Global Blood Therapeutics Inc GBT shares were sliding Friday after the release of updated data for the company's sickle cell disease candidate that will be presented at the European Hematology Association Congress.
What Happened
Global Blood announced 24-week data from its ongoing HOPE-KIDS 1 Study, an open-label Phase 2a study that evaluated its voxelotor in treating adolescents aged 6-17 years with SCD.
Part B of the study evaluated 22 patients who received voxelotor 900mg/day and showed that 43 percent achieved hemoglobin response of greater than 1g/dL at 24 weeks, with a median hemoglobin change from a baseline of 0.7 g/dL.
Interim data presented at the American Society of Hematology in December looked at 12 patients in the 900mg cohort who were treated for 16 weeks and showed that 55 percent of the patients achieved hemoglobin response rate greater than 1g/dL.
Why It's Important
The sell-side was upbeat on the prospects for voxelotor, with William Blair's Raju Prasad estimating revenues of $1.5 billion in the U.S., $260 million in the EU and another $11 million in licensing revenue in 2027. The optimism is hinged on a lack of competition and a once-daily, oral small molecule approach that is likely to endear itself to patients.
What's Next
Global Blood has an ongoing Phase 3 Hope study that's evaluating voxelotor doses of 900mg and 1,500mg per day in adolescents and adults.
"We continue to expect to announce top-line clinical data from Part A of the HOPE Study by the end of this quarter," the company said.
Global Blood Therapeutics shares were down 12.33 percent at $41.25 at the time of publication Friday.
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