Global Blood Therapeutics is making a big gamble on its sickle cell drug

Late-stage trial results for Global Blood Therapeutics’ sickle cell disease therapy, released on Wednesday, were mostly positive.

Patients on the therapy, called voxelotor, showed a statistically significant improvement on the trial’s primary endpoint, measuring an increase in the oxygen-transporting protein hemoglobin, compared with the placebo.

And a majority of patients — 58% — on the higher dose of voxelotor demonstrated an increase in hemoglobin after 12 weeks of treatment, relative to 9% of patients on the placebo. That’s also better than a 35% response rate that the company had discussed with the Food and Drug Administration.

Those results came from Part A of the of the phase 3 HOPE study, which stands for Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization.

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