Table 1.
Overview of indoximod clinical data (phase 1b/2, phase 2 trials).
| Disease | Design | Combination | Number of Patients Evaluated | Evidence of Efficacy? | NCT Reference Number | References |
|---|---|---|---|---|---|---|
| Melanoma cutaneous, mucosal, uveal | Phase 2, single arm, 1200 mg bid | SOC Pembrolizumab (evaluated), Nivolimumab or Ipilimumab (non-evaluated) | 85 | Yes ORR 53% (PD-L1+ 77%, PD-L1– 37%) CR 18% | 03301636 | (53) |
| Prostate metastatic CRPC | Phase 2, dual arm, randomized 1200 mg bid or placebo | SOC sipuleucel-T vaccine | 46 (24 placebo, 22 indoximod) | Yes 10.3 mos treatment vs 4.1 mos placebo (p = 0.011) | 0156092 | (54) |
| Acute Myeloid Leukemia | Phase 1b/2 dual arm (var. doses ± placebo in Phase 2) | SOC Induction + Maintenance Chemotherapy | 6 | High occurrence of MRD after one cycle of induction therapy in 5/6 patients | 02835729 | (55) |
| Brain Adult glioma | Phase 1b/2a single arm (var. doses) | SOC Temozolomide + Bevacizumab + Radiotherapy | 12 | SD (5-10 mos) 3/12 patients previously refractory to SOC; near PR, 1/12 pts with progressive ongoing reduction in tumor size | 02052648 | (56) |
| Brain Pediatric | Phase 1b/2 single arm (var. doses) | SOC Temozolomide ± Radiotherapy | 29 (12 chemo, 17 chemo+radio) | Yes TTRF = 12 mos vs. 3.2 mos chemo+radio vs. chemo-only | 02502708 | (57) |
| Pancreas PDAC | Phase 2 single arm 1200 bid | SOC Gemcitabine or Nab-Paclitaxel | 104 | Some, but did not meet pre-specifed goal 30% reduction in HR. Median OS = 10.9 ORR = 46.2% | 02077881 | (58) |
| Breast Stage IV (naïve) | Phase 2 single arm 1200 bid | SOC Taxotere | 169 | No No difference in ORR, PFS, or OS | 01191216 | NA |
SOC, standard of care; SD, stable disease; PR, partial response; CR, complete response; HR, hazard ratio; MRD, minimal residual disease; PFS, progression-free survival; TTRF, time to regimen failure.