On Thursday, Agios Pharmaceuticals, Inc. (NASDAQ:AGIO) announced that the ACTIVATE-Kids Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are not regularly transfused achieved its primary endpoint of hemoglobin response.

Pyruvate kinase (PK) deficiency is a rare, inherited disorder that affects red blood cells.

The safety results were consistent with the safety profile for mitapivat previously observed for adult patients with PK deficiency who are not regularly transfused.

The study’s primary endpoint was hemoglobin response, defined as a ≥1.5 g/dL ​increase in hemoglobin concentration from baseline ​ sustained at two or more scheduled assessments at​ Weeks 12, 16, and 20.

• In addition, the pre-specified supportive analysis based on traditional methodology comparing the hemoglobin response rate for mitapivat versus placebo provided further evidence that the primary endpoint was met.
• 31.6% (6/19) of patients in the mitapivat arm achieved a hemoglobin response compared to 0% (0/11) of patients in the placebo arm; the 95% confidence interval for the difference in hemoglobin response rates between mitapivat and placebo was >0 (95% CI=10.8% to 52.7%). 
• In addition, improvements in changes from baseline for markers of hemolysis (indirect bilirubin, lactate dehydrogenase and haptoglobin) were observed in the mitapivat arm compared to the placebo arm.

In August 2024, Agios reported topline results from the ACTIVATE-KidsT Phase 3 study of mitapivat in children aged 1 to <18 years with PK deficiency who are regularly transfused.

• In the ACTIVATE-KidsT study, 28.1% of patients in the mitapivat arm achieved the primary endpoint of transfusion reduction response, compared to 11.8% of patients in the placebo arm.
• Transfusion-free and normal hemoglobin response were secondary endpoints in this study and were only observed in patients in the mitapivat arm.

Based on the clinically meaningful results observed in both the ACTIVATE-Kids and ACTIVATE-KidsT Phase 3 studies, Agios intends to submit a marketing application for mitapivat in pediatric patients with PK deficiency.

Concurrently, the company reported fourth-quarter Pyrukynd revenues of $10.7 million, up 20% sequentially, beating the consensus of $9.33 million. 223 unique patients have completed prescription enrollment forms, up 6% sequentially.

A total of 130 patients are on PYRUKYND therapy, inclusive of new prescriptions and continued therapy.

Cash, cash equivalents, and marketable securities as of December 31, 2024, were $1.5 billion.

Price Action: AGIO stock is down 1.86% at $32.20 at the last check on Thursday.

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