Confluence Gets $200K from University-affiliated Venture Fund to Develop Fragile X Treatment

An Indiana University-affiliated venture fund has invested $200,000 in Confluence Pharmaceuticals to continue development of the company’s lead product for the treatment of fragile X syndrome.

Along with AOP Orphan Pharmaceuticals, Confluence is developing a novel, clinically discovered adult and pediatric therapy for social and communication impairments related to fragile X and autism spectrum disorders.

Based on the generic compound acamprosate — used in the United States to reduce alcohol cravings — ACP has received Food and Drug Administration Orphan Drug Designation in Europe and the United States. Capital from the Indiana University (IU) Philanthropic Venture Fund, which invests in IU-related projects, will be used to speed up clinical development and regulatory activity of the investigational treatment.

The therapy is currently in Phase 2/3 of a clinical trial (NCT01813318) for autism spectrum disorders. The double-blind phase calls for each participant to receive 10 weeks of treatment with acamprosate or a matching placebo. Subjects will then have the option of receiving acamprosate for 16 weeks.

As the leading single-gene known cause of autism, fragile X is associated with numerous intellectual and emotional issues affecting 180,000 individuals in the United States.

“Today, no medication exists to treat these core impairments,” Steve Johns, Confluence’s CEO, said in a news release. “Early pilot studies have demonstrated the potential of our product to improve focus, communication, social withdrawal and attention deficit hyperactivity disorder.”

Confluence’s intellectual property is licensed at Indiana University through the IU Innovation and Commercialization Office. The company is based in Carmel, Indiana.

“We, too, are delighted to be supporting the team at Confluence in their efforts to bring this critical therapy to patients, and to have Confluence as our 10th investment since launching the fund a few months ago,” said Terry Willey, manager of the IU fund, which was launched in February.

In related news, a clinical trial found that a cognitive training program provides benefits for young fragile X patients. Specifically, the trial discovered that the computer game-like program called Cogmed can improve working memory and increase the attention span of children with the syndrome.

Developed by Pearson Corporation, Cogmed consists of disparate computerized visuospatial memory training tasks involving temporary storage and manipulation of sequences, based on the patient’s developmental level.

Trial findings were published in the study, “Cognitive training for children and adolescents with fragile X syndrome: a randomized controlled trial of Cogmed,” in the Journal of Neurodevelopmental Disorders.