boundary area. We know we were above the boundary area.
Mike Olds, Biotech Analyst, Morgan Stanley: Okay. Maybe just on pruritus in general, what’s considered a meaningful difference? Remind us what you showed in the VANTAGE.
Chris Peetz, CEO, Mirum Pharmaceuticals: It is measured on, as mentioned, a 0 to 10 scale. It is expected that a 2-point change would be clinically meaningful. In the VANTAGE analysis, we saw a 3.8 change from baseline, a really strong clinically meaningful effect coming out of that interim analysis.
Mike Olds, Biotech Analyst, Morgan Stanley: Great. Maybe just picking with VISTAS, you completed enrollment, which you announced today, and now you’re heading towards data in the second quarter of next year. Maybe just talk a little bit about what to expect from that readout and what you’re looking for and what’s a positive readout in your view.
Chris Peetz, CEO, Mirum Pharmaceuticals: The study is designed to really look at the pruritus as the primary outcome. As mentioned from looking at VANTAGE, we think that this dose level is highly active. We’re confident that we’ll be seeing these, you know, effect size that’s going to be meaningful for patients. The analysis was really just looking at placebo-adjusted difference on that itch score so that the top line expect to be announcing that. From the tolerability profile, at this point, we have a pretty good handle on what IBAT looks like in clinic. The most common treatment-related effect is actually mechanistic, right? There is diarrhea related to flushing the bile acids out of the body. We found that if you’re coaching and helping patients expect it, it can be very manageable. It’s not something that we see as a real barrier, in particular, if there’s good awareness around it.
That’s another thing we’ll be looking at is the headline kind of GI adverse event rates.
Mike Olds, Biotech Analyst, Morgan Stanley: Okay. Maybe just shifting to PBC and maybe just similar questioning, you know, market opportunity there, unmet need.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah. For PBC, it’s a much larger indication, but it’s busier from a competitive standpoint. Where PFIC, because of the endpoint issues, there are no approved therapies. In PBC, there has been a surrogate using alkaline phosphatase as an endpoint for recent approvals. To break it down, we think about in line with therapy. Probably about 100,000 patients in the U.S., roughly, is the rounded way to make the math pretty easy to follow. Of that 100,000, maybe a third or 40% have progressive alkaline phosphatase levels, so they’re eligible for some of these more recent approvals of seladelpar and elafibranor, the PPARs that were recently out there. The volixibat’s role really goes across all of these lines of treatment. You see pruritus in PBC really happen at similar rates in both first line and second line.
We kind of are approaching the market segmentation quite differently from the recent approvals. That means that in the first line setting right now, there’s no other approved therapies. We have one other IBAT that has a PDUFA date next year, so there’ll be one other product out there that we can sort of touch on, the relative positioning. The IBAT role in PBC really cuts across all lines of therapy. Similar to the other indications, probably about two-thirds of patients have pruritus. That’s nearly 60,000 patients, roughly, is the target market for launch.
Mike Olds, Biotech Analyst, Morgan Stanley: For the VANTAGE study, can you just remind us the current status there in terms of where you are in enrollment and kind of timelines around that?
Chris Peetz, CEO, Mirum Pharmaceuticals: Yep. Expect to complete enrollment next year. That would put us on track for top-line data in the first half of 2027. We’ll look to try to refine that as we get closer, but that’s the rough guidance that we have at this point. The study conduct’s going well. As mentioned, for PBC, we’re looking for a bigger total safety database, and it just takes a little bit more time than the VISTAS study to enroll to get to that target patient number. I point that out because the size of the study really is not about efficacy powering, right? As we saw from the interim analysis, it’s highly significant, even with only a 30-patient analysis. This is really about sizing up for the safety database.
Mike Olds, Biotech Analyst, Morgan Stanley: Makes sense. Maybe you can touch on the competitor in the space or the recent data we’ve seen and kind of how you’re positioned there.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah. The other IBAT that’s been developed in PBC is coming out of GSK. They have a PDUFA date next year, the first half of next year. What they saw from their Phase 3 data really looks like an IBAT, right? They’re seeing improvements in pruritus. Their approval strategy and kind of patient population is really similar to kind of how we’re approaching it. From what we’ve seen from their dose selection, they did not take their highest dose forward. I think that really shows up in the headline effect size. The VANTAGE study in its interim analysis, the effect size we saw there was just really striking. I think that puts us in a position, assuming it holds up in a similar range in the final data set, to be a fast follower with a much stronger effect size.
Mike Olds, Biotech Analyst, Morgan Stanley: Yeah. Makes sense. Maybe we can just shift quickly to your MRM-3379 program. Sorry, maybe just talk about that a little bit, you know, how it’s differentiated and kind of next steps there.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah. MRM-3379 is the PD4D that we’re starting a phase two study now for Fragile X. Really unique program here. We’re going to come back and talk about endpoints as kind of what’s got us excited about diving into this program. In Fragile X, you know, there have been a number of different studies, largely looking at behavioral tools, so surveys from the caregivers. Those studies, we think, in large part because of the endpoint, have had a lot of noise in the final datasets. There was a key dataset that actually came out of Shionogi from a competing program where they used a cognitive tool called the NIH Toolbox that was really designed for settings of cognitive impairment. It adjusts to provide a scale for the level of where the patient’s at. That resulted in a pretty striking phase two result for their program.
We think the MRM-3379 program has a differentiated CNS-penetrant profile, so gets more of the drug where you want it to be, and so compelling profile compared to that program, looking to play that out in the phase two study.
Mike Olds, Biotech Analyst, Morgan Stanley: Could you quickly remind us where you are in the Phase 2? Has it started yet, or when were you started?
Chris Peetz, CEO, Mirum Pharmaceuticals: We just cleared the IND. After in-licensing the program, we did some work on having formulation and drug product ready to go, conversation with FDA confirming their view on endpoints, what they want to validate this NIH Toolbox. Feel great about that being the viable registration endpoint. I want to have all that squared away before we started the study. Kicking it off now. It’ll be three doses versus placebo to do that dose-ranging work to understand where we’re at on playing out this, we think, the stronger drug profile for MRM-3379. Want to make sure we do the dose-ranging work to figure that out.
Mike Olds, Biotech Analyst, Morgan Stanley: Makes sense. Maybe in the last few minutes, we can talk about some of these macro issues. We’re asking a couple of questions to all our companies at the conference. Maybe if we just start at the top with the first question, China’s rise in biotech innovation, how are you thinking about your competitive position here? Will this influence your R&D or BD strategy?
Chris Peetz, CEO, Mirum Pharmaceuticals: Overall, it seems most relevant for BD strategy for us, where you know the more potential drug candidates out there, and that’s our business model, is to go find kind of underappreciated programs and maybe reposition a mechanism or find something where the dataset was misunderstood that we can take it forward. I’d say from rare disease competition, we haven’t seen as much of that out of China. Those companies have been more focused on bigger indications. It doesn’t mean that might not happen over time. What the result of that is, you know, more product candidates that are available for us to go try to bring into the company.
Mike Olds, Biotech Analyst, Morgan Stanley: Maybe next question, how are you currently leveraging, you know, artificial intelligence or thinking about AI’s future disruption potential in your business?
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah. To all the Mirum employees out there listening, you know, make sure you’re logging in and using it. We’ve been trying to roll it out or really a lot of kind of first-round drafting of documents. You know, as all the work that we’re doing here, there’s a lot of writing on the regulatory side, on data review, on analytics. Having some of these tools as a first pass to add efficiency, we think will help us out quite a bit. It’s really about adding efficiency for the team we have. We’re ambitious about bringing in more programs and doing more. It’s not necessarily that we’re trying to replace people’s jobs. We’re just trying to make our strong team even stronger.
Mike Olds, Biotech Analyst, Morgan Stanley: Gotcha. Maybe last question, just, you know, what’s been most impactful for Mirum Pharmaceuticals on the regulatory side in terms of, you know, would it be FDA? Would it be MFN if you have any impacts there or tariffs?
Chris Peetz, CEO, Mirum Pharmaceuticals: Mostly, it’s been about how much time we have to answer questions about it. I think that’s been the biggest impact for us so far. Just from how our business is set up, I feel like we have somewhat less exposure on a lot of these topics. We certainly track them. For the FDA, interactions continue to be constructive, and direct. Our teams that we deal with are all there. We’ve had two approvals and a recent interaction on multiple programs. That’s all gone smoothly. No impacts there. On the other ones, MFN, I think hard to tell where that one goes. Our relative pricing in the European markets is really not that far off of where our U.S. government prices already, frankly. Overall, I think we’re pretty well positioned.
Mike Olds, Biotech Analyst, Morgan Stanley: Okay. Great. Looks like we’re just about out of time. Why don’t we end it there, Chris? Thanks so much. Appreciate it.
Chris Peetz, CEO, Mirum Pharmaceuticals: Yeah, thanks for the time.
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