Horizon Therapeutics plc recently announced positive topline data from two clinical trials supporting the efficacy and safety of TEPEZZA® (teprotumumab-trbw) for the treatment of Thyroid Eye Disease (TED). TEPEZZA is the first and only medicine approved by the U.S. Food and Drug Administration (FDA) for the treatment of TED – a serious, progressive and vision-threatening rare autoimmune disease.
The OPTIC Phase 3 confirmatory clinical trial and the OPTIC-X open-label extension clinical trial are part of Horizon’s development program to evaluate the safety and efficacy of TEPEZZA in people living with TED.
OPTIC (Treatment of Graves’ Orbitopathy [Thyroid Eye Disease] to Reduce Proptosis with Teprotumumab Infusions in a Randomized, Placebo-Controlled, Clinical Study) was a multicenter, randomized, double-blind, placebo-controlled trial. This Phase 3 confirmatory clinical trial included a 24-week treatment period and a 48-week off-treatment follow-up period. At the end of the 24-week treatment period, patients who were proptosis responders entered into the 48-week off-treatment follow-up period, without receiving additional TED treatment, including TEPEZZA.
OPTIC-X evaluated the safety and efficacy of TEPEZZA in TED patients who were enrolled in OPTIC and were either proptosis non-responders at Week 24 of OPTIC, or were proptosis responders at Week 24 but relapsed during the 48-week off-treatment follow-up period. Non-responders were defined as patients who did not achieve at least a 2 mm proptosis improvement from baseline at Week 24 of OPTIC. Relapse was defined as patients who lost at least 2 mm of their Week 24 proptosis improvement during the 48-week off-treatment follow-up period – even if their proptosis was still substantially better than at baseline of OPTIC – or who had a substantial increase in the number of inflammatory signs or symptoms without worsening proptosis. Patients could qualify as relapsing at any point during the 48-week off-treatment follow-up period of OPTIC.
Among the topline findings, 89 percent of patients who received placebo during the OPTIC Phase 3 clinical trial and then received TEPEZZA in the OPTIC-X extension trial achieved clinically significant proptosis reduction with an average of 12 months of disease, compared with six months in OPTIC. Majority of TEPEZZA patients who were proptosis responders in OPTIC at week 24 maintained their response at week 72, nearly a year off treatment. For the small number of TEPEZZA patients who relapsed during the OPTIC follow-up period, the majority experienced improvements in proptosis with an additional course of TEPEZZA in OPTIC-X.
“The similarity in results between those who received TEPEZZA in the OPTIC trial and those who transitioned from placebo in OPTIC to TEPEZZA in OPTIC-X is remarkable,” said Raymond Douglas, M.D., Ph.D., the trial’s co-principal investigator and director of the Orbital and Thyroid Eye Disease Program, Cedars-Sinai Medical Center. “Previously, people diagnosed with TED had no FDA-approved treatments and could expect to experience many years of life-altering symptoms while undergoing multiple surgeries in an attempt to restore their vision. Data from the OPTIC and OPTIC-X clinical trials, as well as observations from our real-world use of TEPEZZA following the FDA approval, have provided very compelling reasons to completely change expectations for people living with TED.”
Full news release: https://ir.horizontherapeutics.com/news-releases/news-release-details/new-topline-tepezzar-teprotumumab-trbw-data-underscore-its
Source: Horizon Therapeutics