Skip to main content
. 2021 Mar 29;78(5):1–10. doi: 10.1001/jamaneurol.2021.0405

Table 2. Summary of Efficacy Results in the Full Analysis Set.

End point from baseline to week 108 Ponesimod, 20 mg (n = 567) Patients included in analysis, No. Teriflunomide, 14 mg (n = 566) Patients included in analysis, No. RR (99% CL) or Difference, RR, or HR (95% CL) P value
Primary end point
Mean annualized relapse rate/y (95% CL)a 0.202 (0.173-0.235) 567 0.290 (0.254-0.331) 566 0.695 (0.536-0.902)b <.001
Secondary end points
LS mean FSIQ-RMS weekly symptoms score change (95% CL)c −0.01 (−1.60 to 1.58) 449 3.56 (1.96-5.16) 458 −3.57 (−5.83 to −1.32)d .002
Mean cumulative combined unique active lesions/y (95% CL)e 1.405 (1.215-1.624) 539 3.164 (2.757-3.631) 536 0.444 (0.36-0.54)f <.001
Patients with first 12-wk confirmed disability accumulation, No. (%)g 57 (10.1) 567 70 (12.4) 566 0.83 (0.58-1.18)h .29
Exploratory end points
Patients with first 24-wk confirmed disability accumulation, No. (%)g 46 (8.1) 567 56 (9.9) 566 0.84 (0.57 to 1.24)h .37
Mean cumulative new gadolinium-enhancing T1 lesions/scan (95% CL) 0.18 (0.141-0.224) 540 0.43 (0.351-0.525) 538 0.42 (0.31-0.56)f <.001
LS mean change in brain volume, % (95% CL)i −0.91 (−1.03 to −0.79) 436 −1.25 (−1.36 to −1.13) 434 0.34 (0.17 to 0.50)d <.001
Estimated mean NEDA-3, % (95% CL)i 25.0 (21.4-29.0) 564 16.4 (13.5-19.8) 558 1.70 (1.27 to 2.28)j <.001
Estimated mean NEDA-4, % (95% CL)k 11.4 (8.7-14.6) 526 6.5 (4.7-9.0) 532 1.85 (1.24-2.76)j .003

Abbreviations: CL, confidence limit; DMT, disease-modifying therapy; EDSS, Expanded Disability Status Scale; FSIQ-RMS, Fatigue Symptom and Impact Questionnaire–Relapsing Multiple Sclerosis; HR, hazard ratio; LS, least squares; NEDA, no evidence of disease activity; OR, odds ratio; RR, rate ratio.

a

Confirmed relapses up to the end of the study; a negative binomial model was applied with Wald CLs and P value, the offset log time (years) to the end of the study, and covariates of EDSS strata (≤3.5 or >3.5), DMT in the last 2 years prior to randomization strata, and the number of relapses in the year prior study entry (≤1 or ≥2).

b

RR (99% CL).

c

Mixed-effects, repeated-measurements model with unstructured covariance, treatment, visit, treatment by visit interaction, baseline by visit interaction as fixed effects, baseline FSIQ-RMS score, EDSS strata (≤3.5 or >3.5), and DMT in the 2 years prior to randomization as covariates. A negative change from baseline indicates an improvement in fatigue symptoms.

d

Difference (95% CL).

e

Negative binomial model was applied with Wald 95% CIs, a P value, and an offset of log time (years) up to last magnetic resonance imaging scan and covariates of EDSS strata (≤3.5 or >3.5), DMT within the 2 years prior to randomization, and gadolinium-enhancing T1 lesions at baseline.

f

RR (95% CL).

g

Change from baseline to end of study; stratified by EDSS category and DMT within 2 years, Cox regression with 95% Wald CLs and a log-rank P value.

h

HR (95% CL).

i

Mixed model with linear time effect and covariates of EDSS strata (≤3.5 or >3.5), DMT within the 2 years prior to randomization strata, gadolinium-enhancing T1 lesions at baseline, and baseline brain volume.

j

OR (95% CL).

k

Logistic regression with treatment as factor, adjusted for covariates EDSS strata (≤3.5 or >3.5), DMT in the 2 years prior to randomization, the number of relapses in the year prior to study entry (≤1 or ≥2), and the presence of gadolinium-enhancing T1 lesions at baseline.