A Phase 2b, Randomized, Double-blind, Placebo-Controlled Multicenter Study Evaluating Antiviral Effects, Pharmacokinetics, Safety, and Tolerability of Presatovir in Hematopoietic Cell Transplant Recipients with Respiratory Syncytial Virus (RSV) Infection of the Lower Respiratory Tract

Marty, Francisco M; Chemaly, Roy F; Mullane, Kathleen M; Lee, Dong-Gun; Hirsch, Hans H; Small, Catherine B; Bergeron, Anne; Shoham, Shmuel; Ljungman, Per; Waghmare, Alpana; Blanchard, Elodie; Kim, Yae-Jean; McKevitt, Matt; Porter, Danielle P; Jordan, Robert; Guo, Ying; German, Polina; Boeckh, Michael; Watkins, Timothy R; Chien, Jason W; Dadwal, Sanjeet S

Twenty-four presatovir-treated patients (80.0%) and 23 placebo-treated patients (79.3%) experienced ≥1 AE, whereas 7 presatovir-treated patients (23.3%) and 7 placebo-treated patients (24.1%) experienced serious AEs (SAEs). Adverse events ≥grade 3 occurred in 7 presatovir-treated patients (23.3%) and 9 placebo-treated patients (31.0%). Individual AEs occurred in ≤10% of presatovir-treated patients (Table 3) . Numerically more frequent AEs in patients treated with presatovir versus placebo were pneumonia, increased alanine aminotransferase, hypokalemia, nausea, acute sinusitis, and epistaxis (3 patients each, 10%), and increased aspartate aminotransferase, dry mouth, and increased alkaline phosphatase (2 patients each, 6.7%; Table 3 ). Except for SAE pneumonia in 3 presatovirtreated patients (10%), grade 3 or 4 AEs and SAEs occurred in 1 patient each and were numerically less frequent overall in patients treated with presatovir versus placebo (Supplemental Tables 11-12 ). There were no significant imbalances in electrocardiogram and troponin results during the study. No patients treated with presatovir and 2 patients treated with placebo (6.9%) died during the 28-day study period; 1 death was due to respiratory failure and 1 to progressive acute leukemia. Another 2 patients (6.9%) who received placebo died after day 28, 1 of respiratory failure, and 1 of invasive fusariosis.