Horizon Pharma has announced positive Phase III clinical trial data for teprotumumab, as a treatment for active thyroid eye disease (TED).
The positive Phase III results move teprotumumab one step closer to becoming the first-ever Food and Drug Administration (FDA) approved treatment for TED, a rare, autoimmune disease that affects 15,000-20,000 people a year.
According to the data, 82.9% of the teprotumumab dosed patients found their proptosis, or bulging of the eye, reduced compared to just 9.5% of placebo patients.
The drug is a fully human monoclonal antibody (mAb) and a targeted inhibitor of the insulin-like growth factor 1 receptor (IGF-1R).
The company has revealed plans to submit a Biologics License Application to the FDA in mid 2019, and Teprotumumab has already received Breakthrough Therapy, Orphan Drug and Fast Track designations.
“The dramatic results of the Teprotumumab Phase III confirmatory trial, in addition to positive Phase 2 data, form a highly convincing body of clinical evidence supporting Teprotumumab for the treatment of active thyroid eye disease,” said Timothy Walbert, chairman, president and chief executive officer, Horizon Pharma.
He continued, “This is a key milestone as we evolve into a research-focused company developing innovative new medicines to address challenging diseases with very few effective options.
TED is a progressive, debilitating autoimmune disease which often occurs in people living with hyperthyroidism or Graves’ disease and is a distinct disease that is caused by autoantigens activating an IGF-1R-mediated signalling complex on cells within the orbit.