Cantex Pharmaceuticals, Inc. has received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for azeliragon, aimed at treating brain metastasis originating from breast cancer. This designation marks the third orphan drug status for azeliragon, following previous designations for pancreatic cancer and glioblastoma.
Azeliragon is an orally administered compound designed to inhibit the receptor for advanced glycation end products (RAGE). The RAGE receptor, when bound to S100 proteins and other ligands on cancer cells, has been implicated in radiation resistance, disease advancement, and the development of metastasis in breast cancer. By blocking this interaction, azeliragon aims to improve treatment outcomes for patients with brain metastasis.
Addressing Unmet Needs in Breast Cancer Treatment
Brain metastasis represents a severe complication of breast cancer, particularly challenging in aggressive subtypes like triple-negative breast cancer. While advancements have been made in treating brain metastasis from some breast cancer forms, triple-negative breast cancer remains a significant therapeutic hurdle.
"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," said 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."
Benefits of Orphan Drug Designation
The FDA's Orphan Drug Designation not only grants azeliragon seven years of market exclusivity upon launch for the specified indication but also offers Cantex advantages such as aid in the drug development process, tax incentives for clinical trial expenses, and waivers for certain FDA fees.
Azeliragon's Mechanism and Clinical Development
Azeliragon functions by disrupting the interactions between RAGE and ligands like HMGB1 and S100 proteins within the tumor microenvironment. Originally developed by vTv Therapeutics Inc. for Alzheimer's disease, Cantex Pharmaceuticals licensed the worldwide rights to azeliragon. Prior clinical trials involving over 2000 participants have indicated that azeliragon is generally well-tolerated, with dosing periods extending up to 18 months.
Cantex Pharmaceuticals is currently conducting clinical trials to evaluate azeliragon's efficacy in treating brain metastasis, glioblastoma, breast cancer, pancreatic cancer, and pneumonia in hospitalized patients. These trials are supported by preclinical data and safety information derived from randomized, placebo-controlled clinical trials.
