Huaan: Breakthrough in small nucleic acid delivery technology and extension of indications suggests continued focus on the siRNA field.

Huaan releases research report stating that breakthroughs in small nucleic acid technology and indications have been made, extending from rare diseases and cardiovascular diseases to major metabolic areas such as weight loss, as well as extending into the CNS and even tumor fields. Overseas products are entering the commercial validation stage, with domestic and foreign industry trends resonating. Domestic companies are expected to benefit from the capital market layout, thereby accelerating technological updates and pipeline progress. Continued attention is recommended for investment opportunities in the small nucleic acid field, with a suggestion to continue monitoring the siRNA track. Key points from Huaan: Landmark events Overseas, multinational pharmaceutical company Novartis is making a big move in small nucleic acids by 2025, with 3 BD transactions in one year and a $12 billion acquisition case that shook the industry. Its listed product Inclisiran achieved $555 million in sales in H1 2025 (up 66% year-on-year). Novartis' Schweizerhalle site in Switzerland recently received an $80 million investment to expand siRNA production. Small nucleic acid company Arrowhead's Plozasirna has entered commercialization, with positive clinical data announced for ARO-INHBE and ARO-ALK7. Wave Life Science's INHBE siRNA muscle-building and fat-reducing drug WVE-007 has shown excellent data, making siRNA a new direction for weight loss molecules after GLP-1. Domestically, Boai Pharmaceutical's $5 billion total out licensing to Novartis broke the record for non-tumor field BD transaction amount. Novartis' siRNA drug Inclisiran has been officially included in China's 2025 medical insurance directory, marking small nucleic acids as a technology track with both commercialization and BD-driven logic. Technological iteration and application expansion In terms of delivery technology, there have been breakthroughs in liver-targeted delivery to extrahepatic targets such as cross-brain delivery and lung-targeted delivery. In terms of indications, the extension from rare diseases and cardiovascular diseases to major metabolic areas such as weight loss, as well as extending into the CNS and tumor fields, has been achieved. Drug targeting layout In terms of mature targets, following the approval of Inclisiran (PCSK9 proprotein convertase subtilisin/kexin type 9) in 2020, which opened up the application of siRNA in the cardiovascular field, ApoC3 (apolipoprotein C3), Lpa (lipoprotein a), and ANGPTL3 (angiopoietin-like 3) related to lipid-lowering therapy have become standard targets for pharmaceutical companies. AGT (angiotensinogen), FXI (coagulation factor XI) deepen the application in hypertension and thrombotic diseases. In terms of emerging targets, preliminary data has been disclosed for INHBE (inhibin subunit beta E), ALK7 in obesity, PNPLA3 in NASH development, C3 in complement-related diseases, and an increase in the IgA nephropathy pipeline, continuously expanding the application range and prospects of siRNA. Risk warnings: risks of new drug clinical development failures, BD negotiation progress falling short of expectations, and risks of technological development falling short of expectations.