Global Blood Therapeutics, Inc. announced that data from its Phase 1/2 study of voxelotor in patients with sickle cell disease (SCD) were published online in Blood, a peer-reviewed publication of the American Society of Hematology. Voxelotor is being developed as a potentially disease-modifying therapy for SCD. GBT intends to submit a New Drug Application (NDA) for voxelotor under an accelerated approval pathway to the U.S. Food and Drug Administration (FDA) later this year 2019. The findings from the Phase 1/2 study and the open-label extension played an important role in development of voxelotor. A phase 1/2 ascending dose study and open-label extension study of voxelotor in patients with sickle cell disease the safety, tolerability, pharmacokinetic and pharmacodynamic properties of voxelotor were demonstrated in patients with SCD. Thirty-eight patients with SCD received voxelotor at doses of 500, 700 or 1000 mg per day or placebo for 28 days; 16 patients received voxelotor at doses of 700 or 900 mg per day or placebo for 90 days. Four patients from the 90-day cohort were subsequently enrolled in an extension study and treated with a once-daily 900 mg dose of voxelotor for six months. Patients treated with voxelotor for =90 days showed a 1 g/dL increase in hemoglobin and a substantial and durable reduction in hemolysis and sickled red blood cells. Low hemoglobin (anemia) and chronic hemolysis (red blood cell destruction) are powerful predictors of chronic organ damage, including stroke, silent cerebral infarction, renal failure and pulmonary hypertension, as well as early mortality in SCD. Voxelotor was well tolerated with no treatment-related serious adverse events and no evidence of tissue hypoxia. SCD is a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, which leads to the formation of abnormal hemoglobin known as sickle hemoglobin (HbS). In its deoxygenated state, HbS has a propensity to polymerize, or bind together, forming long, rigid rods within a red blood cell (RBC). The polymer rods deform RBCs to assume a sickled shape and to become inflexible, which causes hemolytic anemia (low hemoglobin due to RBC destruction) that can lead to multi-organ damage and early death. This sickling process also causes blockage in capillaries and small blood vessels. Beginning in childhood, SCD patients typically suffer unpredictable and recurrent episodes or crises of severe pain due to blocked blood flow to organs, which often lead to psychosocial and physical disabilities. 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 recently completed its evaluation of 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.