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Agios Pharma Sets 2026 Strategic Priorities After AQVESME Approval

Story Highlights
  • Agios is launching newly approved AQVESME for thalassemia in the U.S. in 2026.
  • Agios plans multiple 2026 clinical milestones to expand its rare disease pipeline and path to profitability.
  • Looking for the best stocks to buy? Follow the recommendations of top-performing analysts.
Agios Pharma Sets 2026 Strategic Priorities After AQVESME Approval

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The latest update is out from Agios Pharma ( (AGIO) ).

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.

Spark’s Take on AGIO Stock

According to Spark, TipRanks’ AI Analyst, AGIO is a Neutral.

Agios Pharma’s overall stock score is driven by its financial performance challenges, particularly in profitability and cash flow, and bearish technical indicators. Positive sentiment from the earnings call and pipeline progress provide some support, but valuation concerns and regulatory risks weigh heavily.

To see Spark’s full report on AGIO stock, click here.

More about Agios Pharma

Agios Pharmaceuticals, based in Cambridge, Massachusetts, is a commercial-stage biopharmaceutical company focused on developing and delivering innovative medicines for rare diseases, with a core expertise in hematology and a growing pipeline targeting hematologic and other rare disorders. The company already has a commercial presence in thalassemia and pyruvate kinase deficiency and is positioning its pyruvate kinase activator franchise, including AQVESME (mitapivat) and tebapivat, as key drivers of long-term growth in high-value, underserved indications.

Average Trading Volume: 1,563,652

Technical Sentiment Signal: Sell

Current Market Cap: $1.64B

See more data about AGIO stock on TipRanks’ Stock Analysis page.

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