Small Nucleic Acid Leader: Performance Loss of 3.5 Billion Yuan

Recently, the small nucleic acid leader Ionis Pharmaceuticals released its 2024 performance report, with annual revenue of $705 million, a decrease of 11% from $788 million in 2023. The fourth-quarter revenue was $227 million, down 30% year-on-year. The total operating expenses for the year were $1.18 billion, an increase from $1.14 billion in 2023. The operating loss for the year amounted to $475 million (approximately 3.46 billion RMB, based on the latest exchange rate of 1 USD ≈ 7.2729 RMB).

Ionis' product commercialization revenue in 2024 decreased from $309 million in the previous year to $293 million. R&D expenses were $412 million, down from $479 million in 2023, mainly due to the reduction in the cost of the joint development of Wainua.

Product Sales

1. Spinraza

Spinraza is an antisense oligonucleotide drug used to treat spinal muscular atrophy (SMA) and is the world’s first approved treatment for SMA. It was approved by the US FDA in December 2016. In 2024, the global sales reached $1.6 billion, generating $216 million in royalty income for the company.

2. Wainua

Wainua works by binding to transthyretin (TTR) mRNA to reduce the production of TTR protein, used for the treatment of hereditary transthyretin amyloidosis (ATTRv-PN). In 2024, it generated $85 million in sales, bringing in $20 million in royalty income and has been launched in several countries.

3. Qalsody

Qalsody is an antisense oligonucleotide used to treat SOD1-ALS. The global sales in 2024 were $32 million, generating $4 million in royalty income. The sales showed a quarter-over-quarter growth trend, and it has been approved for market launch in China and Japan.

4. Tryngolza

Tryngolza (generic name: volanesorsen) is a subcutaneous antisense oligonucleotide used to treat familial chylomicronemia syndrome (FCS), and was approved by the US FDA for market launch on December 19, 2024. It has already started selling as an adjunct to diet therapy.

5. Tegsedi

Tegsedi is an antisense oligonucleotide drug developed in collaboration with Akcea Therapeutics. It is primarily used to treat hereditary transthyretin amyloidosis (hATTR) polyneuropathy in adults. Approved in 2018 in the EU, the US, and Canada, it is the second-generation antisense RNA therapy developed by Ionis and the first RNA-targeting drug for treating hATTR. 

In 2024, the combined revenue of Tegsedi? (inotersen) and Waylivra? (volanesorsen) was $34 million, down from $35 million the previous year.

As of December 31, 2024, Ionis' cash, cash equivalents, and short-term investments amounted to $2.3 billion. Ionis also provided guidance for its 2025 performance, expecting total revenue for 2023 to exceed $600 million.

About Ionis Pharmaceuticals

Ionis Pharmaceuticals Inc. is a leading developer of antisense technology for new drug development. As a biopharmaceutical company, it focuses on exploring and developing RNA-based drugs for its product line and its partners. The company’s drug development programs mainly target cardiovascular and metabolic diseases, as well as cancer, inflammation, and other diseases.

Ionis has a broad clinical and preclinical product pipeline treating various diseases, with a focus on cardiovascular, metabolic, and severe rare diseases.