SEATTLE, May 20, 2026
Atossa Therapeutics announced that a new scientific manuscript exploring the utrophin-modulation potential of (Z)-endoxifen in Duchenne Muscular Dystrophy (DMD) has been accepted for publication in the journal Degenerative Neurological and Neuromuscular Disease. The publication strengthens the scientific rationale behind the company’s ongoing research into mutation-agnostic treatment strategies for DMD, a rare and progressive neuromuscular disorder with significant unmet medical need.
New Research Supports Utrophin-Based DMD Treatment Strategy
The accepted manuscript, titled “(Z)-Endoxifen as a Potential Modulator of Utrophin Pathways in Duchenne Muscular Dystrophy: A Mechanistic and Transcriptomic Perspective,” examines how (Z)-endoxifen may help regulate biological pathways linked to utrophin, a protein closely related to dystrophin. Since DMD patients lack functional dystrophin, researchers believe utrophin could partially compensate for muscle-cell instability and degeneration.
The paper explains that (Z)-endoxifen, the primary active metabolite of tamoxifen, may promote a cellular environment that supports utrophin expression, localization, and muscle membrane stabilization. Researchers also highlighted potential effects on several disease-relevant biological mechanisms, including protein kinase C beta-1 signaling, estrogen receptor signaling, calcium regulation, inflammation, fibrosis, mitochondrial function, and muscle regeneration.
According to the publication, these findings position (Z)-endoxifen as a promising mechanistic candidate for Duchenne Muscular Dystrophy treatment, particularly because the approach is not dependent on a specific gene mutation. The manuscript also recommends future preclinical studies in dystrophin-deficient models and further biomarker development to evaluate utrophin-related therapeutic effects.
Atossa Expands Rare Disease Research Beyond Oncology
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Atossa Therapeutics stated that this publication builds upon its previously published DMD research article, which first introduced the broader therapeutic rationale for evaluating (Z)-endoxifen in DMD. The new manuscript places stronger emphasis on utrophin biology as a central mechanism connecting the compound’s multiple molecular effects.
Dr. Steven C. Quay, President and CEO of Atossa Therapeutics, said the publication advances the company’s understanding of how (Z)-endoxifen may support muscle stabilization and repair pathways in DMD patients. He emphasized that DMD remains a devastating disease with limited treatment options and significant unmet clinical need.
Duchenne Muscular Dystrophy is caused by mutations in the DMD gene, resulting in the absence of dystrophin, a critical structural protein required for muscle integrity. The disease progressively weakens skeletal and cardiac muscles, leading to loss of mobility, respiratory complications, cardiomyopathy, and premature death.
Regulatory Advantages and Commercial Potential
Atossa’s (Z)-endoxifen program has already received both Orphan Drug Designation and Rare Pediatric Disease designation from the U.S. FDA for DMD treatment. These regulatory designations may provide future development incentives, including eligibility for a Priority Review Voucher (PRV) upon approval. Recent PRV transactions in the biotech sector have reportedly ranged between $100 million and $205 million, adding potential commercial value to the program.
The company noted that its proprietary oral formulation of (Z)-endoxifen has demonstrated a favorable safety profile and distinct pharmacological properties compared with tamoxifen, including ER-targeted activity and PKC inhibition. The drug candidate is currently under investigation across multiple oncology and rare disease settings but has not yet received regulatory approval for any indication.
Source: Atossa Therapeutics press release
