Pipeline Milestones And Expanding Patient Pools Will Broaden Market Reach

Key Takeaways

• Expanding into larger patient populations and strong physician acceptance position Ionis for substantial market share gains and sustained top-line growth.
  
• Strategic partnerships, growing royalties, and a competitive edge in rare disease commercialization enhance financial resilience and support margin expansion.
  
• Expanding into broader indications and dependence on key late-stage assets heighten pricing pressures, regulatory risks, and limit revenue and margin growth amid market and partner challenges.

Catalysts

A commercial-stage biotechnology company, provides RNA-targeted medicines in the United States.

• The rapid revenue growth and positive launch trajectory for Tryngolza in familial chylomicronemia syndrome (FCS), along with the imminent launch of Donidalorsen for HAE and multiple late-stage pipeline assets reading out or launching by 2027, are set to drive sustained, stepwise increases in top-line revenue and operating leverage as Ionis transitions from R&D-heavy to commercial-stage.
  
• Expanding addressable patient populations-from rare diseases to larger segments like severe hypertriglyceridemia (sHTG)-combined with favorable physician feedback and significant unmet need, position Ionis to capture substantial market share and revenue growth from trends tied to the rise in chronic disease and an aging population.
  
• Greater acceptance of RNA-targeted and antisense oligonucleotide (ASO) therapies among physicians, regulators, and payors, demonstrated by positive reimbursement outcomes, coverage expansions, and robust adoption, supports Ionis' potential to improve gross and net margins over time as its innovative therapies become increasingly mainstream.
  
• Ionis' growing stream of high-margin royalty and milestone revenues from partnerships (e.g. Biogen, AstraZeneca, Roche), as well as recent substantial out-licensing deals, provides both capital efficiency and financial resilience-helping shore up net margins and reducing risk as operating expenses scale with new launches.
  
• The company's proven ability to rapidly identify, educate, and enroll patients in rare diseases-leveraging omnichannel and specialty networks-signals a first-mover and competitive advantage in precision medicine, supporting revenue durability and premium pricing amid expanding payor willingness to reimburse high-value, personalized therapies.

Ionis Pharmaceuticals Future Earnings and Revenue Growth

Assumptions

• Analysts are assuming Ionis Pharmaceuticals's revenue will grow by 16.7% annually over the next 3 years.
• Analysts are not forecasting that Ionis Pharmaceuticals will become profitable in next 3 years. To represent the Analyst Price Target as a Future PE Valuation we will estimate Ionis Pharmaceuticals's profit margin will increase from -28.4% to the average US Biotechs industry of 16.1% in 3 years.
• If Ionis Pharmaceuticals's profit margin were to converge on the industry average, you could expect earnings to reach $241.3 million (and earnings per share of $1.48) by about September 2028, up from $-268.2 million today.
• In order for the above numbers to justify the analysts price target, the company would need to trade at a PE ratio of 56.5x on those 2028 earnings, up from -35.9x today. This future PE is greater than the current PE for the US Biotechs industry at 15.5x.
• Analysts expect the number of shares outstanding to grow by 0.95% per year for the next 3 years.
• To value all of this in today's terms, we will use a discount rate of 6.98%, as per the Simply Wall St company report.

Ionis Pharmaceuticals Future Earnings Per Share Growth

Risks

• Ionis faces a significant risk of revenue compression as it expands Olezarsen from a rare-disease (FCS) to a broader (sHTG) indication, with management openly discussing that a price reduction will be necessary to address the much larger patient populations-potentially leading to a step-down in average net price and limiting long-term margin growth and earnings expansion.
  
• The company's increasing reliance on a few late-stage assets and upcoming FDA approvals (notably for Donidalorsen and Olezarsen in sHTG) highlights high regulatory and execution risk: setbacks or delays in pivotal data (e.g., insufficient acute pancreatitis outcomes, ambiguity around meaningful endpoints, or label negotiations) could disrupt the launch cadence and result in "lumpy" or stagnating revenue and net income.
  
• Slowdown in new patient identification beyond "low hanging fruit" populations (e.g., initial trial participants or previously diagnosed rare disease patients) suggests that longer-term growth in rare indications like FCS may be constrained by diagnosis rates and market penetration challenges, dampening the sustainability of revenue momentum.
  
• Ionis admits it is not currently building out ex-U.S. infrastructure and will continue to rely on third-party partners (e.g., Sobi, AstraZeneca) for global commercialization, meaning any changes in strategy, performance, or prioritization by these partners could materially impact royalties, milestone revenues, and overall revenue diversification.
  
• Anticipated large-scale pricing negotiations for expanded indications and payer resistance, especially as Ionis targets larger, less severe patient populations, expose the company to global drug pricing pressures and reimbursement complexities, elevating the risk that gross-to-net realization and ultimate revenue growth may fall short of expectations as biosimilars, generics, or competitive modalities (such as CRISPR and new RNA therapies) emerge.

Valuation

• The analysts have a consensus price target of $68.346 for Ionis Pharmaceuticals based on their expectations of its future earnings growth, profit margins and other risk factors. However, there is a degree of disagreement amongst analysts, with the most bullish reporting a price target of $96.0, and the most bearish reporting a price target of just $43.0.
• In order for you to agree with the analyst's consensus, you'd need to believe that by 2028, revenues will be $1.5 billion, earnings will come to $241.3 million, and it would be trading on a PE ratio of 56.5x, assuming you use a discount rate of 7.0%.
• Given the current share price of $60.49, the analyst price target of $68.35 is 11.5% higher.
• We always encourage you to reach your own conclusions though. So sense check these analyst numbers against your own assumptions and expectations based on your understanding of the business and what you believe is probable.