Mirum Pharmaceuticals, Inc. Common Stock 12th Annual Cantor Fitzgerald Global Healthcare Conference

Yeah. Thanks for hosting and happy to jump in. Mirum, you are talking about by design, a bit about our kind of mission and the business strategy on how we came to be really is based around executing on finding overlooked rare disease assets, bringing them into a platform like Mirum, where we can focus on really high-impact medicines that otherwise get missed by larger companies. The small to midsize products that are overlooked by those that are chasing some of the larger indications like weight loss, for example. I see this as really a high impact and sustainable business model. Put it in the context of all the rare diseases, probably only 5% actually have an approved medicine, and even those with approved medicines have room for improvement. We think there is a long stretch of impact that we can have with that model.

To date, we have now grown the commercial business to we are getting towards $680 million to $700 million for the year this year, across three approved medicines. We have a lot more coming. We have a PDUFA date for a recently acquired program called zilurgisertib for FOP later this month, so that is the next potential launch in front of us. We have potentially pivotal data from a PSC program that we are discussing with FDA, working towards a potential NDA in the first half of next year. It has been a big discussion point for us, so I am sure we will spend some time on it today. Other phase III readouts very near term. brelovitug for hepatitis delta virus with a phase III readout this quarter and a second study next quarter.

A life cycle indication for LIVMARLI, one of our commercial medicines, has a phase III readout in Q4, and then volixibat in PBC, a data readout in the first quarter of next year. It is a packed window for us right now. A lot going on clinically, commercially, and excited about everything that we have pulled together. One other comment I would make on kind of rolling this forward with all of these launches, we do see a real potential for a financial inflection point as we go through next year and into the years that follow, as the commercial contribution starts to come online from all of these new potential medicines.

Breaking down LIVMARLI and where it's approved and where we're taking it, current approvals are for cholestatic pruritus and Alagille syndrome and PFIC, and a third leg to the stool with EXPAND being the phase III that reads out later this year in other cholestatic pruritus settings. To date, what we've seen over the time since approval of LIVMARLI is in Alagille syndrome, there's been just a nice steady cadence of patient adds and growth over time as we both see patients grow on drug as well as new patient starts, new geographies coming online. Recent acceleration to that has been in PFIC, and we've been talking about this now for a bit over a year on how we found more PFIC patients out there than was originally understood. We recently put pen to paper on really coming to some numbers of estimating what that could be.

Largely in the adult setting is where it's, we think, pretty dramatically underdiagnosed. When you go to some of the adult hepatology or GI clinics, there's not just as much of adoption of genetic testing to date. That's starting to change, and what we're seeing is as that's changing, those patients that were previously diagnosed as idiopathic cholestasis actually have PFIC genetics. You're seeing that these insufficiently diagnosed patients actually can have a genetic diagnosis. A lot of those patients have the symptomatic burden that is a great fit for LIVMARLI. That's what's been this acceleration of growth that we've seen recently, and we're continuing to deploy against it. Expect to get further into that. We've estimated at about 2,000 adult patients that would be the addressable patient population.

The short answer, I don't think we're that deep yet. From the launch of LIVMARLI, which was initially focused on pediatrics, we are probably covering about the top decile of the adult hepatology and GIs who see liver disease, the top decile of those prescribers. Over the past year or so, we've expanded a lot of the medical education around genetic testing as one of the ways to build awareness of the presence of genetic disease in the adult clinics. What we're doing now, starting later this year, is expanding the commercial footprint as well. I think that's going to be a real driver of seeing how much of this 2,000 adult patient estimate we can address with LIVMARLI by actually having Mirum commercial presence in those more community GI clinics outside of what to date has really just been KOL-driven transplant centers.

I think a lot of the first groundwork comes from what we've been active doing already on the medical education side, and we've seen that start to be echoed with research efforts on understanding genetic cholestasis. That's been showing up at AASLD and EASL, some of the venues where you see a lot of the more research interest in understanding what do these genetic hits mean if they were to find them. I think that's putting a genetic test into the hand of a community prescriber. Interpreting those results is going to be one of the first questions. I think it's important to have a backdrop of research and publications on how to interpret those results, as well as the support system of other prescribers who have familiarity with it that could be referenced or called on.

That's a big part of building the familiarity comfort to test. I think the other piece we also already have, which is you have an approved medicine that can help address a need that some of their patients have. Where you find these patients is those that already have a diagnosis of idiopathic cholestasis. They're in care. There's really not a satisfactory diagnosis, and so there is a desire to refine the diagnosis, and if there's a treatment there for it, we think that is the pull as to why you should consider genetic testing. All those pieces pulled together, knowing what to do with the test, access to the test, which we provide support for, and then a medicine to change an outcome for a patient if you do find a diagnosis.

I think there is potential parallels here. When we have designed EXPAND, the primary cohort is in pediatric patients. That is where a lot of the initial interest came from. We have had compassionate use case studies and some of the prescriber interest and demand that has driven the EXPAND study design that we have talked about before. We also have some adult patients enrolled in the study that frankly we focused on pediatric centers for the study, so it is not fully representative of what I think that profile could look like. Just back to how you are framing the question, we have sized this around the pediatric markets, but there is undoubtedly demand in the adult setting. I think it ties into, again, some of the efforts with PFIC on trying to refine diagnosis in the idiopathic cholestatic patients.