TABLE 3

Summary of primary, secondary and exploratory efficacy end-points (modified intention-to-treat population)

PlaceboSAR156597 Q2WSAR156597 QW
Subjects n109108108
Primary end-point
 Primary analysis (RANCOVA)#
  Death n (%)10 (9.2)7 (6.5)10 (9.3)
  Lung transplant n (%)01 (0.9)3 (2.8)
  Change from baseline to 52 weeks in FVC % pred
   Mean±sd–4.4±5.8–4.7±6.4–4.9±6.9
   p-value versus placebo0.420.39
 Sensitivity analysis
  Change from baseline to 52 weeks in FVC % pred
   LS mean±se–5.8±0.7–5.2±0.7–6.3±0.8
   LS mean difference versus placebo±se0.6±1.0–0.5±1.1
   95% CI–1.5–2.6–2.6–1.6
   p-value versus placebo0.590.63
Secondary end-points
 Disease progression at 52 weeks
  Number of events353542
  Probability of events0.5120.4600.537
  HR (95% CI) versus placebo1.023 (0.640–1.636)1.255 (0.801–1.966)
  p-value versus placebo0.900.31
 Decomposition of the disease progression composite end-point
  Absolute decrease in FVC % pred ≥10
   Number of events141320
   Probability of events0.1580.2620.389
   HR versus placebo0.9551.501
  Absolute decrease in DLCO % pred ≥15
   Number of events131512
   Probability of events0.3620.2060.138
   HR versus placebo1.1970.981
  Lung transplant
   Number of events012
   Probability of events0.0000.0110.028
  Death
   Number of events868
   Probability of events0.0890.0650.093
   HR versus placebo0.7481.018
Exploratory end-points n (%)
 Acute exacerbations of IPF
  Any suspected IPF exacerbation TEAE12 (11.0)7 (6.5)9 (8.3)
  Confirmed acute IPF exacerbation9 (8.3)5 (4.6)7 (6.5)
 Respiratory and non-elective hospitalisations
  Any hospitalisation18 (16.5)22 (20.4)38 (35.2)
  Type of hospitalisation
   Elective3 (2.8)9 (8.3)10 (9.3)
   Non-elective16 (14.7)17 (15.7)32 (29.6)
  Reason for hospitalisation
   Respiratory15 (13.8)13 (12.0)24 (22.2)
   Other5 (4.6)12 (11.1)22 (20.4)

Q2W: once every 2 weeks; QW: once every week; RANCOVA: rank-based analysis of covariance; FVC: forced vital capacity; LS: least squares; HR: hazard ratio; DLCO: diffusing capacity of the lung for carbon monoxide; IPF: idiopathic pulmonary fibrosis; TEAE: treatment-emergent adverse event. #: RANCOVA analysis is based on a global ranking combining both clinical events (death and transplantation) and continuous data (change from baseline in FVC % pred). See Methods section for more detail.