Agios scraps sickle cell treatment after trial shows limited benefit

Truist's Gregory Renza said the discontinuation removes Agios' "second shot" in sickle cell disease and increases reliance on mitapivat, its other PK activator, as "the bar for differentiation" rises following strong late-stage results for Novo Nordisk's etavopivat.

"The setback today means that Novo is likely to remain best-in-class," said RBC Capital Markets analyst Luca Issi, adding etavopivat showed stronger efficacy data than tebapivat in reducing painful sickle cell complications.

Sickle cell disease is a painful, inherited blood disorder in which the body makes sickle-shaped hemoglobin, preventing red blood cells from properly carrying oxygen to the body's tissues.

"We remain focused on mitapivat, our foundational PK activator," Chief Medical Officer Sarah Gheuens said, adding that the company expects a U.S. approval later this year.

PK activators are designed to boost the activity of an enzyme that helps red blood cells produce energy.

The U.S. Food and Drug Administration's target action date for mitapivat in sickle cell disease is November 1.

In 2023, the FDA approved Vertex's and Genetix Biotherapeutics' gene therapies for sickle cell disease in patients 12 years and older.

The sickle cell disease mid-stage trial was designed to characterize the dose-response relationship of tebapivat and assess whether its profile was meaningfully differentiated from other drugs in the pyruvate kinase (PK) activator class.

Over the 12-week treatment period, improvements in hemoglobin levels and hemolysis - premature destruction of red blood cells - were observed across all tebapivat dose levels.

However, the trial did not demonstrate the level of differentiation required to support continued development, said the company.