Efficacy outcomes in final analysis data set of OPTiM

Talimogene laherparepvec (n = 295)GM-CSF (n = 141)Descriptive P-valueaDifference
%95% CIb
Response per investigator assessment in the intent-to-treat population (Stage IIIB–IVM1c disease)
 DRR, n (%)57 (19.3)2 (1.4)< 0.000117.912.0–23.1
 CR, n (%)50 (16.9)1 (0.7)
 PR, n (%)43 (14.6)8 (5.7)
 ORR, % (95% CI)b31.5 (26.3–37.2)6.4 (3.0–11.8)< 0.000125.117.4–31.7
 SD, n (%)132 (44.7)71 (50.4)
 DCR, n (%)225 (76.3)80 (56.7)19.59.7–29.3
 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 months73.7 (68.3–78.4)69.1 (60.6–76.2)4.6−4.7–13.8
 At 24 months49.8 (44.0–55.4)40.3 (32.0–48.4)9.5−0.5–19.6
 At 36 months38.9 (33.3–44.4)30.4 (22.9–38.3)8.4−1.2–18.0
 At 48 months34.5 (28.9–40.1)23.9 (16.8–31.7)10.61.2–20.0
 At 60 months33.4 (27.7–39.2)NENENE
DRR, ORR, CR and DCR per investigator assessment according to disease stage
 DRR, n/N (%)
  IIIB/C29/88 (33.0)0/43 (0)< 0.000133.019.1–43.9
  IVM1a18/75 (24.0)0/43 (0)0.000324.010.5–35.5
  IIIB–IVM1a47/163 (28.8)0/86 (0)< 0.000128.820.3–36.5
  IVM1b4/64 (6.3)1/26 (3.8)1.00002.4−15.8–12.8
  IVM1c6/67 (9.0)1/29 (3.4)0.67105.5−11.5–16.2
 ORR, n/N (%)
  IIIB/C46/88 (52.3)2/43 (4.7)< 0.000147.631.1–59.0
  IVM1a29/75 (38.7)2/43 (4.7)< 0.000134.017.6–46.6
  IIIB–IVM1a75/163 (46.0)4/86 (4.7)< 0.000141.430.6–49.9
  IVM1b9/64 (14.1)2/26 (7.7)0.50026.4−13.8–19.5
  IVM1c9/67 (13.4)3/29 (10.3)1.00003.1−16.3–16.5
 CR, n/N (%)
  IIIB/C31/88 (35.2)0/43 (0)
  IVM1a15/75 (20.0)1/43 (2.3)
  IIIB–IVM1a46/163 (28.2)1/86 (1.2)
  IVM1b2/64 (3.1)0/26 (0)
  IVM1c2/67 (3.0)0/29 (0)
 PR, n/N (%)
  IIIB/C15/88 (17.0)2/43 (4.7)
  IVM1a14/75 (18.9)1/43 (2.3)
  IIIB–IVM1a29/163 (17.8)3/86 (3.5)
  IVM1b7/64 (10.9)2/26 (7.7)
  IVM1c7/67 (10.4)3/29 (10.3)
 DCR, n/N (%)
  IIIB/C75/88 (85.2)23/43 (53.5)31.713.9–48.6
  IVM1a54/75 (72.0)24/43 (55.8)16.2−2.7–34.6
  IIIB–IVM1a129/163 (79.1)47/86 (54.7)24.511.5–37.0
  IVM1b50/64 (78.1)16/26 (61.5)16.6−4.9–39.3
  IVM1c46/67 (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