Review Article

Pivotal Roles of GM-CSF in Autoimmunity and Inflammation

Table 2

Effects of GM-CSF inhibitors in RA.

Drug Regimen Patients Efficacy (versus placebo)

Mavrilimumab
(CAM-3001)
Phase II
[121]
10, 30, 50, or 100 mg, SC
every other week
= 233
On stable MTX
DAS28 change: −1.2 at week 12
41.0%, 61.0%, 53.8%, and 66.7% versus 34.7%
( = 0.543, 0.011, 0.071, and 0.001)
ACR at week 12 100 mg dose versus Placebo
ACR20: 69.2% versus 40.0% ()
ACR50: 30.8% versus 12.0% ()
ACR70: 17.9% versus 4.0% ()

MOR103
Phase Ib/IIa
[122]
0.3, 1.0, or 1.5 mg/kg, IV
once a week for 4 weeks, with follow-up to 16 weeks
= 96Significant differences in DAS28 change between placebo and MOR-103:
None, weeks 4 through 10, weeks 4 through 6
ACR at week 4, 1.0 mg/kg versus placebo
ACR20: 68.2% versus 7.4% ()
ACR50: 22.7% versus 3.7% (ns)
ACR70: 4.5% versus 0.0% (ns)

SC: subcutaneous injection; IV: intravenous injection; MTX: methotrexate; DAS28: 28-joint disease activity score; ACR20: American College of Rheumatology 20% response rate; ACR50: American College of Rheumatology 50% response rate; ACR70: American College of Rheumatology 70% response rate.