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Atossa Highlights Emerging Opportunity for (Z)-Endoxifen in Duchenne Muscular Dystrophy, Including Symptomatic Female Carriers, Following Peer-Reviewed Publication and Scientific Presentation
Published article outlines rationale for multi-pathway efficacy for (Z)-endoxifen in Duchenne Muscular Dystrophy (DMD); November scientific presentation to
About this update from Atossa Therapeutics, Inc.
[{"type":"text","content":"\n Published article outlines rationale for multi-pathway efficacy for (Z)-endoxifen in Duchenne Muscular Dystrophy (DMD); November scientific presentation to spotlight potential in Duchenne carrier–associated pathologies\n \n SEATTLE, Nov. 17, 2025 /PRNewswire/ -- Atossa Therapeutics, Inc. (Nasdaq: ATOS) (\"Atossa\" or the \"Company\"), a clinical-stage biopharmaceutical company developing innovative medicines in oncology, announces a growing body of scientific work supporting the potential role of its investigational therapy (Z)-endoxifen in DMD, a severe, progressive, and ultimately fatal neuromuscular disease, in addition to Duchenne carrier–associated pathologies (D-CAPs) that affect a subset of female carriers. The momentum is anchored by a newly published, peer-reviewed hypothesis article and an upcoming invited scientific presentation.\n \n \n \n \n \n \n \n \n Newly published hypothesis article outlines why (Z)-endoxifen may matter in DMDThe article about (Z)-Endoxifen in Duchenne Muscular Dystrophy (DMD), surveys the DMD treatment landscape and details how (Z)-endoxifen's pharmacology could address multiple downstream drivers of disease, including inflammation, fibrosis, calcium dysregulation, mitochondrial dysfunction, and lipid abnormalities. A video abstract of the paper can be found here.\n The paper emphasizes (Z)-endoxifen's direct estrogen-receptor (ER) modulation, allosteric inhibition of PKC (notably PKC-β1), and effects along AKT/mTOR and NF-κB axes, mechanisms that together may help slow disease progression when used as an adjunct to standard care. Notably, the authors underscore (Z)-endoxifen's potential to deliver more consistent therapeutic exposures than tamoxifen by bypassing CYP2D6 metabolic variability, an important limitation of the pro-drug approach. As illustrated in the mechanistic diagram, page 7 of the publication, the paper maps (Z)-endoxifen's ER-dependent and ER-independent signaling effects relevant to dystrophic muscle.\n \n Clinical context: Prior tamoxifen studies in DMD showed safety and encouraging trends but were underpowered due to premature termination during the pandemic, supporting continued exploration of more potent, exposure-reliable metabolites like (Z)-endoxifen.\n Tissue exposure: (Z)-endoxifen tissue concentrations may substantially exceed plasma levels in certain settings, ...