
Core Insights - Moleculin Biotech, Inc. announced promising preclinical data for its lead drug candidate, Annamycin, showing significant efficacy against various liver cancers, including hepatocellular carcinoma, colorectal liver metastases, and pancreatic ductal adenocarcinoma liver metastases [1][4][5] - Annamycin is currently in late-stage clinical development for the treatment of acute myeloid leukemia (AML) in combination with cytarabine, with preliminary data expected in the second half of 2025 [1][7] Company Overview - Moleculin Biotech, Inc. is a Phase 3 clinical stage pharmaceutical company focused on developing therapeutic candidates for hard-to-treat tumors and viruses, with Annamycin being a next-generation anthracycline designed to avoid multidrug resistance and cardiotoxicity [6][8] - The company is also developing WP1066, an immune/transcription modulator targeting brain tumors and other cancers, along with a portfolio of antimetabolites for potential treatment of pathogenic viruses and certain cancer indications [8] Preclinical Findings - Annamycin demonstrated targeted accumulation in organs, particularly the liver, spleen, lungs, and pancreas, leading to higher concentrations compared to doxorubicin, which is critical for treating liver-localized tumors [5] - In orthotopic hepatocellular carcinoma models, Annamycin showed excellent anti-tumor activity, significantly reducing tumor progression and improving survival rates [5] - The drug also exhibited significant efficacy in inhibiting metastatic growth in colorectal liver metastasis models and showed promising results in managing advanced pancreatic cancer with liver involvement [5] Safety Profile - Annamycin has a favorable safety profile, showing low or no cardiotoxicity, which is a significant advantage over traditional anthracyclines like doxorubicin, often limited by dose-dependent cardiac side effects [5]