Janssen will present data from the trial at a September conference in Stockholm. The company plans to seek FDA approval later this year.
In a Phase III head-to-head trial, Janssen Pharmaceutical’s ponesimod stood up to Sanofi’s Aubagio (teriflunomide) in adults with relapsing multiple sclerosis. Late Thursday, Janssen said the late-stage OPTIMUM study met its primary and most secondary endpoints.
With the head-to-head data in hand, Janssen said it plans to seek regulatory approval for ponesimod as a treatment for relapsing forms of multiple sclerosis in the United States and Europe later this year. Ponesimod is a selective sphingosine-1-phosphate receptor 1 (S1P1) modulator. It is a member of a class of drugs believed to inhibit S1P activity and reduce the number of circulating lymphocytes by trapping them in the lymph nodes. Limiting the ability of inflammatory cells available to cross into the central nervous system, it will protect myelin, the protective sheath that insulates nerve cells and is damaged in patients with multiple sclerosis.
The Phase III OPTIMUM study was a head-to-head, multicenter, randomized, double-blind, active-controlled, parallel-group, 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. In its announcement, Janssen did not release the details of how well ponesimod fared in the trial versus Aubagio. The company will reveal the data during a presentation at the 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis. The congress will be held Sept. 11-13 in Stockholm.
Sanofi’s Aubagio was first approved by the U.S. Food and Drug Administration in 2012 as a treatment for multiple sclerosis, a chronic autoimmune inflammatory disease of the central nervous system that affects more than 2.3 million people worldwide. Last year, Sanofi presented additional late-stage data showing that Aubagio significantly slowed whole brain volume loss compared with placebo in patients with a first clinical episode suggestive of multiple sclerosis.
The main subtype of multiple sclerosis is the relapsing forms of MS, which represents approximately 85% of MS patients. Relapses are defined as new, worsening or recurrent neurological symptoms that last for more than 24 hours with the absence of fever or infections.
With a wide population, relapsing MS is a popular target for drug companies. Earlier this year, Novartis won approval for its new multiple sclerosis treatment Mayzent (siponimod). Mayzent was approved for the treatment of adults with relapsing forms of multiple sclerosis, including secondary progressive multiple sclerosis with active disease, relapsing-remitting multiple sclerosis and clinically isolated syndrome.
The primary endpoint of the OPTIMUM study was annualized relapse rate (ARR) up to the end of the study, with a key secondary endpoint being the change from baseline to week 108 in fatigue-related symptoms. Fatigue is considered a significant unmet need from the patients’ perspective. Other secondary endpoints evaluated in the trial include a diagnosis of the cumulative number of combined unique active lesions in the patient, as well as 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, Janssen said.
