By By Colin Kellaher
Shares of Agios Pharmaceuticals saw a significant rise in premarket trading on Wednesday following the announcement of positive outcomes from a late-stage study of its Pyrukynd anemia drug. The study demonstrated successful achievement of its goals in certain patients with thalassemia, an inherited blood disorder.
Phase 3 Study Highlights
Agios reported that the Phase 3 study of Pyrukynd in adults with non-transfusion-dependent alpha- or beta-thalassemia reached its primary endpoint of hemoglobin response. Additionally, the study also showed statistical significance for a pair of key secondary endpoints.
These study results suggest that Pyrukynd may hold potential as the first oral therapy for all non-transfusion-dependent thalassemia patients, including those with alpha- or beta-thalassemia.
Future Plans and Regulatory Approval
Agios stated that it anticipates announcing topline data from a separate Phase 3 study of Pyrukynd in adults with transfusion-dependent alpha- or beta-thalassemia by mid-2024. Furthermore, the company plans to submit the drug for regulatory approval as a thalassemia treatment by the end of this year.
Pyrukynd’s Current Approvals
Currently, Pyrukynd is approved in the U.S. for treating hemolytic anemia in adults with pyruvate-kinase deficiency. It is also approved in the European Union for the treatment of pyruvate-kinase deficiency in adults.
Stock Performance
Agios shares closed at $22.83 on Tuesday and experienced a 14% surge to $26 during premarket trading.
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