WESTON, Fla., March 6, 2023 /PRNewswire/ -- Cantex Pharmaceuticals, Inc., 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.

About Azeliragon
Azeliragon, previously known as TTP488, is an orally active, small molecule, antagonist of the receptor for advanced glycation end products (RAGE) licensed by Cantex from vTv Therapeutics Inc. (NASDAQ:VTVT). vTv Therapeutics discovered azeliragon and carried out phase 3 clinical trials for Alzheimer's disease. Although these trials did not demonstrate efficacy in Alzheimer's disease, clinical safety data from these trials, involving over 2000 patients dosed for periods up to 18 months, indicate that azeliragon is very well tolerated. 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.

About Cantex Pharmaceuticals, Inc. 
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.

About Miami Cancer Institute

Miami Cancer Institute brings to South Florida access to personalized clinical treatments and comprehensive support services delivered with unparalleled compassion. No other cancer program in the region has the combination of cancer-fighting expertise and advanced technology—including the first proton therapy center in South Florida, Latin America and the Caribbean, and one of the only radiation oncology programs in the world with each of the newest radiation therapies in one place—to diagnose and deliver precise cancer treatments that achieve the best outcomes and improve the lives of cancer patients. The Institute offers an impressive roster of established community oncologists and renowned experts, clinical researchers and genomic scientists recruited from the nation's top cancer centers. Selected as Florida's only member of the Memorial Sloan Kettering Cancer (MSK) Alliance, Miami Cancer Institute is part of a meaningful clinical collaboration that affords patients in South Florida access to innovative treatments and ensures that the standards of care developed by their multidisciplinary disease management teams match those at MSK. For more information, please visit https://cancer.baptisthealth.net/miami-cancer-institute.

Miami Cancer Institute is part of Baptist Health Cancer Care, the largest cancer program in South Florida, with locations from the Florida Keys to the Palm Beaches.

Contacts:

Miami Cancer Institute, part of Baptist Health Cancer Care
Bianca Hernandez
305.846.6118

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Stephen G. Marcus, M.D.
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