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Agios Pharma (AGIO)
NASDAQ:AGIO

Agios Pharma (AGIO) AI Stock Analysis

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AGIO

Agios Pharma

(NASDAQ:AGIO)

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Neutral 49 (OpenAI - 5.2)
Rating:49Neutral
Price Target:
$28.00
▲(0.72% Upside)
The score is held down primarily by weak financial performance (losses and negative operating/free cash flow) and a bearish longer-term technical trend. Offsetting these, the earnings outlook is more constructive due to meaningful regulatory and clinical catalysts and evidence of commercial traction, with a positive corporate update around U.S. approval and launch plans.
Positive Factors
High gross margin
An 85% gross margin indicates the company’s therapies have low incremental production costs and substantial pricing power. This supports durable unit economics, enabling Agios to fund commercialization and R&D while maintaining margin sustainability as sales scale over the next several quarters.
Strong cash runway
About $1.3B of cash and investments provides a meaningful runway to support U.S. launches, REMS implementation, and multiple clinical programs. This reduces near-term financing risk, allowing strategic investments in commercial infrastructure and pipeline advancement over the next 2–6 months.
First FDA-approved thalassemia therapy
Achieving sole FDA approval for thalassemia is a structural competitive advantage: establishes lead product, prescriber relationships, and formulary positioning. A successful launch can create durable revenue streams and a platform for label expansions and further rare-disease commercialization.
Negative Factors
Negative operating and free cash flow
Persistent negative operating and free cash flow undermines self-sustaining growth despite strong margins. Over months, continued cash burn forces reliance on the cash runway or external financing, raising dilution or balance sheet risk if commercial uptake or trial milestones slip.
REMS requirement adding launch friction
A REMS requirement imposes additional monitoring, distribution and prescriber constraints that can slow adoption, complicate payer access, and increase commercial costs. Over the launch period, REMS can materially reduce uptake velocity and delay reaching projected sales milestones.
Rising operating expenses and weak ROE
Increasing R&D and SG&A while returns remain negative (ROE reported as -28%) pressures the path to profitability. Elevated spend to support launches and trials can extend the period before positive returns, requiring sustained commercial success or additional funding to justify long-term investment.

Agios Pharma (AGIO) vs. SPDR S&P 500 ETF (SPY)

Agios Pharma Business Overview & Revenue Model

Company DescriptionAgios Pharmaceuticals, Inc., a biopharmaceutical company, engages in the discovery and development of medicines in the field of cellular metabolism and adjacent areas of biology. The company offers PYRUKYND (mitapivat) an activator of both wild-type and a variety of mutant pyruvate kinase, PK, enzymes for the treatment of hemolytic anemias; and AG-946 that is in Phase I clinical study for treating hemolytic anemias and other indications. Agios Pharmaceuticals, Inc. was incorporated in 2007 and is headquartered in Cambridge, Massachusetts.
How the Company Makes MoneyAgios generates revenue primarily through the commercialization of its approved therapies, particularly IDHIFA and TIBSOVO, which contribute to sales revenue from patients and healthcare providers. Additionally, the company may receive milestone payments and royalties from partnerships with larger pharmaceutical companies for the development and commercialization of its drug candidates. Collaborations, licensing agreements, and research partnerships can also provide significant revenue streams, contributing to Agios's overall earnings. The company is focused on expanding its portfolio of therapies and may generate further income through pipeline products as they advance through clinical trials and gain regulatory approval.

Agios Pharma Earnings Call Summary

Earnings Call Date:Oct 30, 2025
(Q3-2025)
|
% Change Since: |
Next Earnings Date:Feb 12, 2026
Earnings Call Sentiment Positive
The earnings call reflects a positive sentiment with strong financial performance, global regulatory progress, and significant pipeline advancements. However, there are challenges with increased expenses and potential regulatory hurdles related to the REMS program.
Q3-2025 Updates
Positive Updates
PYRUKYND Revenue Growth
Third quarter net PYRUKYND revenue was $12.9 million, an increase of 44% compared to the third quarter of 2024 and a 3% increase compared to the second quarter of 2025.
Global Regulatory Approvals
PYRUKYND received approval for adults with thalassemia in Saudi Arabia and a positive CHMP opinion recommending marketing authorization in Europe for thalassemia treatment.
Strong Financial Position
Agios maintains a strong balance sheet with approximately $1.3 billion in cash and investments, enabling strategic investments in pipeline growth and potential U.S. launches.
Pipeline Progress
Enrollment completed for the Phase IIb trial of Tebapivat in lower-risk MDS, with top line data expected early next year. Progress continues in early-stage programs AG-181 for phenylketonuria and AG-236 for polycythemia vera.
Negative Updates
Increased Expenses
R&D expenses increased by $14.3 million compared to the third quarter of 2024, primarily due to increased clinical trial costs. SG&A expenses also rose by $2.7 million.
Potential REMS Program Impact
The FDA requested a REMS program for PYRUKYND due to hepatocellular injury risk, which could impact the U.S. launch strategy for thalassemia.
Company Guidance
During the Agios Pharmaceuticals third-quarter 2025 conference call, significant guidance was provided, highlighting multiple upcoming catalysts and financial metrics. The company is anticipating a PDUFA date for PYRUKYND (a PK activator) for thalassemia on December 7, following the FDA's request for a REMS program, and expects top-line results from the RISE UP Phase III trial for sickle cell disease by year-end. The third quarter saw $12.9 million in net revenue for PYRUKYND, marking a 44% year-over-year increase. The company maintains a strong financial position with approximately $1.3 billion in cash and investments, positioning them to support potential U.S. launches and advance their rare disease pipeline. Additionally, they achieved enrollment completion in the Phase IIb trial of Tebapivat for lower-risk myelodysplastic syndromes. The team has focused on building a capital-efficient global commercial model and is actively engaging with the thalassemia and sickle cell communities to ensure a successful launch, while continuing to explore strategic business development opportunities to expand their pipeline.

Agios Pharma Financial Statement Overview

Summary
Agios Pharma shows revenue growth potential with a 9.58% increase in TTM. However, profitability challenges persist with negative EBIT and EBITDA margins, and a net profit margin of 15.90% due to significant losses. The balance sheet is stable with low leverage, but cash flow inefficiencies and negative return on equity highlight areas for improvement.
Income Statement
45
Neutral
Agios Pharma shows a positive revenue growth trend with a 9.58% increase in TTM, indicating potential market expansion. However, the company struggles with profitability, as evidenced by negative EBIT and EBITDA margins, and a net profit margin of 15.90% due to significant losses. The gross profit margin remains strong at 85.10%, suggesting efficient cost management in production.
Balance Sheet
55
Neutral
The balance sheet reflects a stable financial structure with a low debt-to-equity ratio of 0.036, indicating low leverage and financial risk. However, the return on equity is negative at -28.35%, highlighting challenges in generating returns for shareholders. The equity ratio is healthy, suggesting a strong equity base relative to total assets.
Cash Flow
40
Negative
Agios Pharma's cash flow statement reveals challenges in cash generation, with negative operating and free cash flows. The free cash flow to net income ratio is slightly above 1, indicating that cash flow aligns with reported earnings. However, the negative operating cash flow to net income ratio suggests inefficiencies in converting earnings into cash.
BreakdownTTMDec 2024Dec 2023Dec 2022Dec 2021Dec 2020
Income Statement
Total Revenue44.79M36.50M26.82M14.24M0.000.00
Gross Profit37.81M32.33M17.32M12.54M-18.78M-18.77M
EBITDA-441.84M-420.08M-384.86M-380.48M-337.73M-310.53M
Net Income-401.26M673.73M-352.09M-231.80M1.60B-327.37M
Balance Sheet
Total Assets1.39B1.66B937.12M1.24B1.44B852.95M
Cash, Cash Equivalents and Short-Term Investments952.86M893.71M776.93M783.12M1.02B572.93M
Total Debt44.52M56.99M72.00M85.66M97.09M105.20M
Total Liabilities101.38M122.24M126.10M137.90M145.76M453.45M
Stockholders Equity1.28B1.54B811.02M1.10B1.29B399.50M
Cash Flow
Free Cash Flow-414.09M-391.53M-297.06M-314.36M-413.06M-304.87M
Operating Cash Flow-409.93M-389.84M-296.06M-309.48M-407.32M-290.76M
Investing Cash Flow237.95M363.44M239.57M243.26M1.25B75.75M
Financing Cash Flow10.96M14.44M5.43M2.35M-765.77M261.52M

Agios Pharma Technical Analysis

Technical Analysis Sentiment
Negative
Last Price27.80
Price Trends
50DMA
30.45
Negative
100DMA
35.03
Negative
200DMA
34.06
Negative
Market Momentum
MACD
-0.35
Negative
RSI
47.48
Neutral
STOCH
54.14
Neutral
Evaluating momentum and price trends is crucial in stock analysis to make informed investment decisions. For AGIO, the sentiment is Negative. The current price of 27.8 is above the 20-day moving average (MA) of 27.15, below the 50-day MA of 30.45, and below the 200-day MA of 34.06, indicating a neutral trend. The MACD of -0.35 indicates Negative momentum. The RSI at 47.48 is Neutral, neither overbought nor oversold. The STOCH value of 54.14 is Neutral, not indicating any strong overbought or oversold conditions. Overall, these indicators collectively point to a Negative sentiment for AGIO.

Agios Pharma Risk Analysis

Agios Pharma disclosed 53 risk factors in its most recent earnings report. Agios Pharma reported the most risks in the "Tech & Innovation" category.
Finance & Corporate - Financial and accounting risks. Risks related to the execution of corporate activity and strategy
Latest Risks Added 0 New Risks

Agios Pharma Peers Comparison

Overall Rating
UnderperformOutperform
Sector (51)
Financial Indicators
Name
Overall Rating
Market Cap
P/E Ratio
ROE
Dividend Yield
Revenue Growth
EPS Growth
65
Neutral
$5.63B-18.70-38.41%-58.35%
55
Neutral
$3.62B-15.59-39.94%
52
Neutral
$2.52B-2.56-91.06%-32.98%-18.46%
51
Neutral
$7.86B-0.30-43.30%2.27%22.53%-2.21%
49
Neutral
$1.68B-4.00-27.57%36.26%-158.98%
49
Neutral
$2.97B-9.391025.95%52.91%
46
Neutral
$2.85B-6.45-44.44%-5.30%
* Healthcare Sector Average
Performance Comparison
Ticker
Company Name
Price
Change
% Change
AGIO
Agios Pharma
27.80
-4.62
-14.25%
IBRX
ImmunityBio
3.95
1.01
34.35%
DNLI
Denali Therapeutics
18.78
-3.15
-14.36%
NAMS
NewAmsterdam Pharma Company
31.83
8.28
35.16%
RXRX
Recursion Pharmaceuticals
4.64
-1.89
-28.94%
APGE
Apogee Therapeutics
80.75
43.69
117.89%

Agios Pharma Corporate Events

Business Operations and StrategyProduct-Related Announcements
Agios Pharma Sets 2026 Strategic Priorities After AQVESME Approval
Positive
Jan 12, 2026

On January 12, 2026, Agios outlined its 2026 strategic priorities following the December 2025 U.S. approval of AQVESME (mitapivat) for anemia in adults with alpha- or beta-thalassemia, marking the first and only FDA-approved treatment for both non-transfusion-dependent and transfusion-dependent forms of the disease. The company is launching AQVESME in the U.S. with a REMS program in place due to hepatocellular injury risks and expects the drug to become commercially available in late January 2026, while also highlighting a path to profitability supported by its existing rare disease portfolio and the potential to exceed $1 billion in peak global sales. Agios plans to advance mitapivat into sickle cell disease with a U.S. regulatory submission anticipated after a pre-sNDA meeting in early 2026, report key 2026 clinical milestones for tebapivat in sickle cell disease and lower-risk myelodysplastic syndromes, and generate early-stage data in polycythemia vera and phenylketonuria, reinforcing its strategy to diversify across hematologic and rare diseases and strengthen its competitive position in the rare disease therapeutics market.

The most recent analyst rating on (AGIO) stock is a Buy with a $35.00 price target. To see the full list of analyst forecasts on Agios Pharma stock, see the AGIO Stock Forecast page.

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: Jan 12, 2026