Overview
- In the 207-patient Rise Up study, roughly two-thirds received mitapivat and one-third received placebo.
- Forty-one percent of treated participants achieved at least a 1 g/dL hemoglobin increase versus 3% on placebo, with responders averaging about a 1.6 g/dL gain.
- The annualized rate of vaso-occlusive pain crises was about 2.6 on mitapivat versus 3.1 on placebo, a difference that was not statistically significant.
- Agios reported liver test abnormalities but said the findings were not suggestive of drug-induced hepatocellular injury.
- Agios plans to discuss a potential filing with U.S. regulators, as its separate beta thalassemia application awaits an FDA decision expected on Dec. 7 and its shares fell sharply on the news.