Pharma Focus Europe

AMO Pharma and Population Health Research Institute Collaborate on Tideglusib Clinical Trial for Arrhythmogenic Cardiomyopathy Treatment Efficacy Assessment

Friday, February 16, 2024

AMO Pharma Limited, a leading clinical-stage biopharmaceutical company specializing in rare genetic disorders, has announced a groundbreaking collaboration with the Population Health Research Institute (PHRI) of McMaster University and Hamilton Health Sciences in Canada. This partnership aims to conduct a clinical proof-of-concept trial for assessing the effectiveness of AMO-02 (tideglusib), an investigational oral inhibitor of glycogen synthase kinase 3 beta (GSK3β), in treating arrhythmogenic cardiomyopathy (ACM) among genotype-positive individuals.

The study, titled "Targeted Therapy with Glycogen Synthase Kinase-3 Inhibition for Arrhythmogenic Cardiomyopathy" (TaRGET), will be overseen by PHRI's research team, with collaboration from various organizations including the Hearts in Rhythm Organization (HiRO). Scheduled to commence enrollment in mid-2024 across 20 sites in Canada, the trial aims to recruit 120 participants, who will be randomly assigned in a 1:1 ratio.

Dr. Jason Roberts, a scientist at PHRI and the principal investigator of the TaRGET study, expressed optimism about tideglusib's potential based on previous animal studies. He emphasized the urgent need for effective treatments for ACM, a genetic heart condition characterized by heart muscle scarring that increases the risk of life-threatening heart rhythms and sudden cardiac death. Currently, there are no approved therapies for halting ACM progression, with implantable cardioverter defibrillators (ICDs) recommended for high-risk patients.

Earlier research demonstrated the efficacy of a GSK3β inhibitor in preventing and reversing ACM-related symptoms in animal models. GSK3β plays a crucial role in regulating various intracellular signaling pathways implicated in ACM pathogenesis, including the Wnt/β-catenin pathway.

AMO Pharma, underscored the company's commitment to advancing research on AMO-02, particularly in the context of myotonic dystrophy. He expressed enthusiasm for supporting this significant ACM research endeavor with PHRI and highlighted the potential of tideglusib to offer new therapeutic options for individuals and families affected by ACM. Snape also noted the promising cardiac efficacy data from previous studies in Duchenne muscular dystrophy, led by Professor Val Fajardo's team at Brock University, which informs the current investigation's direction.



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