FDA Orphan Drug Status Granted to Azeliragon for Breast Cancer Brain Metastasis

WESTON, Fla., Dec. 9, 2024 – Cantex Pharmaceuticals, Inc., a company in the clinical development phase, has announced that the U.S. Food and Drug Administration (FDA) has awarded Orphan Drug Designation to its drug azeliragon for treating brain metastasis originating from breast cancer. This new designation follows two previous Orphan Drug Designations for azeliragon, granted for treating pancreatic cancer and glioblastoma in mid-2024 and early 2023, respectively.

Azeliragon, administered orally once a day, works by inhibiting the receptor for advanced glycation end products (RAGE). The interaction between RAGE on cancer cells and S100 proteins, among other ligands, has been linked to resistance to radiation, progression of disease, and metastasis development in breast cancer.

Brain metastasis is a severe complication of breast cancer. Despite significant advancements in extending life for patients with certain types of brain metastasis from breast cancer, triple-negative breast cancer—a particularly aggressive subtype—remains a significant therapeutic challenge, necessitating better treatment options.

Stephen G. Marcus, M.D., Chief Executive Officer of Cantex, commented on the significance of the FDA's designation, highlighting the ongoing need for new treatment options for patients with life-threatening conditions. He emphasized the company's commitment to developing azeliragon as a new treatment option for these patients.

In addition to the existing patents on azeliragon, the FDA's Orphan Drug Designation provides Cantex with seven years of marketing exclusivity once the product is launched for the orphan indication. The designation also offers several other benefits, such as assistance in the drug development process, tax credits for clinical research costs, and exemptions from certain FDA fees.

Azeliragon is an orally administered capsule taken once a day, designed to inhibit interactions between RAGE and specific ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Originally developed by vTv Therapeutics Inc. for Alzheimer's disease, Cantex subsequently licensed the worldwide rights to azeliragon. Clinical safety data from Alzheimer’s trials, which included over 2000 participants dosed for up to 18 months, indicate that azeliragon is well tolerated.

Cantex is currently conducting clinical trials for azeliragon in several areas, including brain metastasis, glioblastoma, breast cancer, pancreatic cancer, and pneumonia in hospitalized patients. These trials are underpinned by strong preclinical data and extensive clinical safety information derived from previous randomized placebo-controlled trials.

Cantex Pharmaceuticals, Inc. is a privately-held, clinical-stage pharmaceutical company dedicated to developing revolutionary therapies for cancer and other severe medical conditions that urgently require new treatments.

vTv Therapeutics Inc. is a biopharmaceutical company in late-stage development, concentrating on developing oral, small molecule drug candidates. Their leading project is cadisegliatin, a potential adjunctive therapy to insulin for type 1 diabetes. vTv and its partners are also exploring additional indications, including type 2 diabetes and other chronic conditions.