Core Viewpoint - Moleculin Biotech, Inc. is advancing its pivotal Phase 3 clinical trial, named the "MIRACLE" trial, for Annamycin in combination with cytarabine for treating relapsed or refractory acute myeloid leukemia (AML), with the first subject expected to be treated in Q1 2025 [1][2]. Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses, with Annamycin as its lead program targeting relapsed or refractory AML and soft tissue sarcoma [4][5]. Clinical Trial Details - The MIRACLE trial has received Institutional Review Board (IRB) approval and will include global sites, with an initial adaptive design involving 75 to 90 subjects randomized to receive high-dose cytarabine combined with either placebo or two different doses of Annamycin [1][2]. - The trial will consist of two parts: Part A will determine the optimum dose of Annamycin, while Part B will randomize approximately 240 additional subjects to receive either high-dose cytarabine plus placebo or the optimum dose of Annamycin [2][5]. 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 (EMA) [3]. Future Development - The company is also developing WP1066, an immune/transcription modulator targeting various cancers, and WP1122, an antimetabolite for potential treatment of pathogenic viruses and certain cancer indications [6].
Moleculin Receives Institutional Review Board Approval for MIRACLE Phase 3 Pivotal Trial of Annamycin in Combination with Cytarabine for the Treatment of R/R Acute Myeloid Leukemia (AML)