Mirum Pharmaceuticals (MIRM) H.C. Wainwright 4th Annual BioConnect Investor Conference summary

Corporate strategy and growth outlook

• Focused on rare disease therapies, especially for liver and genetic disorders, aiming for sustainable, high-growth operations and shareholder value.

• Net sales have grown from $10 million five years ago to a 2026 guidance of $660–$780 million, with a target to return to cash flow positivity by end of next year.

• Capital allocation prioritizes investments in late-stage pipeline assets, notably brelovitug for hepatitis delta virus.

Pipeline and portfolio development

• Commercial portfolio includes LIVMARLI for cholestatic pruritus, with additional assets like volixibat and brelovitug targeting PSC, PBC, and HDV.

• Zilurgisertib for FOP genetics was recently in-licensed, leveraging existing commercial infrastructure for rare genetic diseases.

• Pipeline expected to deliver four registrational readouts in the next 12 months, including VISTAS, EXPAND, and VANTAGE studies.

Revenue drivers and franchise structure

• Business is organized into two units: liver diseases (LIVMARLI, volixibat, brelovitug) and rare genetic diseases (CTEXLI, CHOLBAM, zilurgisertib).

• LIVMARLI is projected to reach at least $1 billion in peak sales, serving as the portfolio's foundation.

• Volixibat and brelovitug each have significant commercial potential, with volixibat targeting billion-dollar peak sales and brelovitug estimated at $750 million or more.

• Zilurgisertib is expected to be a $200 million opportunity, with FDA priority review and a PDUFA date in September.