Phase III HOPE study of GBT 440 shows significant impact on sickle cell disease.- Global Blood Therapeutics.

Global Blood Therapeutics announced the completion of a planned review of Part A of the Phase III HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, which is evaluating GBT 440 (voxelotor) for the treatment of sickle cell disease (SCD). On the primary endpoint (the proportion of patients with greater than 1 g/dL increase in hemoglobin versus baseline), a statistically significant increase was demonstrated with voxelotor at both the 1500 mg and 900 mg doses after 12 weeks of treatment versus placebo. It showed that 58 percent of patients taking the 1500 mg dose (p<0.0001) and 38 percent of patients taking the 900 mg dose p="0.0021)" achieved a greater than 1 g dl increase in hemoglobin at 12 weeks versus 9 percent of patients taking placebo. this compares favorably to the hemoglobin increase assumption agreed to with the u.s. food and drug administration fda in the hope study protocol of a 35 percent response. statistically significant and dose-dependent improvements in hemoglobin reticulocytes and bilirubin occurred with both voxelotor doses further demonstrating an improvement in hemolytic anemia.>

Improvements in these clinical measures of anemia and hemolysis were similar in patients with or without background use of hydroxyurea. Approximately 64 percent of patients enrolled in Part A are on background use of hydroxyurea. There were numerically fewer VOC episodes in both voxelotor groups than in the placebo group, which as anticipated did not reach statistical significance due to limited patient follow-up. The patient reported outcomes (PRO) data were difficult to interpret due to low baseline symptom scores and high inter-subject and intra-subject variability. Given this, GBT does not plan to utilize the PRO as a key secondary endpoint. Voxelotor was generally safe and well tolerated with similar safety profiles between the two doses. There was no evidence of tissue hypoxia at either dose.