Genfit disclosed early data from its Phase 1b/2a study of the PPT1 inhibitor GNS561 in combination with the MEK inhibitor trametinib in patients with advanced, KRAS‑mutated cholangiocarcinoma (CCA).
The study enrolled nine heavily pre‑treated patients; four of them reached the week‑6 assessment, at which point disease control and measurable tumor reduction were observed in a subset of the cohort. The data are preliminary but suggest that the dual‑targeting strategy can produce clinical benefit in a population with limited options.
GNS561 blocks late‑stage autophagy by inhibiting the lysosomal enzyme PPT1, while trametinib suppresses MAPK signaling downstream of KRAS. The combination is designed to induce cancer‑cell death more effectively than either agent alone, a hypothesis that the early results appear to support.
These findings reinforce Genfit’s plan to move the combination into a Phase 2 trial, which the company expects to initiate in the second half of 2026. The data will inform dose selection and safety monitoring for the next‑phase study.
Cholangiocarcinoma is a rare, aggressive bile‑duct cancer with a median overall survival of less than a year after standard chemotherapy. KRAS‑mutated disease lacks approved targeted therapies, and Genfit’s orphan‑drug designation for GNS561 in CCA provides a regulatory incentive to advance the program. The preliminary efficacy signals therefore carry substantial therapeutic and commercial implications.
Genfit remains optimistic that the combination will demonstrate meaningful activity in a broader cohort and that the Phase 2 study will confirm the safety and efficacy profile needed to support regulatory approval and market access.
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