12 Most Promising Biotech Stocks According to Wall Street Analysts

2. Ionis Pharmaceuticals, Inc. (NASDAQ:IONS)

Analyst Upside: 77.30% 

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) develops RNA-targeted therapeutics for neurological, cardiovascular, and rare diseases. It generates revenue from royalties on drugs like SPINRAZA, sales of TEGSEDI and WAYLIVRA, and research collaborations. The company’s innovative approach uses modified RNA to target genetic causes of diseases, offering potential breakthroughs for conditions with limited treatments.

Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) reported strong financial results for Q4 and full-year 2024, with total revenue of $227 million for Q4 and $705 million for the year. The increase in commercial revenue was mainly driven by the launch of new products like WAINUA and TRYNGOLZA. Despite a decrease in R&D revenue due to the winding down of joint development for WAINUA, the business maintains a solid cash position of $2.3 billion.

The corporation’s operating expenses for Q4 2024 were $337 million, with a total of $1.18 billion for the year, reflecting the company’s ongoing commercialization efforts for new products. R&D expenses remained stable as Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) continued to invest in its promising pipeline. Key target markets include Familial Chylomicronemia Syndrome (FCS), Hereditary Transthyretin-Mediated Amyloidosis (ATTRv-PN), Spinal Muscular Atrophy (SMA), and Severe Hypertriglyceridemia (sHTG). Notably, TRYNGOLZA became the first-ever treatment for FCS, and WAINUA is gaining traction for ATTRv-PN.

Recent developments include the FDA approval of TRYNGOLZA in December 2024, Ionis Pharmaceuticals, Inc. (NASDAQ:IONS)’s first independent commercial product, and significant progress for WAINUA with regulatory milestones in Europe. Additionally, ION582, a treatment for Angelman syndrome, is on track for Phase 3 development in 2025.