Yamo Pharma Reports Positive Phase 2 Results for Autism Treatment L1-79

Yamo Pharmaceuticals, a company specializing in clinical-stage pharmaceuticals, has announced promising topline results from its Phase 2 clinical trial evaluating L1-79 in adolescents and young adults with autism spectrum disorder (ASD). The trial's findings demonstrated a statistically significant improvement with L1-79 compared to placebo on the Vineland Adaptive Behavior Scale (Vineland-3) Socialization standard score. Specifically, the difference compared to placebo was 7.09 with a p-value of 0.02 during the first 12 weeks of the study.

The Vineland-3 Socialization Domain is a reputable assessment tool that measures an individual's coping skills, ability to engage in play and leisure, and social interactions. Beyond the primary endpoint, other metrics such as the Clinical Global Impression of Severity of Illness (CGI-S) and the Caregiver Global Impression of Change of 3 Most Bothersome Symptoms of ASD (CGI-3P) also showed significant improvements with L1-79 compared to placebo. Importantly, L1-79 was generally safe and well tolerated, with no serious adverse events or withdrawals from the trial.

Dr. J. Thomas Megerian, Yamo’s Chief Medical Officer, expressed optimism about the more than seven-point improvement in the Socialization Domain of the Vineland-3, noting that it represents one of the most significant effects observed to date for a medication targeting ASD. This improvement is viewed as clinically meaningful, addressing a core domain of function affected by ASD. The company is planning to discuss Phase 3 plans with the FDA and intends to file for Breakthrough Therapy designation.

Chuck Bramlage, Yamo’s Chief Executive Officer, expressed enthusiasm over the trial results, noting the potential to offer a new treatment option for patients and their families that can impact the core symptoms of ASD.

L1-79 is a tyrosine hydroxylase inhibitor designed to modulate catecholaminergic pathways, which are believed to play a role in the core symptoms of ASD. The FDA granted Fast Track designation to L1-79 in May 2018.

The Phase 2 study was a multi-center, randomized, and chronic-dosing clinical trial that enrolled 58 adolescents and young adults with ASD. Conducted over two 12-week periods with a six-week washout period in between, the crossover trial randomized participants to receive either L1-79 or placebo in each period. 

Autism spectrum disorder (ASD) is a group of complex neurodevelopment disorders characterized by difficulties in social communication and interaction, as well as restricted and repetitive behavior, interests, or activities. In the U.S., it is estimated that 1 in 36 children are affected by ASD, with boys being 3.8 times more likely to be diagnosed than girls. Symptoms of ASD typically begin in early childhood and persist throughout life. Both genetic and environmental factors are believed to contribute to ASD, but no pharmacological therapies are currently approved to address its core symptoms.

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