Shares Agios Pharmaceuticals Inc.’s climbed after it was revealed a Phase 3 ENERGIZE-T study of mitapivat in adults with transfusion-dependent (TD) alpha-or beta-thalassemia achieved its primary endpoint of transfusion reduction response.

Statistical significance was also achieved for all key secondary endpoints evaluating additional measures of reduction of transfusion burden compared to placebo.

The 258-patient study met its primary endpoint of transfusion reduction response: TRR, defined as a ≥50% reduction in transfused red blood cell (RBC) units with a reduction of ≥2 units of transfused RBCs in any consecutive 12-week period through Week 48 compared with baseline.

Treatment with mitapivat demonstrated a statistically significant reduction in transfusion burden compared to placebo, as measured by the TRR endpoint.

In the mitapivat arm, 30.4% of patients achieved a transfusion reduction response, compared to 12.6% of patients in the placebo arm.

Treatment with mitapivat also demonstrated a statistically significant reduction in additional measures of transfusion reduction response compared to placebo.

In addition, a higher proportion of patients in the mitapivat arm (9.9%) than the placebo arm (1.1%) achieved the secondary endpoint of transfusion independence.

Overall, during the 48-week double-blind period, the incidence of adverse events (AEs) was similar across mitapivat and placebo arms.

Based on the safety and efficacy data observed in ENERGIZE-T, the company will proceed with the previously stated plans of U.S. regulatory submission by the end of this year.

The company intends to submit a marketing application for Pyrukynd (mitapivat) in the U.S. by the end of 2024 based on all available data from both studies, including Phase 3 ENERGIZE study.

The company also plans to submit marketing applications in Europe and the Gulf Cooperation Council (GCC) countries.

Price Action: AGIO shares are up 16.87% at $42.47 at last check Monday.