MAIA Biotechnology Inc (NYSE American: MAIA), a a targeted therapy, immuno-oncology company, announced on Monday that it has received FDA Rare Pediatric Disease designation for its investigational therapy THIO as a treatment for pediatric-type diffuse high-grade gliomas (PDHGG), including the aggressive subtype diffuse intrinsic pontine glioma (DIPG).

The designation positions MAIA to obtain a highly valuable priority review voucher (PRV) upon FDA approval of THIO for PDHGG. PRVs can be redeemed for expedited review of another product or sold, with past transactions averaging USD100m.

Preclinical studies, conducted in collaboration with Nationwide Children's Hospital, demonstrated significant anticancer effects when combining THIO with ionizing radiation (IR) in DIPG models. These findings were presented at the April 2024 American Association for Cancer Research Annual Meeting.

THIO is a first-in-class telomere-targeting agent that induces telomerase-dependent DNA damage, leading to selective cancer cell death and immune activation. Beyond PDHGG, THIO holds orphan drug designations for hepatocellular carcinoma (HCC), small cell lung cancer (SCLC) and glioblastoma. It is currently in clinical trials as a second-line treatment for telomerase-positive non-small cell lung cancer (NSCLC).

MAIA Biotechnology focuses on innovative cancer therapies with novel mechanisms of action, aiming to provide transformative treatments for patients with advanced cancers.