Efficacy
Pooled analysis results in patients with MAS in Still's disease1
Gamifant helped control hyperinflammation in patients with MAS in Still’s disease with an inadequate response or intolerance to glucocorticoids, or with recurrent MAS.1
Key efficacy measure: 8-component composite endpoint
Complete response (CR) at week 8
53%
of patients achieved CR at week 8
(95% CI: 37.2, 69.9)1
CR was observed as early as day 10 in 1 patient2,*
*The results and observations related to this endpoint were not specifically designed or statistically powered to evaluate efficacy outcomes.
Individual components of the composite endpoint at week 81
MAS clinical activity at week 8†
Clinical MAS remission was assessed using a visual analog scale (VAS) where 0 cm referred to no clinical signs or symptoms of MAS and 10 cm referred to the worst possible clinical signs and symptoms of MAS.1
MAS clinical activity VAS of ≤1/10 cm was observed in 82% (32/39) of patients1
Exploratory endpoint
Change in mean prednisolone-equivalent glucocorticoid dose†: Mean dose was 9.7 mg/kg/day at baseline and 0.8 mg/kg/day at week 8.2
†Assessment was not statistically powered or designed to evaluate treatment effect. These data are descriptive in nature and are observational only. No formal conclusion can be drawn.
Pharmacodynamic profile: CXCL9 levels observed in the clinical trials
CXCL9 can be a useful surrogate marker for IFNγ because it is selectively induced by the cytokine. Reduction of CXCL9 levels may indicate neutralization of IFNγ activity.3
In the pivotal trials, a decrease in serum CXCL9 levels was observed. Reductions in CXCL9 were consistent with early onset of clinical action and time to response seen with Gamifant in the clinical trials.2
Levels of CXCL9 were studied as a secondary endpoint for pharmacodynamic assessments.
Reference values‡: Average CXCL9 levels in patients with AOSD and sJIA without MAS as measured in separate external studies:
....595
Average value in patients with AOSD: (595.6±790.8)4
....837
Average value in patients with sJIA: 837 (471-2505)5
AOSD=adult-onset Still’s disease; sJIA=systemic juvenile idiopathic arthritis.
‡The average CXCL9 levels above should be used for reference only and were not collected as part of Gamifant trials.
IFNγ and CXCL9 brochure
For more information about IFNγ and CXCL9, download this guide