Date: 2016-08-07
Type of information: Publication of results in a medical journal
phase: preclinical
Announcement: publication of results in the British Journal of Haematology
Company: Global Blood Therapeutics (USA - CA)
Product: GBT440
Action
mechanism: GBT440 is a novel small molecule that binds specifically to hemoglobin (the oxygen-carrying protein in red blood cells) and is designed to inhibit sickle hemoglobin (HbS) polymer formation.
Disease: sickle cell disease
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Country:
Trial details:
Latest
news: * On July 8, 2016, Global Blood Therapeutics announced publication of preclinical GBT440 results in sickle cell disease (SCD) in the July 5th online edition of the British Journal of Haematology. GBT is developing GBT440 as a potentially disease-modifying therapy for sickle cell disease and is currently evaluating it in an ongoing Phase 1/2 study in adults (GBT440-001) and a Phase 2a study in adolescents, age 12 to 17 years (GBT440-007). 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 sickle cell disease, 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 sickle cell disease patients. Global Blood Therapeutics now intends to initiate a pivotal trial in adults with sickle cell disease later this year.
