Mirum Pharmaceuticals, Inc. Common Stock 2026 Q2 Earnings Call

Please go ahead. Thank you, Alexandra, and good afternoon, everyone.

I'd like to welcome you to Mirum Pharmaceuticals' second quarter 2026 conference call. I'm joined today by our Chief Executive Officer, Chris Peetz, our President and Chief Operating Officer, Peter Radovich, and Eric Bjerkholt, our Chief Financial Officer. Lara Longpre, our Chief Development Officer, will be joining us for the Q&A portion of the call. Joanne Quan, our Chief Medical Officer, could not be with us today due to a family matter. Earlier today, Mirum issued a press release announcing the company's results for the second quarter of 2026. Copies of the press release and our SEC filings are available on the Investors section of our website.

Before we start, I'd like to remind you that during the course of this conference call, we will be making certain forward-looking statements based on management's current expectations, including statements regarding Mirum's programs and market opportunities for its approved medicines and product candidates and financial guidance. These statements represent our judgment and knowledge of events as of today and inherently involve risks and uncertainties that may cause actual results to differ materially from the results discussed. We are under no duty to update these statements. Please refer to the risk factors in our latest Form 10-Q and subsequent SEC filings for more information about these risks and uncertainties. With that said, I'd like to turn the call over to Chris.

Chris? Thanks, Andrew, and good afternoon, everyone.

At Mirum, we're growing a rare disease leader focused on delivering high-impact medicines for often-overlooked diseases. This quarter demonstrates continued progress with strong commercial execution on our approved medicines as we head into the potential launch of our fourth commercial medicine later this year. We have a busy pipeline with multiple pivotal readouts in the quarters ahead, all delivered with a strengthened capital structure and overall financial performance, giving us greater capacity to invest throughout the business. In the second quarter, our commercial business generated $176 million in net product sales, reflecting strong demand across the portfolio and excellent execution by our team. Based on this performance, we are increasing our full year 2026 net product sales guidance to $680 million-$700 million.

Fueled by the strong commercial performance, we're driving towards the next phase of Mirum's growth with multiple milestones over the coming months. Our next commercial milestone will be the potential launch of zilurgisertib for FOP with a PDUFA date next month. This is fast progress for a program added to our rare genetic business only in the second quarter. Peter will cover more of the launch profile in his remarks. Moving to the pipeline for our rare liver business, it's important to spend some time today on volixibat and PSC, which just had some key U.S. regulatory interactions. First, as a reminder of the background of the VISTAS study of volixibat and cholestatic pruritus in PSC, we designed this adaptive study with input from the FDA as a pivotal trial for this difficult clinical setting, including alignment on study duration, endpoints, and analysis plan.

As we've announced previously and presented at EASL this year, VISTAS met its primary endpoint, showing highly significant improvement in pruritus in the primary cohort, with consistent significant results also observed in a second cohort of patients with milder baseline pruritus. We are excited to share that the FDA has now granted breakthrough therapy designation for volixibat in cholestatic pruritus due to PSC based on these strong results. We see this as recognition of the potential for volixibat to address a serious unmet need in PSC. As planned, we recently held a pre-NDA discussion with the agency about the submission of an NDA based on the VISTAS study. In the meeting, the FDA recommended conducting a Phase III study.

We believe the VISTAS study provides a robust and clear data set to characterize the use of volixibat in patients with pruritus due to PSC and is a clinically and statistically highly persuasive study. VISTAS is the largest randomized clinical study conducted in patients with pruritus due to PSC, with an extensive overall data package that includes more than 180 PSC patients randomized, one-year safety exposure data for over 100 PSC patients and growing, results from an independent committee evaluating liver safety, all totaling over 600 subjects across the clinical program to date. While we are not currently aligned on the NDA submission package, we will be engaging in discussions with the FDA on how to further supplement our planned submission based on the VISTAS study. This engagement will delay the planned timing of our NDA submission, which we are now targeting for the first half of next year.

We're positioned to move quickly once we have further clarity from the agency. We'll provide updates as we work towards our goal of bringing a much-needed therapy to this unaddressed clinical setting. In parallel, the VANTAGE study in PBC is progressing well and has completed enrollment, reaching over 330 patients randomized. In PBC, our earlier breakthrough therapy designation has enabled more dialogue with the agency during the conduct of the study. We have recent feedback from FDA for VANTAGE to serve as a pivotal study of volixibat in pruritus due to PBC if the study is successful at its first-quarter top-line readout next year. Our next clinical readout for the rare liver business is expected to be brelovitug as AZURE-1 top-line results later this quarter. This is the readout of the Phase III portion, following strong results of the Phase IIb portion earlier this year.

We also continue to expect the year four data in the fourth quarter, which keeps us on track for a potential BLA submission for this breakthrough therapy-designated program in the first half of next year. Rounding out the rare liver pipeline highlights, the phase III EXPAND study of LIVMARLI in additional rare cholestatic conditions remains on track for top-line data in the fourth quarter. Putting this all together, we are advancing these clinical programs from a position of financial strength. Our commercial business continues to generate meaningful cash, providing the capacity to invest in potential launches, clinical development, and opportunistic business development where we see a compelling strategic fit and the potential to create value. I am proud of the team's progress and the promise of our current medicines and pipeline, and I am excited about what lies ahead for Mirum.

With that, I will turn the call over to Peter to discuss our commercial performance and launch readiness in more detail.

Peter? Thanks, Chris. The second quarter was another strong quarter for Mirum's commercial business, with total net product sales of $176 million.

LIVMARLI and the bile acid medicines both continued to perform well, and based on the demand we see, we are increasing our full-year 2026 net product sales guidance to $680 million-$700 million. Second quarter net product sales for LIVMARLI were $129 million, with the U.S. contributing $92 million. Alagille growth remains durable, supported by continued new patient starts, sustained persistence on therapy, and weight-based dose increases. PFIC continues to be an important driver of growth, fueled by new diagnoses. We are particularly encouraged by the growing contribution from adult PFIC patients. We are seeing an increase in prescriptions from adult liver providers as awareness of later-onset PFIC grows and genetic testing becomes more routine.