“OI is a rare, genetic disorder that negatively affects the body’s ability to produce type I collagen which leads to bone fragility causing bone deformities and frequent fractures, often with minimal or no trauma. There are multiple subtypes of OI ranging from Mild to Severe where the most severe types are not compatible with life and patients die shortly after birth having suffered from multiple fractures. There is a significant unmet medical need as there are no approved treatment options for patients diagnosed with OI. We are delighted that MIV-711 has been granted ODD by the FDA, which strengthens the evidence base for its ability to positively impact bone remodeling and its potential to treat bone-related disorders”, states Jens Lindberg, CEO of Medivir.

MIV-711

MIV-711 has shown, in an OI-specific animal model, significant and dose-dependent positive impact on bone strength and quality as well as bone morphology, supporting potential benefit in treating patients with OI. Clinical data in osteoarthritis further support that MIV-711 has a positive effect in the prevention of bone degradation, translating to potential clinical benefit in OI.

Inhibits cathepsin K

MIV-711 inhibits cathepsin K, which is the main enzyme by which osteoclasts, that are involved in bone resorption and cartilage degradation, cleave collagen in the bone matrix. Cathepsin K inhibition protects the damaged bone by reducing bone resorption and promoting bone formation, thereby addressing the key mechanisms causing pathological changes in OI, with potential to minimize long-term negative effects.