CMX001: Phase II data

The double-blind, dose-escalation, U.S. Phase II Study 201 trial in 230 allogeneic HSCT recipients showed that twice-weekly 100 mg CMX001 met the primary endpoint of significantly reducing the proportion of patients with CMV viremia >200 copies/mL or with CMV disease at 13 weeks post-transplant vs. placebo (10% vs. 37%, p=0.001). The once-weekly 40, 100 and 200 mg and twice-weekly 200 mg doses of CMX001 missed the endpoint (52%, 22%, 31% and 23%, respectively; p=0.34, p=0.19, p=0.31 and p=0.09). Additionally, the once-weekly 200 mg and twice-weekly 100 and 200 mg doses of CMX001 significantly reduced the proportion of patients with CMV viremia >=1,000 copies/mL at any time during treatment vs. placebo (18%, 8% and 7%, respectively, vs. 42%; p=0.002, p<0.001 and p<0.001).

On secondary endpoints, several dose regimens of CMX001 significantly reduced the proportion of patients who had CMV viremia >=1,000 copies/mL at any time during treatment vs. placebo in the subgroups of patients who were CMV-negative and CMV-positive at baseline, respectively. Specifically, in CMV-negative patients at baseline (n=181), 9%, 7%, 0% and 0% of patients receiving once-weekly 100 and 200 mg and twice-weekly 100 and 200 mg doses of CMX001 achieved the endpoint vs. 31% for placebo (p<=0.04 for all). In CMV-positive patients at baseline (n=49), 44% and 25% of patients receiving twice-weekly 100 and 200 mg doses of CMX001 achieved the endpoint vs. 91% for placebo (p=0.05 and p=0.006, respectively). ...