Company Reports

Ionis Pharmaceuticals is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.

Ionis posted revenue of $268 million in the second quarter, up 56% year over year, excluding last year's one-time sapablursen upfront payment. Ionis maintained its 2026 guidance of roughly $890 million of revenue at the midpoint, including Tryngolza (olezarsen) net product sales of $105 million.

Biogen and Ionis reported nuanced phase 2 data for diranersen in early Alzheimer's disease at the Alzheimer's Association International Conference, which sent shares down 8% and 3%, respectively, on July 14. Biogen holds global commercialization rights, while Ionis is eligible for tiered royalties.

Ionis Pharmaceuticals is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.

Ionis and AstraZeneca's phase 3 study of Wainua (eplontersen) in ATTR-CM missed its primary endpoint, showing no statistically significant benefit when added to standard of care. This drove significant stock movement with Ionis and AstraZeneca falling nearly 24% and 6%, respectively on July 9.

Ionis received US approval for the label expansion of Tryngolza in adults with severe hypertriglyceridemia (triglyceride levels of 500 mg/dL or higher). Tryngolza had already been approved in the much rarer indication of familial chylomicronemia syndrome. Shares traded up 2% after hours on June 24.

Ionis posted first‑quarter revenue of $246 million, up 86% year over year. Management raised its peak sales outlook for olezarsen to over $3 billion, from at least $2 billion as announced in January, and increased its 2026 revenue guidance by 9% at the midpoint.

Ionis Pharmaceuticals is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.

Ionis generated $944 million in 2025 revenue, up 34% year over year, driven in part by $108 million in first-year sales of Tryngolza (for familial chylomicronemia syndrome). Despite strong top-line growth, Ionis ended the year with an adjusted operating loss of $248 million.

Ionis Pharmaceuticals is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.

Ionis' third quarter delivered $116 million in commercial revenue, including $32 million in sales for Tryngolza, for familial chylomicronemia syndrome (up 68% sequentially). Management raised its 2025 revenue guidance by 6% at the midpoint and lowered operating loss guidance by 8% at the midpoint.

Ionis reported top-line data from the phase 3 study of olezarsen in severe hypertriglyceridemia. Compared with the placebo, olezarsen lowered triglycerides by up to 72% and resulted in an 85% reduction in acute pancreatitis events. Ionis' shares rose nearly 35% on Sept. 2.

Ionis is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.

Ionis received approval from the US Food and Drug Administration for Dawnzera (pipeline candidate donidalorsen), marking the first and only RNA-targeted prophylactic treatment for hereditary angioedema, or HAE.

Ionis' second quarter delivered $103 million in commercial revenue, including $19 million in sales for recently approved Tryngolza, for familial chylomicronemia syndrome. Management raised its 2025 revenue guidance by 14% at the midpoint, and shares traded up nearly 5% on July 30.

Narrow-moat Ionis reported strong first-quarter results, highlighted by a 10% increase in revenue year over year, driven by the strong launch of Tryngolza (olezarsen). Ionis is tracking our expectations, and we maintain our fair value estimate of $69 per share. We view the shares as very undervalued, trading at an attractive entry point for long-term investors.

Narrow-moat Ionis Pharmaceuticals reported solid 2024 results highlighted by total revenue of $705 million and a portfolio that continues to make progress. Ionis is tracking our expectations, and we maintain our $69 fair value estimate. We view the shares as very undervalued, trading at an attractive entry point for long-term investors with a high degree of risk tolerance.

Ionis is a leader in RNA-based therapies. Its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.

Ionis Pharmaceuticals received approval from the US Food and Drug Administration for Tryngolza (pipeline candidate olezarsen). This is the first treatment approved for adults with familial chylomicronemia syndrome, a rare genetic disorder that leads to dangerously high triglyceride levels, risking life-threatening pancreatitis and other complications. We are maintaining our fair value estimate of $69 per share, and we view shares as very undervalued, currently trading in 5-star territory at an attractive entry point for long-term investors with a high degree of risk tolerance.

Ionis is a leader in RNA-based therapies, and its spinal muscular atrophy drug Spinraza, marketed by partner Biogen, is the first RNA-based therapy to achieve blockbuster status. The firm's antisense oligonucleotide, or ASO, technology faces strong competition from RNA interference technology emerging from Alnylam, Arrowhead, and Novo Nordisk (Dicerna), as well as gene editing and gene therapy pipelines at multiple firms. However, Ionis has built a massive pipeline of promising new drugs that are rapidly moving toward the market, securing a narrow moat.