December 4, 2015

Cambridge, MA — H3 Biomedicine Inc., a biopharmaceutical company specializing in the discovery and development of precision medicines for oncology, announced today that data from one of its lead oncology programs targeting SF3B1, found in multiple cancers, will be presented at the upcoming 2015 American Society of Hematology (ASH) Meeting and Exposition in Orlando, December 4–8, 2015. The poster presentation from Buonamici et al., (abstract 1643) will be take place Saturday, December 5, 2015, 5:30–7:30 p.m.

Data to be presented at ASH will extend on pre-clinical findings recently published in the November 3 issue of Cell Reports. ( which detailed the ongoing work of H3 Biomedicine scientists in identifying defective splicing mechanisms of SF3B1 mutations. New data will be presented indicating that SF3B1 hotspot mutations induce aberrant splicing and downregulation of several genes that contribute to a block in erythroid differentiation, one of the key biological defects observed in myelodysplastic syndromes (MDS).

Furthermore, additional data highlighting the potential of H3 Biomedicine’s approaches to targeting splicing factor mutated hematological malignancies will be presented in the Plenary Session by Dr. Omar Abdel-Wahab, a hematologist at Memorial Sloan Kettering Cancer Center (Abstract #4). Dr. Abdel-Wahab’s work has characterized the disease mechanisms of SRSF2 hotspot mutations and demonstrated the therapeutic potential of targeting SF3B1 in pre-clinical models of acute myeloid leukemia. The Plenary Session will take place on Sunday December 6, 2015, 4:00–6:00 p.m., more information on this work can be found at:

“We are pleased to be sharing this important data at the ASH meeting and discuss the progress made by H3 scientists and collaborators towards targeting spliceosome mutated cancers,” said Markus Warmuth, M.D., President and CEO of H3 Biomedicine. “The recent publication of this work and our ASH presentations represent an important step towards advancing our program into clinical trials with the potential to make a difference in patients with a range of hematologic cancers.”

H3 is expected to submit an Investigational New Drug (IND) Application in 2016.


Presentation Title: Heterozygous Hotspot SF3B1 Mutations Found in Myelodysplastic Syndromes Downregulate Genes Involved in Differentiation of Erythroid Cells
Date & Time: Saturday, December 5, 2015, 5:30–7:30 p.m.
Session Title: Session: 636. Myelodysplastic Syndromes – Basic and Translational Studies: Poster I
Abstract Number: 1643
Location: PM Hall A, Level 2 (Orange County Convention Center)

Presentation Title: Therapeutic Targeting of Spliceosomal Mutant Myeloid Leukemias through Modulation of Splicing Catalysis
Date & Time: Sunday, December 6, 2015, 2:00–4:00 p.m.
Session Title: Plenary Scientific Session
Abstract Number: 4
Location: Hall D, Level 2 (Orange County Convention Center)

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, secondary AML 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 programs in splicing are designed to develop drugs that target the vulnerabilities related to SF3B1’s disease-causing mechanisms.

About H3 Biomedicine Inc.

H3 Biomedicine is a Cambridge, Massachusetts based 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. For more information, please visit

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