China-based Lepu Biotechnology Co., Ltd (HKG: 2157) has announced that it has received Breakthrough Therapy Designation (BTD) from the Center for Drug Evaluation for its core product MRG003 in recurrent/metastatic nasopharyngeal carcinoma (R/M NPC). The epidermal growth factor receptor (EGFR) targeted antibody drug conjugate (ADC) had previously obtained Orphan Drug Designation (ODD) from the US FDA for the same indication.
Global Impact of Nasopharyngeal Carcinoma
China accounts for nearly half of the global cases of nasopharyngeal carcinoma, with approximately 60,000 new cases each year. The prognosis for patients with R/M NPC is very poor, with a median overall survival (OS) of about 20 months. Systemic chemotherapy is currently the main treatment method for R/M NPC patients. However, after the failure of platinum-containing treatment, alternative treatment options and therapeutic effects for second-line or third-line treatments are limited.
MRG003: Mechanism and Clinical Progress
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, where the cytotoxic small molecule MMAE is released under the action of pE/lysosome. MMAE targets and inhibits tubulin polymerization, leading to tumor cell death. MRG003 has shown excellent data in a Phase II exploratory study in NPC, completing patient enrollment in March this year.
Company Overview and Pipeline
Lepu Biotechnology is a specialist in tumor immunotherapy with a full industrial platform for target discovery, drug preparation, development, and manufacturing. The company’s product pipeline covers immunotherapy, ADC targeted therapy, and oncolytic virus drugs. It includes one commercialized drug, seven drug candidates at the clinical stage, and two combination therapies in the clinics.
Future Outlook
The receipt of Breakthrough Therapy Designation for MRG003 underscores Lepu Biotechnology’s commitment to advancing innovative treatments for nasopharyngeal carcinoma. This designation is expected to accelerate the development and regulatory review process, bringing hope to patients with this challenging disease.-Fineline Info & Tech
