Atossa’s Manuscript on (Z)-Endoxifen Utrophin Modulation Accepted for Publication
ATOS•Atossa Therapeutics announced the acceptance of its manuscript on (Z)-endoxifen’s utrophin-modulation potential in Duchenne muscular dystrophy for publication in Degenerative Neurological and Neuromuscular Disease. The review outlines next steps including preclinical evaluation in dystrophin-deficient models and development of biomarkers measuring utrophin expression, calcium handling, and muscle composition.
1. Manuscript Acceptance Highlights Utrophin Pathway Focus
Atossa’s manuscript on (Z)-endoxifen as a potential modulator of utrophin pathways in Duchenne muscular dystrophy was accepted for publication in Degenerative Neurological and Neuromuscular Disease, emphasizing utrophin’s role as a dystrophin homolog to support muscle-cell membrane stability.
2. Mechanistic Insights of (Z)-Endoxifen
The review details how (Z)-endoxifen may enhance utrophin expression and localization by influencing protein kinase C beta-1 signaling, estrogen receptor pathways, calcium homeostasis, inflammatory responses, fibrosis, mitochondrial function, and muscle regeneration.
3. Planned Preclinical Evaluation and Biomarker Development
Atossa plans preclinical studies in dystrophin-deficient models along with biomarker development focused on utrophin expression and localization, calcium handling, PKC activity, developmental myosin levels, transcriptomic signatures, and muscle composition imaging.
4. Regulatory Incentives via RPD Designation and PRV
(Z)-Endoxifen holds Rare Pediatric Disease designation for Duchenne muscular dystrophy, potentially granting a Priority Review Voucher upon approval, with recent sales of such vouchers ranging from $100 million to $205 million.
