- For patients taking placebo, median time to flare was 100 days
Rapid resolution of index flare at Day 15—with no new flares through Week 16—was achieved by significantly more patients receiving ILARIS1
Percentage of Patients Achieving Complete Response* vs Placebo at Week 161
150 mg or 2 mg/kg
(OR [95% CI]; 23.75 [4.38, 227.53])
(OR [95% CI]; 8.94 [1.72, 86.41])
(OR [95% CI]; 9.17 [1.51, 94.61])
- At Day 15, the majority of patients with FMF (81%, n/N=25/31), HIDS/MKD (65%, n/N=24/37), and TRAPS (64%, n/N=14/22) who received ILARIS achieved rapid resolution of index disease flare vs placebo: FMF (31%, n/N=10/32), HIDS/MKD (37%, n/N=13/35), and TRAPS (21%, n/N=5/24)
In the same study:
After the initial dose at Day 15, ILARIS improved disease activity as measured by PGA, as well as CRP levels1,2
Percentage of Patients Showing No or Minimal Signs of Disease Activity (PGA score <2) vs Placebo at Day 151
150 mg or 2 mg/kg
- 10% of patients with FMF in the ILARIS group had mild disease vs 19% in the placebo group
- In the ILARIS group, 55% had moderate disease and 36% had severe disease compared with 59% and 22%, respectively, in the placebo group
- 27% of patients with HIDS/MKD in the ILARIS group had mild disease vs 20% in the placebo group
- In the ILARIS group, 60% had moderate disease and 14% had severe disease compared with 60% and 20%, respectively, in the placebo group
- 41% of patients with TRAPS in the ILARIS group had mild disease vs 46% in the placebo group
- In the ILARIS group, 50% had moderate disease and 9% had severe disease compared with 46% and 8%, respectively, in the placebo group
At Day 15, CRP ≤10 mg/L Achieved by1:
CRP treatment comparisons (OR [95% CI]): FMF (22.51 [5.41, 93.62]), HIDS/MKD (6.05 [2.14, 17.12]), and TRAPS (3.88 [1.05, 14.26]).
The efficacy of ILARIS was assessed in patients with PFS across 3 disease cohorts: FMF, HIDS/MKD, and TRAPS. In the 16-week, double-blind, placebo-controlled treatment period, patients were randomized to receive ILARIS 150 mg (2 mg/kg for a body weight of ≤40 kg) or placebo every 4 weeks for 16 weeks and were allowed uptitration to ILARIS 300 mg (or 4 mg/kg) every 4 weeks for patients whose disease flare did not resolve or who had persistent disease, or active treatment.
The primary endpoint of the 16-week treatment period was the proportion of patients achieving a complete response without any dose adjustments.
Complete response was defined as resolution of index flare (PGA <2 and CRP ≤10 mg/L or a ≥70% reduction from baseline) at Day 15 and no new flare (PGA ≥2 and CRP ≥30 mg/L) throughout the 16-week treatment period.
A 5-point PGA scale was used by physicians to assess overall disease severity, where 0=no disease-associated signs and symptoms, 1=minimal, 2=mild, 3=moderate, and 4=severe. The key signs and symptoms assessed in the PGA for each condition were the following: FMF: abdominal pain, skin rash, chest pain, and arthralgia/arthritis; HIDS/MKD: abdominal pain, lymphadenopathy, and aphthous ulcers; TRAPS: abdominal pain, skin rash, musculoskeletal pain, and eye manifestations.
In Study Part 1:
The majority of patients achieved complete clinical response at Weeks 1 and 8 after the first dose of ILARIS1,3
Percentage of Patients Achieving Complete Clinical Response With ILARIS
- Physician’s assessment of disease activity ≤ minimal (rated on a 5-point scale consisting of absent, minimal, mild, moderate, and severe)
- Assessment of skin disease ≤ minimal (rated on a 5-point scale consisting of absent, minimal, mild, moderate, and severe)
- Normal serum values of CRP and SAA (<10 mg/L)
Assessment of disease activity included a composite of the following symptoms: urticarial skin rash, headache/migraine, fatigue/malaise, conjunctivitis, arthralgia, myalgia, and other symptoms related or unrelated to CAPS.
In Study Part 2:
After 3 doses† of ILARIS, 100% of patients remained flare free through 24 weeks3‡
Percentage of Patients Who Were Flare Free vs Placebo at Week 32
- During the open-label treatment period (Part 3), 97% (n/N=30/31) of patients were without disease relapse4||
Physician’s Global Assessment of Disease Activity and Assessment of Skin Disease1:
- 93% (n/N=14/15) had no rash and 7% (n/N=1/15) had minimal rash after treatment with ILARIS vs 31% (n/N=5/16) and 19% (n/N=3/16), respectively, with placebo
- 100% (n/N=15/15) had no or minimal disease activity after treatment with ILARIS vs 25% (n/N=4/16) with placebo
- 94% (n/N=29/31) had no rash and 6% (n/N=2/31) had minimal rash after treatment with ILARIS
- 97% (n/N=30/31) had no or minimal disease activity after treatment with ILARIS
- Analysis has not been adjusted for multiple comparisons. No conclusions of statistical or clinical significance can be drawn
A 3-part study in patients with CAPS (MWS) treated with a subcutaneous dose of ILARIS 150 mg (in patients weighing >40 kg) or ILARIS 2 mg/kg (in patients weighing ≥15 kg and ≤40 kg) every 8 weeks.
An 8-week open-label treatment period in which 35 patients were treated with a single injection of ILARIS 150 mg.
A double-blind, randomized withdrawal phase in which patients who achieved a complete clinical response and did not relapse by Week 8 in Part 1 were randomized to ILARIS 150 mg or 2 mg/kg in patients weighing ≥15 kg and ≤40 kg (n=15) or placebo (n=16) every 8 weeks for 24 weeks. During Part 2, patients continued with blinded treatment unless a relapse occurred to prompt early entry into Part 3.
An open-label treatment period in which patients received ILARIS 150 mg (n=31) every 8 weeks. Patients entered Part 3 at the end of Part 2 or at the time of relapse, whichever occurred first. For patients who completed Part 2 without disease flare, Part 3 had a duration of 16 weeks. For patients who had disease relapse in Part 2, Part 3 had a duration of up to 40 weeks. The total study duration was 48 weeks.
The primary endpoint was the proportion of patients experiencing disease flare or relapse in Part 2.