China Post Securities: siRNA Therapeutics Demonstrate Distinct Mechanistic Advantages with Delivery as the Core Technology

Intelligent Finance APP learned that China Postal Securities issued a research report stating that siRNA drugs can precisely silence pathogenic proteins with significant advantages, and the delivery system is the core technology. Overseas giants, based on the GalNAc liver delivery platform, have entered the harvest period for drugs targeting cardiovascular indications. In China, enterprises are flourishing, becoming important global participants, and frequent collaborations with MNCs are occurring.

The main viewpoints of CPMC Securities are as follows:

siRNA Drugs Highlight Mechanism Advantages, Delivery as Core Technology

Compared with traditional small molecules and biologics, nucleic acid therapies represented by siRNA drugs can precisely silence pathogenic proteins, degrade efficiently, and have a long-lasting effect. They have now become a popular direction in new drug development. Technically, they mainly include modification and delivery, with delivery being the core technology. Extrahepatic delivery is currently the focus of breakthroughs. Technical pathways include LNP, antibodies, peptides, and VLP delivery, each with its own advantages and disadvantages, among which AOC is developing slightly faster.

Foreign Giants Enter Harvest Period, Await Proof of Concept for Extrahepatic Delivery

GalNAc liver delivery developed by siRNA pioneer Alnylam has become the "standard solution" for liver delivery due to its extremely high delivery efficiency, strong specificity, and long-lasting effects. Other overseas giants such as Arrowhead and Dicerna have entered the late clinical or commercial harvest phase for siRNA drugs targeting common cardiovascular targets like PCSK9, AGT, Lp(a), and ApoC3. However, research on delivery to extrahepatic organs such as muscle, the nervous system, and the eye is still in its early stages. Currently, Alnylam's C16 delivery platform has shown initial potential in the CNS field, while Arrowhead’s TRiM platform has demonstrated promise in adipose tissue. Future competition will focus on the discovery of new targets and advancements in extrahepatic delivery platforms.

China-produced siRNA Drugs with GalNAc Delivery Blossom, Dual-target and Extrahepatic Delivery Strive for the Lead

The Development of China-Produced siRNA Drugs Shows a Flourishing Landscape: Startups Pursue Diverse Directions, Big Pharma Joins the Race, and Chinese Companies Have Become Extremely Important Global Participants in the siRNA Field. Against the Backdrop of Many Chronic Disease Drugs Entering the Late Stages of Sales and the Urgent Need for New Technological Iterations, Multinational Corporations (MNCs) in the Liver Delivery Space Exhibit Strong Business Development (BD) Intentions. Collaborations Such as Boyo with Novartis, Shengyin with Roche, Ribo with Madrigal, and Frontier with GSK Have Been Established. In Emerging Areas, Chinese Companies Are Actively Advancing Dual-Target and Extrahepatic Delivery Technologies, Gradually Narrowing the Gap with Overseas Competitors or Even Taking the Lead, and May Potentially Guide Future Industry Trends.

Investment Advice:It is recommended to focus on: A-share — Hengrui Pharma, Bribet, Frontier Biotechnologies, Yikang Pharmaceuticals, Sunshine Nuhe, Fuyuan Pharmaceuticals, etc.; Hong Kong stocks — Ribo Life Science, CSPC Pharmaceutical Group, China Biologic Products Holdings, Genrui Biotech (already applied for listing).

Risk Warning:1) The clinical progress of innovative drugs is slower than expected; 2) The clinical data of innovative drugs is less impressive than expected; 3) The sales performance of innovative drugs after market launch is below expectations; 4) Geopolitical risks.