Shares in Global Blood jump after positive results for trial sickle cell drug

pharmafile | June 13, 2016 | News story | Research and Development, Sales and Marketing Global Blood Therapeutics, drug trial, sickle cell disease 

Global Blood Therapeutics (Nasdaq: GBT) shares jumped to close up over 9% Friday after the company said its trial drug to treat sickle cell disease boosted hemoglobin production in a small study.

Chief Executive Ted Love said: “Overall, the data collected to date in study GBT440-001 indicate that we have a drug candidate that we can move into a pivotal trial later this year.

“All SCD patients dosed with GBT440 have shown a positive hematologic response. Additionally, a rapid and durable reduction in hemolytic anemia and sickling has been shown over 90 days. We continue to see a linear, dose proportional relationship between pharmacokinetics and pharmacodynamics, and the data continue to support the inhibition of polymerization of sickle hemoglobin through increased oxygen affinity as the mechanism of action of GBT440.”

The data presented at the European Hematology Association (EHA) congress, showed the oral drug significantly inhibited haemoglobin polymer formation.

Dr. Paul Telfer, consultant in hematology and pediatric hematology at Barts Health NHS Trust, said:  “These new GBT440 clinical data continue to support the hypothesis that GBT440 inhibits sickle hemoglobin (HbS) polymer formation, allowing it to potentially stop red blood cell hemolysis, improve blood flow and transform the treatment of the disease. Based on the data we have seen to date, GBT440 has the potential to be a once-daily treatment that could improve the devastating clinical course of sickle cell disease.”

Shares in the company rose 30% in pre-market trading Friday.

Sickle cell is a group of inherited red blood cell disorders where a person has abnormal hemoglobin, the protein that transports oxygen in the blood. Those with the disease experience sudden severe pain attacks and poor oxygen delivery can cause organ damage.

Anjali Shukla

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