Adenylosuccinic Acid Is a Non-Toxic Small Molecule In Vitro and In Vivo

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Timpani, Cara ORCID: 0000-0003-4567-4319, Rasmussen, Lorna and Rybalka, Emma ORCID: 0000-0002-4854-0036 (2023) Adenylosuccinic Acid Is a Non-Toxic Small Molecule In Vitro and In Vivo. Pharmaceuticals, 16 (10). ISSN 1424-8247


Adenylosuccinic acid (ASA) is a small molecule dicarboxylate that could be a strong clinical development candidate for inherited myopathies involving dysregulated purine nucleotide metabolism. Currently, there are no published pharmacokinetic/dynamic or toxicology data available, although 10-year clinical trial data on Duchenne muscular dystrophy patients suggests it is a chronically safe drug. In this study, we tested the toxicity of ASA to cultured myoblasts in vitro and its acute systemic toxicity in mice. ASA is a non-toxic small molecule with an LD50 > 5000 mg/kg. Some background necrotic foci in the liver, kidney and gastrointestinal tract were shown that are likely incidental but warrant follow-up sub-/chronic oral exposure studies.

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Item type Article
DOI 10.3390/ph16101458
Official URL
Subjects Current > FOR (2020) Classification > 3209 Neurosciences
Current > Division/Research > Australian Institute of Musculoskeletal Science (AIMSS)
Current > Division/Research > Institute for Health and Sport
Keywords small molecule metabolite, muscular dystrophy, in vitro, in vivo
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