The partnership between Indianapolis-based Confluence Pharmaceuticals Inc. and a Vienna-based AOP Orphan Pharmaceuticals AG will develop a treatment for fragile X syndrome, which is the most commonly known genetic cause of autism, according to the release.
The agreement between the two focuses on using Confluence’s lead product to develop a treatment, according to the release. Fragile X impairs social and communication abilities in those affected, which is currently estimated at 1.7 million ?globally.
Fragile X is identified often by anxiety, attention deficit disorder, aggression, seizures, self-injurious behavior and physical deficits. It’s also been linked to X chromosome mutations, which leads to a decreased or absent level of fragile X mental retardation protein, according to the release.
“There is a clear unmet medical need in fragile X and related disorders where no effective targeted treatment exists,” said Dr. Craig A. Erickson, Confluence co-founder and lead scientific adviser, in the release. “This partnership is an important move in the right direction to begin to address this need.”
Erickson has developed a treatment for fragile X in the past In 2010, he developed a therapeutic application while he served as an assistant professor of psychiatry at the IU School of Medicine, according to the release.
Since receiving intellectual property rights from IU’s Research and Technology Corps, Confluence has created a drug development team, a clinical trial and regulatory affairs experts to accelerate the development of novel drugs and formulas, according to ?the release.
AOP specializes in developing and marketing medicines for complex diseases, specifically in Central Europe and the Middle East. Since Confluence has received Orphan Designation for its product in Europe and in the U.S., Confluence and AOP expect fast approval to treat fragile X.
“This relationship we have established with AOP is an exciting opportunity for Confluence to leverage AOP’s knowledge and expertise in Europe and the Middle East to accelerate this breakthrough treatment for the benefit of fragile X patients,” Confluence president and co-founder Steven Johns said in a press release.
AOP CEO Rudi Widmann said the partnership will also help accomplish his company’s mission to develop therapies for rare diseases.
“AOP has a unique record of developing and distributing products for rare and complex diseases and Confluence will benefit from AOP’s access to treatment centers and its long-term expertise in commercializing drugs for rare and complex diseases,” Widmann said in the release.
Fragile X is one of the most commonly inherited forms of an intellectual disability.
Currently, one out of 3,600 to 4,000 males globally are born with the mutation and will eventually develop the syndrome. One in 4,000 to 6,000 females have the mutation at birth, but only half will develop the syndrome, according to the release.
Suzanne Grossman
