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2B3-101 is a brain-targeted formulation of doxorubicin, in which the drug is encapsulated in a PEGylated liposome and further functionalized with glutathione to enhance delivery across the blood-brain barrier. This design aims to increase the concentration of doxorubicin within brain tumors while reducing systemic toxicity. The drug has shown increased delivery of doxorubicin to the brain and improved anti-tumor activity in preclinical models compared to non-targeted formulations. Clinical trials have demonstrated that 2B3-101 is well tolerated and shows preliminary evidence of anti-tumor activity in patients with recurrent high-grade gliomas (HGG) and breast cancer brain metastases (BCBM). The most common adverse events are neutropenia, palmar-plantar erythrodysesthesia (PPE), fatigue, stomatitis, and infusion reactions; no neurotoxicity or cardiotoxicity was observed. Development has focused on multiple brain cancer indications including glioma, meningeal carcinomatosis, breast cancer brain metastases, and other solid tumor CNS metastases[1][4][5][6][8].
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