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1. Company Snapshot

1.a. Company Description

Ionis Pharmaceuticals, Inc.discovers and develops RNA-targeted therapeutics in the United States.The company offers SPINRAZA for spinal muscular atrophy (SMA) in pediatric and adult patients; TEGSEDI, an injection for the treatment of polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; and WAYLIVRA, a treatment for familial chylomicronemia syndrome and familial partial lipodystrophy.


It also develops medicines for various indications that are in phase 3 study, including Eplontersen as a monthly self-administered subcutaneous injection to treat all types of TTR amyloidosis; Olezarsen for patients with severe hypertriglyceridemia (SHTG); Donidalorsen for patients with hereditary angioedema; ION363 for patients with amyotrophic lateral sclerosis; Pelacarsen for patients with established cardiovascular disease and elevated lipoprotein(a); and Tofersen to inhibit the production of superoxide dismutase 1.In addition, the company develops medicines for metabolic diseases, infectious diseases, renal diseases, ophthalmic diseases, and cancer.It has a strategic collaboration with Biogen Inc.; and collaboration and license agreement with AstraZeneca, Bayer AG, GlaxoSmithKline plc, Novartis AG, Roche, Janssen Biotech, Inc., and Flamingo Therapeutics, Inc.


Ionis Pharmaceuticals, Inc.was founded in 1989 and is based in Carlsbad, California.

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1.b. Last Insights on IONS

Ionis Pharmaceuticals' recent performance was negatively impacted by a wider-than-expected Q4 loss of $1.14 per share, despite beating revenue estimates. The company's guidance for 2026 sales views was below expectations, which weighed on sentiment. Additionally, CEO Brett Monia's sale of 29,430 shares, valued at $2.5 million, may have raised concerns among investors. The company's adjusted loss of $1.15 in Q4 was better than the Wall Street estimate, but the soft 2026 revenue outlook and slower uptake for new drugs have raised concerns.

1.c. Company Highlights

2. Ionis Pharmaceuticals' 2025 Earnings: A Year of Significant Progress

Ionis Pharmaceuticals reported a robust financial performance in 2025, with revenue increasing by 34% year-over-year to $944 million. The company's commercial products generated $436 million in revenue, while R&D collaborations contributed $508 million. The earnings per share (EPS) came in at -$1.14, beating estimates of -$1.23667. TRYNGOLZA, launched in 2025, earned $108 million in product sales, with a 56% quarter-over-quarter increase in Q4. DAWNZERA, launched in August, also had an encouraging start with strong participation in its free trial program.

Publication Date: Mar -02

📋 Highlights
  • 2025 Revenue Growth: Ionis generated $944 million in revenue (34% YoY), with commercial products contributing $436 million and R&D collaborations $508 million.
  • TRYNGOLZA Performance: Achieved $108 million in full-year revenue with 56% Q4 QoQ growth, priced at $20,000 net, targeting >$2 billion peak sales.
  • DAWNZERA Launch: Launched in August 2025 with $8 million in product sales, leveraging a free trial program for market penetration.
  • 2026 Financial Projections: Anticipates $800–825 million revenue (20% growth), driven by olezarsen (potential >$2B annualized) and zilganersen (>$100M peak revenue).
  • Balance Sheet Strength: Ends 2026 with $1.6 billion in cash, targeting cash flow breakeven by 2028 amid expanding commercial and partnered pipelines.

Revenue Growth Drivers

The company's revenue growth was driven by its marketed medicines, with TRYNGOLZA and DAWNZERA contributing to total commercial revenue. Royalty revenues increased 11% to $285 million, driven by contributions from SPINRAZA and growing royalties from WAINUA. Ionis expects to continue this momentum in 2026, with projected revenue between $800 million and $825 million, representing approximately 20% growth over 2025.

Pipeline Progress and Upcoming Milestones

Ionis made significant progress in its pipeline in 2025, with positive Phase III results for olezarsen in severe hypertriglyceridemia. The company submitted a supplemental NDA and expects a decision from the FDA shortly, with launch preparations underway. Additionally, Ionis expects to launch zilganersen for Alexander disease in the second half of 2026, with an NDA submitted and acceptance expected soon.

Valuation and Growth Expectations

With a P/S Ratio of 13.93, Ionis' valuation reflects the market's expectations for the company's growth potential. Analysts estimate next year's revenue growth at 70.0%, indicating a significant increase in revenue. The company's ROE (%) is -68.82, and ROIC (%) is -12.05, indicating that Ionis is still in an investment phase. However, with a rich pipeline and multiple partnered programs advancing, Ionis is well-positioned to drive long-term value creation.

Future Outlook

Ionis is confident in its goal of accelerating revenue growth and achieving cash flow breakeven by 2028. The company expects to end 2026 with a well-capitalized balance sheet, including cash and investments of approximately $1.6 billion. With multiple value-driving events expected in 2026, including results from two major cardiovascular outcome trials and potential launches from its partnered pipeline, Ionis is poised for a pivotal year.

3. NewsRoom

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Ionis Pharmaceuticals, Inc. (IONS) Presents at TD Cowen 46th Annual Health Care Conference Transcript

Mar -02

Source: seekingalpha.com

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Ionis Pharmaceuticals, Inc. $IONS Shares Bought by Artisan Partners Limited Partnership

Feb -28

Source: defenseworld.net

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Ionis to present new data supporting DAWNZERA™ (donidalorsen) in hereditary angioedema at 2026 AAAAI Annual Meeting

Feb -27

Source: businesswire.com

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Ionis Pharmaceuticals: Digesting Recent Events

Feb -27

Source: seekingalpha.com

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Ionis Pharmaceuticals, Inc. (IONS) Presents at Oppenheimer 36th Annual Healthcare Life Sciences Conference Transcript

Feb -26

Source: seekingalpha.com

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Ionis Q4 Earnings & Sales Beat, Stock Down on Soft 2026 Outlook

Feb -26

Source: zacks.com

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Olezarsen sNDA accepted by the FDA for Priority Review for the treatment of severe hypertriglyceridemia (sHTG)

Feb -26

Source: businesswire.com

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Ionis Pharmaceuticals (IONS) Reports Q4 Earnings: What Key Metrics Have to Say

Feb -25

Source: zacks.com

4. Business Breakdown

4.a. Revenues by Country

4.b. Revenues by Segment

5. Expected revenues mid-term growth (10.43%)

6. Segments

Human Therapeutic Medicines

Expected Growth: 10.43%

Ionis Pharmaceuticals' Human Therapeutic Medicines segment growth of 10.43% is driven by increasing demand for Spinraza, a treatment for spinal muscular atrophy, and strong sales of Tegsedi, a treatment for polyneuropathy. Additionally, the company's pipeline of novel antisense medicines and collaborations with major pharmaceutical companies, such as Biogen and AstraZeneca, contribute to its growth momentum.

7. Detailed Products

SPINRAZA

A prescription medicine used to treat spinal muscular atrophy (SMA) in pediatric and adult patients.

TEGSEDI

A prescription medicine used to treat the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults.

WAYLIVRA

A prescription medicine used to treat familial chylomicronemia syndrome (FCS) in adults.

VOLanesorsen

An investigational antisense medicine for the treatment of familial chylomicronemia syndrome (FCS).

AKCEA-APO(a)-LRx

An investigational antisense medicine for the treatment of high lipoprotein(a) levels.

AKCEA-ANGPTL3-LRx

An investigational antisense medicine for the treatment of certain cardiovascular diseases.

8. Ionis Pharmaceuticals, Inc.'s Porter Forces

Forces Ranking

Threat Of Substitutes

Ionis Pharmaceuticals, Inc. has a moderate threat of substitutes due to the presence of alternative treatments and therapies for the diseases they are targeting. However, their proprietary antisense technology provides a competitive advantage.

Bargaining Power Of Customers

Ionis Pharmaceuticals, Inc. has a low bargaining power of customers due to the specialized nature of their products and the lack of alternative treatment options for patients.

Bargaining Power Of Suppliers

Ionis Pharmaceuticals, Inc. has a moderate bargaining power of suppliers due to the presence of multiple suppliers for raw materials and equipment. However, the company's reliance on a few key suppliers for certain components may limit their negotiating power.

Threat Of New Entrants

Ionis Pharmaceuticals, Inc. has a low threat of new entrants due to the high barriers to entry in the biotechnology industry, including the need for significant investments in research and development and regulatory approvals.

Intensity Of Rivalry

Ionis Pharmaceuticals, Inc. operates in a highly competitive industry with several established players, leading to a high intensity of rivalry. The company must continually innovate and invest in research and development to stay ahead of competitors.

9. SWOT Analysis

Strength

Weaknesses

Opportunities

Threats

10. Capital Structure

10.a. Balance Sheet

10.b. Weighted Average Cost of capital

Value
Debt Weight 78.99%
Debt Cost 4.74%
Equity Weight 21.01%
Equity Cost 5.99%
WACC 5.00%
Leverage 375.90%

11. Quality Control: Ionis Pharmaceuticals, Inc. passed 3 out of 9 key points

12.a Historical Valuation

12.b Price/Earnings Ratio

12.c Margin Valuation

12.d Peers Valuation

Peers Group Analysis

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Peers Metrics

12.e Scoring Insights

12.f DCF BETA

Parameters

Short Term Growth

Short term Time

Long-Term Growth

WACC

Target Price

78.66$

Current Price

78.66$

Potential

-0.00%

Expected Cash-Flows