P2/3, N=61, Terminated, RedHill Biopharma Limited | N=310 --> 61 | Suspended --> Terminated; Part A complete; corporate decision made to cancel Part B.
Mouse weights and PAX165 tumor volumes were measured. Tumor volumes in the upamostat, opaganib, and upamostat plus opaganib groups were significantly decreased compared to the control group.
Upamostat and its active metabolite WX-UK1 have been shown to inhibit serine proteases. Upamostat and opaganib in combination have significant inhibitory effects on both CCA cell migration and viability in vitro which may prevent tumor progression. Both drugs have an established clinical safety profile and may be effective in targeted treatment of CCA patients.