The U.S. FDA has granted orphan drug designation to MAIA Biotechnology, Inc., for its lead asset THIO, a cancer telomere-targeting agent for the treatment of glioblastoma. This is the third orphan drug designation granted to THIO, including designations for hepatocellular carcinoma (HCC) and small cell lung cancer (SCLC) in 2022.

A Phase 2 trial of THIO, THIO-101, is evaluating the drug candidate in patients with advanced non-small cell lung cancer (NSCLC). THIO is the only direct telomere targeting agent currently in clinical development.

“We are pleased to receive a third orphan drug designation for THIO, further highlighting FDA’s recognition of THIO’s potential in the treatment of multiple cancer indications, including rare ones such as glioblastoma,” said Vlad Vitoc, MAIA’s Chairman and CEO. “Each year, globally, more than 300,000 people are diagnosed with brain tumors, of which, 25,000 are in the U.S. Glioblastoma represents the majority of these cases in the U.S. Given this prevalence there is significant room for growth in the $2.2 billion glioblastoma market, which is expected to reach $3.2 billion globally in the next three years. We consider this ODD an important milestone for our development strategy and for glioblastoma patients who could benefit from a potentially revolutionary therapy.”

Sergei Gryaznov, MAIA’s chief scientific officer said, “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. 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.”