Mirum Pharmaceuticals, Inc. Common Stock H.C. Wainwright 28th Annual Global Investment Conference

Yeah, thanks for hosting. It is a busy year for Mirum, 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. To dive into first the strategy of Mirum and who we are, what led to the creation of the company about eight years ago, it was really started from a search to look for underappreciated opportunities in rare disease.

Our strategy as a company is to pull together these small to mid-size products that really fly below the radar of most of the larger companies. There are a lot of opportunities out there like this. In fact, across rare disease, probably only about 5% of those rare diseases have approved medicines.

And there's even opportunity to improve on those that have existing medicines. There's really a long list of opportunities that we look at and are excited about across the rare disease settings. Today in Mirum, as you covered some of the highlights, the way I describe it is we have a thriving commercial business based on three approved medicines, LIVMARLI being the real highlight of the growth- Yeah 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- Yeah complete diagnosis.

Behind that, across the pipeline, all of these really game-changing potential opportunities for new medicines. The first up is the PDUFA date for zilurgisertib, which would lead to the potential approval of zilurgisertib for FOP.

Yeah. We can talk about the launch profile there.

Really efficient add for our rare team. Then data readouts also starting later this month for bulevirtide with AZURE-1. AZURE-4 follows next quarter, EXPAND next quarter. VANTAGE for PBC, first quarter next year. It is pretty stacked right now with a lot coming on.

Yeah. Looking at LIVMARLI's profile and breaking down what we have seen over time. LIVMARLI's first approval was for cholestatic pruritus and Alagille syndrome, and followed with a label expansion in cholestatic pruritus due to PFIC. The profile for response for patients in both of these indications can be really quite profound in addressing the pruritus that is a hallmark symptom of these diseases. Both Alagille syndrome and PFIC can drive highly elevated circulating bile acid levels, and LIVMARLI interrupts the recirculation of those bile acids to reduce systemic bile acid levels. 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 are 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 is typically seen as a favorable trade-off for the itch benefit that patients see. How it differentiates from others, there is another IBAT inhibitor that is approved in the same indications, and I credit a lot to our clinical profile and our team's performance and the growth profile that LIVMARLI has been able to demonstrate, really quarter over quarter continuing 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 LIVMARLI. We've been spending a lot of time talking about the adult PFIC opportunity Yeah this year.

It's something that's emerged really over the past year and a half, where as we had the PFIC label expansion for LIVMARLI and were 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.