Global Blood Takes HOPE In Sickle Cell After Pulmonary Fibrosis Failure

GBT440 is not efficacious enough in studies of idiopathic pulmonary fibrosis, but the drug was well-tolerated at twice the exposure in the treatment of sickle cell disease, the drug's lead indication.

Sickle cell anemia, 3D illustration showing blood vessel with normal and deformated crescent-like red blood cells

Global Blood Therapeutics Inc. has decided to quash development of GBT440 in idiopathic pulmonary fibrosis due to disappointing mid-stage trial results, but the company sees positive aspects of the data that bode well for success in the Phase III HOPE study of the drug in sickle cell disease, which is due to report in the first half of 2019.

The company announced Oct. 23 data from three proof-of-concept studies – the Phase I Basecamp study in healthy volunteers and two Phase IIa studies (GBT440-006 and ZEPHYR) – do not support moving forward with development in idiopathic pulmonary fibrosis (IPF)

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