The Janssen Pharmaceutical Companies of Johnson & Johnson announced positive top-line results from the Phase 3 OPTIMUM study, which evaluated the efficacy and safety of ponesimod compared to Aubagio® (teriflunomide) in adults with relapsing multiple sclerosis. The study met its primary and most secondary endpoints.
Ponesimod is a selective sphingosine-1-phosphate receptor 1 (S1P1) modulator, a class of drugs that is believed to functionally inhibit S1P activity and reduce the number of circulating lymphocytes by trapping them in the lymph nodes. Therefore, there are less inflammatory cells available to cross into the central nervous system (CNS) where they could damage myelin. Myelin is a protective sheath that insulates nerve cells and is damaged in patients with multiple sclerosis.
The primary endpoint of OPTIMUM was annualized relapse rate (ARR) up to the end of the study. A key secondary endpoint was change from baseline to week 108 in fatigue-related symptoms. Fatigue is considered a significant unmet need from patients’ perspective. Additionally, the study evaluated other secondary endpoints: cumulative number of combined unique active lesions (CUALs) using magnetic resonance imaging (MRI), time to first 12-week confirmed disability accumulation (CDA) and time to first 24-week CDA from baseline to end of the study.
The safety profile observed for ponesimod in the OPTIMUM study was consistent with previous studies of ponesimod, and the known safety profile for other S1P receptor modulators.
OPTIMUM was a head-to-head, prospective, multicenter, randomized, double-blind, active controlled, parallel-group, Phase 3 superiority study to compare efficacy, safety and tolerability of ponesimod 20 mg versus teriflunomide 14 mg in adults with relapsing multiple sclerosis. The study included 1,133 participants with the treatment duration of 108 weeks.
Data from the OPTIMUM study have been accepted for presentation at the 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) 2019.
Data from the OPTIMUM study will serve as the basis of submissions to the U.S. Food and Drug Administration and European Medicines Agency seeking approval of ponesimod as a treatment for relapsing forms of multiple sclerosis, which are anticipated for later this year.