We are investigating transformative scientific approaches to better treat deadly diseases.

In oncology, Chimerix is investigating smarter ways to treat  acute myeloid leukemia (AML), and potentially other hematologic indications. With more than 21,000 new cases of AML diagnosed annually in the U.S. and a five-year survival rate in elderly patients of less than 30%, there is a clear and urgent need for life-extending and life-saving treatment options.1

What is AML?

AML is a type of cancer in the blood and bone marrow that rapidly progresses and interferes with the production of normal white blood cells, red blood cells and platelets.

How is AML treated?

Currently, most patients receive chemotherapy, sometimes in combination with a targeted therapy, as treatment for AML. The goal is to eradicate as many AML cancer cells and leukemic stem cells as possible. If patients respond well, they may go on to receive a stem cell transplant.

What is the prognosis of AML with treatment?

Patients with AML receiving chemotherapy treatment may experience up to a 70% mortality rate within the first year of treatment, depending on age and comorbidities.2 Even patients who respond well to chemotherapy are likely to relapse in 12 months or less.3

Why is AML so difficult to treat?

  • AML is considered a heterogeneous disease, meaning there are a multitude of chromosomal abnormalities and gene mutations that can cause the disease
  • AML cells develop from stem cells that undergo both genetic and epigenetic changes resulting in subpopulations of cells with different phenotypes
  • Subpopulations of AML cells may contain distinct cellular abnormalities or mutations contributing to treatment resistance

What are targeted therapies?

  • Targeted therapies target specific genes, mutations and proteins that contribute to cancer growth and survival while conventional chemotherapies affect a broader population of rapidly dividing cells
  • Doctors often use targeted therapies in combination with chemotherapy to target and kill as many cancer cells as possible

Why do existing targeted therapies fall short?

  • Targeted treatments are designed to target specific mutations in AML
  • In many cases some of the diseased cells within a patient do not express the targeted mutation or the mutations themselves change during the course of the disease.
  • Relapse can occur if not all AML blasts and leukemic stem cells are eradicated


Dociparstat sodium (DSTAT)

Chimerix is currently developing dociparstat sodium, also known as DSTAT, a new chemical entity that has the potential to treat heterogeneous AML via multiple pathways that contribute to chemotherapy resistance and frequent relapse in this disease.

In contrast to targeted therapies, DSTAT may enhance eradication of leukemic blasts and dormant leukemic stem cells (LSC) by making them more sensitive to chemotherapy. DSTAT is thought to increase the depth of response to standard chemotherapy, thereby reducing the potential for relapse, and improving long-term outcomes for patients with AML. Specifically, DSTAT inhibits binding and/or interactions of proteins including CXCL12, P-selectin, high mobility group box 1 (HMGB1), platelet factor 4 (PF4) and human leukocyte elastase (HLE) which are involved in leukemic blast adhesion, survival and proliferation. Additionally, these DSTAT binding interactions may reverse quiescence of LSC, inducing cell division which renders the cells sensitive to the cytotoxic mechanisms of chemotherapy.a

Promoting LSC/resistant AML blast replication with DSTAT would complement multiple current and emerging therapies, including conventional chemotherapy and targeted agents.

Read more about DSTAT.


  1. NIH National Cancer Institute. Cancer Stat Facts: Leukemia – Acute Myeloid Leukemia (AML) Retrieved from https://seer.cancer.gov/statfacts/html/amyl.html
  2. Meyers J, Yu Y, Kaye JA, Davis KL. Medicare fee-for-service enrollees with primary acute myeloid leukemia: an analysis of treatment patterns, survival, and healthcare resource utilization and costs. Appl Health Econ Health Policy. 2013;11(3):275-286. doi: 10.1007/s40258-013-0032-2.
  3. Walter RB, Othus M, Burnett AK, et al. Resistance prediction in AML: analysis of 4601 patients from MRC/NCRI, HOVON/SAKK, SWOG and MD Anderson Cancer Center. Leukemia. 2015;29(2):312-320. doi: 10.1038/leu.2014.242.


  aZhang 2012 JBC 287(8); Kovacsovics 2018 Blood Adv 2(4); Yasinska 2018 Oncoimmunology 7(6); Rao 2010 Am J Physiol Cell Physiol 299; Zheng 2016 Am J Cell and Mol Bio 56(1); Griffin 2014 Am J Resp Cell and Mol Bio 50(4); Lakshmi 2010 J Biomed Mat Res 95(1); Yu 2005 Blood 105(9);  Lapierre 1996 Glycobiology 6(3); Tavor 2005 Blood 106(6); Kummarapurugu 2018 JBC 293(32)