Moleculin(US:MBRX) Globenewswireยท2025-05-12 12:40Core Viewpoint - Moleculin Biotech, Inc. has received approval from the European Medicines Agency (EMA) for its pivotal Phase 2B/3 clinical trial of Annamycin in combination with cytarabine for treating relapsed or refractory acute myeloid leukemia (R/R AML) [1][2] Group 1: Clinical Trial Details - The "MIRACLE" trial is a multi-center, randomized, double-blind, placebo-controlled study aimed at evaluating Annamycin for R/R AML patients, particularly those who have failed venetoclax regimens [1][2] - The trial has been approved in all nine EU countries submitted, including Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain [2] - Patient enrollment and dosing have commenced, with initial data readout expected in the second half of 2025 [1][5] Group 2: Trial Design and Protocol - The trial utilizes an adaptive design, with the first 75 to 90 subjects randomized to receive high dose cytarabine combined with either placebo or two different doses of Annamycin (190 mg/m and 230 mg/m) [2][3] - The protocol allows for unblinding of preliminary primary efficacy data at 45 subjects, with the first unblinding expected in the second half of 2025 [3] - Part B of the trial will randomize approximately 220 additional subjects to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin [4] Group 3: Regulatory Status and Designation - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML and soft tissue sarcoma [6] - The EMA has also granted Orphan Drug Designation for Annamycin for the treatment of R/R AML [6] Group 4: Company Overview - Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company focused on developing therapeutic candidates for hard-to-treat tumors and viral infections [7] - The lead program, Annamycin, is designed to avoid multidrug resistance mechanisms and lacks the cardiotoxicity associated with current anthracyclines [7][8]