Moleculin Advances MIRACLE Phase 3 AML Trial with 78% Enrollment, First Data Readout Expected Q1 2026
Key Insights
Moleculin Biotech has achieved 78% enrollment in its pivotal MIRACLE Phase 2B/3 trial testing Annamycin plus cytarabine for relapsed/refractory acute myeloid leukemia, up from 60% in November.
The company expects to complete treatment of the first 45 patients in Q1 2026, triggering the first interim data unblinding to assess preliminary efficacy and safety.
Annamycin, designed to overcome multidrug resistance and avoid cardiotoxicity of current anthracyclines, holds FDA Fast Track and Orphan Drug designations for AML treatment.
Moleculin Biotech has reported significant progress in its pivotal MIRACLE Phase 2B/3 trial, achieving 78% enrollment for the first interim analysis of Annamycin in combination with cytarabine for treating relapsed or refractory acute myeloid leukemia (R/R AML). The enrollment represents a substantial increase from 60% reported in November 2025, with the company targeting 45 subjects for the first planned interim unblinding expected in the first quarter of 2026.
Trial Design and Progress
The MIRACLE study (Moleculin R/R AML AnnAraC Clinical Evaluation) is a global multi-center, randomized, double-blind, placebo-controlled, adaptive designed clinical trial. The protocol allows for unblinding of preliminary primary efficacy data measuring complete remission (CR) and safety/tolerability across three treatment arms at the 45-subject milestone.
The first early unblinding will include 30 subjects treated with Annamycin at two different dosages (190mg/m² and 230 mg/m²) in combination with high-dose cytarabine (HiDAC (search)), and 15 subjects treated with HiDAC plus placebo as the control arm. Walter Klemp, Chairman and CEO of Moleculin, noted that while the 45-subject data is not designed to hold statistical significance, the company expects at least one of the two Annamycin arms to outperform the control arm.
Geographic Expansion and Enrollment Momentum
The trial has expanded to include consented subjects across seven countries, providing a diverse patient population base. Klemp highlighted the enthusiasm of investigators across Europe and the United States, noting that early enrollment challenges due to bed shortages in some European sites are now improving in certain EU countries.
The company continues to identify additional subjects beyond the 78% milestone while working toward completing Part A of the MIRACLE trial with up to 90 patients within the first half of 2026. The adaptive design allows for continued recruitment of the remaining 45 subjects for the full 90-subject Part A cohort while conducting the first unblinding analysis.
Annamycin's Therapeutic Profile
Annamycin, also known by its non-proprietary name naxtarubicin, represents a next-generation anthracycline designed to address key limitations of current AML treatments. The drug is engineered to avoid multidrug resistance mechanisms and lacks the cardiotoxicity commonly associated with currently prescribed anthracyclines.
The therapeutic candidate has received significant regulatory recognition, including FDA Fast Track Status and Orphan Drug Designation for relapsed or refractory AML treatment, as well as Orphan Drug Designation for soft tissue sarcoma. The European Medicines Agency has also granted Orphan Drug Designation for relapsed or refractory AML treatment.
Regulatory Pathway and Market Protection
Annamycin benefits from composition of matter patent protection through 2040, with potential extension to 2045. The company believes it has substantially de-risked the development pathway toward potential approval for AML treatment following a successful Phase 1B/2 study (MB-106) and input from the FDA.
The adaptive trial design combines data from the Phase 2B (Part A) portion with the Phase 3 (Part B) portion for measuring primary efficacy endpoints. Before each unblinding, trial data will undergo audit, database lock, and review procedures to ensure data integrity.
Clinical Trial Access
The MIRACLE trial is registered on clinicaltrials.gov under identifier NCT06788756, with the European clinical trial listed on euclinicaltrials.eu under reference identifier 2024-518359-47-00. The trial addresses a significant unmet medical need in R/R AML, where patients have limited treatment options after failing initial induction therapy.