Table 2.
Main safety and tolerability findings for each ETC‐206 dose level, administration conditions, and Pbo assignment
| Parameters | ETC‐206 10 mg FASTED (N = 16) | ETC‐206 20 mg FASTED (N = 14) | ETC‐206 10 mg FED (N = 9) | Pbo [entire study] (N = 8) |
|---|---|---|---|---|
| SAEs: seizure (%) | 1 (6)a | 0 | 0 | 0 |
| AEs grade 3 CTCAE: ↑CK,a % | 0 | 1 (7)b | 0 | 0 |
| AEs grade 1–2 CTCAE, %c , h | 9 (56) | 11 (79) | 9 (100) | 5 (62) |
| Gastrointestinal disorders,d% | 4 (25) | 3 (21) | 2 (22) | 0 |
| Diarrhea,e % | 3 (19) | 3 (21)g | 2 (22)f | 0 |
| Investigations, %i | 5 (31) | 1 (7) | 1 (11) | 0 |
| ↓WBC (%) | 3 (19) | 0 | 1 (11) | 0 |
| ↑ALT (%) | 1 (6) | 0 | 0 | 0 |
| ↑CK (%) | 1 (6) | 0 | 0 | 0 |
AEs, adverse events; ALT, alanine aminotransferase; CK, creatine kinase; CTCAE, Common Terminology Criteria for Adverse Events; Pbo, placebo; SAE, serious adverse events; WBC, white blood cell.
The panel shows that one SAEa not drug‐related (seizure) was reported in one subject with ETC‐206 at 10 mg dose level. One grade 3 AEb not drug‐related (CK increase) was reported in one subject with ETC‐206 at 20 mg dose level. AEs, grade 1–2,c were reported across all doses and conditions with the exception of gastrointestinal disordersd that were not reported with subjects receiving placebo (Pbo). Diarrheae (grade 1–2) was reported in 19%, 21%, and 22% of subjects receiving ETC‐206 at the dose of 10 and 20 mg in fasted condition and 10 mg in fed condition, respectively, but not in subjects receiving Pbo. About 50% of diarrhea cases were reported as at least possibly drug‐related (as per clinical judgment of the treating physician), with one grade 2 case at the dose of 10 mg in fed condition definitely relatedf to ETC‐206 administration, and with another grade 2 case at the dose of 20 mg in fasted condition, not relatedg. A decrease of white blood cells was observed in 19% of subjects at the dose of 10 mg in fasted condition and in 11% of subjects at the dose of 10 mg in fed condition. ALT and CK increases were observed, each in 6% of subjects, at the dose of 10 mg in fasted condition. Not‐clinically‐significant, out‐of‐the‐reference‐range laboratory values were reported with all doses and conditions except with Pbo (not shown). hAEs observed in >15% of subjects (and not related to local procedures) are reported; iinvestigation alterations observed in >5% of subjects are reported.