Mereo BioPharma Receives FDA Rare Pediatric Disease Designation for Setrusumab for the Treatment of Osteogenesis Imperfecta - Seite 2

About Setrusumab
Setrusumab is a fully humanized monoclonal antibody that inhibits sclerostin, a protein which inhibits the activity of bone-forming cells. The mechanism of action of setrusumab could be particularly well suited for the treatment of OI and setrusumab has the potential to become the first approved treatment option that could reduce fractures and improve the quality of life for individuals with OI. Mereo has obtained orphan drug designation in OI for setrusumab in both the United States and the EU. Setrusumab was accepted into the EMA’s Adaptive Pathways program in the EU and was accepted into the EMA’s Priority Medicines scheme (PRIME). In the Phase 2b ASTEROID study, setrusumab demonstrated a dose-dependent bone building effect and a trend of reduction in fractures in addition to being safe and well tolerated adults with OI. Following the review of the data from the Phase 2b ASTEROID study, the FDA agreed on the principles of a design of a Phase 3 pediatric study in OI to be completed prior to the submission of a BLA in the United States, which is also in line with Mereo’s proposed pivotal pediatric study design that has been agreed to in principle with the European Medicines Agency (“EMA”). Mereo intends to partner setrusumab prior to conducting a pivotal trial of setrusumab in children with severe OI.

About Mereo BioPharma
Mereo BioPharma is a biopharmaceutical company focused on the development and commercialization of innovative therapeutics that aim to improve outcomes for oncology and rare diseases. Mereo's lead oncology product candidate, etigilimab (Anti-TIGIT), has completed a Phase 1a dose escalation clinical trial in patients with advanced solid tumors and has been evaluated in a Phase 1b study in combination with nivolumab in select tumor types. Mereo's rare disease product portfolio consists of setrusumab, which has completed a Phase 2b dose-ranging study in adults with OI, as well as alvelestat, which is being investigated in a Phase 2 proof-of-concept clinical trial in patients with alpha-1 antitrypsin deficiency (AATD) and in a Phase 1b/2 clinical trial in COVID-19 respiratory disease.

Additional Information
The person responsible for arranging the release of this information on behalf of the Company is Charles Sermon, General Counsel.

Forward-Looking Statements
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