Our patient focus relies on a deep commitment to exploring fundamental science.

Scientific Approach

Unlocking cancer genomics, real-world patient data, and biomarkers in clinical research to deliver on the promise of precision medicine.

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H3 Biomedicine Clinical Programs

Through its deep understanding of cancer genomics and its target-centric drug discovery approach, H3 has created a compelling portfolio of clinical assets. Each program is tailored to a genomically and clinically defined population to increase the chances of bringing meaningful advances to the individual patients most likely to benefit. Click below to learn more about each of our ongoing clinical programs.

All agents are investigational. The information you are viewing is what is known or hypothesized about each agent’s proposed site of action and suggested biologic activity, and is not meant to convey conclusions of safety or effectiveness prior to any regulatory approval from a health authority. There is no guarantee that any of these agents will successfully complete clinical development or be available commercially.

Product Candidates
Phase 1
Phase 2
Breast Cancer
H3B-6545 | Phase 2
Breast Cancer

Estrogen receptor alpha (ERα) plays an important oncogenic role in the genesis and progression of luminal breast cancers,1 and historically has been a target of endocrine therapies. However, recently, hotspot mutations in ERα have been detected in nearly 30% of endocrine-therapy resistant metastases. Functional studies have shown that these ERα mutations can confer ligand-independent activation of the ERα pathway and can promote partial resistance to existing classes of ER-directed therapies.2,3 H3B-6545, a first-in-class small molecule selective estrogen receptor covalent antagonist (SERCA) demonstrates activity in tumor models that harbor wild-type or mutant ERα.4 H3B-6545 activity against ERα mutants resistant to standard therapy provides an opportunity to target a currently unmet medical need both as a single agent and in combination with other breast cancer therapies.

Hepatocellular Carcinoma
H3B-6527 | Phase 1
Hepatocellular Carcinoma

Receptor tyrosine kinases (RTKs) can be dysregulated in cancer cells and can frequently promote abnormally rapid tumor growth and development. Hepatocellular carcinoma (HCC) can be driven in this way by hyperactivation of the fibroblast growth factor receptor 4 (FGFR4). H3B-6527 is a selective, orally bioavailable, and covalent inhibitor of FGFR4 that has demonstrated tumor regression in several preclinical models of HCC.5 H3B-6527 is being tested specifically in patients with FGFR4-dysregulated advanced HCC.

Myelodysplastic Syndromes, Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia
H3B-8800 Program | Phase 1
Myelodysplastic Syndromes, Acute Myeloid Leukemia, Chronic Myelomonocytic Leukemia

H3B-8800 is a selective, and orally bioavailable small molecule modulator of wild-type and mutant SF3b complex.6 The SF3b complex is a key component of the spliceosome that is found in the nucleus of cells and is responsible for the removal of noncoding introns from a transcribed pre-messenger RNA.7,8 Recurrent heterozygous mutations in several core members (SF3B1, U2AF1, SRSF2, ZRSR2) of the spliceosome have been identified in both hematological malignancies, including myelodysplastic syndrome, acute myeloid leukemia, chronic myelomonocytic leukemia and chronic lymphocytic leukemia, as well as select solid tumors such as those found in uveal melanoma, lung, breast and pancreatic cancers.9,10,11 Mutations in the core spliceosome components lead to aberrant mRNA splicing that may contribute to disease pathogenesis.9,10 Preclinical data indicates that H3B-8800 modulates RNA splicing and shows preferential antitumor activity in a range of spliceosome mutant cancer models.6 Initial clinical development is ongoing in patients with hematological malignancies (e.g. MDS, AML, and CMML) that may carry mutations in the core spliceosome genes and will assess the safety and preliminary efficacy of H3B-8800.12

Advanced Solid Tumors or Lymphomas
E7766 Program | Phase 1
Advanced Solid Tumors or Lymphomas

The human immune system is capable of detecting and eliminating cancer cells, but tumors evolve strategies to evade the immune surveillance. STING (Stimulator of Interferon Genes) is an innate immune sensor of aberrant cytosolic dsDNA, and its activation by binding to ligand bridges the innate immunity and adaptive T cell response. E7766 is a STING agonist with broad specificity to all major genetic variants of human STING.13 Preclinical studies suggest that treatment with E7766 by intratumoral administration has anti-tumor activity at both local and systemic levels with induction of effective tumor-specific memory immune response. Intratumoral E7766 is currently being evaluated in patients with advanced solid tumors or lymphomas.

Non-Muscle Invasive Bladder Cancer
E7766 Program | Phase 1
Non-Muscle Invasive Bladder Cancer

The human immune system is capable of detecting and eliminating cancer cells, but tumors evolve strategies to evade the immune surveillance. STING (Stimulator of Interferon Genes) is an innate immune sensor of aberrant cytosolic dsDNA, and its activation by binding to ligand bridges the innate immunity and adaptive T cell response. E7766 is a STING agonist with broad specificity to all major genetic variants of human STING13. Preclinical studies suggest that treatment with E7766 by intravesical administration has anti-tumor activity with induction of effective tumor-specific memory immune response. Intravesical E7766 is currently being evaluated in patients with intermediate risk or Bacillus Calmette-Guerin-unresponsive non-muscle invasive bladder cancer.

Scientific Publications

Explore the science that underlies our promising programs.

Nature Chemical Biology | March 18, 2013

Translational Synthetic Chemistry

Providing chemical matter to modulate newly identified biological targets—as well as pre-existing but chemically intractable ones—remains a challenge in the… Read More


H3 Biomedicine continues to build and grow its preclinical and clinical portfolio. We are actively collaborating with our existing partners and seeking additional partnering opportunities with a goal to advance our pipeline to efficiently deliver novel precision cancer therapies to patients.

With our open and innovative collaboration model, we seek to bring together the best scientists, academic institutions and biotechnology and pharmaceutical companies to join our expertise, insights and resources together. H3 has entered into collaborative relationships with:

In December 2018, Bristol Myers Squibb (BMS), H3 Biomedicine and Eisai entered into a multi-year research collaboration leveraging H3’s RNA splicing platform. The collaboration will explore modulating RNA splicing to target cancer cells and help more patients experience the benefits of immunotherapy. Research will be jointly conducted by H3 and BMS. BMS will be responsible for development and commercialization of selected compounds, while H3 Biomedicine/Eisai retain the right to co-develop and co-commercialize certain compounds.

Read Press Release

In February 2015, Foundation Medicine, Inc. (NASDAQ:FMI) and H3 Biomedicine Inc. announced a multi-year collaboration for the discovery and development of precision medicines in oncology. The collaboration marries Foundation Medicine’s comprehensive clinical genomic knowledgebase of genomic profiles, the largest of its kind, FoundationCORE™, with H3 Biomedicine’s drug discovery engine and computational biology platform. Based on the unique genomic information of individual cancers, the approach aims to identify potential drug targets and patient selection biomarkers, rapidly accelerate clinical development, and lead to the commercialization of new, safe and effective precision medicines for individuals living with cancer.

In July 2019, H3 Biomedicine joined the Cancer Dependency Map (DepMap) Consortium, a new academic-industry partnership program established by the Cancer Program of the Broad Institute of MIT and Harvard. H3 Biomedicine joins other pharmaceutical and biotechnology partners to tap into the DepMap project’s extensive datasets and know-how to gain insights into cancer targets. DepMap project includes access to genomically-characterized, patient-derived cell line models, massively-parallel compounds screens, genome-wide CRISPR dependency screens and computational methods to help integrate and identify cancer targets.

Read Press Release


  1. Spicer D, Pike M. Breast cancer prevention through modulation of endogenous hormones. Breast Cancer Res TR. 28:179-193.
  2. Toy W, Shen Y, Won H, Green B, Sakr R, Will M, Li Z, Gala K, Fanning S, King T, Hudis C, Chen D, Taran T, Hortobagyi G, Green G, Berger M, Baselga J, Chandarlapaty S., et al. ESR1 ligand-binding domain mutations in hormone-resistant breast cancer. Nat Genet. 45(12):1439-1447.
  3. Puyang X, Furman C, Zheng G, Wu Z, Banka D, Aithal K, Agoulnik S, Bolduc D, Buonamici S, Caleb B., et al. Discovery of Selective Estrogen Receptor Covalent Antagonists for the Treatment of ERaWT and ERaMUT Breast Cancer. Cancer Discov. 2018(8):1176-1193.
  4. Korpal M, Puyang X, Furman C, Zheng G, Banka D, Wu J, Caleb B, Karr C, Mackenzie C, Rimkunas V., et al. Development of First-in-Class Oral Selective ER Covalent Antagonist (SERCA) for the Treatement of ERaWT and ERaMUT Breast Cancer. Poster presented at: San Antonio Breast Cancer Symposium; 2017 Dec 5-9; San Antonio TX.
  5. Joshi J, Coffey H, Corcoran E, Tsai J, Huang C, Ichikawa K, Prajapati S, Hao M, Bailey S, Wu J, et al. H3B-6527 Is a Potent and Selective Inhibitor of FGFR4 in FGF19-Driven Hepatocellular Carcinoma. Cancer Res 2017(77):6999-7013.
  6. Seiler M, Yoshimi A, Darman R, Chan B, Keaney G, Thomas M, Agrawal AA, Caleb B, Csibi A, Sean E, et al. H3B-8800, an orally available small-molecule splicing modulator, induces lethality in spliceosome-mutant cancers. Nat Med. 2018 May;24(4):497-504. doi: 10.1038/nm.4493. Epub 2018 Feb 19.
  7. Sharp PA. Split genes and RNA splicing. Cell. 1994 Jun 17;77(6):805-15. doi: 10.1016/0092-8674(94)90130-9.
  8. Wilkinson ME, Charenton C, Nagai K. RNA Splicing by the Spliceosome. Annu Rev Biochem. 2019 Dec 3. doi: 10.1146/annurev-biochem-091719-064225.
  9. Agrawal AA, Yu L, Smith PG, Buonamici S. Targeting splicing abnormalities in cancer. Curr Opin Genet Dev. 2018 Feb;48:67-74. doi: 10.1016/j.gde.2017.10.010.
  10. Lee SC, Abdel-Wahab O. Therapeutic targeting of splicing in cancer. Nat Med. 2016 Sep 7;22(9):976-86. doi: 10.1038/nm.4165.
  11. Seiler M, Peng S, Agrawal AA, Palacino J, Teng T, Zhu P, Smith PG; Cancer Genome Atlas Research Network, Buonamici S, Yu L. Somatic Mutational Landscape of Splicing Factor Genes and Their Functional Consequences across 33 Cancer Types. Cell Rep. 2018 Apr 3;23(1):282-296.e4. doi: 10.1016/j.celrep.2018.01.088.
  12. A Phase 1 Study to Evaluate H3B-8800 in Participants With Myelodysplastic Syndromes, Acute Myeloid Leukemia, and Chronic Myelomonocytic Leukemia.
  13. Huang K, Endo A, Kim D, Chandra D, Wu J, Albu D, Ingersoll C, Tendyke K, Loiacono K, Noland T et al. Discovery And Characterization of E7766, A Novel Macrocycle-bridged STING Agonist With Pan-genotypic And Potent Antitumor Activity Through Intravesical and Intratumoral Administration. Poster presented at: American Association for Cancer Research Annual Meeting; 2019 Mar 29-Apr 3; Atlanta, GA.

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