Cantex and Miami Cancer Institute Announce FDA Clearance for a Clinical Trial Investigating Azeliragon Combined with Stereotactic Radiosurgery for the Treatment of Brain Metastases

Cantex Pharmaceuticals, a clinical-stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions, and Baptist Health Miami Cancer Institute, part of Baptist Health Cancer Care, the largest cancer program in South Florida, announced today that the U.S. Food and Drug Administration (FDA) has issued a “Study May Proceed” letter for the Miami Cancer Institute’s investigator initiated clinical trial to assess the safety and effectiveness of azeliragon combined with stereotactic radiosurgery in patients with brain metastases.

“Brain metastases are a devastating diagnosis for many people in their cancer journey,” said Rupesh Kotecha, M.D., the principal investigator of the trial and Chief of Radiosurgery in the Department of Radiation Oncology and Director of the Central Nervous System Metastasis program at Miami Cancer Institute. “At Miami Cancer Institute, we are fortunate in that we have the latest radiotherapy technologies used to treat brain metastases. Stereotactic radiosurgery allows the radiation oncologist to deliver an intense, precisely targeted dose of radiation to tumor cells inside the brain while sparing healthy surrounding tissues including the brain itself, arteries, nerves and other important structures. Typically, patients receive corticosteroids along with the treatment to reduce the risk of peri-tumoral edema and inflammation. The clinical trial of azeliragon, with its differentiated mechanism of action, will offer patients a unique approach to the treatment of brain metastases that may protect against inflammation as well as potentiate radiation response in the tumor. This clinical trial represents a novel way of enhancing the effectiveness of radiation therapy of cancer.”

Cantex’s azeliragon is an orally administered small molecule, taken as a pill once daily, that is being studied as a treatment for glioblastoma, brain metastases, and other cancers. Azeliragon inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the cancer microenvironment. S100A9, a protein released from cells within the microenvironment of brain metastasis, binds to RAGE receptors on the surface of cancer cells, making them resistant to the effectiveness of radiation treatments. Azeliragon blocks the interaction of S100A9 with RAGE, potentially restoring sensitivity to radiation therapy. In addition, interaction of RAGE with proteins that bind to it, trigger inflammatory responses, which may worsen brain swelling associated with radiation therapy. The Miami Cancer Institute’s investigator-initiated trial is designed to determine whether azeliragon, when administered in combination with stereotactic radiosurgery, can prevent or eliminate the need for a powerful steroid, dexamethasone, to control brain swelling as well as improve the effectiveness of radiation therapy to treat brain metastases.

Brain metastases occur when cancer spreads from their original location. Brain metastases are the most common type of cancer in the brain, with over 150,000 people each year developing brain metastasis from their primary cancer (most commonly lung and breast cancer).

“We are excited to receive FDA authorization to proceed with this trial. With this approval, Cantex expands the cancer indications for which azeliragon is being investigated, including glioblastoma, breast cancer, and pancreatic cancer, furthering our work to better understand azeliragon as a treatment of very aggressive and difficult to treat cancers,” said Stephen G. Marcus, M.D., Chief Executive Officer of Cantex. “The team of renowned oncologists at Miami Cancer Institute is committed to expanding innovative, cutting-edge tools for cancer treatment as indicated by their interest in RAGE inhibition and treatment of brain metastases.”

Cantex recently announced that the FDA had provided the company with a “Study May Proceed” letter to assess azeliragon for the treatment of glioblastoma, the most common primary brain cancer. Azeliragon was also recently granted FDA Orphan Drug Designation for the treatment of glioblastoma.