Moleculin Doses First EU Patient in Phase 3 Pivotal MIRACLE Trial for Treatment of Acute Myeloid Leukemia

Core Insights - Moleculin Biotech, Inc. has initiated the pivotal Phase 2B/3 MIRACLE trial for Annamycin in combination with cytarabine for treating relapsed or refractory acute myeloid leukemia (R/R AML) [1][10] - The trial aims to recruit 45 subjects by the end of 2025 for initial data unblinding, with the first two subjects already enrolled in Spain [2][3] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML and soft tissue sarcoma [7] Trial Details - The MIRACLE trial is a multi-center, randomized, double-blind, placebo-controlled study designed to evaluate the efficacy and safety of Annamycin combined with cytarabine [1][10] - The trial will consist of two parts: Part A will involve 75 to 90 subjects, while Part B will randomize approximately 220 additional subjects [5][10] - The first unblinding of preliminary efficacy data is expected in the second half of 2025, with a second unblinding anticipated in the first half of 2026 [3][6] Regulatory and Design Aspects - The trial is subject to future filings with the FDA and EMA, with the EMA requiring nonclinical GLP study results before initiating Part B [4][10] - The adaptive design allows for the unblinding of data at 45 subjects, focusing on Complete Remission (CR) and safety/tolerability across three treatment arms [3][5] Company Overview - Moleculin Biotech is a late-stage pharmaceutical company focused on developing therapies for hard-to-treat cancers and viral infections [9] - The lead program, Annamycin, is designed to avoid multidrug resistance and cardiotoxicity associated with traditional anthracyclines [9][10] - The company is also developing additional therapeutic candidates, including WP1066 and WP1122, targeting various cancers and viral infections [11]