Clinical Trials

Evaluating potential of investigational medicines

  • DSTAT for Acute Myeloid Leukemia (AML)

    Chimerix initiated a Phase 3 trial of DSTAT in combination with standard chemotherapy (cytarabine plus anthracycline, or “7+3”) in newly diagnosed AML patients, DASH-AML. The scientific literature and recent clinical studies suggest that adding DSTAT to standard chemotherapy for AML may improve patient outcomes. DSTAT has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA) for the treatment of AML.

    About DSTAT for AML

    DSTAT may enhance eradication of leukemic cells through inhibition of resistance pathways and eliminate LSCs by sensitizing them 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.

    DASH AML is a randomized, double-blind trial of approximately 570 newly diagnosed AML patients. The trial enrolls patients 60 years of age or older who have an intermediate or adverse genetic risk profile and patients between 18 and 60 years of age who have an adverse genetic risk profile. Patients will be randomized 1:1 to receive DSTAT in combination with standard of care cytarabine plus anthracycline (7+3) induction and cytarabine consolidation chemotherapy or to receive standard of care (7+3) induction and consolidation chemotherapy alone. Patients with FLT-3 mutations will be allowed in the study and will be eligible to receive midostaurin.

    The primary endpoint of the trial is overall survival (OS). In addition, FDA has indicated that event-free survival (EFS), using complete response with hematologic recovery to define induction success (CR), is acceptable as an endpoint for regulatory approval. Other endpoints to be evaluated in the proposed trial include minimal residual disease (MRD), relapse-free survival (RFS), time to hematologic recovery, and induction response.
    In order to supplement the previously reported data from the pilot and Phase 2 trials and provide additional evidence of DSTAT’s mechanism of action, the proposed Phase 3 trial includes an early assessment of comparative CR and MRD rates among the first 80 evaluable patients.

    For more information on our clinical trials, contact us at or ( NCT04571645).

  • ONC201

    The current clinical program for ONC201 includes a 50 subject registration cohort of patients greater than 2 years of age with recurrent diffuse midline glioma who harbor the H3 K27M-mutation in their tumor, measurable disease, prior radiation that was greater than 90 days from starting ONC201 and evidence of progressive disease, among other criteria. This registration cohort is comprised of patients from multiple clinical studies and has completed enrollment. A BICR analysis of objective response rate is expected to take place in 2021 which, if favorable, may form the basis for approval of ONC201 in the United States. A BICR of the first 30 patients was completed and presented at the Society of Neuro-Oncology meeting held in November 2020. ONC201 has demonstrated a favorable safety profile with a database of over 350 treated patients. The most commonly reported adverse events (AEs) were nausea/vomiting, fatigue and decreased lymphocyte counts. There have been no reports of discontinuation due to drug-related AEs among patients who have received continuous weekly dosing for more than a year.

    The FDA has granted ONC201 Fast Track Designation for the treatment of adult recurrent H3 K27M-mutant high-grade glioma, Rare Pediatric Disease Designation for treatment of H3 K27M-mutant glioma, and Orphan Drug Designations for the treatment of glioblastoma and for the treatment of malignant glioma.

    Over 300 subjects with recurrent high-grade gliomas, including gliomas with H3 K27M-mutations, have been treated with ONC201 across three company-sponsored studies and expanded access.

    About ONC201 for Cancer

    ONC201 is a highly selective antagonist of dopamine receptor D2 (DRD2) and ClpP agonist that is able to penetrate the blood-brain-barrier effectively. ONC201 engages proven anti-cancer pathways that lead to apoptosis in cancer cells. ONC201 is currently in a registration program for H3 K27M-mutant gliomas and is in Phase II trials in a variety of oncology indications, such as neuroendocrine tumors.

    For more information on our clinical trials, contact us at

  • ONC206

    ONC206 is a DRD2 antagonist and ClpP agonist with nanomolar potency that demonstrates enhanced non-competitive DRD2 antagonism relative to ONC201 and disruption of DRD2 homodimers. ONC206 exhibits a distinct gene expression profile as well as single agent and combinatorial efficacy with ONC201 in cells with acquired resistance to ONC201. ONC206 is effective in preclinical models of difficult-to-treat neuroendocrine tumors and high-grade gliomas. It affects some of the same downstream pathways as ONC201, including activation of the integrated stress response and inhibition of Ras signaling, leading to selective killing of tumor cells.

    The first-in-human clinical trial for adults with recurrent primary central nervous system tumors is ongoing at the National Institute of Health.