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Global Blood admits first sickle cell disease patient under the GBT440 HOPE Study.

M2 PHARMA-January 19, 2017-Global Blood admits first sickle cell disease patient under the GBT440 HOPE Study

(C)2017 M2 COMMUNICATIONS

Biopharmaceutical company Global Blood Therapeutics Inc (NasdaqGS:GBT) stated on Wednesday that it has enrolled the first patient under the HOPE (Hemoglobin Oxygen Affinity Modulation to Inhibit HbS PolymErization) Study for the treatment of people with sickle cell disease (SCD), a lifelong inherited blood disorder caused by a genetic mutation in the beta-chain of hemoglobin, leading to formation of abnormal hemoglobin known as sickle hemoglobin, or HbS.

The company said the HOPE Study, which is a pivotal Phase 3 clinical trial of GBT440 in people with sickle cell disease (SCD), will be conducted at leading SCD sites globally and is expected to enroll up to 400 adults and adolescents with SCD who have had at least one episode of vaso-occlusive crisis (VOC) in the previous year.

This randomised, double-blind, placebo-controlled, multi-national trial will enroll patients age 12 and older with SCD who have had at least one episode of VOC in the previous year. The Part A will compare GBT440 administered at doses of 900 or 1,500 mg per day vs. placebo in up to 150 patients treated for at least 12 weeks and Part B will include 250 patients randomised to placebo or a dose of GBT440 selected from Part A, according to the company.

In conjunction with the study, the primary efficacy endpoint of the company's HOPE Study is the proportion of patients who achieve a greater than 1 g/dL increase in hemoglobin at 24 weeks of treatment compared with baseline.

Since oxygenated sickle hemoglobin does not polymerize, the company believes GBT440 blocks polymerization and the resultant sickling of red blood cells. With the potential to restore normal hemoglobin function and improve oxygen delivery, GBT440 may dramatically modify the course of SCD. GBT440 has recieved the US Food and Drug Administration's (FDA) both fast track and orphan drug designations and the European Commission's (EC) orphan medicinal product.

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Publication:M2 Pharma
Date:Jan 19, 2017
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