Why Is AGIO Stock Falling Today?

The biotech firm said it will discontinue development of its therapy for sickle cell disease following the outcome of a Phase 2 trial.

• Agios said the results failed to demonstrate a sufficiently differentiated profile from other pyruvate kinase activators.
• The company said the drug’s hemoglobin response rates and safety profile were consistent with the PK activator class but did not justify further investment in the program.
• The company is now turning its attention to Mitapivat, with a key regulatory decision expected by November.

Shares of Agios Pharmaceuticals (AGIO) fell more than 4% in early trading, after the biotech firm said it will discontinue development of its therapy for sickle cell disease after a Phase 2 trial showed disappointing results.

Why Is Agios Discontinuing Tebapivat For Sickle Cell Disease?

On Monday, Agios said it is discontinuing the development of Tebapivat after results failed to demonstrate a sufficiently differentiated profile from other pyruvate kinase (PK) activators, despite showing improvements in hemoglobin levels.

The company said the drug’s hemoglobin response rates and safety profile were consistent with the PK activator class but did not justify further investment in the program.

Response Rates From The Trial

The 12-week, placebo-controlled Phase 2 study enrolled 59 patients aged 16 years and older with sickle cell disease. The trial evaluated three once-daily dose levels of Tebapivat and measured hemoglobin response.

Response rates were 43.8%, 47.1%, and 29.4% across the three treatment groups, compared with 33.3% for placebo.

“These Phase 2 data further reinforce PK activation as a clinically validated mechanism in sickle cell disease, with Tebapivat demonstrating hematologic activity consistent with this class of medicine,” said Sarah Gheuens, Chief Medical Officer and Head of R&D, Agios.

Focus Shifts to Mitapivat

The company is now turning its attention to Mitapivat, its lead PK activator, which is under the United States Food and Drug Administration (FDA) Priority Review for the treatment of sickle cell disease.

Earlier this month, the FDA accepted Agios’ supplemental New Drug Application (sNDA) submitted under the accelerated approval pathway and assigned a PDUFA target date of Nov. 1, 2026.

Retail’s Take On AGIO

Retail sentiment surrounding AGIO turned ‘neutral’ from ‘bearish’ over the past 24 hours, amid ‘high’ message volumes.

The stock has seen strong buying interest so far this year, gaining more than 39%.

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