PRT543 is designed to be a potent, selective, and oral PRMT5 inhibitor with dose-dependent pharmacodynamics (PD) and predictable pharmacokinetics (PK). PRT543 has demonstrated preclinical activity both as a single agent and in combination therapy.

PRT543 is being studied in a Phase 1 clinical trial in advanced solid tumors and select myeloid malignancies, including adenoid cystic carcinoma (ACC) and splicing mutated solid tumors and myeloid malignancies.