Genoscience Pharma has announced that its lead candidate, ezurpimtrostat, a PPT-1 (Palmitoyl Protein Thioesterase-1) inhibitor, has been granted orphan drug designation (ODD) by the US Food and Drug Administration (FDA) for the treatment of hepatocellular carcinoma (HCC). \n\n\n\nODD qualifies ezurpimtrostat for a potential seven years of market exclusivity after approval.\n\n\n\nThe FDA’s ODD program provides orphan status to drugs and biologics intended for the treatment, prevention or diagnosis of a rare disease or condition; those affecting less than 200,000 people in the U.S. There were 35,563 new cases of primary liver cancer reported in 2019, with 27,958 deaths. According to the Centers for Disease Control and Prevention, liver cancer is the sixth most common cancer worldwide and the third leading cause of cancer-related death.\n\n\n\nWithout treatment, HCC is rapidly fatal. In the disease’s untreated progression, the median survival time for patients with advanced HCC ranges from four to eight months. The approved combination of atezolizumab and bevacizumab has more than doubled this life expectancy and improved the patient-reported outcome. However, progression-free survival remains short and new treatment options are needed.\n\n\n\nEzurpimtrostat (GNS561) is a first-in-class, first-in-human autophagy inhibitor whose anticancer activity is linked to PPT-1 inhibition. It displayed high liver tropism and potent anti-tumor activity against a panel of human cancer cell lines and in HCC in vivo models – alone and in combination with immune checkpoint inhibitors. \n\n\n\nPhase 2b trial\n\n\n\nRecent investigations show that autophagy inhibitors in combination with immune checkpoint inhibitors provide opportunities for enhancing anti-tumor activity. Preliminary data from a phase 1b trial on primary and secondary liver tumors has confirmed that administration of ezurpimtrostat as a monotherapy is both feasible and well tolerated.\n\n\n\nThe drug candidate is currently being trialed, as a first-line treatment in combination with an anti-PDL1 and an anti-angiogenic, in ABE-Liver, a phase 2b clinical trial sponsored by Grenoble University Hospital in France, which will enroll up to 196 patients.\n\n\n\n“FDA Orphan Drug Designation is a significant milestone for both Genoscience and for our product, ezurpimtrostat. It recognizes that our treatment has the potential to improve the lives of individuals living with HCC,” said Philippe Halfon, CEO of Genoscience Pharma. \n\n\n\n“We have recently launched our phase 2b clinical trial using ezurpimtrostat in conjunction with the standard atezolizumab/bevacizumab treatment. We are looking forward to sharing the intermediate results in 2024.”\n\n\n\nAbout Genoscience Pharma\n\n\n\nGenoscience Pharma is a French clinical-stage biotechnology company developing novel lysosomotropic treatments to establish a new standard of care in cancer, fibrosis and autoimmune diseases. Its lead candidate, GNS561/ezurpimtrostat, is a best-in-class drug candidate that has entered phase 2b clinical trials. It works by attacking cancer cells through autophagy modulation. Genoscience Pharma is developing other molecules in its portfolio for use in oncology and fibrosis indications.