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Ionis Pharmaceuticals (IONS)
NASDAQ:IONS
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Ionis Pharmaceuticals (IONS) AI Stock Analysis

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IONS

Ionis Pharmaceuticals

(NASDAQ:IONS)

Rating:52Neutral
Price Target:
$43.00
▲(0.84% Upside)
Ionis Pharmaceuticals' overall stock score is primarily impacted by its financial challenges, including persistent losses and high leverage. However, positive earnings call sentiment and recent corporate events provide a counterbalance, indicating potential for future growth. The technical analysis and valuation scores reflect a cautious market outlook.
Positive Factors
FDA Approval
FDA approval of Dawnzera for hereditary angioedema is a key milestone for Ionis, representing the second of four independent launches expected in the coming years.
Market Opportunity
Severe hypertriglyceridemia is an indication with a high unmet need and a potential $1B+ commercial opportunity.
Market Position
Dawnzera has become the HAE product with the longest dosing interval that is self-administered via an autoinjector.
Negative Factors
Clinical Data
Dawnzera's 55% attack-rate reduction at 24 weeks is not competitive compared to Takhzyro's 87% at 26 weeks.
Competitive Landscape
HAE is a very crowded space, with upcoming competitors offering long-term durability.
Safety Concerns
The label for Dawnzera includes a safety warning for anaphylaxis, which occurred in 2 patients during the clinical study.

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:Jul 30, 2025
(Q2-2025)
|
% Change Since: |
Next Earnings Date:Oct 30, 2025
Earnings Call Sentiment Positive
The earnings call reflected strong commercial and financial performance, driven by the success of the Tryngolza launch and a robust pipeline. Despite some uncertainties in clinical study outcomes and competitive landscapes, the overall sentiment is positive due to significant revenue growth and strategic execution.
Q2-2025 Updates
Positive Updates
Successful Launch of Tryngolza
Tryngolza, the first FDA-approved treatment for familial chyloymicronemia syndrome, exceeded revenue expectations with $19 million in net product sales for the second quarter, reflecting a threefold increase quarter-over-quarter.
Record Revenue and Increased Financial Guidance
Ionis reported $452 million in revenue for Q2, a twofold increase year-over-year, and raised the 2025 revenue guidance by $100 million to $850 million.
Strong Pipeline Progress
Anticipated FDA approval for Donidalorsen for HAE next month, with potential launches for Olezarsen and Zilganersen in the pipeline, representing significant revenue potential.
Early Commercial Success and Strategic Execution
The commercial team reached over 3,000 physicians, with favorable payer dynamics leading to over 90% of patients paying $0 out-of-pocket for Tryngolza.
Negative Updates
Uncertainty in AP Event Rates in sHTG Studies
CORE and CORE2 studies have not definitively provided information on the acute pancreatitis event rate in sHTG, complicating the understanding of potential study outcomes.
Regulatory and Competitive Challenges
Ongoing discussions with the FDA regarding Donidalorsen's labeling and the competitive landscape with existing treatments in the HAE market present challenges.
Company Guidance
During Ionis's second quarter 2025 financial results conference call, the company highlighted significant momentum, resulting in an upward revision of their 2025 financial guidance. The standout performance of Tryngolza, the first FDA-approved treatment for familial chylomicronemia syndrome (FCS), exceeded expectations with $19 million in net product sales for the quarter, marking a threefold increase from the previous quarter. The company now anticipates total 2025 Tryngolza revenues between $75 million and $80 million. The call also covered the anticipated FDA approval of Donidalorsen for hereditary angioedema, expected next month, which, alongside the strong Tryngolza performance, supports Ionis's elevated financial outlook. The company reported a twofold year-over-year revenue increase to $452 million for the quarter, contributing to a $154 million non-GAAP net income. This robust performance enabled Ionis to improve its operating loss projection to between $300 million and $325 million and forecast a year-end cash balance of approximately $2 billion.

Ionis Pharmaceuticals Financial Statement Overview

Summary
Ionis Pharmaceuticals is facing significant financial challenges, characterized by persistent losses, high leverage, and negative cash flows. Despite strong cost management, the inability to achieve net profitability and positive cash flow is concerning. The balance sheet reflects high debt levels and declining equity, limiting financial flexibility.
Income Statement
35
Negative
Ionis Pharmaceuticals has shown a trend of declining revenues and persistent losses. The TTM data shows a gross profit margin of 98.88%, indicating strong cost management relative to revenue. However, the company is experiencing significant operating losses, with a negative EBIT margin and net profit margin, highlighting profitability challenges. Revenue growth is inconsistent, with fluctuations year-on-year, and recent declines are concerning.
Balance Sheet
40
Negative
The company is heavily leveraged with a high debt-to-equity ratio of 2.96 in the TTM period. The stockholders' equity has been decreasing, indicating financial strain. Return on equity is negative due to net losses. Although cash and equivalents are substantial, the equity ratio of 16.90% indicates a weaker capital structure.
Cash Flow
30
Negative
Negative operating and free cash flows signal challenges in generating sufficient cash from operations. The operating cash flow to net income ratio is negative, reflecting cash flow issues. Free cash flow has been consistently negative, indicating ongoing capital expenditure and funding requirements, which could pose liquidity risks.
BreakdownTTMDec 2024Dec 2023Dec 2022Dec 2021Dec 2020
Income Statement
Total Revenue717.44M705.14M787.65M587.00M810.00M729.00M
Gross Profit701.33M693.92M778.51M573.00M799.00M717.00M
EBITDA-376.51M-475.08M-230.01M-227.76M-239.00K-107.88M
Net Income-458.03M-453.90M-366.29M-270.00M-29.00M-487.00M
Balance Sheet
Total Assets2.81B3.00B2.99B2.53B2.61B2.39B
Cash, Cash Equivalents and Short-Term Investments2.15B2.30B2.33B1.99B2.11B1.89B
Total Debt1.41B1.42B1.45B1.37B1.25B908.93M
Total Liabilities2.34B2.42B2.60B1.96B1.84B1.65B
Stockholders Equity475.73M588.35M386.69M572.89M771.74M743.28M
Cash Flow
Free Cash Flow-569.17M-546.23M-335.52M-294.47M12.90M-5.16M
Operating Cash Flow-501.78M-500.95M-307.51M-274.37M30.80M35.89M
Investing Cash Flow-39.67M-134.03M-214.13M-262.64M194.91M274.48M
Financing Cash Flow456.69M478.06M644.08M-55.30M245.93M-596.61M

Ionis Pharmaceuticals Technical Analysis

Technical Analysis Sentiment
Positive
Last Price42.64
Price Trends
50DMA
41.85
Positive
100DMA
37.08
Positive
200DMA
35.41
Positive
Market Momentum
MACD
0.31
Positive
RSI
52.87
Neutral
STOCH
29.38
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 42.64 is above the 20-day moving average (MA) of 42.43, above the 50-day MA of 41.85, and above the 200-day MA of 35.41, indicating a bullish trend. The MACD of 0.31 indicates Positive momentum. The RSI at 52.87 is Neutral, neither overbought nor oversold. The STOCH value of 29.38 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
82
Outperform
$4.78B13.9123.94%-0.17%23.84%
75
Outperform
$4.27B30.0515.61%116.09%35.29%
58
Neutral
$6.05B-280.68%69.83%22.16%
52
Neutral
$6.80B-59.83%16.04%30.61%
51
Neutral
$7.86B-0.26-41.41%2.22%22.87%-2.01%
49
Neutral
$1.78B19.61-4.76%64.89%-221.56%
47
Neutral
$4.23B435.49%2635.74%4.66%
* Healthcare Sector Average
Performance Comparison
Ticker
Company Name
Price
Change
% Change
IONS
Ionis Pharmaceuticals
42.64
-4.67
-9.87%
ALKS
Alkermes
28.97
0.85
3.02%
CYTK
Cytokinetics
35.33
-19.23
-35.25%
SRPT
Sarepta Therapeutics
18.20
-116.90
-86.53%
AXSM
Axsome Therapeutics
121.28
31.63
35.28%
KRYS
Krystal Biotech
147.70
-39.08
-20.92%

Ionis Pharmaceuticals Corporate Events

Product-Related AnnouncementsBusiness Operations and Strategy
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.

Product-Related AnnouncementsBusiness Operations and Strategy
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.

Executive/Board Changes
Ionis Pharmaceuticals Announces Leadership Transition
Neutral
Jun 12, 2025

On June 12, 2025, Ionis Pharmaceuticals announced the retirement of Richard Geary, Ph.D., effective January 2026, after a 30-year tenure during which he led the development of several key medicines. Holly Kordasiewicz, Ph.D., will succeed him as executive vice president and chief development officer, bringing her extensive experience in neurology drug development to support Ionis’ growth and innovation in the biotechnology sector.

Shareholder MeetingsBusiness Operations and Strategy
Ionis Pharmaceuticals Holds Annual Stockholders Meeting
Positive
Jun 10, 2025

Ionis Pharmaceuticals held its virtual Annual Meeting of Stockholders on June 5, 2025, where four proposals were considered. The stockholders elected directors to serve until the 2028 Annual Meeting, approved executive compensation on an advisory basis, authorized an increase in shares under the 2011 Equity Incentive Plan, and ratified the selection of Ernst & Young LLP as independent auditors for the 2025 fiscal year. These approvals reflect continued support for the company’s strategic direction and governance structure.

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: Sep 02, 2025