GBT Announces Updated 24-Week Efficacy Data

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All Patients Enrolled in Phase 3 HOPE Study Showing Statistically Significant and Sustained Improvements in Hemoglobin with Voxelotor

Global Blood Therapeutics, Inc. (GBT) today announced new results from its Phase 3 HOPE Study of voxelotor in patients ages 12 and older with sickle cell disease (SCD). The findings from 274 adolescents and adults treated with voxelotor showed the HOPE Study met its primary endpoint of an improvement in hemoglobin greater than 1 g/dL at 24 weeks with voxelotor 1500 mg compared with placebo, with a favorable safety and tolerability profile. In the study, voxelotor provided a rapid, statistically significant and sustained improvement in hemoglobin levels and reduced the incidence of worsening anemia and hemolysis.

The data were published today in The New England Journal of Medicine and featured in the press briefing prior to presentation at 4:15 p.m. CEST as part of the Presidential Symposium at the 24th European Hematology Association (EHA) Congress in Amsterdam.

“These additional data from our multi-national, Phase 3 HOPE Study support and strengthen the 24-week findings from 154 patients that were presented at the American Society of Hematology Annual Meeting in December 2018. These updated results form the basis of the rolling submission of our New Drug Application for voxelotor, which we are on track to complete in the second half of this year for review under an accelerated approval pathway,” said Ted W. Love, M.D., president and chief executive officer of GBT.

“We are excited about the potential for voxelotor to improve the major morbid outcomes for people with SCD, given that lower levels of hemoglobin are associated with greater risk of overt stroke, silent infarct and increased mortality. We are also pleased to announce that we have reached final agreement with the FDA on the design of our transcranial doppler post-approval confirmatory study, which we will share more information about during our corporate update webcast later today.”

“These positive data from more than 270 patients enrolled in the HOPE Study provide strong evidence that voxelotor, by significantly improving anemia and hemolysis, has the potential to be a disease-modifying treatment for SCD by preventing chronic organ damage and prolonging survival,” said Jo Howard, MB BChir, MRCP, FRCPath, of Guy’s and St. Thomas’ NHS Foundation Trust and King’s College London and a HOPE Study investigator.

“Given the observed ability of voxelotor to reduce anemia, hemolysis and red blood cell sickling, as well as a favorable safety profile, I am confident that voxelotor could potentially become a new standard-of-care for treating adolescents and adults with SCD.”

About Voxelotor in Sickle Cell Disease

Voxelotor (previously called GBT440) is being developed as an oral, once-daily therapy for patients with SCD. Voxelotor works by increasing hemoglobin’s affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes voxelotor blocks polymerization and the resultant sickling and destruction of red blood cells. With the potential to improve hemolytic anemia and oxygen delivery, GBT believes that voxelotor may potentially modify the course of SCD. In recognition of the critical need for new SCD treatments, the U.S. Food and Drug Administration (FDA) has granted voxelotor Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. The European Medicines Agency (EMA) has included voxelotor in its Priority Medicines (PRIME) program, and the European Commission (EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD.

GBT is currently evaluating voxelotor in the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study, a Phase 3 clinical study in patients age 12 and older with SCD. Additionally, voxelotor is being studied in the ongoing Phase 2a HOPE-KIDS 1 Study, an open-label, single- and multiple-dose study in pediatric patients (age 4 to 17) with SCD. The HOPE-KIDS 1 Study is assessing the safety, tolerability, pharmacokinetics and exploratory treatment effect of voxelotor.

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