China's Small Nucleic Acid Drug BD Era Begins with Argo Biopharma's Landmark $4.165B Deal with Novartis

On January 7, Argo Biopharma announced: the signing of two exclusive license and cooperation agreements with Novartis,Grant Novartis the overseas rights to a Phase 1/2a cardiovascular pipeline, the global rights to a Phase 1 cardiovascular pipeline, and options for no more than two other target cardiovascular pipelines.

These pipelines are all siRNA drugs. Argo Biopharma will receive an upfront payment of $185 million, potential milestone payments totaling up to $4.165 billion, and tiered royalty fees.

This is the largest licensing-out deal in terms of transaction amount since the beginning of 2024.In terms of the upfront payment, it has already ranked among the Top 10 overseas transactions by Chinese pharmaceutical companies.

Unlike before, the subject of this deal is the newly emerging siRNA drug. The chairman and CEO of Argo Biopharma stated: "This is the first major overseas licensing deal by a Chinese biotech company in the RNAi field."

Novartis is a global leader in the field of cardiovascular diseases and RNAi therapies. This deal also indicates that, apart from bispecific antibodies and ADCs, China's small nucleic acid drug companies are increasingly attracting foreign investment attention.

Favored by Giants

siRNA drugs are a type of small nucleic acid drugs, featuring multiple druggable targets, strong efficacy, good safety, and low dosing frequency.

In 2019, Novartis spent $9.7 billion to acquire The Medicines Company, obtaining the siRNA drug inclisiran sodium developed by Alnylam. This is the "cholesterol-lowering miracle drug" that only needs to be administered once every six months and has already been launched in China.

Small nucleic acid drugs were mainly used to treat rare diseases in the past,Inclisiran sodium is a rare small nucleic acid drug for chronic diseases, and its market prospects are naturally very broad.After Novartis made its move, it continued to expand its portfolio in chronic disease small nucleic acid drugs.

In April 2021, Argo Biopharma was established in Zhangjiang, Shanghai, with a team of about 50 people. Core members come from Arrowhead, a pioneer in siRNA drugs, and multinational pharmaceutical companies such as Merck.In November 2023, the company had just completed its A+ round of financing.Led by SDIC Capital, with Huagai Capital, Sany Innovation Investment and others following.

Argo has advanced four projects into clinical trials, and the fifth project has also been submitted for clinical application, covering therapeutic areas such as cardiovascular diseases, rare diseases, hepatitis B, autoimmune conditions, and neurological disorders.The successfully BD products this time are mainly cardiovascular disease drugs.

Based on the pipeline projects disclosed on the company's official website, the authorized products are likely to be BW-01 and BW-02, which are advanced in the cardiovascular field.

Source: Argo Biopharma's official website

According to the publicly filed patent application by Argo Biopharma, the company’s potential target layout may include ANGPTL3, AGT, LPA, PKK, C3, XDH, etc. It is not yet clear which targets are involved in this transaction.

However, what the giants are eyeing may be the potential of Argo Biopharma's nucleic acid drug development platform and core products.

Argo previously claimed that, compared head-to-head with other international companies' technology platforms, the drugs generated by its self-developed RADS technology platform can achieve better efficacy and longer duration at lower doses.One of the products is expected to achieve a dosing frequency of once a year.

New Force Company Exerts Efforts

Currently,The core technology and patents of siRNA drugs are concentrated in a few leading enterprises.Later entrants basically can't bypass their patent layout.

Among them, Alnylam is undoubtedly the leader in the siRNA field, holding the most siRNA delivery patents.Five of the six siRNA drugs currently on the market are related to Alnylam.

In recent years, innovative pharmaceutical companies in China have also begun to focus on the RNAi track represented by siRNA drugs, attempting to bypass strong patent barriers through the independent development of innovative technology platforms.

Among them, a group of biotech companies have achieved results and thus gained significant attention from investment institutions.In 2022, under the biopharmaceutical winter, the siRNA field made a逆势突围 with 33 related companies securing financing, totaling nearly 5 billion yuan., five companies raised over 100 million RMB in funding, most of which have established mature technology platforms, including Argo Biopharma, which has achieved this licensing deal.

Another company with high financing is the "star enterprise" SinoBio, which gained significant popularity in 2023. Also established in 2021, it recently secured an $80 million Series A+ round of financing.

November 9,Saint Biotech and Hangzhou Zhongmei Huadong Pharmaceutical, a wholly-owned subsidiary of Huadong Medicine, Reach Strategic CooperationThe two parties will jointly develop siRNA drugs for chronic metabolic diseases based on Saint Biologics' LEADTM innovative technology platform.

On December 27, Saint Gene Biotech and Innovent Biologics reached a strategic cooperation agreement to jointly develop the siRNA candidate drug SGB-3908 targeting angiotensinogen (AGT) for the treatment of hypertension.

Although still in the early stages technically, with strong market confidence and substantial financing, these emerging siRNA technology companies in China are rapidly advancing their clinical layouts and pipeline progress.

By this year, foreign giants have also started to set their sights on China's small nucleic acid drug companies. At the beginning of the new year,German company Boehringer Ingelheim announces collaboration with Suzhou Ribo Life ScienceReach a cooperation to jointly develop treatments in the NASH/MASH field.Small nucleic acid innovative therapies, with a total transaction amount exceeding 2 billion US dollars.

It is believed that soon, more BD transactions from small nucleic acid companies in China are on the way.

Author｜Li Ao

Editor｜Jiang Yun, Jia Ting

Operation｜Han Jinrui

Image source: Visual China

Statement: Original content by Jian Shi Ju, please do not reprint without permission.

A New Round of Flu Arrives; China's Central Commission for Discipline Inspection to Crack Down on Medical Corruption This Year; Senior Executives of Multiple Pharmaceutical Companies Replaced

From this year, pharmaceutical companies involved in bribery will face heavier penalties, with a maximum sentence of life imprisonment.

Repeated Talks, Significant Price Cuts: China's National Healthcare Security Administration Cracks Down on Drug Companies' "Shortage-Driven Price Hikes"