Table 1.
Baseline characteristics of subjects in the single and multiple ascending dose cohorts
| AKCEA-APOCIII-LRX, single-dose cohort | ||||||
|---|---|---|---|---|---|---|
| Pooled placebo (n = 10) | 10 mg (n = 6) | 30 mg (n = 6) | 60 mg (n = 6) | 90 mg (n = 6) | 120 mg (n = 6) | |
| Gender (male:female) | 3:7 | 4:2 | 4:2 | 5:1 | 4:2 | 3:3 |
| Age (years), mean (SD) | 54.3 (8.9) | 59.0 (5.5) | 50.5 (15.8) | 51.5 (10.0) | 48.8 (7.4) | 53.3 (14.1) |
| BMI (kg/m2), mean (SD) | 29.4 (2.5) | 29.8 (3.0) | 28.8 (4.0) | 30.5 (2.6) | 28.5 (4.0) | 27.5 (2.4) |
| Lipids and lipoproteins (mg/dL) | ||||||
| Apo CIII, mean (SD) | 10.4 (2.5) | 11.3 (2.3) | 8.5 (2.2) | 8.8 (3.6) | 12.4 (5.3) | 14.8 (1.7) |
| Apo CIII, median (IQR) | 10.5 (8.0–13.0) | 10.5 (9.5–13.0) | 7.5 (7.5–11.0) | 7.8 (7.0–9.5) | 11.8 (9.0–16.0) | 14.5 (14.0–16.0) |
| Triglycerides, mean (SD) | 134.7 (48.1) | 173.3 (67.3) | 127.3 (50.1) | 139.1 (87.8) | 245.4 (130.8) | 234.7 (86.6) |
| Triglycerides, median (IQR) | 118.8 (95.5–194.5) | 163.8 (144.5–234.5) | 108.8 (93.0–157.5) | 105.0 (93.5–121.0) | 192.8 (171.0–341.5) | 197.0 (173.5–318.5) |
| VLDL-C (direct), mean (SD) | 29.8 (13.3) | 35.9 (17.9) | 26.6 (5.1) | 30.9 (18.5) | 59.3 (29.2) | 55.4 (15.8) |
| Non-HDL-C, mean (SD) | 160.7 (24.2) | 173.2 (36.0) | 160.8 (31.3) | 160.4 (30.1) | 163.3 (33.7) | 198.4 (15.9) |
| Total cholesterol, mean (SD) | 213.2 (33.3) | 218.6 (43.7) | 205.6 (44.6) | 198.8 (21.9) | 203.8 (28.6) | 238.9 (22.3) |
| LDL-C (ultracentrifugation), mean (SD) | 131.0 (25.0) | 137.3 (41.5) | 134.2 (31.4) | 129.5 (25.1) | 104.0 (13.3) | 143.0 (28.3) |
| HDL-C (precipitation), mean (SD) | 52.5 (19.1) | 45.4 (12.5) | 44.8 (17.8) | 38.3 (9.1) | 40.5 (8.7) | 40.5 (11.9) |
| ApoB, mean (SD) | 106.9 (22.3) | ND | ND | ND | 99.2 (16.8) | 127.4 (12.7) |
| Lp(a) (nmol/L), mean (SD) | 17.0 (12.2) | ND | ND | ND | 34.8 (36.6) | 48.4 (46.0) |
| AKCEA-APOCIII-LRX, multiple-dose cohort | |||||
|---|---|---|---|---|---|
| Pooled placebo weekly (n = 4) | 15 mg/week (n = 6) | 30 mg/week (n = 7) | Placebo every 4 weeks (n = 4) | 60 mg/every 4 weeks (n = 6) | |
| Gender (male:female) | 2:2 | 4:2 | 7:0 | 2:2 | 3:3 |
| Age (years), mean (SD) | 53.0 (9.6) | 53.3 (8.0) | 41.9 (6.6) | 57.0 (5.0) | 49.8 (7.5) |
| BMI (kg/m2), mean (SD) | 27.5 (3.2) | 28.7 (4.7) | 30.6 (3.0) | 28.2 (3.3) | 27.7 (2.3) |
| Lipids and lipoproteins (mg/dL) | |||||
| Apo CIII, mean (SD) | 12.5 (2.8) | 14.4 (3.1) | 10.3 (3.2) | 12.8 (1.6) | 15.0 (7.2) |
| Apo CIII, median (IQR) | 11.5 (10.5–14.5) | 15.3 (11.5–17.0) | 10.0 (8.0–13.5) | 13.3 (11.8–13.8) | 12.0 (10.5–18.5) |
| Triglycerides, mean (SD) | 225.3 (96.2) | 223.2 (144.1) | 188.7 (73.3) | 190.8 (58.7) | 300.8 (232.6) |
| Triglycerides, median (IQR) | 210.5 (151.0–299.5) | 176.3 (107.5–307.5) | 155.5 (124.5–270.0) | 197.0 (140.8–240.8) | 225.3 (150.5–357.0) |
| VLDL-C (direct), mean (SD) | 45.9 (25.5) | 44.9 (20.1) | 39.3 (14.3) | 38.5 (20.2) | 53.4 (36.5) |
| Non-HDL-C, mean (SD) | 192.1 (46.9) | 172.8 (28.4) | 189.0 (33.9) | 211.1 (32.0) | 230.8 (72.6) |
| Total cholesterol, mean (SD) | 229.8 (40.5) | 225.4 (25.1) | 228.5 (33.7) | 257.1 (27.1) | 270.6 (73.3) |
| LDL-C (ultracentrifugation), mean (SD) | 146.3 (36.4) | 127.9 (21.1) | 149.7 (25.7) | 172.6 (32.7) | 177.4 (61.6) |
| HDL-C (precipitation), mean (SD) | 37.6 (10.3) | 52.6 (20.7) | 39.5 (8.5) | 46.0 (8.1) | 39.8 (17.7) |
| ApoB, mean (SD) | 118.8 (26.2) | 106.4 (20.1) | 117.2 (16.7) | 127.1 (15.3) | 144.2 (42.5) |
| Lp(a) (nmol/L), mean (SD) | 86.5 (78.3) | 32.4 (43.7) | 33.6 (23.8) | 17.8 (17.1) | 30.7 (30.4) |
ND, not determined.