Global Blood Therapeutics Rises on Solid Mid-Stage Results

Shares of Global Blood Therapeutics Inc. (NASDAQ: GBT) saw a handy gain going into the weekend following positive results from its mid-stage clinical trial. The company announced new results from its ongoing Phase 1/2 GBT440-001 study in sickle cell disease (SCD) that further support the company’s plans to develop GBT440 as a potential disease-modifying therapy for SCD.

The data from this study were just presented at the European Hematology Association’s (EHA) 21st Congress in Copenhagen. This included a poster presentation of 90-day data from a cohort of patients in the Phase 1/2 study who were taking 700 mg/day of GBT440 and 28-day results from three dosing cohorts of GBT440. Data on the pharmacokinetics and pharmacodynamics of GBT440 were highlighted in a separate poster presentation.

Ultimately, these new GBT440 clinical data support the hypothesis that GBT440 inhibits sickle hemoglobin polymer formation, allowing it to potentially stop red blood cell hemolysis, improve blood flow and transform the treatment of the disease.

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Ted W. Love, M.D., CEO of Global Blood Therapeutics, commented:

All SCD patients dosed with GBT440 have shown a positive hematologic response. Additionally, a rapid and durable reduction in hemolytic anemia and sickling has been shown over 90 days. We continue to see a linear, dose proportional relationship between pharmacokinetics and pharmacodynamics, and the data continue to support the inhibition of polymerization of sickle hemoglobin through increased oxygen affinity as the mechanism of action of GBT440. Overall, the data collected to date in study GBT440-001 indicate that we have a drug candidate that we can move into a pivotal trial later this year. We look forward to discussing the design of that trial with the U.S. Food and Drug Administration.

Global Blood Therapeutics is a clinical-stage biopharmaceutical company dedicated to discovering, developing and commercializing novel therapeutics to treat grievous blood-based disorders with significant unmet need. It is developing its lead product candidate, GBT440, as an oral, once-daily therapy for SCD.

Shares of Global Blood Therapeutics were trading up 11% at $24.63 Friday morning, with a consensus analyst price target of $59.75 and a 52-week trading range of $12.24 to $57.00.

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