PRT543 Trials

Proven success in rapidly advancing novel therapeutics to clinical trials for cancer patients.

Phase 1 Study of Oral PRT543 in Patients with Advanced Solid Tumors and Hematologic Malignancies– PRT543-01 (NCT03886831)


Oral PRT543

Trial Status:

Actively Recruiting

Trial Information:

PRT543 is designed to be an oral, potent, and selective Protein Arginine Methyl Transferase 5 (PRMT5) inhibitor. PRMT5 is believed to mediate cancer progression in solid tumors and hematologic malignancies.

This Phase 1 dose-escalation, dose-expansion study is evaluating PRT543 in patients with advanced cancers who have exhausted available treatment options. The purpose of this study is to define a safe dose and schedule to be used in subsequent development of PRT543. The study will enroll patients with certain advanced solid tumors and hematologic malignancies, including:

  • Adenoid cystic carcinoma (ACC)
  • Homologous recombination deficient positive (HRD+) tumors
  • Heme malignancies including myelofibrosis (MF), diffuse Large B-Cell Lymphoma (DLBCL) and mantle cell lymphoma (MCL)
  • Splicing positive tumors including myelodysplastic syndrome (MDS), acute myeloid leukemia (AML), and others
  • MYC/MYB amplified tumors

For full trial information, please visit or contact us directly at [email protected].