TLDR; EMD AML Trial Clinical trialdesign: open label,...

TLDR; EMD AML Trial

Clinical trialdesign: open label, non-randomised study with two clinical arms:

1. Utilises Zantrene as a high dose for 7 days inpatients with EMD AML who can tolerate chemotherapy, followed by cycles ofconsolidation treatment of Zantrene in combination with Ara-C (SOC)

Aim: bridging the patient to an allogeneichematopoietic stem cell transplant.

2. Utilises Zantrene as a low dose FTO-targetedagent in combination with ASTX727 (an oral formulation of decitabine, anucleoside metabolic inhibitor, and cedazuridine, a cytidine deaminaseinhibitor which has been FDA approved for use in adult patients with MDS) forthose who are unable or unwilling to tolerate chemotherapy. This arm willevaluate further the synergistic effects of optimal dosing of decitabine andZantrene in leukaemic cells which were identified by Prof Chen’s COH/BRI data,and validated earlier this year in an EMD setting by A/Prof VerrillsLaboratory.

Aim: Safety and tolerability of theASTX727/Zantrene regimen.

NB: ASTX727 — supply agreement with AstexPharm was announced last week 30/3/22.

· Primary Endpoint: completeresponse and complete response with incomplete haematological recovery

· Secondary Endpoint: safety andtolerability of Zantrene, overall and event-free survival, and the correlationof FTO expression or other biomarkers with response to treatment.

· Timeframe: 36-40 months tocomplete study. Full recruitment over the next 18 months.

· Open label nature means that patient outcomesare obtained as patients are treated. So we will expect updates on regularbasis, with first patient expected to begin treatment soon after governanceapproval is obtained.

· Comparator is not used as patient populationis considered without existing treatment options.