Global Blood Therapeutics, Inc. ( GBT Quick Quote GBT - Free Report) initiated its planned phase II/III study on its next-generation sickle hemoglobin (HbS) polymerization inhibitor, GBT02160 (GBT601) in patients with sickle cell disease (SCD).
Global Blood Therapeutics has initiated the phase II portion of the phase II/III study to identify the optimal safe and effective dose of GBT601 for the pivotal phase III portion of the trial
Per the company, GBT601 has the potential to improve on the clinical results achieved with Oxbryta, the company’s oral once-daily treatment for SCD, at a lower daily dose
The phase II portion of the study will enroll up to 60 patients with SCD between 18 and 65 years of age. The study’s primary outcome measure is the number of participants with a change from baseline in Hb through Week 12. After the optimal dose is selected, the phase III portion will assess the efficacy and safety of the dose compared with placebo in adult and pediatric SCD patients for 48 weeks.
The company expects initial data from the phase II/III study before the end of the year.
Shares of Global Blood have gained 10.3% year to date against the
industry’s decline of 22.9%.
Last December, Global Blood reported positive results from its phase I study of GBT601. The data demonstrated average Hb occupancy greater than 30% and improvements in hematologic parameters in a cohort of six patients with SCD receiving multiple ascending doses (MAD) of GBT601.
The study also showed that single ascending doses (SAD) and MAD of GBT601 were well tolerated in both SCD patients and healthy volunteers.
GBT also restarted the phase I study of GBT601 to study a 150-mg daily dose, increasing over the 100-mg daily maintenance dose previously studied.
Earlier this month, Global Blood announced that the FDA had granted both orphan drugs and rare pediatric designations to GBT601 for treating SCD.
Zacks Rank & Stocks to Consider
Global Blood currently carries a Zacks Rank #3 (Hold). Some better-ranked stocks in the same sector are
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