Study published in The Lancet shows new treatment for later stage multiple sclerosis significantly improves outcomes

Novartis has announced that the full results from the EXPAND study of siponimod (BAF312) in secondary progressive multiple sclerosis (SPMS) were published in The Lancet.

These results show significant reductions in the risk of three and six-month disability progression with siponimod versus placebo and favourable outcomes in other relevant measures of MS disease activity.

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

Novartis plans to file for a European license in 2018. Secondary progressive MS is a form of MS that leads to progressive, irreversible disability, largely independent of relapses. Patients transition to secondary progressive MS after an initial phase of relapsing-remitting MS, the most commonly diagnosed type of MS.

Secondary progressive MS is different from primary progressive MS, which is progressive from the beginning. There is a high unmet medical need for new treatments that are safe and effective for patients with secondary progressive MS. MS affects approximately 2.3 million people worldwide and about 9,000 people in Ireland.

“Currently, Irish people with secondary progressive MS have limited treatment options. These data, published today, shows that siponimod delays the advancement of disability for these patients, representing an important milestone in MS care”, says Professor Orla Hardiman, Consultant Neurologist, Beaumont Hospital and Professor of Neurology, Trinity College, Dublin.

“These data are all the more impressive when considering that the majority of patients already had advanced disability when starting treatment on the study”.

Siponimod is an oral selective modulator of sphingosine1-phosphate (S1P) receptor subtypes one and five (S1P1 and S1P5).

Full data from EXPAND show that siponimod reduced the risk of three-month confirmed disability progression by a statistically significant 21% versus placebo, efficacy was consistent across many sub groups.

Other clinically relevant endpoint data show that siponimod, when compared to placebo:

 

• Reduced the risk of six-month confirmed disability progression by 26%

 

• Slowed the rate of brain volume loss (brain shrinkage) by 23%

 

• Limited the increase of brain lesion volume by approximately 80%

 

• Reduced annualised relapse rate by 55%

 

• Did not show a significant difference in the Timed 25-Foot Walk test and MS Walking Scale

 

• Demonstrated a safety profile that was overall consistent with the known effects of S1P receptor modulation

 

“Novartis is dedicated to advancing MS research and pioneering solutions for people living with secondary progressive MS a complex, debilitating disease,” said Danny Bar-Zohar, Global Head, Neuroscience Development for Novartis. “The pivotal EXPAND data provides patients, and the medical community alike, with hope that a much needed, safe and effective treatment option is on the horizon for secondary progressive MS, 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.”

Novartis plans to file for regulatory approval of siponimod for SPMS with the US Food and Drug Administration (FDA) in early 2018. Novartis has initiated a scientific advice consultation with the European Medicines Agency (EMA) and, pending its outcome, plans to file in Q3 2018. The EXPAND results have previously been presented at scientific congresses.