First Patient in Phase 3 Hope Study in Sickle Cell

Global Blood Therapeutics Announces Enrollment of First Patient in Phase 3 HOPE Study in Sickle Cell Disease SOUTH SAN Francisco, Calif. – January 17, 2017 – Global Blood Therapeutics, Inc. (GBT) (NASDAQ:GBT) today announced enrollment of the first patient in the HOPE (Hemoglobin Oxygen Affinity Modulation to inhibit Hbs PolymErization) Study, a pivotal Phase 3 clinical trial of GBT440 in people with sickle cell disease (SCD).  The HOPE Study will be conducted at leading SCD sites globally and is expected to enroll up to 400 adults and adolescents with SCD who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year.  “Enrolling the first patient in our pivotal Phase 3 HOPE Study is an important milestone for GBT as we continue to work to bring GBT 440 to the SCD community,” said Ted W. Love, M.D., president and chief executive officer of GBT.  “The innovative HOPE Study trial design has strong grounding in the mechanist of action of GBT 440 and is designed to assess the efficacy of GBT 440 in producing meaningful clinical improvements in anemia and daily symptoms.  GBT 440 has the potential to fundamentally modify the course of this devastating disease by habiting sickle hemoglobin polymerization, the fundamental cause of SCD pathophysiology.