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H3 Biomedicine Announces Data from Lead Development Program of H3B-8800 in Pre-Clinical Models of Hematologic Cancers

Data Presented at American Society of Hematology Meeting Supports Initiation of Phase 1 Clinical Trials in Patients with Spliceosome Mutant Cancers

Cambridge, Mass., December 5, 2016 — H3 Biomedicine Inc., a biopharmaceutical company specializing in the discovery and development of precision medicines for oncology and a member of Eisai's global Oncology Business Group, announced today that data from the company’s lead cancer program were presented at the 2016 American Society of Hematology (ASH) Meeting in San Diego, Calif. The oral presentation detailed pre-clinical findings from the company’s lead cancer compound, H3B-8800, demonstrating its potential efficacy in models of spliceosome mutant myeloid malignancies including a novel patient-derived xenograft system for chronic myelomonocytic leukemia (CMML).

Also collaborating in this program along with H3 Biomedicine, were additional cancer research groups from Memorial Sloan Kettering Cancer Center (New York, NY) and Moffitt Cancer Center, (Tampa, Fla.).

“The data presented at ASH highlights the continued progress of our H3B-8800 program and establishes the clinical rationale for development of the compound in myeloid malignancies which has recently begun clinical trials,” said Markus Warmuth, M.D., Chief Executive Officer and President of H3 Biomedicine. “Approximately 50% of myelodysplastic syndrome, secondary AML and CMML patients harbor a spliceosome mutation and these populations are in need of new treatment options.”

H3’s ASH presentation provides an overview of the pre-clinical discovery of H3B-8800, an orally available modulator of the SF3b complex that shows potent splicing modulation in vitro and preferential cell killing of spliceosome mutant cells.

“Our deep expertise in RNA biology has uncovered a mechanistic rationale for the targeted treatment of spliceosome mutant cancers with H3B-8800,” said Pete Smith, PhD, Vice President, Biology for H3Biomedicine. “The data presented at ASH demonstrates the compelling activity of H3B-8800 in cell line and patient-derived xenograft models of spliceosome mutant hematological malignancies.”

H3B-8800 is currently in Phase I clinical trials in advanced myeloid malignancies. The early clinical studies will evaluate safety, pharmacokinetics, pharmacodynamics and efficacy in patients with mutations in SF3B1, SRSF2, U2AF1 and ZRSR2 spliceosome genes.

About H3B-8800

H3B-8800 is an oral, potent, and selective small molecule modulator of splicing factor 3b subunit 1 (SF3B1) that is being developed by H3 Biomedicine as a potential anticancer therapeutic agent. In pre-clinical studies, H3B-8800 showed dose dependent modulation of canonical and aberrant splicing when dosed orally at tolerated doses. More importantly, oral administration of H3B-8800 demonstrated preferential antitumor activity in several pre-clinical xenograft models carrying spliceosome mutations. H3 Biomedicine’s lead research and discovery programs in splicing are designed to develop drugs that target the vulnerabilities related to deregulated RNA homeostasis in cancer.

About H3 Biomedicine Inc.

H3 Biomedicine is a Cambridge, Massachusetts-based biopharmaceutical company specializing in the discovery and development of precision oncology treatments, which was established as a subsidiary of Eisai's U.S. pharmaceutical operation, Eisai Inc. 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.

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