ATOS News & Events

"During the quarter, we made meaningful progress advancing our (Z)-endoxifen development strategy across both oncology and rare disease indications," stated Dr. Steven Quay, M.D., Ph.D., Atossa Therapeutics' President and CEO. "We continued to advance (Z)-endoxifen in the clinic for the treatment of breast cancer, while also generating data to support its potential in rare diseases, including Duchenne Muscular Dystrophy (DMD) and McCune-Albright Syndrome. Importantly, we secured both Orphan Drug and Rare Pediatric Disease designations from the FDA for (Z)-endoxifen in DMD, and subsequently we've received Rare Pediatric Disease designation from the FDA for McCune-Albright Syndrome, reinforcing the potential of our programs in areas of high unmet need. Building on this momentum, we remain focused on identifying additional indications where our platform can deliver meaningful therapeutic benefit to patients with limited treatment options." Dr. Quay continued, "Our balance sheet remains strong, positioning us to continue to execute across our strategic plans, and deliver value to shareholders in upcoming quarters."

Atossa Therapeutics announced the publication of results from the KARISMA Endoxifen trial in the Journal of the National Cancer Institute. The Phase 2 study evaluated daily oral Endoxifen and demonstrated that low-dose Endoxifen significantly reduced MBD, a key risk factor for breast cancer. The article highlighted data from a study funded by Atossa. The study evaluated placebo, 1 mg, and 2 mg Endoxifen administered daily for six months in 240 healthy premenopausal women enrolled through the Swedish national mammography screening program. Elevated MBD is an established risk factor for breast cancer and a recognized pharmacodynamic marker of response to endocrine risk-reduction therapy. In the KARISMA Endoxifen trial, both the 1 mg and 2 mg Endoxifen dose levels produced statistically significant reductions in MBD compared with placebo. The 1 mg dose reduced MBD by 19.3% vs. placebo, while the 2 mg dose reduced MBD by 26.5% vs. placebo. These reductions were comparable to those previously reported with standard-dose tamoxifen, but were achieved using direct administration of Endoxifen, the most therapeutically active metabolite of tamoxifen. Both levels demonstrated a favorable tolerability profile. Adverse events were generally vasomotor in nature, consistent with those previously reported for tamoxifen.

Atossa Therapeutics announced that the U.S. FDA has granted Rare Pediatric Disease designation to (Z)-endoxifen for the treatment of McCune-Albright Syndrome in females. RPD designation is granted to drug candidates intended to treat serious or life-threatening diseases that primarily affect individuals from birth to 18 years of age.