Overview of the Endoxifen Trial#
Atossa Therapeutics Inc, a biopharmaceutical company based in Seattle, has announced the publication of results from its Phase 2 trial of the drug candidate endoxifen. The findings were published in the Journal of the National Cancer Institute. The trial, known as the KARISMA Endoxifen trial, involved 240 healthy premenopausal women in Sweden.
Trial Details and Results#
Participants in the trial were divided into three groups: one received a placebo, while the others received either 1 mg or 2 mg of daily oral endoxifen for six months. The main goal was to measure changes in mammographic breast density, which is a known risk factor for breast cancer. The results showed that the 1 mg dose led to a 19.3% reduction in breast density compared to the placebo group, while the 2 mg dose resulted in a 26.5% reduction.
Safety and Side Effects#
While some participants experienced adverse events related to the study drug, these were generally mild and included symptoms like hot flashes. Specifically, four participants in the placebo group, five in the 1 mg group, and eleven in the 2 mg group discontinued due to these effects. Importantly, no significant changes were observed in various health tests, including blood pressure and heart rate.
Future Directions#
Endoxifen is the active form of tamoxifen, a drug already approved for reducing breast cancer risk. The researchers emphasized the need for further studies to determine if endoxifen can actually lower breast cancer rates in women at higher risk. The trial is registered under ClinicalTrials.gov identifier NCT05068388.
Atossa Therapeutics, which focuses on developing treatments for breast cancer and rare diseases, has faced challenges in the market, with its stock price declining significantly in recent months. However, the company has also received a Rare Pediatric Disease designation from the FDA for another formulation of endoxifen, which could provide future financial benefits.