Drugs / Ezurpimtrostat
Ezurpimtrostat
Trials 5
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT05353894 | May → Nov 2022 | liver cancer | Genoscience Pharma | Unknown | No outcome recorded |
| Phase 1/22 trials | ||||||
| Phase 1/2 | NCT05874414 | Aug 2023 → Dec 2027 expected | cholangiocarcinoma | Genfit | Recruiting | No outcome recorded |
| Phase 1/2 | NCT03316222 | Apr 2018 → Jan 2021 | hepatocellular carcinoma, intrahepatic cholangiocarcinoma | Genoscience Pharma | Terminated | No outcome recorded |
| Phase 22 trials | ||||||
| Phase 2 | NCT05448677 | Dec 2022 → Mar 2024 | hepatocellular carcinoma | University Hospital, Grenoble | Terminated | No outcome recorded Stop: Enrollment |
| Phase 2 | NCT04637828 | Nov 2020 → Jul 2021 | COVID-19 | Genoscience Pharma | Unknown | No outcome recorded |
News releases announcing trial results or a regulatory action · 4
| Date | Issuer | Release |
|---|---|---|
| 2026-06-23 | Genfit | Results GENFIT: Positive Phase 1b Data with GNS561 in Combination Therapy in Heavily Pretreated Patients with Cholangiocarcinoma genfit.com ↗
today announced positive Phase 1b results for GNS561 in combination therapy in patients with heavily pretreated cholangiocarcinoma, demonstrating a favorable safety and tolerability profile and early signs of antitumor activity. |
| 2025-12-10 | Genfit | Results GENFIT: GNS561 Shows Promising Antitumor Activity in Combination Therapy genfit.com ↗
Achieving disease control and tumor reduction in such heavily pretreated patient population with advanced CCA is a significant signal of antitumor activity. |
| 2025-02-06 | Genfit | Results GENFIT Outlines Anticipated New Clinical Trial Initiations, Development Milestones and Data Readouts in 2025 genfit.com ↗
Preliminary safety data analyzed end of 2024 from the patients treated in the first cohort of Phase 1b with the combination of GNS561 and trametinib are supportive |
| 2022-09-13 | Genfit | Regulatory FDA Grants GENFIT’s GNS561 Orphan Drug Designation for the Treatment of Cholangiocarcinoma genfit.com ↗
Lille (France ), Cambridge (Massachusetts, United States), September 1 3 , 20 22 – GENFIT (Nasdaq and Euronext: GNFT) , a late-stage biopharmaceutical company dedicated to improving the lives of patients with severe chronic liver diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to GNS561 1 (ezurpimtrostat), a novel clinical-stage autophagy/PPT1 inhibitor, for the treatment of cholangiocarcinoma. |
All press releases naming this drug 4 releases
Evidence & citations 5 cited values
Every value below carries the sentence it was read from. 6 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Ezurpimtrostat | “GNS561/Ezurpimtrostat is a first-in-class, orally bioavailable, small molecule that blocks cancer cell proliferation by inhibiting late-stage autophagy and dose-dependent...” PMID 35949290 ↗ Feb 2022 |
| Known as | Ezurpimtrostat (GNS561) | “Ezurpimtrostat (GNS561)” NCT05874414 ↗ |
| Known as | GNS561 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT05874414 ↗ |
| Modality | Small molecule | “a first-in-class, orally bioavailable, small molecule” PMID 35949290 ↗ Feb 2022 |
| Route | Oral | “single oral intake 3 times a week” PMID 35949290 ↗ Feb 2022 |