Table 1.
Key completed studies evaluating carfilzomib in MM
| Study | Population | Patients | ORR (%) | CR + VGPR | PFS (months) | OS (months) |
|---|---|---|---|---|---|---|
| PX-171-001 (Phase I) carfilzomib (single agent)13 | Hematologic malignancies (MM, NHL, WM, HL) | 29 | NR | NR | NR | NR |
| PX-171-002 (Phase I) carfilzomib (single agent)12 | Hematologic malignancies (MM, NHL, HL) | 48 | NR | NR | NR | NR |
| PX-171-003-A0 (Phase IIB) carfilzomib (single agent)35 | RRMM | 46 | 16.7 | 0 (0 + 0) | 3.5 | NR |
| PX-171-003-A1 (Phase II) carfilzomib (single agent)15 | RRMM | 266 | 23.7 | 5.5% (0.4% + 5.1%) | 3.7 | 15.6 |
| PX-171-004 (Phase II) carfilzomib (single agent, 20 mg/m2 versus 27 mg/m2)17 | RRMM | 129 | 42.4 versus 52.2 | 17% (3.4% + 13.6%) versus 28.4% (1.5% + 26.9%) | 8.2 versus NR | NR |
| PX-171-005 (Phase II) carfilzomib (single agent)33 | RRMM: renal dysfunction | 50 | 25.5 | 0 (0 + 0) | NR | NR |
| PX-171-006 (Phase IB) carfilzomib + Rd22 | Relapsed or progressive MM | 40 | 62.5 | 35% (2.5% + 32.5%) | 10.2 | NR |
| PX-171-006 (Phase II) carfilzomib + Rd21 | Relapsed or progressive MM | 52 | 76.9 | 42.2% (5.7% + 36.5%) | 28.7 | 37 |
| PX-171-007 (Phase IB/II) carfilzomib (single agent) dose-expansion cohort16 | Relapsed MM, solid tumors or lymphoma | 33 | 55 | 28% (5% + 23%) | 7 | NR |
| Carfilzomib + Rd (Phase I/II)26 | Newly diagnosed | 53 | 98 | 81% (62% + 19%) | NR | NR |
| Carfilzomib + clarithromycin + Rd (Phase I)27 | Newly diagnosed | 24 | 87 | 61% (13% + 48%) | NR | NR |
| FOCUS: PX-171-011 (Phase III) carfilzomib versus best supportive care18 | RRMM | 315 | NR | NR | NR | NR |
| ASPIRE: PX-171-009 (Phase III) carfilzomib + lenalidomide + dexamethasone versus lenalidomide + dexamethasone Planned interim analysis25 | Relapsed MM | 792 | NR | NR | 26.3 versus 17.6 | NR |
Abbreviations: ORR, overall response rate; CR, complete response; VGPR, very good partial response; PFS, progression-free survival; OS, overall survival; RRMM, relapsed and refractory multiple myeloma; NHL, non-Hodgkin’s lymphoma; WM, Waldenström’s macroglobulinemia; Rd, Revlimid and dexamethasone; NR, not reported.