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Additional Relevant Details Results of the preclinical studies showed that in vitro, GBT440 dose-dependently increased the affinity of HbS for oxygen, delayed polymerization of HbS and reduced the number of sickled red blood cells (RBCs) in whole blood from SCD patients. Additionally, in an animal model of SCD, GBT440 inhibited RBC sickling, prolonged the half-life of RBCs and reduced reticulocyte counts. It also exhibited favorable pharmacokinetic properties in various animal species, suggesting the potential for once-daily oral dosing in SCD patients.
"Our preclinical work has developed a foundation of evidence that GBT440 is a potent inhibitor of the polymerization of HbS. We continue to build on these data with our ongoing Phase 1/2 study, which has shown that GBT440 was well tolerated over 90 days of dosing, and that all SCD patients who received multiple doses of GBT440 exhibited improvements in one or more clinical markers of hemolysis and anemia," said Ted W. Love, M.D., chief executive officer of GBT. "Our next step is to initiate a pivotal trial in adults with SCD later this year."

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Clinical Data
A major driver of the pathophysiology of sickle cell disease (SCD) is polymerization of deoxygenated haemoglobin S (HbS), which leads to sickling and destruction of red blood cells (RBCs) and end-organ damage. Pharmacologically increasing the proportion of oxygenated HbS in RBCs may inhibit polymerization, prevent sickling and provide long term disease modification. We report that GBT440, a small molecule which binds to the N-terminal α chain of Hb, increases HbS affinity for oxygen, delays in vitro HbS polymerization and prevents sickling of RBCs. Moreover, in a murine model of SCD, GBT440 extends the half-life of RBCs, reduces reticulocyte counts and prevents ex vivo RBC sickling. Importantly, oral dosing of GBT440 in animals demonstrates suitability for once daily dosing in humans and a highly selective partitioning into RBCs, which is a key therapeutic safety attribute. Thus, GBT440 has the potential for clinical use as a disease-modifying agent in sickle cell patients.

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