Agios’ next-gen PK drug fails blood cancer study but sickle cell disease ambitions remain undimmed

Agios Pharmaceuticals’ next-gen pyruvate kinase (PK) activator has failed a phase 2 study in a group of rare blood cancers, although the biopharma is still holding out hope in sickle cell disease (SCD).

The company was evaluating various once-daily doses of the oral therapy, called tebapivat, in a 24-week trial of 65 patients with lower-risk myelodysplastic syndromes (LR-MDS). The phase 2b study missed its primary endpoint as measured by the number of patients who didn’t require a blood transfusion for eight consecutive weeks.