L1-79 is a novel therapy that targets the core symptoms of autism. Previous experience with open-label administration of L1-79 in patients with autism demonstrated that L1-79 is a well-tolerated oral treatment that has the potential to improve the core symptoms of autism. In a recently completed Phase II study, multiple independent efficacy measures assessed using commonly accepted and validated psychometric tests demonstrated positive trends supporting improvements in the target core symptom domains affected by autism, especially social domains, despite a short treatment period and small number of patients. This data supported the granting of a Fast Track Designation by the Food and Drug Administration (FDA) in May of 2018.
Underlying the safety and efficacy of L1-79 is a novel mechanism of action theory that integrates extensive pre-existing medical research into a more unified theory of autism than has been previously possible. This theory includes the ability to modulate catecholamines and impact the functions of the following:
- Mechanisms in the central nervous system (CNS) which regulate mood, attention, reward, irritability, attention, motor function and other elements in the brain which appear to be affected by autism;
- Different systems in the CNS which serve to modify brain function through inhibition and which are believed to be deficient in the hyperactive CNS state that characterizes autism;
- Mechanisms in the gut which have been hypothesized to play an important role in some forms of autism;
- Mechanisms in the liver and pancreas that are altered in the presence of some individuals with autism, and;
- Importantly, a novel system of integrated neurologic and metabolic signaling that unites the gut, the mesenteric organs, the central nervous system and the autonomic nervous system.