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MAIA Biotechnology have announced that the U.S. Food and Drug Administration has granted orphan drug designation to its lead asset THIO, a cancer telomere-targeting agent, for 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.
“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, M.D., MAIA’s Chairman and Chief Executive Officer. “Each year, globally, more than 300,000 people are diagnosed with brain tumours, of which, 25,000 are in the United States. Glioblastoma represents the majority of these cases in the U.S., with 15,000 new patients diagnosed and more than 10,000 deaths yearly, making it an orphan indication. 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.”
“In the data presented to the FDA, THIO successfully penetrated the blood brain barrier (BBB) in syngeneic and humanised 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,” added 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 tumours in vivo and potentially in glioblastoma patients.”
Enrolment is ongoing in a Phase 2 trial of THIO, THIO-101, 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.