Moleculin says MIRACLE trial nears first unblinding with blinded CRc above 40%
Moleculin said its MIRACLE Phase 2B/3 AML trial is nearing first unblinding, with blinded CRc above 40% and CR around 30%. The first 45-subject readout is expected before June 30, 2026.
Moleculin Biotech reported preliminary blinded efficacy from 45 subjects in its pivotal Phase 2B/3 MIRACLE trial of Annamycin plus cytarabine in second-line relapsed/refractory AML. Blinded data show CRc >40% and CR ~30%, versus historical cytarabine CR ~17–18%. First 45-subject unblinding is expected before June 30, 2026.
The company said it is evaluating Annamycin in combination with cytarabine, collectively referred to as AnnAraC, in adult patients with acute myeloid leukemia who are refractory to or relapsed after induction therapy. The MIRACLE trial is a global, adaptive Phase 2B/3 clinical study being conducted across the United States, Europe, and other international sites.
In February 2026, the company reported a blinded preliminary composite complete remission rate of 40% in the MIRACLE trial’s first 30 subjects treated and with blinded, preliminary efficacy data. This CRc rate is comprised of a complete remission rate of 30% and complete remission with partial hematological recovery of 10%. The efficacy rates were observed across six countries.
The company said roughly 35% of the subjects treated to date are relapsed or refractory from a venetoclax regimen. It also said the subjects treated to date presented with a high degree of genetic markers that are considered predictive of poor treatment response.
The protocol allows for early unblinding of data at 45 subjects, enabling an initial readout of approximately 30 subjects treated with Annamycin plus cytarabine and 15 subjects in the control arm receiving cytarabine plus placebo. In Part A, approximately 75 to 90 subjects are randomized in a 1:1:1 ratio to receive high-dose cytarabine combined with either placebo, 190 mg/m² Annamycin, or 230 mg/m² Annamycin. As of May 1, 2026, 56 of 90 Part A subjects had been enrolled, with recruitment of Part A continuing to the target 90 subjects in Q3 2026.
For Part B of the trial, 222 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin, randomized 1:1. The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s Project Optimus initiative.
The company also said Annamycin holds FDA Fast Track and multiple Orphan Drug designations in relapsed or refractory AML. It reported continued absence of cardiotoxicity, with a new independent assessment bringing the total number of Annamycin-treated subjects reviewed by its independent expert to 90.
For the year ended December 31, 2025, the company reported full-year financial results and provided a clinical update highlighting progress toward the upcoming MIRACLE trial milestone.