Core Viewpoint - Moleculin Biotech, Inc. has amended its clinical trial protocol for the Phase 3 "MIRACLE" trial evaluating Annamycin in combination with Cytarabine for treating relapsed or refractory acute myeloid leukemia (AML), aiming for accelerated approval and earlier data unblinding [1][2][8] Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses, with its lead program Annamycin targeting relapsed or refractory AML and soft tissue sarcoma [7][8] Clinical Trial Details - The MIRACLE trial will utilize an adaptive design, initially randomizing 75 to 90 subjects to receive high dose cytarabine combined with either placebo or two different doses of Annamycin (190 mg/m2 and 230 mg/m2) as recommended by the FDA [3][4] - The amended protocol allows for the unblinding of preliminary primary efficacy data and safety/tolerability results at 45 subjects, with expectations to reach this number in the second half of 2025 [3][5] - Part B of the trial will involve approximately 244 additional subjects randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin, with the optimum dose determined based on safety, pharmacokinetics, and efficacy [4] Regulatory Status - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory AML, as well as Orphan Drug Designation from the European Medicines Agency for the same condition [6]
Moleculin Accelerates Planned Unblinded Data Readout for MIRACLE Phase 3 R/R Acute Myeloid Leukemia (AML) Pivotal Trial to H2 2025