1. GNS561 combined with MEK inhibitors shows promising antitumor activity. 2. All evaluable patients demonstrated disease stabilization, with some showing tumor shrinkage. 3. Phase 2 initiation is targeted for 2H26 to explore treatment efficacy. 4. Significant unmet medical need for advanced KRAS mutated cholangiocarcinoma exists. 5. GENFIT aims to rapidly advance GNS561 for patients with limited options.
FAQ
Why Bullish?
GNS561's promising early results in a challenging patient population could enhance investor confidence. Previous drug trials that showed strong efficacy in early stages have often led to stock price increases.
How important is it?
The article discusses ongoing trials and preliminary results which are directly relevant for GNFT's growth potential and market perception, indicating a strong chance of influencing the stock price.
Why Long Term?
Successful Phase 2 results could establish GNS561's position in a niche market, leading to sustained interest over time. Long-term viability of such therapies often hinges on the outcomes of later phase studies.
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GENFIT Reports Positive Phase 1b Results for GNS561 in Cholangiocarcinoma Treatment
GENFIT (Euronext: GNFT), a prominent biopharmaceutical company focused on rare and life-threatening liver diseases, has announced encouraging preliminary results from its ongoing Phase 1b study. The study evaluates the investigational drug GNS561 in combination with a MEK inhibitor (MEKi) for treating KRAS mutated cholangiocarcinoma (CCA), positioning this novel approach as a potential breakthrough for patients with limited treatment options.
Overview of Clinical Trial and Objectives
Cholangiocarcinoma is a rare and aggressive cancer of the bile ducts often diagnosed in advanced stages. The current treatment landscape is hampered by limited options and a poor prognosis for patients. GNS561 is an innovative small molecule that targets PPT1, inhibiting autophagy and disrupting cancer cell survival mechanisms. The combination with MEKi aims to harness synergistic effects by simultaneously targeting both autophagy and MAPK signaling pathways.
The Phase 1b study is enrolling patients with advanced KRAS mutated CCA who have previously failed one or two lines of standard care therapies. The main goals are to assess the safety, tolerability, and recommended doses of GNS561 combined with trametinib, a MEK inhibitor, ahead of a planned Phase 2 trial.
Preliminary Results
Data from the analysis included 9 patients with measurable disease at baseline; 4 of these patients underwent tumor assessment by week 6. The key findings showcased:
- Disease stabilization in all evaluated patients, who had previously shown disease progression.
- Tumor shrinkage observed in a subgroup, with the best response showing a 20% reduction, approaching the partial response (PR) benchmark.
These outcomes are indicative of GNS561's potential antitumor activity, particularly in a heavily pretreated patient population.
Clinical Implications and Future Steps
The preliminary results underscore a promising ability to meet a critical unmet medical need for patients with advanced solid tumors who have limited treatment options. With the potential of GNS561 in combination with a MEK inhibitor to achieve disease control, this strategy could represent a significant advancement in treating cholangiocarcinoma.
Dr. Mark Yarchoan, Associate Professor of Oncology at Johns Hopkins Medicine and principal investigator, stated, "The emerging activity seen in this initial study is encouraging. The early signs of benefit with dual targeting of autophagy and MAPK signaling provide a meaningful rationale for continued evaluation of this combination strategy."
GENFIT CEO Pascal Prigent added, "These early results suggest a potential breakthrough for patients with limited options. We are committed to advancing this program rapidly to individuals affected by cholangiocarcinoma."
Next Development Steps
GENFIT plans to continue the Phase 1b dose escalation to confirm the activity signal, with new data expected in Q1 2026. These findings will aid in establishing the recommended doses for the upcoming Phase 2 trial, targeted for initiation in 2H 2026.
About Cholangiocarcinoma
Cholangiocarcinoma represents approximately 15% of all primary liver tumors and 3% of gastrointestinal cancers. It is characterized by a poor prognosis, especially in advanced stages. Surgical resection remains the only curative treatment, but only about 25% of patients qualify for surgery upon diagnosis. The 5-year survival rates plummet to between 5-15% in advanced and unresectable cases.
About GNS561
GNS561 is a first-in-class investigational agent with a unique mechanism of action. By combining with MEK inhibitors, it targets key pathways involved in cancer cell survival, potentially leading to significant antitumor activity. In December 2021, GENFIT licensed the rights to develop and commercialize GNS561 for CCA in major markets, and by early 2025, it fully acquired the intellectual property rights from Genoscience Pharma.
About GENFIT
GENFIT is dedicated to enhancing the lives of patients with rare, life-threatening liver diseases, focusing on diseases like Acute on-chronic Liver Failure (ACLF) and cholangiocarcinoma (CCA). With more than two decades of experience, GENFIT is committed to developing innovative therapeutic solutions through its comprehensive research and development pipeline.