We began clinical testing of verdinexor in May 2015 in healthy human volunteers and we are preparing to advance verdinexor for certain viral indications with an initial focus on influenza. Preclinical data provide strong support for other potential indications for verdinexor, including HIV, RSV, HCV, and VEEV.
Verdinexor as an Antiviral Agent
Verdinexor is our lead compound in development for the treatment of viral indications. Several viruses exclusively utilize XPO1 to shuttle cargos necessary for virion assembly such as viral ribonucleoproteins, or vRNA, and proteins from the nucleus to the cytoplasm. Verdinexor has the potential to treat viral diseases through both inhibition of viral replication and suppression of inflammatory cytokine-mediated symptoms and shows significant anti-influenza activity in murine and ferret models.
In 2015, we conducted a randomized, double-blind, placebo-controlled, dose-escalating Phase 1 clinical trial of verdinexor in healthy human volunteers in Australia. This study was designed to evaluate the safety and tolerability of verdinexor in healthy adult subjects. Verdinexor was found to be generally safe and well tolerated. Mild-to-moderate adverse events of similar number and grade as placebo were reported, but no serious or severe adverse events. We plan to continue the clinical development of verdinexor as a treatment for influenza. Preclinical data also show efficacy of verdinexor and related SINE compounds in additional viral models, including HIV.
Verdinexor as an Anti-cancer Therapy in Dogs
Verdinexor is an oral SINE compound that is closely-related to selinexor and is currently being evaluated for the treatment of lymphomas and other cancers in companion animals in partnership with Anivive Lifesciences.
It is widely known that canine lymphomas respond to chemotherapy similarly to their human counterpart (human NHL) and display a comparable genetic profile. Lymphomas are one of the most common tumors in pet dogs. Lymphoma in dogs is very aggressive and, without treatment, the tumors are often fatal within weeks. The majority of dog lymphomas are DLBCL and most of the others are T-cell lymphomas.
We have conducted a Phase 2b clinical trial of verdinexor in dogs with newly-diagnosed or first time relapse lymphomas. Verdinexor has received a Minor Use / Minor Species, or MUMS, designation from the center for Veterinary Medicine (CMV) of the FDA for the treatment of lymphomas in canines.