CANTEX PHARMACEUTICALS RECEIVES FDA ORPHAN DRUG DESIGNATION FOR AZELIRAGON FOR THE TREATMENT OF BRAIN METASTASIS FROM BREAST CANCER

Rhea-AI Summary

Cantex Pharmaceuticals has received FDA Orphan Drug Designation for azeliragon to treat brain metastasis from breast cancer. This is the third such designation for azeliragon, following previous designations for pancreatic cancer and glioblastoma in mid-2024 and early 2023.

Azeliragon is an oral, once-daily compound that inhibits the RAGE receptor. The drug's interaction with RAGE on cancer cells has been linked to radiation resistance, disease progression, and metastasis development in breast cancer. This designation provides Cantex with seven years of marketing exclusivity upon product launch, along with development assistance, tax credits, and FDA fee exemptions.

Positive

• Received FDA Orphan Drug Designation for brain metastasis treatment
• Third Orphan Drug Designation for azeliragon, showing expanded therapeutic potential
• Seven years of marketing exclusivity upon product launch
• Additional benefits including tax credits and FDA fee exemptions

Negative

• None.

Insights

Medical Research Analyst

The FDA's Orphan Drug Designation for azeliragon in treating brain metastasis from breast cancer is scientifically significant. The drug's mechanism targeting RAGE receptors represents an innovative approach, particularly promising for triple-negative breast cancer patients where treatment options are Previous orphan designations for pancreatic cancer and glioblastoma suggest broader therapeutic potential. Clinical development benefits include 7-year marketing exclusivity, tax credits and FDA fee exemptions - valuable assets that enhance the drug's commercial viability. The oral once-daily administration format offers practical advantages over more invasive treatments. However, it's important to note this is an early-stage development and successful clinical trials will be important for market approval.

Financial Analyst

This development has strategic implications for vTv Therapeutics (VTVT), the original developer of azeliragon. While Cantex now holds the rights, VTVT could potentially benefit from milestone payments or royalties depending on their agreement terms. The orphan drug designation's market exclusivity period and financial incentives significantly enhance the commercial prospects. For a micro-cap company with $41.3M market value, any revenue from azeliragon's success could be material. The multiple orphan designations across different indications expand the potential market opportunity. However, investors should note that clinical development in the brain metastasis space is historically challenging with high failure rates.

WESTON, Fla., Dec. 9, 2024 /PRNewswire/ -- Cantex Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions, announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to Cantex's azeliragon for the treatment of brain metastasis from breast cancer. This new azeliragon designation adds to azeliragon's two other Orphan Drug Designations for the treatment of pancreatic cancer and glioblastoma, received in mid-2024 and early 2023, respectively.

Cantex's azeliragon is a well-tolerated compound, administered orally once-a-day, that inhibits the receptor for advanced glycation end products (known as RAGE). The binding of RAGE on cancer cells to S100 proteins and other ligands has been linked to resistance to radiation, disease progression, and development of metastasis in breast cancer.

The development of brain metastasis is a life-threatening complication of breast cancer. Although critically important life-extending advances in the treatment of brain metastasis from some forms of breast cancer have recently been made, brain metastasis from triple-negative breast cancer, an aggressive subtype of breast cancer, remains a therapeutic challenge greatly in need of improved treatments.

"Receiving FDA Orphan Drug Designation for azeliragon for the treatment of brain metastasis from breast cancer highlights a continued need for new treatment options for these patients," commented Stephen G. Marcus, M.D., Chief Executive Officer of Cantex. "This designation reflects our continued commitment to developing new azeliragon treatment options for patients with life-threatening cancer."

In addition to azeliragon's already issued composition of matter and other patents, FDA Orphan Drug Designations provide 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.

About Azeliragon
Azeliragon is an orally administered capsule, taken once daily, that inhibits interactions of the receptor for advanced glycation end products (known as RAGE) with certain ligands, including HMGB1 and S100 proteins in the tumor microenvironment. Azeliragon was discovered by and originally under development for Alzheimer's disease by vTv Therapeutics Inc. (NASDAQ: VTVT) from which Cantex licensed worldwide rights to azeliragon. Clinical safety data from those trials, involving more than 2000 individuals dosed for periods up to 18 months, indicate that azeliragon is very well tolerated.

Cantex has ongoing clinical trials in brain metastasis, glioblastoma, breast cancer, pancreatic cancer, and hospitalized patients with pneumonia. These trials are based on azeliragon's robust preclinical data as well as its extensive clinical safety information from randomized placebo-controlled clinical trials.

About Cantex Pharmaceuticals, Inc.
Cantex Pharmaceuticals, Inc. 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 vTv Therapeutics Inc.
vTv Therapeutics Inc. is a late-stage biopharmaceutical company focused on developing oral, small molecule drug candidates. vTv's clinical pipeline is led by cadisegliatin, a potential adjunctive therapy to insulin for the treatment of type 1 diabetes patients. vTv and its development partners are investigating additional indications including type 2 diabetes and other chronic conditions. To learn more please visit vtvtherapeutics.com.

Contacts:

Cantex Pharmaceuticals, Inc.

Stephen G. Marcus, M.D.
+1 954-315-3660
info@cantex.com

Juan F. Rodriguez
+1 954-315-3660
info@cantex.com

Tiberend Strategic Advisors, Inc.
Investors
Jon Nugent
+1 646-577-8520
jnugent@tiberend.com

Media
Casey McDonald
+1 646-577-8520
cmcdonald@tiberend.com

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SOURCE Cantex Pharmaceuticals, Inc.

FAQ

What is the new FDA Orphan Drug Designation granted to Cantex's azeliragon?

Azeliragon received FDA Orphan Drug Designation for the treatment of brain metastasis from breast cancer.

How many Orphan Drug Designations does azeliragon now have?

Azeliragon now has three Orphan Drug Designations: for brain metastasis from breast cancer, pancreatic cancer (mid-2024), and glioblastoma (early 2023).

What are the benefits of the Orphan Drug Designation for Cantex?

The designation provides seven years of marketing exclusivity upon product launch, assistance in drug development, tax credits for clinical costs, and exemptions from certain FDA fees.

How is azeliragon administered to patients?

Azeliragon is administered as a well-tolerated, oral, once-daily compound.