Core Viewpoint - Moleculin Biotech 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 (R/R AML), with the first subject expected to be treated in Q1 2025 [1][8]. Company Developments - The FDA has provided feedback that allows Moleculin to reduce the size of Part B of the trial by approximately 10%, while major aspects of the trial remain unchanged [2]. - The trial will be global, with sites in the US, Europe, and the Middle East, facilitating an accelerated timeline for new drug approval [2][3]. - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML and soft tissue sarcoma, as well as Orphan Drug Designation from the European Medicines Agency [5]. Trial Design and Expectations - The MIRACLE trial will utilize 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 [3][4]. - Preliminary primary efficacy data will be unblinded at 45 subjects, with the first unblinding expected in the second half of 2025 and a second unblinding in the first half of 2026 [3]. - Part B of the trial will involve approximately 220 additional subjects randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin [4]. Product Overview - Annamycin is a next-generation anthracycline designed to avoid multidrug resistance mechanisms and eliminate cardiotoxicity associated with current anthracyclines, targeting R/R AML and soft tissue sarcoma [7][8].
Moleculin Receives Positive FDA Guidance for Acceleration of its Registration-Enabling MIRACLE Trial for R/R Acute Myeloid Leukemia (AML)