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 four 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 subsequent cohorts. 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, with sustained active drug species in the 2-3µM range at the fourth dose level of 100mg/m2. 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 fifth dose level of 150mg/m2. View Clinical Trial