On December 9, 2024 Cantex Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions, reported that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to Cantex’s azeliragon for the treatment of brain metastasis from breast cancer (Press release, Cantex, DEC 9, 2024, View Source [SID1234648940]). 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.
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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 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., Chief Executive Officer 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.
About Azeliragon
Azeliragon is an orally administered capsule, taken once daily, that inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Azeliragon was discovered by and originally under development for Alzheimer’s disease by vTv Therapeutics Inc. (NASDAQ: VTVT) from which Cantex licensed worldwide rights to azeliragon. Clinical safety data from those trials, involving more than 2000 individuals dosed for periods up to 18 months, indicate that azeliragon is very well tolerated.
Cantex has ongoing clinical trials in brain metastasis, glioblastoma, breast cancer, pancreatic cancer, and hospitalized patients with pneumonia. These trials are based on azeliragon’s robust preclinical data as well as its extensive clinical safety information from randomized placebo-controlled clinical trials.