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H3 Biomedicine Receives FDA Acceptance for Investigational New Drug Application Novel Hematologic Compound Bolsters Company’s Emerging Drug Pipeline

H3 Biomedicine Inc., a biopharmaceutical company specializing in the discovery and development of precision medicines for oncology and a member of the Eisai Group, announced today that the U.S. Food and Drug Administration (FDA)

Cambridge, Mass., May 11, 2016 — H3 Biomedicine Inc., a biopharmaceutical company specializing in the discovery and development of precision medicines for oncology and a member of the Eisai Group, announced today that the U.S. Food and Drug Administration (FDA) accepted the company’s Investigational New Drug (IND) application to begin Phase 1 clinical trials for its lead oncology drug candidate H3B-8800. The compound is an oral and selective small molecule modulator of splicing factor 3b subunit 1 (SF3B1), which is being developed by H3 Biomedicine as an anticancer therapeutic agent for the potential treatment of select hematologic malignancies.

“There is a high unmet need for new medicines to treat patients afflicted with a number of hematologic malignancies, and who have very few treatment options,” said Markus Warmuth, M.D., President and CEO of H3 Biomedicine. “Mutations in the spliceosome are a hallmark of various hematological malignancies, and the results from our initial pre-clinical studies targeting these through splice modulation with H3B-8800 are encouraging.”

The SF3B1 program represents H3’s most advanced effort in its strategy to impact deregulated RNA homeostasis in cancer through RNA splicing modulation. The spliceosome is a complex molecular machine found in the nucleus of cells and is responsible for the removal of noncoding introns from a transcribed pre-mRNA. Recurrent heterozygous mutations in several core members (SF3B1, U2AF1, SRSF2, ZRSR2) of the spliceosome have been identified in various cancers and lead to aberrant mRNA splicing that may contribute to disease pathogenesis. H3 is building on its discoveries around targeting SF3B1 and splice modulation to design novel treatments for various cancers.

“We are pleased the FDA has accepted H3’s application for the evaluation of H3B-8800 in patients with blood cancers,” said Pete Smith, PhD, Vice President, Drug Discovery Biology for H3 Biomedicine. “H3 is committed to developing medicines like H3B-8800 and looks forward to the evolution of all our pipeline programs.”

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 an 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, 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 h3biomedicine.com.

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