Full positive results of EXPAND study for siponimod in secondary progressive multiple sclerosis .- Novartis.

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 significant reductions in the risk of three- (primary endpoint) and six-month confirmed disability progression with siponimod versus placebo and favorable 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 SPMS patients, including many who had reached a non-relapsing stage and high level of disability. SPMS is a form of MS that leads to progressive, irreversible disability, largely independent of relapses. Patients transition to SPMS after an initial phase of relapsing-remitting MS (RRMS), the most commonly diagnosed type of MS. There is a high unmet medical need for new treatments that are safe and effective for patients with SPMS.

Siponimod is an oral selective modulator of sphingosine-1-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 (p=0.013; primary endpoint); efficacy was consistent across many pre-defined 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% (p=0.0058).� Slowed the rate of brain volume loss by 23% (relative difference; mean across 12 and 24 months, p=0.0002).� Limited the increase of T2 lesion volume by approximately 80% (mean over 12 and 24 months, p<0.0001). reduced annualized relapse rate arr by 55 p><0.0001).� 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 plans to file for regulatory approval of siponimod for SPMS with the 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.

See- Kappos L et al. "Siponimod versus placebo in secondary progressive multiple sclerosis: a double-blinded randomized, phase 3 study". The Lancet. 2018; DOI 10.1016/S0140-6736(18)30475-6.