Agios Fails to Reach Primary Objective in Pediatric Study

In spite of the fact that it failed to meet the primary goal in a pediatric research, Agios has stated that it is continuing to make efforts to expand the scope of its pyruvate kinase (PK) activation portfolio.

Pyrukynd (mitapivat), the first disorder-modifying drug for treating hemolytic anemia in individuals with the rare blood condition, is being developed by Agios with the intention of addressing the limited therapeutic options available for children who have PK deficiency.

Although it was the first trial to disclose safety and effectiveness results in the pediatric PK deficient population, the ACTIVATE-KidsT research conducted by the firm did not succeed in accomplishing its primary objective.

To investigate the treatment, researchers looked at 49 children, ranging in age from one to 18 years old, who were receiving frequent transfusions for the blood condition. Transfusion reduction response (TRR) is a metric that was used to determine whether or not the treatment was successful in reducing patients’ dependency on red blood cell transfusions. The company said that the drug did not demonstrate statistical significance in this regard.

In comparison, just two out of 17 people (12%) in the placebo cohort were able to reach a TRR, whereas 9 out of 32 participants (28%) in the mitapivat group were able to obtain a TRR according to the study design.

The findings, according to Agios, did not satisfy a predetermined statistical requirement, despite the fact that they were clinically significant.

“With our focus on lifelong, debilitating rare diseases, we hope that this study will be the first of several pediatric studies to make a positive impact in the lives of children facing rare hemolytic anemias, including PK deficiency, thalassemia and sickle cell disease,” Agios’ CMO Sarah Gheuens stated in a release.

Following that is the ACTIVATE-Kids research, which will be conducted on children who have PK deficiency but do not get frequent transfusions. Agios has finished enrolling participants for the study, and anticipates receiving a topline report in the coming year.

Pyrukynd, which was granted approval in 2022 for the treatment of hemolytic anemia in adults with PK issues, earned $8.6 million in revenues during the second quarter, representing a 5% increase over the first quarter. Up to this point, 128 patients have been receiving the treatment, and 201 of them have filled out the prescription enrollment forms.

CEO Brian Goff stated in a release that the firm is consistently making strides to achieve its vision of becoming one of the world’s top rare disease firms with a possible billion-dollar PK activation franchise.

Agios’ medication reportedly fulfilled its objectives in a late-stage study for individuals with transfusion-dependent alpha- and beta-thalassemia. According to Goff, the findings will provide basis for a file with the FDA before the end of the year.

Additionally, Agios anticipates that recruitment for a late-stage sickle cell disease study will be completed by the end of this year.

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