Moleculin Biotech, Inc.
For the period ended Mar 31, 2026. Filed May 14, 2026.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2026 · Filed May 14, 2026
MBRX$8.70-0.3%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-09-30; filed 2025-11-13.
0001437749-25-034839
Item 2. Management's Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-03-31; filed 2026-05-14.
0001437749-26-017034
Item 2. Management's Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 105 previous paragraphs and 60 current paragraphs.
21 unchanged; 11 reworded; 28 added; 72 removed; 1 previous and 0 current paragraphs rearranged.
Tables are not compared: 18 previous and 23 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (28)
Current report
We have two additional portfolios of technologies for hard-to-treat cancers and viruses with clinical and preclinical research funded by investigators at academic institutions. One of these portfolios has an ongoing investigator-initiated trial, another one planned and additional active preclinical testing. Since our inception, drug candidates from each of the three core portfolios are in active planning for, have approval to begin, are currently in or have successfully completed eighteen clinical trials.
Current report
We believe Annamycin is a first-in-class, next-generation anthracycline and DNA-binding agent designed to overcome key limitations associated with currently approved anthracyclines. Whereas first- and second-generation anthracyclines are currently used to treat approximately half of all cancers, they carry with them the burden of dangerous cardiotoxicity and efficacy limitations associated with multidrug resistance mechanisms and poor tissue/organ distribution. Later generation anthracyclines are comprised mainly of attempts to reduce cardiotoxicity or enhance anticancer activity, but we believe have failed to deliver the necessary safety and efficacy profiles to win approval or gain market acceptance. Annamycin is an entirely new chemical entity integrating a new molecular structure intended to enhance its ability to selectively kill cancer cells and reduce toxic side effects. Annamycin possesses a unique and innovative multilamellar lipid-based delivery system designed to improve bioavailability and to increase therapeutic window. The result is a next-generation anthracycline that we believe effectively addresses critical problems associated with currently used anthracyclines. It is our lead drug candidate, and we have concluded a Phase 1B/2 clinical trial for treating AML and are now conducting a Phase 3 clinical trial for R/R AML, which we believe will be registration enabling. We have also sponsored two Phase 1B/2 clinical trials of Annamycin for treating Soft Tissue Sarcoma metastasized to the lungs (STS lung metastases, STS lung mets, or Advanced STS), the results of which we believe supports further clinical development beyond Phase 1 and further evaluation of Annamycin’s therapeutic potential in this indication.
Current report
We believe that our lead drug candidate Annamycin has:
Current report
Core Technologies and Focus
Current report
Our Current Clinical Trial Focus
Current report
We are focused on the development of Annamycin and WP1066 through internally and externally funded programs (“internally” and “externally” are defined in the Funding Strategy section below) as follows:
Current report
We have established a Recommended Phase 2 Dose for WP1122 to potentially enable future externally funded oncology and virology trials. Beyond this, we support development of our core technologies through sponsored (internally funded) non-clinical research at MD Anderson.
Current report
Clinical and Regulatory Updates
Current report
On May 13, 2026, we announced that the first unblinding of data from our pivotal Phase 2B/3 “MIRACLE” trial remains on track to occur prior to June 30, 2026, as expected. The MIRACLE trial is evaluating Annamycin in combination with cytarabine compared to cytarabine plus a placebo for the treatment of subjects that have been relapsed or refractory to their primary line of treatment for acute myeloid leukemia (R/R AML). The trial incorporates two arms of Annamycin at different doses plus cytarabine compared to the control arm of cytarabine plus a placebo.
Current report
We also reported that preliminary blinded efficacy data for the first 45 subjects treated continue to approximate previously disclosed results, including a composite complete remission rate (CRc) exceeding 40% and a complete remission (CR) rate of approximately 30%. These results compare favorably to historical CR rates from two major independent trials of approximately 17–18% observed with cytarabine alone in similar patient populations. Cytarabine monotherapy is considered a standard of care for second line treatment of AML. Based on preliminary data, the median age for subjects enrolled is in the mid-60’s, with over 30% entering the trial after becoming relapsed from or refractory to a prior venetoclax regimen as first line therapy, which is considered a particularly challenging patient group. As of May 5, 2026, 56 subjects (62% of Part A) have been recruited and randomized in the MIRACLE trial, keeping us on track to recruit the 90th subject in Part A in Q3 2026.
Current report
On March 23, 2026, we announced that the 45th subject was enrolled in the MIRACLE trial, a milestone triggering the final phase of preparation for the trial’s highly anticipated interim 45 subject data unblinding, which remains on track for mid-2026.
Current report
Abstract on Cardiac Safety of Annamycin at EHA
Current report
On May 12, 2026, we announced the publication of an abstract at theEuropean Hematology Association (EHA) 2026 Congress highlighting the cardiac safety profile of Annamycin (or as known in scientific journals "L-Annamycin"). The abstract titled, "Cardiac Safety of L-Annamycin Across High Cumulative Anthracycline Exposure: Implications for Relapsed/Refractory AML," highlighted pooled cardiac safety findings from five completed clinical trials evaluating Annamycin in heavily pretreated patients, including those with substantial prior anthracycline exposure. Importantly, the analysis demonstrated no clinically significant treatment-related cardiotoxicity across cumulative anthracycline-equivalent doses that exceeded conventional lifetime exposure limits associated with traditional anthracyclines.
Current report
The independent cardiac review, conducted by a cardio-oncology laboratory at the Cleveland Clinic, analyzed comprehensive cardiac monitoring data from 90 patients treated with Annamycin. Among 78 patients with source-data verified pre- and post-treatment ejection fraction assessments, no patients met criteria for clinically significant left ventricular dysfunction. Mean ejection fraction remained stable, and no association was observed between cumulative dose and cardiac function decline. Additional analyses of serial ECG's, troponins, and global longitudinal strain assessments similarly demonstrated no evidence of drug-induced cardiotoxicity.
Current report
Data Presented at AACR 2026 Annamycin Extends Survival in Metastatic Pancreatic Cancer Preclinical Models
Current report
On April 23, 2026, we announced the presentation of new preclinical data at the American Association for Cancer Research (AACR) Annual Meeting 2026 highlighting the potential of our lead drug candidate, Annamycin, in pancreatic cancer. Access the poster here. The data demonstrate that liposomal Annamycin (L-ANN or naxtarubicin), a novel, non-cardiotoxic anthracycline, produced significant tumor growth inhibition across multiple pancreatic ductal adenocarcinoma (PDAC) models, including orthotopic human PDAC and syngeneic systems, with strong statistical significance (p < 0.001). These findings were accompanied by a meaningful survival benefit in a metastatic model, where treatment extended median survival by more than 60% compared to control (29 days versus 18 days; p = 0.0003), underscoring the potential clinical relevance of L-ANN in aggressive disease settings. Pharmacokinetic analyses further demonstrated enhanced tumor penetration and retention of Annamycin compared to doxorubicin, with significantly higher accumulation observed in pancreatic tissue and tumors (p<0.0001). These data provide a mechanistic basis for the observed anti-tumor activity and highlight a key differentiating feature of Annamycin relative to traditional anthracyclines, which have historically shown limited efficacy in pancreatic cancer. In addition to its direct cytotoxic effects, L-ANN was shown to induce immune activation within the tumor microenvironment, including increased infiltration of CD8+ cytotoxic T cells and CD4+ helper T cells. These findings suggest the potential for Annamycin to convert immunologically “cold” pancreatic tumors into more responsive phenotypes, supporting its evaluation both as a monotherapy and in combination with other drugs, including immune checkpoint and KRAS inhibitors.
Current report
Consistent with prior studies, Annamycin also demonstrated a favorable safety profile, including the absence of cardiotoxicity, a well-documented limitation of conventional anthracyclines such as doxorubicin. This differentiated safety profile may enable broader therapeutic use and synergistic combination strategies.
Current report
Abstract Accepted for Poster Presentation at the 2026 ASCO Annual Meeting
Current report
On April 21, 2026, we announced that an abstract highlighting data on our lead drug candidate, Annamycin, was accepted for poster presentation at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting, taking place May 29 – June 2, 2026, in Chicago, Illinois. The abstract, titled “Cardiac safety of L-annamycin at high cumulative anthracycline exposure: Pooled analysis,” will be presented in a poster session focused on Symptom Science and Palliative Care. The abstract presents a pooled analysis evaluating the cardiac safety profile of annamycin in patients with high cumulative exposure to anthracyclines, an important consideration given the known risk of cardiotoxicity associated with this class of chemotherapeutic agents.
Current report
Closing Australian Subsidiary
Current report
In June 2018, we formed Moleculin Australia Pty. Ltd., a wholly owned subsidiary to oversee pre-clinical development in Australia. In April 2026, we began the process of closing this subsidiary to reduce corporate overhead costs. Both the board of directors of Moleculin and the subsidiary approved the closing of the subsidiary. The subsidiary did not have any employees or conduct a separate line of business. With the MIRACLE trial focused in the US, Europe, and, possibly, the Middle East future activities are not anticipated to include Australia.
Current report
Patents for Annamycin Globally
Current report
We announced on May 8, 2026, an advancement in our intellectual property portfolio with the issuance of a Hong Kong patent covering our proprietary method of reconstituting liposomal Annamycin. The newly granted patent (No. 40073244), titled “Method of Reconstituting Liposomal Annamycin,” extends protection through June 25, 2040, further reinforcing Moleculin’s long-term global exclusivity strategy. The invention is jointly owned with The University of Texas System.
Current report
Business transactions
Current report
In May 2026, we sold 354,757 shares under the 2025 ATM Agreement for net proceeds of approximately $0.8 million where the lowest price sold was $2.01 per share which reset the Series G and H warrants from $2.40 to $2.01 per share.
Current report
In May 2026, we entered into a consulting agreement to provide clinical services pursuant to which it issued the consultant a five-year warrant to purchase 100,000 shares of common stock at an exercise price of $2.48 per shares vesting in twelve equal installments over a one-year period.
Current report
Moleculin Biotech, Inc.
Current report
On February 19, 2026, we entered into warrant inducement agreements with certain holders of our existing equity-classified Series E and Series F warrants. Pursuant to the agreements, the holders exercised warrants to purchase an aggregate of 2,122,652 shares of our common stock at an exercise price of $3.90 per share, resulting in aggregate gross proceeds of approximately $8.3 million. In consideration for such exercises, we issued new Series H warrants to purchase up to 6,367,956 shares of common stock. The Series H warrants have a five-year term and an exercise price of $2.3976 per share. The warrants contain customary anti-dilution adjustments and beneficial ownership limitations and became exercisable upon stockholder approval in April 2026. As a result of the completion of this transaction, the exercise price of our outstanding Series E warrants was adjusted to $3.00 per share, and the number of shares underlying the remaining outstanding Series E warrants increased, in each case pursuant to the anti-dilution provisions contained in such warrants. In addition, the exercise price of our outstanding Series F warrants was adjusted to $2.75 per share, and the exercise price of our outstanding Series G warrants was adjusted to $2.3976 per share. We recorded a deemed dividend of $1.8 million resulting from the downward adjustment to the exercise price of certain warrants and the increase in the number of shares underlying certain outstanding warrants, in each case triggered by the anti-dilution provisions contained in the original warrant agreements. The deemed dividend was recorded within additional paid-in capital and recognized as a reduction to income available to common stockholders.
Removed paragraphs (72)
Previous report
Annamycin is in a class of drugs referred to as Anthracyclines, which are inhibitors of topoisomerase II, enabling them to cause DNA damage in rapidly replicating tumor cells. Annamycin, in a unique multilamellar lipid formulation, is our lead molecule and we have recently concluded treating subjects in several Phase 1B/2 clinical trial for treating AML and are embarking on a Phase 3 clinical trial for the treatment of AML, which we believe will be pivotal. Annamycin has also been in two Phase 1B/2 clinical trials for treating Soft Tissue Sarcoma metastasized to the lungs (STS lung metastases, STS lung mets, STS or Advanced STS). Our Phase 1B/2 STS lung mets trial MB-107 is completed, and a clinical study report has been issued. The other trial is an investigator initiated Advanced STS trial which is closed to recruitment.
Previous report
Focus and Core Technologies
Previous report
Annamycin and the Lack of Cardiotoxicity
Previous report
As part of our Annamycin clinical trials, we have engaged an independent expert at the Cleveland Clinic to assess cardiotoxicity associated with chemotherapy (Expert or Independent Expert). The data made available to the Expert include left ventricular ejection fraction (LVEF) as determined by echocardiograms, and ECHO strain imaging, as well as serum Troponin levels (a biochemical marker of acute heart damage). “ECHO strain imaging” is a method in echocardiography (medical ultrasound) for measuring regional or global deformation (contraction or beating) of the myocardium (heart muscle). By strain rate imaging, the simultaneous function of different regions can be displayed and measured. Cardiac health biomarkers such as blood Troponin levels are considered an indicator of potential long-term heart damage. The Expert issued periodic reports during our MB-106 clinical trial and will also issue updated reports over the course of the MIRACLE trial. Such data from clinical trials where a clinical study report has not yet been issued include some data which are preliminary and subject to change.
Previous report
We now have independent assessments covering 84 subjects that have been treated with Annamycin in five different clinical trials in the US and Europe with no evidence of cardiotoxicity. To date of the 77 subjects treated in our internally funded trials, 56 were treated above the FDA’s lifetime maximum anthracycline limit of 550 mg/m 2, including one subject whose repeated Annamycin treatments took that subject to a total cumulative anthracycline exposure of 3,420 mg/m 2 (or roughly five times the FDA approved lifetime anthracycline exposure) and there has been no evidence of cardiotoxicity reported. After review of the data provided, the Independent Expert concluded that there was no evidence of drug-related cardiotoxicity in any subjects. Furthermore, additional subjects have been treated in the MIRACLE trial and, to date, no cardiotoxicity has been observed, bringing the total subjects treated safely with Annamycin to almost 100 subjects.
Previous report
We believe the Expert's reports are particularly relevant in light of a recently published retrospective study showing that the incidence of heart failure more than doubles for cancer patients treated with anthracyclines compared to cancer patients not receiving anthracyclines (C Larson, et al. Anthracycline and Heart Failure in Patients Treated for Breast Cancer or Lymphoma, 1985-2010. JAMA Network Open. 2023;6(2):e2254669. doi:10.1001/jamanetworkopen.2022.54669). Given the heart-damaging impact of prior treatment with currently prescribed anthracyclines, and considering that the subject population that we are enrolling in our Annamycin trials (multiple prior therapies, including anthracyclines known to be cardiotoxic, many elderly, and other comorbidities) we believe that there is a high likelihood that a cardiac event will eventually occur in the future that we will not be able to disassociate from Annamycin. We believe that the potential for such future incidences, however, does not outweigh the significant lack of cardiotoxicity to date as reflected in the Expert’s reports.
Previous report
Although we have not definitively concluded that Annamycin is incapable of cardiotoxicity, we discuss Annamycin’s lack of demonstrated cardiotoxicity in this document, corporate presentations and other public communications. Such discussions reflect the information in the preceding paragraphs but in summary are based on the following: 1) Annamycin was initially designed to be non-cardiotoxic; 2) Preclinical studies have demonstrated Annamycin lacks cardiotoxicity as compared to a currently prescribed anthracycline; and, 3) Our Expert’s conclusions from analyzing the data from 84 subjects treated with Annamycin in five clinical studies conducted in the US and in the EU. Our discussions will reflect any additional data or analyses as they become available.
Previous report
Clinical Trials Summary
Previous report
We have multiple active INDs/CTAs ("Investigational New Drug" authorization in the US or "Clinical Trial Authorization" in other countries). These INDs/CTAs are under development, approved, in progress, or completed and comprise a total of fourteen clinical trials, internally and externally funded. With Annamycin, we closed one AML clinical trial: MB-106 which was a Phase 1B/2 treating AML with the combination of Annamycin and Cytarabine (together referred tp as “AnnAraC”). The Clinical Study Report is now being written. Additionally, MB-108 is a Phase 2B/3 pivotal clinical trial treating R/R AML as 2 nd line therapy (recruitment has started and additional sites are being added) with AnnAraC. Finally, there is one externally funded Phase 1B/2 trial treating STS Lung Mets with Annamycin as monotherapy. This trial is closed and is in follow-up on the trial’s subjects. With WP1066, we have an externally funded a Phase 1B/2 trial in combination with radiation treating glioblastoma (GBM) at Northwestern University that is active.
Previous report
MIRACLE Trial
Previous report
We have a Phase 3 pivotal trial for the treatment, with Annamycin in combination with Cytarabine, of AML patients who are R/R after induction therapy. This Phase 3 MIRACLE trial is a global study. It is an adaptive trial designed with a lead-in Part A portion of the study having three arms (control arm with cytarabine, and two test arms with cytarabine plus two different doses of Annamycin) intended to determine an “optimum dose” to be used in Part B of the study. Once an optimum dose for the Annamycin is determined the study will continue into Part B with two arms (control arm with cytarabine and a test arm with cytarabine plus Annamycin). In some contexts, we refer to Part A as Phase 2B and Part B as Phase 3, however the adaptive design enables the combining of data from Part A (for the optimum dose) with Part B such that data from both parts A and B will comprise Phase 3 data.
Previous report
MIRACLE Trial
Previous report
Clinical and Regulatory
Previous report
On November 13, 2025, we announced an update, as of November 4, 2025, on recruitment for MIRACLE with 60% of the targeted 45 subjects consented into the trial with additional subjects identified by site investigators. We believe that the blinded complete remission response activity to date is within the randomized expectations, based on historical clinical data for the treatment arms. The currently consented subjects are from across sites in five countries, providing a diverse base of subjects. With the upcoming holidays in addition to unexpected bed shortages at certain EU sites, we expect treatment of the last of the first 45 subjects in the first quarter of 2026. Such data will be audited, locked, and reviewed prior to release. The release of the unblinded data will be thereafter. The final unblinded data of the first 90 subjects will be the basis for the Data Safety Monitoring Committee’s (DSMC) recommendation of the treatment arms going forward and, most importantly, on any needed actions related to subject safety. Subjects have been identified in the US, and to date, none have passed our pre-screening protocol.
Previous report
We announced on September 9, 2025, updates to our site status and recruitment and as of early September 2025, 13 subjects had been screened, enrolled, or dosed, with a target of reaching 20 active/screening sites by month-end. On September 1, 2025, and treated our first subject in the European Union (EU).
Previous report
As of July 25, 2025, we selected over 35 sites to conduct Part A of the MIRACLE trial in the US, the EU, and Eastern Europe. Four sites (one each in Ukraine, the country of Georgia, Spain and the US) were actively recruiting, screening and/or treating subjects. Besides these four sites the MIRACLE trial was approved by over 25 ethics committees of various clinics in the US and the EU. Eight subjects were treated as of July 25, 2025 in the MIRACLE trial.
Previous report
Since February and through October of 2025, we received a total of ten separate communications from the FDA including additional comments and information requests, all of which we addressed. These requests focused mainly on safety, subject monitoring, clinical pharmacology, statistics, and inclusion/exclusion criteria. None of the requests resulted in significant changes to overall trial design or dosing of Annamycin. Such requests require from time to time, an update to our protocol, which must be processed through each regulatory/clinical jurisdiction, and we believe such requests and responses will continue as we progress the MIRACLE trial through multiple countries. The US and EU protocols with the FDA and EMA, respectively, are currently “harmonized” and future requests as we proceed may require future “harmonization”.
Previous report
In November 2024, we amended our existing US investigational new drug application or IND for inclusion of the MIRACLE trial protocol. In the amendment with the new MIRACLE protocol, the trial will allow, for the first time in the US for AML subjects, dosing above the lifetime maximum allowable dose for currently prescribed anthracyclines. Then in February 2025 we received FDA feedback and guidance on that amendment. Such feedback allowed a reduction in the size of our Part B of the Phase 3 pivotal trial protocol to 222 subjects. Guidance from FDA included a recommendation to alter the statistical plan that reduced the initially proposed size of Part B of our trial by approximately 10%. Moreover, the nature of the feedback helps us move forward quickly to open sites in the US, in addition to the non-US sites we are expecting to open. Any reduction in recruitment enables shortening the time for completion of the trial.
Previous report
On July 9, 2025, we announced that the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in the country of Georgia has approved our Clinical Trial Application (CTA) to conduct the MIRACLE trial, further expanding our active sites beyond Ukraine.
Previous report
On May 12, 2025, we announced that the European Medicines Agency (EMA) approved its Clinical Trial Application to conduct the MIRACLE trial in all nine countries submitted in the European Union (EU). We received the final Report for the Application and Evaluation Assessment (part of the EMA’s Clinical Trial Information System or CTIS process) of "Acceptable" for Belgium, Czechia, France, Germany, Italy, Lithuania, Poland, Romania, and Spain. Such approval was under the condition that we present results of appropriate nonclinical GLP studies before initiating the Phase 3 portion (Part B) of the MIRACLE study. Such results will be submitted as a substantial modification. Along with the individual country committee and/or ethics approvals for these nine countries in the EU, this allows us to proceed. While there are minor differences between the US and EU protocols with the FDA and EMA, respectively, we do not view these as a barrier to conducting the study and are working to harmonize the protocols as appropriate.
Previous report
MB-106 Clinical Study with AnnAraC for the Treatment of AML (1st through 7th Linse Subjects)
Previous report
We completed our Phase 1B/2 (MB-106) clinical trial evaluating AnnAraC for the treatment of subjects with AML with the database for the trial locked in September 2025. The data below is subject to the final Clinical Study Report (CSR) being published which is projected to be in the first quarter of 2026. In total, 23 subjects were enrolled and 22 dosed in the trial, with all subjects (1st Line through 7th Line or 1L-7L) who received AnnAraC per protocol having completed their efficacy evaluations (n=20). The updated overall survival (OS) data revealed the following:
Previous report
Industry publications note that the typical OS for relapsed AML patients is roughly 4-6 months. As previously reported, 8 subjects in the intent-to-treat (ITT) population of 22 (36%) achieved a complete remission (CR) following treatment with AnnAraC. Among the subjects treated in the second line (2L) setting (n=10), the CR rate (the same primary efficacy endpoint as in the ongoing Phase 2B/3 pivotal MIRACLE trial), was 50%. Median durability for the 8 subjects achieving a CR was 10 months and continuing at the end of the study. CR durability ranged from 2 months to 22 months and continuing. CRs were achieved in subjects with a variety of prior treatments, including traditional 7+3 and venetoclax regimens.
Previous report
This data is considered preliminary until a final CSR is published.
Previous report
Pediatric Study for Annamycin in Combination Therapy with Cytarabine for the Treatment R/R AML
Previous report
Received Positive FDA Feedback on Pediatric Study Plan for Annamycin in Children with R/R AML
Previous report
We announced on June 18, 2025, that we received a written response from the Office of Oncologic Diseases – Pediatric Oncology of the FDA regarding our Initial Pediatric Study Plan (iPSP), which was submitted after a June 2024 end-of-phase 1/2 meeting. The FDA has agreed to a single pediatric approval study in which Annamycin (also known as naxtarubicin) in combination with Cytarabine will be evaluated as second line therapy in pediatric patients with R/R AML.
Previous report
We believe that this is particularly important in pediatric oncology because about 60% of children with cancer are treated with anthracyclines that present a high risk of causing heart damage, we expect to commence this trial in the second half of 2027. As part of the iPSP, we proposed a single-arm study evaluating pharmacokinetics (PK), efficacy, and safety of AnnAraC in patients between 2 and 16 years of age. The FDA response calls for including patients from 6 months of age and older, with no minimum number of patients between 6 months and 2 years old. FDA also clarified that drug concentration exposure and safety profile that are comparable would allow extrapolation from efficacy in adults. Importantly, FDA said we would be able to start the pediatric trial before having the full two years of follow-up data from the adult trial. Between August 2025 and September 2025, we corresponded with the FDA regarding certain specifics of this trial. On September 26, 2025 we received the iPSP agreement letter from the FDA.
Previous report
MB-107 Annamycin in Monotherapy for the Treatment of STS Lung Mets
Previous report
We recently hosted a Key Opinion Leader (KOL) call announcing and reviewing the final top line data from the MB-107 trial. The MB-107 trial involved Annamycin monotherapy treatment for STS Lung Mets. The final top line results of MB-107 demonstrated median overall survival (OS) of 13.5 months for subjects as median 7th line therapy (n=36). This compares to OS of 8-12 months in standard of care treatments and 13.4 months for experimental treatments for advanced STS as 2nd line (Comandone A, et al; “Salvage Therapy in Advanced Adult Soft Tissue Sarcoma: A Systematic Review and Meta-Analysis of Randomized Trials”; The Oncologist 2017;22:1518–1527).
Previous report
Investigator-Initiated Study Annamycin for the Treatment of Pancreatic Cancer
Previous report
On November 2025 we announced grant funded research evaluating Annamycin for the treatment of pancreatic cancer at University of North Carolina - Chapel Hill (UNC). In early November we entered into a research and material transfer agreement with the UNC for investigator-initiated preclinical research evaluating Annamycin for the treatment of pancreatic cancer. The studies covered under this agreement will evaluate the ability of novel treatment agents and modalities to enhance the tumor delivery of liposomal Annamycin (L-Annamycin) and Free-Annamycin as compared to Doxil and Free-doxorubicin in the PDAC GEMM models. Under the terms of the agreement the Company will supply Annamycin and the UNC Eshelman School of Pharmacy, UNC Lineberger Comprehensive Cancer Center, and Carolina Institute of Nanomedicine will conduct the planned preclinical research as part of a series of funded grants*.*
Previous report
On October 23, 2025, we announced that the Company is working with Atlantic Health on an investigator-initiated Phase 1B/2 single-arm study evaluating Annamycin for third-line (3L) treatment of advanced pancreatic cancer. Pancreatic cancer continues to present significant treatment challenges, being the cancer with the highest mortality rate globally and the seventh leading cause of cancer death. However, recent preclinical studies indicate that Annamycin may target critical factors associated with this disease, showcasing its potential as a novel therapeutic option.
Previous report
In connection with the planned study, Moleculin will supply Annamycin and be responsible for submitting and maintaining an Investigational New Drug Application (IND) with the US Food and Drug Administration (FDA), and Atlantic Health will be responsible for conducting the Phase 1B/2 study. Moleculin estimates the additional cost to run this trial from 2026 into 2030 (for long-term follow-up) will be approximately $1 million, mainly for drug supply and outside laboratory testing. This trial is subject to the successful filing of an IND with the FDA, both parties agreeing on a clinical trial agreement, ensuring sufficient drug supply and other critical items leading up to expected commencement of the clinical trial in 2026.
Previous report
Promising Preclinical Data of Annamycin in Liver Cancer Treatment Presented at a Symposium at MD Anderson
Previous report
On August 6, 2025, we announced the presentation of encouraging preclinical data for its lead drug candidate, Annamycin which demonstrated significant efficacy against various primary and metastatic liver cancers, including hepatocellular carcinoma (HCC), colorectal liver metastases, and pancreatic ductal adenocarcinoma (PDAC) liver metastases. This is believed to be the result of targeted accumulation in the liver and other organs. These findings were highlighted in a poster titled "Liposomal Annamycin (L-ANN) Efficacy Against Primary and Metastatic Liver Cancers," presented by Dr. Waldemar Priebe, Lead Author and Chairman of the Scientific Advisory Board at Moleculin at the recently held Shelby-Lavine Pancreatic Cancer Symposium at MD Anderson Cancer Center.
Previous report
AACR Presentation of Pre-Clinical Data for Annamycin Market Expansion Potential, including Pancreatic Cancer
Previous report
We announced on April 29, 2025, that new pre-clinical data for Annamycin demonstrating market expansion potential including treatment for pancreatic cancer was presented at the American Association for Cancer Research (AACR) Annual Meeting in Chicago, IL. With five previous or current investigator-initiated clinical trials supporting development of our drug candidates, we believe that our next investigator-initiated trials could be Annamycin for the treatment of pancreatic cancer or advanced soft tissue sarcomas.
Previous report
The study, presented in poster form, was designed to assess the efficacy of Annamycin in combination with approved anticancer agents in order to identify novel potentially highly efficacious clinical applications of Annamycin alone and with a therapeutic partner. Annamycin in its non-liposomal form (free drug;in vitro) and Liposomal Annamycin (L-ANN;in vivo) were tested in combination with selected FDA approved drugs. Several of the most efficacious drug combinations from thein vitro studies were then tested using well developed in vivo models of leukemia and solid tumors, including sarcoma and pancreatic cancer. It should be noted that in a separate set of previous experiments, Annamycin activity was testedin vitro, and appeared to be highly active, against drug resistant cell lines, including cells resistant to cytarabine and venetoclax.
Showing the first 40 of 72 passages. Open the source sections for the rest.
Reworded paragraphs (11)
Previous wording
We are a late-stage pharmaceutical development company currently conducting a pivotal Phase 3 trial evaluating Annamycin (also known by its non-proprietary name "naxtarubicin") in combination with Cytarabine for the second line (2L) treatment of subjects with relapsed/refractory (R/R) acute myeloid leukemia (AML). This Phase 3 trial plans to have an interim unblinding of data upon the recruitment of the first 45 subjects and the readout of the data from the sites, expected shortly thereafter. We plan an additional unblinding on the first 90 subjects (includes the previously mentioned first 45 subjects). We believe such early visibility for a pivotal registration-enabling trial is highly unique in that stakeholders will receive preliminary safety and efficacy data in the “MIRACLE” trial (derived from Moleculin R/R AMLA nnAraCCl inical Evaluation) within roughly one year of dosing the first subject. We have two additional portfolios of technologies for hard-to-treat cancers and viruses currently with clinical and preclinical research funded by investigators at academic institutions.
Current wording
We are a late-stage pharmaceutical development company currently evaluating Annamycin, also known as L-Annamycin and by its generic name "naxtarubicin”, which we believe is a "next-generation” anthracycline and, with it, are conducting a pivotal Phase 3 trial in combination with cytarabine for the treatment of subjects with relapsed/refractory (R/R) acute myeloid leukemia (AML). We call this the "MIRACLE” trial (derived from Moleculin R/R AML AnnAraC Clinical Evaluation). A blinded preliminary data readout has already been released for the MIRACLE trial, and we expect to have an interim unblinding of data in mid-2026 and an additional unblinding in the second half of 2026, thereby concluding Part A of the two-part trial. We believe such early visibility for a pivotal registration-enabling trial is unique in that stakeholders will receive preliminary safety and efficacy data long before the conclusion of the trial.
Previous wording
Each of our three core technologies is based substantially on discoveries made at and licensed from MD Anderson in Houston, Texas, and features one or more drugs that have successfully completed a Phase 1 clinical trial. Some of the intellectual property was co-developed by us with MD Anderson. Three of our six drug candidates have shown human activity in clinical trials and are currently or have been in Phase 1B/2 or Phase 2 clinical trials. One Phase 2B/3 trial and one Phase 2 trial are currently underway. Since our inception, our drugs have completed, are currently in, or have been permitted to proceed in, fourteen clinical trials. This is expected to increase in 2026 with an expansion of treated indications with Annamycin.
Current wording
Each of our three core technologies is based substantially on discoveries made at, made in conjunction with, and/or licensed from the University of Texas MD Anderson Cancer Center (MD Anderson) in Houston, Texas. For each of the core technologies, one or more drug candidates have successfully completed a Phase 1 or greater clinical trial. Three of our drug candidates have shown human activity in clinical trials and are currently or have been in Phase 1B/2, Phase 2, Phase 2B/3 clinical trials. One of those drug candidates is Annamycin, which is currently in the MIRACLE trial.
Previous wording
One of our core management beliefs is that anthracyclines represent one of the most important treatments for nearly half of all cancers, including AML and Advanced STS, and that Annamycin may increase the efficacy, safety and tolerability of anthracycline therapy fora majority of these patients. We believe that such a benefit could be disruptive to the competitive landscape for these markets. This belief, coupled with our limited resources, leads us to currently focus mainly on the development of Annamycin. We intend to advance our other drug candidates via investigator led studies – both preclinically and clinically.
Current wording
A core management belief is that anthracyclines represent one of the most important treatments available for AML and Advanced STS, as well as many other cancers, and we believe Annamycin may, for the first time ever, allow a majority of these patients to benefit from Annamycin’s improved safety and efficacy by allowing increased dosing (and even maintenance dosing), improved tolerability and allowing access for patients who otherwise would not be eligible for an anthracycline (including the elderly and those with impaired cardiac function). We believe that such benefits will be disruptive to the competitive landscape for these markets. This belief leads us to focus our available resources mainly on the development of Annamycin. We seek to advance our other drug candidates via investigator led studies, both clinically and preclinically, and as available resources will allow.
Previous wording
a) Annamycin or L-Annamycin is a “next generation” anthracycline (one of the most widely used classes of chemotherapy), designed to be different than currently approved anthracyclines, which are limited in utility because of cardiotoxicity risks and their susceptibility to multidrug resistance mechanisms. Annamycin was designed to avoid multidrug resistance and to be non-cardiotoxic and, with intensive cardiac monitoring, has shown no cardiotoxicity in subjects treated in our six Annamycin clinical trials to date. Furthermore, we have demonstrated safe dosing significantly beyond the lifetime dose limitations imposed by regulatory authorities upon commonly prescribed anthracyclines due to their inherent cardiotoxicity. The clinical activity for Annamycin is discussed below.
Current wording
a) Annamycin is what we believe to be a “next-generation” anthracycline (one of the most widely used classes of chemotherapy), designed to be different than currently approved anthracyclines, which are limited in utility because of cardiotoxicity risks, tolerability, their susceptibility to multidrug resistance mechanisms and a lack of efficacy in certain tumor models.
Previous wording
b) Our WP1066 Portfolio includes WP1066, WP1193 and WP1220, three of several Immune/Transcription Modulators in the portfolio designed to inhibit p-STAT3 (phosphorylated signal transducer and activator of transcription 3) among other transcription factors associated with tumor activity. These also stimulate a natural immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs).We have an oral formulation of WP1066 that has been in three clinical trials to date and we are currently performing preclinical work on an intravenous formulation, all of which are funded and performed by investigator-initiated studies.
Current wording
b) Our WP1066 Portfolio includes WP1066, WP1193 and WP1220, three of several Immune/Transcription Modulators in the portfolio designed to inhibit p-STAT3 (phosphorylated signal transducer and activator of transcription) among other transcription factors associated with tumor activity. These also stimulate a natural immune response to tumors by inhibiting the errant activity of Regulatory T-Cells (TRegs).
Previous wording
Research and Development Expense. Research and development (R&D) expensewas$3.7 million and$4.9 million for the three months ended September 30, 2025 and 2024, respectively. The decrease of $1.2 million is mainly related to lower drug production costs during the current quarter as compared to the prior year quarter due to timing of production and offset by higher clinical operations costs in the current period quarter.
Current wording
Research and Development Expense. Research and development (R&D) expensewas$5.4 million and$3.4 million for the three months ended March 31, 2026 and 2025, respectively. The increase of $2.0 million is mainly related to the MIRACLE clinical trials in Europe of $1.4 million and additional nonclinical studies of $0.3 million plus other research costs during the current quarter as compared to the prior year quarter.
Previous wording
Gain from Change in Fair Value of Warrant Liability. We recorded a net gain of$1.6 million inthe third quarter of 2025 due to the change in fair value of the warrant liability. The liability-classified warrants are valued at the time of each warrant issuance date, if applicable, and at the end of each reporting period. Changes in fair value are reflected in the statement of operations as a gain or loss from the change in fair value of the warrant in the period in which the change occurred. A gain results principally from a decline in our share price during the period and a loss will result principally from an increase in our share price.
Current wording
Gain from Change in Fair Value of Warrant Liability. We recorded a net gain of$10.8 million inthe first quarter of 2026 due to the change in fair value of the Series H warrant liability after the February 2026 inducement. Changes in fair value are reflected in the statement of operations as a gain or loss from the change in fair value of the warrant in the period in which the change occurred. A gain results principally from a decline in our share price during the period and a loss will result principally from an increase in our share price.
Previous wording
Transaction costs allocated to warrant liabilities and Loss on issuance of warrant liabilities. Proceeds of offerings are allocated between common shares and warrants first by allocating to the warrants classified as a liability based on their fair value and then allocating the residual to the equity instruments, which would include pre-funded warrants. Transaction costs on the Series F warrants in August 2025 amounted to $0.5 million as compared to no loss in the prior quarterly period. The loss on issuance of the warrant liabilities for the Series F warrants was $20.6 million as compared to no loss for the prior quarterly period in 2024.
Current wording
Transaction costs allocated to warrant liabilities and Loss on issuance of warrant liabilities. Proceeds of offerings are allocated between common shares and warrants first by allocating to the warrants classified as a liability based on their fair value and then allocating the residual to the equity instruments. Transaction costs on the Series H warrants in February 2026 amounted to $0.7 million. The loss on issuance of the warrant liabilities for the Series H warrants was $15.2 million as compared to no loss for the prior quarterly period in 2025.
Previous wording
Cash used in operationswas $17.4 million for thenine months ended September 30, 2025. This $1.4 million decrease over the prior year period of $18.8 million was primarily due to the timing of costs incurred and associated payments primarily for clinical trial expenses and drug production costs.
Current wording
Cash used in operationswas $6.1 million for thethree months ended March 31, 2026. This $1.5 million increase over the prior year period of $4.6 million was primarily due to the timing of costs incurred and associated payments for clinical trial expenses and drug production and other testing related expenses.
Previous wording
In July 2025, we entered into an at-the-market equity agreement (the 2025 ATM Agreement) with Roth Capital Partners, LLC (Roth). Pursuant to the terms of the 2025 ATM Agreement, we may offer and sell shares of our common stock having an aggregate offering price of up to $6.5 million from time to time through or to Roth acting as our sales agent or principal. Roth may also sell shares of our common stock in negotiated transactions at market prices prevailing at the time of sale at prices related to such prevailing market prices. We will pay a commission to Roth equal to 3.0% of the gross proceeds from the sale of our common stock under the 2025 ATM Agreement. We sold 736,549 shares of common stock pursuant to the 2025 ATM agreement in October for gross proceeds of $0.4 million at prices ranging from $0.4349 to $0.5601.
Current wording
On December 23, 2025, we increased the maximum aggregate gross sales price of our common stock that may be offered, issued and sold under our at the market equity offering agreement (the “2025 ATM Agreement”) with Roth Capital Partners, LLC (“Roth”), which we initially entered into in July 2025, from $6.5 million to $8.2 million. Pursuant to the terms of the 2025 ATM Agreement, we may offer and sell shares of our common stock having an aggregate offering price of up to $8.2 million from time to time through or to Roth, acting as sales agent or principal. Roth may sell shares of our common stock in negotiated transactions at market prices prevailing at the time of sale or at prices related to such prevailing market prices. We pay Roth a commission equal to 3.0% of the gross proceeds from any shares of common stock sold under the 2025 ATM Agreement. During the first quarter of 2026, we sold 3,785 shares of common stock pursuant to the 2025 ATM Agreement for gross proceeds of approximately $18,000.
Previous wording
We believe that our cash on hand and cash equivalents as of September 30, 2025, is sufficient to fund our planned operations into the first quarter of 2026. This takes into account cash outlays for preparations for clinical trials beyond the current active trials. The continuation of our Company as a going concern is dependent upon our ability to obtain necessary financing to continue operations and the attainment of profitable operations. We must seek additional funds of approximately $7 million, to support MIRACLE and our operations into the second quarter of 2026 through the combination of equity offerings, debt financings, government or other third-party funding, commercialization, marketing and distribution arrangements, other collaborations, strategic alliances and licensing arrangements and delay planned cash outlays or a combination thereof to continue our operations in near term. We cannot provide assurance that such events or a combination thereof can be achieved.
Current wording
We believe that our cash on hand and cash equivalents as of March 31, 2026, plus the $0.8 million raised subsequent to the quarter via issuance of common stock under its ATM, is sufficient to fund our planned operations into the third quarter of 2026. This takes into account cash outlays for preparations for clinical trials beyond the current active trials. The continuation of our Company as a going concern is dependent upon our ability to obtain necessary financing to continue operations and the attainment of profitable operations. We must seek additional funds of approximately $25 million, to support MIRACLE and our operations into the first quarter of 2027 through the combination of equity offerings, debt financings, government or other third-party funding, commercialization, marketing and distribution arrangements, other collaborations, strategic alliances and licensing arrangements and delay planned cash outlays or a combination thereof to continue our operations in near term. We cannot provide assurance that such events or a combination thereof can be achieved.
Rearranged previous paragraphs (1)
Previous report — wording carried elsewhere
Gain from Change in Fair Value of Warrant Liability. We recorded a net gain of $1.6 million for the nine months ended September 30, 2025 as compared to a netgain of $1.0 million in the nine months ended September 30, 2024, due to the change in fair value on revaluation of our warrant liability. We are required to revalue our liability-classified warrants at the time of each warrant issuance, if applicable, and at the end of each reporting period and reflect in the statement of operations a gain or loss from the change in fair value of the warrant in the period in which the change occurred. We calculated the fair value of the warrants outstanding using the Monte Carlo model for Series E and F warrants. A gain results principally from a decline in our share price during the period and a loss results principally from an increase in our share price.