Novartis BAF312 reduces the risk of disability progression in pivotal phase III study in secondary progressive MS patients

News Hour:

Novartis  announced positive results of the Phase III EXPAND study showing that oral once-daily BAF312 (siponimod) significantly reduced the risk of disability progression compared with placebo in people with secondary progressive multiple sclerosis (SPMS).SPMS is a form of MS characterized by continuous worsening of neurological function over time, independent of relapses.Topline results of EXPAND were presented at the 32^nd Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), in London, UK.

BAF312 is a scientifically designed, selective sphingosine-1-phosphate (S1P) receptor modulator.Initial data from the EXPAND studyshow:

• Treatment with BAF312 reduced the risk of three-month confirmed disability progression by 21% compared with placebo (p=0.013). The risk reduction for six-month confirmed disability progression was greater, further supporting robustness of the data.
• A consistent reduction in the risk of confirmed disability progression across predefined subgroups, including patients without relapses.
• A significant difference in favor of BAF312 compared to placebo in annualized relapse rate, the percent change in brain volume, and change from baseline in the volume of T2 lesions (brain lesions identified by a T2-weighted magnetic resonance imaging scan). Difference in change from baseline in the Timed 25-Foot Walk test (T25FW) was not significant.
• BAF312 was generally safe and well tolerated, with a profile comparable to other drugs in the same class.

“There are very few available treatment options to delay disease progression in SPMS, and there is a high unmet need for effective therapies with an acceptable safety profile for people with the condition,” said Vasant Narasimhan, Global Head Drug Development and Chief Medical Officer for Novartis. “Novartis is the global leader in understanding the role of S1P receptor modulation in the treatment of MS, and the positive results of the EXPAND study are a continuation of our ongoing efforts to innovate and meet the needs of patients. These data are a positive stride forward in an unserved disease area, and we look forward to evaluating next steps with health authorities.”

EXPAND is the largest randomized, controlled study in secondary progressive multiple sclerosis to date. Patients enrolled in EXPAND were representative of a general SPMS population. They must have been diagnosed with SPMS and also demonstrated progression of disability in the two years prior to study. The majority of patients had non-relapsing SPMS. The mean age at study entry was 48 years,and patients had a median Expanded Disability Status Scale (EDSS) score of 6.0, which corresponds to the use of walking aid.

 Novartis will complete full analyses of the EXPAND data and evaluate next steps in consultation with health authorities. The full study results, including data from primary and secondary endpoints, will be submitted for publication.