Mirum Pharmaceuticals, Inc. (NASDAQ:MIRM) Q1 2023 Earnings Call Transcript

Chris Peetz: Hi, thanks for the question. We — the 600 number really we see as a really strong statement on familiarity with and interest in prescribing LIVMARLI. That number comes from all sources over the history of the program for patients with Alagille syndrome who have been prescribed or been part of the clinical study protocol. So it’s really kind of all sources that feed into that. Not really a number that makes sense to parse out to the — by country or by source. But what we can say is that across all of the different ways that patients had found their way to LIVMARLI clinical study, expanded access program, commercial products, compassionate use products in different formats. You do see the same profile of really high compliance, high persistence. Some of these patients six plus years staying with the program, because of the profound impact that it can have.

Unidentified Analyst: Thanks for the proper finishing . Perhaps sticking with Alagille syndrome. With the U.S. label expansion down to three months of age and above. Can you just tell us what were your expectations are both for top-line and also from a strategic standpoint as well?

Chris Peetz: Yes, on the label expansion for Alagille down to three months of age, really important step because that’s where most patients are diagnosed. So it allows LIVMARLI as a treatment option early on. Overall, top-line impacts. Keep in mind that these are younger, smaller patients, really important for the long-term view, but staying consistent with our guidance of 50% year-over-year growth in the U.S. And international, as we get further in, we’ll look at how we fold that into guidance as well.

Unidentified Analyst: Got it. And then perhaps switching gears to PFIC with the application submitted to the EMA in Europe. When should we expect your next milestone, including CHMP opinion perhaps?

Chris Peetz: Yes. So the PDUFA date we shared in December, excited about that, and the potential timing for an opinion would be — from CHMP would be late Q3 or in Q4.

Unidentified Analyst: Got it. Perhaps one final question. This one is financial. With the convertible offering growth in April, can you tell us what’s your estimated cash runway out to?

Chris Peetz: Yes. So similar to described in the prepared remarks, really are fully funded with the current business to cash flow breakeven and positive. So three plus years really meant to just be an indicator of the strength that we have there and see runway to operate the business for the long-term here and continue to grow the products.

Unidentified Analyst: Got it. Understood. Thank you so much. Thank you again for taking our questions. Looking forward to the Biliary Atresia data.

Chris Peetz: Yes. Thanks for the question.

Operator: Thank you. We have our next question comes from Brian Skorney from Baird. Brian your line is now open.

Unidentified Analyst: Hi, this is Luke on for Brian. Just a couple on EMBARK. Do you have an update on the scope of detail you might expect to include with the top-line readout beyond just bilirubin in particular or maybe any outcomes data? And then are you able to generally characterize the geographic dispersion of the enrolled patients in terms of U.S. versus other territories? Thanks.

Chris Peetz: So thanks for the question. And overall, on top-line, other than the primary, you can’t really speak to specifically what else would be in there other than getting material updates disclosed. The study is not powered for outcomes at that time point. So unclear if we would have anything to share on that point. And then geographic presence of sites really around the globe, and we’re seeing activity across all geographies, nothing really to point to there.