Agios Announces Phase 3 ACTIVATE-T Trial of Mitapivat Achieved Primary Endpoint in Adults with Pyruvate Kinase Deficiency Who Are Regularly Transfused - Seite 2

Results from the ACTIVATE-T trial were as follows:

• 37 percent of patients dosed with mitapivat (n = 10 of 27) achieved a ≥33% reduction in transfusion burden in the 24-week fixed dose period compared with individual historical transfusion burden standardized to 24 weeks (1-sided p=0.0002).
• 22 percent of patients dosed with mitapivat (n = 6 of 27) were transfusion-free during the 24-week fixed dose period.
• Treatment with mitapivat showed a reduction in the annualized total number of red blood cell units transfused during the study compared with the historical transfusion burden.
• The safety profile observed in the study was consistent with previously reported data.

Agios is conducting a full analysis of the ACTIVATE-T results and expects to submit the complete results of the trial for presentation at the European Hematology Association (EHA) Virtual Congress, which is being held June 9-17, 2021.

ACTIVATE-T Trial Design
ACTIVATE-T is a global, multicenter, open-label study to evaluate the efficacy and safety of mitapivat in adult patients with PK deficiency who are regularly transfused, defined as receiving six or more transfusions in the past 52 weeks. The trial enrolled 27 patients across North America, Europe and Asia.

The study was designed with two parts. Part 1 was a dose escalation period in which patients started at 5 mg twice daily of mitapivat, with two potential dose increases to 20 mg twice daily and 50 mg twice daily for up to 16 weeks. After the dose escalation period, patients received a fixed dose for an additional 24 weeks in Part 2.

The primary endpoint of the study was reduction in transfusion burden, defined as a reduction of ≥33 percent in the number of red blood cell units transfused during the 24-week fixed dose period compared with the historical transfusion burden standardized to 24 weeks. Participants who discontinued the study before completing at least 12 weeks of treatment in the fixed dose period were considered non-responders. The p-value is based on the binomial exact test of H₀: transfusion reduction response rate≤10% vs. H₁: transfusion reduction response rate>10% at a 1-sided α=0.025.