Global Blood Therapeutics Meets Primary Endpoints In Sickle Cell Study, Seeks Accelerated FDA Approval

Global Blood Therapeutics Inc GBT shares were trading down off the open Wednesday after the company released data from its sickle cell disease candidate.

What Happened

Part A of a Phase 3 study of voxelotor in sickle cell disease achieved the primary endpoints, the company said. 

Relative to the placebo, the candidate demonstrated a statistically significant increase in the proportion of patients achieving a targeted rise in hemoglobin.

Why It’s Important

STAT News reporter Adam Feuerstein said much of the data looks strong, but patient reported outcomes were "a total bust.”

The $GBT hemoglobin and hemolysis data look very good. Positive trend on VOC reduction. PRO was a total bust.

— Adam Feuerstein (@adamfeuerstein) June 27, 2018

Nonetheless, GBT CEO and President Ted Love called the results “highly encouraging.”

"Based upon voxelotor's robust impact on hemolytic anemia, we believe it meets the standard for accelerated approval, and we look forward to providing further updates on our regulatory discussions as soon as possible, but no later than year-end,” Love said in a press release.

The firm is in talks with Food and Drug Administration to accelerate approval.

What’s Next

GBT will present results from Part A of the study at a medical meeting later in the year. In the meantime, it continues to dose Part A patients but does not intend to expand enrollment before completing discussions with the FDA.

Shares initially popped 21 percent on the news before falling 30 percent. GBT shares were down 4.39 percent and trading around the $37 level at the time of publication Wednesday morning. 

Related Links:

Global Blood Therapeutics Shares Plunge After Sickle Cell Candidate Shows Reduced Effect

Turning Neutral: Morgan Stanley Downgrades Sarepta, Loxo Oncology, Global Blood Therapeutics