The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Cantex Pharmaceuticals Inc.’s azeliragon for the treatment of brain metastasis from breast cancer. This new azeliragon designation adds to azeliragon’s two other Orphan Drug Designations for the treatment of pancreatic cancer and glioblastoma, received in mid-2024 and early 2023, respectively.
 
Cantex’s azeliragon is a well-tolerated compound, administered orally once a day, that inhibits the receptor for advanced glycation end products (known as RAGE). The binding of RAGE on cancer cells to S100 proteins and other ligands has been linked to resistance to radiation, disease progression, and the development of metastasis in breast cancer.
 
The development of brain metastasis is a life-threatening complication of breast cancer. Although critically important life-extending advances in the treatment of brain metastasis from some forms of breast cancer have recently been made, brain metastasis from triple-negative breast cancer, an aggressive subtype of breast cancer, remains a therapeutic challenge greatly in need of improved treatments.
 
“Receiving FDA Orphan Drug Designation for azeliragon for the treatment of brain metastasis from breast cancer highlights a continued need for new treatment options for these patients,” commented Stephen G. Marcus, M.D., CEO of Cantex. “This designation reflects our continued commitment to developing new azeliragon treatment options for patients with life-threatening cancer.”
 
In addition to azeliragon’s already issued composition of matter and other patents, FDA Orphan Drug Designations provide Cantex with seven years of azeliragon marketing exclusivity from the time of product launch for the orphan indication, and several other important benefits, including assistance in the drug development process, tax credits for clinical costs, and exemptions from certain FDA fees.