GBT Announces Positive 24-week Results from Phase 3 HOPE Study

Global Blood Therapeutics (Nasdaq:GBT) today announced updated efficacy and safety results from Part A of its Phase 3 HOPE Study of voxelotor in patients age 12 and older with sickle cell disease (SCD). Preliminary results from 154 adolescents and adults with SCD treated with voxelotor for 24 weeks demonstrated rapid, robust and sustained improvements in hemoglobin levels and measures of hemolysis with a favorable safety and tolerability profile.

As quoted in the press release:

“These longer-term efficacy and safety data from more than 150 patients enrolled in the HOPE Study continue to support the potential of voxelotor to be a disease-modifying treatment for SCD. These major improvements in anemia and hemolysis have the potential to prevent the chronic organ damage that is the leading cause of death in patients with SCD in the United States,” said Elliott Vichinsky, M.D., lead investigator of the HOPE Study and Director of Hematology/Oncology at UCSF Benioff Children’s Hospital in Oakland, Calif. “In addition to the large improvements in hemolytic anemia, I am greatly encouraged in seeing a very good safety profile.”

“These data from the Phase 3 HOPE Study, including the clinically meaningful and statistically significant increase in hemoglobin, have been a key element in the discussions with the FDA which led to the agency’s agreement with GBT’s proposal for voxelotor under the subpart H accelerated approval pathway,” said Ted W. Love, M.D., president and chief executive officer of GBT. “To further support our NDA submission, we are continuing to generate efficacy and safety data on an additional 118 patients in the HOPE Study.”

Click here to read the full press release.