Core Insights - The company has reported an increase in enrollment for the MIRACLE trial, reaching 78% of the target number of subjects for the first interim unblinding, up from 60% in November [1][2] - The trial aims to evaluate Annamycin in combination with cytarabine for treating adult patients with relapsed or refractory acute myeloid leukemia (AML) [1][9] - The first unblinding is expected to occur in the first quarter of 2026, with a target of 45 subjects [1][4] Enrollment and Trial Progress - As of December 3, 2025, the company has consented subjects from seven countries, indicating a diverse patient population [4] - The company anticipates completing treatment of the first 45 subjects by the first quarter of 2026 and aims to finish Part A of the trial with up to 90 patients in the first half of 2026 [4][2] - The trial design allows for unblinding of preliminary efficacy data at 45 subjects, with a focus on complete remission as a primary endpoint [3] Annamycin's Potential and Designation - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory AML, as well as Orphan Drug Designation from the EMA [6][7] - The drug is designed to avoid multidrug resistance mechanisms and lacks the cardiotoxicity associated with current anthracyclines [8] - The company believes Annamycin could provide a safer and more effective treatment option for AML patients [2]

Core Viewpoint - Moleculin Biotech, Inc. has entered into a research agreement with CIC biomaGUNE to evaluate Annamycin for treating glioblastoma multiforme, a challenging brain cancer with significant unmet medical needs [2][4]. Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viral infections [2][6]. - The company's lead program, Annamycin, is a next-generation anthracycline designed to avoid multidrug resistance and cardiotoxicity associated with existing treatments [6]. Research Agreement Details - Under the agreement, Moleculin will supply Annamycin, and CIC biomaGUNE will conduct preclinical studies comparing the effects of intra-arterial delivery of liposomal Annamycin and Free-Annamycin against Doxil and Free-doxorubicin in mouse models [3][4]. - The research aims to explore Annamycin's potential to address drug delivery challenges and resistance in central nervous system tumors [4]. Glioblastoma Multiforme Insights - Glioblastoma is the most aggressive primary brain tumor, accounting for 54% of all gliomas and 16% of all primary brain tumors, with a median survival of only 15 months [4]. - The average annual age-adjusted incidence rate of glioblastoma in the U.S. is 3.19 per 100,000 persons, and survival rates have not significantly improved in the last three decades [4]. Annamycin's Regulatory Status - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases [5]. - The drug also holds Orphan Drug Designation from the EMA for the same indications [5]. Clinical Trials - The company has initiated the MIRACLE trial, a Phase 3 study evaluating Annamycin in combination with cytarabine for relapsed or refractory AML [7]. - Following a successful Phase 1B/2 study, the company believes it has reduced risks associated with the development pathway for Annamycin's approval [7].

Core Insights - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat tumors and viruses [4] - The company will host a live video webcast featuring CEO Walter Klemp on December 2, 2025, to provide a corporate overview and business outlook [1][2] Company Overview - Moleculin Biotech is advancing a pipeline of therapeutic candidates, with its lead program Annamycin targeting relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases [4] - Annamycin is designed to avoid multidrug resistance mechanisms and lacks the cardiotoxicity associated with current anthracyclines [4] Clinical Trials - The company has initiated the MIRACLE Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for the treatment of relapsed or refractory AML [5] - Following a successful Phase 1B/2 study, the company believes it has de-risked the development pathway for Annamycin's potential approval [5] Additional Developments - Moleculin is also developing WP1066, an immune/transcription modulator targeting brain tumors and other cancers, and a portfolio of antimetabolites including WP1122 for treating pathogenic viruses [6]

Core Viewpoint - Moleculin Biotech, Inc. announced a 1-for-25 reverse stock split of its common stock, effective December 1, 2025, to consolidate shares and potentially enhance stock performance [1][2]. Group 1: Reverse Stock Split Details - The reverse stock split will consolidate every 25 shares into one share, maintaining the nominal par value of $0.001 per share [2]. - The number of outstanding shares will decrease from approximately 51.7 million to about 2.1 million, while the authorized shares will remain at 500 million [3]. Group 2: Company Overview - Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company focused on developing therapies for hard-to-treat tumors and viruses [4]. - The lead program, Annamycin, is designed to treat relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma (STS) lung metastases [4]. Group 3: Clinical Trials and Pipeline - The company has initiated the MIRACLE Trial (MB-108), a pivotal Phase 3 trial evaluating Annamycin in combination with cytarabine for AML treatment [5]. - Moleculin is also developing WP1066, an immune/transcription modulator targeting various cancers, and has a portfolio of antimetabolites, including WP1122 for treating pathogenic viruses [6].

Clinical Trials and Drug Development - The company is conducting a pivotal Phase 3 trial for Annamycin in combination with Cytarabine for the treatment of relapsed/refractory acute myeloid leukemia (AML), with an interim unblinding expected after recruiting the first 45 subjects[85]. - As of November 4, 2025, 60% of the targeted 45 subjects have consented to participate in the MIRACLE trial, with treatment of the last subject expected in Q1 2026[98]. - Annamycin has been administered to 84 subjects across five clinical trials with no evidence of cardiotoxicity, including subjects treated above the FDA's lifetime maximum anthracycline limit of 550 mg/m2[93]. - The company has a total of fourteen clinical trials, with ongoing studies for Annamycin and other drug candidates, including a Phase 2B/3 trial for AML and a Phase 1B/2 trial for soft tissue sarcoma[96]. - The MIRACLE trial is designed as an adaptive study with a lead-in Part A to determine the optimum dose of Annamycin, followed by Part B for further evaluation[97]. - The company has received ten communications from the FDA regarding the MIRACLE trial, focusing on safety and monitoring, with no significant changes to the trial design or dosing of Annamycin[101]. - Annamycin is designed to be non-cardiotoxic and has shown no cardiotoxicity in clinical trials, which is a significant advantage over currently prescribed anthracyclines[89]. - The MIRACLE trial protocol was amended to allow dosing above the lifetime maximum allowable dose for anthracyclines, with FDA feedback reducing the size of Part B of the Phase 3 trial to 222 subjects, a 10% reduction[102]. - The European Medicines Agency approved the Clinical Trial Application for the MIRACLE trial in nine EU countries, contingent on presenting results of nonclinical GLP studies before initiating Part B[104]. - In the Phase 1B/2 clinical trial of AnnAraC for AML, 36% of subjects achieved complete remission, with a median durability of 10 months for those in remission[106]. - The FDA approved a single pediatric study plan for Annamycin in combination with Cytarabine for children with relapsed/refractory AML, expected to commence in the second half of 2027[108]. - The MB-107 trial for Annamycin in STS Lung Mets showed a median overall survival of 13.5 months, compared to 8-12 months for standard treatments[110]. - Annamycin is being evaluated for pancreatic cancer in a grant-funded research project at UNC, with a Phase 1B/2 study planned to start in 2026[113][115]. Financial Performance - Research and development (R&D) expenses decreased to $3.7 million in Q3 2025 from $4.9 million in Q3 2024, a reduction of approximately 24.5%[134]. - General and administrative expenses slightly decreased to $2.1 million in Q3 2025 from $2.2 million in Q3 2024, a decrease of about 4.6%[135]. - The net loss for Q3 2025 was $25.4 million, compared to a net loss of $7.0 million in Q3 2024, indicating a significant increase in losses[133]. - The company recorded a net gain of $1.6 million from the change in fair value of warrant liability in Q3 2025, compared to no gain in Q3 2024[136]. - Cash used in operating activities was $17.4 million for the nine months ended September 30, 2025, a decrease of $1.4 million from $18.8 million in the prior year[145]. - The company raised gross proceeds of $6.0 million from the exercise of Series E warrants in August 2025[146]. - The company reported a net increase in cash and cash equivalents of $2.4 million for the nine months ended September 30, 2025, compared to a decrease of $14.1 million in the prior year[144]. - The company must seek additional funds of approximately $7 million to support operations into the second quarter of 2026[152]. Intellectual Property and Collaborations - The Australian Patent Office granted a patent for preliposomal Annamycin lyophilizate, extending the patent term until June 2040[122]. - The World Health Organization approved "naxtarubicin" as the international non-proprietary name for Annamycin, facilitating its recognition in clinical documents[125]. - The US Patent and Trademark Office granted two additional patents covering methods of making liposomal Annamycin, extending the patent term until June 2040[127]. - The company received a Notice of Intent to Grant for a European patent for "Method of Reconstituting Liposomal Annamycin," enhancing global exclusivity with a base patent term extending until June 2040[128]. - The company has additional patent applications related to Annamycin pending in major jurisdictions worldwide[128]. - The company has established collaborations with the University of Texas MD Anderson Cancer Center for research and development of its drug candidates[84]. Regulatory and Compliance - The company received a subpoena from the SEC in March 2022 regarding information related to its COVID-19 drug candidate, with expenses of approximately $0.1 million and $0.2 million for the three and nine months ended September 30, 2024, respectively[153]. - There have been no material changes to the company's critical accounting policies and estimates from those disclosed in the Form 10-K for the year ended December 31, 2024[154]. - The company is classified as a smaller reporting company, and thus, quantitative and qualitative disclosures about market risks are not applicable[156]. Strategic Focus and Growth - The company plans to expand treated indications with Annamycin in 2026, indicating a focus on growth and market expansion[86]. - The company aims to attract and retain key personnel to effectively manage its business and support future growth[84].

Core Insights - Moleculin Biotech, Inc. is progressing with its pivotal Phase 2B/3 "MIRACLE" study of Annamycin for treating adult patients with relapsed or refractory acute myeloid leukemia (AML), with 60% of the target enrollment completed as of November 4, 2025 [1][2][4] - The company expects to complete treatment for the first 45 subjects by Q1 2026, with initial unblinded data anticipated shortly thereafter [1][4][5] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for AML treatment, and it is designed to avoid common cardiotoxicity associated with existing anthracyclines [7][9] Enrollment and Study Progress - The MIRACLE study is a global, multi-center, randomized, double-blind, placebo-controlled trial, combining data from its Phase 2B and Phase 3 portions to measure primary efficacy endpoints [3][10] - The first unblinding will involve 30 subjects treated with Annamycin in combination with HiDAC and 15 subjects treated with HiDAC plus placebo [3] - Recruitment is ongoing, with subjects enrolled from five countries, although some European sites are experiencing bed shortages affecting enrollment [5][2] Future Expectations - The company anticipates a second unblinding in the first half of 2026, following the initial unblinding of data [4] - The accelerated timeline for recruitment is attributed to positive feedback from potential investigators [4] - Annamycin is positioned as a potentially safer and more effective treatment option for AML, addressing a significant gap in current therapies [2][4]

Core Insights - Moleculin Biotech, Inc. has entered into a research agreement with the University of North Carolina at Chapel Hill to conduct preclinical studies on Annamycin for pancreatic cancer treatment [1][2][3] - Annamycin is being evaluated for its ability to enhance tumor delivery compared to existing treatments like Doxil and Free-doxorubicin [2] - The company aims to leverage Annamycin's high affinity for the pancreas and its correlation with poor survival rates in pancreatic cancer patients to expand its clinical applications [3] Company Overview - Moleculin Biotech is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viral infections [1][6] - Annamycin is a next-generation anthracycline designed to avoid multidrug resistance and cardiotoxicity associated with traditional anthracyclines [6][8] - The company is currently conducting a pivotal Phase 2B/3 trial named MIRACLE, evaluating Annamycin in combination with cytarabine for relapsed or refractory acute myeloid leukemia (AML) [4][8] Research and Development - The preclinical research at UNC will assess the effectiveness of Annamycin in conjunction with novel agents to improve treatment outcomes for pancreatic cancer [1][2] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for treating relapsed or refractory AML and soft tissue sarcoma lung metastases [3][6] - The company is also developing WP1066, an immune/transcription modulator targeting various cancers, including pancreatic cancer [9]

Core Viewpoint - Moleculin Biotech, Inc. is advancing its lead program Annamycin, a non-cardiotoxic anthracycline, through a pivotal Phase 3 trial for treating relapsed or refractory acute myeloid leukemia (AML) [1][4] Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viral infections [1][3] - The company’s lead candidate, Annamycin, is designed to avoid multidrug resistance mechanisms and lacks the cardiotoxicity associated with current anthracyclines [3] Clinical Trials - The ongoing MIRACLE trial is a Phase 2B/3, multi-center, randomized, double-blind, placebo-controlled study evaluating Annamycin in combination with cytarabine for adult patients with R/R AML [1][4] - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for R/R AML and STS lung metastases, as well as Orphan Drug Designation from the EMA [2] Upcoming Presentation - Dr. Paul Waymack, Senior Chief Medical Officer, will present details of the MIRACLE trial at the 14th Annual Acute Leukemia Meeting on October 30, 2025 [1]

Core Insights - Moleculin Biotech, Inc. has been granted an Australian patent for preliposomal Annamycin lyophilizates, enhancing its global intellectual property portfolio with a base patent term extending until June 2040 [1][2] - Annamycin is being developed as a non-cardiotoxic anthracycline for treating acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases, with potential applications for various other cancers [2][4] - The company has initiated the MIRACLE trial, a Phase 3 study evaluating Annamycin in combination with cytarabine for relapsed or refractory AML, following a successful Phase 1B/2 study [5] Patent and Intellectual Property - The newly granted Australian patent covers preliposomal Annamycin with improved stability and high purity, contributing to the company's competitive position in oncology [1][2] - The patent family includes expected Canadian patents and previously issued U.S. and European patents, with additional applications pending in major jurisdictions worldwide [2] Drug Development and Clinical Trials - Annamycin has received Fast Track Status and Orphan Drug Designation from the FDA for relapsed or refractory AML and STS lung metastases, as well as Orphan Drug Designation from the EMA [3] - The MIRACLE trial aims to evaluate the efficacy of Annamycin in combination with cytarabine, which is expected to de-risk the development pathway towards potential approval [5] Additional Pipeline Candidates - The company is also developing WP1066, an immune/transcription modulator targeting various cancers, and WP1122, an antimetabolite for treating pathogenic viruses and certain cancer indications [6]

Core Insights - Moleculin Biotech, Inc. is collaborating with Atlantic Health to conduct a Phase 1B/2 study of Annamycin for third-line treatment of advanced pancreatic cancer, addressing a significant unmet medical need in this high-mortality disease [1][3][2] Company Overview - Moleculin Biotech, Inc. is a late-stage pharmaceutical company focused on developing drug candidates for hard-to-treat cancers and viral infections, with Annamycin as its lead program [11] - Annamycin is a next-generation anthracycline designed to avoid multidrug resistance and cardiotoxicity, currently in development for relapsed or refractory acute myeloid leukemia (AML) and soft tissue sarcoma lung metastases [11][4] Study Details - The Phase 1B/2 study will evaluate Annamycin's efficacy in advanced pancreatic cancer, with an estimated cost of approximately $1 million for the trial from 2026 to 2030 [3][1] - Atlantic Health will conduct the study, while Moleculin will supply Annamycin and manage the Investigational New Drug Application (IND) with the FDA [3][1] Preclinical Data - Recent preclinical studies suggest that Annamycin targets critical factors in pancreatic cancer, showing a high level of activity against the disease and associated liver metastases [2][3] - The upregulation of topoisomerase II, the primary target of Annamycin, correlates with poor survival in pancreatic cancer patients, validating the target's relevance [3][2] Industry Context - Pancreatic cancer is the cancer with the highest mortality rate globally and has limited treatment options, particularly in the third-line setting where no approved standard of care exists [2][3] - The collaboration aims to drive innovation in pancreatic cancer treatment, addressing a significant gap in available therapies for patients who have progressed beyond first- and second-line treatments [3][2]