Global Blood reports Phase IIa data for voxelotor in sickle cell disease

Global Blood Therapeutics Inc. (NASDAQ:GBT) reported interim data from 11 evaluable patients with sickle cell disease in part B of the open-label, U.S. Phase IIa HOPE-KIDS 1 (GBT440-007) trial showing that once-daily 900 mg oral voxelotor (formerly GBT440) increased hemoglobin levels from baseline to week 16. Specifically, 55% of patients achieved an increase in hemoglobin of >1 g/dL, with a median increase in hemoglobin of 1.1 g/dL. Voxelotor also improved clinical measures of hemolysis at week 16 as measured by changes from baseline in reticulocytes and unconjugated bilirubin. No treatment-related serious adverse events were reported. Data were presented at the American Society of Hematology meeting in Atlanta.

Part A of the trial is evaluating a single dose of 600 mg GBT440, with pharmacokinetics defined as the primary endpoint. The secondary endpoint of part A is safety. Part B is evaluating once-daily doses of 900 and 1,500 mg GBT440 for 24 weeks in patients ages 12-17, with change in hemoglobin from baseline to week 24 defined as the primary endpoint. Part B’s secondary endpoints will assess the effect of multiple doses of GBT440 on clinical measures of hemolysis and pharmacokinetics...