Global Blood Therapeutics Inc. (NASDAQ: GBT) (GBT) has announced new data from the first phase GBT021601 (GBT601) study. GBT601 is the Company’s experimental next-gen sickle hemoglobin polymerization inhibitor that has shown mean hemoglobin occupancy of more than 30% with significant hematologic parameters enhancement in a group of six sickle cell disease patients that have received multiple ascending GBT601 doses.
Positive GBT601 study results
The trial also demonstrated that single ascending and multiple ascending doses of GBT601 had a good tolerability profile in SCD patients and healthy volunteers. The com[any presented the data in a poster at this year’s 63rd American Society of Haematology Annual Meeting and Exposition that took place in Atlanta, Georgia, and virtually.
Lead study investigator Clark Brown stated, “These Phase 1 data of GBT601 are very encouraging and demonstrate that in sickle cell disease patients we can achieve a high target hemoglobin occupancy at daily doses lower than 500 mg, while maintaining a favorable safety and tolerability profile. Therefore, GBT601 has the potential to improve clinical outcomes in people living with SCD, while reducing pill burden. I’m very excited to continue to study GBT601 in more patients and with longer follow up to seek to confirm the potential for GBT601.”
GBT presents Oxbryta study results
The Company also announced the presentation of positive findings from long-term and real-world studies with Oxbryta (voxelotor) tables in sickle cell disease treatment. Oxbryta is a first-in-class oral therapy given daily that inhibits polymerization of sickle hemoglobin, which is the cause of red blood cells sickling and destruction in SCD. Findings from the analysis of the 3,128 patients that received Oxbryta showed considerable improvement in Hb levels and substantial reduction in vaso-occlusive crises, transfusions, and hospitalization.
EVP and R&D head Kim Smith-Whitley said, “We’re thrilled with the data presented at ASH, which demonstrate the clinically meaningful impact of Oxbryta for patients living with sickle cell disease. The benefits of Oxbryta were reinforced in this large retrospective study to evaluate the impact of an SCD medicine on clinical outcomes and healthcare resource utilization in a real-world setting.”
