Ionis Pharmaceuticals Inc
XMUN:ISI
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Ionis Pharmaceuticals Inc
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Ionis Pharmaceuticals Inc
Ionis Pharmaceuticals is a drug company that makes medicines based on antisense technology, a way to turn off harmful proteins by targeting RNA before they are made. It spends most of its effort on discovering and developing treatments for serious diseases, especially rare genetic, neurological, cardiovascular, and metabolic conditions. Its pipeline is the main business asset: it creates drug candidates and then tests them through clinical development. Ionis usually makes money in two ways. It earns partnership income when larger pharmaceutical companies help fund development or commercialize a medicine, and it can also collect royalties, milestones, and, for some products, sales revenue. That means it is not a broad drug seller with many large in-house brands; it is more of a specialist research company that turns RNA science into medicines and often shares the later stages of development and marketing with partners. What makes Ionis different is its focus on antisense drugs, which can be designed to hit very specific disease targets. This gives it a clear role in the biotech value chain: it invents and advances the science, while partners often help with large-scale clinical trials, manufacturing, and sales. For investors, the business is easiest to think of as a research-driven platform company that aims to convert a steady stream of new drug ideas into licensing deals and marketed therapies.
Ionis Pharmaceuticals is a drug company that makes medicines based on antisense technology, a way to turn off harmful proteins by targeting RNA before they are made. It spends most of its effort on discovering and developing treatments for serious diseases, especially rare genetic, neurological, cardiovascular, and metabolic conditions. Its pipeline is the main business asset: it creates drug candidates and then tests them through clinical development.
Ionis usually makes money in two ways. It earns partnership income when larger pharmaceutical companies help fund development or commercialize a medicine, and it can also collect royalties, milestones, and, for some products, sales revenue. That means it is not a broad drug seller with many large in-house brands; it is more of a specialist research company that turns RNA science into medicines and often shares the later stages of development and marketing with partners.
What makes Ionis different is its focus on antisense drugs, which can be designed to hit very specific disease targets. This gives it a clear role in the biotech value chain: it invents and advances the science, while partners often help with large-scale clinical trials, manufacturing, and sales. For investors, the business is easiest to think of as a research-driven platform company that aims to convert a steady stream of new drug ideas into licensing deals and marketed therapies.
TRYNGOLZA launch: Ionis said the new severe hypertriglyceridemia launch is off to an encouraging start, with prescriptions on day 1, both doses in channel within about 1 week, and early payer coverage building as expected.
Guidance intact: Management reaffirmed full-year 2026 revenue guidance of $875 million to $900 million, including TRYNGOLZA product sales of $100 million to $110 million and DAWNZERA sales of $110 million to $120 million.
DAWNZERA momentum: DAWNZERA continued to gain share in hereditary angioedema, with second-quarter sales of $26 million, up 63% from Q1, and management said repeat prescribing is growing.
Pipeline catalysts: Ionis highlighted upcoming catalysts including Zilganersen's PDUFA date of September 22, Bepirovirsen's PDUFA date of October 26, and multiple data readouts at ESC later this year.
CARDIO-TTRansform miss: Management reiterated disappointment that eplontersen did not meet the primary endpoint in the overall ATTR cardiomyopathy population, but said analysis continues and a presentation is planned at ESC.
Cash and losses: The company ended Q2 with $2.1 billion in cash and still expects 2026 non-GAAP operating loss of $425 million to $475 million and cash flow breakeven in 2028.