MAIA Biotechnology (NYSE: MAIA) Has Received FDA Orphan Drug Designation For THIO As A Treatment For Most Aggressive Brain Cancer

More than 300,000 people are diagnosed with brain cancer across the globe each year. Glioblastoma represents the majority of these cases in the U.S., with 15,000 new patients diagnosed and more than 10,000 deaths annually. 

A potentially revolutionary therapy option could help treat the many individuals who are fighting glioblastoma, the most aggressive and common type of cancer that originates in the brain.

Earlier in November, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to a cancer telomere-targeting agent for the treatment of glioblastoma. MAIA Biotechnology Inc. MAIA is the company behind THIO, a first-in-class cancer telomere-targeting agent with the potential to revolutionize the treatment of glioblastoma. 

This is the third orphan drug designation granted to THIO, following the receipt of orphan drug designations for hepatocellular carcinoma (HCC) and small-cell lung cancer (SCLC) in 2022. Receiving three designations highlights the FDA’s recognition of THIO’s potential to treat multiple types of cancers, including rare ones like glioblastoma.

A form of brain cancer with limited treatment options and a poor prognosis, glioblastoma is the most aggressive and most common type of cancer that originates in the brain. With very limited treatment options available, just 7% of patients survive for more than five years after being diagnosed. 

MAIA’s lead program, THIO has the potential to improve outcomes for glioblastoma patients.

“In the data presented to the FDA, THIO successfully penetrated the blood-brain barrier (BBB) in syngeneic and humanized mouse models of telomerase-expressing brain cancers. Treatment with THIO resulted in potent anticancer activity and significant expansion of the animal lifespan for several difficult-to-treat cell lines and xenograft mouse models,” said Sergei Gryaznov, Ph.D., MAIA’s Chief Scientific Officer. “These results stem from THIO’s remarkable mechanism of action and its BBB penetrating property that allows for direct targeting of brain tumors in vivo and potentially in glioblastoma patients.”

The FDA’s Orphan Drug Act of 1983 was designed to incentivize the development of therapies that demonstrate promise for the treatment of rare (orphan) diseases or conditions, those that affect fewer than 200,000 people total in the U.S. About 15,000 people are diagnosed with glioblastoma in the U.S. annually. 

With the orphan drug designation from the FDA, MAIA will receive a number of financial incentives, including up to seven years of market exclusivity for THIO, if approved. This would help MAIA capitalize on the glioblastoma market, expected to grow from $2.2 billion to $3.2 billion globally in the next three years. 

MAIA Biotechnology reports that THIO is the only direct telomere-targeting agent currently in clinical development. Focused on the development and commercialization of drugs that improve and extend the lives of people with cancer, MAIA is currently conducting a phase 2 clinical trial of THIO in patients with advanced Non-Small Cell Lung Cancer (NSCLC). 

On Oct. 24, MAIA Biotechnology reported an unprecedented Disease Control Rate (DCR) in second-line treatment for NSCLC in its ongoing phase 2 trial, THIO-101. The promising preliminary efficacy data included a 100% Disease Control Rate (DCR) observed in the second-line treatment of NSCLC, a number that dramatically surpasses the standard-of-care DCR, which ranges from 53% to 64%. KEYTRUDA, a prescription medicine used to treat NSCLC, has a DCR of 71% in first-line patients. 

Featured photo by benjamin lehman on Unsplash.

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