Moleculin Biotech has completed dosing in Part A of its pivotal Phase 2/3 MIRACLE trial, which is testing Annamycin plus cytarabine in adults with relapsed or refractory acute myeloid leukemia (AML). The company expects to report comprehensive results from the 90-patient part of the study in the first quarter of 2027.
The results are intended to help Moleculin choose an Annamycin dose for the next part of the trial and assess the treatment across patients with different prior treatment histories. No efficacy results were included in the announcement.
Key details
- Trial design: Part A is randomized and double-blind. It compares two doses of Annamycin, each combined with cytarabine, against cytarabine plus placebo. Each treatment arm is evaluated after one cycle of therapy.
- Patient mix: Moleculin expects the completed 90-patient group to include roughly equal numbers of patients whose AML relapsed or resisted intensive “7+3” chemotherapy and those whose disease relapsed or resisted venetoclax-based treatment. Among the first 45 patients, the mix was about 70% and 30%, respectively.
- Purpose of the upcoming analysis: The Q1 2027 readout is expected to examine efficacy and safety across the full Part A population and inform dose selection for Part B.
- Safety comment: Moleculin said blinded interim data have continued to show no cardiotoxicity. Because the data are blinded, the announcement did not provide treatment-specific safety results.
- How to interpret remission rates: The trial measures outcomes after one treatment cycle. Moleculin cautioned that remission rates may therefore be lower than those in some historical studies that allowed multiple cycles. It says the most relevant comparison will be between the Annamycin groups and the trial’s concurrent control group, which is assessed on the same one-cycle basis.
What changes
Completion of dosing sets up the planned full Part A analysis, but it does not yet establish whether Annamycin is effective or which dose will be advanced. The next major milestone is the comprehensive data readout expected in the first quarter of 2027.