Mereo Biopharma Group (MREO) 43rd Annual J.P. Morgan Healthcare Conference 2025 summary

Strategic focus and pipeline overview

• Focus on unlocking deprioritized pharma assets for rare diseases, with two lead programs: setrusumab for osteogenesis imperfecta (OI) and alvelestat for alpha-1 antitrypsin deficiency (AATD), both targeting prevalent rare diseases with high unmet needs.

• Setrusumab is in phase III for OI, with Mereo retaining European rights and a partnership with Ultragenyx; alvelestat is phase III ready for AATD, with ongoing partnering efforts.

• Both programs benefit from strong prior investment, enabling rapid development and a capital-efficient, outsourced operational model.

• Cash runway extends into 2027, supporting key milestones through 2025.

Clinical progress and data highlights

• Setrusumab demonstrated significant increases in bone mineral density (BMD) and a 67% median reduction in annualized fracture rate in phase II OI studies.

• Phase III studies (ORBIT and COSMIC) are fully enrolled, with interim and final analyses scheduled for 2024; primary endpoint is annualized fracture rate.

• Alvelestat showed 50% reduction in exacerbations in pooled phase II data for AATD, outperforming current standard of care (augmentation therapy).

• Phase III for alvelestat will target both early and late-stage patients, with independent primary endpoints for FDA (SGRQ) and EMA (lung density by CT).

Regulatory and market access strategy

• Setrusumab has received multiple regulatory designations (orphan, PRIME, PRV), and is part of the EUnetHTA pilot for rare disease therapy access in Europe.

• Alvelestat recently recommended for orphan drug designation in Europe after demonstrating potential superiority over standard of care.

• Early and ongoing engagement with patient organizations and HTA bodies to support reimbursement and market access.

• Targeted commercial strategy in Europe, with a focused in-field force and robust data collection on disease burden and treatment outcomes.