Autism drug advances in testing

Indianapolis-based Confluence Pharmaceuticals Inc. signed an agreement with a European orphan drug company that should give it the capital to take its experimental autism drug through at least the first two phases of human testing and possibly beyond.

Financial details of Confluence’s agreement with Austria-based AOP Orphan Pharmaceuticals AG were not disclosed. But Confluence CEO Steve Johns said the deal provides both money and access to AOP’s network of health care providers in Europe and the Middle East.

Confluence is reformulating an existing drug known as acamprosate, which is on the market as a treatment for alcohol dependency. Confluence licensed rights to the drug from the Indiana University School of Medicine. A former IU professor, Dr. Craig Erickson, discovered that the drug was an effective treatment for fragile X syndrome, which stems from a genetic mutation and is a cause of autism.

Confluence has received orphan drug status in the United States and Europe, which allows it to conduct clinical trials with fewer patients to receive regulatory approval. That helps companies bring drugs to market faster and with less spent on research.

Johns said Confluence has raised investment capital of more than $1 million. He said the company expects to start its first human testing later this year, with a Phase 2 clinical trial starting in the middle of 2016.