Full Breakdown
BioNTech-OncoC4 Report Survival Benefit for Gotistobart in Late-Stage Lung Cancer Trial
By Drooid · · How we work
Trial Results Presented at the World Conference on Lung Cancer
On September 14, BioNTech announced that the non-pivotal stage of the PRESERVE-003 Phase 3 trial showed a median overall survival (OS) of 18.5 months for patients receiving the investigational anti-CTLA-4 antibody gotistobart, compared with 10.0 months for those treated with standard docetaxel chemotherapy. The hazard ratio (HR) versus docetaxel was 0.56, corresponding to a 44 % lower risk of death (nominal p-value = 0.0295). The data were presented at the International Association for the Study of Lung Cancer 2026 World Conference on Lung Cancer in Seoul.
Background & Context
Gotistobart is a next-generation CTLA-4 monoclonal antibody designed to bind the target in a pH-sensitive manner, depleting suppressive regulatory T cells within the tumor while sparing normal tissue. The drug is being co-developed by BioNTech and OncoC4 and has received Fast Track, orphan-drug, and breakthrough-therapy designations from the U.S. Food and Drug Administration (FDA) and a breakthrough-therapy designation from China’s National Medical Products Administration. The PRESERVE-003 trial enrolls patients whose squamous non-small cell lung cancer (sqNSCLC) has progressed after both PD-(L)1-directed immunotherapy and platinum-based chemotherapy, a setting where median survival with existing therapies is typically under one year.
Data & Statistics
| Metric | Gotistobart (n = 45) | Docetaxel (n = 42) |
|---|---|---|
| Median OS (months) | 18.5 | 10.0 |
| Hazard Ratio (OS) | 0.56 | — |
| Nominal p-value | 0.0295 | — |
| Grade >= 3 treatment-related adverse events | 44.4 % (20 patients) | 48.8 % (20 patients) |
| Follow-up cut-off | July 17, 2026 (median 25.4 months) | — |
| Randomized patients | 87 with metastatic squamous NSCLC | — |
The trial was open-label, with patients and investigators aware of treatment allocation. The 87-patient sample represents a small, early-stage comparison within a larger confirmatory effort that continues at more than 160 sites worldwide.
Official Statements & Responses
Both companies noted that the pivotal second-stage of PRESERVE-003 is ongoing, but no public date for its readout has been provided.
Conflicting Reports & Gaps
- Sample size and design – The trial’s 87-patient enrollment is modest for a survival claim in lung cancer, and the open-label format may introduce bias.
- Statistical significance – The reported p-value is nominal and was not adjusted for multiplicity, a requirement for confirmatory analyses.
- Regulatory status – Gotistobart remains investigational; it has not received FDA or any other regulatory approval, and the results do not yet alter treatment guidelines or insurance coverage.
- Generalizability – The study population is limited to patients with squamous histology who have progressed after PD-(L)1 inhibitors and platinum chemotherapy, restricting applicability to broader NSCLC cohorts.
What’s Next
The pivotal portion of PRESERVE-003, which will evaluate overall survival as its primary endpoint, continues to enroll at over 160 global sites. Results from this stage will determine whether gotistobart advances toward regulatory review and potential incorporation into standard-of-care treatment algorithms. No specific timeline for the pivotal readout has been disclosed.