Advancing Telomere Targeting Therapy For Non-Small Cell Lung Cancer
By Erin Harris, Editor-In-Chief, Cell & Gene
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Non-small cell lung cancer (NSCLC) remains one of the greatest challenges in oncology, particularly for patients resistant to chemotherapy and checkpoint inhibitors who face limited treatment options and poor outcomes. I recently caught up with Dr. Vlad Vitoc, CEO of MAIA Biotechnology, an immuno-oncology company pioneering telomere-targeting therapies with its lead candidate, ateganosine. With compelling Phase 2 data from the THIO-101 trial showing both survival benefits and strong tolerability, Dr. Vitoc shares insights into how MAIA is advancing a novel therapeutic strategy that could redefine the treatment landscape for hard-to-treat cancers.
Tell us about MAIA Biotechnology.
Dr. Vitoc: MAIA is an immuno-oncology company focused on the development and commercialization of drugs intended to meaningfully improve and extend the lives of people with hard-to-treat cancers.
We are exploring new science for cancer therapy utilizing a novel dual mechanism of action (MoA): telomere targeting and immunogenicity. Our lead program is ateganosine, a first-in-class anticancer agent in clinical development for the treatment of non-small cell lung Cancer (NSCLC) in patients resistant to checkpoint inhibitors and chemo.
Ateganosine’s MoA is very robust and has applicability in multiple cancer indications. We’re also planning trials in other tumor types, such as hepatocellular carcinoma (liver cancer), colorectal cancer and the deadliest form of lung disease, small cell lung cancer.
The recently released data from THIO-101 showed a median progression-free survival of 5.6 months. Walk us through what these results mean for patients who have exhausted multiple treatment options? How do these results compare to other third-line therapies currently available for advanced NSCLC?
Dr. Vitoc: Progression-Free Survival (PFS) is the time from treatment initiation until disease progression or death. In this patient population, third-line NSCLC patients resistant to chemo and immunotherapies, other therapies have shown a PFS of around 2.5 months. Ours more than doubles that to 5.6 months.
We believe PFS is a good surrogate for overall survival advantage and our trial results to date suggests that is the case. Ateganosine’s observed median overall survival (OS) is 17.8 months, with a 95% confidence interval (CI) lower bound of 12.5 months and a 99% CI lower bound of 10.8 months. Other therapies in scientific literature present OS of around 6 months. Ours is almost 3x the standard.
We have robust treatment that is safe, with excellent PFS and OS. We look forward to bringing our treatment to more patients.
Checkpoint inhibitor resistance remains a huge challenge in advanced NSCLC. What are you learning from THIO-101 about the timing and dosing needed to sustain an immune response in these difficult-to-treat patients?
Dr. Vitoc: Ahead of starting the THIO-101 trial we tested several permutations of the sequence ateganosine and checkpoint inhibitor (CPI) in a preclinical setting. The chosen schedule (3 days of ateganosine, a short break on day 4 for the immunogenic effect to take place, and CPI on day 5) was the most efficacious with high confidence.
We brought that to the trial and focused on identifying the optimal dose to sustain an immune response. We learned that there is no direct relation between dose and response, but we found 180mg per cycle to be the optimal dose that activates the immune system and doesn’t exhaust it, maximizing the benefits of the treatment.
You recently began enrolling patients in the THIO-101 expansion trial. What are the main objectives for this phase, and how will the data you gather guide potential regulatory pathways or partnerships moving forward?
Dr. Vitoc: Our key objective in the expansion of the THIO-101 trial is to further validate with statistical power the efficacy of ateganosine sequenced with cemiplimab in a third-line patient setting.
We believe that with more patients, our data may support the case for accelerated approval by the FDA for the treatment with ateganosine.
The treatment landscape for advanced NSCLC has evolved rapidly, with targeted therapies, checkpoint inhibitors, and now telomere-targeting agents. From your perspective, what are the biggest unmet needs that still exist for these patients? Where do you see the next breakthroughs coming?
Dr. Vitoc: We believe that this population represents patients with the highest unmet medical needs; they can’t enjoy the benefits of targeted therapies, as they don’t have actionable mutations, and they are CPI and chemo-resistant. Telomere-targeting is the breakthrough, and our trial with ateganosine is set to confirm that.
We are already working on the next generation of telomere-targeting molecules, which have the potential to bring even better benefits to a variety of cancer indications. We’re confident we’ll have breakthroughs from those programs in the near future.
Many of the patients enrolled in THIO-101 were heavily pre-treated, which can complicate both efficacy and safety. Can you share what you’ve seen so far regarding tolerability, low toxicity, and the potential for extended treatment cycles. What could this mean for patient quality of life?
Dr. Vitoc: We recently updated the safety profile observed in our trial at the IASLC World Conference on Lung Cancer 2025. We saw minimal clinical side effects, and treatment with ateganosine sequenced with cemiplimab has been generally well tolerated in a heavily pre-treated population, with most events being Grade 1–2 in severity. We saw minimal clinical side effects of Grade 3.
The most commonly reported adverse events were liver function elevation (AST 27%, AST 23%), followed by nausea (13%). The treatment with ateganosine is very safe and can be taken for extended periods of time. In THIO-101, we’ve had patients on the treatment for more than 2 years.
Having great safety is highly beneficial for the patients in the trial, as they can continue the trial as scheduled without interruptions.
What’s next for MAIA?
Dr. Vitoc: As we’re about to start our Phase 3 trial in this heavily pre-treated NSCLC population, we recently used a Bayesian Assurance Calculation to analyze the probability of technical success (PTS) based on our current readings from the THIO-101 Phase 2 trial.
We estimate that for a robust case for approval of ateganosine in a Phase 3 trial with 300 patients, the observed OS needs to be greater than 9.4 months compared to the 5.8 to 6.1 months shown in chemotherapy (HR=0.62).
Based on our current OS data of 17.8 months, we have a 96% probability of success if we pursue an approval with an interim read of the trial with 131 death events, and a 99% probability to succeed at a final analysis after 186 death events.
This is great news, and we believe that it’s only a matter of continuing execution of our trial to be able to bring this treatment to third-line NSCLC patients.