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Global Blood Therapeutics begins Phase 2a GBT440 study in adolescent sickle cell disease patients.

M2 EQUITYBITES-June 30, 2016-Global Blood Therapeutics begins Phase 2a GBT440 study in adolescent sickle cell disease patients

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Biopharmaceutical company Global Blood Therapeutics (NasdaqGS:GBT) reported on Wednesday the launch of a Phase 2a study of GBT440 in adolescents with sickle cell disease (SCD), an inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, leading to formation of abnormal hemoglobin known as sickle hemoglobin, or HbS.

GBT440 is being developed by the company as a potential disease-modifying therapy for SCD for patients of all ages and genotypes. It is being developed as an oral, once-daily therapy for patients with SCD. The drug works by increasing hemoglobin's affinity for oxygen. With the potential to restore normal hemoglobin function and improve oxygen delivery, it may be capable of modifying the progression of SCD.

The US Food and Drug Administration (FDA) has granted GBT440 both Fast Track and Orphan Drug designation for the treatment of patients with SCD in recognition of the critical need for new treatments, according to the company.

This open-label, single and multiple dose GBT440-007 study is evaluating the safety, tolerability, pharmacokinetics and exploratory treatment effect of GBT440 in adolescents age 12 to 17 years with SCD.

The study is being conducted in two parts: in Part A, six subjects will receive a single dose of GBT440, and in Part B, 24 subjects will receive multiple doses of GBT440 for up to 28 days. The primary objective of Part A is to characterize the pharmacokinetics of GBT440, while the primary objective of Part B is to explore the safety of multiple doses of GBT440 administered to adolescent SCD patients.

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Publication:M2 EquityBites (EQB)
Date:Jun 30, 2016
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