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CARLSBAD, Calif. - Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) outlined its growth strategy at its 2025 Innovation Day in New York City, highlighting recent product launches and its pipeline development plans. The company’s stock has shown remarkable momentum, surging over 150% in the past six months and currently trading near its 52-week high of $70.14. According to InvestingPro analysis, the stock appears overvalued at current levels, with technical indicators suggesting overbought conditions.
The company has achieved two independent product launches in less than nine months and plans two more by 2026, according to CEO Brett P. Monia. Ionis also expects four partner-led launches by the end of 2027. With a market capitalization of $11 billion and revenue growth of 16% over the last twelve months, the company’s expansion strategy is gaining traction. For deeper insights into Ionis’s growth potential and comprehensive analysis, InvestingPro subscribers can access detailed research reports and additional metrics.
Ionis reported that its TRYNGOLZA (olezarsen) launch for familial chylomicronemia syndrome in the U.S. is showing strong patient uptake and favorable physician engagement. The drug recently received approval in the European Union, where Sobi will lead commercialization.
The company plans to submit a supplemental new drug application to the FDA by year-end for olezarsen to treat severe hypertriglyceridemia (sHTG) following positive Phase 3 trial results. Data showed up to 72% placebo-adjusted reduction in fasting triglycerides and an 85% reduction in acute pancreatitis events.
Ionis also reported an "encouraging start" to the U.S. launch of DAWNZERA (donidalorsen), its treatment for hereditary angioedema. The European launch, led by Otsuka, is expected in 2026.
The company’s neurology portfolio includes zilganersen for Alexander disease, with plans to submit a new drug application in Q1 2026. Ionis also shared positive 18-month extension data from its Phase 2 HALOS study of ION582 for Angelman syndrome.
With approximately $2 billion in cash and short-term investments, Ionis projects its recent and upcoming launches could generate more than $5 billion in potential annual peak revenue. The company expects to reach cash flow breakeven in 2028. Financial data from InvestingPro shows the company maintains a healthy current ratio of 2.87, indicating strong liquidity to support its growth initiatives, though it’s currently operating at a loss with negative earnings per share of $1.72.
The information in this article is based on a company press release statement.
In other recent news, Ionis Pharmaceuticals has been the focus of various analyst updates following significant developments in its treatment pipeline. Notably, the company received positive attention for its Phase 3 clinical trial data on zilganersen, a treatment for the ultra-rare Alexander disease, which led Guggenheim to raise its price target to $92 while maintaining a Buy rating. Similarly, H.C. Wainwright reiterated a Buy rating and upheld a $95 price target, citing the treatment’s disease-modifying benefits. Meanwhile, Leerink Partners increased its price target to $68, maintaining an Outperform rating, after the release of topline results for the same treatment.
Further bolstering Ionis Pharmaceuticals’ outlook, Goldman Sachs upgraded the stock from Sell to Neutral, with a new price target of $65. This upgrade was influenced by the company’s promising Phase 3 data for Tryngolza in severe hypertriglyceridemia, which is projected to achieve peak sales of $1.7 billion. Additionally, Stifel adjusted its price target for Ionis to $67 from $43, reflecting a reassessment of the TTR market and the competitive landscape. These developments underscore the growing confidence in Ionis Pharmaceuticals’ innovative treatments and potential market impact.
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