naxtarubicin (L-ANN) / Moleculin - LARVOL DELTA

Cardiac safety of L-Annamycin: a pooled analysis of five clinical trials. (PubMed, Front Cardiovasc Med) - "In this pooled analysis of patients with AML and STS, L-Annamycin was not associated with clinical or subclinical evidence of cardiotoxicity at cumulative doses exceeding traditional anthracycline thresholds. These data support continued clinical evaluation of L-Annamycin as a potentially safer anthracycline platform."

Journal • Retrospective data • Acute Myelogenous Leukemia • Cardiovascular • Hematological Malignancies • Leukemia • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

Expected Milestones for the Annamycin Development Program: 2028 & 2029 (GlobeNewswire) - "(i) 2028: End recruitment of Part B; (ii) 2028: Primary efficacy data for MIRACLE 2nd line subjects' (iii) 2028: Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE; (iv) 2029: NDA submission complete."

FDA filing • P2/3 data • Trial status • Acute Myelogenous Leukemia

Expected Milestones for the Annamycin Development Program: 2026 & 2027 (GlobeNewswire) - "(i) September 2026: Completion of enrollment in Part A of MIRACLE; (ii) December 2026 to February 2027 timeframe: MIRACLE - data unblinding for Part A 90 subjects completed; (iii) 2H 2026: Atlantic Health pancreatic cancer clinical trial begins; (iv) 1H 2027: MIRACLE - Start of Part B; (v) 2027: Begin 3rd line R/R AML subject trial; (vi) 2027: Begin pediatric AML clinical study."

New trial • P2/3 data • Trial status • Acute Myelogenous Leukemia • Pancreatic Cancer

Moleculin Reports Positive Phase 2/3 MIRACLE Interim Results, With Annamycin Complete Remission Rates 3-fold Greater than Control (Moleculin Press Release) - "The interim analysis demonstrated a clear efficacy advantage for both Annamycin treatment arms, 190 mg/m² plus HiDAC and 230 mg/m² plus HiDAC, over the HiDAC control arm. CR reached 43% and 36% in the respective Annamycin cohorts, compared with 12% for control, while CRc reached 50% and 57%, respectively, versus 29% for the control arm. The n=45 population contained 75.6% over 60 years of age, 55.6% 7+3 and 31.1% venetoclax regimens for first line (1L) therapies....The interim review was conducted by the trial’s Independent Data Monitoring Committee (iDMC)."

P2/3 data • Acute Myelogenous Leukemia

CARDIAC SAFETY OF L-ANNAMYCIN ACROSS HIGH CUMULATIVE ANTHRACYCLINE EXPOSURE: IMPLICATIONS FOR RELAPSED/REFRACTORY AML (EHA 2026) - "These findings support continued evaluation of L-Annamycin as a second-generation anthracycline in R/R AML, including in patients previously treated with anthracycline-based therapy and venetoclax-based regimens, where prior cumulative exposure and limited salvage efficacy remain significant clinical challenges. ."

Clinical • Acute Myelogenous Leukemia • Heart Failure • Hematological Malignancies • Leukemia

Cardiac safety of L-annamycin at high cumulative anthracycline exposure: Pooled analysis. (ASCO 2026) - "In a Phase 1b/2 study in patients with acute myeloid leukemia (AML), L-Annamycin administered in combination with cytarabine as second-line therapy achieved a 50% complete remission (CR) rate and a 60% composite CR (CRc) rate with a median overall survival of 12.39 months (95% CI, 2.07–13.96) in the intent-to-treat population. L-Annamycin was not associated with any detectable cardiotoxicity despite high cumulative anthracycline exposure. These findings suggest that L-Annamycin represents a next-generation anthracycline capable of delivering effective anthracycline therapy without traditional cumulative dose limitations, thus further supporting its ongoing pivotal Phase 2b/3 trial in AML patients."

Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Sarcoma • Soft Tissue Sarcoma • Solid Tumor

Moleculin Highlights Abstract Accepted for Poster Presentation at the 2026 ASCO Annual Meeting Highlighting Cardiac Safety Data for Annamycin (GlobeNewswire) - "The abstract presents pooled cardiac safety analyses from sponsor- and investigator-initiated clinical trials evaluating Annamycin...in patients with acute myeloid leukemia (AML) and soft tissue sarcoma....Independent cardiac safety review conducted in 90 patients treated with L-Annamycin across five completed clinical trials. Source-verified pre-and-post-treatment left ventricular ejection fraction (LVEF) assessments available for 78 patients. Median cumulative L-Annamycin dose of 660 mg/m2 (95% CI, 645–690; range, 210–2,970 mg/m2), with most cumulative doses exceeding conventional lifetime anthracycline limits. No statistically significant change in LVEF from baseline to final assessment (mean difference, -0.12%; 95% CI, -1.34 to 1.09; p = 0.84)."

Retrospective data • Acute Myelogenous Leukemia • Soft Tissue Sarcoma

MIRACLE Trial Progress and Next Steps (Yahoo Finance) - "The unblinding of the first 45 subjects is on track to occur in late Q2 2026, as the data are subject to final data entry and audit. The second group of 45 subjects in Part A are expected to be fully recruited in the third quarter of 2026 with unblinding expected late in the second half of 2026. Recruitment of the second group of 45 subjects in Part A will continue uninterrupted while the first 45 subjects with efficacy are being unblinded. Unblinding for the full 90 subjects in Part A may require more time than for the first 45 as it involves more data to support the transition from Part A to Part B."

Trial status • Acute Myelogenous Leukemia

Moleculin Announces Imminent MIRACLE Trial Unblinding as Blinded Data Continue to Significantly Outperform Historical Benchmarks (Yahoo Finance) - "The trial incorporates two arms of Annamycin at different doses plus cytarabine compared to the control arm of cytarabine plus a placebo. The Company continues to expect this unblinding to occur prior to June 30, 2026, as previously communicated...The Company also reported that preliminary blinded efficacy data for the 45 subjects continue to approximate previously disclosed results, including a composite complete remission rate (CRc) exceeding 40% and a complete remission (CR) rate of approximately 30%...As of May 1st, a total of 56 subjects have been recruited in the MIRACLE trial to date keeping the Company on track to recruit the 90th subject in Part A in Q3 2026."

P2/3 data • Trial status • Acute Myelogenous Leukemia

Moleculin Biotech Secures Strategic Additional Patent, Strengthening Global Positioning of Annamycin (GlobeNewswire) - "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."

Patent • Acute Myelogenous Leukemia • Soft Tissue Sarcoma

A potent TOP2 poison with robust preclinical efficacy and a favorable therapeutic window supporting clinical evaluation in pancreatic cancer (AACR 2026) - "Several TOP2A targeting drugs, including doxorubicin (DOX) are available, but none represent effective PDAC treatment modalities. Annamycin (ANN), a potent, non-cardiotoxic analog of DOX, but in contrast to DOX exhibits high accumulation in the pancreas and displays robust antitumor activity in orthotopic human PDAC models...Our preclinical studies demonstrate robust antitumor activity of L-ANN in different PDAC models, including prior human PDAC models and now an implantable syngeneic immunocompetent KPC model, that correlates with high tumor uptake of L-ANN. Along with low toxicity prolife of L-ANN (e.g. lack of cardiotoxicity) established in ongoing clinical trials, these data substantiate the clinical evaluation of L-ANN in PDAC patients."

Preclinical • Oncology • Pancreatic Cancer • Pancreatic Ductal Adenocarcinoma • Solid Tumor • CD4 • CD8 • TOP2A

Moleculin’s Annamycin Extends Survival by More Than 60% in Metastatic Pancreatic Cancer Preclinical Models - Data Presented at AACR 2026 (GlobeNewswire) - "The data demonstrate that liposomal Annamycin (L-ANN), 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)."

Preclinical • Pancreatic Cancer • Pancreatic Ductal Adenocarcinoma

Non-cardiotoxic properties of annamycin, a clinically evaluated anthracycline and potent topoisomerase 2β poison (AACR 2024) - P1/2 | "Cardiotoxicity is an important side effect limiting the clinical use of all anthracyclines, including doxorubicin (DOX)...In contrast, DOX caused a dramatic change in heart beating rate and decreased in contractility and field potential amplitude...Interestingly, ANN appeared to be a significantly more potent inhibitor of Top2β than DOX, suggesting that Top2β may not be a universal mediator of cardiotoxicity of anthracyclines. Additional studies are needed to reevaluate the role of Top2β in anthracycline-induced cardiotoxicity."

Clinical • Late-breaking abstract • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Sarcoma • Soft Tissue Sarcoma • Solid Tumor • TOP2A

Combining annamycin, a non-cardiotoxic potent topo II poison, with azacitidine, cytarabine, gemcitabine, ifosfamide, trabectedin, or vincristine to synergize anticancer effects and identify potential clinical applications (AACR 2025) - "Background: Annamycin (ANN) is a potent topoisomerase II poison structurally related to doxorubicin (DOX) but displaying superior biological properties, such as high in vivo efficacy, reduced toxic side effects, and activity against cancers resistant to common therapeutic agents...The most efficacious drug combinations from the in vitro studies were then tested using in vivo models of leukemia and solid tumors, including sarcoma and pancreatic cancer. The in vitro screening performed in set of cancer cell lines led to selection of the following agents combined with ANN for testing efficacy in clinically relevant animal models: 1) Cytarabine, 2) Azacitidine, 3) Venetoclax, 4) Vincristine in AML model; 5) Ifosfamide in fibrosarcoma; 6) Irinotecan and 7) Gemcitabine in PATC53 pancreatic cancer model... ANN appears to be a versatile drug forming efficacious combinations with numerous mechanistically different anticancer agents. Our studies are working toward identifying new..."

Clinical • Fibrosarcoma • Hematological Malignancies • Leukemia • Oncology • Pancreatic Cancer • Sarcoma • Solid Tumor

Moleculin Biotech…announced that the 45th subject has been enrolled in its pivotal Phase 2B/3 MIRACLE trial evaluating Annamycin in combination with cytarabine (AnnAraC) for the treatment of adult subjects with relapsed or refractory acute myeloid leukemia (R/R AML) (GlobeNewswire) - "This milestone triggers the final phase of preparation for the trial’s interim 45 subject data unblinding, which remains on track for mid-2026 and represents a potentially defining inflection point for the Company."

Enrollment status • P2/3 data • Acute Myelogenous Leukemia

Expected Milestones for Annamycin AML Development Program (GlobeNewswire) - "Q1 2026: MIRACLE - 45th subject treated with unblinding of data for 45 subjects expected in mid-2026; Q3 2026: MIRACLE - 90 subjects treated and unblinding thereafter; 2H 2026: MIRACLE - Start of Part B; 2H 2026: Atlantic Health pancreatic cancer clinical trial begins; 2027: Begin recruitment of 3^rd line AML subjects; 2027: Begin pediatric AML clinical study; 2028: End recruitment of Part B; 2028: Primary efficacy data for MIRACLE 2^nd line subjects; 2028: Begin submission of a Rolling New Drug Application (NDA) for the treatment of R/R AML for accelerated approval on primary endpoint of CR from MIRACLE."

FDA filing • New trial • Trial status • Acute Myelogenous Leukemia • Pancreatic Cancer

Moleculin Biotech releases CEO update on leukemia trial (Investing.com) - "The study protocol calls for an interim unblinding after treatment of the first 45 patients is completed to evaluate safety and efficacy. Enrollment and dosing across clinical sites are progressing as planned, with completion of treatment for the initial 45 patients expected in the near term."

Trial status • Acute Myelogenous Leukemia

Moleculin Biotech…announced that it is approaching the first unblinding of data in its pivotal Phase 2B/3 'MIRACLE' study of Annamycin in combination with cytarabine 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)… (GlobeNewswire) - "Additionally, the Company reports a preliminary composite complete remission (CRc) rate of 40% in the MIRACLE trial’s first 30 subjects treated and with blinded, preliminary efficacy data. This CRc rate is comprised of a complete remission (CR) rate of 30% and complete remission with partial hematological recovery (CRh) of 10%....Of particular note is that roughly 35% of the subjects treated to date are relapsed or refractory (R/R) from a venetoclax regimen....The Company expects to reach the recruitment and treatment of the first 45 subjects in the first quarter of 2026."

Enrollment status • P2/3 data • Acute Myelogenous Leukemia

Moleculin Biotech…announced that the Japan Patent Office (JPO) has issued a notice of allowance for Patent Application No. 2021-577862 titled, 'Method of Reconstituting Liposomal Annamycin' (GlobeNewswire) - "A patent from the application is expected to be issued in the coming months. The allowed claims cover proprietary methods for reconstituting and preparing liposomal Annamycin from a preliposomal lyophilizate under controlled temperature conditions to achieve precise concentrations suitable for intravenous administration. These methods are designed to ensure consistent dosing, stability, and handling during preparation and delivery..."

Patent • Acute Myelogenous Leukemia

Moleculin Announces Completion of Treatment for the 45 Patients in Pivotal “MIRACLE” Phase 3 AML Trial on Pace for Q1 2026 (GlobeNewswire) - "The Company expects...to complete Part A of the MIRACLE trial with up to 90 patients within the first half of 2026."

Trial status • Acute Myelogenous Leukemia

Moleculin Biotech…announced it has entered into a research and material transfer agreement with CIC biomaGUNE…for investigator-initiated preclinical research evaluating Annamycin for the treatment of glioblastoma multiforme (GBM), a form of brain cancer (GlobeNewswire) - "Under the terms of the agreement Moleculin will supply Annamycin and Jesús Ruiz-Cabello, Principal Investigator at CIC biomaGUNE will conduct the planned preclinical research. The studies covered under this agreement will evaluate tumor progression following intra-arterial delivery of liposomal Annamycin (L-Annamycin) and Free-Annamycin as compared to Doxil and Free-doxorubicin in mouse models."

Licensing / partnership • Preclinical • Glioblastoma

Annamycin, a Non-Cardiotoxic Anthracycline Demonstrates Unique Organotropism and Activity Against Ara-C and Venetoclax Resistant AML (ASH 2024) - P1/2 | "This hurdle inspired us to develop novel highly efficacious agents with improved safety profiles, such as Annamycin (ANN), a doxorubicin (DOX) analog. Interestingly, rechallenging these animals with AML-Turq-2 cells resulted in extended survival compared to naïve mice, suggesting immune-memory inducing properties of L-ANN therapy and warranting further examination.Conclusion : This study confirms that L-ANN effectively targets both Ara-C and VEN resistant AML cell lines and heavily pretreated Relapse/Refractory primary AML patients in vitro. Furthermore, lack of apparent cardiotoxicity, improved organotropism, synergy with Ara-C, and possible immune-memory reinforcing properties contributes to the favorable performance of L-ANN in clinical settings (NCT03388749; NCT05319587)."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • Sarcoma • Solid Tumor • FLT3 • TP53

Preliminary Results from a Phase 1/2 Study of Liposomal Annamycin (L-ANN) in Combination with Cytarabin for the Treatment of Patients with Acute Myeloid Leukemia (AML) That Is Refractory to or Relapsed (r/r) after Induction Therapy (ASH 2023) - P1/2 | "The design of the novel liposomal L-annamycin (L-ANN) was the replacement of a basic amine at the C-3' position with a hydroxy group, which was shown to significantly reduce cardiotoxicity when compared with doxorubicin...All patients will also receive cytarabine 2... Initial data suggested that L-ANN is safe and active in heavily pretreated r/r AML patients with no signs of cardiotoxicity. This study is currently being conducted in Europe across 5 sites in Poland and 3 in Italy. Results from the Phase 2 portion of this study will be presented at the meeting."

Clinical • Combination therapy • P1/2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • ABCB1

Moleculin Reports 60% of First 45 Subjects in Pivotal “MIRACLE” Phase 3 AML Trial Consented (GlobeNewswire) - "The Company expects to reach the recruitment and treatment of the first 45 subjects in the first quarter of 2026 with unblinding thereafter, in addition to the second unblinding, which is expected in the first half of 2026....The currently enrolled subjects are from sites across five countries, providing a diverse base of subjects. With the upcoming holidays in addition to unexpected bed shortages at certain EU sites, the Company expects treatment of enrolled subjects to slow, pushing the completion of the first 45 subjects into 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."

P2/3 data • Trial status • Acute Myelogenous Leukemia

Moleculin Biotech…provided an enrollment update with 60% of the target number of subjects for the first planned interim unblinding of data having consented to its pivotal Phase 2B/3 'MIRACLE' study of Annamycin in combination with cytarabine for the treatment of adult patients with…R/R AML (GlobeNewswire) - "The targeted number for the first unblinding of data is 45 subjects. Additional subjects continued to be identified by site investigators. This update is as of November 4, 2025, as identification and recruitment are ongoing....The Company expects to reach the recruitment and treatment of the first 45 subjects in the first quarter of 2026 with unblinding thereafter, in addition to the second unblinding, which is expected in the first half of 2026."

Trial status • Acute Myelogenous Leukemia