TABLE 2.
Overall summary of adverse events
| Safety outcomes, n (%) | Berotralstat 110 mg (n = 100) | Berotralstat 150 mg (n = 127) | Total (N = 227) |
|---|---|---|---|
| Any AE | 91 (91) | 115 (91) | 206 (91) |
| Most common AEs with medical concepts (>10% of patients in any treatment group) a | |||
| Upper respiratory tract infection | 34 (34) | 57 (45) | 91 (40) |
| Abdominal pain | 31 (31) | 26 (21) | 57 (25) |
| Headache | 21 (21) | 19 (15) | 40 (18) |
| Diarrhea | 13 (13) | 18 (14) | 31 (14) |
| Drug‐related AE | 56 (56) | 57 (45) | 113 (50) |
| Grade 3 or 4 AE | 14 (14) | 20 (16) | 34 (15) |
| Drug‐related grade 3 or 4 AE b | 6 (6) | 8 (6) | 14 (6) |
| Investigator‐identified drug‐related rash (EOSI) c | 2 (2) | 0 (0) | 2 (1) |
| SAE | 18 (18) | 12 (9) | 30 (13) |
| Drug‐related SAE | 2 (2) | 1 (1) | 3 (1) |
Abbreviations: AE, adverse event; EOSI, event of special interest; SAE, serious adverse event.
Adverse events are coded using MedDRA version 19.1. The terms “abdominal pain,” “diarrhea,” and “upper respiratory tract infection” are medical concepts containing multiple preferred terms. “Abdominal pain” contains the preferred terms “abdominal pain,” “abdominal discomfort,” “abdominal pain upper,” “abdominal pain lower,” and “epigastric discomfort.” “diarrhea” contains the preferred terms, “diarrhea” and “feces soft.” “upper respiratory tract infection” contains the preferred terms “nasopharyngitis,” “upper respiratory tract infection,” “viral upper respiratory tract infection,” “respiratory tract infection,” and “respiratory tract infection viral.”
Berotralstat 110 mg (n = number of patients): Alanine aminotransferase increased (n = 3), hepatic enzyme increased and gastroenteritis (n = 1), liver function test abnormal (n = 1), abdominal pain (n = 1). Berotralstat 150 mg (n = number of patients): Alanine aminotransferase increased (n = 2), four events of increased alanine aminotransferase (n = 1), hepatic enzyme increased (n = 1), liver function test abnormal (n = 1), transaminases increased (n = 1), arthralgia and myalgia (n = 1), dizziness and headache (n = 1). Patients may have experienced the same coded event more than once.
Diffuse maculopapular rash assessed as at least possibly related to berotralstat treatment.