Small Nucleic Acid Therapeutics: Global Industry Achieves Multidimensional Breakthroughs, Chinese Innovative Biopharma Firms Demonstrate Global Competitiveness

Sep 28, 2025 18:50 CST Updated 18:50

Argo

RNAi Drug Developer

Text｜He Juying, Yuan Qinghui, Wang Zaicun, Liu Ruofei, Tang Ran

From a commercialization perspective, Amvuttra and Leqvio demonstrate blockbuster drug potential, with global leader Alnylam expected to achieve profitability this year. From an indication standpoint, 2025-2026 will be a critical validation period for indication breakthroughs. The weight loss indication is approaching its Proof of Concept (POC) moment, while Central Nervous System (CNS) indications are expected to begin exploration successively. Technologically, extracellular liver delivery is making gradual progress. Extracellular delivery methods such as TRiM and AOC are emerging. Del-zota, an AOC drug developed by Avidity, shows remarkable efficacy as the first AOC drug, with a Biologics License Application (BLA) anticipated by the end of 2025 and commercial launch expected in 2026. Among China’s small nucleic acid innovative pharmaceutical companies, in recent years, Argo, Ribo, and MNC have reached BD deals, showcasing the global competitiveness of China’s small nucleic acid sector.

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Industry Policy Risk: The risk of changes in research design requirements, price fluctuations, bulk procurement policy adjustments, and modifications in the scope and proportion of medical insurance reimbursement due to industry policy changes. Particularly, changes in centralized procurement and medical insurance payment policies significantly impact the industry's development expectations.

Risk of R&D Falling Short of Expectations: During the R&D process of new drugs and devices, there are risks associated with uncertain clinical enrollment progress, as well as uncertain efficacy and safety data outcomes.

Risk of Approval Delays: The risk of prolonged approval cycles due to factors such as the need for additional documentation and changes in the approval process.

Risk of Macroeconomic Volatility: A further slowdown in global economic growth may impact downstream demand. Additionally, risks related to international relations, climate change, inflation, as well as exchange rates and interest rates need to be considered.

Name of the securities research report: "Weekly Report on the Pharmaceutical Industry: Small Nucleic Acid Drugs: Global Industry Achieves Multi-dimensional Breakthroughs, Chinese Innovative Pharmaceutical Companies Demonstrate Global Competitiveness》

Public Release Date: September 28, 2025

Report Issuing Institution: CITIC Securities Co., Ltd.

Analyst of this report:

Ju Ying He SAC No.: S1440517050001

SFC No.: ASZ591

Yuan Qinghui SAC No.: S1440520030001

SFC No.: BPW879

Wang Zaicun SAC No.: S1440521070003

Liu Ruofei SAC No.: S1440519080003

SFC No.: BVX723

Tang Ran SAC No.: S1440524100001

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