Horizon Therapeutics presents additional data from Phase 4 TEPEZZA trial

Horizon Therapeutics announced the presentation of new data from the randomized, double-masked, placebo-controlled Phase 4 clinical trial evaluating TEPEZZA in patients with long disease duration and low Clinical Activity Score, a measure of disease activity. The oral presentation at the Endocrine Society Annual Meeting supports the efficacy and safety of TEPEZZA in TED patients regardless of disease activity or duration. TEPEZZA is the first and only medicine approved by the U.S. Food and Drug Administration for the treatment of TED – a serious, progressive, debilitating and potentially vision-threatening rare autoimmune disease. As previously reported, the Phase 4 clinical trial met its primary efficacy endpoint and key secondary efficacy endpoint. At Week 24, per the pre-specified primary analysis method, patients treated with TEPEZZA achieved a 2.41 mm reduction in proptosis from baseline compared with 0.92 mm for placebo and 62% of patients treated with TEPEZZA had a meaningful improvement in proptosis compared with placebo. In the pre-specified per-protocol analysis, patients treated with TEPEZZA achieved a 2.44 mm reduction in proptosis from baseline compared with 0.69 mm for those receiving placebo and 63% of patients treated with TEPEZZA had a meaningful improvement in proptosis compared with placebo at Week 24. At Week 24, in the pre-specified analysis in the intent-to-treat population, patients who received TEPEZZA experienced a significantly greater average improvement from baseline for visual functioning compared with placebo. At Week 24, the change from baseline for appearance, also measured by GO-QOL, was 10.03 for TEPEZZA and 7.19 for placebo. The appearance subscale endpoint was not statistically significant: at Week 24, per the pre-specified primary analysis method, patients who received TEPEZZA experienced a 10.03 improvement in appearance compared with 7.19 for placebo. No new safety signals were observed.