Phase III OPTIC trial of RG 1507 confirms benefits in active thyroid eye disease.- Horizon Therapeutics

Horizon Therapeutics announced new data from the Phase III OPTIC confirmatory clinical trial showing that RG 1507 (teprotumumab) provided significant benefit on several devastating effects of active thyroid eye disease (TED) compared with placebo, including diplopia (double vision), quality of life (QoL) and clinical activity score (CAS).

The following new data on three secondary endpoints were presented at ASOPRS: Diplopia: At Week 24, 68% of patients receiving teprotumumab had an improvement from baseline of at least one grade in diplopia, compared to 29% of patients receiving placebo (p=0.001). This endpoint measured the percentage of patients who reported at least some diplopia at baseline in the study eye and who had a reduction of at least 1 grade with no corresponding deterioration (at least 1 grade worsening) in the fellow eye at Week 24. Quality of Life: Patients receiving teprotumumab had a mean change of 13.79 on the Graves' Ophthalmopathy Quality of Life (GO-QoL) scale compared with a change of 4.43 for patients receiving placebo (p<0.001). these scores indicate a statistical and clinically meaningful improvement over placebo in these qol measures. the go-qol scale consists of two subscales to evaluate the quality of life of ted graves ophthalmology patients including impacts on visual function and self-assessment of appearance. a change of 6 points is considered clinically significant. cas score: at week 24 more patients achieved a cas value of 0 or 1 with teprotumumab treatment 59 vs 21 of placebo participants p><0.001). cas is a scale used to assess the disease activity of ted and measures the degree of inflammation including pain swelling and redness. the cas scale ranges from 0 to 7 with a score of 0 representing no swelling or activity.>

Significant improvement in other secondary endpoints, including average change in proptosis and overall response rate over the 24-week treatment period, were presented during the 2019 American Association of Clinical Endocrinologists (AACE) Annual Scientific & Clinical Congress. As previously reported, teprotumumab was generally well tolerated; the majority of adverse events were mild or moderate, manageable and resolved during or after treatment. Previously presented primary endpoint data from OPTIC (Treatment of Graves� Orbitopathy (Thyroid Eye Disease) to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study) showed that significantly more patients treated with teprotumumab had a meaningful improvement in proptosis, the primary study endpoint, as compared with placebo (82.9% of teprotumumab patients compared to 9.5% of placebo patients; p?0.001). These data were presented during the American Society of Ophthalmic Plastic and Reconstructive Surgery (ASOPRS) 50th Anniversary 2019 Fall Scientific Symposium.