Moleculin Biotech has received Institutional Review Board (IRB) approval to proceed with its Phase 3 pivotal trial, known as the MIRACLE trial, evaluating Annamycin in combination with cytarabine (Ara-C) for patients with relapsed or refractory acute myeloid leukemia (R/R AML). The study, titled "Moleculin R/R AML AnnAraC Clinical Evaluation," is designed as a global trial with sites in the US, and aims for potential accelerated approval of Annamycin.
Walter Klemp, Chairman and CEO of Moleculin, stated, "IRB approval marks an important milestone towards the launch of our MIRACLE pivotal Phase 3 trial of Annamycin in AML patients. Our team is focused on getting clinical trial sites up and running, and we believe we will be in a position to commence enrollment in the first quarter of 2025."
Trial Design
The MIRACLE trial will employ an adaptive design. The initial phase (Part A) will randomize 75 to 90 subjects 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 during the end of Phase 1B/2 meeting. Following unblinding, the optimal Annamycin dose will be selected 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 optimized dose of Annamycin.
Regulatory Status and Background
Annamycin has been granted Fast Track Status and Orphan Drug Designation by the FDA for R/R AML, along with Orphan Drug Designation for soft tissue sarcoma. It also holds Orphan Drug Designation from the European Medicines Agency (EMA) for R/R AML. Annamycin is a next-generation anthracycline designed to overcome multidrug resistance mechanisms and reduce cardiotoxicity associated with traditional anthracyclines.
About Moleculin Biotech
Moleculin Biotech, Inc. is a Phase 3 clinical-stage pharmaceutical company focused on developing therapeutic candidates for hard-to-treat tumors and viruses. In addition to Annamycin, Moleculin is developing WP1066, an Immune/Transcription Modulator targeting brain tumors, pancreatic cancer, and other cancers, and a portfolio of antimetabolites, including WP1122, for potential treatment of pathogenic viruses and cancer indications.
