Oligo Insights | Global and Domestic Oligonucleotide Industry Update – Issue 8, 2025

Sep 11, 2025 13:50 CST Updated 13:50

Argo

RNAi Drug Developer

Innovation in China

01 Dongyang Light Medicine

Dongyang Light Pharma Self-Developed Hepatitis B Small Nucleic Acid New Drug Submitted for Clinical Trial in China

On August 2, 2025, HECN30227, an siRNA therapy for hepatitis B independently developed by Dongyangguang Pharmaceutical, officially submitted its Investigational New Drug (IND) application, marking a crucial step forward for the company in the field of small nucleic acid drug development.

HECN30227 is a Class 1 new drug independently developed by Dongyang Guangya Pharmaceutical with global intellectual property rights. It is also the first siRNA drug developed by Dongyang Guangya Pharmaceutical based on its small nucleic acid technology platform, capable of simultaneously eliminating HBsAg derived from cccDNA and intDNA. Preclinical data shows that HECN30227 exhibits pan-genotypic activity, effectively reducing HBV surface antigen levels, maintaining significant efficacy against nucleoside drug-resistant strains, and demonstrating superior in vivo and in vitro efficacy compared to clinical competitors. The drug utilizes Dongyang Guangya Pharmaceutical's uniquely designed HEC-GalNova (N-acetylgalactosamine) liver-targeting delivery system, which achieves precise and efficient liver delivery while significantly reducing off-target risks.

02 Preact

Shenji Changhua Collaborates with Mr. Cai Lei to Establish a Joint Laboratory, Aiding in ALS Drug Development

Argo Biopharma's Small Nucleic Acid Drug LDR2402 Injection for Hypertension Treatment Enters Phase 2 Clinical Trials

August 28, 2025，Phase 2 Clinical Trial Application for LDR2402 Injection, a Class 1 New Drug Independently Developed by Chengdu Xianyan Biotechnology Co., Ltd. (hereinafter referred to as "Xianyan Biotech"), has been publicly disclosed on the official website of the Center for Drug Evaluation (CDE) under the National Medical Products Administration (NMPA). This drug is a small nucleic acid innovative medicine targeting angiotensinogen (AGT) for the treatment of primary hypertension and represents the first siRNA-based antihypertensive drug in China to enter Phase 2 clinical trials. The advancement of LDR2402 Injection into Phase 2 clinical trials marks a significant breakthrough for Xianyan Biotech in the field of small nucleic acid drug development, officially moving into a new stage of clinical development.

LDR2402 Injection is a novel small nucleic acid drug developed based on RNA interference (RNAi) technology. In preclinical experiments, it significantly reduces the level of angiotensinogen (AGT), with effects lasting over six months after a single injection. AGT is the origin of the renin-angiotensin-aldosterone system (RAAS) mechanism. LDR2402 selectively enters the liver to silence AGT expression at its source, thereby achieving a blood pressure-lowering effect. In clinical treatment, LDR2402 may improve patient compliance by extending the dosing interval (once every quarter or half a year). It also provides steady blood pressure reduction within 24 hours, decreases blood pressure variability (BPV), and offers greater cardiovascular benefits to patients with hypertension.

03 Argo Biopharma

Shenji Changhua Collaborates with Mr. Cai Lei to Establish a Joint Laboratory, Aiding in ALS Drug Development

Argo Biopharma Reaches New Licensing Agreement with Novartis

On September 3, 2025, Argo Biopharma Co., Ltd. ("Argo Biopharma," "the Company") announced a new strategic collaboration agreement with Novartis (NYSE: NVS) to jointly develop multiple cardiovascular products. This newly reached agreement represents a further expansion of the existing cooperation between the two parties.

According to the agreement, Argo Biopharma granted Novartis an option for the rights outside of China to two early-stage molecules for the treatment of severe hypertriglyceridemia (sHTG) and mixed dyslipidemia, as well as the priority negotiation rights for the BW-00112 (ANGPTL3) product. This product is currently in Phase II clinical trials in the United States and China, and subsequent combination therapy clinical trials will be led by Argo Biopharma. The trials will target another siRNA candidate drug currently in the preclinical research stage, with the product expected to initiate Phase I clinical trials in 2026. Argo Biopharma will receive an upfront payment of $160 million and is eligible to receive potential milestone and option payments, as well as tiered royalties on commercial sales, with a total potential milestone value of up to $5.2 billion.

Overseas Hotspots

01 Novartis

Novartis siRNA Drug Inclisiran Sodium Launches New Phase III Clinical Trial

On August 4, 2025, the ClinicalTrials.gov website showed that Novartis (NVS.US) registered a Phase III clinical trial of inclisiran sodium. This trial aims to evaluate the efficacy and safety of early initiation of inclisiran sodium treatment in patients hospitalized with acute coronary syndrome (ACS). This is a multicenter, randomized, double-blind, placebo-controlled study. Participants are patients who have recently experienced acute coronary syndrome (ACS), including those with ST-segment elevation myocardial infarction (STEMI) and non-ST-segment elevation myocardial infarction (NSTEMI).。

Inclisiran Sodium Injection is a first-in-class siRNA drug targeting PCSK9, which requires only two subcutaneous administrations per year. It is suitable for patients with atherosclerotic cardiovascular disease (ASCVD) and heterozygous familial hypercholesterolemia (HeFH). In July 2023, its use was expanded to include patients with primary hyperlipidemia. To date, the drug has been approved for four indications in the EU, three indications in the US, and four indications in China.。

02 Ionis

Donidalorsen: Approved for Marketing by the U.S. FDA

2On August 21, 2025, Ionis Pharmaceuticals announced that the FDA had approved Dawnzera (donidalorsen) for the prevention of hereditary angioedema (HAE) attacks in adult and pediatric patients aged 12 years and older. According to the press release, donidalorsen is the first RNA-targeting drug approved for HAE.

Donidalorsen is an antisense oligonucleotide ligand conjugate designed to precisely target and silence the expression of prekallikrein (PKK), interrupting the signaling pathway that leads to HAE attacks. PKK plays a significant role in activating inflammatory mediators associated with acute HAE episodes.。

03 Regeneron

New Drug Cemdisiran for Myasthenia Gravis Announces Phase 3 Clinical Trial Data

2On August 26, 2025, Regeneron Pharmaceuticals announced that cemdisiran, a complement C5-targeted RNAi therapy jointly developed with Alnylam Pharmaceuticals, met the primary and key secondary endpoints in the NIMBLE Phase 3 clinical trial for the treatment of adult generalized myasthenia gravis (gMG). Regeneron plans to submit a regulatory application for cemdisiran as a monotherapy in the first quarter of 2026 after consulting with the FDA.

Cemdisiran is a small interfering RNA (siRNA) drug co-developed by Regeneron and Alnylam, which can treat diseases related to the abnormal activation of the C5 pathway by reducing the level of complement factor 5 (C5).。

Selected Highlights

1. Small Nucleic Acid Drugs Overseas Market 2025 Outlook

For more industry information, please refer to:

OliView Industry Trends

Clinical Progress of Small Nucleic Acid Drugs in China

(Source: Compiled from official websites and publicly available information of various companies)

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Oli Biopharmaceuticals (Suzhou) Co., Ltd. is a professional medicinal nucleic acid CRDMO company that provides customers with "one-stop" services ranging from nucleic acid drug discovery, laboratory R&D, process and analytical development, CMC services, API production, to pharmaceutical registration. Oli Biopharmaceuticals' technical team originates from one of the earliest groups in China to engage in the development of nucleic acid drug manufacturing processes and CMC research, possessing extensive experience in project development and product registration. Currently, the company has established four industry-leading core technology platforms: nucleic acid solid-phase synthesis, chemical modification and conjugation, process development and analysis, as well as CMC pharmaceutical research. It has also initiated in-depth collaborations with multiple pharmaceutical enterprises and biotechnology companies both domestically and internationally.

In 2023, the company's 3,000-square-meter GMP-compliant pilot platform officially began offering services, focusing on helping clients address critical challenges such as scaling up small nucleic acid drug production processes and CMC pharmaceutical research. The company will continue to provide compliant, high-quality, reliable, and efficient services to help clients improve R&D efficiency, accelerate product registration and market entry, jointly supporting the entire process of small nucleic acid drugs from preclinical to commercial production.

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