Comprehensive assessment of amino acid substitutions in the trimeric RNA
polymerase complex of influenza virus detected in clinical trials of
baloxavir marboxil
Abstract
Background: Baloxavir marboxil (BXM) is an approved drug that
selectively targets cap-dependent endonuclease on PA subunit in the RNA
polymerase complex of influenza A and B viruses. Amino acid
substitutions at position 38 in the PA subunit were identified as a
major pathway for reduced susceptibility to baloxavir acid (BXA), the
active form of BXM. Additionally, substitutions found at positions E23,
A37, and E199 in the PA subunit impact BXA susceptibility by less than
10-fold. Methods: We comprehensively evaluated the impact of novel amino
acid substitutions identified in PA, PB1 and PB2 subunits in BXM
clinical trials and influenza sequence databases by means of drug
susceptibility and replicative capacity. Results: PA/I38N in A(H1N1) and
PA/I38R in A(H3N2) were newly identified as treatment-emergent
substitutions in the CAPSTONE-2 study. The I38N substitution conferred
reduced susceptibility by 24-fold, whereas replicative capacity of the
I38N-substituted virus was impaired compared to the wild-type. The
I38R-substituted virus was not viable in cell culture. All other
mutations assessed in this extensive study did not significantly affect
BXA susceptibility (< 2.4-fold change). Conclusion: These
results provide additional information on the impact of amino acid
substitutions in the trimeric viral polymerase complex to BXA
susceptibility and will further support influenza surveillance.