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Global Blood (GBT) Initiates Phase II Study on SCD Candidate

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Global Blood Therapeutics, Inc. (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 Aridis Pharmaceuticals (ARDS - Free Report) , Anavex Life Sciences (AVXL - Free Report) and Scholar Rock (SRRK - Free Report) , each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

Aridis Pharmaceuticals’ loss per share estimates for 2022 have narrowed from $1.78 to 34 cents in the past 30 days. The same for 2023 has narrowed from 75 cents to 60 cents in the same time frame. Shares of ARDS have plunged 51.5% in the year-to-date period.

Earnings of Aridis missed estimates in two of the trailing four quarters and beat the same on the remaining two occasions, the average negative surprise being 75.16%.

Anavex Life Sciences’ loss per share estimates for 2022 have narrowed from 85 cents to 60 cents in the past 30 days. Shares of AVXL have plunged 42.9% year to date.

Earnings of Anavex beat estimates in two of the last four quarters and missed the mark on the other two occasions, the average surprise being 0.48%.

Scholar Rock’sloss per share estimates for 2022 have narrowed from $2.94 to $2.59 in the past 30 days. The same for 2023 has narrowed from $3.27 to $2.85 in the same time frame. Shares of SRRK have plunged 78.1% in the year-to-date period.

Earnings of Scholar Rock missed estimates in two of the trailing four quarters and beat the same on the remaining two occasions, the average surprise being 13.12%.