Developing Treatment for Fragile X

Indianapolis-based Confluence Pharmaceuticals www.confluencepharma.com and Vienna-based AOP Orphan Pharmaceuticals AG (AOP) www.aoporphan.com/en.html have signed an agreement to co-develop and market Confluence’s product for the treatment of Fragile X Syndrome (FXS) in Europe and the Middle East.

Confluence is a biopharmaceutical company focused on developing therapeutic treatments for FXS and Autism spectrum disorders while AOP an Austrian based company specializes in developing and marketing medicines for rare and complex diseases.

FXS is associated with an array of intellectual and emotional disabilities impacting 180,000 individuals in the U.S and 213,000 in Europe. Most often FXS is accompanied by anxiety, attention deficit disorder, aggression, seizures, self-injurious behavior, and physical problems.

The cause of FXS has been linked to a genetic mutation of the X chromosome leading to a decreased or absent level of fragile X mental retardation protein. “There is a clear unmet medical need to understand FXS and related disorders where no effective targeted treatment exists”, said Dr. Craig A. Erickson, Confluence Co-Founder and lead scientific adviser.

Initial intellectual property rights were exclusively licensed to Confluence by the Indiana University Research and Technology Corporation (IURTC) http://iurtc.iu.edu, a not-for-profit agency to help IU faculty and researchers realize the commercial potential of their discoveries. IURTC is part of the Innovate Indiana http://innovateindiana.iu.edu initiative which engages strategic partners to advance IU’s intellectual resources and expertise,

Confluence has since assembled a team of drug development and clinical trial and regulatory affairs experts to accelerate the development of novel drugs and formulas for FSX and autism spectrum disorders.

AOP works with representatives throughout Central Europe and the Middle East with distribution ties to South Africa, Southeast Asia, and South America. With Confluence having received Orphan Designation for their lead product in Europe and the U.S., both Confluence and AOP expect accelerated regulatory approval to treat FSX.