September 15, 2017

Cambridge, MA — 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 company scientists will present data from pre-clinical studies involving one of its oncology drug candidates, H3B-6527. This data was accepted as an oral presentation at the 2017 International Liver Cancer Association (ILCA) annual meeting taking place September 15-17, 2017 in Seoul, Republic of Korea.

H3 Biomedicine’s oral presentation is scheduled at 3:30 p.m. on Friday September 15, in Grand Ballroom 1 & 2, Lobby Level. The presentation will overview H3B-6527, an orally bioavailable, highly selective and potent inhibitor of fibroblast growth factor receptor 4 (FGFR4), which is currently in an ongoing Phase I clinical study in advanced hepatocellular carcinoma (HCC) and intrahepatic cholangiocarcinoma (IHCC). The presentation will be given by H3 scientist and lead author, Anand Selvaraj, Ph.D.

“The data being presented at ILCA highlight the excellent progress H3’s scientists have made within this program and demonstrate that FGF19 expression is a predictive biomarker for response to H3B-6527 therapy in non-clinical studies,” said Markus Warmuth, M.D., President and CEO of H3 Biomedicine. “These results highlight the potential for FGFR4 inhibition with H3B-6527, and we are excited to continue with our research to determine if it may provide clinical benefit to patients who have been diagnosed with HCC and IHCC.”

The study revealed that biochemical and cellular selectivity assays showed that H3B-6527 is >250 fold selective towards FGFR4 compared to other FGFR isoforms. Addition of H3B-6527 to FGF19 amplified HCC cell lines led to dose dependent inhibition of FGF19/FGFR4 signaling and concomitant reduction in cell viability. In a panel of 40 HCC cell lines, H3B-6527 selectively reduced the viability of cells that harbor FGF19 amplification and showed no effect in FGF19 non-amplified HCC cell line models. Oral dosing of H3B-6527 to mice led to dose-dependent pharmacodynamic modulation of FGFR4 signaling and tumor regression in FGF19 altered HCC cell line derived xenograft models. H3B-6527 demonstrated inhibition of tumor growth in an orthotopic liver xenograft model of FGF19 altered HCC grown in nude mice. Importantly, the inhibition of tumor growth occurred at doses that were well tolerated in mice and no evidence of FGFR1-3 related toxicities were observed at efficacious doses.

“We are extremely pleased with the results of this work and the progress the program has made, to date,” said Pete Smith, Ph.D., Chief Scientific Officer, H3 Biomedicine. “H3B-6527 demonstrated tumor regressions in multiple patient-derived xenograft models with high FGF19 expression and has shown excellent combination potential with standard-of-care and experimental agents. The data described in the presentation support the ongoing clinical development of H3B-6527 and highlight our aim to select patients most likely to respond to H3B-6527 treatment.”

About H3B-6527

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. H3B-6527 is currently in Phase 1 clinical trials. For more information on the clinical trial, please click here.

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

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