Based on the FDA’s review of a new drug application, a phase 2 study will evaluate the safety and efficacy of azeliragon for patients with glioblastoma.
Trial Name: A Phase I/II Open Label Study to Assess Safety and Preliminary Evidence of a Therapeutic Effect of Azeliragon Combined With Conventional Concurrent Radiation and Temozolomide in Patients With Newly Diagnosed Glioblastoma
ClinicalTrials.gov Identifier: NCT05635734
Sponsor: Cantex Pharmaceuticals
Recruitment Contact: A responsible person Designated by the Sponsor +34 93 434 44 12, investigacion@mfar.net
Completion Date: March 2023
After reviewing the new drug application for a phase 2 trial assessing the safety and efficacy of azeliragon (previously TTP488) in patients with newly diagnosed unmethylated glioblastoma (GBM), the FDA has issued a "study may proceed" letter for the phase 2 clinical trial of the agent.1
Azeliragon is an orally administered, once-a-day pill, which is a small molecule antagonist of the receptor for advanced glycation end products (RAGE). Azeliragon inhibits RAGE interactions with certain ligands, including HMGB1 and S100 proteins in the GBM microenvironment. Treatment with azeliragon has the potential to inhibit GBM and overcome its resistance to effective treatment through preventing the interaction of RAGE with these ligands.
In January 2023, azeliragon was granted an orphan drug designation from the FDA for the treatment of GBM. Now, the agent is being evaluated in a phase 2, open-label study among patients with newly diagnosed GBM.
"We are very pleased to have received this favorable response from the FDA enabling Cantex to initiate a phase 2 clinical trial of azeliragon for the treatment of glioblastoma. Today's news significantly advances Cantex's azeliragon development program. We look forward to soon commencing enrollment in this trial given the urgent need for novel therapies that can better address this devastating disease," stated Stephen G. Marcus, MD, chief executive officer of Cantex Therapeutics, Inc, in the press release.
GBM is the most common type of primary brain cancer. Around 13,000 cases are diagnosed in the United States each year. The highly malignant brain tumor does not currently have many treatment options to extend survival benefit.
Currently, the median survival is 15-18 months for patients with GBM after their diagnosis. With the 5-year survival also less than 10%, there is an urgent unmet need for new treatments for this patient population. Investigators are hopeful that this now approved phase 2 study of azeliragon will provide a new option for patients with GBM.
Previous trials have evaluated azeliragon in over 2000 patients with Alzheimer’s. While these trials did not demonstrate efficacy in Alzheimer's disease, the clinical safety data were promising and showed the agent to be well-tolerated when evaluating patients with Alzheimer’s who were administered up to 18 months of treatment with azeliragon.2
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Cantex is also developing azeliragon for patients with other cancer types where RAGE-mediated resistance and treatment-related complications have led to poor outcomes for patients.1
"The phase 2 trial of azeliragon in glioblastoma is 1 of several promising clinical programs we continue to advance with azeliragon to treat cancer and other diseases where RAGE is implicated. Additional azeliragon indications include pancreatic cancer, breast cancer, and cancers such as lung cancer and breast cancer that have metastasized to the brain. These programs highlight the versatility of azeliragon and our commitment to develop new treatment options for these cancers. We expect significant progress on these clinical trials during 2023 and 2024," added Marcus in the press release.
