Mereo BioPharma's Setrusumab Phase 3 Trial Progresses; Alvelestat Receives Positive EMA Opinion
Key Insights
Mereo BioPharma's setrusumab Phase 3 Orbit study for osteogenesis imperfecta (search) is ongoing, with a second interim analysis expected in mid-2025.
Alvelestat, for Alpha-1 Antitrypsin Deficiency-associated Lung Disease (search), received a positive EMA opinion on its European Orphan Designation application.
The European Commission is expected to make a final decision on Alvelestat's Orphan Designation in the first quarter of 2025.
Mereo BioPharma Group plc (NASDAQ: MREO) has provided an update on its lead clinical programs, including setrusumab for osteogenesis imperfecta (search) (OI) and alvelestat for alpha-1 antitrypsin deficiency-associated lung disease (search) (AATD-LD). The company's partner, Ultragenyx Pharmaceutical, Inc. (search), is continuing to dose patients in the Phase 3 Orbit study of setrusumab, with a second interim analysis anticipated in mid-2025 and a potential final analysis in the fourth quarter of 2025.
Setrusumab Development for Osteogenesis Imperfecta
The Phase 3 Orbit Study is progressing as planned, with ongoing patient dosing. Additionally, treatment is continuing in the Cosmic study, an open-label Phase 3 trial evaluating setrusumab against intravenous bisphosphonate therapy in patients aged 2 to <7 years. Data from the Cosmic study will be evaluated in parallel with the Orbit interim and final analyses. Setrusumab has received orphan designation for osteogenesis imperfecta (search) from the EMA and FDA, PRIME designation from the EMA, and Breakthrough Therapy designation and rare pediatric disease designation from the FDA.
Dr. Denise Scots-Knight, Chief Executive Officer of Mereo BioPharma, stated, "Based on the highly promising data from completed studies of setrusumab in OI, including the Phase 2 portion of the Orbit Study, we remain confident in the potential of setrusumab to become the standard-of-care in OI. We look forward to the second interim analysis expected mid-year as we continue our launch readiness activities in the key European markets."
Alvelestat and European Orphan Designation
The European Medicines Agency (EMA)’s Committee for Orphan Medicinal Products (COMP) has issued a positive opinion on Mereo BioPharma’s application for Orphan Designation for alvelestat. The COMP recommendation has been provided to the European Commission, which is expected to issue a final decision on the Orphan Designation in the first quarter of 2025. Alvelestat previously received Orphan Drug Designation and Fast Track Designation from the U.S. FDA in 2021 and 2022, respectively.
European Orphan Designation is awarded to therapeutic candidates targeting the treatment, prevention, or diagnosis of life-threatening or chronically debilitating diseases with a prevalence of fewer than 5 in 10,000 people in the European Union which provide a significant benefit over available therapies, or for which no approved therapies exist. Therapeutics receiving EU Orphan Designation are eligible for ten years of marketing exclusivity upon approval, as well as fee reductions for various centralized activities including the Marketing Authorization Application, inspections, and protocol assistance. Individual EU Member States also provide specific incentives to support the development, review, and availability of Orphan Medicinal Products at the time of HTA evaluations and Pricing and Reimbursement negotiations.
Financial Outlook
Mereo BioPharma also reiterated its previous cash runway guidance, stating that its current cash and cash equivalents are expected to fund operations into 2027, through multiple key inflection points. Dr. Scots-Knight added, "With our cash runway into 2027, we continue to be in a strong position to execute on our key milestones through 2025."