Our lead molecule, APR-246, has been shown to reactivate mutant p53 and thereby induce programmed cell death in human cancer cells. APR-246 has demonstrated compelling pre-clinical anti-tumor activity in a wide variety of solid and hematological (blood) tumors, including MDS, AML and ovarian cancer, among others. Additionally, strong synergy has been seen with both traditional anti-cancer agents, such as chemotherapy, as well as newer mechanism-based anti-cancer drugs and immuno-oncology checkpoint inhibitors.
A Phase I clinical study has been completed, demonstrating a favorable safety profile and both biological and clinical responses in hematological tumors with mutations in the p53 gene. Phase Ib/II clinical studies in MDS / AML and a Phase III clinical study in MDS are ongoing. Additional hematologic malignancy indications are planned.