Moleculin Completes Dosing Patients in Part A of Pivotal MIRACLE Trial; Comprehensive Data Readout on Track for Q1 2027

The planned Q1 2027 efficacy and safety analysis is expected to support Annamycin dose selection for Part B.

Rhea-AI Summary

Moleculin Biotech (Nasdaq: MBRX) completed dosing in Part A of its pivotal Phase 2/3 MIRACLE trial in adults with relapsed or refractory leukemia. The randomized, double-blind study evaluates Annamycin plus cytarabine for acute myeloid leukemia (AML). Part A includes 90 patients and compares two Annamycin doses with cytarabine plus placebo.

Moleculin expects a comprehensive efficacy and safety readout in Q1 2027 to support dose selection for Part B. Blinded interim data continued to show no cardiotoxicity, or heart damage. The company expects an approximately 50/50 split between patients who failed intensive 7+3 chemotherapy and venetoclax-based regimens, versus approximately 70/30 among the first 45 subjects.

Remission outcomes cover only one treatment cycle. Moleculin expects lower absolute remission rates than historical studies allowing multiple cycles and considers the concurrent randomized control the most meaningful comparison.

3 points · 0 major

PositiveiHow this balance works

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Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.

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Rhea-AI Sentiment measures something else, the tone of the wording.

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0 major · 1 point

Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate pointPart A dosing completed across the 90-patient randomized, double-blind MIRACLE population.
• Moderate point. Forward-looking: it has not happened yet and may not happen.Q1 2027 comprehensive readout remains on track; Moleculin expects it to support Part B dose selection.
• Minor pointNo cardiotoxicity continued to be observed in blinded interim data.

Negative

• Minor point. Forward-looking: it has not happened yet and may not happen.Single-cycle remission assessment limits historical comparisons; Moleculin expects lower absolute rates than multi-cycle datasets.

Key Figures

Part A population: 90 patients Data readout timing: Q1 2027 Treatment-group representation: Approximately 50/50 vs. approximately 70/30

Part A population

90 patients

Full randomized MIRACLE Part A population

Data readout timing

Q1 2027

Comprehensive Part A data readout remains on track

Treatment-group representation

Approximately 50/50 vs. approximately 70/30

Final Part A population versus the first 45 subjects; 7+3 chemotherapy and venetoclax-based regimens, respectively

Historical Context

2 past events · Latest: Oct 20

2 events

1. Oct 20

   Trial enrollment update

   24h Move

   +16.8%

   Reported completed Part A enrollment and maintained the Q1 2027 full-population readout timeline.

2. Sep 29

   Trial enrollment update

   24h Move

   -7.5%

   Enrollment closed at 89 subjects, with one additional subject in screening before final dosing.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

aml, cardiotoxicity, double-blind, placebo-controlled

4 terms

aml medical

"adults with relapsed or refractory acute myeloid leukemia (R/R AML)"

AML stands for anti-money laundering — the laws, rules and internal checks that banks and businesses use to spot and stop illicit cash flows, such as proceeds from crime or funding of illegal activities. Think of it as a security checkpoint for money: investors care because poor AML controls can lead to heavy fines, frozen assets and reputational harm that hurt profits and share value, while strong controls reduce legal and operational risk.

cardiotoxicity medical

"continued absence of cardiotoxicity in blinded interim data"

Cardiotoxicity is damage to the heart caused by a drug, chemical or medical treatment that can weaken heart function, disrupt heartbeat or cause inflammation. It matters to investors because evidence of cardiotoxicity can halt or delay product approvals, trigger costly additional testing, recalls or legal risk, and reduce future revenue potential—similar to how rust in an engine can undermine a machine’s reliability and resale value.

double-blind technical

"Part A of the trial is a randomized, double-blind portion"

A double-blind process means that neither the people conducting an activity nor the people involved know certain key details, such as who is receiving a treatment or a placebo. This approach helps prevent bias from influencing the results, making the outcome more trustworthy. For investors, it ensures that decisions or judgments are based on unbiased information rather than preconceived opinions or expectations.

placebo-controlled technical

"multi-center, randomized, double-blind, placebo-controlled Phase 2/3 trial"

"Placebo-controlled" describes a testing method where one group receives the actual treatment or intervention, while another group receives a harmless, inactive version called a placebo. This approach helps determine whether the real treatment has genuine effects beyond psychological expectations. For investors, understanding this ensures confidence that reported benefits are real and not influenced by bias or false perceptions.

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Milestone underscores execution of pivotal Phase 2/3 trial evaluating Annamycin in combination with cytarabine for relapsed or refractory acute myeloid leukemia (AML)

Fully treated, randomized, double-blind n=90 Part A population sets up Annamycin dose selection for Part B, with balanced representation of AML patients who failed 7+3 chemotherapy and venetoclax-based regimens

HOUSTON, Oct. 30, 2026 (GLOBE NEWSWIRE) -- 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.

Forward-Looking Statements

Some of the statements in this release are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, Section 21E of the Securities Exchange Act of 1934 and the Private Securities Litigation Reform Act of 1995, which involve risks and uncertainties. Forward-looking statements in this press release include, without limitation, the potential efficacy and safety of Annamycin and AnnAraC in R/R AML, the continued absence of cardiotoxicity based on blinded interim data, expected timing of the comprehensive Part A data readout in Q1 2027, the anticipated composition of the Part A population including the expected approximate 50/50 split between patients who failed intensive 7+3 chemotherapy and those who failed venetoclax-based regimens, the potential for Part A data to support dose selection for Part B, and the potential for Part A data to support dose selection for Part B. Moleculin will require significant additional financing, for which the Company has no commitments, in order to conduct its clinical trials as described in this press release, and the milestones described in this press release assume the Company’s ability to secure such financing on a timely basis. Although Moleculin believes that the expectations reflected in such forward-looking statements are reasonable as of the date made, expectations may prove to have been materially different from the results expressed or implied by such forward-looking statements. The Company relies on the reports of its expert with regard to the absence of cardiotoxicity. The dataset referenced in this press release is subject to the review of the data from future subjects in its current and future clinical trials and long-term follow-up with subjects in its current trials. Moleculin has attempted to identify forward-looking statements by terminology including ‘believes,’ ‘estimates,’ ‘anticipates,’ ‘expects,’ ‘plans,’ ‘projects,’ ‘intends,’ ‘potential,’ ‘may,’ ‘could,’ ‘might,’ ‘will,’ ‘should,’ ‘approximately’ or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements are only predictions and involve known and unknown risks, uncertainties, and other factors, including those discussed under Item 1A. “Risk Factors” in our most recently filed Form 10-K filed with the Securities and Exchange Commission (SEC) and updated from time to time in our Form 10-Q filings and in our other public filings with the SEC. Any forward-looking statements contained in this release speak only as of its date. We undertake no obligation to update any forward-looking statements contained in this release to reflect events or circumstances occurring after its date or to reflect the occurrence of unanticipated events.

Investor Contact:
JTC Team, LLC
Jenene Thomas
(908) 824-0775
MBRX@jtcir.com

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

When will Moleculin report comprehensive MIRACLE Part A results?

Moleculin remains on track to report comprehensive Part A results in Q1 2027. The planned analysis covers efficacy and safety across the complete 90-patient randomized population and is expected to support selection of the Annamycin dose for Part B.

What did Moleculin complete in Part A of the MIRACLE trial?

Moleculin completed dosing in Part A, which includes 90 patients. This randomized, double-blind portion compares two doses of Annamycin combined with cytarabine against cytarabine plus placebo in adults with relapsed or refractory acute myeloid leukemia.