Chimerix

CMX001

CMX001 combines Chimerix’s PIM (Phospholipid Intramembrane Microfluidization) Conjugate Technology with cidofovir, an approved antiviral agent, with the aim of creating a well-tolerated and highly potent new chemical entity with broad-spectrum antiviral activity.  We believe that if we can successfully develop a product with increased efficacy and reduced toxicities we can provide a powerful new option for patients and their physicians that may dramatically change antiviral treatment, particularly among immunocompromised patients.  In clinical testing to date, CMX001 has shown oral bioavailability in humans and has demonstrated a positive safety profile.

CMX001 is being developed for both commercial and medical preparedness uses. We are currently conducting clinical trials of CMX001 for the treatment of cytomegalovirus (CMV) and BK virus in immunocompromised transplant patients. 

We have received a $36.1 million grant from the National Institute of Allergy and Infectious Diseases (NIAID) of the National Institutes of Health (NIH) to support the development of CMX001 as a biodefense countermeasure in the event of a smallpox release. 

CMX001 is a mimic of a naturally occurring lipid, lysolecithin, formed by linking a lipid, 3-hexadecyloxy-1-propanol, to the phosphonate group of cidofovir. CMX001 is designed to readily cross the intestinal wall and penetrate target cells before being cleaved to free the antiviral, cidofovir.  Cidofovir is phosphorylated by host cell nucleoside kinases to form the active antiviral agent cidofovir diphosphate.

Improved Potency Demonstrated in Preclinical Studies

The antiviral activity of CMX001 has been characterized in both in vitro and in vivo studies.  In cell culture assays, CMX001 is significantly more active than cidofovir against double-stranded DNA viruses including orthopoxviruses (variola, monkeypox, vaccinia, cowpox, and ectromelia), herpes viruses (CMV, herpes simplex virus (HSV)-1,-2,-6, -8, HSV-2, varicella zoster virus (VZV), Epstein-Barr virus (EBV), and multiple adenoviruses.  Against variola major, CMX001 is approximately 250 fold more potent than cidofovir in inhibiting viral DNA replication.

Clinical development for CMV, BK virus and Smallpox

A Phase 1 clinical study of orally-administered CMX001 in 84 healthy volunteers is complete.  The blinded, randomized, parallel-group, placebo-controlled study evaluated the safety and pharmacokinetics of single and multiple doses of CMX001.  CMX001 was well tolerated at all doses.  CMX001 was well absorbed after oral dosing, with dose-dependent pharmacokinetics.  A second study to demonstrate the bioequivalence of CMX001 solution and tablet dosage forms is also complete.  

Currently, we are conducting a Phase 1 clinical trial of multi-dose CMX001 in transplant recipients with BK viruria.  We are also initiating a Phase 2 clinical trial of CMX001 in immunocompromised patients with CMV.

We are actively pursuing development of CMX001 for the prevention and treatment of smallpox under the “Animal Efficacy Rule,” which allows the U.S. Food and Drug Administration to rely on efficacy data from animal models of human disease in the development and approval of a compound.