WESTON, Fla., Jan. 9, 2023 /PRNewswire/ -- Cantex Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions for which new treatments are urgently needed, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to Cantex' azeliragon, a well-tolerated once-a-day pill, for the treatment of glioblastoma.

Glioblastoma is a highly malignant primary brain tumor for which current therapeutic options provide a limited life extension benefit. New treatments of glioblastoma are urgently needed.  

Azeliragon is an orally administered small molecule, taken once daily, that inhibits the receptor for advanced glycation endproducts (known as RAGE) interactions with certain ligands, including HMGB1 and S100 proteins in the glioblastoma microenvironment. By preventing interaction of RAGE with these ligands, azeliragon may inhibit glioblastoma and overcome its resistance to effective treatment. Azeliragon was originally under development for Alzheimer's disease by the company from which Cantex licensed it. Clinical safety data from these trials, involving more than 2000 individuals dosed for periods up to 18 months, indicate that azeliragon is very well tolerated. Cantex is also developing azeliragon for the treatment of other major cancers in need of improved treatments where RAGE has been implicated in disease progression and in complications of cancer treatment.

"Receiving FDA orphan drug status for azeliragon highlights the significant unmet need for novel treatment options for patients with glioblastoma, the most common and lethal primary brain cancer," commented Stephen G. Marcus, M.D., Chief Executive Officer of Cantex. "This designation validates our continued commitment to developing new treatment options for patients with glioblastoma, as well as for other cancers and their complications, including breast cancer, cancer chemotherapy related cognitive decline, pancreatic cancer, and cancers, such as breast and lung cancer, that have metastasized to the brain."

FDA Orphan Drug Designation provides 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.

Azeliragon, previously known as TTP488, is an orally active, small molecule, antagonist of the receptor for advanced glycation endproducts (RAGE) licensed by Cantex Pharmaceuticals, Inc. from vTv Therapeutics Inc. (NASDAQ: VTVT). vTv Therapeutics discovered azeliragon and developed it into phase 3 clinical trials. A broad range of evidence suggests that RAGE—ligand interactions play a critical role in cancer and its complications as well as in a range of inflammatory diseases.

Cantex Pharmaceuticals is a privately held, clinical-stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions for which new treatments are urgently needed. For more information, please visit www.cantex.com.

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Stephen G. Marcus, M.D. 
+1 954-315-3660