In a setback, Global Blood Therapeutics scraps IPF program as lead drug flounders in proof-of-concept trials

Ted Love

It’s all or nothing for Global Blood Therapeutics’ clinical trial program for sickle cell disease.

The South San Francisco-based biotech announced this morning that it is scrapping an ambitious early-stage effort to develop their lead drug GBT440 as a therapy for hypoxemia — low levels of oxygen in the blood of patients suffering from idiopathic pulmonary fibrosis. That leaves sickle cell disease as the sole indication now being studied in the clinic.

Global Blood’s stock $GBT swiftly took a 12% hit Monday morning in pre-market trading as investors sized up the setback.

According to researchers, they were able to track an increase in the level of oxygen in the blood of people taking the therapy, reviewing two Phase IIa studies and a Phase I in healthy volunteers. But that improvement in oxygen levels didn’t actually translate into clinically meaningful benefits for the patients in the proof-of-concept study.

Their Phase III study for sickle cell disease is expected to read out in 2019.

Last March Reuters reported that the Danish pharma company Novo Nordisk was kicking the tires at GBT, possibly interested in making a play for the biotech. But nothing came out of that, yet. That story hit 5 months after Novartis bought out sickle cell rival Selexys, which had a mid-stage drug in the clinic for symptoms of sickle cell disease. GBT440, though, looks to treat a root cause of the disease.

“From the outset, we set a high bar for success in our IPF program. The results re-affirm our confidence in the mechanism of action of GBT440. However, the data from these proof-of-concept studies did not demonstrate sufficient overall clinical benefit to justify continuing the program,” said GBT CEO Ted Love.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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