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Recently, ASO leader Ionis Pharmaceuticals announced that it has signed a licensing agreement with Japanese pharmaceutical company Ono, granting the latter exclusive global rights to develop and commercialize sapablarsen. Ionis will receive a prepayment of $280 million, as well as potential additional payments and royalties of up to $660 million. It is reported that sapablarsen is an antisense oligonucleotide drug used to treat polycythemia (PV), which has been granted fast track and orphan drug designation by the US FDA.

 

 

 

Another leading small nucleic acid company, Alnylam, has recently obtained a major approval, with vutrisiran (trade name Amvuttra) approved by the FDA for the treatment of transthyretin amyloidosis cardiomyopathy (ATTR-CM). It is reported that Vutrisiran is a subcutaneous siRNA therapy, which is the first FDA approved therapy that can simultaneously treat polyneuropathy and cardiomyopathy in patients with ATTR amyloidosis.

 

 

 

Small nucleic acid drugs have the advantages of high specificity, short research and development cycle, low susceptibility to drug resistance, long-lasting effects, and a wide range of treatment fields, and have great development prospects. Currently in China, many pharmaceutical companies have also ushered in new development opportunities in the field of small nucleic acid drugs.

 

 

 

On March 14th, Yuekang Pharmaceutical released its annual performance report for 2024, revealing that the ultra long acting lipid-lowering small nucleic acid drug YKY015 injection has been approved for clinical trials in China and the United States. Phase I clinical trials in China are currently underway, and it is expected to achieve effective lipid-lowering with only two injections per year. It is coupled with Yuekang Pharmaceutical's independently developed GalNAc delivery system, which can achieve efficient liver targeted delivery.

 

 

 

Frontier Biotech is also steadily advancing the research and development of small nucleic acid drugs, and has successfully identified candidate chemicals (PCC) for some projects. One of the selected targets has the potential to be a first in class small nucleic acid candidate drug, which has successfully entered the IND enabling stage of new drug clinical trial application.

 

 

 

Analysts point out that after years of development, with the continuous advancement of technology, nucleic acid drugs are also becoming the next breakthrough point for domestic innovative drugs. It is reported that the Chinese small nucleic acid drug market is expected to reach around 4 million US dollars in 2022 and over 300 million US dollars in 2025. The huge market size will attract domestic pharmaceutical companies to accelerate their entry and expand their research and development focus from rare diseases to chronic diseases and more disease areas, bringing new treatment options and hope to patients.