Yamo Pharmaceuticals announced positive Phase 2 trial results for L1-79, a drug targeting core autism spectrum disorder (ASD) symptoms, at the 2025 INSAR Annual Meeting. The study demonstrated statistically significant improvements in socialization skills for adolescents and young adults with ASD, a significant advancement given the lack of FDA-approved therapies for this core symptom domain. The drug also maintained a favorable safety profile.
This news holds substantial promise for individuals with ASD and their families. The observed improvements in socialization, nearly double the minimal clinically important difference, suggest the potential for meaningful changes in daily social interactions and overall quality of life. This addresses a critical unmet need for effective therapies that target the core social challenges faced by those with ASD.
In a 12-week randomized, double-blind, placebo-controlled crossover study of 58 participants aged 12-21, L1-79 demonstrated a 7.94-point advantage over placebo on the Vineland-3 Socialization Standard Score (VSSS), a gold-standard measure of social skills. Additional measures, including clinician and caregiver assessments, further confirmed the positive impact on socialization and a reduction in symptom severity. L1-79, a tyrosine hydroxylase inhibitor, works by modulating catecholamine signaling, a pathway believed to contribute to social and sensory difficulties in ASD.
These positive Phase 2 results pave the way for Phase 3 trials and discussions with the FDA. The development of L1-79 represents a potential paradigm shift in ASD treatment, offering hope for a future where core social deficits can be effectively addressed, leading to increased independence and improved social functioning for individuals with ASD.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
