China-based Lepu Biotechnology Co., Ltd (HKG: 2157) has announced that it has received orphan drug designation (ODD) status from the US FDA for its core product MRG003, an epidermal growth factor receptor targeted antibody drug conjugate (ADC) for the treatment of recurrent/metastatic nasopharyngeal carcinoma (R/M NPC).
Drug Mechanism and Development
MRG003 is a novel ADC drug that targets EGFR on the surface of tumor cells and delivers cytotoxic small molecules to these cells through its antibody component. Upon binding to EGFR, MRG003 triggers Fc segment-mediated immunotoxicity, such as antibody-dependent cell-mediated cytotoxicity (ADCC). The ADC-EGFR complex is then endocytosed into the cell, and the cytotoxic small molecule MMAE is released under the action of pE/lysosome. MMAE targets and inhibits tubulin polymerization in the cell, leading to tumor cell death.
Clinical Progress and Future Outlook
MRG003 has shown promising results in multiple exploratory studies across various indications. In a Phase II exploration study for nasopharyngeal carcinoma (NPC), which completed patient enrollment in March of this year, MRG003 demonstrated excellent data. The orphan drug designation from the FDA underscores the potential of MRG003 to address significant unmet needs in the treatment of R/M NPC.-Fineline Info & Tech
