Argo Biopharma Sets Sail for RNAi's 'Golden Fleece' with $4.1 Billion Outbound Deal

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‍‍‍‍丨Organized by the PharmaHunter Club Research Team

"Argo" is the name of a ship in ancient Greek mythology, and the people on the ship were called "Argonauts." These heroes successfully overcame many challenges and obtained the "Golden Fleece."In Greek mythology, a treasure is a divine beast with golden fleece, and legend has it that only the brave Jason could obtain it.）。" Argonauts"The story of bravery, courage, and triumph is the inspiration for "Argo Biopharma."

In the RNAi field,Argonaute is the most important protein in the RNAi mechanism.In the RNA interference (RNAi) pathway, siRNA interacts withArgonaute (AGO2) protein interaction forms an RNA-induced silencing complex (RICS) that suppresses target mRNA expression. Subsequently, siRNA unwinds the double strands, and after its antisense strand binds to the target mRNA, AGO2 specifically degrades the target mRNA, thereby inhibiting its translation.

RNAi therapies that function through Argonaute have significant potential for development and can be used to design treatments for a variety of diseases. Investment institutions predict that the global market size for RNAi therapies will reach $X billion by 2030.$25 billion, the domestic market size has reached$3 billion。

Based on breakthrough technology and promising commercial prospects, around 2005, a group of scientists in the small nucleic acid field began returning to China to start businesses, gradually giving rise to China's own leading companies. Among them, Argo Biopharma...AchievementStandAlthough it is not early among a group of companies in China, asFounder of the post-80s generation and experience in the RNAi industry, Argo's founding team has distinct characteristics.

After graduating from the chemistry department of Peking University in 2007, founder Dongxu Shu pursued his Ph.D. and postdoctoral studies at the University of Wisconsin-Madison and Northwestern University, respectively, and later...Joined Arrowhead Pharmaceuticals in 2017 as a Senior Scientist and Project Leader, accumulating extensive experience in the field of siRNA drug discovery.Arrowhead is one of the pioneers in siRNA drugs and currently has more than 10 projects in clinical development, including two therapies in Phase III clinical trials.

Back to small nucleic acids, the prominent R&D features and advantages of small nucleic acid technology lie in,The development of technology has shown platform-based characteristics, and the speed of developing similar drugs will greatly accelerate after the technology platform has been validated.Therefore, in the past three years, as advanced small nucleic acid technology platforms from abroad have been widely validated, RNAi companies with localized technology platforms have been highly sought after by investors.

As more and moreWith multipleYears of experienceSmall Nucleic Acid FieldThe joining of professional talents,ArgoArgo has established a complete nucleic acid drug development platform, covering the entire process of RNAi drug development., including nucleic acid sequence design, chemical modification, GalNAc delivery technology, extrahepatic delivery technology, oligonucleotide synthesis, CMC, etc. The industry-leading RNAi platform technology developed by the company is namedRADS (RNAi Molecules with Exceptional Activity, Duration, and Safety), reflecting ArgoLook forward to continuously generating top-tier RNAi therapies through technical capabilities in delivery, chemical modification, and sequence design.

According to an article by "Zhi Yao Tou," in comparing Argo's self-developed RADS platform withHead-to-head comparison of Alnylam's technology platform reveals that the company's technology platform generates drugs that are expected to achieve better efficacy at lower doses.。

Based on the RADS platform, Argo has established a rich and differentiated product pipeline., including cardiovascular diseases, rare diseases, viral infections, metabolic diseases, and central nervous system diseases.

It is worth mentioning that Argo's pipeline is advancing extremely rapidly.According to the official website of Argo Biopharma, in just over two years, five pipelines have entered the clinical stage, with multiple pipelines confirming PCC. The therapeutic areas cover cardiovascular diseases, rare diseases, viral infections, neurological disorders, etc., and clinical approvals have been obtained in various regions including China, the United States, and Australia.

Among them, BW-01 and BW-02 in the cardiovascular field have entered the clinical stage and are expected to be the core products of this authorization. According to the filing information, these two cardiovascular products are respectively used for treating dyslipidemia and hypertension.

With a founding team backed by industry experience, a full-process drug R&D technology platform, a rich and differentiated pipeline, and highly efficient progress, Argo Biopharma has successfully secured three rounds of financing in less than three years since its establishment, with a total amount exceeding 700 million RMB., including well-known institutions such as Apricot Capital, Loyal Valley Capital, CPE Yuanfeng, Daoyuan Capital, 3E Innovations Investment, and金沙江联合创投.

Novartis TOP20 Best-Selling Drugs in the First Half of 2023

LeqvioThis year, it continues to secure new indications.In July 2023, Leqvio received an updated label from the FDA, expanding its indicated population from patients with atherosclerotic cardiovascular disease (ASCVD) and mixedHeterozygous familial hypercholesterolemia (HeFH) patients are expanded to include those with primary hyperlipidemia. With the continuous expansion of indications and ongoing market penetration, Leqvio is also expected to maintain its growth momentum.Evaluate Pharma predicts its sales peak will reach $3 billion.

siRNAIn addition, Novartis has also acquired the lipid-lowering ASO drug Pelacarsen.Pelacarsen, developed by Lonis, is a drug that can reduce lipoprotein(a).(i.e., Lp(a))Antisense oligonucleotide formulation.The Lp(a) target is known as "the final frontier in the field of blood lipids." Reducing the expression of Lp(a) in patients with dyslipidemia can preventCardiovascular disease risk. As the first ASO drug to lower Lp(a), it significantly reduces Lp(a) by targeting and blocking the synthesis of Apo(a), and has received Breakthrough Therapy Designation from China's CDE.

It is worth mentioning that Novartis' layout in RNAi can be described as "far-sighted."The first ASO drug, fomivirsen (withdrawn from the market), and the first common disease siRNA drug, Inclisiran, both have Novartis' involvement.

In 2022, Novartis announced a key strategic plan:Transform into an "innovative pharmaceutical company".To this end, Novartis will5Five core therapeutic areas: cardiovascular, immunology, neuroscience, solid tumors, and hematology, and continuously advanceEmerging Technology Platform(Gene therapy, cell therapy, radioligand therapy, targeted protein degradation andxRNA)'s strength, these platforms are being prioritized for investment, thereby forming new R&D capabilities and production scales.According to the data disclosed at Novartis Investor Day,Currently, Novartis has accumulated 13 pipelines in xRNA.。

xRNA includes RNA targeting LMWs, ASOs, siRNA, mRNA cancer vaccines. 2. Exploratory to Ph1/2 (December 2022).

For innovative drug exports, 2023 was a milestone year. The number of License-out deals by Chinese companies exceeded License-in for the first time, with multiple new drugs successfully entering the European and American markets, and the scale of outbound licensing cooperation reaching new heights.

However, the overseas licensing of small nucleic acid products has yet to break the ice. Compared with other innovative drug sectors, especially the high-value overseas licensing deals worth hundreds of millions seen in ADCs and monoclonal antibodies, aside from a few technology licenses, the development of domestically produced small nucleic acid products had not garnered significant attention in the overseas market before Ribo Life Science's major BD deal.

2023 Review of BD Projects by Small Nucleic Acid Enterprises in China

However, while collaborations in the domestic market are thriving, the shrinking of the domestic market and the necessity and urgency of going overseas are undeniable facts. The continuous implementation of centralized procurement and medical insurance cost control is constantly compressing the space of the domestic pharmaceutical market, and relying solely on the domestic market can no longer satisfy pharmaceutical companies' need to realize the value of innovation.

Especially in the capital winter, going global to maximize the value of innovative drugs has become a common choice for many big pharma companies seeking innovative transformation and biotech/biopharma firms pursuing first-in-class innovations. After zanubrutinib achieved "zero breakthrough" for Chinese innovative drugs going overseas, the number of Chinese innovative drugs challenging the FDA can be described as incessant.

Especially for the frontier technology of small nucleic acids, historically, the BD between biotech companies and MNCs has been proven to enhance enterprises' self-sustaining capabilities to a certain extent.From the development history of Alnylam and Arrowhead, licensing out part of their pipelines and engaging in collaborative development have enabled these two companies to experience very little financing needs over the years.

Therefore, with two major BD deals at the beginning of the year, in this era of ADC and GLP-1 hype, it is enough to make China's small nucleic acid industry and capital circle "proud and confident." MNCs have turned their attention to Chinese small nucleic acid companies, and the first collaboration was a significant transaction, which also means,Strong enterprises are expected to initiate a virtuous cycle by expanding overseas, and the "domestically produced era" of small nucleic acids is also expected to begin.

After more than a decade of trials and explorations, China's small nucleic acid industry has finally embarked on the "license-out" journey, with two significant BD deals and rapid progress. Of course, while "going global" brings vast market prospects, it also places higher demands on companies' foundational technologies and product R&D capabilities. In 2024, the story of small nucleic acid BD may continue to unfold, and the grand era of small nucleic acids is still on the move.

Source:

Argo Biopharma Official Website

【PharmaSmart Investment】Taking on a Giant Worth Tens of Billions! A Peking University Entrepreneur Born in the 1980s Secures 700 Million in Financing Within Two Years of Starting Up

【Listening to the Tide of Medicine】Honesty, Creating Value: Argo Licenses Cardiovascular Assets to Novartis for $185 Million Upfront Payment

【Hunter's Club】The Era of Significant BD for Small Nucleic Acids Has Arrived! Attached: A Review of Global Small Nucleic Acid Transactions in 2023

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