Table 1.
Efficacy and Safety Outcomes
| Efficacy | Sonidegib | Placebo |
|---|---|---|
| n = 7a | n = 2 | |
| Patients with clinical clearance of target BCC at week 16b | ||
| Complete (100% improvement) | 3 (43) | 0 |
| Marked (76%–99% improvement) | 3 (43) | 0 |
| Moderate (26%–75% improvement) | 1 (14) | 0 |
| Slight (1%–25% improvement) | 0 | 1 (50) |
| Worsening | 0 | 1 (50) |
| Patients with histological clearance of target BCC at week 16 | 4 (57) | 0 |
| Number of total BCCs | ||
| Baseline | 566 | 510 |
| Week 12 | 341 | 571 |
| Week 16 | 309 | 619 |
| Safety | n = 8 | n = 2 |
| Any AEs (through long-term follow-up period) | 7 (88) | 1 (50) |
| Mild | 3 (38) | 0 |
| Moderate | 3 (38) | 0 |
| Severe | 1 (13) | 1 (50) |
| Related to treatment | 6 (75) | 1 (50) |
| Leading to discontinuation | 0 | 1 (50) |
| Serious | 1 (13) | 1 (50) |
| AEs in ≥20% of patients (core study period) | ||
| Muscle spasms | 3 (38) | 1 (50) |
| Alopecia | 2 (25) | 0 |
| CK increased | 2 (25) | 0 |
| Fatigue | 2 (25) | 0 |
| Headache | 2 (25) | 0 |
| Nasopharyngitis | 2 (25) | 0 |
| Nausea | 2 (25) | 0 |
Notes: Data presented as n (%) of total patients in treatment arm unless otherwise indicated. aOne patient was excluded from efficacy analysis due to receipt of placebo in 5 of 13 doses. bOne target BCC was counted per patient and assigned to 1 of 5 listed clearance categories.
Abbreviations: AE, adverse event; BCC, basal cell carcinoma; CK, creatine kinase.