HOUSTON, Texas — Moleculin Biotech, Inc. (Nasdaq: MBRX) (“Moleculin” or the “Company”) today announced 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.
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.
About Moleculin Biotech, Inc.
Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company advancing a pipeline of therapeutic candidates addressing hard-to-treat tumors and viruses. The Company’s lead program, Annamycin (also known as naxtarubicin), has shown to be an efficacious and well tolerated anthracycline designed to avoid multidrug resistance mechanisms and to lack the cardiotoxicity common with currently prescribed anthracyclines. Annamycin is currently in development for the treatment of relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases.
The Company is conducting the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design, multi-center, randomized, double-blind, placebo-controlled Phase 2/3 trial evaluating Annamycin in combination with cytarabine (also referred to as “Ara-C”), together referred to as AnnAraC, for the treatment of relapsed or refractory acute myeloid leukemia. Following a successful Phase 1B/2 study (MB-106), with input from the FDA, the Company believes it has substantially de-risked the development pathway toward a potential approval for Annamycin for the treatment of AML. This study remains subject to appropriate future filings with potential additional feedback from the FDA and their foreign equivalents.
Additionally, the Company is developing WP1066, an Immune/Transcription Modulator capable of inhibiting p-STAT3 and other oncogenic transcription factors while also stimulating a natural immune response, targeting brain tumors, pancreatic and other cancers. Moleculin also has in its pipeline a portfolio of antimetabolites, including WP1122 for the potential treatment of pathogenic viruses, as well as certain cancer indications.
For more information about the Company, please visit www.moleculin.com and connect on X, LinkedIn and Facebook.
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