MOUNTAIN VIEW, Calif., April 12, 2022 /PRNewswire/ -- Adge Pharmaceuticals ("Adge") continues to advance its rare disease programs in the Rett Syndrome and Osteogenesis imperfecta in support of the regulatory requirements of the US FDA to move its lead asset expeditiously to the clinic.
In preparation for launching of the clinical program in the Rett Syndrome, Dr. Kalev Kask, CEO and founder of Adge, will be attending the ASCEND 2022 Rett Syndrome National Summit held in Nashville on April 27-30, 2022.
As announced earlier (https://adgepharm.com/news/mar-22-2022/), Adge has secured a global license to RO269228, a clinical stage oral small molecule originally developed by Roche for osteoporosis. RO269228, also known as Elocalcitol, is a vitamin D analog which has demonstrated efficacy and safety in multiple Phase 2 clinical trials. Furthermore, its mechanism of actions are highly relevant to addressing pathogenesis of multiple rare diseases including Rett Syndrome and Osteogenesis imperfecta that Adge is targeting as priority indications.
Rett syndrome is a neurodevelopmental disorder that is caused by a mutation in the MECP2 gene and almost exclusively affects girls. It is characterized by normal early growth and development followed by a slowdown in development, slowed brain and head growth, loss of purposeful use of the hands, distinctive hand movements, problems with walking, seizures, autistic behavior and intellectual disability. Rett syndrome is the second most prevalent neurodevelopmental disorder in girls after Down syndrome affecting an estimated 1 in 9,000 to 10,000 females.
Osteogenesis imperfecta, also known as brittle bone disease, is a group of genetic diseases that primarily affect the bones. People with this condition have bones that fracture easily, often from mild trauma or with no apparent cause. Multiple fractures are common, and in severe cases, can occur even before birth. Osteogenesis imperfecta affects approximately 1 in 10,000 to 20,000 people worldwide. An estimated 25,000 to 50,000 people in the United States have the condition.
Adge Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development of first-in-class therapies for orphan indications.