Safety and activity of nivolumab (nivo) monotherapy and nivo in combination with ipilimumab (ipi) in recurrent glioblastoma (GBM): Updated results from checkmate-143.

2014Background: Patients (pts) with recurrent/progressive GBM have a poor prognosis and limited therapeutic options. The phase 1 CheckMate-143 study evaluates safety and tolerability of the anti-PD-1 monoclonal antibody nivo, alone or in combination with the anti-CTLA-4 antibody ipi, in pts with recurrent GBM. Preliminary results from a phase 1 safety cohort (Cohort 1, Methods) were presented previously. Herein, we present updated safety, overall survival (OS) data, and PD-L1 biomarker status for the completed phase 1 Cohorts 1 and 1b. Methods: Eligible pts had a first recurrence of GBM after radiation and temozolomide. In Cohort 1, 20 pts were randomized 1:1 to receive nivo 3 mg/kg (N3) every 2 weeks (Q2W) or nivo 1 mg/kg + ipi 3 mg/kg every 3 weeks (Q3W; N1+I3) for 4 doses followed by N3 Q2W. Pts in Cohort 1b (n = 20) received nivo 3 mg/kg + ipi 1 mg/kg Q3W (N3+I1) for 4 doses followed by N3 Q2W. Treatment continued until disease progression or unacceptable toxicity. PD-L1 status was determined by immun...