Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that it initiated the Phase 2 portion of a planned Phase 2/3 trial of GBT021601 (GBT601), the company’s investigational next-generation sickle hemoglobin (HbS) polymerization inhibitor. The study (NCT05431088) is a randomized, multicenter Phase 2/3 clinical trial evaluating the safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of GBT601.
“Based on compelling preclinical and clinical data, we believe GBT601 has the potential to be a best-in-class therapy for patients with sickle cell disease,” said Kim Smith-Whitley, M.D., executive vice president and head of research and development of GBT. “Specifically, it has the potential to improve on the clinical results achieved with Oxbryta® at a lower daily dose. The initiation of our Phase 2/3 trial is an important milestone in our efforts to bring GBT601 to patients. We anticipate that initial data from this trial as well as data from the continuation of our GBT601 Phase 1 study will be available before the end of the year.”
The study is planned to include sites in Africa, Europe, the Middle East, South America, and the United States, including several sites that are expected to begin enrolling patients in the near term. The Phase 2 portion of the study will evaluate the safety, tolerability, and efficacy of GBT601 and enroll up to 60 patients with SCD who are 18 to 65 years of age. Patients with hemoglobin (Hb) levels between 5.5 g/dL and 10.5 g/dL and 10 or fewer vaso-occlusive crises in the prior year are eligible for enrollment. Patients will be randomized 1:1:1 to a daily maintenance dose of GBT601 of 100, 150 or up to 200 mg. The primary outcome measure is the number of participants with a change from baseline in Hb through Week 12. Secondary outcomes measures include assessments of PK and PD, as well as an assessment of the relationship between GBT601 and measures of anemia and hemolysis.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.