Atossa Therapeutics, a clinical-stage biopharmaceutical company developing novel therapies in oncology and other areas of significant unmet clinical need, has announced that a manuscript titled, ‘(Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy: A Mechanistic and Transcriptomic Perspective’ has been accepted for publication in Degenerative Neurological and Neuromuscular Disease.
The review centres on utrophin, a structural and functional homolog of dystrophin. Utrophin may partially compensate for dystrophin deficiency by supporting sarcolemmal and muscle-cell membrane stability when dystrophin is absent or deficient.
The manuscript describes how (Z)-endoxifen, the primary active metabolite of tamoxifen, could create a cellular environment that favours utrophin expression, localization, and function, which could potentially influence disease-relevant processes, such as protein kinase C beta-1 signalling, oestrogen receptor signalling, calcium homeostasis, inflammation, fibrosis, mitochondrial function, and muscle regeneration.
The authors describe (Z)-endoxifen as a compelling mechanistic candidate in treating DMD and identified next steps including preclinical evaluation in dystrophin-deficient models and biomarker development focused on utrophin expression and localization, calcium handling, PKC activity, developmental myosin, transcriptomic signatures, and muscle composition imaging.
The manuscript was authored by H Lawrence Remmel, Sandra S Hammer, Scott M Blackburn, and Steven C Quay.
