FDA Grants Orphan Drug Designation to THIO in Small Cell Lung Cancer

The FDA has granted an orphan drug designation to THIO (6-thio-dG, 6-thio-2’-deoxyguanosine) for the treatment of patients with small cell lung cancer (SCLC), according to an announcement from MAIA Biotechnology.1

THIO is a potentially first-in-class telomere targeting agent. Telomeres, along with the enzyme telomerase, which is present in more than 85% of human cancers, 2 play a key role in cancer cell growth and their resistance to current treatments. The agent acts through recognition of telomerase, allowing its entrance into telomeres in cancer cells. Once inside, THIO inhibits the telomere structure and function, causing uncapping of the chromosome ends and cell death.

“Patients with SCLC have exceptionally poor prognosis, with only 6% of patients remaining alive five years after diagnosis,” Mihail Obrocea, MD, chief medical officer of MAIA, stated in a press release. “While clinical progress in SCLC treatment has been incredibly slow to evolve, an increased understanding of the disease biology has demonstrated that targeted therapies, like THIO, may offer a novel therapeutic option for SCLC.”

In April 2022, the FDA granted THIO its first orphan drug designation for the treatment of patients with hepatocellular carcinoma (HCC).3

“Receiving our second orphan drug designation is an impressive regulatory milestone that highlights the FDA’s recognition of THIO’s potential to improve outcomes for patients with SCLC and HCC,” Vlad Vitoc, MD, MAIA’s chairman and chief executive officer of MAIA, stated in a press release. “We believe there is a significant, multi-billion-dollar opportunity for THIO across many difficult-to-treat cancers, and we look forward to advancing THIO towards the market and to patients in need for more effective therapies.”