A Primer On Endpoints: Global Blood Therapeutics

The 90 mg GBT440 data presented by Global Blood Therapeutics Inc GBT at the American Society of Hematology meeting showed “consistent and durable responses”.

Wedbush’s Heather Behanna reiterated an Outperform rating on the company, with a price target of $73.

What The Data Revealed

The longer-term data for three months suggest durability of response, the analyst mentioned, while adding, “With no responders in the placebo group, based on these data, we expect the primary endpoint of the pivotal trial to be highly statistically significant.”

Regarding the more direct measures of the mechanism of GBT440, Behanna noted hemoglobin was the primary endpoint for the Phase III study.

Global Blood Therapeutics is also looking at dense red blood cells (RBCs), which is a measure of damaged cells that could correlate with disease morbidity.

Although the numbers are small, the data appear to demonstrate GBT440 is able to stop sickling.

Additional Information

“Our conversations with Global Blood management suggest that there is a correlation between exposure and response of GBT440; however, with variable exposure, this does not closely mirror dose,” Behanna went on to say.

Regarding safety, the company presented safety data when dosed for 28 days or more, with increased dosing durations not revealing any new safety signals. “No one has discontinued due to side effects and GI effects have been mild and transient,” the analyst stated.

The data also revealed there was no on-target toxicities at the studied doses, while most of the drug was cleared within a week after treatment.

At last check, Global Blood shares were down 10 percent at $15.52.