Shares of Ionis Pharmaceuticals (NASDAQ: IONS) have fallen from roughly $86 in July to about $56, leaving the stock down approximately 30% year to date. Since early July, Ark Invest’s ARK Genomic Revolution ETF has purchased $15.3 million worth of Ionis shares, with all buying occurring after the surprise late-stage failure of eplontersen in ATTR-CM. Cathie Wood’s contrarian move has put the spotlight on whether the market has overreacted.
The second-quarter report was a mixed bag. Revenue came in at $268 million, down 41% from the prior-year period. However, the year-ago quarter included a one-time $280 million upfront payment from Ono Pharmaceutical. Excluding that item, revenue actually grew about 56% year over year. The company posted a net loss of $115 million, compared with net income of $124 million a year earlier, as commercialization and pipeline spending ramped up. Management reiterated full-year revenue guidance of $875 million to $900 million.
Tryngolza is now the central story. On June 24, the FDA approved the therapy for severe hypertriglyceridemia, expanding its label beyond the ultra-rare FCS indication to a U.S. patient population exceeding 3 million. In clinical trials, Tryngolza lowered triglycerides by up to 72% and reduced acute pancreatitis events by 85% to 91%, making it the only approved therapy shown to cut that specific pancreatic risk. Ionis also lowered the annual price from $595,000 to $40,000 to accelerate payer coverage. William Blair projects peak sales of $3 billion, supporting management’s goal of reaching cash flow breakeven by 2028.
Dawnzera, approved for hereditary angioedema prevention, posted a 63% sequential increase in sales and is nearing its first full year on the market. Elsewhere in the pipeline, the FDA has accepted the zilganersen new drug application with priority review for Alexander disease, and phase 3 data for pelacarsen, partnered with Novartis, is expected soon. The eplontersen miss removes potential profit sharing and milestones in ATTR-CM, though the drug has been approved since 2023 for another form of transthyretin amyloidosis.
Wall Street maintains a strong buy rating with a high target price of $115, implying potential upside of more than 100% from current levels. Risks remain: Tryngolza faces competition from Arrowhead Pharmaceuticals, commercialization costs are pressuring cash—down from $2.7 billion to $1.9 billion—and the company must rely on approved products after the eplontersen setback. Wood’s buying signals confidence in the long-term thesis, but execution will be key.
