Vepoloxamer Fails Primary Endpoint for Treatment of Sickle Cell Disease

Treatment with the investigational drug vepoloxamer did not reduce the mean duration of vaso-occlusive crisis, compared with placebo, for patients with sickle cell disease (SCD), according to results from the randomized, double-blind, placebo-controlled, phase III EPIC study.

The study included 388 patients with SCD (age range = 4-46 years), most of whom (71%) were younger than 18 years old.

Researchers found that the mean duration of vaso-occlusive crisis (the study’s primary endpoint) was 82 hours in the vepoloxamer, compared with 78 hours with placebo. There were no significant differences in treatment-related serious adverse events between the treatment groups, and no patients died, the authors added.

The drug’s manufacturer said it will likely terminate the study based on these outcomes.

Source: Mast Therapeutics, Inc. press release, September 26, 2016.