Results of early clinical trials of novel agents
| Drug . | Phase of study . | Patient population . | Prior lines of therapy . | N . | ORR, % . | VGPR, % . | DOR, mo . | PFS, mo . |
|---|---|---|---|---|---|---|---|---|
| Venetoclax13 | Phase 1 | Relapsed, PI and IMiD exposed | 5 (1-15) | 66 | 21 | 15 | 9.7 | 2.6 (TTP) |
| Venetoclax + bortezomib | Phase 1 | Relapsed, PI and IMiD exposed | 3 (1-13) | 66 | 67 | 23 | NA | NA |
| Selinexor17 | Phase 1 | Refractory to Btz, Cfz, Len, and Pom | 7 (3-16) | 79 | 21 | 5 | 5 | 2.3 |
| Selinexor + bortezomib | Phase 1 | ≥1 line of prior therapy, not refractory to bortezomib | 4 (1-11) | 22 | 77 | 18 | NA | NA |
| Afuresertib22 | Phase 1 | Refractory to available standard therapy | NA | 34 | 9 | 0 | NA | NA |
| Trametinib25 | Phase 1 | Relapsed MM, 1 cohort with Ras mutation, 1 cohort with no mutation | 4 (2-8) | 12/13 | 8 (1/12) | 0 | NA | NA |
| LGH44724 | Phase 1 | Relapsed, refractory MM | 4 (1-16) | 59 | 11 | 2 | 5.5 | NA |
| Isatuximab30 | Phase 1/2 | Double refractory to an IMiD and PI or have received ≥3 prior lines of therapy: 4 dose cohorts | 5-6 | 96 | 9-29 | 8-13 | NA | NA |
| Isatuximab + Pom | Phase 1/2 | ≥2 prior anti-MM therapies, including Len and a PI | 4 (3-11) | 26 | 65 | 27 | 9 | NA |
| MOR202 | Phase 1 | At least 2 prior lines of therapy | 5 | 18 | 28 | 11 | NA | 4.7 |
| MOR202 + Len | Phase 1 | At least 1 prior line | 3 | 17 | 71 | 18 | NA | NR |
| MOR202 + Pom | Phase 1 | At least 2 prior lines of therapy | 4 | 13 | 46 | 8 | NA | 17.5 |
| Nivolumab36 | Phase 2 | Relapsed refractory | 4 (2-6) | 27 | 0 | 0 | NA | NA |
| GSK2857916 | Phase 1 | Relapsed refractory | >4 (70%) | 30 | 27 | 10 | NA | NA |
| Marizomib | Phase 1 weekly | Relapsed, at least 2 prior regimens | 4 (1-11) | 32 | 3 | 0 | NA | NA |
| Phase 1 twice weekly | 6 (2-19) | 36 | 11 | 0 | NA | NA | ||
| Melflufen | Phase 2 | 4 (2-9) | 31 | 33 | 3 | NA | 7.6 |
| Drug . | Phase of study . | Patient population . | Prior lines of therapy . | N . | ORR, % . | VGPR, % . | DOR, mo . | PFS, mo . |
|---|---|---|---|---|---|---|---|---|
| Venetoclax13 | Phase 1 | Relapsed, PI and IMiD exposed | 5 (1-15) | 66 | 21 | 15 | 9.7 | 2.6 (TTP) |
| Venetoclax + bortezomib | Phase 1 | Relapsed, PI and IMiD exposed | 3 (1-13) | 66 | 67 | 23 | NA | NA |
| Selinexor17 | Phase 1 | Refractory to Btz, Cfz, Len, and Pom | 7 (3-16) | 79 | 21 | 5 | 5 | 2.3 |
| Selinexor + bortezomib | Phase 1 | ≥1 line of prior therapy, not refractory to bortezomib | 4 (1-11) | 22 | 77 | 18 | NA | NA |
| Afuresertib22 | Phase 1 | Refractory to available standard therapy | NA | 34 | 9 | 0 | NA | NA |
| Trametinib25 | Phase 1 | Relapsed MM, 1 cohort with Ras mutation, 1 cohort with no mutation | 4 (2-8) | 12/13 | 8 (1/12) | 0 | NA | NA |
| LGH44724 | Phase 1 | Relapsed, refractory MM | 4 (1-16) | 59 | 11 | 2 | 5.5 | NA |
| Isatuximab30 | Phase 1/2 | Double refractory to an IMiD and PI or have received ≥3 prior lines of therapy: 4 dose cohorts | 5-6 | 96 | 9-29 | 8-13 | NA | NA |
| Isatuximab + Pom | Phase 1/2 | ≥2 prior anti-MM therapies, including Len and a PI | 4 (3-11) | 26 | 65 | 27 | 9 | NA |
| MOR202 | Phase 1 | At least 2 prior lines of therapy | 5 | 18 | 28 | 11 | NA | 4.7 |
| MOR202 + Len | Phase 1 | At least 1 prior line | 3 | 17 | 71 | 18 | NA | NR |
| MOR202 + Pom | Phase 1 | At least 2 prior lines of therapy | 4 | 13 | 46 | 8 | NA | 17.5 |
| Nivolumab36 | Phase 2 | Relapsed refractory | 4 (2-6) | 27 | 0 | 0 | NA | NA |
| GSK2857916 | Phase 1 | Relapsed refractory | >4 (70%) | 30 | 27 | 10 | NA | NA |
| Marizomib | Phase 1 weekly | Relapsed, at least 2 prior regimens | 4 (1-11) | 32 | 3 | 0 | NA | NA |
| Phase 1 twice weekly | 6 (2-19) | 36 | 11 | 0 | NA | NA | ||
| Melflufen | Phase 2 | 4 (2-9) | 31 | 33 | 3 | NA | 7.6 |
Btz, bortezomib; Cfz, carfilzomib; DOR, duration of response; IMiD, immunomodulatory drug; Len, lenalidomide; MM, multiple myeloma; NA, not applicable; NR, not reached; ORR, overall response rate; PFS, progression-free survival; PI, proteasome inhibitor; Pom, pomalidomide; TTP, time to progression; VGPR, very-good partial response.