CSPC PHARMA (01093) announced that its self-developed Class 1 chemical new drug SYH2070 injection, a double-stranded small interfering RNA (siRNA) drug, has received approval from the U.S. Food and Drug Administration (FDA) to conduct clinical trials in the United States. The product also obtained approval from China's National Medical Products Administration in September 2025 to initiate clinical trials in China.

The product is a siRNA drug that achieves liver-targeted delivery through N-acetylgalactosamine (GalNAc) conjugation, administered subcutaneously to target angiopoietin-like protein 3 (ANGPTL3), effectively reducing ANGPTL3 levels. Through sequence optimization and chemical modification strategies, the product achieves more durable gene silencing effects and is expected to become an ultra-long-acting siRNA drug for reducing ANGPTL3, suitable for treating hypertriglyceridemia or mixed hyperlipidemia, with potential to effectively reduce the risk of elevated residual cholesterol levels.

Preclinical studies demonstrate that the product outperforms similar siRNA products in both drug activity and pharmacodynamic persistence, showing differentiated advantages including durable drug efficacy, good safety profile, and high patient compliance, indicating significant clinical development value.