Phase 1b Clinical Study of APTO-253
The Phase 1b, multicenter, open-label dose-escalation clinical trial of APTO-253 is designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamic responses and establish the recommended phase 2 dose of APTO-253 as a single agent. APTO-253 is being administered once weekly, over a 28-day cycle. The dose-escalation portion of the study is expected to enroll up to 20 patients with relapsed or refractory AML and high-risk MDS, and is designed to then transition, as appropriate, to single-agent expansion cohorts in AML and MDS, followed by combination studies.
We have completed dosing of the first three cohorts in our Phase 1b trial with APTO-253, with only one patient required in each of the first two cohorts and three patients in the third cohort. In the patients on the first three dose cohorts, no drug-related adverse events had been observed, including no myelosuppression, and dosing was planned to continue to ascend until a maximum tolerated dose is reached. Biomarker data from AML and MDS patients in the first three cohorts have demonstrated reductions of MYC gene expression in their peripheral blood cells. We are currently dosing patients in the fourth cohort. View Clinical Trial