Table 2.
Safety and tolerability.
| Safety overview | Placebo (n = 10) | Efruxifermin (n = 20) |
|---|---|---|
| Study discontinuations | 1a | 1b |
| Deaths | 0 | 0 |
| Any treatment-emergent adverse events, n (%) | 8 (80) | 19 (95) |
| Treatment-emergent adverse events, n (%) | ||
| Life-threatening | 1 (10) | 0 |
| Severe | 0 | 0 |
| Moderate | 5 (50) | 14 (70) |
| Mild | 2 (20) | 5 (25) |
| Study procedure related | 1 (10) | 7 (35) |
| Treatment-emergent events leading to discontinuation, n (%) | 0 | 1 (5) |
| Drug-related treatment-emergent adverse events, n (%) | 3 (30) | 13 (65) |
| Serious adverse events | 1c | 0 |
| Treatment-emergent events occurring in ≥15% of patients in any group, n (%) | ||
| Gastrointestinal disorders | 6 (60) | 14 (70) |
| Diarrhea | 1 (10) | 10 (50) |
| Nausea | 2 (10) | 9 (45) |
| Vomiting | 0 | 4 (20) |
| Abdominal pain | 2 (20) | 3 (15) |
| Constipation | 0 | 4 (20) |
| Gastroesophageal reflux disease | 2 (20) | 0 |
| Nervous system disorders | 4 (40) | 8 (40) |
| Headache | 1 (10) | 4 (20) |
| General disorders and administration site conditions | 1 (10) | 10 (50) |
| Injection site reaction | 0 | 6 (30) |
| Injection site bruising | 1 (10) | 4 (20) |
| Injection site erythema | 0 | 5 (25) |
| Infections and infestations | 2 (20) | 7 (35) |
| Sinusitis | 0 | 4 (20) |
| Skin and subcutaneous tissue disorders | 0 | 9 (45) |
| Pruritus | 0 | 3 (15) |
a
Withdrawal of consent.
b
Abdominal distention, constipation, diarrhea, pruritus.
c
Pulmonary embolism.