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 escalation through the first five dose levels (20, 40, 66, 100, 150mg/m2). The clinical experience to date has shown continued favorable safety and tolerability in heavily pretreated patients with relapsed or refractory AML and MDS, and continued dose-related exposure. Biomarker data from AML and MDS patients have demonstrated reductions in MYC gene expression in their peripheral blood cells. We are currently dosing patients at the sixth dose level of 210mg/m2. View Clinical Trial