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AGIO Halts Tebapivat Development in Myelodysplastic Syndromes
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Key Takeaways
AGIO halted tebapivat development in LR-MDS after a phase IIb study missed to meet efficacy targets.
Tebapivat showed biological activity and was generally well tolerated but lacked sufficient clinical benefit.
AGIO continues to evaluate tebapivat in a phase II sickle cell disease study, with top-line data due in 2H'26.
Agios Pharmaceuticals (AGIO - Free Report) announced that it has discontinued the development of its investigational drug, tebapivat, 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 demonstrate a sufficiently compelling clinical benefit to support further development in the indication. Tebapivat is a next-generation oral pyruvate kinase (PK) activator that uniquely targets both PKR and PKM2 isoforms, aiming to improve outcomes in rare blood disorders.
Myelodysplastic syndromes are a group of blood cancers in which the bone marrow produces abnormal or insufficient numbers of healthy blood cells, leading to conditions such as anemia, infections and bleeding problems.
AGIO’s shares declined 3.4% on Friday following the announcement. Year to date, Agios shares have risen 8.1% compared with the industry’s 0.4% growth.
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Key Highlights of AGIO’s Phase II Study for LR-MDS
The placebo-controlled phase IIb study evaluated once-daily tebapivat at the doses of 10mg, 15mg or 20mg over a 24-week treatment period in heavily pretreated patients with LR-MDS and anemia. The study’s primary endpoint was to determine whether patients could remain free from blood transfusions for at least eight consecutive weeks during the treatment.
Although tebapivat demonstrated biological activity and was generally well tolerated across all dose levels, the candidate failed to achieve Agios’ predefined efficacy threshold, which required a meaningful proportion of patients to attain durable transfusion independence. The company also concluded that the benefit-risk profile did not support advancing tebapivat into later-stage development for LR-MDS.
Agios’ Focus Shifts to SCD Program
The setback allows Agios to remain focused on exploring tebapivat’s potential in other indications. The company is evaluating tebapivat in a phase II study for the sickle cell disease (SCD) indication, with top-line data expected in the second half of 2026. The upcoming SCD data readout could be an important catalyst for tebapivat and Agios' broader rare-disease pipeline.
Over the past 30 days, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.00 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.33 from $3.69. INDV shares have risen 0.4% 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 2026 earnings per share have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have gained 79.4% 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, estimates for Immunocore’s 2026 loss per share have narrowed from 88 cents to 6 cents. Over the same period, EPS estimates for 2027 have risen from 24 cents to 87 cents. IMCR shares have lost 16.8% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 46.66%.
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AGIO Halts Tebapivat Development in Myelodysplastic Syndromes
Key Takeaways
Agios Pharmaceuticals (AGIO - Free Report) announced that it has discontinued the development of its investigational drug, tebapivat, 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 demonstrate a sufficiently compelling clinical benefit to support further development in the indication. Tebapivat is a next-generation oral pyruvate kinase (PK) activator that uniquely targets both PKR and PKM2 isoforms, aiming to improve outcomes in rare blood disorders.
Myelodysplastic syndromes are a group of blood cancers in which the bone marrow produces abnormal or insufficient numbers of healthy blood cells, leading to conditions such as anemia, infections and bleeding problems.
AGIO’s shares declined 3.4% on Friday following the announcement. Year to date, Agios shares have risen 8.1% compared with the industry’s 0.4% growth.
Image Source: Zacks Investment Research
Key Highlights of AGIO’s Phase II Study for LR-MDS
The placebo-controlled phase IIb study evaluated once-daily tebapivat at the doses of 10mg, 15mg or 20mg over a 24-week treatment period in heavily pretreated patients with LR-MDS and anemia. The study’s primary endpoint was to determine whether patients could remain free from blood transfusions for at least eight consecutive weeks during the treatment.
Although tebapivat demonstrated biological activity and was generally well tolerated across all dose levels, the candidate failed to achieve Agios’ predefined efficacy threshold, which required a meaningful proportion of patients to attain durable transfusion independence. The company also concluded that the benefit-risk profile did not support advancing tebapivat into later-stage development for LR-MDS.
Agios’ Focus Shifts to SCD Program
The setback allows Agios to remain focused on exploring tebapivat’s potential in other indications. The company is evaluating tebapivat in a phase II study for the sickle cell disease (SCD) indication, with top-line data expected in the second half of 2026. The upcoming SCD data readout could be an important catalyst for tebapivat and Agios' broader rare-disease pipeline.
AGIO’s Zacks Rank & Stocks to Consider
AGIO currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are Indivior Pharmaceuticals (INDV - Free Report) , Liquidia Corporation (LQDA - Free Report) , and Immunocore (IMCR - Free Report) , 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, estimates for Indivior Pharmaceuticals’ 2026 earnings per share have increased from $3.00 to $3.35. Over the same period, EPS estimates for 2027 have risen to $3.33 from $3.69. INDV shares have risen 0.4% 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 2026 earnings per share have increased to $2.97 from $1.50. Over the same period, EPS estimates for 2027 have risen to $4.81 from $2.91. LQDA shares have gained 79.4% 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, estimates for Immunocore’s 2026 loss per share have narrowed from 88 cents to 6 cents. Over the same period, EPS estimates for 2027 have risen from 24 cents to 87 cents. IMCR shares have lost 16.8% year to date.
Immunocore’s earnings beat estimates in three of the trailing four quarters and missed in the remaining one, with the average surprise being 46.66%.