Verdinexor (KPT-335) is a novel, oral Selective Inhibitor of Nuclear Export (SINE) compound being evaluated for the treatment of canine cancers, including lymphoma, as well as in humans, for a number of different non-cancer conditions. In humans, verdinexor is being evaluated in preclinical studies in a variety of viral indications as well as in autoimmune / inflammatory diseases.
Verdinexor inhibits the nuclear export function of Exportin-1 (XPO1 or CRM1).
Verdinexor is an investigational medicine and has not been approved by The United States Food and Drug Administration (FDA), Health Canada, the European Medicines Agency (EMA), nor any other regulatory agency.
Verdinexor as an Anti-cancer Therapy in Dogs
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.
The U.S. Food and Drug Administration (FDA) Center for Veterinary Medicine (CVM) has found the effectiveness and safety technical sections for verdinexor complete to support conditional approval under a New Animal Drug Application (NADA) for the treatment of canine lymphoma. The use of verdinexor to treat canine lymphoma has been designated a “minor use” in accordance with the Minor Use Minor Species (MUMS) Act. This makes the product eligible for conditional approval similar to orphan drug/accelerated approvals used for submissions of human therapeutics.
In May 2017, Karyopharm announced a collaboration agreement with Anivive Lifesciences, a privately-held biotech company focused on innovations in the veterinary drug and bioinformatics space, whereby Anivive licensed from Karyopharm exclusive worldwide rights to research, develop and commercialize verdinexor (KPT-335) for the treatment of cancer in companion animals.
Verdinexor as an Antiviral Agent
Verdinexor is also Karyopharm’s 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. Preclinical data has shown efficacy of verdinexor and related SINE compounds in a number of viral models, including influenza and HIV.