MapLight's ML-004 Misses Phase 2 Primary Endpoint in Autism but Shows Irritability Signal in Adolescents

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MapLight Therapeutics announced that its Phase 2 IRIS study of ML-004 in autism spectrum disorder did not meet the primary endpoint of improving social communication deficits. However, a prespecified subgroup analysis of adolescents aged 12 to 17 with moderate to severe irritability at baseline showed clinically meaningful improvement over placebo on the Aberrant Behavior Checklist-Irritability subscale, with an effect size of 1.33 and a nominal p-value of 0.013. Consistent improvement was also seen in the clinician-rated CGI-I irritability domain, with an effect size of 1.08 and a nominal p-value of 0.036. The drug was well tolerated, with no severe or serious adverse events and lower mean weight gain than placebo. MapLight plans to meet with the FDA to discuss next steps and remains well-capitalized ahead of Phase 2 results for ML-007C-MA in schizophrenia expected by mid-August 2026.

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