AMO Pharma Announces Promising Results for Investigational Therapy in Duchenne Muscular Dystrophy Treatment

AMO Pharma Announces Promising Results for Investigational Therapy in Duchenne Muscular Dystrophy Treatment

AMO Pharma has released initial preclinical data from a study that investigated the use of their investigational therapy, AMO-02, in the treatment of Duchenne muscular dystrophy (DMD). The study, conducted in collaboration with Brock University, demonstrated improved muscle function, glucose handling, and cardiac muscle function.

DMD is a rare genetic disorder characterized by progressive muscle degeneration and weakness. The research partnership between Brock University and AMO Pharma was established to explore the potential utility of GSK3β inhibitors in treating DMD. The team of researchers at Brock University investigated the effects of AMO-02 in a well-characterized mouse model of DMD.

The findings showed that AMO-02 treatment improved both muscle function and glucose handling in mice, regardless of the stage of disease progression. Additionally, the treatment demonstrated improvements in metabolic function, muscle fat deposition, and cardiac muscle function. Short-term treatment with AMO-02 also significantly improved cognitive function.

Dr. Val Fajardo, one of the lead researchers, emphasized the importance of these findings, stating that the broad profile of efficacy was observed across multiple organ domains, including the heart and brain, as well as metabolic function and skeletal muscle.

AMO Pharma plans to continue its collaboration with Brock University to further investigate the potential of AMO-02 in treating DMD and other muscle-wasting conditions. The company’s Chief Scientific Officer, Dr. Michael Snape, expressed optimism about the therapy’s potential to improve muscle health and function, as well as its ability to benefit patients with adult-onset myotonic dystrophy.

The promising results from the study of AMO-02 in DMD build upon previously announced results from the REACH-CDM clinical study, which showed efficacy benefits in the treatment of children and adolescents with congenital myotonic dystrophy. AMO Pharma is actively working to develop treatments for rare neurogenetic disorders with limited or no treatment options.

AMO Pharma Website: [LINK]

– 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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