Key Points
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The biotech has a strong pipeline and is growing its revenue.
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In June, the FDA approved Ionis’s drug, Tryngolza, to treat severe hypertriglyceridemia.
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The new approval expands the drug’s target market to more than 3 million patients in the U.S. alone.
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Cathie Wood’s ARK Genomic Revolution ETF (NYSEMKT: ARKG) focuses on companies in the genomics sector, especially in healthcare. Since the beginning of July, Wood has, through this Ark Invest exchange-traded fund (ETF), bought $15.3 million worth of Ionis Pharmaceuticals (NASDAQ: IONS), a biotech company based in Carlsbad, California.
The stock is down nearly 37% from its 2026 high earlier this month, with much of that decline coming after it and its partner AstraZeneca announced a surprise late-stage trial failure regarding eplontersen, a medicine used to treat patients with transthyretin amyloidosis cardiomyopathy, or ATTR-CM, a rare heart disease. All of Wood’s recent Ionis purchases came after that announcement, suggesting she is taking advantage of the stock’s decline to buy more shares while maintaining a long-term view that the company is worth investing in.
Let’s see what she may be seeing.
Ionis already had a big market expansion in June
In June 2026, the Food and Drug Administration (FDA) approved Ionis’s drug Tryngolza (olezarsen) for treatment of severe hypertriglyceridemia (sHTG), a condition characterized by elevated blood triglyceride levels. Tryngolza’s initial approval in 2024 was for familial chylomicronemia syndrome (FCS), a rare disease that causes high levels of triglycerides in the blood. FCS affects only about 3,000 people in the U.S., but the new sHTG approval expands its target market to more than 3 million potential patients in the U.S. alone.
In clinical trials, Tryngolza reduced triglyceride levels by up to 72% and, crucially, reduced life-threatening acute pancreatitis events by 85% to 91%. It is the only approved therapy for sHTG that has been shown to dramatically reduce this specific pancreatic risk, giving it a competitive advantage.
While its recent trial setback means that Ionis won’t be able to share profits, sales royalties, or future milestone payments for eplontersen for that indication, the drug was approved in 2023 to treat polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.
A shift to higher-margin, wholly owned commercialization
Historically, Ionis functioned as a research and development (R&D) engine, developing drugs and licensing them off to larger pharmaceutical companies, including Biogen and AstraZeneca, in exchange for royalty cuts.
Ionis is pivoting to focus on and commercialize its own assets. It is controlling the commercial launch of Tryngolza, which means Ionis captures the high-margin revenue directly. Despite proactively slashing the annual list price from $595,000 (its ultra-orphan drug price) to a highly accessible $40,000 per year to ensure rapid insurer coverage, Wall Street firms such as William Blair expect the massive sHTG volume to push Tryngolza’s peak sales to $3 billion. This supports Ionis’s guidance to reach the cash flow break-even point by 2028.
The company is already seeing an impact, with $246 million in revenue in the first quarter, up 86% year over year. It also trimmed its net loss from $146 million in the same quarter a year ago to $118 million.
The company increased its annual Tryngolza peak net sales guidance from $2 million to $3 million, reflecting the potential of the sHTG market.
One concern is the cost of commercializing Tryngolza and other therapies. In the first quarter, Ionis reported that its cash, cash equivalents, and short-term investments fell from $2.7 billion to $1.9 billion. While much of this drop was due to paying off maturing convertible debt, its operating expenses are scaling rapidly to fund launch preparations for Tryngolza, zilganersen, and other wholly owned assets.
Late-stage pipeline catalysts
Beyond Tryngolza, Ionis has a highly diverse, validated RNA-targeted pipeline with major readouts and regulatory catalysts lined up for the remainder of 2026 and 2027. In March, the FDA accepted its New Drug Application (with Priority Review) for zilganersen to treat Alexander disease, a rare neurological disorder.
Another high-profile phase 3 readout is expected shortly for pelacarsen, which is partnered with Novartis. The therapy is being looked at to lower the chance of a heart attack or stroke in people who already have heart disease and also have high levels of a specific, genetic type of cholesterol called Lipoprotein(a).
Because Ionis’s antisense oligonucleotide (ASO) platform is already clinically proven, its pipeline has a much higher probability of success than that of early-stage, speculative biotech firms.
A word of caution
While Ionis has a growing pipeline and a proven discovery platform, it is entering uncharted waters as it transitions to a commercial-stage biotech. A late-stage readout that falters or a revenue decline could send the stock swooning, and higher marketing costs could divert resources from the company’s R&D efforts.
Wood sees opportunity there, particularly at its current share price, but unprofitable biotech stocks pose risks for investors, particularly those who can’t afford to take a long-term position.
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James Halley has no position in any of the stocks mentioned. The Motley Fool has positions in and recommends AstraZeneca Plc and Ionis Pharmaceuticals. The Motley Fool recommends Biogen. The Motley Fool has a disclosure policy.
