naxtarubicin (L-ANN) / Moleculin - LARVOL DELTA

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

Moleculin Biotech…announced it has entered into research and material transfer agreement with the University of North Carolina at Chapel Hill (UNC) for investigator-initiated preclinical research evaluating Annamycin for the treatment of pancreatic cancer (GlobeNewswire) - "Under the terms of the agreement Moleculin will supply Annamycin and...will conduct the planned preclinical research as part of a series of funded grants. 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."

Licensing / partnership • Preclinical • Pancreatic Cancer

Moleculin to Highlight its Ongoing Phase 3 Acute Myeloid Leukemia “MIRACLE” Clinical Trial at the 14th Annual Acute Leukemia Meeting (GlobeNewswire) - "As part of the presentation, Dr. Waymack will discuss the Company’s ongoing pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design study of Annamycin in combination with cytarabine...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)."

Trial status • Acute Myelogenous Leukemia

Moleculin Expands Global IP Portfolio with New Australian Patent for Annamycin (GlobeNewswire) - "...Patent No. 2024203598 titled, 'PREPARATION OF PRELIPOSOMAL ANNAMYCIN LYOPHILIZATE,' with claims covering certain preliposomal Annamycin lyophilizates with improved stability and high purity, with a base patent term currently extending until June 2040, subject to extension to account for time required to fulfill requirements for regulatory approval."

Patent • Acute Myelogenous Leukemia • Soft Tissue Sarcoma

Moleculin Biotech…announced it 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 (GlobeNewswire) - "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 U.S. Food and Drug Administration (FDA), and Dr. Alistar of Atlantic Health will be responsible for conducting the Phase 1B/2 study."

New P1/2 trial • Pancreatic Cancer

Study of Liposomal Annamycin in Combination With Cytarabine for the Treatment of Subjects With Acute Myeloid Leukemia (AML) (clinicaltrials.gov) - P1/2 | N=22 | Completed | Sponsor: Moleculin Biotech, Inc. | Active, not recruiting ➔ Completed | N=63 ➔ 22

Enrollment change • Trial completion • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology

Moleculin Biotech…announced that the Canadian Intellectual Property Office (CIPO) has issued a notice of allowance for Patent Application No. 3,142,510 titled, “PREPARATION OF PRELIPOSOMAL ANNAMYCIN LYOPHILIZATE” (GlobeNewswire) - "A patent from the application is expected to be issued in the coming months. When issued, the patent claims will cover methods of making a preliposomal Annamycin lyophilizate with improved stability and high purity, with a base patent term currently extending until June 2040, subject to extension to account for time required to fulfill requirements for regulatory approval."

Patent • Acute Myelogenous Leukemia • Soft Tissue Sarcoma

Moleculin Accelerates Recruitment in Phase 3 Acute Myeloid Leukemia “MIRACLE” Clinical Trial (GlobeNewswire) - "European expansion in September across Georgia, Italy, Lithuania, Poland, Romania, Spain, and US...To advance this trial to recruiting the 45th subject in Q4 2025 for its first unblinding for early data insights into the trial’s efficacy and safety endpoints, the Company has the following expectations for September: Global Expansion: Expanding recruiting beyond Ukraine to include Spain, Georgia, Poland, Romania, Italy, Lithuania, and the US; Site Expansion: Adding 8 new active sites by the end of September, increasing the total to 20 sites recruiting; Recruitment Goals: Treating, enrolling, or screening a total of 20 subjects by the end of September."

Trial status • Acute Myelogenous Leukemia

Moleculin Doses First EU Patient in Phase 3 Pivotal MIRACLE Trial for Treatment of Acute Myeloid Leukemia (GlobeNewswire) - "The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026."

Trial status • Acute Myelogenous Leukemia

Enantioselective Total Syntheses of Sannamycins A and B. (PubMed, J Am Chem Soc) - "Herein we report enantioselective syntheses of 2-deoxyfortamine-type aminoglycosides sannamycins A and B. The described strategy involves an enantioselective dearomative hydroamination, rapid stereo- and chemoselective introduction of heteroatom functionalities, and a unique skeletal rearrangement to forge the aminocyclitol core. The carbohydrate fragment was elaborated from Cyrene, a readily available enantioenriched starting material, and was used in a stereoselective glycosylation reaction to deliver natural products in 14 and 17 steps, respectively, from benzene."

Journal

Moleculin Issues New Positive AML Overall Survival Data (Moleculin Press Release) - "Database lock for the trial is expected by the end of September, with the final clinical study report (CSR) projected to be published in early Q1 2026....Median OS for Complete Remissions (CR): 15 months (n=8) with 4 subjects alive at study close; Median OS for the intent to treat (ITT) Population (1L-7L): 9 months (n=22; 13 subjects experienced an event, 9 subjects censored); Median OS for 2L Efficacy Evaluable Population: 12 months (n=9)."

P1/2 data • Acute Myelogenous Leukemia

Moleculin Biotech, Inc. Unveils Promising Preclinical Data of Annamycin in Liver Cancer Treatment (GlobeNewswire) - "Moleculin Biotech...announced the presentation of encouraging preclinical data for its lead drug candidate, Annamycin, also known by its non-proprietary name of naxtarubicin, 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...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."

Preclinical • Colorectal Cancer • Hepatocellular Cancer • Pancreatic Ductal Adenocarcinoma

Moleculin Receives Notice of Intent to Grant New European Patent for Annamycin (GlobeNewswire) - "Moleculin Biotech...announced it has received a Notice of Intent to Grant for the European patent application titled, 'PREPARATION OF PRELIPOSOMAL ANNAMYCIN LYOPHILIZATE'. Such grant should solidify the Company’s European Union exclusivity of Annamycin, also known by its non-proprietary name of naxtarubicin, with the potential to become the first non-cardiotoxic anthracycline....The grant is subject to payment of fees and completion of final amendments and formalities. When issued, the patent claims will cover methods of making a preliposomal Annamycin lyophilizate with improved stability and high purity, with a base patent term currently extending until into 2040, subject to extension to account for time required to fulfill requirements for regulatory approval. Moleculin’s novel drug candidate is...currently being developed for the treatment of acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases (STS lung mets)."

Patent • Acute Myelogenous Leukemia • Soft Tissue Sarcoma

Moleculin Expands Phase 3 MIRACLE Clinical Trial into the Country of Georgia, Enrollment Update and Future Plans (GlobeNewswire) - "Enrollment in Part A of MIRACLE reaches seven subjects treated and one additional subject in screening; 16 additional clinical sites in Europe and the US expected to begin recruitment by the end of August; building to an expected 30 plus sites for Part A of MIRACLE...Moleculin...announced that the Regulation Agency for Medical and Pharmaceutical Activities (RAMPA) in the country of Georgia has approved its Clinical Trial Application (CTA) to conduct its pivotal Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled, adaptive design 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....The Company expects to reach the first unblinding (45 subjects) in the second half of 2025, in addition to the second unblinding, which is expected in the first half of 2026."

Trial status • Acute Myelogenous Leukemia

FDA agrees to Moleculin’s pediatric study plan for cancer drug (Investing.com) - "The U.S. Food and Drug Administration has agreed to Moleculin Biotech, Inc.’s...proposal for a single pediatric approval study of its cancer drug Annamycin in combination with Cytarabine for children with relapsed/refractory acute myeloid leukemia (R/R AML). In its response to Moleculin’s Initial Pediatric Study Plan, the FDA recommended including patients as young as 6 months old, younger than the company’s proposed minimum age of 2 years....Moleculin is currently conducting the MIRACLE trial, a pivotal Phase 3 study evaluating the Annamycin-Cytarabine combination (AnnAraC) in adult patients with R/R AML. Initial data from this trial is expected in the second half of 2025. The company plans to submit a revised pediatric study plan to the FDA later this quarter and anticipates beginning the pediatric clinical study in the second half of 2027."

FDA event • New trial • P3 data • Acute Myelogenous Leukemia

Expected Milestones for Annamycin AML Development Program (GlobeNewswire) - "1Q – 3Q 2025 – Update on MIRACLE trial site selection/approvals by countries; 2025 – Recruitment update for MIRACLE trial; 2H 2025 – Data readout (n=45) unblinded efficacy/safety review; 2H 2025 – 2026 – Impact of data readout (n=45) on regulatory pathway; Recruitment update; 1H 2026 – Interim efficacy and safety data (n=~75-90) unblinded and Optimum Dose set for MIRACLE trial; 2027 – Begin enrollment of 3rd line subjects in MIRACLE2; 2027 – Enrollment ends in 2nd line subjects; 2027 – Begin enrollment in pediatric AML trial; 2028 – Primary efficacy data for 2nd line subjects in MIRACLE; 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; 2028 – Primary efficacy data for 2nd line subjects; 2028 – Rolling NDA submission begins."

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

Moleculin Participates in Virtual Investor 'What This Means' Segment (GlobeNewswire) - "Globally recognized Oncology Key Opinion Leader, Brian Andrew Van Tine, MD, PhD, discusses the positive topline results from the U.S. Phase 1B/2 clinical trial evaluating Annamycin for the treatment of Soft Tissue Sarcoma Lung Metastases (MB-107)."

P1/2 data • Lung Cancer • Soft Tissue Sarcoma

Moleculin Releases On-Demand KOL Webcast to Discuss Data from its Phase 1B/2 Study of Annamycin for the Treatment of Soft Tissue Sarcoma (STS) Lung Metastases (MB-107) (GlobeNewswire) - "Moleculin Biotech, Inc...announced the release of its Soft Tissue Sarcoma (STS) Lung Mets KOL Webcast discussing the final data from its US Phase 1B/2 clinical trial evaluating Annamycin as monotherapy for the treatment of soft tissue sarcoma lung metastases (MB-107)."

P1/2 data • Soft Tissue Sarcoma

Moleculin Reports Positive Topline Efficacy Results from U.S. Phase 1B/2 Clinical Trial Evaluating Annamycin for the Treatment of Soft Tissue Sarcoma Lung Metastases (MB-107) (GlobeNewswire) - P1b/2 | N=36 | NCT04887298 | Sponsor: Moleculin Biotech, Inc. | "Clinical Benefit Rate ('CBR') was 59.4% (n=32), comprised of 18 subjects with stable disease and 1 subject with a partial response (no subjects achieved Complete Response or CR). Progression Free Survival ('PFS') & Overall Survival ('OS'): (i) Dose and regimen optimized subjects demonstrated PFS of ~4 months and OS of ~20 months; (ii) Overall (N=36) Median PFS was 63 days, with a 95% Confidence Interval ('CI') between 43 and 105 days; (iii) Median OS was 411 days, with a 95% CI between 241 and 583 days. In Phase 2 (N=17) at 330 mg/m2: (i) Median PFS was 105 days and OS for all-comers (median 6 prior therapies) of 13.5 months exceeded typical results for 2nd line monotherapies (8-12 months); (ii) OS/PFS was higher (19.9 months/127 days) for subjects with fewer prior therapies (≤2) (n=7) and receiving doses of Annamycin ≤ 330 mg/m2 (the RP2D)."

P1/2 data • Soft Tissue Sarcoma

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