Despite missing the primary endpoint in a pediatric study, Agios says it remains steadfast in working to grow the reach of its pyruvate kinase (PK) activation franchise.
Agios hopes to fill the treatment gap for children with PK deficiency with Pyrukynd (mitapivat), the first disease-modifying therapy to treat hemolytic anemia in adults with the rare blood disease.
The company's ACTIVATE-KidsT study represents the first trial to report safety and efficacy data in the pediatric PK deficiency population, but it fell short of meeting its primary goal.
Investigators studied the treatment in 49 patients between one and 18 years of age who receive regular transfusions for the rare blood disorder. In the end, the drug didn't meet the statistical significance bar in helping reduce patients' reliance on red blood cell transfusions on a measure called transfusion reduction response (TRR), the company said.
Nine out of 32 patients (28%) in the mitapivat arm achieved a TRR as outlined in the trial protocol compared to two out of 17 patients (12%) in the placebo group.
While the results were "clinically meaningful," Agios said they did not meet a "prespecified statistical criterion."
“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’ chief medical officer and head of R&D Sarah Gheuens, M.D., Ph.D., said in a release.
Next up is the ACTIVATE-Kids study in children with PK deficiency who are not regularly transfused. For that study, Agios has completed enrollment and expects a topline readout next year.
Approved in 2022 to treat hemolytic anemia in adults with PK deficiency, Pyrukynd generated $8.6 million in second-quarter revenues, a 5% increase from the first quarter. So far, 128 patients are on the therapy and 201 have completed prescription enrollment forms.
“We continue to make significant progress toward our vision of becoming a leading rare disease company with a potential multi-billion-dollar franchise in PK activation,” CEO Brian Goff said in a release.
Besides PK deficiency, Agios' drug recently met its endpoints in a late-stage trial in patients with transfusion-dependent alpha- and beta-thalassemia. The results will support an FDA filing by the end of the year, Goff noted.
In addition, Agios expects to finish enrollment in a late-stage sickle cell disease trial this year.