Moleculin Biotech receives EMA approval to expand Phase 3 MIRACLE trial

Moleculin Biotech (MBRX) announced that the European Medicines Agency has approved its Clinical Trial Application to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine. The study is for the treatment of adult patients with acute myeloid leukemia who are refractory to or relapsed after induction therapy and is approved in all nine countries submitted in the European Union. This Phase 3 “MIRACLE” trial is a global approval trial, including sites in the US, Europe and the Middle East. 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 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. The protocol for the MIRACLE trial allows for the unblinding of preliminary primary efficacy data and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A. The first early unblinding will yield 30 subjects treated with Annamycin and HiDAC and 15 subjects treated with just HiDAC plus placebo. The Company expects to reach the first unblinding in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026. This accelerated estimated timeline is due in part to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial. The clinical trial approval with EMA was granted under the condition that the Company present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion of the study. Results will be submitted as a substantial modification to the existing approved protocol. 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.