Skip to main content

IU-licensed treatment for fragile X to be developed, marketed for Europe and Middle East


 INDIANAPOLIS — An alliance of U.S. and Austrian drug companies will co-develop and market an Indiana University-licensed treatment for fragile X syndrome — the most commonly known genetic cause of autism — throughout Europe and the Middle East.

 The agreement between Indianapolis–based Confluence Pharmaceuticals Inc. and Vienna-based AOP Orphan Pharmaceuticals AG centers on Confluence’s lead product. At present, there are no approved treatments for the social and communication impairments that accompany fragile X, which affects an estimated 1.7 million people globally.

 “There is a clear unmet medical need in fragile X and related disorders where no effective targeted treatment exists. This partnership is an important move in the right direction to begin to address this need,” said Dr. Craig A. Erickson, Confluence co-founder and lead scientific adviser. Erickson developed the therapeutic application in 2010 when he served as an assistant professor of psychiatry at the Indiana University School of Medicine.

 Initial intellectual property rights were exclusively licensed to Confluence by the Indiana University Research and Technology Corp. Confluence has since assembled a team of drug development, clinical trial and regulatory affairs experts to accelerate the development of novel drugs and formulas for fragile X syndrome and autism spectrum disorders.

 AOP specializes in the development and marketing of medicines for rare and complex diseases, with representation throughout Central Europe and the Middle East, and distribution ties to South Africa, Southeast Asia and South America. With Confluence having received Orphan Designation for its lead product in Europe and the United States, both Confluence and AOP expect accelerated regulatory approval to treat fragile X.

 “The 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,” said Steven Johns, Confluence president and co-founder.

 Rudi Widmann, CEO of AOP, said the agreement will further his company’s mission to find and 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.

 Fragile X is often accompanied by anxiety, attention-deficit disorder, aggression, seizures, self-injurious behavior and physical deficits. Its cause has been linked to a genetic mutation of the X chromosome, which leads to a decreased or absent level of fragile X mental retardation protein, or FMRP.

 Fragile X is the most commonly inherited form of intellectual disability, according to the National Fragile X Foundation. About one out of 3,600 to 4,000 males worldwide are born with the full mutation, with most eventually developing fragile X syndrome. About one out of 4,000 to 6,000 females globally are born with the full mutation, but only about half develop features of the syndrome.

 About the Indiana University Research and Technology Corp.

 IURTC is a not-for-profit agency that helps IU faculty and researchers realize the commercial potential of their discoveries. Since 1997, IURTC’s university clients have accounted for more than 2,800 inventions, nearly 1,900 patent applications and 77 startup companies. IURTC is part of the Innovate Indiana initiative, which engages strategic partners to leverage and advance IU’s intellectual resources and expertise, enhance Indiana’s economic growth and contribute to the overall quality of life for Hoosiers.