AMO Pharma Presents Statistical Analysis of Results of Phase 2 Study of AMO-02 in Adolescents with Autism Spectrum Disorder


LONDON and DURHAM, N.C., May 6, 2019 /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare, childhood onset neurogenetic disorders with limited or no treatment options, today announced the presentation of a statistical analysis of Phase 2 study results for AMO-02 in the treatment of autism spectrum disorder (ASD). An overview of the analysis was presented on Friday, May 3rd in a poster presentation titled, "The utility of omnibus statistical approaches in go/no-go decision making in a Phase 2 study in adolescents with autism spectrum disorder: a case example" led by Joseph Horrigan MD, AMO Pharma's chief medical officer, during the International Society for Autism Research (INSAR) 2019 Annual Meeting at the Palais des congres de Montreal in Canada.

"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).

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," explained 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."

About the International Society for Autism Research 2019 Annual Meeting

The International Society for Autism Research (INSAR) 2019 Annual Meeting will bring together more than 2,000 researchers, clinicians, students, parents, and autism advocates from over 30 countries, Wednesday, May 1 through Saturday, May 4, 2019 at the Palais des congrès de Montrèal (Montreal Convention Center) in Canada. The INSAR Annual Meeting is the world's largest gathering of scientists and specialists as they exchange and disseminate the latest scientific discoveries and stimulate progress into the nature, causes, and treatments for autism spectrum disorder.

About Autism Spectrum Disorder

NIMH defines Autism Spectrum Disorder as: "Autism spectrum disorder (ASD) is a developmental disorder that affects communication and behavior. Although autism can be diagnosed at any age, it is said to be a "developmental disorder" because symptoms generally appear in the first two years of life.  According to the Diagnostic and Statistical Manual of Mental Disorders (DSM-5), a guide created by the American Psychiatric Association used to diagnose mental disorders, people with ASD have:

  • Difficulty with communication and interaction with other people
  • Restricted interests and repetitive behaviors
  • Symptoms that hurt the person's ability to function properly in school, work, and other areas of life

Autism is known as a "spectrum" disorder because there is wide variation in the type and severity of symptoms people experience. ASD occurs in all ethnic, racial, and economic groups. Although ASD can be a lifelong disorder, treatments and services can improve a person's symptoms and ability to function. The American Academy of Pediatrics recommends that all children be screened for autism. All caregivers should talk to their doctor about ASD screening or evaluation."

About AMO Pharma

AMO Pharma is a biopharmaceutical company incorporated in February of 2015. The co-founder, Dr. Michael Snape, has extensive experience in senior scientific and operational roles in both large pharma and biotech companies spanning more than twenty-five years, and has brought together a targeted and experienced senior management team with a proven track record of success in all phases of product development and acquisition. The company is working to identify and advance promising therapies for the treatment of serious and debilitating diseases in patient populations with significant areas of unmet need, including rare, debilitating childhood onset neurogenetic disorders with limited or no treatment options. AMO-02 is an investigational medicine that has not yet been approved for the treatment of patients anywhere in the world.  For more information, please visit the AMO Pharma website at


Mike Snape, PhD
Chief Executive Officer
AMO Pharma Ltd.
+44 1483 898 448

Kelly Wakelee
Berry & Company Public Relations


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SOURCE AMO Pharma Limited


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