On September 29, 2026 Moleculin Biotech, Inc. (Nasdaq: MBRX) ("Moleculin" or the "Company") reported that it has completed and closed enrollment 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), with 89 subjects enrolled and an additional subject currently in screening. With Part A enrollment closed, the Company remains on track for its planned comprehensive data readout of Part A in the first quarter of 2027.
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The rapid pace of enrollment underscores the significant unmet need in R/R AML as well as strong engagement from investigators and clinical sites participating in MIRACLE. The Company expects the expanded Part A dataset to provide important insight into the potential efficacy of AnnAraC across subjects who have relapsed from or are refractory to different first-line (1L) AML treatment approaches, including subjects previously treated with intensive 7+3 chemotherapy and those previously treated with venetoclax-based regimens. Such treatment approaches associated with 1L patient populations are commonly, while not universally, deemed as "fit" and "unfit", respectively.
"Completing enrollment in Part A of MIRACLE is an important milestone for Moleculin and a strong demonstration of the operational execution behind this pivotal program," said Walter Klemp, Chairman and Chief Executive Officer of Moleculin. "We have maintained a rapid pace of enrollment in a global, randomized AML trial and, importantly, remain on track in the first quarter of 2027 for our planned comprehensive data readout – one much deeper in data than our first interim n45 readout. Our focus turns to the maturation and analysis of this substantially larger dataset and what it may tell us about the potential of AnnAraC across distinct and clinically important R/R AML patient populations such as "fit" and "unfit" subjects."
Mr. Klemp continued, "We are particularly excited about the composition of the additional subjects enrolled in Part A and the opportunity that the full Part A population may provide to better understand efficacy based on prior 1L treatment. We estimate that Part A will end with approximately a 50/50 split between "fit" and "unfit" subjects, compared to a roughly 70/30 split, respectively, in the first 45 subjects in Part A. While we are mindful that this shift in composition to more "unfit" subjects represents a more challenging environment for achieving treatment responses, it should provide valuable insight into Annamycin’s ability to fill critical unmet needs. Lastly, we continue to see a lack of cardiotoxicity in MIRACLE’s blinded data."
"Completing Part A enrollment reflects the execution of our team and investigators and moves us directly toward what we believe will be one of the most important milestones in Annamycin’s development," Mr. Klemp added. "We are grateful to the patients and families participating in MIRACLE and to the investigators and clinical teams whose commitment has enabled us to reach this milestone at such a rapid pace. With enrollment complete and the trial on track for a comprehensive Q1 2027 readout, we believe Moleculin is entering a potentially defining period for the MIRACLE program."
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, SEP 29, 2026, View Source [SID1234671152])