Moleculin to Highlight its Ongoing Phase 3 Acute Myeloid Leukemia “MIRACLE” Clinical Trial at the 14th Annual Acute Leukemia Meeting

Rhea-AI Summary

Moleculin (Nasdaq: MBRX) will present at the 14th Annual Acute Leukemia Meeting in Madrid on October 30-31, 2025. Dr. Paul Waymack, senior chief medical officer, will speak on Thursday, October 30 at 2:35 PM CEST about L-Annamycin (naxtarubicin) and the ongoing pivotal Phase 2B/3 “MIRACLE” trial.

The MIRACLE study is a multi-center, randomized, double-blind, placebo-controlled, adaptive design trial evaluating Annamycin combined with cytarabine (AnnAraC) in adult patients with relapsed or refractory acute myeloid leukemia (R/R AML). It is a global approval trial with sites in the US, Europe and the Middle East (clinicaltrials.gov NCT06788756; euclinicaltrials.eu 2024-518359-47-00). Annamycin currently holds FDA Fast Track status and Orphan Drug designations from FDA and EMA for R/R AML.

Insights

Clinical Development Strategist

Company will present ongoing Phase 2B/3 global MIRACLE trial details at a conference; no outcome data disclosed.

The presentation highlights Moleculin progress with Annamycin (naxtarubicin) in a randomized, double-blind, placebo-controlled Phase 2B/3 study for R/R AML, indicating the program remains on the regulatory path toward approval. The trial is described as a global approval study with sites in the US, Europe, and the Middle East and is registered as NCT06788756 and 2024-518359-47-00, which supports its scale and formal regulatory orientation.

The near-term value of this news lies in increased visibility and scientific engagement rather than new efficacy or safety data; the release contains no endpoints, interim results, or enrollment updates. Key dependencies include eventual disclosure of prespecified efficacy and safety readouts and any adaptive-design decision points; until those appear, impact on approval probability or commercial value remains indeterminate. Watch for presentation content from October 30, 2025 at 2:35 PM CEST, any announced interim analyses, enrollment milestones, or protocol amendments within the trial registry entries over the next 6–18 months.

HOUSTON, Oct. 30, 2025 (GLOBE NEWSWIRE) -- Moleculin Biotech, Inc., (Nasdaq: MBRX) (“Moleculin” or the “Company”), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat cancers and viral infections, today announced it will present at the 14^th Annual Acute Leukemia Meeting being held October 30-31, 2025 at the MD Anderson Cancer Center Spain Foundation in Madrid, Spain.

Details of the presentation are as follows:

Title: L-Annamycin - Non-Cardiotoxic Anthracycline; MIRACLE Pivotal AML Study
Session: Lunch Session: Moleculin
Presenter: Dr. Paul Waymack, Senior Chief Medical Officer of Moleculin
Date and Time: Thursday, October 30^th at 2:35 PM CEST

As part of the presentation, Dr. Waymack will discuss the Company’s ongoing pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine (also known as “Ara-C” and for which the combination of Annamycin and Ara-C is referred to as “AnnAraC”) for the treatment of adult patients with acute myeloid leukemia (AML) who are refractory to or relapsed (R/R) after induction therapy (R/R AML). This Phase 3 “MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is a global approval trial, including sites in the US, Europe and the Middle East (clinicaltrials.gov NCT06788756; euclinicaltrials.eu 2024-518359-47-00).

Annamycin, also known by its non-proprietary name of naxtarubicin, currently has Fast Track Status and Orphan Drug Designation from the FDA for the treatment of relapsed or refractory AML, in addition to Orphan Drug Designation for the treatment of STS lung mets. Furthermore, Annamycin has Orphan Drug Designation for the treatment of relapsed or refractory AML from the EMA.

For more information about the 14^th Annual Acute Leukemia Meeting, please visit the meeting website.

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, is a next-generation highly 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 has begun the MIRACLE (Moleculin R/R AML AnnAraC Clinical Evaluation) Trial (MB-108), a pivotal, adaptive design Phase 3 trial evaluating Annamycin in combination with cytarabine, 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 towards 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. 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. 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

When is Moleculin (MBRX) presenting at the 14th Annual Acute Leukemia Meeting?

Moleculin will present on Thursday, October 30, 2025 at 2:35 PM CEST.

What will Moleculin (MBRX) present about at the October 30, 2025 meeting?

Dr. Paul Waymack will present on L-Annamycin (naxtarubicin) and the pivotal Phase 2B/3 MIRACLE trial of AnnAraC in R/R AML.

What is the design of the MIRACLE Phase 3 trial for Moleculin (MBRX)?

The MIRACLE trial is a multi-center, randomized, double-blind, placebo-controlled, adaptive Phase 2B/3 study.

Which patients are eligible for the MIRACLE (MBRX) Annamycin trial?

The trial enrolls adult patients with relapsed or refractory acute myeloid leukemia (R/R AML) after induction therapy.

Where is the MIRACLE trial for Moleculin (MBRX) being conducted and what are the trial IDs?

It is a global trial with sites in the US, Europe and the Middle East: clinicaltrials.gov NCT06788756 and euclinicaltrials.eu 2024-518359-47-00.

What regulatory designations does Annamycin (MBRX) have for R/R AML?

Annamycin has FDA Fast Track status and Orphan Drug designations from both the FDA and EMA for relapsed or refractory AML.