Treatment with teprotumumab, a human monoclonal antibody inhibitor of insulin-like growth factor I receptor (IGF-IR), was more effective vs. placebo in reducing proptosis and the Clinical Activity Score in patients with active ophthalmopathy.

• Multicenter, double-masked, randomized, placebo-controlled trial conducted to evaluate teprotumumab in patients with active, moderate-to-severe ophthalmopathy
• 88 patients randomized to placebo or active drug given intravenously (IV) once every 3 weeks for 8 infusions
• Primary endpoint: response in the study eye defined as reduction of ≥2 points in the Clinical Activity Score (0-7) and a reduction of ≥2mm in proptosis at Week 24
• Secondary endpoints: proptosis, Clinical Activity Score, results on Graves’ ophthalmopathy-specific quality-of-life questionnaire

• 69% of teprotumumab patients (29/42) had a response at Week 24 (P<0.001) vs. 20% of placebo patients (9/45)
• 43% of teprotumumab patients (18/42) had a response at Week 6 (P<0.001) vs. 4% of placebo patients (2/45)
• Differences between treatment arms increased at subsequent time points
• Hyperglycemia was reported in patients with diabetes but was controlled by adjusting antidiabetic medications