Table 1.
Summary of efficacy outcomes from clinical studies of copanlisib in patients with indolent lymphoma
| Study | Histology | Number of patients | ORR, % (CR; PR) | DCR, % (SD) |
|---|---|---|---|---|
| Phase I | ||||
| NCT00962611 [34] | FL | 6 | 100 (2; 4) | 100 (0) |
| NCT02155582 [47] | FL | 1 | 100 (0; 1) | 100 (0) |
| Phase II | ||||
| NCT01660451; part A [52] | FL | 16a | 40.0 (3b; 3) | 93.3 (8) |
| CLL | 13 | 38.5 (0; 5) | 84.6 (6) | |
| MZL | 3 | 66.7 (0; 2) | 100 (1) | |
| SLL | 1 | 100 (0; 1) | 100 (0) | |
| NCT01660451; part B [35] | FL | 104 | 58.7 (15; 46) | 87.5c (35) |
| MZL | 23 | 69.6 (2; 14) | 87.0 (4) | |
| SLL | 8 | 75.0 (0; 6) | 87.5 (1) | |
| LPL/WM | 6 | 16.7 (0; 1) | 66.7 (3) | |
| NCT01660451; part B long-term follow-up [36] | FL | 104 | 58.7 (21; 40) | 87.5 (35d) |
| MZL | 23 | 78.3 (3; 15) | 87.0 (2) | |
| SLL | 8 | 75.0 (0; 6) | 87.5 (1) | |
| LPL/WM | 6 | 16.7 (0; 1) | 66.7 (3) | |
| NCT01660451; part B FL POD24 subset (Leppä et al.; submitted) | FL (POD24) | 68 | 60.3 (15; 26) | 88.2 (21) |
CLL chronic lymphocytic leukemia, CR complete response, DCR disease control rate, FL follicular lymphoma, LPL/WM lymphoplasmacytic lymphoma/Waldenstrӧm’s macroglobulinemia, MZL marginal zone lymphoma, ORR objective response rate, POD24 progression within 24 months, PR partial response, SD stable disease, SLL small lymphocytic lymphoma
aOne patient was excluded because they did not have any measurable lesion as per Cheson criteria at baseline; therefore, 15 patients were evaluable for response
bOne patient achieved an unconfirmed CR
cOne patient with unconfirmed SD and four with SD or PR recorded > 35 days from the last treatment were excluded
dIncludes one patient with unconfirmed early SD (SD was assessed earlier than 7 weeks after start of treatment, with no radiologic assessment of SD, PR, or CR at later cycles)