Agios Pharmaceuticals' Rare Blood Disorder Drug Misses Primary Goal In Pediatric Study, Stock Slides

On Thursday, Agios Pharmaceuticals Inc AGIO released topline results from the global Phase 3 ACTIVATE-KidsT study of mitapivat in children aged 1 to <18 years with PK deficiency who are regularly transfused.

Pyruvate kinase deficiency is a rare genetic disorder characterized by the premature destruction of red blood cells.

Using Bayesian methodology, the prespecified statistical criterion for the primary endpoint in ACTIVATE-KidsT was not met using low or moderate borrowing of data from the ACTIVATE-T study in adults.

Also Read: Why Is Agios Pharmaceuticals Stock Trading Higher On Monday?

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 response and normal hemoglobin response were secondary endpoints in this study and were only observed in patients in the mitapivat arm.

Mitapivat was generally safe and well-tolerated, with safety results consistent with the safety profile for mitapivat previously observed in adults with PK deficiency who are regularly transfused.

Six patients (18.8%) in the mitapivat arm compared to 0 in the placebo arm had a transfusion-free response, defined as no red blood cell transfusions from Week 9 through Week 32 of the double-blind period.

Four patients (12.5%) in the mitapivat arm achieved a normal hemoglobin response compared to 0 in the placebo arm.

Pyrukynd (mitapivat) is a pyruvate kinase activator indicated for hemolytic anemia in adults with pyruvate kinase (PK) deficiency in the U.S. and PK deficiency in adult patients in the European Union.

In addition, Agios has completed enrollment in the ACTIVATE-Kids study of mitapivat in children with PK deficiency who are not regularly transfused and expects to report topline data in 2025.

In the second quarter, the company reported Pyrukynd sales of $8.6 million, up from $6.71 million a year ago, missing the consensus of $9.31 million. 

The company reported an EPS loss of $(1.69), up from $(1.51) a year ago and missing the consensus of $(1.60).

Price Action: AGIO stock is down 4.87% at $44.14 at last check Thursday.

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