Wall Street Analysts Are Bullish on Top Healthcare Picks

Ionis Pharmaceuticals' Zanvastro, the first approved treatment for Alexander disease, targets the underlying genetic cause. Clinical trials showed improved motor function. The drug, developed with University of Wisconsin-Madison researchers, will be available in the U.S. and internationally via Recordati. Alexander disease is rare, affecting fewer than 1 in a million people.

Ionis Pharmaceuticals (IONS) shares rose 4.39% as investors reassess its prospects following recent FDA approvals and Phase 3 trial successes. The company's growing product slate and RNA-based medicine platform are noted, but cash burn and debt remain concerns. YTD performance is -45.27%.

Ionis Pharmaceuticals and Roche reported positive Phase 3 interim data for sefaxersen in treating primary IgA nephropathy, meeting proteinuria endpoints. The company is still loss-making, with a net loss of $565.2 million on $874.1 million revenue. Analysts project $2.2 billion revenue and $261.6 million earnings by 2029, assuming 36.3% annual growth. Success depends on execution, pricing, and partner coordination.

The FDA approved several neurology drugs in September 2026. Ionis Pharmaceuticals' Zilganersen (Zanvastro) was approved for Alexander disease, Scholar Rock's apitegromab-mstn (Isembyld) for SMA, Ultragenyx's rebisufligene etisparvovec-hopf (Fayuvi) for Sanfilippo A, IntraBio's levacetylleucine (Aqneursa) for ataxia-telangiectasia, and AbbVie's Tavapadon (Juvmo) for Parkinson's. Azurity Pharmaceuticals' Lamlique, a liquid lamotrigine, was also approved for epilepsy.

Sobi UK announced that NICE recommended Tryngolza (olezarsen) for treating familial chylomicronaemia syndrome (FCS) in adults in England and Wales. The recommendation follows positive Phase 3 trial results showing significant triglyceride reduction and fewer pancreatitis events. Sobi has exclusive rights to commercialize the drug in ex-U.S. markets except Canada and China, developed by Ionis Pharmaceuticals.

Ionis Pharmaceuticals (IONS) and partners reported positive Phase 3 results for two RNA-targeted medicines, ulefnersen and sefaxersen, meeting primary endpoints. The company's revenue was US$874.1 million with a net loss of US$565.2 million. Analysts project US$2.3 billion in revenue and US$222.2 million in earnings by 2029, with a 37.1% annual growth rate. The stock is down 42.4% year-to-date as of late September 2026.