Atossa Therapeutics (NASDAQ: ATOS) has unveiled new preclinical data for its investigational drug, (Z)-Endoxifen, as a potential treatment for McCune-Albright syndrome (MAS). The findings were presented in a poster session at a recent special conference on rare cancers hosted by the American Association for Cancer Research (AACR). This research points to a novel therapeutic strategy for this rare endocrine disorder that primarily affects children and adolescents.
The released data underscore the potential dual mechanism of action of (Z)-Endoxifen. McCune-Albright syndrome is primarily caused by mutations in the GNAS gene, leading to autonomous overproduction of estrogen and resulting in issues such as peripheral precocious puberty. The study indicates that (Z)-Endoxifen, as a potent selective estrogen receptor modulator/degrader, not only blocks estrogen receptor-mediated transcriptional activity but also effectively inhibits PKC-β/AKT-related proliferation and cell cycle signaling pathways.
Dr. Steven Quay, President and CEO of Atossa, stated that peripheral precocious puberty associated with MAS is a rare disease with a significant unmet medical need. Existing therapies mainly focus on reducing estrogen production but may not fully address the downstream estrogen receptor dependency and proliferative signaling. He believes these data support (Z)-Endoxifen as a mechanism-driven, differentiated candidate capable of simultaneously modulating estrogen receptor biology and PKC-β/AKT-related cell cycle signaling.
Based on its potential, the U.S. Food and Drug Administration (FDA) has previously granted (Z)-Endoxifen a Rare Pediatric Disease designation for the treatment of MAS. This designation means that if the drug is eventually approved, Atossa would be eligible to receive a Priority Review Voucher. This voucher can be sold or transferred, with a market value estimated between $100 million and $200 million, offering a potential avenue for non-dilutive financing.
Atossa Therapeutics is a clinical-stage biopharmaceutical company focused on developing innovative therapies for significant unmet needs in oncology. Its lead candidate, (Z)-Endoxifen, is currently being developed for multiple indications.
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