Clinical programs

BIT225 is in mid‑stage clinical development for treatment of HIV-1 and Hepatitis C virus (HCV) infections, with seven clinical trials completed. BIT225 has been dosed in 55 healthy human volunteers and 94 subjects infected with either HIV-1, HCV or co-infected with HIV-1/HCV.

Clinical trial results for BIT225 include:


  • BIT225 is a novel first-in-class HIV-1 Vpu protein inhibitor with the aim of inhibiting virus replication in cellular reservoirs.
  • In vitro studies have shown BIT225
    • Significantly inhibits HIV-1 release in a dose dependent manner from CD14+ and CD16+ monocyte subsets at various stages of differentiation and monocyte-derived dendritic cells (MDDC)
    • Significantly reduces cell-cell transmission of virus from HIV-1 infected myeloid lineage cells to CD4+ T cell targets
    • Has broad spectrum activity against clinical isolates from different virus clades and selected drug resistant strains
    • Has additive or synergistic inhibition of virus replication in combination with marketed HIV-1 anti-retroviral drugs from different classes
  • Studies in the clinic have demonstrated
    • BIT225 treated isolated cells from HIV-1 infected patients show reduced HIV-1 release from CD14+ monocytes
    • Following 10 days of monotherapy in HIV-1 infected patients, BIT225 can reduce viral copy numbers and inhibit replication in myeloid lineage cells (including dendritic cells)
    • BIT225 may impact on macrophage associated immune activation
    • BIT225 is able to cross the blood-brain barrier, with potential to treat HIV-associated neurocognitive disorder (HAND)


  • BIT225 is a novel first-in-class viral assembly inhibitor that targets HCV p7 protein
  • In vitro studies have shown
    • Pan genotypic activity in HCV infectious clone assay
    • Synergy with marketed drugs for the treatment of HCV
    • High barrier to antiviral resistance
  • Studies in the clinic have demonstrated
    • Clinical antiviral efficacy against HCV genotypes 1 and 3
    • BIT225 treatment is associated with faster viral clearance in treated HCV patients
    • Substantial safety, tolerability and pharmacokinetic data in both Phase 1 and 2 clinical trials of up to 3 months dosing duration


Completed Human Clinical Trials: