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Ionis Pharmaceuticals Inc. (IONS)
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Ionis Pharmaceuticals (IONS) AI Stock Analysis

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IONS

Ionis Pharmaceuticals

(NASDAQ:IONS)

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Neutral 57 (OpenAI - 4o)
Rating:57Neutral
Price Target:
$76.00
▲(4.28% Upside)
Ionis Pharmaceuticals' overall stock score is driven by strong earnings call performance and positive technical indicators. However, financial performance and valuation concerns, including ongoing losses and high leverage, weigh down the score. The company's strategic advancements and product launches provide a positive outlook, but financial risks and valuation challenges remain significant.
Positive Factors
Revenue Growth
The consistent revenue growth reflects the company's expanding market presence and successful product adoption, supporting long-term business sustainability.
Product Launch Success
Successful product launches like Tringolza demonstrate the company's ability to innovate and capture market share, enhancing its competitive position.
Regulatory Approvals
Regulatory approvals expand market access and potential revenue streams, strengthening Ionis's position in the biopharmaceutical industry.
Negative Factors
Operating Loss
Ongoing operating losses highlight challenges in achieving profitability, which could impact long-term financial health and investment capacity.
High Leverage
High leverage indicates financial risk, potentially limiting strategic flexibility and increasing vulnerability to economic downturns.
Cash Flow Challenges
Negative cash flow growth can strain liquidity, impacting the company's ability to invest in R&D and future growth opportunities.

Ionis Pharmaceuticals (IONS) vs. SPDR S&P 500 ETF (SPY)

Ionis Pharmaceuticals Business Overview & Revenue Model

Company DescriptionIonis Pharmaceuticals, Inc. is a biopharmaceutical company headquartered in Carlsbad, California, specializing in the development of innovative RNA-targeted therapies. The company focuses on treating a range of diseases, including neurological disorders, cardiovascular diseases, and metabolic conditions, with a robust pipeline of drug candidates that leverage its proprietary antisense technology. Ionis collaborates with various pharmaceutical companies to enhance drug development and expand its therapeutic reach.
How the Company Makes MoneyIonis Pharmaceuticals generates revenue primarily through collaborations and partnerships with other pharmaceutical companies, which involve licensing fees, milestone payments, and royalties on sales of marketed products. The company has established significant collaborations, including those with Biogen, AstraZeneca, and Novartis, which contribute to its revenue through shared development costs and profit-sharing agreements. Additionally, Ionis earns revenue from the commercialization of its own products, including the marketed drug Tegsedi (inotersen) for the treatment of hereditary ATTR amyloidosis. The company’s revenue model is bolstered by its extensive pipeline of antisense oligonucleotide therapies, some of which are in late-stage development and have the potential for future commercialization.

Ionis Pharmaceuticals Key Performance Indicators (KPIs)

Any
Any
Revenue by Product
Revenue by Product
Shows revenue generated from individual products, highlighting which drugs are driving sales and indicating the company's reliance on specific treatments for growth.
Chart InsightsIonis Pharmaceuticals is experiencing a shift in its revenue dynamics. While Spinraza's revenue has been declining, the introduction of Tryngolza has shown strong early performance, significantly boosting revenue projections for 2025. The earnings call highlights Tryngolza's successful launch and increased revenue guidance, indicating a strategic pivot towards new product launches. However, challenges in patient identification and pricing strategy for other treatments could impact future growth. Overall, Ionis is leveraging its robust pipeline to drive revenue, with potential FDA approvals on the horizon further supporting its growth trajectory.
Data provided by:Main Street Data

Ionis Pharmaceuticals Earnings Call Summary

Earnings Call Date:Oct 29, 2025
(Q3-2025)
|
% Change Since: |
Next Earnings Date:Feb 25, 2026
Earnings Call Sentiment Positive
Ionis Pharmaceuticals demonstrated strong revenue growth and successful product launches, with significant advancements in their clinical pipeline. Despite these achievements, the company faces challenges with operating losses and patient identification for Tringolza. Overall, the positive developments outweigh the challenges.
Q3-2025 Updates
Positive Updates
Strong Revenue Growth
Ionis Pharmaceuticals reported a 17% increase in revenue year over year for the third quarter of 2025, totaling $157 million. The first nine months saw a 55% increase in revenue compared to the previous year.
Successful Launch of Tringolza
Tringolza, the treatment for familial chylomicronemia syndrome (FCS), achieved $32 million in net product sales in Q3, a nearly 70% increase from the second quarter.
Regulatory and Market Approvals
Tringolza received European approval, and Donzara was approved by the FDA for Hereditary Angioedema (HAE).
Positive Clinical Trial Results
Olazarsen showed significant reductions in triglycerides in severe hypertriglyceridemia (SHTG) patients, achieving a 72% reduction in triglycerides and an 85% reduction in acute pancreatitis events.
Advancements in Neurology
Zilgarnason showed the first-ever disease-modifying effect in Alexander disease, achieving a 33% mean benefit in gait speed.
Negative Updates
Operating Loss
Ionis Pharmaceuticals expects an operating loss between $275 million and $300 million for the full year 2025.
Challenges in Patient Identification
Despite the success of Tringolza, the vast majority of the estimated 3,000 people living with FCS in the US remain unidentified, indicating challenges in patient identification.
Company Guidance
During the Ionis Pharmaceuticals, Inc. Third Quarter 2025 Financial Results Conference Call, the company provided updated guidance reflecting their strong performance and strategic advancements. Ionis announced an increase in their 2025 financial guidance, projecting total revenue between $875 million and $900 million, a $50 million increase from previous guidance. TRINGOZA product sales are expected to reach $85 to $95 million for the year, driven by a nearly 70% quarter-over-quarter increase in net product sales. The company anticipates an operating loss between $275 million and $300 million, with a planned cash balance of over $2.1 billion by year-end. Ionis highlighted key achievements, including FDA approval and European launch plans for Tringolza, as well as the approval of Donzara for Hereditary Angioedema. Positive Phase III results for Olazarsen and Zilgarnason pave the way for future launches, with olanzarcen expected to be a blockbuster with over $1 billion in peak sales. The company remains focused on expanding its pipeline and achieving cash flow breakeven by 2028.

Ionis Pharmaceuticals Financial Statement Overview

Summary
Ionis Pharmaceuticals exhibits strong gross profit margins but struggles with profitability and cash flow sustainability. High leverage and negative returns on equity pose financial risks. While revenue shows modest growth, the company needs to address its profitability and cash flow issues to improve its financial health.
Income Statement
45
Neutral
Ionis Pharmaceuticals shows a strong gross profit margin of 98.78% in TTM, indicating efficient cost management. However, the company struggles with profitability, as evidenced by a negative net profit margin of -26.51% and EBIT margin of -19.71%. Revenue growth is modest at 2.43% TTM, but the consistent negative margins highlight ongoing challenges in achieving profitability.
Balance Sheet
40
Negative
The company's balance sheet reveals a high debt-to-equity ratio of 2.41 TTM, suggesting significant leverage and potential financial risk. Return on equity is negative at -44.32%, reflecting challenges in generating returns for shareholders. The equity ratio is not provided, but the high leverage indicates a need for improved capital structure.
Cash Flow
35
Negative
Ionis Pharmaceuticals faces cash flow challenges, with negative operating cash flow and free cash flow in TTM. The free cash flow to net income ratio is positive at 1.23, indicating some ability to cover net losses, but the overall negative cash flow growth and operating cash flow coverage ratio of -0.27 highlight liquidity concerns.
BreakdownTTMDec 2024Dec 2023Dec 2022Dec 2021Dec 2020
Income Statement
Total Revenue966.96M705.14M787.65M587.37M810.46M729.26M
Gross Profit955.14M693.92M778.51M573.25M799.61M717.32M
EBITDA-162.60M-353.72M-235.87M-227.76M-5.19M-111.19M
Net Income-256.34M-453.90M-366.29M-269.72M-28.60M-444.26M
Balance Sheet
Total Assets3.03B3.00B2.99B2.53B2.61B2.39B
Cash, Cash Equivalents and Short-Term Investments2.24B2.30B2.33B2.00B2.13B1.89B
Total Debt2.05B1.42B1.45B1.37B1.25B908.93M
Total Liabilities2.42B2.42B2.60B1.96B1.84B1.65B
Stockholders Equity618.00M588.35M386.69M572.89M771.74M743.28M
Cash Flow
Free Cash Flow-302.66M-546.23M-335.52M-294.47M12.90M-5.16M
Operating Cash Flow-247.01M-500.95M-307.51M-274.37M30.80M35.89M
Investing Cash Flow209.94M-134.03M-214.13M-262.64M194.91M274.48M
Financing Cash Flow40.55M478.06M644.08M-55.30M245.93M-596.61M

Ionis Pharmaceuticals Technical Analysis

Technical Analysis Sentiment
Positive
Last Price72.88
Price Trends
50DMA
65.21
Positive
100DMA
53.27
Positive
200DMA
42.66
Positive
Market Momentum
MACD
2.42
Positive
RSI
62.25
Neutral
STOCH
64.34
Neutral
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For IONS, the sentiment is Positive. The current price of 72.88 is above the 20-day moving average (MA) of 72.16, above the 50-day MA of 65.21, and above the 200-day MA of 42.66, indicating a bullish trend. The MACD of 2.42 indicates Positive momentum. The RSI at 62.25 is Neutral, neither overbought nor oversold. The STOCH value of 64.34 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Positive sentiment for IONS.

Ionis Pharmaceuticals Risk Analysis

Ionis Pharmaceuticals disclosed 33 risk factors in its most recent earnings report. Ionis Pharmaceuticals reported the most risks in the "Legal & Regulatory" category.
Finance & Corporate - Financial and accounting risks. Risks related to the execution of corporate activity and strategy
Latest Risks Added 0 New Risks

Ionis Pharmaceuticals Peers Comparison

Overall Rating
UnderperformOutperform
Sector (51)
Financial Indicators
Name
Overall Rating
Market Cap
P/E Ratio
ROE
Dividend Yield
Revenue Growth
EPS Growth
79
Outperform
$5.89B30.4919.66%116.09%35.29%
74
Outperform
$5.02B15.0422.35%1.08%3.40%
57
Neutral
$11.80B-39.99%20.39%34.00%
54
Neutral
$6.80B-28.87-275.50%69.83%22.16%
53
Neutral
$7.13B-11.642635.74%4.66%
51
Neutral
$7.86B-0.30-43.30%2.27%22.53%-2.21%
48
Neutral
$1.58B-5.60-21.37%64.89%-221.56%
* Healthcare Sector Average
Performance Comparison
Ticker
Company Name
Price
Change
% Change
IONS
Ionis Pharmaceuticals
72.88
34.08
87.84%
ALKS
Alkermes
30.39
2.02
7.12%
CYTK
Cytokinetics
59.62
7.10
13.52%
SRPT
Sarepta Therapeutics
16.20
-108.81
-87.04%
AXSM
Axsome Therapeutics
134.93
45.69
51.20%
KRYS
Krystal Biotech
203.26
28.85
16.54%

Ionis Pharmaceuticals Corporate Events

Ionis Pharmaceuticals Faces Investor Risks Amid Market Volatility and Milestone Uncertainties
Oct 31, 2025

Ionis Pharmaceuticals Inc. faces significant risks related to its securities and the global credit markets, particularly if it fails to progress in its programs as anticipated. The company’s share price is vulnerable to fluctuations based on the timing and achievement of various clinical, regulatory, and commercial milestones, which are often based on assumptions outside its control. Failure to meet these milestones or analysts’ forecasts could lead to a decrease in the market price of its common stock, affecting investor confidence. Additionally, the volatility of its securities could complicate liquidation efforts and increase the risk of financial loss for investors.

Ionis Pharmaceuticals Reports Strong Q3 2025 Growth
Oct 30, 2025

Ionis Pharmaceuticals Inc. is a biotechnology company focused on discovering and developing RNA-targeted therapies for serious diseases, with a strong presence in neurology and cardiometabolic disease sectors.

Ionis Pharmaceuticals Shines in Q3 2025 Earnings Call
Oct 30, 2025

Ionis Pharmaceuticals’ recent earnings call reflected a generally positive sentiment, underscored by strong revenue growth and successful product launches. The company made significant advancements in its clinical pipeline, although it continues to face challenges with operating losses and patient identification for its drug Tringolza. Overall, the positive developments seem to outweigh the challenges, painting an optimistic picture for the company’s future.

Ionis Pharmaceuticals’ Phase 3 Study on Angelman Syndrome: A Potential Game-Changer
Oct 27, 2025

Ionis Pharmaceuticals Inc. is conducting a Phase 3 study titled ‘REVEAL: A Phase 3 Study of ION582 in Angelman Syndrome’ to evaluate the efficacy and safety of ION582 in children and adults with Angelman syndrome. This genetic disorder is caused by a deletion or mutation of the UBE3A gene, and the study aims to provide a potential treatment option for affected individuals.

Ionis Pharmaceuticals Advances in MECP2 Duplication Syndrome Treatment
Oct 27, 2025

Ionis Pharmaceuticals Inc. is conducting a study titled ‘ATTUNE: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Participants With Methyl CpG Binding Protein 2 (MECP2) Duplication Syndrome (MDS).’ The primary goal is to assess the safety and tolerability of ION440, a promising treatment for MECP2 Duplication Syndrome, a rare genetic disorder. This study is significant as it addresses a critical unmet medical need.

Ionis Pharmaceuticals’ HALOS Study: A New Hope for Angelman Syndrome
Oct 27, 2025

Ionis Pharmaceuticals Inc. is conducting a clinical study titled HALOS: A Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION582 in Patients With Angelman Syndrome. The study aims to assess the safety and tolerability of ascending doses of ION582, a drug administered via intrathecal injection, in individuals with Angelman syndrome. This research is significant as it explores potential therapeutic options for a condition with limited treatment alternatives.

Ionis Pharmaceuticals’ Olezarsen Study: A Potential Game-Changer for Severe Hypertriglyceridemia
Oct 27, 2025

Ionis Pharmaceuticals Inc. is conducting an open-label extension study titled An Open-Label Extension Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Patients With Severe Hypertriglyceridemia (SHTG). The study aims to evaluate the safety and tolerability of olezarsen in patients with severe hypertriglyceridemia, a condition characterized by extremely high levels of triglycerides in the blood, which can lead to pancreatitis and other cardiovascular issues.

Ionis Pharmaceuticals’ Rocket Study: Advancing Research in Rare Disease Treatment
Oct 27, 2025

Study Overview: The Rocket Study, officially titled ‘Integrated Prospective and Retrospective Observational Study to Characterize Biomarkers and Disease Progression in Patients With Pelizaeus-Merzbacher Disease,’ aims to assess changes in specific biomarkers and neuroimaging parameters in patients with Pelizaeus-Merzbacher Disease (PMD). This study is significant as it seeks to inform the development of therapies for PMD, a rare genetic disorder affecting the central nervous system.

Ionis Pharmaceuticals’ Orbit Study: A Promising Step for Rare Disease Treatment
Oct 27, 2025

Study Overview: The Orbit Study, officially titled ‘A Phase 1b Study to Evaluate the Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION356 in Patients With Pelizaeus Merzbacher Disease,’ aims to assess the safety and tolerability of the drug ION356. This study is significant as it targets Pelizaeus-Merzbacher Disease (PMD), a rare genetic disorder affecting the central nervous system.

Ionis Pharmaceuticals’ ION464 Study: A Potential Breakthrough for Multiple System Atrophy
Oct 27, 2025

Ionis Pharmaceuticals Inc. is conducting a Phase 1 study titled ‘A Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of ION464 Administered Intrathecally to Adults With Multiple System Atrophy.’ The study aims to evaluate the safety and tolerability of ION464, a drug administered via intrathecal injection, in patients with multiple system atrophy (MSA). This study is significant as it explores a potential new treatment for MSA, a rare neurodegenerative disorder.

Business Operations and StrategyProduct-Related Announcements
Ionis Pharmaceuticals Reports Positive Study Results for Zilganersen
Positive
Sep 22, 2025

On September 22, 2025, Ionis Pharmaceuticals announced positive results from a pivotal study of zilganersen, an investigational medicine for Alexander disease (AxD), a rare neurological condition. The study showed statistically significant stabilization in gait speed and favorable safety, marking the first time a treatment has demonstrated a disease-modifying impact in AxD. Ionis plans to submit a new drug application to the FDA in early 2026, potentially transforming the treatment landscape for AxD and reinforcing Ionis’ position in addressing severe neurological diseases.

The most recent analyst rating on (IONS) stock is a Buy with a $95.00 price target. To see the full list of analyst forecasts on Ionis Pharmaceuticals stock, see the IONS Stock Forecast page.

Business Operations and StrategyProduct-Related Announcements
Ionis Pharmaceuticals Announces Positive Phase 3 Study Results
Positive
Sep 2, 2025

On September 2, 2025, Ionis Pharmaceuticals announced positive results from its Phase 3 CORE and CORE2 studies of olezarsen for severe hypertriglyceridemia (sHTG). The studies revealed significant reductions in fasting triglycerides and acute pancreatitis events, positioning olezarsen as a potential new standard for sHTG treatment. The results demonstrated a 72% reduction in triglycerides and an 85% reduction in pancreatitis events, with favorable safety and tolerability. Ionis plans to submit a supplemental new drug application to the FDA by the end of the year, marking a significant step in their efforts to address sHTG and expand their market presence.

The most recent analyst rating on (IONS) stock is a Buy with a $55.00 price target. To see the full list of analyst forecasts on Ionis Pharmaceuticals stock, see the IONS Stock Forecast page.

Business Operations and StrategyProduct-Related Announcements
Ionis Pharmaceuticals Gains FDA Approval for DAWNZERA
Positive
Aug 21, 2025

On August 21, 2025, Ionis Pharmaceuticals announced that the FDA approved DAWNZERA™ (donidalorsen) for preventing hereditary angioedema (HAE) attacks in patients aged 12 and older. DAWNZERA is the first RNA-targeted medicine for HAE, significantly reducing attack rates and offering a convenient dosing schedule. The approval was based on successful Phase 3 trial results, showing an 81% reduction in monthly HAE attack rates. This approval strengthens Ionis’ position in the market, offering a new treatment option for HAE patients, and is part of Ionis’ broader strategy to deliver life-changing medicines.

The most recent analyst rating on (IONS) stock is a Hold with a $45.00 price target. To see the full list of analyst forecasts on Ionis Pharmaceuticals stock, see the IONS Stock Forecast page.

Ionis Pharmaceuticals Reports Strong Earnings and Growth
Aug 1, 2025

Ionis Pharmaceuticals Inc. recently held its earnings call, revealing a strong overall performance characterized by significant revenue growth, successful product launches, and positive pipeline developments. Despite these achievements, the company faces challenges in patient identification and pricing strategy, alongside uncertainties in data outcomes.

Ionis Pharmaceuticals Reports Strong Q2 2025 Results
Jul 31, 2025

Ionis Pharmaceuticals Inc. is a biotechnology company specializing in RNA-targeted drug discovery and development, with a focus on creating innovative therapies for patients with severe diseases. The company operates primarily in the biopharmaceutical sector, known for its pioneering work in RNA-based medicines.

Glossary
BuyA stock rated as a "Buy" is expected to perform better than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock is likely to deliver higher returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
HoldA stock rated as a "Hold" is expected to perform in line with the overall market or a specific benchmark. This rating indicates that the stock is neither particularly compelling nor unfavorable for investment. Note: This is not investment advice; please consult a financial advisor before making investment decisions.
SellA stock rated as a "Sell" is expected to perform worse than the overall market or a specific benchmark over the near-to-medium term. This rating suggests the stock may deliver lower returns compared to other stocks in the same sector or market index. Note: This is not investment advice; please consult a financial advisor before making investment decisions.

Disclaimer

This AI Analyst Stock Report is automatically generated by our AI systems using advanced algorithms and publicly available financial, technical, and market data. While the information provided aims to be accurate and insightful, it is intended for informational purposes only and should not be considered financial advice. Any content created by an AI (Artificial Intelligence) system may contain inaccuracies and/or contain errors. Investing in stocks carries inherent risks, and past performance is not indicative of future results. This report does not account for your personal financial circumstances, objectives, or risk tolerance. Always conduct your own research or consult with a qualified financial advisor before making investment decisions. The analysis and recommendations provided are based on historical and current data and may not fully reflect future market conditions or unexpected developments. Neither the creators of this report nor its affiliated entities guarantee the accuracy, completeness, or reliability of the information presented. Use this report at your own discretion and risk.Date of analysis: Oct 31, 2025