Atossa Therapeutics announced that its investigational drug (Z)-endoxifen may be applicable to Duchenne muscular dystrophy (DMD) and Duchenne carrier‑associated pathologies (D‑CAPs). The announcement was made on November 17, 2025, and the company released a peer‑reviewed hypothesis article and scheduled a presentation at the 2nd International Conference on Women’s Health, Reproduction & Obstetrics in Rome, Italy, from November 17‑19, 2025.
The hypothesis article, originally published in March 2025, outlines how (Z)-endoxifen’s estrogen‑receptor modulation, PKC inhibition, and effects on AKT/mTOR and NF‑κB pathways could address multiple disease drivers in DMD, including inflammation, fibrosis, calcium dysregulation, mitochondrial dysfunction, and lipid abnormalities. It also discusses the drug’s potential to upregulate utrophin, a dystrophin analog, offering a mutation‑agnostic therapeutic strategy.
Atossa plans to use the mechanistic framework to guide preclinical validation and design of future clinical studies that will assess safety, pharmacokinetics, pharmacodynamics, and functional endpoints in upper‑limb, diaphragmatic, and cardiac muscle. The company views this expansion as a strategic shift that could open a new therapeutic area and address a severe unmet need, potentially diversifying its pipeline beyond oncology.
CEO Steven Quay emphasized the scientific rationale, noting that “Duchenne remains one of the highest‑need pediatric diseases. The science points to a coherent, multi‑pathway rationale for (Z)-endoxifen that is distinct from, and potentially synergistic with, genetic strategies.” Senior Vice President of R&D Janet Rea added that the company aims to engage regulators early and pursue rare‑disease pathways to accelerate development for patients and families who have waited too long.
The presentation in Rome will focus on Duchenne carrier‑associated pathologies, where unmet need and biological plausibility intersect. The published hypothesis provides the mechanistic scaffolding, and the next step is to translate this into data‑driven clinical exploration. While no market reaction has been reported yet, the announcement signals Atossa’s intent to broaden its therapeutic portfolio and could position the company to capture a portion of the DMD market, which is estimated to be worth several billion dollars annually.
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