April 3, 2014

Cambridge, MAH3 Biomedicine Inc., a biopharmaceutical company specializing in the discovery and development of precision medicines for oncology, announced today that it has named its lead investigational drug candidate for its program that targets SF3B1, a component of the human splicing machinery that has been implicated and shown to be frequently mutated in multiple cancers. The Company will present data validating the importance of the underlying science behind the program during a mini-symposium to be held on April 7, 2014, at The American Association for Cancer Research (AACR) Annual Meeting 2014 in San Diego.

“We are excited to present the research surrounding SF3B1, a novel drug target in some of the hardest-to-treat cancers, including chronic lymphocytic leukemia (CLL), myelodysplastic syndrome (MDS) and solid tumors such as pancreatic cancer,” said Markus Warmuth, M.D., President and CEO of H3 Biomedicine. “Our research has shed significant light into the mechanism of SF3B1 mutations and their role in aberrant splicing and irregular epigenetic controls. At H3, we have translated these findings into a fast-moving drug discovery project. We have identified a lead drug candidate for the program in less than two years: a remarkable timeframe for a novel drug target.”

“The opportunity to share this data at AACR—a prestigious peer-reviewed conference—validates our approach to developing genomic-based precision medicines,” continued Dr. Warmuth. “We have commenced IND-enabling studies with our top candidate and are hoping to quickly advance the molecule through these studies.”

About SF3B1

SF3B1 (splicing factor 3B subunit 1) is a component of the splicing machinery. Recent publications have implicated mutations in SF3B1 in both hematological malignancies, including myelodysplastic syndrome and chronic lymphocytic leukemia, as well as solid tumors such as those found in skin, breast and pancreatic cancers.H3 Biomedicine’s lead research and discovery program is designed to develop drugs that target the vulnerabilities related to SF3B1’s disease-causing mechanisms. During the AACR mini-symposium, H3 Biomedicine will present findings that support targeting splicing inhibitors as a promising investigational approach for patients with cancer carrying SF3B1 mutations.

H3 Biomedicine at AACR

H3 Biomedicine will present data related to its lead candidate program in a mini-symposium at AACR entitled “SF3B1 mutations induce aberrant mRNA splicing in cancer and confer sensitivity to spliceosome inhibition.” The event will occur on Monday, April 7, 2014, at the San Diego Convention Center starting at 3:35 p.m. PT.

In addition, H3 will also present a poster at AACR discussing findings of its cancer cell line sequencing project—an important tool in developing its lead candidate program—entitled “Exome sequencing of tumor cell lines: optimizing for cancer variants.” This presentation occurs on Tuesday, April 8, 2014, from 1:00–5:00 p.m. PT at the San Diego Convention Center.

About H3 Biomedicine Inc.

H3 Biomedicine is a biopharmaceutical company specializing in the discovery and development of precision oncology treatments. Leveraging an unprecedented collaboration with Eisai, which provides research funding and access to the capabilities and resources of a global pharmaceutical company, H3 Biomedicine combines long-term vision with operational independence. Using modern synthetic chemistry, chemical biology and human genetics, the company seeks to bring the next generation of cancer treatments to market with the goal of improving the lives of patients. H3 Biomedicine is based in Cambridge, Massachusetts. Visit h3biomedicine.com for more information.

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