MAIA Biotechnology, Inc., announced a significant expansion of its THIO-101 Phase 2 trial. The trial is evaluating ategosine (THIO), a novel telomere-targeting agent, for the treatment of advanced non-small cell lung cancer (NSCLC). The first patient in the expansion phase was dosed in Taiwan, marking a crucial step that broadens the patient recruitment pool to include Asia, alongside ongoing screening in Europe.

This expanded study specifically targets heavily pre-treated third-line (3L) NSCLC patients who have previously failed to respond to both checkpoint inhibitors (CPIs) and chemotherapy. The trial features two distinct treatment arms: ateganosine administered sequentially with cemiplimab (Libtayo®), and ateganosine as a monotherapy. This strategic design aims to thoroughly assess ateganosine's potential in a patient population with very limited therapeutic options.

The strategic importance of this trial is underscored by the immense size of the NSCLC market, which was valued at $34.1 billion in 2024 and is projected to reach $68.8 billion by 2033. Preliminary data from the THIO-101 trial has been highly encouraging, with the median overall survival (OS) for 22 patients in the third-line setting observed at 17.8 months. This figure significantly surpasses established benchmarks for chemotherapy in a similar setting, which typically show overall survival rates of 5-6 months.

Ateganosine (THIO) is a first-in-class investigational telomere-targeting agent. It works by inducing telomerase-dependent telomeric DNA modification, which leads to DNA damage responses and selective cancer cell death. Crucially, ateganosine-damaged telomeric fragments accumulate in cytosolic micronuclei, activating both innate (cGAS/STING) and adaptive (T-cell) immune responses. This unique mechanism, especially when followed by PD-(L)1 inhibitors, has shown profound and persistent tumor regression in advanced in vivo cancer models. MAIA's CEO, Dr. Vlad Vitoc, emphasized that ateganosine's observed OS "exceeds all known benchmarks," potentially positioning the company for a first-mover advantage in a multi-billion dollar space that currently lacks an approved standard of care.

The THIO-101 trial is designed to evaluate both the safety and tolerability of ateganosine as an anticancer compound and immune activator, as well as its clinical efficacy using Overall Response Rate (ORR) as the primary endpoint. The expansion of the study will further assess ORR in these challenging-to-treat patients, building on the promising results seen so far.