On October 30, 2026 Moleculin Biotech, Inc. (Nasdaq: MBRX) ("Moleculin" or the "Company") reported that dosing has been completed in Part A of its pivotal Phase 2/3 MIRACLE trial evaluating Annamycin in combination with cytarabine (AnnAraC) for the treatment of adults with relapsed or refractory acute myeloid leukemia (R/R AML). The Company remains on track to report comprehensive Part A data in the first quarter of 2027.
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Reaching this treatment milestone represents continued execution in the MIRACLE trial as the Company advances toward a comprehensive analysis of the full 90-patient Part A population. The planned Q1 2027 data readout is expected to provide important insights into the potential of AnnAraC across clinically distinct R/R AML patient populations and support selection of the optimal Annamycin dose for advancement into Part B of the trial.
The expanded Part A dataset is expected to provide further insight into AnnAraC in patients who have relapsed from or are refractory to different first-line AML treatment approaches, including intensive 7+3 chemotherapy and venetoclax-based regimens. The Company expects the final Part A population to reflect an approximately 50/50 split between these treatment groups, compared with an approximately 70/30 split, respectively, among the first 45 subjects. This broader representation is expected to help characterize AnnAraC’s potential across patient populations with differing treatment histories and clinical challenges.
"Reaching the completion of dosing in Part A represents another important step toward our planned Q1 2027 comprehensive n=90 data readout," said Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "Combined with the continued absence of cardiotoxicity in blinded interim data, we believe our Q1 2027 readout positions us to select our Part B dose and to define Annamycin’s potential role across today’s R/R AML landscape."
About the MIRACLE Trial
MIRACLE is a pivotal Phase 2/3 study of Annamycin in combination with cytarabine for the treatment of adult subjects with AML who are refractory to, or have relapsed (R/R) after, induction therapy. Part A of the trial is a randomized, double-blind portion of the study that compares two different doses of Annamycin plus cytarabine to a control arm of cytarabine plus placebo, all with just one cycle of therapy.
Importantly, as specified by the MIRACLE protocol, remission rates for all three arms, including the control arm, reflect outcomes measured after only a single cycle of therapy. The most commonly cited historical benchmarks in this setting, including the MIRROS and CLASSIC I studies, as well as Moleculin’s own MB-106 study, permitted multiple cycles of treatment. The Company therefore expects absolute remission rates for both the control and Annamycin arms to be lower than those reported in such multi-cycle datasets and believes the most meaningful comparison is the performance of the Annamycin arms relative to the concurrent, randomized control arm evaluated on the same single-cycle basis.
The comprehensive Part A analysis is expected to evaluate efficacy and safety across the complete randomized population and support selection of the optimal Annamycin dose for advancement into Part B of MIRACLE.
For additional information on the MIRACLE trial, please visit ClinicalTrials.gov and reference Identifier NCT06788756.
(Press release, Moleculin, OCT 30, 2026, View Source [SID1234674003])