Shares of Agios Pharmaceuticals AGIO have risen 35% over the past six months, driven by growing investor confidence in the commercial potential of its two marketed products. Positive regulatory developments have strengthened the company's near-term growth outlook.
The company’s lead drug, mitapivat, is marketed under two brand names — Pyrukynd and Aqvesme. While Pyrukynd is approved for the treatment of hemolytic anemia in adult patients with pyruvate kinase (PK) deficiency, Aqvesme is approved for the treatment of anemia in adults with alpha- or beta-thalassemia. Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications. Agios is seeking to expand mitapivat’s label into sickle cell disease (SCD).
Year to date, the stock has gained 21.6% against the industry’s 1.5% decline.
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Mitapivat Drives Agios' Growth Engine
The biggest catalyst behind the stock rally was the FDA approval of Aqvesme (mitapivat) in December 2025 for the treatment of adult patients with alpha- or beta-thalassemia. With this approval, Aqvesme became the first FDA-approved drug for the treatment of non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. Although Agios did not provide the breakdown of its sales numbers in first-quarter 2026, it reported strong early adoption of Aqvesme, with prescription growth exceeding expectations and worldwide mitapivat revenues more than doubling year over year.
Investors have responded positively to Agios' efforts to expand mitapivat globally. While Pyrukynd was already approved in the European Union for PK deficiency, it secured approval in the thalassemia indication last month. The company has partnered with Avanzanite Bioscience B.V. to market the drug across the European Economic Area, the United Kingdom and Switzerland. In addition, regulatory approvals for thalassemia have already been secured across the Gulf Council Countries.
Beyond its approved indications, Agios is also seeking to expand the label of its mitapivat products. Last month, the company submitted a regulatory filing with the FDA seeking to expand mitapivat’s label to include the SCD indication.
Agios’ Expansion in the ITP Market
AGIO inked an agreement with Oscotec, a South Korea-based biotech, earlier this month in-licensing exclusive global rights to develop and commercialize cevidoplenib, a next-generation, late-stage, highly selective oral spleen tyrosine kinase inhibitor for immune thrombocytopenia (ITP).
The agreement expands and diversifies Agios’ rare hematology portfolio by adding a potential treatment for ITP, with an estimated peak annual U.S. sales potential of up to $1 billion. Agios intends to advance cevidoplenib into a phase III study for ITP, with initiation expected in the first half of 2028 following additional chemistry, manufacturing and controls work.
Agios’ LR-MDS Study Failure: A Major Setback
While the company’s development around mitapivat has been encouraging, it is not devoid of pipeline setbacks. Last month, Agios discontinued the development of tebapivat, a next-generation PK activator, for the treatment of patients with lower-risk myelodysplastic syndromes (LR-MDS). The decision was based on the findings from a mid-stage dose-finding study that failed to meet efficacy targets.
Despite this setback, Agios remains focused on exploring tebapivat’s potential in other indications. The company is evaluating tebapivat in a phase II study for the SCD indication, with top-line data expected in the second half of 2026.
Agios Pharmaceuticals, Inc. Price and Consensus
Agios Pharmaceuticals, Inc. price-consensus-chart | Agios Pharmaceuticals, Inc. Quote
AGIO’s Zacks Rank & Stocks to Consider
Agios currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Indivior Pharmaceuticals INDV, Liquidia Corporation LQDA and Immunocore IMCR, each currently sporting a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, earnings per share estimates for Indivior Pharmaceuticals remained unchanged at $4.05 for 2026 and $4.27 for 2027. INDV shares have risen 5.7% year to date.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 65.44%.
Over the past 30 days, estimates for Liquidia’s earnings per share remained unchanged at $2.97 for 2026 and $4.81 for 2027. LQDA shares have gained 99.1% year to date.
Liquidia’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 54.40%.
Over the past 30 days, earnings per share estimates for Immunocore’s 2026 were unchanged at 6 cents for 2026 and 87 cents for 2027. IMCR shares have lost 17.7% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, the average surprise being 46.66%.
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This article originally published on Zacks Investment Research (zacks.com).