Agios Touts Encouraging Action From Sickle Cell Disease Study With Mitapivat

Agios Pharmaceuticals Inc AGIO announced that the Phase 2 portion of the global Phase 2/3 RISE UP study of mitapivat in sickle cell disease met its primary endpoint of hemoglobin response.

These results support proceeding with the Phase 3 portion of the study.

79 patients were enrolled in the study, with 26 each in the 50mg and 100mg BID mitapivat arms and 27 in the placebo arm.

Both mitapivat doses demonstrated a significant increase in hemoglobin response rate compared to placebo.

46.2% of patients in the 50 mg arm and 50% in the 100 mg achieved a hemoglobin response, compared to 3.7% of patients in the placebo arm.

The annualized rates of sickle cell pain crises for patients in the 50mg and 100mg BID mitapivat arms were lower than those in the placebo arm.

No adverse events led to discontinuation in either the mitapivat or the placebo arms.

Of the 79 patients enrolled in the study, 73 continued into the Phase 2 open-label extension period.

Given promising results from both mitapivat dose arms, Agios plans to analyze the data further to select a dose for the Phase 3 study.

The Phase 3 portion of the RISE UP study is expected to enroll 198 patients, with the first patient to be enrolled in Q4 of this year, Phase 3 data to be reported in 2025, and potential U.S. approval in 2026.

In November, the European Commission approved Agios' Pyrukynd (mitapivat) for PK deficiency in adult patients, and it scored FDA approval for hemolytic anemia in adults with PK deficiency in February 2022.

Price Action: AGIO shares are down 1.38% at $26.43 on the last check Monday.

Disclaimer: This content was partially produced with the help of AI tools and was reviewed and published by Benzinga editor.