Complete 72-Week Results from Phase 3 HOPE Study of Oxbryta (voxelotor) Tablets Published in The Lancet Haematology Show Significantly Improved Hemoglobin, Hemolysis and Overall Health Status in Sickle Cell Disease Patients

SOUTH SAN FRANCISCO, Calif., April 08, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced The Lancet Haematology has published the complete analysis of 72-week data from the Phase 3 HOPE Study of Oxbryta (voxelotor) tablets in patients with sickle cell disease (SCD). The results showed significant and sustained improvement in hemoglobin levels, reduction in hemolysis and improved overall health status in patients treated with Oxbryta. These findings support the long-term use of Oxbryta to reduce hemolytic anemia and hemolysis in SCD, potentially mitigating life-threatening complications of the condition.

Oxbryta, a first-in-class oral, once-daily therapy, directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. Oxbryta is approved in the United States for the treatment of SCD in patients ages 12 years and older.

“Sickle cell disease is a devastating disease that can lead to organ damage and a shortened life expectancy and is complicated by significant disparities in access to quality care,” said lead author Professor Jo Howard of Guy’s and St. Thomas’ NHS Foundation Trust and King’s College London. “Fortunately, we have entered a new era of treatment. The HOPE Study is the longest registrational trial to date among recently approved therapies for sickle cell disease, and these results further demonstrate that by sustainably improving both the hemolysis and anemia manifestations of the disease, Oxbryta has the potential to be a safe and effective disease-modifying treatment in patients with sickle cell disease.”

The HOPE Study, a randomized, double-blind, placebo-controlled, international, multicenter trial in 274 patients ages 12 to 65 years with SCD, showed treatment with the U.S. FDA-approved dose of Oxbryta 1500 mg resulted in rapid and durable improvements in hemoglobin levels throughout 72 weeks of treatment. Approximately 90 percent of patients treated with Oxbryta achieved a hemoglobin improvement of >1 g/dL from baseline at one or more time points during the study compared to placebo (25 percent). In addition, approximately 59 percent of patients treated with Oxbryta 1500 mg were able to achieve a hemoglobin increase of >2 g/dL and 20 percent achieved >3 g/dL at one or more time points, compared to approximately 3 percent and no patients in the placebo group, respectively. The analysis also showed that study participants treated with Oxbryta had numerically fewer vaso-occlusive crises (VOCs), consistent with the trends at 24 weeks, and were three times less likely to experience an acute anemic episode (decrease in hemoglobin >2 g/dL from baseline).