04:38 AM EST, 12/09/2024 (MT Newswires) -- Agios Pharmaceuticals ( AGIO ) said Sunday the phase 3 study of its experimental drug mitapivat to treat adults with transfusion-dependent alpha- or beta-thalassemia, a rare blood disease, met primary and secondary goals.

In the study, mitapivat demonstrated a statistically significant reduction in transfusion burden compared with placebo, the company said.

The company also said the study met all the key secondary endpoints, with mitapivat demonstrating a "statistically significant" reduction in additional measures of transfusion response compared with placebo.

Based on results from two phase 3 studies, the drug developer said it plans to submit regulatory applications for mitapivat to treat transfusion-dependent and non-transfusion-dependent thalassemia patients in the US, EU, Saudi Arabia and the UAE.