MAIA Biotechnology, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its lead asset THIO, a cancer telomere-targeting agent, for the treatment of glioblastoma. 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. 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.

The FDA's ODD program provides multiple incentives to make orphan drug development more financially possible for companies to pursue, such as up to seven years of market exclusivity for the approved orphan drug, up to 20 years of 25% federal tax credit for expenses incurred in conducting clinical research within the U.S. and waiver of Prescription Drug User Fee Act (PDUFA) fees for orphan drugs, a value of approximately $2.9 million in 2021. THIO-101 is a multicenter, open-label, dose finding Phase 2 clinical trial. The trial design has two primary objectives: (1) to evaluate the safety and tolerability of THIO administered as an anticancer compound and a primarily immune activator (2) to assess the clinical efficacy of THIO using Overall Response Rate (ORR) as the primary clinical endpoint.

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