Global Blood Therapeutics Demonstrates 'Compelling Potential' In Sickle Cell Treatment

Cantor Fitzgerald’s Elemer Piros believes Global Blood Therapeutics Inc’s GBT lead pipeline candidate, GBT440, “has demonstrated compelling potential for the treatment of sickle cell disease (SCD), based on its reported clinical efficacy and safety data.”

The analyst initiated coverage of the company with an Overweight rating and price target of $61.

Strongly Validated Mechanism

Piros mentioned that the mechanism of action used by GBT440 of oxygen affinity modulation has been validated strongly in scientific literature.

SCD presents a meaningful unmet need, with the only approved therapy being carcinogenic.

Global Blood Therapeutics’ lead candidate prevents the formation of abnormal shape in red blood cells, also known as sickling, by altering hemoglobin to retain oxygen better and prevent the cells from becoming completely deoxygenated.

“This mechanism of action is well-validated by studies of fetal hemoglobin (HbF), which is associated with a milder form of the disease for the small minority of patients who retain HbF into adulthood. GBT440 addresses the root cause of SCD,” the analyst explained.

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Immense Potential

Despite Global Blood Therapeutics having used a limited sample size of patients, GBT440 was able to demonstrate an increase in sickle hemoglobin’s oxygen affinity and reduce markers of red blood cell death or hemolysis and sickling.

“In Part A of the Phase 3 study, we expect initial results of the drug across the patient reported outcomes (PRO) tool, which could more-sensitively characterize the clinical benefit of GBT440,” Piros stated.

With an estimated 100,000 patients with SCD in the U.S. alone, and the approved therapy being associated with significant adverse effects, the analyst believes that GBT440 could “profoundly and safely” change the course of SCD, potentially improving survival meaningfully.

Piros expects sales of GBT440 to reach $2 billion in the U.S. when the drug achieves peak penetration of 30 percent.