US-based Biopharma Presents Positive Data on Potential Sickle Cell Therapy at European Hematology Congress

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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Company presents data on potential sickle cell therapy

Global Blood Therapeutics, a biopharma developing novel therapeutics for the treatment of blood-based disorders, recently announced new results from its ongoing Phase 1/2 study of GBT440-001 in sickle cell disease (SCD).

The data, presented at the European Hematology Association’s 21st Congress in Copenhagen, further supports the company’s plans to develop GBT440 as a potential once-daily, oral disease-modifying therapy for sickle cell disease.

GBT440, currently in development, works by improving hemoglobin’s affinity for oxygen. The proposed mechanism of action states that the drug blocks the sickling of red blood cells, which potentially restores normal hemoglobin function, improves oxygen delivery, and modifies the progression of sickle cell disease.

The GBT440-001 clinical study is a randomized, placebo-controlled, double-blind and multiple ascending dose trial, with the aim of evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of GBT440 in sickle cell patients and healthy controls.

The study was divided into three parts. Part A consists of a single-dose administration; Part B, a multiple dose administration, daily for 15 days in healthy subjects and 28 days in sickle cell patients; and Part C, a multiple dose administration for 90 days in sickle cell patients.

The company’s presentation included a poster presentation describing the 90-day data from patients receiving 700 mg a day, and 28-day results from three dosing cohorts of GBT440.

The 90-day data points to significant improvements in several disease parameters, including a sustained reduction in irreversibly sickled cells, with a median decrease of 70 percent compared to an increase of about 15 percent with a placebo. GBT440 was also well tolerated, with no drug-related serious adverse events.

“We continue to see a linear, dose proportional relationship between pharmacokinetics and pharmacodynamics, and the data continues to support the inhibition of polymerization of sickle hemoglobin through increased oxygen affinity as the mechanism of action of GBT440,” said GBT CEO Ted W. Love, M.D.

“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. We look forward to discussing the design of that trial with the U.S. Food and Drug Administration,” Love said.

In a separate poster presentation, Global Blood Therapeutics describes data on the pharmacokinetics and pharmacodynamics of GBT440. The detailed data and study’s results can be read in the company’s press release.