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GENFIT: GNS561 Shows Promising Antitumor Activity in Combination Therapy
Highly encouraging early data from the ongoing Phase 1b study evaluating investigational drug GNS561 with a MEK inhibitor (MEKi) in KRAS mutated cholangiocarcinoma (CCA), positioning this novel combination as a potential new therapeutic approach for difficult-to-treat cancers: No dose limiting toxicity reached to date, enabling recruitment of a third patient cohortGNS561 and MEKi combination demonstrated disease stabilization in all evaluable patients with evidence of tumor shrinkage in a subset
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[{"type":"list","items":[{"val":[{"type":"text","content":"Highly encouraging early data from the ongoing Phase 1b study evaluating investigational drug GNS561 with a MEK inhibitor (MEKi) in KRAS mutated cholangiocarcinoma (CCA), positioning this novel combination as a potential new therapeutic approach for difficult-to-treat cancers:","length":277,"tagName":"p"},{"type":"list","items":[{"val":[{"type":"text","content":"No dose limiting toxicity reached to date, enabling recruitment of a third patient cohort","length":89,"tagName":"p"}]},{"val":[{"type":"text","content":"GNS561 and MEKi combination demonstrated disease stabilization in all evaluable patients with evidence of tumor shrinkage in a subset of patients, warranting further investigation","length":179,"tagName":"p"}]},{"val":[{"type":"text","content":"Recommended Phase 2 doses expected for 1H26","length":43,"tagName":"p"}]}],"tagName":"ul","bulletedList":true,"length":311,"olType":false}]}],"tagName":"ul","bulletedList":true,"length":588,"olType":false},{"type":"text","content":"Lille (France), Cambridge (Massachusetts, United States), Zurich (Switzerland), December 10, 2025 - GENFIT (Euronext: GNFT), a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, today reports encouraging preliminary Phase 1b data from its CCA clinical trial evaluating GNS561 in combination.","length":358,"tagName":"p"},{"type":"text","content":"Clinical trial context and objectiveCCA is a rare and aggressive cancer of the bile ducts, often diagnosed at an advanced stage. The unmet medical need is characterized by strong limitations in current treatments and poor prognosis. GNS561 is an investigational small molecule that targets PPT1, leading to autophagy inhibition and lysosomal dysfunction, which disrupt cancer cell survival mechanisms. By blocking autophagy, GNS561 aims to promote cancer cell death and may enhance sensitivity to other treatments. Combining GNS561 with a MEKi aims to unlock synergistic potential by simultaneously targeting autophagy and MAPK signaling pathways. In the on-going Phase 1b study, patients with advanced KRAS mutated CCA who have previously failed one or two lines of prior standard of care therapies are enrolled to evaluate the safety and tolerability of GNS561 when given in combination with trametinib, a MEKi, and to identif...