Agios' Pyrukynd falls short in initial pediatric blood disorder study

Agios Pharmaceuticals is committed to expanding the application of its pyruvate kinase (PK) activation franchise despite not meeting the primary endpoint in a recent pediatric study. The company aims to address the treatment gap for children with PK deficiency through its drug, Pyrukynd (mitapivat), which is the first disease-modifying therapy for hemolytic anemia in adults with this rare blood disorder.

The ACTIVATE-KidsT study, conducted by Agios, was the first trial to evaluate the safety and efficacy of Pyrukynd in children with PK deficiency. The trial included 49 patients aged between one and 18 who regularly receive blood transfusions due to the disorder. Unfortunately, the study did not achieve the primary objective of significantly reducing the reliance on red blood cell transfusions, known as transfusion reduction response (TRR). Only 28% of patients in the mitapivat group reached the TRR benchmark compared to 12% in the placebo group. Despite these results being "clinically meaningful," they did not meet the "prespecified statistical criterion."

Sarah Gheuens, M.D., Ph.D., the Chief Medical Officer and Head of R&D at Agios, expressed optimism about the future impact of their studies on children suffering from PK deficiency and other hemolytic anemias such as thalassemia and sickle cell disease. She indicated that the current study is just the beginning of several pediatric research endeavors aimed at improving the lives of children with rare blood disorders.

Moving forward, Agios has completed enrollment for the ACTIVATE-Kids study, which focuses on children with PK deficiency who do not require regular transfusions. The results of this study are expected to be available next year.

Pyrukynd, which was approved in 2022 for treating hemolytic anemia in adults with PK deficiency, generated $8.6 million in revenue during the second quarter, marking a 5% increase from the previous quarter. Currently, 128 patients are being treated with Pyrukynd, and 201 have completed prescription enrollment forms.

Brian Goff, the CEO of Agios, highlighted the company's progress towards becoming a leading entity in the rare disease sector with a potential multi-billion-dollar PK activation franchise. Beyond PK deficiency, Agios has achieved favorable outcomes in a late-stage trial for patients with transfusion-dependent alpha- and beta-thalassemia. These positive results will form the basis of an FDA filing planned for later this year. Additionally, Agios aims to complete enrollment in a late-stage trial for sickle cell disease before the end of the year.

In summary, Agios Pharmaceuticals is unwavering in its mission to enhance the reach of its PK activation franchise. Despite the setback in the pediatric PK deficiency trial, the company remains focused on developing effective treatments for rare blood disorders, with several promising studies and potential regulatory approvals on the horizon.

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