H3 Biomedicine Receives FDA Acceptance for Investigational New Drug Application

CAMBRIDGE, Mass.–(BUSINESS WIRE)–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 https://www.h3biomedicine.com/.