Moleculin Biotech receives approval for pivotal Phase 2B/3 study of Annamycin

Moleculin Biotech (MBRX) announced that the Regulation Agency for Medical and Pharmaceutical Activities, or RAMPA, in the country of Georgia has approved its Clinical Trial Application, or CTA, to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine for the treatment of adult patients with acute myeloid leukemia, or AML, who are refractory to or relapsed, or R/R, after induction therapy, or R/R AML. This Phase 3 “MIRACLE” trial is a global approval trial, including sites in the US, Europe and the Middle East. Additionally, the Company expects the first patient to be treated in Georgia to occur by the end of August, if not sooner. The MIRACLE study is a Phase 2B/3 clinical trial whereby data from the 2B portion will be combined with the Phase 3 portion for purposes of measuring its primary efficacy endpoint. MIRACLE is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 75 to 90 subjects will be randomized in Part A of the trial to receive high dose cytarabine, or HiDAC, combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting. For Part B of the trial, approximately 220 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin. The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative. Patient dosing has commenced, and the initial data readout is on track for the second half of 2025. For more information about the MIRACLE trial, visit clinicaltrials.gov and reference identifier NCT06788756. Additionally, the clinical trial in the EU is on euclinicaltrials.eu and the reference identifier there is 2024-518359-47-00.