Moleculin Biotech accelerates unblinding of Phase 3 efficacy data in AML

Moleculin Biotech (MBRX) has amended the clinical trial protocol with the FDA for its Phase 3 pivotal trial protocol evaluating Annamycin in combination with Cytarabine for the treatment of AML patients who are refractory to or relapsed after induction therapy. The company believes that the unblinding of data from the Phase 3 “MIRACLE” trial at 45 subjects will enable it to begin assessing all three arms of the study and provide a clear path forward in understanding the potential of Annamycin for AML patients. This change puts the company potentially less than 12 months away from definitive unblinded data that could be a strong indicator of the likelihood of approval. The company expects to reach 45 subjects in the second half of 2025, in addition to the planned unblinding expected in 2026 of the next 30-45 subjects. The increase from 240 to 244 subjects represents the statistical “cost” of the additional unblinding. The amended protocol is currently being reviewed by the Institutional Review Board. Once approved, the amended protocol will be filed with the amendment for the company’s Initial New Drug application in the US with the FDA. Annamycin currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory acute myeloid leukemia, in addition to Orphan Drug Designation for the treatment of soft tissue sarcoma.