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

LONDON and REDWOOD CITY, Calif., Sept. 24, 2020 (GLOBE NEWSWIRE) -- Mereo BioPharma Group plc (NASDAQ: MREO, AIM: MPH), “Mereo” or “the Company”, a clinical-stage biopharmaceutical company focused on oncology and rare diseases, today announces that the U.S. Food and Drug Administration (“FDA”) has granted Rare Pediatric Disease designation to setrusumab for the treatment of osteogenesis imperfecta (“OI”). Setrusumab is a fully humanized monoclonal antibody that inhibits sclerostin, a protein which inhibits the activity of bone-forming cells. OI is a genetic rare disorder with no approved treatments that is characterized by reduced bone mass and fragile bones that break easily. In Mereo’s 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 in adults with OI.

The FDA grants Rare Pediatric Disease Designation for serious and life-threatening diseases that primarily affect children aged 18 years or younger and fewer than 200,000 people in the United States. If a Biologics License Application (“BLA”) in the United States for setrusumab is approved, Mereo may be eligible to receive a priority review voucher from the FDA, which can be redeemed to obtain priority review for any subsequent marketing application and may be sold or transferred to other companies for their programs, as has been done by other voucher recipients.

“Receiving Rare Pediatric Disease designation from the FDA highlights the significant unmet medical need facing children with OI and underscores the potential of setrusumab to become the first approved treatment option specifically for these patients,” said Dr. Denise Scots-Knight, Chief Executive Officer of Mereo. “Following the completion of our Phase 2b ASTEROID study, we are pleased that both the FDA and EMA have agreed on the principles of a design of a single Phase 3 pivotal pediatric study in OI. We believe there is a clear path forward for setrusumab in OI and are continuing discussions with potential partners prior to the initiation of a Phase 3 study consistent with our Company strategy.”

About Osteogenesis Imperfecta
Osteogenesis Imperfecta (OI) is a rare genetic disorder that is characterized by fragile bones and reduced bone mass resulting in bones that break easily, loose joints and weakened teeth. In severe cases, those with OI may experience hundreds of fractures in a lifetime. In addition, people with OI often suffer muscle weakness, early hearing loss, fatigue, curved bones, scoliosis, respiratory problems and short stature, leading to significant impacts on overall health and quality of life. The majority of cases of OI (estimated at approximately 90%) are caused by a dominant mutation in a gene coding for type I collagen, a key component of healthy bone. Current treatment of OI is supportive, focusing on minimizing fractures and maximizing mobility, but to date, there are no FDA or EU approved treatments.