Novartis announced that the full results from the Phase III EXPAND study of oral, once-daily siponimod (BAF312) in secondary progressive multiple sclerosis (SPMS) were published in the peer-reviewed journal The Lancet. These pivotal results show siponimod significantly reduced the risk of three-month confirmed disability progression versus placebo (primary endpoint). Siponimod also meaningfully delayed the risk of six-month confirmed disability progression and demonstrated favorable outcomes in other relevant measures of multiple sclerosis (MS) disease activity. 

If approved, siponimod would be the first disease-modifying therapy to delay disability progression in a large trial of typical SPMS patients, including many who had reached a non-relapsing stage and high level of disability.

SPMS is a form of MS that is associated with progressive, irreversible physical and cognitive disability, largely independent of relapses. Patients transition to SPMS after an initial phase of relapsing-remitting MS (RRMS), the most commonly diagnosed type of MS3. There is a high unmet medical need for new treatments that are safe and effective for patients with SPMS.

“Today’s published full EXPAND results show that siponimod can delay disability progression in typical established SPMS patients, where other approaches tested so far have been unsuccessful,” said Professor Ludwig Kappos, University Hospital Basel and Principal Investigator of EXPAND. “These data are all the more impressive when considering that the majority of patients had already advanced disability when starting treatment in EXPAND.”

Siponimod is an investigational selective modulator of specific subtypes of the sphingosine-1-phosphate (S1P) receptor. Full data from EXPAND show that siponimod reduced the risk of three-month confirmed disability progression by a statistically significant 21% versus placebo (p=0.013); efficacy was overall consistent across pre-defined patient subgroups. 

“Novartis is dedicated to advancing MS research and pioneering solutions for people living with SPMS – a complex, debilitating disease,” said Danny Bar-Zohar, global head Neuroscience Development at Novartis. “The pivotal EXPAND data provide patients, and the medical community alike, with hope that a much needed, safe and effective treatment option is on the horizon for SPMS, for which treatment options are scarce. We look forward to continuing to work with regulatory agencies to make siponimod available for these patients as fast as possible.”