Mereo Biopharma Group (MREO) Jefferies 2024 Global Healthcare Conference summary

Program updates and clinical progress

• Lead program Setrusumab targets osteogenesis imperfecta, with two phase III studies (ORBIT and COSMIC) now fully enrolled; phase III data expected in 2025.

• Mid-year 2024 will see 14+ month phase II data from the ORBIT study, with 23 of 24 patients continuing treatment.

• Alvelestat, an oral neutrophil elastase inhibitor for Alpha-1 antitrypsin deficiency, has shown promising results and is supported by recent external data in bronchiectasis.

• Validation work for Alvelestat's patient-reported outcome measures is complete, with a phase III IND update to be submitted to the FDA by month-end.

• Cash position at end of Q1 was $49 million, not including future deal income or Alvelestat phase III costs.

Commercialization and market opportunity

• Setrusumab is partnered with Ultragenyx, with $245 million in milestones remaining and tiered double-digit royalties outside Europe; Europe is owned outright with a flat royalty paid to Ultragenyx.

• Patient identification in Europe has found 10,000 potentially treatable patients in key markets, with expansion ongoing.

• Early access program in France will allow pre-approval revenue generation and early treatment catalyzation.

• Commercialization strategy in Europe involves a field force of about 70, excluding Central Eastern Europe, which will be managed via distributors.

• Market opportunity is estimated at $1.5–$2 billion globally, using analogs from similar rare disease launches.

Regulatory and payer engagement

• Early and ongoing engagement with European HTA bodies and payers since 2018 aims to streamline pricing and reimbursement, using fracture as a key endpoint.

• EUnetHTA process is leveraged to accelerate access and ensure equitable treatment across Europe, with Crysvita-like pricing as a base case.