Baseline Subgroup Characteristics of EXPAND: A Phase 3 Study of Siponimod (BAF312) for the Treatment of Secondary Progressive Multiple Sclerosis (P3.084)

OBJECTIVE: To present baseline subgroup analysis of the EXPAND study population. BACKGROUND: EXPAND is the largest, controlled phase 3 study in secondary progressive multiple sclerosis (SPMS) investigating the therapeutic potential of siponimod. DESIGN/METHODS: Patients with SPMS aged 18-60 years and Expanded Disability Status Scale (EDSS) score between 3.0-6.5 were randomized (2:1) to receive either siponimod or placebo. The primary outcome measure is time to 3-month confirmed disability progression as measured by increase in EDSS. RESULTS: As of August 2015 (end of recruitment), 1649 patients were enrolled (mean age 48.0±7.9 years). Relapses in the 2 years prior to study start were documented in 35.1[percnt] patients (n=578) whereas 64.5[percnt] were free of clinical relapses (n=1064) [information was missing for 0.4[percnt] (n=7)], and 55.2[percnt] had an EDSS ≥6. In patients with prior relapses, median duration (years) of MS since first symptom/conversion to SPMS were 14.3/1.8. Results of baseline clinical/MRI measures were (mean±SD): EDSS 5.4±1.0, T25FW 17.0±20.9 seconds (s), 9Hole Peg Test (9HPT) in dominant hand (DH) 32.0±22.7s, 12-item MS Walking Scale (MSWS12) 68.0±22.7, and T2 lesion volume (T2LV) 16306.8±17164.0 mm 3 . The corresponding values for patients without prior relapses were: median duration (years) of MS since first symptom/conversion to SPMS 16.9/3.12, EDSS 5.4±1.1, T25FW 17.7±31.6s, 9HPT-DH 35.4±37.1s, MSWS12 68.5±23.2, and T2LV 14701.5±15267.8 mm 3 . Most patients had no Gd+T1 lesions at baseline (patients with vs. without prior relapse: 71.5[percnt] vs. 81.5[percnt]; difference[95[percnt]CI]: 10.1[percnt][5.6;14.5]). In patients with EDSS of 3.0-5.5 (n=717) vs. those with high EDSS (6.0-6.5, n=910): median duration (years) from MS symptom-onset was 14.6 vs. 17.0; median duration (years) since conversion to SPMS was 1.94 vs. 3.35; T2LV (mean±SD) 13723.0±15052.3 mm 3 vs. 16557.7±16683.8 mm 3 and no Gd+T1 lesions in 79.2[percnt] vs. 72.9[percnt]. CONCLUSIONS: Subgroup analysis of the EXPAND baseline characteristics confirms the consistency of the SPMS profile, and considerable representation of non-relapsing patients. Study Supported by Novartis Pharma AG Disclosure: Dr. Kappos9s institution (University Hospital Basel) has received royalty payments from Neurostatus Systems GmbH. Dr. Bar-Or has received personal compensation for activities with Bayer, Bayhill Therapeutics, Berlex, Biogen Idec, BioMS, Diogenix, Eli Lilly as a consultant, speaker and advisory board member. Dr. Cree has received personal compensation for activities with Abbvie, Biogen Idec, EMD Serono, Genzyme/sanofi aventis, Medimmune, Novartis and Teva. Dr. Cree has received research support from Acorda, EMD Serono, Hoffman La Roche, MedImmune, Novartis a Dr. Fox has received personal compensation for activities with Actelion, Biogen Idec, MedDay, Novartis, Questcor, Teva, and Xenoport as a consultant; served on advisory committees for Actelion, Biogen Idec and Novartis. Dr. Fox has received research suppo Dr. Giovannoni has received personal compensation for activities with AbbVie Biotherapeutics Inc., Biogen, Bayer HealthCare, Genzyme, Merck Serono, Sanofi-Aventis, Teva, Ironwood, and Novartis. Dr. Gold has received research support from Bayer HealthCare, Biogen Idec, Merck Serono, Novartis, and Teva Neuroscience. Dr. Vermersch has received personal compensation for activities with Biogen Idec, Sanofi, Bayer, Novartis, Merck Serono, GlaxoSmithKline, and Almirall. Dr. Vermersch has received research support from Biogen Idec, Sanofi, Bayer, and Merck Serono. Dr. Bhasin has received personal compensation for activities with Novartis Healthcare Pvt. Ltd. as an employee. Dr. Arnould has received personal compensation for activities with Novartis Pharma AG as an employee. Dr. Sidorenko has received personal compensation for activities with Novartis Pharma AG, Basel Switzerland as an employee. Dr. Wallstroem has received personal compensation for activities with Novartis as an employee.