I'm LongbridgeAI, I can summarize articles.Dongfang Securities released a research report indicating that small nucleic acid drugs are expected to address clinical needs that traditional drugs cannot meet, becoming a new technological pathway, especially in the field of chronic diseases. With advancements in modification and delivery technologies, small nucleic acid drugs are expanding from rare diseases to common diseases, and it is recommended to pay attention to high-quality targets in the domestic market related to chronic hepatitis B and cardiovascular diseases. Small nucleic acid drugs such as ASO and siRNA have advantages such as broad targets and strong specificity, with huge market potential
According to the Zhitong Finance APP, Dongfang Securities released a research report stating that small nucleic acid drugs can address clinical needs that traditional drugs such as small molecules and antibodies cannot meet, and are highly likely to become a new technological pathway for generating a large number of products. With advancements in modification and delivery technologies, their therapeutic areas are at a turning point, expanding from rare diseases to common chronic diseases with many patients. Overseas multinational corporations (MNCs) are accelerating their small nucleic acid layout through collaborative research and development and licensing. Domestic pharmaceutical companies are also actively researching and directly entering the common chronic disease market, suggesting attention to high-quality targets related to small nucleic acid drug pipelines in the domestic layout for chronic hepatitis B and cardiovascular fields.
The main viewpoints of Dongfang Securities are as follows:
Small nucleic acids are expected to become the third major class of drugs after small molecules and antibodies
Small nucleic acid drugs refer to ASO (antisense oligonucleotides), siRNA (small interfering RNA), miRNA (micro RNA), and other short-chain nucleic acids that regulate gene expression by interacting with mRNA. They possess unique advantages such as broad targets, strong specificity, high research and development efficiency, and long dosing intervals, making them highly potential as a new technological pathway for generating a large number of products.
Commercialization of rare diseases matures, long-acting advantages of chronic diseases emerge
Currently, there are 11 ASOs, 7 siRNAs, and 1 Aptamer (aptamer) on the market. Among them, Spinraza, targeting spinal muscular atrophy, has been the most successful in commercialization, with cumulative global sales nearing USD 13.8 billion by 2024; Leqivo (Inclisiran), with its long-term safety advantages and stable blood lipid control with just two injections a year, is rapidly gaining market share (USD 260 million global sales in Q1 2025, up 72% year-on-year). Additionally, with breakthroughs in modification and delivery technologies, small nucleic acids are at a turning point, expanding from rare diseases to common chronic diseases with many patients.
Overseas: An important turning point in the TTR field, CVD phase III data readout imminent
First, the supplemental marketing application for Vutrisiran's ATTR-CM (transthyretin amyloid cardiomyopathy) indication has been approved by the FDA, expected to achieve a "tripartite balance" in the future USD 10 billion market alongside Tafamidis and Acoramidis; secondly, Pelacarsen, the first drug targeting ApoA developed jointly by Novartis and Ionis, is expected to read out phase III clinical data in 2025, with Cortellis predicting its peak sales could reach USD 2.58 billion by 2030. Additionally, Olezarsen (for treating severe hypertriglyceridemia) will also read out phase III data in the second half of 2025.
Domestic: New cornerstone therapy for chronic hepatitis B, intense competition in the cardiovascular field
In the chronic hepatitis B field, GSK836 is expected to read out complete phase III clinical data and gain approval in 2026, while AHB-137 from Haobo Pharmaceutical has recently been approved to initiate phase III clinical trials in China. The pipeline progress of companies like Tengsheng Bo Pharmaceutical, Hengrui Pharma, SINO BIOPHARM, and Ruibo Bio is also worth noting; in the cardiovascular field, several early pipelines from domestic pharmaceutical companies focus on targets such as PCSK9, AGT, and ANGPTL3, aiming at lipid-lowering and blood pressure-lowering tracks; in the gout field, LIVZON PHARMA's YJH-012 domestic IND application has been accepted, with preclinical data indicating that a single dose of YJH-012 can achieve sustained uric acid reduction for 3-6 months, and with good safety, it is expected to provide a longer-lasting, safer, and more thorough solution for the clinically unmet needs in the gout field Frequent Large External BD Highlights the Potential of Early Chronic Disease Pipeline
On January 3, 2024, Reebio reached a cooperation agreement with Boehringer Ingelheim to jointly develop innovative therapies for MASH/NASH small nucleic acids, with a total cooperation amount exceeding $2 billion. On January 10, 2025, the first milestone was achieved; on January 7, 2024, Bozhong Pharmaceutical authorized Novartis the option rights for two early pipelines and up to two cardiovascular target drugs in the cardiovascular field, receiving a $185 million upfront payment and up to $4.2 billion in potential milestones. As the therapeutic potential of small nucleic acid drugs in common chronic diseases is released, it is expected that significant BD (referring to the external authorization of innovative drugs, i.e., pipeline going overseas) will continue to emerge in the future.
Related Targets
Hengrui Pharma (600276.SH), SINO BIOPHARM (01177), YKYY (688658.SH), Innovent Biologics (01801), LIVZON PHARMA (000513.SZ), Junshi Biosciences-U (688180.SH), CSPC Pharmaceutical Group (01093), Terns Pharmaceuticals (02137), and Sanofi (02257), etc.
Risk Warning
Risks of failure in innovative drug development, risks of significant price reductions due to intensified market competition, and risks of product sales and BD falling short of expectations