Moleculin Receives Positive FDA Guidance to Accelerate MIRACLE Trial for Relapsed or Refractory AML

Moleculin Biotech, Inc., has received positive FDA guidance on its Investigational New Drug amendment, which has allowed a size reduction of the phase III MIRACLE trial evaluating annamycin combined with cytarabine for relapsed or refractory acute myeloid leukemia (AML).

The global trial is divided into two parts: part A and part B. The amended protocol includes reducing the size of part B by approximately 10%, according to a statement by Walter Klemp, chairman and chief executive officer of Moleculin.

In part A, the first 75 to 90 patients will be randomized 1:1 to receive high-dose cytarabine (HiDAC) combined with placebo, 190 mg/m² of annamycin, or 230 mg/m² of annamycin.

“The amended protocol allows for the unblinding of preliminary primary efficacy data (Complete Remission or CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (at 75 to 90 subjects),” Moleculin wrote in a press release.

In part B, approximately 220 additional patients will be randomized 1:1 to receive either HiDAC plus placebo or HiDAC plus the optimum dose of annamycin. In alignment with the FDA’s Project Optimus initiative, the optimum dose will be determined based on the balance of safety, pharmacokinetics, and efficacy.

The FDA previously granted Fast Track Status and Orphan Drug Designation to annamycin for relapsed or refractory AML.

Reference

Moleculin receives positive FDA guidance for acceleration of its registration-enabling MIRACLE trial for R/R acute myeloid leukemia (AML). PR Newswire. February 13, 2025. Accessed February 18, 2025.