BioNTech SE and partner OncoC4 disclosed that the non‑pivotal dose‑confirmation stage of the global Phase 3 PRESERVE‑003 trial has shown a clinically meaningful overall survival benefit for gotistobart (BNT316/ONC‑392) in patients with metastatic squamous non‑small cell lung cancer (sqNSCLC) who had progressed after anti‑PD‑(L)1 therapy and platinum‑based chemotherapy.
The data, presented at the IASLC/ASCO 2025 North America Conference, report that median overall survival has not yet been reached in the gotistobart arm versus 10 months for docetaxel. At 12 months, overall survival was 63.1% for gotistobart compared with 30.3% for chemotherapy. The hazard ratio for death was 0.46 (95% CI 0.25–0.84; nominal p = 0.0102), indicating a 54% reduction in the risk of death. Grade ≥ 3 treatment‑related adverse events occurred in 42.2% of gotistobart patients versus 48.8% in the docetaxel group, demonstrating a manageable safety profile.
Gotistobart is a tumor‑microenvironment‑selective CTLA‑4 antibody designed to deplete regulatory T‑cells (Tregs) within the tumor microenvironment, a strategy that aims to enhance anti‑tumor immune responses while sparing systemic immune regulation. The observed survival benefit validates this approach in a population with limited treatment options and underscores BioNTech’s broader oncology strategy of developing next‑generation immunotherapies beyond its COVID‑19 vaccine platform.
The trial is now progressing to the pivotal stage, which will enroll over 500 patients worldwide. The pivotal study will evaluate overall survival as the primary endpoint and is expected to complete enrollment in 2026, with results anticipated in 2027. The data reinforce BioNTech’s commitment to expanding its oncology portfolio, which has seen significant R&D investment—€2.3 billion in 2024 and guidance of €2.6‑2.8 billion for 2025—while maintaining a strong cash position.
BioNTech’s oncology pipeline has been a key focus for diversifying revenue streams. In Q3 2024, the company reported €1.2448 billion in revenue and €198.1 million in net profit, and it raised its full‑year 2025 revenue guidance to €2.6‑2.8 billion. The positive gotistobart data provide a tangible milestone that could accelerate regulatory approval and market entry, potentially generating new revenue streams and strengthening the company’s competitive position against other immuno‑oncology players.
OncoC4, BioNTech’s partner, recently closed a Series B financing round of nearly $50 million, underscoring confidence in the partnership and the broader pipeline. The collaboration exemplifies BioNTech’s strategy of leveraging external expertise to accelerate development of novel therapeutics.
The overall survival benefit, combined with a manageable safety profile, positions gotistobart as a promising candidate for regulatory approval in a high‑need patient population. If approved, the drug could become a new standard of care for sqNSCLC patients who have exhausted first‑line therapies, thereby expanding BioNTech’s oncology market share and supporting long‑term growth.
The announcement also signals to investors that BioNTech’s investment in tumor‑microenvironment‑selective immunotherapies is yielding clinical dividends, potentially enhancing the company’s valuation and reinforcing confidence in its long‑term strategic direction.
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