Global Blood Therapeutics announced it is shuttering its GBT440 program to treat idiopathic pulmonary fibrosis (IPF). However, it will continue investigating the drug in sickle cell disease.
Global Blood Therapeutics was investing GBT440 in IPF in three proof-of-concept clinical studies. One was a Phase I trial in healthy volunteers dubbed Basecamp. There were two other Phase IIa studies, one in patients with IPF named GBT440-006 and the other named ZEPHYR.
The drug was well tolerated and no safety concerns were observed. GBT440 was designed to improve oxygen saturation and in all three studies seemed to show it was effective at doing so, but not to an extent that would be clinically beneficial.
“From the outset, we set a high bar for success in our IPF program,” said Ted Love, president and chief executive officer of GBT, in a statement. “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. While we are disappointed that we didn’t meet our high bar for success, and we are disappointed that we will not be able to help the IPF community, we are grateful to the patients, healthy volunteers and healthcare professionals who participated in the trials and supported us in these efforts.”
The company is continuing its Phase III HOPE trial in sickle cell disease and expects top-line results in the first half of 2019.
Company stock dropped 12 percent this morning in pre-market trading at the news.
John Carroll, with Endpoints News, writes, “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 five 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.”
Novartis exercised rights to buy Selexys after the company announced results of its SUSTAIN trial, a Phase II study of its SelG1, an anti-P-selectin antibody, in the reduction of vaso-occlusive pain crises in patients with sickle cell disease. It had picked up the exclusive right to buy Selexys and SelG1 in 2012. Selexys, before the acquisition, was a privately held company in Oklahoma City, Oklahoma. Specifics of the deal weren’t released, but could total up to $665 million in upfront payments, acquisition and milestone payments. The deal was announced in November 2016.
Sickle cell disease is an inherited blood disorder noted by sickle-shaped red blood cells. It can range from asymptomatic to life-threatening. Vaso-occlusive crises, also called pain crises, are a big cause of health care interactions in SCD and are caused when sickle-shaped red blood cells block blood flow through blood vessels.
GBT’s approach, however, is to try to ameliorate a significant issue with SCD. The sickle shape of the red blood cells prevents them from carrying oxygen as effectively as the normal red blood cells.


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