A Multicenter, Open-label Study of CMX001 Treatment of Serious Diseases or Conditions Caused by dsDNA Viruses

CMX001 is an orally administered lipid conjugate of the synthetic nucleotide analog
cidofovir (CDV). The conjugate is believed to be absorbed in the small intestine then
delivered to target organs throughout the body where it crosses cell membranes by
facilitated and passive diffusion. Inside the cell, CMX001 is cleaved by intracellular
phospholipases to release CDV which is converted to the active antiviral agent,
CDV-diphosphate (CDV-PP), by intracellular anabolic kinases. CMX001 will be administered to
adults in tablet or oral solution formulation at doses of 200mg, or 300mg for patients with
CNS involvement, disseminated ADV infection, or known resistance to CDV, not to exceed
4mg/kg. For pediatric patients, CMX001 will be administered in tablet or oral solution
formulation at 4mg/kg.