Atossa Therapeutics Targets Rare McCune-Albright Syndrome

The research, presented by Dr. Sandra Hammer in Vancouver, focuses on the syndrome’s underlying GNAS mutations, which drive chronic proliferative signaling. While current treatments primarily aim to suppress estrogen production, they often fail to address downstream cellular activity. The study indicates that (Z)-endoxifen may bridge this gap by simultaneously modulating estrogen receptors and inhibiting PKC-β1, effectively promoting the dephosphorylation and degradation of proliferative signals.

Atossa CEO Dr. Steven C. Quay noted that these results offer a potential path forward for patients facing peripheral precocious puberty, a hallmark symptom of the syndrome. By integrating transcriptomic and kinase-network data, the company is positioning (Z)-endoxifen as a mechanism-driven candidate capable of addressing not only rare endocrine diseases but potentially broader estrogen-driven tumor types as well.