New Preclinical Data Shows Promising Results for AMO-02 in the Treatment of Duchenne Muscular Dystrophy

New Preclinical Data Shows Promising Results for AMO-02 in the Treatment of Duchenne Muscular Dystrophy

AMO Pharma Limited has announced initial preclinical data from a study investigating the use of their investigational therapy, AMO-02, in the treatment of Duchenne muscular dystrophy (DMD). In collaboration with Brock University, the study showed improvements in muscle function, glucose handling, cardiac muscle function, and central nervous system (CNS) function in a mouse model.

The research partnership between Brock University and AMO Pharma began in January 2023 and aimed to explore the potential utility of GSK3β inhibitors in treating DMD. The study used the mdx.D2 mouse, a well-characterized model of DMD. The researchers investigated the effects of AMO-02 and a follow-on compound on the mouse model at different stages of the disease.

The results demonstrated that treatment with AMO-02 improved muscle function and glucose handling, as well as showed improvements in metabolic areas, muscle fat deposition, and cardiac muscle. These benefits were observed in both early and late-stage DMD mice. Additionally, the study showed that short-term treatment with AMO-02 significantly improved cognitive function.

Dr. Val Fajardo, one of the lead researchers at Brock University, highlighted the broad profile of efficacy of AMO-02 across multiple organ domains, including the heart and brain, along with metabolic function, skeletal muscle, and glucose and fat handling.

The findings from this preclinical study provide further evidence of the potential of AMO-02 in treating muscle damage and weakness associated with DMD and other muscle-wasting conditions. AMO Pharma plans to continue its collaboration with Brock University and looks forward to advancing the research.

In addition to the promising results in DMD, AMO Pharma recently announced positive results from the REACH-CDM clinical study of AMO-02 in the treatment of children and adolescents with congenital myotonic dystrophy. These results showed significant efficacy benefits in multiple areas, including cognitive performance and improvements in biomarkers and walk/run measurements.

AMO Pharma aims to develop AMO-02 for the treatment of multiple muscle disorders, including adult-onset myotonic dystrophy, while also progressing AMO-01 for the treatment of Phelan-McDermid syndrome and AMO-04 as a potential treatment for Rett syndrome and related disorders.

This research collaboration with Brock University and the promising preclinical and clinical data highlight the potential of AMO-02 as a treatment option for various muscle-related disorders, where there are currently limited or no approved therapies available.

Duchenne muscular dystrophy (DMD): A rare genetic disorder characterized by progressive muscle degeneration and weakness.
GSK3β inhibitor: A drug that inhibits the activity of an enzyme called glycogen synthase kinase 3 beta (GSK3β), which plays a role in various cellular processes.
CNS function: Refers to the function of the central nervous system, which includes the brain and spinal cord.

Bianca M. Marcella, Briana L. Hockey, Jessica L. Braun, Kennedy C. Whitley, Mia S. Geromella, Ryan W. Baranowski, Colton J.F. Watson, Sebastian Silvera, Sophie I. Hamstra, Luc J. Wasilewicz, Robert W.E. Crozier, Amelie Marais, Rene Vandenboom, Brian D. Roy, Adam J. MacNeil, Rebecca E.K. MacPherson, Val A. FajardoGSK3 inhibition improves skeletal muscle function and whole-body metabolism in the severe DBA/2J mdx mouse model bioRxiv 2022.02.16.480726
Hayward GC, Caceres D, Copeland EN, Baranowski BJ, Mohammad A, Whitley KC, Fajardo VA, MacPherson REK. 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. doi: 10.1002/2211-5463.13317. Epub 2021 Nov 11. PMID: 34668666; PMCID: PMC8727939.

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