Oligonucleotide drugs are poised to initiate a new industry wave in the cardiovascular field over the next three years, as Phase III clinical data for related products are successively disclosed. Combining oligonucleotide therapies with novel cccDNA clearance methods or low-dose interferon may further elevate the functional cure rate while maintaining superior safety, offering a new potential option for the functional cure of chronic hepatitis B. The domestic oligonucleotide drug sector is entering a period of rapid development and is positioned to become a significant leader within this evolving landscape. Investors are advised to focus on leading domestic oligonucleotide R&D companies.
GLMS SEC's primary viewpoints are outlined below.
Inclisiran Demonstrates Significant Commercial Uptake; Novartis's Oligonucleotide Performance Stands Out
Cardiovascular diseases remain the leading cause of death globally. Novartis's Inclisiran, the world's first cardiovascular oligonucleotide drug approved in 2021, has seen its sales steadily increase year-over-year, reaching $1.198 billion in 2025, a 59% year-on-year growth. This success is attributed to its advantages of long-acting lipid-lowering efficacy with a "twice-yearly" dosing regimen and a strong safety profile. The dual validation of Inclisiran's clinical efficacy and market value has bolstered confidence among multinational pharmaceutical companies to continue intensifying their focus on the cardiovascular oligonucleotide therapeutic area.
Oligonucleotide Drugs Outline a New Blueprint for Comprehensive Cardiovascular Risk Management
Oligonucleotide pipelines under development by multinational corporations comprehensively cover multiple cardiovascular risk factors, including hypertension, kidney disease, elevated Lp(a), and hypertriglyceridemia, and have demonstrated clear efficacy in Phase II clinical trials. Roche's zilebesiran, targeting AGT, achieves sustained blood pressure reduction with a single dose. Sefaxersen, targeting complement factor B, reduces 24-hour UPCR by 40% in IgA nephropathy patients, matching the efficacy of the approved small-molecule drug Iptacopan targeting the same pathway, with a favorable safety profile. Eli Lilly's Lepodisiran, administered every six months, reduces Lp(a) levels by 93.9%, showing strong performance. Solbinsiran, targeting ANGPTL3, halves triglyceride levels, reduces LDL-C by 16.8%, lowers non-HDL cholesterol by 25%, and decreases ApoB by 14%, with therapeutic effects persisting for at least three months post-treatment cessation, enabling long-term lipid management. Over the next three years, as Phase III clinical data for these products are released, oligonucleotide drugs are expected to catalyze a new industry surge in the cardiovascular domain.
Oligonucleotide Drugs Offer a New Potential Pathway for Functional Cure of Chronic Hepatitis B
Chronic hepatitis B virus infection is a major contributor to hepatitis, cirrhosis, liver failure, and liver cancer, affecting over 250 million people globally and causing approximately 1.1 million deaths annually. Current mainstream therapies have limitations: nucleos(t)ide analogs (NAs) require long treatment durations, with nearly 30% of patients on NAs experiencing low-level viremia, and functional cure is difficult to achieve. Interferon therapy can improve functional cure rates but is associated with certain adverse reactions. GSK's oligonucleotide drug Bepirovirsen has shown significant functional cure efficacy in Phase III trials, and its marketing applications in China and Japan have been accepted. Combining oligonucleotide therapies with novel cccDNA clearance strategies or low-dose interferon holds promise for further increasing the functional cure rate while maintaining better safety, potentially providing a new therapeutic choice.
Multinational Pharma Increases Focus on Oligonucleotide Therapies for Neurological Disorders
The number of patients with neurodegenerative diseases such as Alzheimer's and Parkinson's has been consistently rising in recent years. The abnormal intracellular aggregation of pathological proteins (such as tau or a-synuclein) is a core pathogenic mechanism in these neurological conditions. Oligonucleotide drugs possess the potential to slow disease progression by endogenously inhibiting the mRNA of these pathological proteins. Currently, multinational corporations have advanced several oligonucleotide pipelines into clinical stages and are enhancing the efficiency of blood-brain barrier penetration for these drugs through the optimization of antibody-oligonucleotide conjugate (AOC) technology.
Risk Factors to Consider
1) Potential for drug pricing to fall short of expectations due to national reimbursement and centralized procurement policies.
2) Clinical development progress or market launch timelines may be slower than anticipated.
3) Deterioration of the competitive landscape within the industry.