California biotech Global Blood Therapeutics is canning work in idiopathic pulmonary fibrosis (IPF) after its medicine came up short in testing and is switching its focus to sickle cell disease.
GBT said that the discontinuation of its GBT440 in IPF comes after clinically meaningful results were not seen in patients with the lung-scarring disease across several early- to midstage trials.
In a statement, the biotech said, “Overall, these studies demonstrate proof-of-concept regarding improvement in oxygen saturation in healthy volunteers and patients with IPF. However, based on the totality of the data, GBT believes this improvement is unlikely to translate into a transformative clinical benefit for IPF patients.”
It said it now will make “no further investments in additional clinical studies in hypoxemia with GBT440 and intends to stop further enrollment in any ongoing studies.”
GBT440 will, however, remain in the clinic for sickle cell disease, in which GBT hopes will prove “disease-modifying.” Top-line data from its phase 3 in this setting are due next year.
Here, the med looks to become a once-daily, oral med for the inherited disease and works by increasing hemoglobin’s affinity for oxygen.
Ted Love, M.D., president and CEO of GBT, said, “From the outset, we set a high bar for success in our IPF program. The results reaffirm 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 biotech, which had a market cap of around $1.4 billion at the end of last week, was back in March said to be in the M&A crosshairs of Novo Nordisk; this update may now dampen that speculation for now, with much resting on its sickle cell readout in 2019.