January 9, 2018

Cambridge, MA — H3 Biomedicine Inc., a clinical stage biopharmaceutical company specializing in the discovery and development of next generation cancer medicines using its data science and precision chemistry product engine, today announced that it will outline its key 2018 strategies and goals that build upon its success in advancing three differentiated, internally generated cancer therapies into clinical trials. Markus Warmuth, M.D., President and CEO of H3 Biomedicine, will provide an overview of the company and further details on its 2018 goals during its presentation at the 36th Annual J.P. Morgan Healthcare Conference on Thursday, January 11, 2017, at 9:00 a.m. PST in the Elizabethan D conference room at the Westin St. Francis Hotel in San Francisco.

“Over the past six years, we built a leading oncology product engine at the interface of data science and precision chemistry with the goal of discovering cancer therapies that address molecular traits driving cancer in select patient segments. Last year, we continued to see the successful translation of this innovation as we advanced our third internally generated therapy into clinical trials and garnered significant interest from the scientific and medical community as evidenced by a large number of key publications, papers and presentations on our product engine and clinical pipeline,” said Dr. Warmuth. “With initial clinical data expected from at least two of our three therapies and our product engine continuing to drive forward new programs to achieve additional investigational new drug (IND) filings in the future, 2018 will be a pivotal year for H3 as we continue to advance toward our vision — as embodied in our name — of improving Health and providing Hope for Humans.”

2017 Accomplishments

  • Received FDA acceptance for our IND application and dosed first patients with H3B-6545, an oral, first-in-class ESR1 covalent antagonist targeting wild-type and mutant estrogen receptor α in endocrine-therapy resistant metastatic breast cancer patients. H3 has two additional therapies in Phase I clinical trials including:
    - H3B-6527, an oral, potent and highly selective small molecule covalent inhibitor of FGFR4 for treatment of hepatocellular carcinoma (HCC) patients with overexpression of FGF19; and
    - H3B-8800, an oral, first-in-class, selective SF3b modulator treating hematological malignancies with spliceosome mutations;
  • Achieved orphan drug designation in the U.S. from the Food and Drug Administration for H3B-8800
    in acute myelogenous leukemia and chronic myelomonocytic leukemia and H3B-6527 in HCC;
  • Garnered significant interest from the medical and scientific community with numerous publications
    accepted in leading scientific and medical journals, with highlights as follows:
    - Publication in Cancer Research highlighting an oral dosing of H3B-6527 in mice led to dose-dependent pharmacodynamic modulation of FGFR4 signaling and tumor regression in FGF19 altered HCC xenograft models;
    - Publication in Nature Communications highlighting preclinical data describing a novel immune evasion mechanism in bladder cancer;
    - Publication in Nature Communications reporting preclinical data from novel discovery research around RNA splicing modulators;
    - Oral presentation at the American Association for Cancer Research’s (AACR) New Drugs on the Horizon session on data from H3B-6545;
    - Four poster presentations at AACR on preclinical programs highlighting splicing platform and lead position in drugging RNA splicing defects in cancer;
    - Oral presentation at the International Liver Cancer Association (ILCA) annual meeting demonstrating FGFR4 inhibition with H3B-6527;
    - Poster presentation at the San Antonio Breast Cancer Symposium (SABCS) demonstrating potent inhibition of ERα with H3B-6545;
  • Extended multi-year collaboration with Foundation Medicine to continue strengthening leadership position in discovering previously unknown molecular traits driving cancers to select cancer patient populations.

Key 2018 Strategies and Goals

Drive forward clinical pipeline of differentiated cancer therapies to achieve proof of concept rapidly

  • Achieve Phase 2 dose for all three clinical stage programs and initiate expansion cohorts to critical decision points;
  • Present initial clinical data from the Phase 1/2 dose escalation and/or expansion cohorts for at least two of the three programs; and
  • Initiate Phase 1B combination studies for H3B-6545 and H3B-6527 as key component of strategy for these therapies.

Continue to advance our big data science and precision chemistry product engine to deliver additional INDs in the future

  • Advance FGFR4 mutant inhibitor program addressing hot spot mutations leading to resistance to first generation FGFR4 inhibitors to IND-enabling studies to support 2019 IND filing;
  • Initiate lead optimization on one additional program;
  • Achieve preclinical proof-of-concept for splice modulator-loaded antibodies, an expansion of H3’s capabilities in splice modulation and a key area of differentiation for H3; and
  • Continue to expand data science engine to empower initiation of additional exploratory research projects.

About H3 Biomedicine Inc.

H3 Biomedicine, a Cambridge, Massachusetts-based biopharmaceutical company specializing in the discovery and development of precision oncology treatments, was established in 2011 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 genomics-based 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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