GBT Presents New Data on the Long-Term and Real-World Use of Oxbryta (voxelotor) Tablets in Patients with Sickle Cell Disease at 62nd ASH Annual Meeting and Exposition

Final 72-Week Analyses of Phase 3 HOPE Study Demonstrate Durable Improvements in Hemoglobin Levels and Significant Improvements in Overall Health Status

Real-World Experience Study Results Consistent with HOPE Study and Show Improved Patient Health Status

SOUTH SAN FRANCISCO, Calif., Dec. 06, 2020 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced new data from the 72-week analyses of the Phase 3 HOPE Study of Oxbryta (voxelotor) tablets in patients with sickle cell disease (SCD). These data, as well as new findings from real-world experience studies of Oxbryta, are being presented at the all-virtual 62^nd American Society of Hematology (ASH) Annual Meeting and Exposition.

“We are pleased that the longer term, 72-week HOPE Study data are consistent with the previously reported 24-week primary analyses, confirm the durability of effect and justify the sustained use of Oxbryta for treatment of sickle cell disease,” said Ted W. Love, M.D., president and chief executive officer of GBT. “Since Oxbryta was approved in late 2019, we are also excited by the growing body of real-world evidence that shows similar increases in hemoglobin levels as were observed in the HOPE study and demonstrates that Oxbryta has the potential to significantly improve overall health status for patients with this devastating disease.”

72-Week Analyses of Phase 3 HOPE Study
The analyses of the complete data from the Phase 3 HOPE Study support the long-term use of Oxbryta to reduce anemia and hemolysis, with the potential to mitigate the associated morbidity and mortality of SCD.

An analysis of the 72-week data (Abstract #1716) demonstrated that Oxbryta at 1500 mg resulted in durable improvements in hemoglobin levels and markers of hemolysis over 72 weeks of treatment. A large majority of patients (approximately 90 percent) achieved a Hb improvement of >1 g/dL from baseline at one or more time points during the study as compared to placebo (approximately 25 percent). The study also found:

• Significant improvements in markers of hemolysis in indirect bilirubin and reticulocyte percentage.
• Consistent with the 24-week data previously reported, treatment with Oxbryta remained well tolerated. The most common side effects reported were headache, diarrhea, abdominal pain, nausea, arthralgia, rash and pyrexia.

“The underlying cause of sickle cell disease and the root of the devastating, life threatening complications of the disease is hemoglobin polymerization and the resulting anemia and hemolysis,” said Elliott Vichinsky, M.D., director of hematology/oncology at UCSF Benioff Children’s Hospital in Oakland, Calif. “The longer-term 72-week data presented at ASH this week provide additional support for the chronic use of this novel disease modifying therapy in the treatment of this serious condition.”