Mereo BioPharma Provides Update on Lead Clinical Programs

Clinical Programs Update - Setrusumab, a monoclonal antibody for Osteogenesis Imperfecta (OI), is progressing in its Phase 3 Orbit Study with the second interim analysis expected in mid-2025 and a potential final analysis in Q4 2025 [1][2] - The Cosmic study, an open-label Phase 3 trial comparing setrusumab to intravenous bisphosphonate therapy in patients aged 2 to <7 years, is ongoing with data to be evaluated alongside Orbit results [2] - Alvelestat, an oral neutrophil elastase inhibitor for Alpha-1 Antitrypsin Deficiency-associated Lung Disease (AATD-LD), received a positive opinion from EMA's Committee for Orphan Medicinal Products (COMP) for Orphan Designation, with a final decision expected in Q1 2025 [1][3] Regulatory Milestones and Designations - Setrusumab has received Orphan Designation from both EMA and FDA, PRIME designation from EMA, and Breakthrough Therapy and rare pediatric disease designations from FDA [6] - Alvelestat previously received Orphan Drug Designation and Fast Track Designation from FDA in 2021 and 2022, respectively [3][6] - The company has aligned with FDA and EMA on primary endpoints for a Phase 3 pivotal study of alvelestat, which could enable full approval in both US and Europe if successful [6] Financial and Partnership Updates - The company's current cash and cash equivalents are expected to fund operations into 2027, supporting multiple key inflection points [1] - The partnership with Ultragenyx includes potential additional milestone payments of up to $245 million and royalties on commercial sales in Ultragenyx territories [6] - Mereo has retained EU and UK commercial rights for setrusumab and will pay Ultragenyx royalties on commercial sales in those territories [6] Market Potential and Development Strategy - Setrusumab has the potential to become the standard-of-care in OI based on promising data from completed studies, including the Phase 2 portion of the Orbit Study [1] - Alvelestat's Orphan Designation in Europe could benefit earlier stage patients who are not currently eligible for augmentation therapy in many countries [1] - The company is preparing for launch readiness activities in key European markets for setrusumab [1] Additional Pipeline Information - The company has two oncology product candidates: etigilimab (anti-TIGIT) and navicixizumab for late-line ovarian cancer, with navicixizumab partnered with Feng Biosciences [6] - Mereo has entered into an exclusive global license agreement with ReproNovo SA for the development and commercialization of leflutrozole, a non-steroidal aromatase inhibitor [6]