AMO Pharma Reports Positive Data from TIDE Study of AMO-02 in Autism Spectrum Disorder

Rare Daily Staff

AMO Pharma, a company focusing on the development of therapies for rare childhood onset neurogenetic disorders with limited or no treatment options, reported an update on results of the recently-completed TIDE study of AMO-02 (tideglusib) in the treatment of autism spectrum disorder (ASD). The updates were presented at the 65th Annual Meeting of the American Academy of Child and Adolescent Psychiatry in Seattle, Washington.

The phase 2 clinical 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 83 adolescents with ASD between the ages of 12 and 18 years-old. 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 randomized, 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 and repetitive behaviors, as well as daily living skills, memory, and sleep quality. 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.

The results provide 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, according to AMO Pharma Chief Medical Officer Joseph Horrigan.

Autism spectrum disorder 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, CEO of AMO Pharma.

AMO-02 is also in development for the treatment of congenital myotonic dystrophy and is positioned to enter clinical stage development for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease.

This study was funded by the Ontario Brain Institute, Brain Canada, and the Azrieli foundation.

AMO-02 is in development for the treatment of congenital myotonic dystrophy and is positioned to enter clinical stage development for the treatment of the severe form of congenital myotonic dystrophy known as DM1 or Steinert disease.

November 1, 2018