CAMBRIDGE, Mass.--(BUSINESS WIRE)--H3 Biomedicine Inc., a clinical stage biopharmaceutical company specializing in the discovery and development of precision medicines for oncology and a member of Eisai’s global Oncology Business Group, today announced that the U.S. Food and Drug Administration (FDA) has granted the company an orphan drug designation for H3B-6527, its first solid tumor clinical compound, for the treatment of patients with Hepatocellular Carcinoma (HCC). H3B-6527, a selective, orally bioavailable, and potent covalent inhibitor of fibroblast growth factor receptor 4 (FGFR4), is currently in Phase 1 clinical trials.
“Receiving orphan drug designation for H3B-6527 is a significant step forward in H3’s efforts to bring a new potential treatment option to HCC patients and their families,” said Markus Warmuth, M.D., President and CEO of H3 Biomedicine. “We believe that the progress we have made thus far by targeting FGFR4 has set the stage for further clinical research, and we look forward to the continued study of H3B-6527 to determine its impact on patients living with liver cancer. We remain committed to advancing this clinical program and providing clinically meaningful benefit to patients.”
The FDA’s Office of Orphan Drug Products grants orphan status to support development of medicines for rare diseases or conditions that affect fewer than 200,000 people in the U.S. The orphan drug designation provides H3 Biomedicine with certain benefits, including market exclusivity upon regulatory approval if received, exemption of FDA application fees, and tax credits for qualified clinical trials.
This is the second clinical compound to receive Orphan Drug designation by the FDA for H3 Biomedicine. In August, 2017, the company’s lead clinical compound, H3B-8800, received orphan designation for Acute Myelogenous Leukemia (AML) and Chronic Myelomonocytic Leukemia (CMML). H3B-8800, a potent, selective and orally bioavailable small molecule modulator of wild-type and mutant SF3b complexes, is currently in Phase 1 clinical trials.
About Hepatocellular Cancer (HCC)
According to the American Cancer Society, Hepatocellular Cancer (HCC) is the most common form of liver cancer in adults and estimates for primary liver cancer and intrahepatic bile duct cancer in the United States for 2017 are about 40,710 new cases (29,200 in men and 11,510 in women) will be diagnosed, while about 28,920 people (19,610 men and 9,310 women) will die of these cancers. More than 700,000 people are diagnosed with this cancer each year throughout the world. Liver cancer is also a leading cause of cancer deaths worldwide, accounting for more than 600,000 deaths each year.
H3B-6527 is a selective, orally bioavailable, and potent inhibitor of fibroblast growth factor receptor 4 (FGFR4) that is being investigated for the treatment of advanced hepatocellular carcinoma (HCC). Aberrant signaling through the FGF19-FGFR4 axis has been shown to drive tumor development and dependency in pre-clinical models of HCC. H3B-6527 has shown sustained tumor regressions in several preclinical models of HCC where FGF19-FGFR4 signaling is aberrantly activated. The safety and preliminary efficacy of H3B-6527 will be explored in patients that are selected using a companion diagnostic that identifies HCC with activated FGF19-FGFR4 pathway activity.
About H3 Biomedicine Inc.
H3 Biomedicine is a Cambridge, Massachusetts-based biopharmaceutical company specializing in the discovery and development of precision oncology treatments, and was established as a subsidiary of Eisai's U.S. pharmaceutical operation, Eisai Inc. Leveraging this collaboration with Eisai, Co., Ltd., who through this partnership provides essential research funding and access to the capabilities and resources of this global pharmaceutical company, H3 Biomedicine combines long-term vision with operational independence. Using modern synthetic chemistry, chemical biology, and human genetics, H3 Biomedicine seeks to bring the next generation of cancer treatments to market with the goal of improving the lives of patients. For more information, please visit www.h3biomedicine.com.