AMO Pharma announced the presentation of a statistical analysis of Phase 2 study results for AMO-02 in the treatment of autism spectrum disorder (ASD).
"We are very pleased that these results reinforce that AMO-02 offers a consistent pattern of efficacy that gives further clinical validation of GSK3beta as a molecular target for novel therapeutic treatments for ASD," said Michael Snape, chief executive officer of AMO Pharma. "Previous clinical studies have lacked a gold standard measurement to assess efficacy. Our approach to clinical research and our analysis of results reflect AMO Pharma's commitment to meeting the highest achievable standards of scientific integrity in our clinical programs."
In this analysis, a step-wise omnibus approach was combined with a concordant trend analysis to verify mean results from seven pre-specified outcome measures identified in a previous Phase 2 trial. Permutation tests involving 1,000 computerized simulations were conducted and study participants were randomly re-assigned to a treatment group. Results were consistent with previous findings, showing patients treated with AMO-02 outperformed placebo in measures of social withdrawal (ABC-Social) and repetitive behaviors (RBS-R), as well as daily living skills (Vineland), memory (NEPSY) and sleep quality (CSHQ).
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Autism spectrum disorder (ASD) is a neurodevelopmental disorder characterized by social deficits and repetitive behaviors. No medications have been approved for the treatment of core symptoms of this disorder. AMO-02 is a novel once-daily orally available GSK3 beta inhibitor. Recent preclinical studies indicate that GSK3 beta is an enzyme that is overactive in multiple different genetically determined syndromic autisms, suggesting the importance of this molecular target in the field of ASD research.
"The most important takeaway from this study was the extremely low probability that there were false-positives reported within our initial Phase 2 results," said Dr. Horrigan. "We look forward to continuing the development of AMO-02 with even more confidence and are hopeful that resulting data will provide essential insights to guide future clinical research."