Table 1.
Efficacy outcomes in final analysis data set of OPTiM
| Talimogene laherparepvec (n = 295) | GM-CSF (n = 141) | Descriptive P-valuea | Difference | ||
|---|---|---|---|---|---|
| % | 95% CIb | ||||
| Response per investigator assessment in the intent-to-treat population (Stage IIIB–IVM1c disease) | |||||
| DRR, n (%) | 57 (19.3) | 2 (1.4) | < 0.0001 | 17.9 | 12.0–23.1 |
| CR, n (%) | 50 (16.9) | 1 (0.7) | – | – | – |
| PR, n (%) | 43 (14.6) | 8 (5.7) | – | – | – |
| ORR, % (95% CI)b | 31.5 (26.3–37.2) | 6.4 (3.0–11.8) | < 0.0001 | 25.1 | 17.4–31.7 |
| SD, n (%) | 132 (44.7) | 71 (50.4) | – | – | – |
| DCR, n (%) | 225 (76.3) | 80 (56.7) | – | 19.5 | 9.7–29.3 |
| Progressive disease, n (%) | 62 (21.0) | 42 (29.8) | – | – | – |
| Not assessed, n (%) | 8 (2.7) | 19 (13.5) | – | – | – |
| Estimated OS probability in the intent-to-treat population (Stage IIIB–IVM1c disease), % (95% CI) | |||||
| At 12 months | 73.7 (68.3–78.4) | 69.1 (60.6–76.2) | – | 4.6 | −4.7–13.8 |
| At 24 months | 49.8 (44.0–55.4) | 40.3 (32.0–48.4) | – | 9.5 | −0.5–19.6 |
| At 36 months | 38.9 (33.3–44.4) | 30.4 (22.9–38.3) | – | 8.4 | −1.2–18.0 |
| At 48 months | 34.5 (28.9–40.1) | 23.9 (16.8–31.7) | – | 10.6 | 1.2–20.0 |
| At 60 months | 33.4 (27.7–39.2) | NE | – | NE | NE |
| DRR, ORR, CR and DCR per investigator assessment according to disease stage | |||||
| DRR, n/N (%) | |||||
| IIIB/C | 29/88 (33.0) | 0/43 (0) | < 0.0001 | 33.0 | 19.1–43.9 |
| IVM1a | 18/75 (24.0) | 0/43 (0) | 0.0003 | 24.0 | 10.5–35.5 |
| IIIB–IVM1a | 47/163 (28.8) | 0/86 (0) | < 0.0001 | 28.8 | 20.3–36.5 |
| IVM1b | 4/64 (6.3) | 1/26 (3.8) | 1.0000 | 2.4 | −15.8–12.8 |
| IVM1c | 6/67 (9.0) | 1/29 (3.4) | 0.6710 | 5.5 | −11.5–16.2 |
| ORR, n/N (%) | |||||
| IIIB/C | 46/88 (52.3) | 2/43 (4.7) | < 0.0001 | 47.6 | 31.1–59.0 |
| IVM1a | 29/75 (38.7) | 2/43 (4.7) | < 0.0001 | 34.0 | 17.6–46.6 |
| IIIB–IVM1a | 75/163 (46.0) | 4/86 (4.7) | < 0.0001 | 41.4 | 30.6–49.9 |
| IVM1b | 9/64 (14.1) | 2/26 (7.7) | 0.5002 | 6.4 | −13.8–19.5 |
| IVM1c | 9/67 (13.4) | 3/29 (10.3) | 1.0000 | 3.1 | −16.3–16.5 |
| CR, n/N (%) | |||||
| IIIB/C | 31/88 (35.2) | 0/43 (0) | – | – | – |
| IVM1a | 15/75 (20.0) | 1/43 (2.3) | – | – | – |
| IIIB–IVM1a | 46/163 (28.2) | 1/86 (1.2) | – | – | – |
| IVM1b | 2/64 (3.1) | 0/26 (0) | – | – | – |
| IVM1c | 2/67 (3.0) | 0/29 (0) | – | – | – |
| PR, n/N (%) | |||||
| IIIB/C | 15/88 (17.0) | 2/43 (4.7) | – | – | – |
| IVM1a | 14/75 (18.9) | 1/43 (2.3) | – | – | – |
| IIIB–IVM1a | 29/163 (17.8) | 3/86 (3.5) | – | – | – |
| IVM1b | 7/64 (10.9) | 2/26 (7.7) | – | – | – |
| IVM1c | 7/67 (10.4) | 3/29 (10.3) | – | – | – |
| DCR, n/N (%) | |||||
| IIIB/C | 75/88 (85.2) | 23/43 (53.5) | – | 31.7 | 13.9–48.6 |
| IVM1a | 54/75 (72.0) | 24/43 (55.8) | – | 16.2 | −2.7–34.6 |
| IIIB–IVM1a | 129/163 (79.1) | 47/86 (54.7) | – | 24.5 | 11.5–37.0 |
| IVM1b | 50/64 (78.1) | 16/26 (61.5) | – | 16.6 | −4.9–39.3 |
| IVM1c | 46/67 (68.7) | 17/29 (58.6) | – | 10.0 | −11.4–32.2 |
aP-values calculated using Fisher’s Exact Test
bThe Clopper-Pearson method was used to calculate exact CIs for binary endpoints. Wilson’s score method with continuity correction was used to calculate an approximate CI for between-group differences in binary rates
CI confidence interval, CR complete response, DCR disease control rate, DRR durable response rate, GM-CSF granulocyte-macrophage colony-stimulating factor, NE not estimable, ORR overall response rate, OS overall survival, PR partial response