Moleculin Biotech, Inc. has announced an amendment to the clinical trial protocol for its Phase 3 MIRACLE trial, evaluating Annamycin in combination with cytarabine (AnnAraC) for patients with relapsed or refractory acute myeloid leukemia (R/R AML). The amended protocol, agreed upon with the FDA, allows for an accelerated unblinding of preliminary data, potentially expediting the drug's path to approval. The company expects to reach 45 subjects in the second half of 2025.
The MIRACLE trial (MB-108) is a global, adaptive Phase 3 study designed to assess the efficacy and safety of Annamycin in combination with cytarabine in R/R AML patients. The trial's adaptive design initially randomizes the first 75 to 90 subjects to receive high-dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin. The amended protocol allows for unblinding of preliminary primary efficacy data (complete remission rate) and safety/tolerability data after the first 45 subjects are evaluated.
Early Unblinding Details
The early unblinding will provide data from 30 subjects treated with Annamycin (at both 190mg/m2 and 230mg/m2 doses) plus HiDAC and 15 subjects treated with HiDAC alone. Following this initial analysis, approximately 244 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimal dose of Annamycin, selected based on safety, pharmacokinetics, and efficacy, aligning with the FDA's Project Optimus initiative.
Management Commentary
"Our team has been thoughtful and strategic with the design of the MIRACLE trial, which may allow for possible accelerated approval of Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML," said Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "This amended protocol enables us to share definitive data earlier, which helps to partially de-risk financing the trial and potentially accelerates the timeline for strategic partnering."
Annamycin's Regulatory Status
Annamycin has been granted Fast Track Status and Orphan Drug Designation by the FDA for the treatment of R/R AML, as well as Orphan Drug Designation for soft tissue sarcoma. It also holds Orphan Drug Designation from the European Medicines Agency (EMA) for R/R AML.
About Annamycin
Annamycin is a next-generation anthracycline designed to overcome multidrug resistance mechanisms and reduce cardiotoxicity, common limitations of currently used anthracyclines. Moleculin Biotech is also developing WP1066, an Immune/Transcription Modulator, and a portfolio of antimetabolites, including WP1122, for potential treatment of pathogenic viruses and certain cancers.
