Recently, there have been frequent BD activities in the small nucleic acid field:Novartis with a potential total transaction value of $5.36 billion andArgoArgo BiopharmaCollaboratively develop multiple cardiovascular siRNA therapies; meanwhile, Novartis introduces with a potential total transaction value of $2.2 billion.ArrowheadThe Company's Small Nucleic Acid DrugsARO-SNCA, This drug is used to treat synucleinopathies such as Parkinson's disease.Besides ArgoChina, currently, Pfizer, Eli Lilly, Merck, NovoNordMNCs have also entered the small nucleic acid track through external collaborations in recent years.The strong entry of MNCs confirms the increasingly prominent clinical and commercial value of the small nucleic acid track.Whether viewed from the perspective of drug research and development technology, therapeutic potential, or market potential, the era of small nucleic acid drugs has arrived.The small nucleic acid drug market has entered a fast lane of growth.
Currently, there are a total of18 Kinds of Small Nucleic AcidsDrug ApprovalBatch Market Release(Excluding delisted products), including 11 ASO drugs, 6 siRNA drugs, and 1 nucleic acid drug.SuitableLigand drug, where genetically rare diseases are targeted.The Most BatchesIndication Categories.In 2024, the global sales totaled approximately US$6 billion, with a compound annual growth rate as high as 30% over the past three years. The small nucleic acid drug market is in a phase of rapid growth.Status of Small Nucleic Acid Drugs Already on the Market Globally(Unit: billion US dollars)
Source: Brokerage FirmResearchReport, Annual Report of Enterprises, Compiled by the AuthorIt is worth noting that,Although, most of the small nucleic acid drugs approved for marketing in the early stage focused on rare disease indications,The patient population is relatively limited,But this did not stop the birth of a "blockbuster." The most typical example isTransthyretin Amyloidosis（ATTR）Small Nucleic AcidDrugMarket.ATTR is a condition caused by misfoldedTransthyretin(TTR) FormationSystemic degenerative diseases caused by amyloid fibrils and their deposition in tissues and organs, The main treatment methods currently include TTR stabilizers and TTR synthesis inhibitors (involving ASO drugs).and siRNA drugs）。Alnylam PharmaceuticalsTTR InhibitorAmvuttraSince its listing in 2022ContinuousWith the increase in volume, it achieved revenue of 0.94 billion US dollars in the first year of its market launch, 5.58 billion US dollars in 2023, and 9.71 billion US dollars in 2024. This shows that ATTR drugs haveVery considerable market potential.In August this year,HuiRui ChinaOptimize the organizational structure of the Oncology and Rare Diseases Division,Integrate the existing hemophilia and ATTR teams to establish a focus onRare Disease FieldAn independent team, integrating internal resources, to further advance the commercialization of groundbreaking innovative drugs in the rare disease field.This organizational restructuring optimization is aligned with Pfizer's innovative drug in the ATTR field, which has significant market potential.TafamidisClosely related,TafamidisSince its launch in 2019, sales have continued to grow, reaching sales of the drug in 2024.$5.5 billion, a year-on-year increase of over 60%.Small Nucleic Acid DrugsAmvuttraIt may have more competitive advantages in terms of drug delivery convenience and mechanisms.Amvuttra requires only 4 subcutaneous injections per year., andTafamidisOnce daily;AmvuttraCan directly target and silence the mRNA of the TTR gene, reducing the production of TTR protein at the source; TafamidisIt is a TTR stabilizer that works by stabilizingTetrameric Structure of TTR ProteinTherebyReduce Misfolded ProteinsDeposition.This shows that,AmvuttraOr will rely onConvenience of administration and mechanism advantages enabled latecomers to surpass in the later market competition.，HopefulBecoming a blockbuster product worth over a billion dollars.Indication Breakthrough
Wide Layout in the Field of Chronic Diseases
Accompanying In Vivo StabilityAs well as the gradual maturity of delivery systems, overseas leading companies in the small nucleic acid drug indications are gradually exploring the broader market of chronic diseases, and the clinical and commercial value of small nucleic acid drugs in the field of chronic diseases has been verified.As the world's firstLaunchThe siRNA lipid-lowering drug, Novartis's long-acting lipid-lowering drugLeqvioThe target of action isProprotein Convertase Subtilisin/Kexin9（PCSK9）ThemRNA。LeqvioApproved for marketing by the FDA in 2021 and by the NMPA in 2023. According to sales data, since its launch,LeqviocShowing an accelerating growth trend, achieving sales of $112 million in 2022, $355 million in 2023, and $754 million in 2024.In addition to the field of hyperlipidemia, leading companies are exploring the therapeutic potential of small nucleic acid drugs in neurodegenerative diseases, metabolic disorders, and other disease areas.AlnylamThe R&D pipeline currently focuses on TTR and rare diseases, while further expanding intoCardiovascular and Cerebrovascular、NerveAndMetabolismAnd other disease areas.AlnylamInResearchProduct Pipeline
Source:AlnylamAmong them,ZILEBESIRANPhase II Clinical Trial for the Treatment of Hypertension (KARDIA-3) The data will be read out in the second half of 2025, and the Phase III clinical trial will be initiated;MIVELSIRANPhase II clinical trials for the treatment of Alzheimer's disease will also be launched in the second half of 2025, with early data showing,MIVELSIRANSustainably reduce the levels of beta-amyloid in the cerebrospinal fluid.Efficacy Data of MIVELSIRAN
Source:AlynlamIonis PharmaceuticalsArgo Biopharma is a pioneer and leading company in the ASO field, with a pipeline primarily focused on central nervous system, cardiovascular, and rare disease areas, where it has several highly competitive products in the neuroscience sector., TargetingAngelman Syndrome's InResearchDrugION582It is currently in Phase III clinical trials, with plans to complete all patient enrollment by next year; targetingAlzheimer's disease, Parkinson's disease inResearchThe drug has entered Phase II clinical trials.。Ionis inResearchProduct Pipeline
Source:Ionis(As of August 1, 2025)Clinical Progress Accelerates for China's Innovative Drug Companies
Currently, there is no domesticSmall Nucleic AcidDrugObtainBatch listing, but in recent years, in ChinaInnovative pharmaceutical companies inThe clinical progress of small nucleic acid drugs is accelerating.。In the selection of indications,Different from overseas companies that have taken the lead in making breakthroughs in the field of rare diseases,In ChinaFocus on Innovative Pharmaceutical EnterprisesIn the field of chronic diseases,DirectTargeting diseases with large patient populations such as hepatitis B, cardio-metabolic disorders, autoimmune conditions, and cancer.Indications。Clinical Research Progress of Small Nucleic Acid Drugs by Chinese Enterprises(Only list Phase II and III stages)
Source: Broker report, compiled by the authorFor example, for the hepatitis B indication, Hengrui,Haobo Medicine、StarYao Kun ZeMany companies, including..., have made arrangements.AHB-137is a targetedHBVASO drug. In May 2025,Haobo MedicinePublishedAHB-137IIPhase Clinical Trial Data，The primary endpoint was a hepatitis B surface antigen level below the lower limit of quantification (<0.05 IU/mL) and a hepatitis B virus DNA level below the lower limit of quantification (<10 IU/mL) at the end of the treatment period. IIPhase clinical trial data show,InDay of TreatmentAt 24 weeks, 75% of the subjects reached this primary endpoint; in the 16-week treatment group, 66% of thePatientReached this primary endpoint.In July this year,Haobo MedicineLaunchedAHB-137Initiate Phase 3 Clinical Trial.StarYao Kun ZeTheHT-101It is a GalNAc-conjugated siRNA.Drug, its Phase IIPreliminary DataSimilarlyDemonstrating excellentHepatitis B Surface AntigenReduced data at 16 weeks of treatment, high-dose group (400mg)Hepatitis B Surface AntigenDecreased by 3.29 log.Hengrui MedicineHRS-5635is asiRNA Drugs Targeting HBV，As of 2024H1 Enters Phase II Clinical Trial.With the gradual maturation of the small nucleic acid drug technology platform，Multiple marketed products have achieved very impressive clinical data and commercialization results. The breakthrough in indications will rapidly expand the small nucleic acid drug market, heralding the arrival of a golden age for small nucleic acid drugs.1. Annual Report of the Enterprise2. In-depth Research on the Small Nucleic Acid Industry, Hua Fu Securities, March 20253. What companies are involved in the small nucleic acid layout?, Guosheng Securities, July 2025*Disclaimer: This article is only intended to introduce research progress in the pharmaceutical and disease fields, provide a brief overview of research, or share pharmaceutical-related information. It neither recommends any treatment or diagnostic plans nor constitutes any advice on related investments. If there are any omissions in the content, please feel free to communicate and point them out!
