Atossa Highlights Emerging Opportunity for (Z)-Endoxifen in Duchenne Muscular Dystrophy, Including Symptomatic Female Carriers, Following Peer-Reviewed Publication and Scientific Presentation

Core Insights - Atossa Therapeutics is advancing its investigational therapy (Z)-endoxifen for Duchenne Muscular Dystrophy (DMD) and associated pathologies, supported by new scientific publications and presentations [1][2][5] Mechanism and Efficacy - The published hypothesis article outlines how (Z)-endoxifen's pharmacology may address multiple disease drivers in DMD, including inflammation, fibrosis, and mitochondrial dysfunction [2][3] - (Z)-endoxifen modulates estrogen receptors and inhibits PKC, potentially slowing disease progression when used alongside standard care [3][6] - The therapy may provide more consistent therapeutic exposure compared to tamoxifen by avoiding CYP2D6 metabolic variability [3] Research Developments - A second manuscript is under review, focusing on (Z)-endoxifen's role in modulating utrophin expression, which could offer a mutation-agnostic therapeutic approach for DMD [4] - Atossa plans to leverage the published framework for preclinical validation and clinical study design, assessing safety and pharmacodynamics relevant to muscle and cardiac performance [9] Target Population - The upcoming presentation will address symptomatic female carriers of DMD, a group that may experience significant health issues, with 2.5–19% showing skeletal muscle symptoms and 7.3–16.7% developing dilated cardiomyopathy [5][10] Strategic Outlook - The company emphasizes the need for a multi-pathway approach to DMD treatment, positioning (Z)-endoxifen as a potentially scalable and accessible option alongside genetic therapies [15] - Management highlights the importance of rigorous testing and regulatory engagement to accelerate development for patients [7][8]