Table 2 Selected clinical trials of PD-1/PD-L1 immunotherapies according to cancer type
From: Development and clinical applications of cancer immunotherapy against PD-1 signaling pathway
Trial | Subject | Study vs. comparison | Result | Reference | FDA approval outcome |
|---|---|---|---|---|---|
Melanoma | |||||
 Keynote 006 | No prior immunotherapy, any PD-L1 level | P 10 mg/kg vs. I | OS: 32.7 vs. 15.9 months PFS: 8.4 vs. 3.4 months | Schachter et al. 2017 | FDA approved pembrolizumab for first-line treatment in advanced melanoma |
 Keynote 002 | With prior ipilimumab, any PD-L1 level | P 2 mg/kg vs. P 10 mg/kg vs C | PFS: 34% vs. 38% vs. 16% | Ribas et al. 2015 | FDA approved pembrolizumab for second-line treatment in advanced melanoma |
 CheckMate 066 | Previous untreated, any PD-L1 level | N 3 mg/kg vs. dacarbazine | OS: 37.5vs. 11.2 months PFS: 5.1 vs. 2.2 months | Ascierto et al. 2019 | FDA approved Opdivo for treatment of BRAF V600 wild-type unresectable or metastatic melanoma |
 CheckMate 037 | With prior ipilimumab, any PD-L1 level | N 3 mg/kg vs. C | OS: 16 vs. 14 months PFS: 3.1 vs. 3.7 months ORR: 27% vs. 10% | Larkin et al. 2018 | FDA approved Opdivo for unresectable or metastatic melanoma following treatment with ipilimumab or BRAF inhibitor |
 CheckMate 067 | Previous untreated, any PD-L1 level | N 3 mg/kg + I 3 mg/kg vs. N 3 mg/kg vs. I 3 mg/kg | OS: not reached vs. 37.6 vs. 19.9 months PFS: 11.5 vs. 6.9 vs. 2.9 months | Larkin et al. 2015 | FDA approved nivolumab in combination with ipilimumab for treatment of BRAFV600 wild-type and BRAF V600 mutation positive unresectable or metastatic melanoma |
 CheckMate 511 | Previous untreated, any PD-L1 level | N 3 mg/kg + I 1 mg/kg vs. N 1 mg/kg + I 3 mg/kg | PFS: 9.9 vs. 8.9 months ORR: 45.6% vs. 50.6% Grade 3 to 5 AEs: 34% vs. 48% | Lebbe et al. 2019 |  |
Renal Cell Carcinoma | |||||
 CheckMate 025 | With prior treatment, any PD-L1 level | N 3 mg/kg vs. everolimus | OS: 25 vs. 19.6 months ORR: 22% vs. 4% | Escudier et al. 2017 | FDA approved nivolumab for treatment of advanced renal cell carcinoma with no prior anti-angiogenic therapy |
 CheckMate 214 | Previous untreated intermediate to poor risk, any PD-L1 level | N 3 mg/kg + I 1 mg/kg vs. sunitinib | OS: not reached vs. 26.6 months PFS: 8.2 vs. 8.3 months ORR: 42% vs. 29% | Motzer et al. 2019; Escudier et al. 2017 | FDA approved nivolumab and ipilimumab for treatment of intermediate or poor risk, previously untreated advanced renal cell carcinoma |
Non-Small Cell Lung Cancer | |||||
 Keynote 024 | Previous untreated, with TPS over 50% | P 200 mg vs. C | OS: 80.2% vs. 72.4% PFS: 10.3 vs. 6 months ORR: 44.8% vs. 27.8% | Reck et al. 2016 | FDA approved pembrolizumab for treatment of metastatic NSCLC whose tumors have high PD-LA expression with no EGFR or ALK genomic tumor aberrations |
 Keynote 189 | Previous untreated, any PD-L1 level | P 200 mg + C vs. C | OS: not reached vs. 11.3 months PFS: 8.8 vs. 4.9 months | Gandhi et al. 2018 | FDA approved pembrolizumab in combination with pemetrexed and platinum chemotherapy for first line treatment of metastatic non squamous NSCLC with no EGFR or ALK genomic tumor aberrations |
 Keynote 010 | With prior treatment, any PD-L1 level | P 2 mg/kg vs. P 10 mg/kg vs. docetaxel | Total population OS: 10.4 vs. 12.7 vs. 8.5 months PFS: 3.9 vs. 4.0 vs. 4.0 months TPS ≥ 50% OS: 14.9 vs. 17.3 vs. 8.2 months PFS: 5.0 vs. 5.2 vs. 4.1 months | Herbst et al. 2016 | FDA approved pembrolizumab as second-line treatment for PD-L1 Positive non-small cell lung cancer |
 IMpower 150 | Nonsquamous, previous untreated, any PD-L1 level | A 1200 mg + C + bevacizumab 15 mg/kg vs. C + bevacizumab | PFS: 8.3 vs. 6.8 months | Socinski et al., 2018 | FDA approved atezolizumab in combination with bevacizumab, paclitaxel, and carboplatin for first line treatment of metastatic non-squamous non-small cell lung cancer with no EGFR or ALK genomic tumor aberrations |
Head and Neck Cancer | |||||
 Keynote 040 | With prior treatment, any PD-L1 level | P 200 mg vs. C | OS: 8.4 vs. 6.9 months | Cohen et al. 2018 | FDA approved pembrolizumab for treatment of recurrent or metastatic squamous cell carcinoma of head and neck with disease progression on or after platinum-based therapy |
 CheckMate 141 | With prior treatment, any PD-L1 level | N 3 mg/kg vs. C | OS7.5 vs. 5.1 months | Kiyota et al. 2017 | FDA approved nivolumab for treatment of recurrent or metastatic squamous cell carcinoma of head and neck with disease progression on or after platinum-based therapy |