Treatment associated with improvement in social withdrawal, repetitive behaviors, daily living skills, memory and sleep quality
LONDON and DURHAM, N.C./PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on rare, childhood onset neurogenetic disorders with limited or no treatment options, recently announced an update on results of the recently-completed TIDE study of AMO-02 (tideglusib) in treatment of autism spectrum disorder (ASD). Updates were presented by principal investigator Evdokia Anagnostou, MD, at the 65thAnnual Meeting of the American Academy of Child and Adolescent Psychiatry in Seattle, Washington. This study was funded by the Ontario Brain Institute, Brain Canada and the Azrieli foundation.
This Phase 2 study was conducted at three Canadian clinical trial facilities and assessed the safety and efficacy of AMO-02, a novel orally available GSK3 beta inhibitor, in adolescents with ASD between the ages of 12 and 18 years-old (n = 83). The once-daily treatment for the core symptoms of ASD was found generally safe and well-tolerated, with adverse event rates that were generally similar between tideglusib and placebo. There were no treatment-associated serious adverse events.
Subjects in the Phase 2 randomized, 1:1 double-blinded study were treated with AMO-02 or placebo across a 12-week treatment period, with follow-up at four weeks. Daily dosing began at 400 mg and was increased incrementally up to 1000 mg based on the subject's weight. Patients treated with AMO-02 consistently 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). Outcome measures were based on measures including caregiver-and clinician-completed rating scales. A permutation test of efficacy results indicated that probability of false-positives was low.
Autism spectrum disorder (ASD) is a common neurodevelopmental disorder, characterized by social deficits and repetitive behaviors. No medications have been approved for the treatment of core symptoms of this disorder. Recent preclinical studies indicate that GSK3 beta is an enzyme that is overactive in key molecular pathways that are germane to neuronal functioning and neuronal plasticity in neurodevelopmental disorders. GSK3 beta also plays an important role in circadian function and in modulating neuroinflammatory processes in the brain.
"This study marks the first time a GSK3 beta inhibitor has been the focus of a well-designed ASD clinical trial. We believe the resulting data represent a positive step forward in validation of GSK3β as a molecular target in treatment of symptoms associated with ASD beyond any existing published pre-clinical data," said Michael Snape, chief executive officer of AMO Pharma. "AMO Pharma would like to thank Dr. Anagnostou, the POND network and the families that took part in this study."
"There has been very little progress in research related to treatment of ASD over the past decade, while the incidence of ASD seemingly continues to rise," stated Dr. Joseph Horrigan, AMO's chief medical officer. "These results provide a new level of hope that treatment with AMO-02 has the potential to offer meaningful, multi-symptom benefit in ASD and merits further study as a potential treatment for ASD."
About POND
The Province of Ontario Neurodevelopmental Disorders (POND) Network is an integrated discovery program funded by the Ontario Brain Institute which aims to understand the neurobiology of neurodevelopment disorders and translate the findings into effective new treatments.
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."
Contacts
Corporate:
Mike Snape, PhD
Chief Executive Officer
AMO Pharma Ltd.
+44 1483 898 448
mike.snape@amo-pharma.com
Media:
Kelly Wakelee
Berry & Company Public Relations
212.253.8881
kwakelee@berrypr.com
