MIRACLE: Pivotal, Adaptive Phase 3 Trial in Acute Myeloid Leukemia

The MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial is a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine for the treatment of relapsed or refractory acute myeloid leukemia (AML). Following a successful Phase 1B/2 study (MB-106), with input from the FDA, we believe we have substantially de-risked the development pathway towards approval for annamycin for the treatment of AML.

The MIRACLE Trial is expected to utilize an adaptive design whereby the first 75 patients will be randomized to receive HiDAC combined with either placebo, 190 mg/m² of Annamycin, or 230 mg/m² of Annamycin. At that point, the trial will be unblinded to select the optimum dose for Annamycin. For the second half of the trial, approximately 120 patients 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 not only on the absence of dose limiting toxicities but also on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

We Believe the Bar for Success is Low

NOTES: 1 – Mirros Trial, 81% 2nd line patients, Konopleva et al, Blood Advances, 26 July 2022, Volume 6, Number 14; 2 – Classic I Trial, Faderl et al, J Clin Oncol, July 2012, Volume 30, Number 20; 3 – MB-106 trial, 50% CR rate for 2nd line patients (n=10), 43% CR rate for 2nd + 3rd line patients  (n=14), and 36% CR rate for all-comers (1st through 7th line, n=22)

The MIRACLE trial is subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents. Annamycin currently has Fast Track Status and Orphan Drug Designation from the U.S. Food and Drug Administration for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory acute myeloid leukemia from the European Medicines Agency (EMA). For more information about the MB-106 Phase 1B/2 trial, visit clinicaltrialsregister.eu and reference EudraCT 2020-005493-10 or clinicaltrials.gov and reference NCT05319587.