Janssen has announced positive top-line results from its Phase III OPTIMUM study, which evaluated the efficacy and safety of ponesimod compared to Aubagio (teriflunomide) in adults with relapsing multiple sclerosis (MS).
The study met its primary and most secondary endpoints, which were an annualised relapse rate (ARR) up to the end of the study and change from baseline to week 108 in fatigue-related symptoms, respectively.
The study also 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 drug 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, which is a protective sheath that insulates nerve cells and is damaged in patients with multiple sclerosis.
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 the disease.
MS is a chronic autoimmune inflammatory disease of the central nervous system affecting 2.3 million people worldwide with females more impacted than males. The disease is characterised by demyelination and axonal loss leading to neurological impairment and severe disability.