Shares of Agios Pharmaceuticals AGIO have plunged 23% over the past six months, primarily due to pipeline and regulatory setbacks, which have dampened investor confidence in the company’s near-term growth prospects.
Agios Pharmaceuticals currently derives revenues solely from its lead drug, mitapivat. The drug is approved for the treatment of hemolytic anemia in adult patients with pyruvate kinase (PK) deficiency under the brand name Pyrukynd. AGIO is also developing mitapivat as a potential therapy for sickle cell disease (SCD) and thalassemia indications.
Over the past year, the stock has lost 8.8% against the industry’s 12.7% growth.
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Mixed Phase III Data on Mitapivat Study in SCD Reduced Confidence in AGIO
In November 2025, the company reported mixed top-line data from the phase III RISE UP study, which evaluated orally administered mitapivat in patients aged 16 years or older with SCD.
While the study achieved its primary endpoint of improving patients’ hemoglobin levels, it failed to achieve the other co-primary endpoint of reducing the annualized rate of sickle cell pain crises (SCPCs).
The results were also mixed across the key secondary endpoints. While Pyrukynd-treated patients showed statistically significant improvements in average hemoglobin concentration and indirect bilirubin levels, mitapivat fell short of improving patient-reported fatigue. These outcomes raised concerns about the drug’s commercial potential in the large SCD market and contributed to the stock’s sharp decline.
Agios intends to engage with the FDA in the first quarter of 2026 before filing for approval. The marketing application in the United States is expected to be filed after the FDA discussion.
Regulatory Delay by FDA for Thalassemia Indication
Investor sentiment was further pressured by a regulatory delay related to the company’s supplemental filing for mitapivat in thethalassemia indication.
In September 2025, the FDA extended the review timeline for the supplemental new drug application (sNDA) seeking label expansion for mitapivat for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia by an additional three months.
The extension was due to the FDA’s request for a Risk Evaluation and Mitigation Strategy (“REMS”) to address the potential risk of hepatocellular (liver) injury mentioned in the sNDA for Pyrukynd. The delay was not associated with any new efficacy or safety concerns, but it deferred anticipated revenues and introduced uncertainty around commercialization timelines.
However, mitapivat was eventually approved by the FDA for the thalassemia indication in December 2025 under the brand name “Aqvesme” and was commercially launched in January following the implementation of the AQVESME REMS program. Outside the United States, mitapivat continues to be marketed as Pyrukynd for both PK deficiency and thalassemia indications.
With the regulatory overhang now removed, investor focus is likely to shift toward the drug’s commercial uptake in thalassemia. The successful launch could support revenue growth and help restore confidence, creating a pathway for the stock to move higher over time.
Agios Pharmaceuticals, Inc. Price and Consensus
Agios Pharmaceuticals, Inc. price-consensus-chart | Agios Pharmaceuticals, Inc. Quote
AGIO’s Zacks Rank & Stocks to Consider
AGIO currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Catalyst Pharmaceuticals CPRX and Indivior Pharmaceuticals INDV, each currently sporting a Zacks Rank #1 (Strong Buy), and ANI Pharmaceuticals ANIP, which presently carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 60 days, estimates for Catalyst Pharmaceuticals’ 2026 earnings per share have risen from $2.55 to $2.82. CPRX shares have gained 3.2% over the past year.
Catalyst Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 35.19%.
Over the past 60 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have risen from $2.79 to $3.03. INDV shares have surged 214.9% over the past year.
Indivior Pharmaceuticals’ earnings beat estimates in each of the trailing four quarters, with the average surprise being 74.53%.
Over the past 60 days, estimates for ANI Pharmaceuticals’ earnings per share have increased from $8.28 to $9.00 for 2026. Over the past year, shares of ANIP have rallied 14.5%.
ANI Pharmaceuticals' earnings beat estimates in each of the trailing four quarters, with the average surprise being 22.21%.
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This article originally published on Zacks Investment Research (zacks.com).