MIV-711

There is no cure for OI and a multidisciplinary approach to treatment including physical therapy, surgical treatments including insertion of rods, vitamin supplements and treatment of other complications is generally taken. Surgical intervention may be required for severe OI cases for resolving fractures and correcting limb deformities. There are no approved pharmacological treatments for OI, with bisphosphonates used off label, mainly during childhood up to adolescence to reduce bone fragility and risk of fracture and deformation, particularly in the vertebral spine.  

Osteogenesis Imperfecta (OI) is driven by disturbances in bone formation and remodelling, most commonly arising from structural or quantitative defects in type I collagen, but also in genes involved in collagen modification or folding, bone mineralization, and osteoblast differentiation. These defects disrupt the tightly coordinated cycle of bone resorption and bone formation, resulting in chronic skeletal fragility, poor bone quality, and a high fracture risk.  

MIV-711 has received Orphan Drug Designation (ODD) from the FDA for OI. In addition, MIV-711 has also received ODD as well as Rare Pediatric Disease Designation (RPDD) for the treatment of Legg-Calvé-Perthes disease (LCPD), a rare pediatric hip disorder affecting children between the ages of 2 and 12, further strengthening the expectation of a positive effect on bone remodeling and bone formation.