Fast Track Designation Granted for L1-79 in Treatment for Autism Spectrum Disorder

Phase 2 clinical study suggests L1-79 has the potential to address an unmet medical need for the treatment of the core symptoms of autism spectrum disorder.

Fast Track Designation was granted by the US Food and Drug Administration (FDA) for Yamo Pharmaceuticals’ investigational drug L1-79, a tyrosine hydroxylase inhibitor intended to mitigate the socialization and communication symptoms in individuals with autism spectrum disorder.

L1-79 has shown the potential to address an unmet medical need for treatment of core symptoms of autism spectrum disorder.

In the US, it’s estimated that 1 in 59 children have autism spectrum disorder, with boys being 3—4 times more susceptible than girls.

“This is an important and exciting step toward developing a much-needed treatment for the core symptoms of autism spectrum disorder. We are encouraged by the results from our first studies with L1-79 and the grant of Fast Track Designation by the FDA,” J. Thomas Megerian MD, PhD, chief medical officer, senior vice president of clinical development, Yamo Pharmaceuticals, said in a statement. “We look forward to working closely with the FDA and our advisors as we advance L1-79 through additional clinical studies.”

The designation is based on data from a recently completed phase 2 clinical study where multiple independent outcome measures demonstrated improvement in the core social domains affected by the disorder.

Positive trends in the efficacy measures were seen, despite the small sample size and short duration of the study—28 days.

Future studies in children, adolescents and adults with autism spectrum disorder are planned.

L1-79 acts as a tyrosine hydroxylase inhibitor and is expected to modulate the catecholaminergic pathways implicated in the disorder.

There are currently no approved pharmacological therapies which address the core symptoms of the spectrum disorder.