Mirum Pharmaceuticals (MIRM) Citigroup’s Biopharma Back to School Summit 2026 summary

Strategic focus and pipeline overview

• Focus on rare diseases with significant unmet needs, targeting small to mid-size medicines overlooked by larger pharma.

• Expected 2024 revenue of $680–$700 million, with multiple key clinical readouts and regulatory milestones ahead.

• Pipeline includes brelovitug for HDV, zilurgisertib for FOP, LIVMARLI for cholestatic pruritus, and volixibat for PBC and PSC.

Key clinical and regulatory milestones

• Phase III brelovitug HDV readouts expected this month and next quarter, supporting a BLA filing in H1 next year.

• Zilurgisertib PDUFA date for FOP is later this month, with approval and launch preparations underway.

• LIVMARLI EXPAND phase III readout in Q4 aims to broaden label for cholestatic pruritus indications.

• Volixibat phase III data for PBC expected in Q1 next year; pivotal intent confirmed with FDA alignment.

Market opportunities and differentiation

• HDV market estimated at 15,000 diagnosed and up to 40,000 total U.S. patients, with underdiagnosis addressed by reflex testing strategies.

• Brelovitug differentiated by single-agent, fully human antibody with strong virologic and ALT normalization responses.

• LIVMARLI expansion driven by improved genetic testing in adults and basket protocol for broader cholestatic pruritus.

• Zilurgisertib positioned for younger FOP patients (age 12+), unlike competitors limited to adults.