Industry Spotlight: Yamo Pharmaceuticals
About Yamo Pharmaceuticals
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.
Ongoing Clinical Trial
On day one of Period 1, subjects in each dosing arm will be randomised and given either L1‑79 or placebo twice a day for 12 weeks. On concluding this period, participants will washout for six weeks and will then be crossed over into Period 2. Subjects who were given a placebo in Period 1 will receive L1‑79 and vice versa for another 12 weeks in Period 2.
Assessment of L1-79’s effect on the key deficits in social communication and interaction as analysed using the BOSCC, Vineland Adaptive Behavior Scale Third Edition (Vineland-3), CGI-S and other social-communication interaction measures are the key goals of the trial.
Yamo CEO Chuck Bramlage said: “The design of this clinical trial builds on the insights gained in previous clinical experience with L1-79, in which a favourable tolerability profile and positive efficacy trends were observed in a smaller 28-day pilot study.
“The ongoing Phase II study will serve as a proof-of-concept to evaluate the impact of two doses of L1-79 in a placebo-controlled crossover study with two 12-week treatment periods in 50 adolescents and adults with ASD.”