Shenwan Hongyuan: Small nucleic acid drugs enter a rapid development stage, domestic companies accelerate to seize the blue ocean

According to the research report released by Shenwan Hongyuan, based on data from the Sullivan report, the global small nucleic acid drug market size has grown from USD 2.7 billion in 2019 to USD 4.6 billion in 2023, with a compound annual growth rate (CAGR) of 14.3%. Driven by continuous technological advancements, increasing approvals for market entry, and ongoing clinical validations, the global small nucleic acid drug market is expected to accelerate its growth, reaching USD 46.7 billion by 2033 at a CAGR of 26.1% starting from 2023. With the gradual commercialization of overseas products, domestic innovative pharmaceutical companies have also been accelerating their layout in small nucleic acid drugs in recent years. Unlike the classic model of overseas giants Alnylam and Ionis moving from rare diseases to common diseases, domestic small nucleic acid research and development has chosen to directly enter the chronic disease track, focusing on indications for major diseases such as cardiovascular diseases, hepatitis B, and cancer.

Shenwan Hongyuan's main viewpoints are as follows:

Small nucleic acid drug technology is gradually maturing and has significant advantages

Compared with traditional small molecule drugs and antibody drugs, small nucleic acid drugs have significant advantages such as shorter research and development cycles, rich targets, lasting effects, and higher success rates in development. From a mechanism of action perspective, small nucleic acid drugs act directly at the mRNA level, targeting "undruggable" targets that traditional drugs find difficult to reach, providing new treatment ideas for many refractory diseases.

From rare diseases to common diseases, small nucleic acid drugs are entering a rapid development stage

Currently, a total of 22 small nucleic acid drugs have been approved globally (of which 3 have been withdrawn), mainly concentrated in the fields of spinal muscular atrophy (SMA), Duchenne muscular dystrophy (DMD), and transthyretin amyloidosis (ATTR), among other rare diseases. In recent years, with the data readouts of small nucleic acid drugs in areas such as hyperlipidemia, hepatitis B, and hypertension, a trend of small nucleic acid drugs crossing from rare diseases to chronic and common diseases has already emerged, and the market is expected to accelerate its expansion.

According to data from the Sullivan report, the global small nucleic acid drug market size has grown from USD 2.7 billion in 2019 to USD 4.6 billion in 2023, with a CAGR of 14.3%. Driven by continuous technological advancements, increasing approvals for market entry, and ongoing clinical validations, the global small nucleic acid drug market is expected to accelerate its growth, reaching USD 46.7 billion by 2033 at a CAGR of 26.1% starting from 2023.

Domestic companies are accelerating their capture of blue oceans, focusing on data readouts and BD progress

With the gradual commercialization of overseas products, domestic innovative pharmaceutical companies have also been accelerating their layout in small nucleic acid drugs in recent years. Unlike the classic model of overseas giants Alnylam and Ionis moving from rare diseases to common diseases, domestic small nucleic acid research and development has chosen to directly enter the chronic disease track, focusing on indications for major diseases such as cardiovascular diseases, hepatitis B, and cancer. Currently, several self-developed products in China are in clinical phases I and II, and it is recommended to pay attention to subsequent clinical data readouts and potential overseas licensing.

Regarding targets

It is recommended to focus on companies with independent technology platforms and more advantageous pipelines: including Reebio, Singlomics-B (02257), Bowe Pharmaceutical, YKYY (688658.SH), CSPC Pharmaceutical Group (01093), Terns Pharmaceuticals-B (02137), and Hengrui Pharma (01276). Outstanding overseas companies include Alnylam (ALNY.US), Ionis (IONS.US), and Arrowhead (AWRW.US) Risk Warning

Risks of research and development progress not meeting expectations, risks of product volume not meeting expectations, and risks of price decline due to intensified product competition