Autifony Therapeutics Limited (“Autifony”), which is pioneering the development of novel pharmaceutical treatments for serious disorders of the central nervous system, has announced that the US Food and Drug Administration (FDA) has granted AUT00206 an Orphan Drug Designation for the treatment of Fragile X Syndrome, the most common known cause of inherited learning disabilities.

Orphan Drug Designation is a special status granted to a drug intended to treat a rare disease or condition, which meets criteria specified in the Orphan Drug Act. Orphan designation qualifies the sponsor of the drug for various development incentives as well as seven years of market exclusivity following approval in the US.

The application was founded on positive results in a range of preclinical studies exploring the efficacy of AUT00206 in a genetic model of Fragile X in mice.

Studies of multiple disease-relevant endpoints showed significant improvement in both cognitive and behavioural functioning of the mice following treatment with AUT00206. This work was supported by the FRAXA Research Foundation, a charity that encourages and funds research into Fragile X Syndrome.

Dr Charles Large, CEO of Autifony, said: “We believe that our promising findings in the preclinical model of Fragile X support investigation of AUT00206 in clinical trials, which we plan to initiate as rapidly as possible in 2018. Orphan Drug designation by the FDA provides us with further encouragement to address this important unmet medical need.”

Dr Mike Tranfaglia, Medical Director and Chief Scientific Officer of FRAXA Research Foundation, said: “We are excited about the potential of AUT00206 as a treatment for Fragile X. With its novel mechanism of action, this compound has demonstrated its ability to rescue many disease-relevant phenotypes in Fragile X animal models.”

“AUT00206 has the potential to be a disease-modifying therapy for people with Fragile X, and we are looking forward to clinical trials in Fragile X patients.”

AUT00206 is also in development for schizophrenia. An experimental medicine study in patients with schizophrenia, involving electrophysiological biomarkers and imaging outcomes, was initiated in May 2017.