FDA Grants MAIA Biotechnology Rare Pediatric Disease Designation for THIO for High-Grade Gliomas

MAIA Biotechnology, Inc., a biopharmaceutical company based in Chicago, has received a significant recognition from the U.S. Food and Drug Administration (FDA). The company's investigational drug, THIO, has been designated as a treatment for pediatric-type diffuse high-grade gliomas (PDHGG), under the FDA’s Rare Pediatric Disease program. This designation highlights THIO's potential as a promising therapeutic option for a type of childhood cancer that is notoriously resistant to treatment.

THIO is a novel anti-cancer agent known for its ability to stimulate the immune system while circumventing the immunosuppressive environment often created by tumors. This unique mechanism is particularly relevant in treating pediatric high-grade glioma, one of the most difficult cancers to treat in children. MAIA’s Chairman and CEO, Dr. Vlad Vitoc, expressed that the FDA’s recognition of THIO as a rare pediatric disease treatment is a crucial step in advancing research for this challenging condition.

K. Robinson Lewis, Vice President and Head of Regulatory and Quality at MAIA, noted that the rare pediatric disease designation provides substantial benefits to the company. It opens up the possibility for MAIA to acquire a priority review voucher from the FDA upon the future approval of a new drug application for PDHGG. These vouchers are highly coveted in the pharmaceutical industry, as they allow for expedited review of another drug product, and can be sold for significant sums, with past transactions averaging around $100 million since 2015.

The effectiveness of THIO has been previously demonstrated in studies targeting a subtype of PDHGG known as diffuse intrinsic pontine glioma (DIPG). Collaborative research conducted with Nationwide Children's Hospital indicated that the combination of THIO and ionizing radiation significantly reduced cell growth and had potent anti-cancer effects in DIPG, a particularly aggressive form of cancer. These findings were presented at the American Association for Cancer Research (AACR) Annual Meeting in April 2024.

In pursuing the FDA designation, MAIA partnered with Only Orphans Cote, a leading provider of regulatory services specializing in orphan drug designations and marketing approvals. This collaboration underscores MAIA’s commitment to advancing THIO’s development for rare and challenging cancer types.

Further emphasizing its potential, THIO has also been granted orphan drug designations for other cancer types, including hepatocellular carcinoma (HCC), small cell lung cancer (SCLC), and glioblastoma. The company touts THIO as the only telomere-targeting agent currently in clinical trials, highlighting its innovative approach to cancer treatment.

THIO, also known as 6-thio-dG or 6-thio-2’-deoxyguanosine, is being evaluated for its effectiveness in targeting telomeres, structures critical for cancer cell survival. By disrupting telomeric DNA and inducing DNA damage, THIO selectively targets cancer cells, triggering both innate and adaptive immune responses. This multi-faceted approach has shown significant potential in preclinical models, particularly when used in conjunction with PD-(L)1 inhibitors, leading to substantial tumor regression and the development of cancer type-specific immune memory.

MAIA Biotechnology is focused on developing and commercializing innovative cancer therapies that offer new treatment options for patients. Their lead product, THIO, is currently under investigation as a second-line treatment for non-small cell lung cancer (NSCLC) in patients with telomerase-positive tumors, aiming to provide new hope for those who have exhausted standard treatment options.