GlaxoSmithKline eyes rare disease to widen its respiratory lead

GlaxoSmithKline ($GSK) is looking to deepen its respiratory pipeline with another late-stage trial of the injectable mepolizumab, this time studying the drug's effects in a rare inflammatory disease that can be life-threatening.

In partnership with the National Institutes of Health, GSK is planning to study its treatment in patients who have eosinophilic granulomatosis with polyangiitis (EGPA), which leads to widespread inflammation in the walls of small blood vessels and can affect multiple organs, the company said. The disease, formerly known as Churg-Strauss syndrome, affects about 4,300 patients in the U.S. and 5 European countries, according to GSK.

Mepolizumab is already in the midst of a Phase III trial in severe asthma, following up on a mid-stage study in which patients in the treatment arm experienced half as many clinically significant attacks as those on placebo. The drug works by blocking the interleukin 5 cytokine and thus lowering white blood cell levels, helping to reduce inflammation in the lungs and other organs.

"This is the first ever double-blind, placebo-controlled study to be conducted in patients with eosinophilic granulomatosis with polyangiitis, marking a significant milestone in our efforts to help patients with this rare systemic inflammatory disease," GSK rare diseases chief Richard Philipson said in a statement.

Meanwhile, GSK is maintaining the world's most successful respiratory drug franchise, led by the multibillion-dollar Advair and its recently approved heirs, the Elliptas Breo and Anoro. Mepolizumab is among the few candidates in the company's respiratory pipeline not developed alongside partner Theravance ($THRX).

- read the announcement