Mirum Pharmaceuticals (MIRM) investor relations material

Strategic overview and recent developments

• Operating as a global rare disease company with 430 employees and three approved medicines, including LIVMARLI, which has expanded from pediatric to adult populations.

• Two major clinical data readouts this quarter: positive pivotal data from the VISTAS study in primary sclerosing cholangitis and a phase II-B readout in hepatitis delta following the Bluejay Therapeutics acquisition.

• Announced a new business development (BD) transaction for an ultra-rare disease medicine, expanding the portfolio.

• BD strategy focuses on high unmet medical needs, conviction in biology, and disciplined deal selection, with recent deals spanning commercial to phase II-ready assets.

• Recent capital raise refinanced convertible notes at a 0% coupon, improving financial flexibility without altering BD approach.

Commercial performance and market dynamics

• LIVMARLI achieved $114 million in Q1 2026 net sales, up 55% year-over-year, with full-year guidance raised to $660–$680 million.

• Growth driven by strong demand in PFIC, especially later-onset cases, and better-than-expected international sales.

• Alagille syndrome market penetration is about 50%, with continued steady patient additions; PFIC market is split 50/50 with competitor BYLVAY.

• Significant growth potential remains, particularly as adult providers increase genetic testing for PFIC.

• The EXPAND phase III study could further boost LIVMARLI, with potential for over $1 billion in peak sales across indications.

Pipeline progress and clinical milestones

• VISTAS phase IIb study in PSC met its primary endpoint, with full data to be presented at EASL; opportunity for volixibat remains over $1 billion.

• Study design elements, such as placebo run-in periods and patient training, have effectively reduced noise in pruritus trials.

• Interim results in the VANTAGE study for PBC are promising, with expectations to outperform competitors on pruritus improvement.

• Pricing strategy for volixibat will be determined after further data and competitor pricing are available, aiming to maximize access and value.

• Combination therapy with PPAR agonists is possible in PBC, with IBATs targeting pruritus where other therapies may not suffice.