Maia Biotechnology has touted the efficacy of its lead candidate, ateganosine, after a positive mid-stage trial outcome in non-small cell lung cancer (NSCLC).
Otherwise known as THIO, the telomerase modifier was tested in sequence with Regeneron’s programmed death-1 (PD-1) inhibitor Libtayo (cemiplimab) in patients with NSCLC who had progressed after two or more standard of care (SoC) therapy regimens.
During the Phase II THIO-101 study (NCT05208944), a sequential regimen of ateganosine and Libtayo triggered a median progression-free survival (PFS) of 5.6 months – a marked increase from the SoC PFS of 2.5 months. Meanwhile, median overall survival (OS) was 17.8 months, which is around 5.8 months more than the highest OS data reported in a recent real-world analysis.
The sequential combination also exhibited a disease control rate (DCR) of 77% versus the 25–35% triggered by chemotherapy.
Ateganosine demonstrated a favourable safety profile – with most treatment-related adverse events (TRAEs) being Grade 1 or 2 – though 21.5% of patients experienced TRAEs marked Grade 3 or above, which are grades considered severe.
A concerning safety signal came from the cohort receiving the drug’s highest dose. Maia had to pause treatment in the 360mg ateganosine arm after a patient experienced a Grade 4 liver function test (LFT) elevation, suggesting that the drug could promote liver toxicity at a high dose.
Despite this, ateganosine showed potential in an extended dose setting, as two patients across all treatment arms successfully completed 33 cycles of therapy, which the biotech suggested could “translate into longer patient survival”.
Data from the THIO-101 trial was presented at the 2025 IASLC World Conference on Lung cancer (WCLC).
Moving forward, Maia’s Chair and CEO, Vlad Vitoc, noted that the company will further explore ateganosine’s efficacy in the THIO-101 Phase II expansion trial, which began enrolling patients in July 2025.
Addressing unmet needs in advanced NSCLC
Despite a wide range of targeted therapies available as first and second line options in NSCLC, there is a distinct lack of treatment options for patients in the third line setting.
Currently, the SoC revolves around chemotherapy agents like pemetrexed, docetaxel and paclitaxel, which are often highly toxic for heavily pretreated patients.
If ateganosine were to be approved, it would be the first in its class, and could be used alongside a PD-1 inhibitor to treat patients with telomerase-positive (TERT) cancer cells – a characteristic found in 78 to 83% of NSCLC cases.
However, it could face stiff competition from other drugs in the late-stage pipeline, including BioNTech’s anti cytotoxic T lymphocyte associated protein 4 (CTLA-4) monoclonal antibody (mAb) gotistobart and AstraZeneca’s ataxia telangiectasia and rad3-related (ATR) inhibitor ceralasertib – both of which Clinical Trials Arena has identified as key trials with upcoming readouts in the NSCLC landscape.
Maia may also have to watch out for Merck’s trophoblast cell surface antigen 2 (TROP-2)-targeting antibody-drug conjugate (ADC) sacituzumab tirumotecan, which is currently being assessed through multiple Phase III trials in previously treated NSCLC with epidermal growth factor receptor (EGFR) mutations.
