Janssen Reports Positive Top-Line Phase 3 Results for Ponesimod

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 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.

Multiple Sclerosis (MS) is a chronic autoimmune inflammatory disease of the central nervous system affecting 2.3 million people worldwide2 with females more impacted than males. The disease is characterized by demyelination1 and axonal loss leading to neurological impairment and severe disability. The main subtype of MS is relapsing forms of MS (RMS), which represents 85% of MS patients and includes clinically isolated syndrome (CIS), relapsing-remitting MS (RRMS) and active secondary progressive MS (SPMS).

Relapses are defined as new, worsening or recurrent neurological symptoms that last for more than 24 hours with the absence of fever or infections. Relapses may be fully resolved over days or weeks or lead to persistent residual deficits and accumulation of disability.

Ponesimod is a selective sphingosine-1-phosphate receptor 1 (S1P1) modulator that functionally inhibits S1P activity and reduces the number of circulating lymphocytes. It is thought that in people with relapsing-remitting multiple sclerosis (RRMS), ponesimod prevents immune cells from crossing the blood-brain barrier and damaging myelin. Myelin is a protective sheath that insulates nerve cells and is damaged in patients with multiple sclerosis.

A member of the Janssen Pharmaceutical Companies of Johnson & Johnson, Actelion Pharmaceuticals Ltd, is party to a revenue sharing agreement with Idorsia Pharmaceuticals Ltd, which provides for certain payments to Idorsia related to the sales of ponesimod.

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