Telomere-Targeting Agent Gets Orphan Drug Status for Glioblastoma Treatment

The Food and Drug Administration (FDA) has granted Orphan Drug designation to THIO (6-thio-2’-deoxyguanosine) for the treatment of glioblastoma.

THIO, a modified nucleotide 6-thio-2’-deoxyguanosine, is a first-in-class investigational telomere-targeting agent designed to induce telomerase-dependent telomeric DNA modification, DNA damage responses, and selective cancer cell death. In preclinical studies, treatment with THIO followed by anti-PD-L1 or anti-PD1 therapy resulted in significant tumor regression in advanced cancer models.

In addition, the FDA has previously granted Orphan Drug designations to THIO for the treatment of hepatocellular carcinoma and small cell lung cancer.

The Company is currently investigating THIO in patients with advanced non-small cell lung cancer (NSCLC) in an ongoing phase 2 trial (ClinicalTrials.gov Identifier: NCT05208944).

“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, MD, MAIA’s Chairman and CEO. “[…] We consider this [Orphan Drug designation] an important milestone for our development strategy and for glioblastoma patients who could benefit from a potentially revolutionary therapy.”