Moleculin Accelerates Outlook Into 2026 With First Pivotal Trial Unblinding on Track, Global Trial Expansion, and Multiple Externally/IIT Funded Clinical Programs

Rhea-AI Summary

Moleculin (Nasdaq: MBRX) outlined 2026–2028 clinical milestones for its anthracycline Annamycin and STAT3 inhibitor WP1066. The pivotal Phase 2B/3 MIRACLE trial for relapsed/refractory AML has expanded to nine countries with >46 sites selected and ~20 sites contracted, and is on track to treat the 45th Part A subject in Q1 2026 with an unblinding shortly thereafter and a second unblinding in Q3 2026. Part A targets 90 subjects; Part B will add 222 subjects. Institutional/externally funded trials include a 2026 Atlantic Health pancreatic trial and investigator‑initiated WP1066 trials at Northwestern and Emory. Primary MIRACLE efficacy data and a rolling NDA are targeted in 2028.

Positive

• MIRACLE trial expanded to 9 countries
• Over 46 sites selected with ~20 contracted
• On track to treat the 45th subject in Q1 2026
• Institutional funding secured for external IIT trials
• WP1066 external Phase 2 GBM trial actively recruiting

Negative

• Primary MIRACLE efficacy data not expected until 2028
• Part B recruitment and key milestones extend into 2027
• Early unblinding yields only ~30 Annamycin vs ~15 control subjects

News Market Reaction

+1.76%

13 alerts

+1.76% News Effect

+9.8% Peak in 28 hr 31 min

+$214K Valuation Impact

$12M Market Cap

0.4x Rel. Volume

On the day this news was published, MBRX gained 1.76%, reflecting a mild positive market reaction. Argus tracked a peak move of +9.8% during that session. Our momentum scanner triggered 13 alerts that day, indicating notable trading interest and price volatility. This price movement added approximately $214K to the company's valuation, bringing the market cap to $12M at that time.

Data tracked by StockTitan Argus on the day of publication.

Key Figures

MIRACLE unblinding 1: Q1 2026 Countries in MIRACLE: 9 countries Sites selected: Over 46 sites +5 more

8 metrics

MIRACLE unblinding 1 Q1 2026 First unblinding of 45 subjects in pivotal Phase 2B/3 MIRACLE AML trial

Countries in MIRACLE 9 countries Global MIRACLE AML trial footprint across US and Europe

Sites selected Over 46 sites Sites selected for MIRACLE AML trial; about 20 contracted

First-part subjects 45 subjects Part A MIRACLE AML subjects for first unblinding in Q1 2026

Total Part A subjects 90 subjects Planned total for Part A before second unblinding

Part B randomization 222 subjects Additional subjects randomized 1:1 to HiDAC±Annamycin in Part B

Annamycin dose level 1 190 mg/m2 Dose arm in Part A MIRACLE trial as per FDA recommendation

Annamycin dose level 2 230 mg/m2 Higher dose arm in Part A MIRACLE trial as per FDA recommendation

Market Reality Check

Price: $4.16 Vol: Pre‑news volume of 113,69...

low vol

$4.16 Last Close

Volume Pre‑news volume of 113,695 shares compares to a 20‑day average of 465,057, indicating subdued trading. low

Technical Price at $3.98 is trading below the $14.29 200‑day moving average, reflecting a weak longer‑term trend.

Peers on Argus

MBRX was down 3.16% pre‑news while biotech peers showed mixed moves: KZIA and LP...

MBRX was down 3.16% pre‑news while biotech peers showed mixed moves: KZIA and LPTX were positive, whereas INTS, MBIO, and SNGX declined. The cross‑currents point to stock‑specific factors rather than a clear sector‑wide move.

Historical Context

5 past events · Latest: Dec 17 (Positive)

Pattern 5 events

Date	Event	Sentiment	Move	Catalyst
Dec 17	Clinical trial update	Positive	-11.7%	Positive Phase 1 WP1066 pediatric brain tumor data with STAT3 suppression.
Dec 10	Financing / warrants	Negative	-27.1%	Immediate warrant exercises raising cash and issuing new unregistered warrants.
Dec 09	Clinical trial progress	Positive	-8.6%	MIRACLE trial enrollment and treatment milestones toward first 45 subjects.
Dec 08	Research collaboration	Positive	+0.1%	New Annamycin GBM collaboration and preclinical intra‑arterial delivery work.
Nov 26	Investor event	Neutral	-22.0%	Announcement of participation in a virtual investor series and corporate overview.

Pattern Detected

Recent history shows several instances where positive clinical or collaboration news coincided with negative price reactions, suggesting a pattern of skepticism or financing/listing overhang.

Recent Company History

Over the past few months, Moleculin has highlighted multiple clinical and corporate milestones. Positive pediatric brain tumor data for WP1066 on Dec 17, 2025 and MIRACLE enrollment progress on Dec 9, 2025 both saw negative next‑day moves. A new Annamycin GBM collaboration on Dec 8, 2025 had little impact, while participation in an investor event on Nov 26, 2025 coincided with a sharp decline. A December warrant exercise for roughly $6.5M gross proceeds also preceded a significant drop, underscoring financing and balance‑sheet concerns alongside the development pipeline.

Regulatory & Risk Context

Active S-3 Shelf

Shelf Active

Active S-3 Shelf Registration 2025-09-19

An effective shelf registration on Form S-3 dated 2025-09-19 remains in place through 2028-09-19. It has 0 recorded usages so far, indicating capacity for future registered offerings if the company chooses to access it.

Market Pulse Summary

This announcement outlines a dense clinical timeline, with MIRACLE AML interim unblindings in Q1 and...

Analysis

This announcement outlines a dense clinical timeline, with MIRACLE AML interim unblindings in Q1 and Q3 2026, Part B expansion to 222 subjects, and a rolling NDA goal in 2028. It also highlights externally funded trials in pancreatic cancer and brain tumors for Annamycin and WP1066. Investors may weigh these catalysts against prior filings noting losses and going‑concern language, while an active Form S-3 through 2028-09-19 provides flexibility for potential future capital raises.

Key Terms

phase 2b/3, acute myeloid leukemia, stat3, c-myc, +4 more

8 terms

phase 2b/3 medical

"pivotal Phase 2B/3 MIRACLE trial treating second line (2L) acute myeloid"

A phase 2b/3 trial is a combined late-stage clinical study that first refines the best dose and measures how well a treatment works (phase 2b) then expands to a larger, definitive test of safety and effectiveness needed for regulatory approval (phase 3). For investors, results from a phase 2b/3 act like a dress rehearsal that turns into opening night: positive, well-controlled outcomes substantially raise the chance of approval and future sales, while failures can sharply reduce a drug’s value.

acute myeloid leukemia medical

"MIRACLE trial treating second line (2L) acute myeloid leukemia (AML)"

A fast‑moving blood cancer that starts in the bone marrow and crowd out healthy blood cell production, leaving the body short of normal red cells, white cells and platelets. It matters to investors because the disease creates urgent medical need, drives demand for new diagnostics and treatments, and so clinical trial results, regulatory decisions and drug pricing can rapidly change the commercial prospects and valuation of companies working on therapies.

stat3 medical

"including p-STAT3 (phosphorylated signal transducer and activator of transcription 3)"

STAT3 is a protein inside cells that acts like a control knob for turning sets of genes on or off, influencing cell growth, survival and immune responses. For investors, STAT3 matters because drugs or tests that block or measure its activity can be used to treat or track cancers and inflammatory diseases, so progress on STAT3-targeted therapies or diagnostics can affect the value and prospects of biotech and pharmaceutical firms.

c-myc medical

"including p-STAT3 ..., c-Myc (oncogene driving many aggressive cancers)"

c-myc is a gene that makes a protein acting like a cell’s “gas pedal,” controlling how fast cells grow, divide, and use energy. Because abnormal c-myc activity is common in many cancers, it is a key target and biomarker in oncology drug development; changes in c-myc can influence whether a therapy works, the size of potential patient populations, and regulatory or commercial prospects for treatments.

hif-1α medical

"and HIF-1α (hypoxia-inducible factor 1α). These transcription factors are widely"

HIF-1α is a protein that acts like a cellular oxygen sensor, switching on genes that help cells survive when oxygen is low. For investors, it matters because drugs or diagnostics that target HIF-1α can change disease outcomes in areas like cancer, heart and vascular disease, and is often a focus of clinical trials and regulatory review; success or failure can materially affect a biotech company's value.

orphan drug regulatory

"Annamycin holds FDA Fast Track status and multiple Orphan Drug designations"

A drug designated for an orphan disease is a medicine developed to treat a rare condition that affects only a small number of people. Regulators often give these drugs special incentives—such as reduced costs, faster review, and temporary exclusive selling rights—to encourage development, which matters to investors because those incentives can make a small market financially viable and reduce competition, much like a temporary patent on a niche product.

fast track regulatory

"Annamycin holds FDA Fast Track status and multiple Orphan Drug designations"

A fast track designation is a regulatory label that speeds up the review and communication between a drug developer and regulators for treatments addressing serious illnesses or unmet medical needs. For investors, it matters because it can shorten development time and reduce regulatory delays—like getting a VIP lane at the airport—raising the chance of earlier market access and potential revenue, though it does not guarantee approval.

nda regulatory

"2028: Rolling NDA begins"

An NDA, or nondisclosure agreement, is a legal contract that keeps certain information private between parties. It’s like a promise not to share sensitive details, helping protect business ideas, strategies, or data from being leaked or used without permission. For investors, NDAs help ensure that confidential information remains secure, enabling trust and open communication during business discussions.

AI-generated analysis. Not financial advice.

– First MIRACLE trial unblinding expected Q1 2026; Global trial now spans nine countries

– Investigator-Initiated (IIT) funded pancreatic and brain cancer trials

HOUSTON, Jan. 12, 2026 (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 tumors and viruses, today provided a business outlook and outlined expected upcoming milestones for its next generation non-cardiotoxic anthracycline Annamycin (also known by its generic name “naxtarubicin”) and its STAT3 inhibitor WP1066.

Annamycin

“Annamycin is set for a number of milestones in 2026 including two data readouts in its pivotal Phase 2B/3 MIRACLE trial treating second line (2L) acute myeloid leukemia (AML) plus embarking in its first clinical trial for the treatment of pancreatic cancer,” commented Walter Klemp, Chairman and CEO of Moleculin. “The two MIRACLE data readouts will set the course for the pivotal trial in two ways – potentially demonstrating the efficacy of the Annamycin arms versus the control arm and setting the dose for the Annamycin arm to be used in Part B of the MIRACLE trial.”

“We have already demonstrated initial efficacy in 2L AML and in soft tissue sarcoma metastases in previous trials so planning an expansion into pancreatic cancer will continue adding to the Annamycin story as a ‘next generation anthracycline.’ Atlantic Health previously indicated their intent to fund and conduct an investigator-initiated trial with Annamycin treating third line pancreatic subjects, building on pre-clinical data where Annamycin demonstrated surprising activity in pancreatic cancer models. This trial should start in 2026. We view this kind of institutional funding of clinical research to be a strong independent endorsement of the potential for Annamycin.”

“Importantly, we believe Annamycin has the potential to become the world’s first ever non-cardiotoxic anthracycline and we are expecting more data supporting its lack of cardiotoxicity to be presented early this year. Considering that nearly half of all cancers and 60% of all childhood cancers are currently treated with cardiotoxic anthracyclines that are known to result in permanent heart damage, we think that makes the market opportunity for Annamycin potentially enormous.”

The Company is advancing the development 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”) with a Phase 3 pivotal trial for the treatment of AML subjects who are refractory to or relapsed after first line induction therapy (R/R AML). This Phase 3 “MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation) is global, including sites in the US, the European Union and Eastern Europe. The trial has expanded to nine countries – the US, Spain, Italy, Poland, Czechia, Romania, Ukraine, Lithuania and Georgia with over 46 sites selected, about 20 sites contracted with more coming online weekly. The Company is on track to treat the 45^th subject in Part A of the MIRACLE trial in the first quarter of 2026.

Expected Milestones for Annamycin Development Program

• Q1 26: Update on non-cardiotoxicity review of subject data by the Company’s independent expert
• Q1 26: MIRACLE - 45th subject treated with unblinding of data for 45 subjects shortly thereafter
• 1H 26: MIRACLE - 90th subject recruited
• Q3 26: MIRACLE - 90 subjects unblinded
• 2H 26: MIRACLE - Start of Part B
• 2H 26: Atlantic Health pancreatic cancer clinical trial begins
• 2027: Begin recruitment of 3^rd line AML subjects
• 2027: End recruitment of Part B
• 2027: Begin pediatric AML clinical study
• 2028: Primary efficacy data for MIRACLE
• 2028: Rolling NDA begins
  
  

WP1066

WP1066 is Moleculin’s flagship Immune/Transcription Modulator designed to stimulate the immune response to tumors by inhibiting the errant activity of regulatory T cells while also inhibiting key oncogenic transcription factors, including p-STAT3 (phosphorylated signal transducer and activator of transcription 3), c-Myc (oncogene driving many aggressive cancers) and HIF-1α (hypoxia-inducible factor 1α). These transcription factors are widely sought targets because of their role in cancer cell survival and proliferation, angiogenesis (coopting vasculature for blood supply), invasion, metastasis, and inflammation associated with tumors.

With Moleculin’s WP1066 oral formulation, an externally funded Phase 2 trial in combination with radiation treating GBM, a form of brain cancer, is recruiting and treating subjects at Northwestern University (Northwestern).

Additionally with the oral formula, Emory University funded and conducted a Phase 1 clinical trial at the Aflac Cancer and Blood Disorders Center at Children’s Healthcare of Atlanta. Positive results of this trial were recently published. Based on the encouraging results of this study, the Emory team has indicated that they would like to conduct a follow-on trial. Further at Emory, the Company signed an agreement with the institution whereby Emory will study various WP1066 intravenous (IV) formulations in preclinical studies with the goal of selecting the best formulation to move into a clinical setting focused, most likely, on brain cancers such as GBM.

Mr. Klemp commented “We are extremely grateful to these two great institutions for their willingness to fund further investigator-initiated clinical activity with WP1066, which has been the focus of over 55 peer reviewed research papers. Despite its importance, STAT3 has been called an ‘undruggable’ target. We believe WP1066 has the potential to solve this issue. Looking forward to 2026, we anticipate the current trial at Northwestern to continue treating subjects and for a second Emory trial to begin in 2026.”

Expected Milestones for WP1066 Development Program

• Q1 2026: Phase 2 GBM at Northwestern continues
• 2H 2026: Phase 2 pediatric trial brain tumor begins at Emory
• 2H 2026: Preclinical data on WP1066 IV formulation
  
  

About the MIRACLE Study

The MIRACLE study, as all clinical trials, subject to appropriate future filings with and potential additional feedback from the FDA and their foreign equivalents, utilizes an adaptive design whereby the first 90 subjects will be randomized (1:1:1) in Part A of the trial to receive high dose cytarabine (HiDAC) combined with either placebo, 190 mg/m2 of Annamycin, or 230 mg/m2 of Annamycin, which Annamycin doses were specifically recommended by the FDA in the Company’s end of Phase 1B/2 meeting. The amended protocol allows for the unblinding of preliminary primary efficacy data (CR) and safety/tolerability of the three arms at 45 subjects, in addition to the conclusion of Part A (90 total subjects). This early unblinding will yield roughly 30 subjects with Annamycin (190mg/m2 and 230/m2) and HiDAC and about 15 subjects with just HiDAC. The Company expects to reach the treating the first 45 subjects in Q1 2026 with the first unblinding shortly thereafter, in addition to the second unblinding, which is expected in Q3 2026. This accelerated estimated timeline is due to the positive response the Company received in meetings during December with potential investigators regarding recruitment for the trial.

For Part B of the trial, 222 additional subjects will be randomized to receive either HiDAC plus placebo or HiDAC plus the optimum dose of Annamycin (randomized 1:1). The selection of the optimum dose will be based on the overall balance of safety, pharmacokinetics and efficacy, consistent with the FDA’s new Project Optimus initiative.

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 (the combination of Annamycin and cytarabine, also referred to as “Ara-C”) and, 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.

In investigator-initiated clinical and preclinical activity, the cost to the Company is generally drugs costs and ancillary services outside of the investigator’s institution. All other costs are generally covered by the investigator and/or their institution via grant funds that are not reflected in the Company’s financial statements.

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, statements regarding the continued recruitment, treatment, and receipt of the unblinded data for the first 45 subjects of the MIRACLE clinical trial as described and the matters set forth in the sections entitled “Expected Milestones for Annamycin Development Program” and “Expected Milestones for WP1066 Development Program”. 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 will Moleculin (MBRX) unblind initial MIRACLE trial data?

Q1 2026 unblinding for 45 treated subjects, with a second unblinding expected in Q3 2026.

How many countries and sites are in the MBRX MIRACLE pivotal trial?

The MIRACLE trial spans 9 countries with over 46 sites selected and about 20 sites contracted.

What is the planned timeline for MBRX Part B and primary efficacy data?

Part B start is planned in 2H 2026, recruitment continues into 2027, and primary efficacy data targeted in 2028.

What investigator‑initiated trials for MBRX drugs are expected in 2026?

An Atlantic Health pancreatic cancer trial for Annamycin and a follow‑on Emory WP1066 trial are expected to begin in 2026.

What is the size and design of MIRACLE Part A for MBRX (MBRX)?

Part A randomizes 90 subjects (1:1:1) to HiDAC plus placebo, 190 mg/m2 Annamycin, or 230 mg/m2 Annamycin, with early unblinding options.

What WP1066 clinical activity does Moleculin (MBRX) have planned for 2026?

The oral WP1066 Phase 2 GBM trial at Northwestern continues in Q1 2026, with a pediatric brain tumor trial at Emory planned for 2H 2026.