Moleculin Biotech, Inc. has announced Institutional Review Board (IRB) approval for its Phase 3 pivotal trial protocol, known as the MIRACLE trial, evaluating Annamycin in combination with cytarabine (Ara-C) for the treatment of acute myeloid leukemia (AML) patients who are refractory to or relapsed after induction therapy (R/R AML). The MIRACLE trial (MB-108) is designed as a global study with sites in the US.
MIRACLE Trial Design
The MIRACLE study will employ an adaptive design. The first 75 to 90 subjects will be randomized in Part A of the trial to receive high-dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin. The doses were specifically recommended by the FDA. Following this initial phase, the trial will be unblinded to select the optimum dose for Annamycin based on safety, pharmacokinetics, and efficacy, aligning with the FDA's Project Optimus initiative. Part B of the trial will involve approximately 240 additional subjects randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin.
Regulatory Status and Company Outlook
Annamycin has been granted Fast Track Status and Orphan Drug Designation by the FDA for the treatment of relapsed or refractory AML, as well as Orphan Drug Designation for soft tissue sarcoma. The European Medicines Agency (EMA) has also granted Annamycin Orphan Drug Designation for relapsed or refractory AML.
Walter Klemp, Chairman and Chief Executive Officer of Moleculin, stated that the company is focused on getting clinical trial sites operational and anticipates commencing enrollment in the first quarter of 2025. He also expressed confidence in potentially bringing a much-needed solution to AML patients, based on positive feedback from clinicians and alignment with the FDA on the MIRACLE study's strategic design.
