Garetosmab is in clinical development for the prevention of abnormal bone formation, outside of the normal skeleton (heterotopic ossification), and soft-tissue flare-ups in patients with fibrodysplasia ossificans progressiva (FOP). FOP is a very rare disease caused by a mutation in the gene ACVR1. The mutation results in the formation of unwanted bone in muscles, tendons, and ligaments throughout the body. Abnormal, misplaced and mis-shapen (heterotopic) bone can bridge across joints causing immobility, scoliosis, and other deformities. Patients usually require a wheelchair by the time they reach their 20s. Death often results in the 40s from complications, such as pneumonia, heart failure and loss of mobility in the chest, neck and jaw.
Garetosmab is administered intravenously. It is designed to attach to activin A, which stops activin A from interacting with ACVR1. This is expected to stop or reduce unwanted bone formation. If licensed, garetosmab will offer a treatment option for preventing abnormal bone formation and soft-tissue flare-ups in patients with FOP.
Edasalonexent is currently in clinical development for the treatment of male paediatric patients with duchenne muscular dystrophy (DMD). DMD is a rare genetic condition caused by a mistake or abnormality in a gene called dystrophin, located on the X chromosome (one of the two sex chromosomes). Chromosomes are tiny structures inside cells made from deoxyribonucleic acid (DNA) and proteins. Males have one X chromosome, while females have two X chromosomes. As males have one X chromosome, DMD is much more common in males. DMD is fatal condition with no cure. It causes progressive muscle weakness and often leads to loss of walking ability by the age of twelve, as well as problems with the heart and lungs. Current treatment options only target a particular mistake or abnormality and focus on slowing progression of the disease.