Autism Spectrum Disorder

Autism Spectrum Disorder (ASD), a neurodevelopmental disorder, is characterized by impaired sociability and altered behavioral patterns, including increased irritability. Approximately 6.6 million children and adults in the United States have been diagnosed with ASD.

Because ASD is a spectrum disorder, each person with autism has a distinct set of strengths and challenges. Some people with ASD may live entirely independently, while others require significant support in their daily lives. For those whose lives are greatly impacted by the social communication deficits that are characteristic of ASD, there is no known therapeutic option and no approved FDA medications.

Two atypical antipsychotics (risperidone and aripiprazole) are approved to treat irritability in ASD. These drugs can improve behavior in some children, but can be associated with significant side effects, including weight gain and sedation. Critically, only about half of children respond to these treatments. These drugs block the brain’s receptors for dopamine and serotonin and were initially developed to treat schizophrenia.

MapLight is making rapid progress toward the treatment of multiple clinical features of Autism. Our platform has demonstrated the promise of ML-004 for the improvement of sociability and irritability in multiple animal models of autism. ML-004 is now in a Phase 2 trial to assess whether it can effectively treat core symptoms in adolescents and adults with ASD, a critical unmet need.

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