Fragile X Syndrome is 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 7 years of market exclusivity following approval in the US.
The application was submitted following positive results in a range of preclinical studies exploring the efficacy of AUT00206 in a genetic model of Fragile X in mice.
AUT00206 treatment for 21 days improved both cognitive and behavioral abnormalities similar to those occurring in children with Fragile X syndrome.
This work was supported by the FRAXA Research Foundation, a charity encouraging and funding research into Fragile X Syndrome.
AUT00206 is a novel, orally active small molecule designed to selectively modulate Kv3 potassium channels. The drug recently completed a first-in-human clinical trial in healthy volunteers and was safe and well tolerated.
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.
The preclinical study using models relevant to the pathophysiology of schizophrenia suggests AUT00206 has the potential to treat cognitive and negative symptoms of schizophrenia, as well as positive symptoms with fewer side effects than current anti-psychotic drugs. Cognitive and negative symptoms are poorly treated by antipsychotic drugs and are associated with significant functional impairment and reduced quality of life for patients.
