New Preclinical Data Shows Promising Results for AMO Pharma’s Investigational Therapy in Duchenne Muscular Dystrophy Treatment

New Preclinical Data Shows Promising Results for AMO Pharma’s Investigational Therapy in Duchenne Muscular Dystrophy Treatment

AMO Pharma Limited, a clinical-stage biopharmaceutical company specializing in rare childhood-onset neurogenetic disorders, has announced initial preclinical data from a study on the use of their investigational therapy AMO-02 in the treatment of Duchenne muscular dystrophy (DMD). The study, conducted in collaboration with Brock University, showed improvements in muscle function, glucose handling, cardiac muscle function, and central nervous system (CNS) function in a mouse-model study.

The research partnership between AMO Pharma and Brock University was supported by a grant from the Brock-Niagara Validating, Prototyping, and Manufacturing Institute. The study aimed to investigate the potential utility of GSK3β inhibitors, specifically AMO-02 (tideglusib), in treating DMD. The researchers at Brock University, led by professors Val Fajardo, PhD, and Rebecca MacPherson, PhD, examined the effects of AMO-02 in the mdx.D2 mouse model, a well-characterized model of DMD.

Results from the study showed that AMO-02 treatment improved muscle function, glucose handling, metabolic areas, muscle fat deposition, cardiac muscle function, and cognitive function. These benefits were observed in both early and late stages of disease progression. The data indicate the broad efficacy of AMO-02 in multiple organ domains, including the heart and brain, as well as in metabolic function, skeletal muscle, and glucose and fat handling.

AMO Pharma’s Chief Scientific Officer, Michael Snape, PhD, expressed enthusiasm for the results and highlighted the potential of AMO-02 in treating muscle damage and weakness in DMD, as well as improving cardiac and skeletal muscle health and function. AMO Pharma plans to further collaborate with Brock University and is grateful for the outstanding work conducted by the research teams at the university.

Alongside this study, AMO Pharma recently announced positive results from the REACH-CDM clinical study of AMO-02 in the treatment of congenital myotonic dystrophy. The company is also developing AMO-01 for Phelan-McDermid syndrome and AMO-04 for Rett syndrome and related disorders. AMO-02, AMO-01, and AMO-04 are investigational medicines that have not yet received approval for the treatment of patients.

The findings from this preclinical study provide promising evidence of the potential of AMO-02 as a therapeutic option for DMD. Further research and collaboration will be conducted to advance the development of AMO-02 and improve the quality of life for individuals with DMD and other muscle-wasting conditions.

– Bianca M. Marcella et al., GSK3 inhibition improves skeletal muscle function and whole-body metabolism in the severe DBA/2J mdx mouse model, bioRxiv 2022.02.16.480726
– Hayward GC et al., Characterization of Alzheimer’s disease-like neuropathology in Duchenne’s muscular dystrophy using the DBA/2J mdx mouse model, FEBS Open Bio 2022. Jan;12(1):154-162.
– Media contact: Bill Berry, Berry & Company Public Relations

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