FDA Grants Orphan Drug Status to Azeliragon for Pancreatic Cancer

Cantex Pharmaceuticals, Inc., a clinical-stage company focused on innovative therapies for cancer and other serious conditions, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to azeliragon, a once-daily oral medication, for the treatment of pancreatic cancer. This designation adds to azeliragon's previous recognition for treating glioblastoma.

Pancreatic cancer is notoriously aggressive and often spreads to other organs if not diagnosed early. Current treatments have limited success once the cancer has metastasized. Therefore, new therapeutic options are crucial. Cantex is currently conducting a clinical trial to assess the safety and effectiveness of azeliragon in patients who have not responded to initial treatments for metastatic pancreatic cancer. This trial is taking place at several esteemed cancer treatment centers across the United States.

Dr. Stephen G. Marcus, CEO of Cantex, emphasized the importance of this FDA designation, stating that it highlights the urgent need for new treatment options, especially for patients with advanced and inoperable pancreatic cancer. He noted that this status continues to bolster Cantex's dedication to developing new uses for azeliragon, not only for pancreatic cancer but also for other cancers and their complications, including glioblastoma, brain metastasis, and breast cancer.

The Orphan Drug Designation by the FDA offers several benefits to Cantex, including seven years of market exclusivity for azeliragon upon its commercial launch for the designated use. Additionally, the company will receive assistance with the drug development process, tax credits for clinical trial costs, and exemptions from certain FDA fees.

Azeliragon is an orally administered capsule designed to inhibit interactions between the receptor for advanced glycation end products (RAGE) and specific ligands, such as HMGB1 and S100 proteins, within the tumor microenvironment. Initially developed by vTv Therapeutics Inc. for Alzheimer’s disease, the rights to azeliragon were subsequently licensed to Cantex. Clinical trials involving more than 2000 individuals have shown that azeliragon is well tolerated, even when administered for up to 18 months.

Cantex is actively running Phase 2 clinical trials of azeliragon for pancreatic cancer, glioblastoma, brain metastasis, and breast cancer, and a Phase 3 trial for hospitalized patients with pneumonia. These trials are supported by substantial preclinical data and extensive clinical safety information from earlier placebo-controlled studies.

Cantex Pharmaceuticals, Inc. remains committed to developing transformative therapies for cancer and other life-threatening conditions, emphasizing the urgent need for new treatments in these areas.

vTv Therapeutics Inc., the original developer of azeliragon, continues to focus on advancing oral small-molecule drug candidates, including cadisegliatin, a promising treatment aimed at reducing hypoglycemic episodes in type 1 diabetes patients.