Trial Launched to Assess Safety and Efficacy of Novel Drug in Rare Type of Head and Neck Cancer

A phase 2 trial was recently launched to examine the safety and efficacy of oral inhibitor VK-2019 in patients with advanced Epstein-Barr Virus-positive nasopharyngeal carcinoma, a rare type of head and neck cancer, and lymphoma.

Earlier this month, The Wistar Institute announced a phase 2 clinical trial of VK-2019, an oral small molecule inhibitor, for patients with advanced Epstein-Barr Virus (EBV)-positive nasopharyngeal carcinoma (NPC), a rare form of head and neck cancer, and lymphoma.

The trial is being led by researchers at Stanford University School of Medicine who plan to enroll 30 patients with recurring disease or disease that has progressed/not responded following standard therapy.

The researchers’ goals are to assess the safety and efficacy of VK-2019. The launch of the phase 2 portion of the trial is supported by prior data from the phase 1 study in patients with advanced NPC, which showed promising results.

The current first-line treatment for NPC after disease recurrence or metastasis is platinum-based chemotherapy, which typically is associated with a duration of response of less than six months.

Patients with this type of cancer have tumor cells that are infected with EBV. VK-2019 works by inhibiting the viral protein Epstein-Barr Nuclear Antigen-1 (EBNA1), which is what regulates EBV replication and maintenance.

EBV is a member of the herpes virus family and can increase the risk of certain cancer types. It has been associated with Hodgkin lymphoma, Burkitt lymphoma and NPC.

“EBNA1 is expressed consistently in all EBV-related cancer and is essential for the cancer to grow," said Paul M. Lieberman — who worked to discover and develop VK-2019 with a team for nearly a decade — in a news release. “By targeting EBNA1, which has a unique protein fold and is present only in the cancer cells, we have so far observed fewer side effects than we typically see with other cancer drugs.”

VK-2019 inhibits the form of EBV that drives the spread of infected cells. Lieberman and his colleagues developed it at The Wistar Institute.

Lieberman, who is the Hilary Koprowski, M.D., Endowed Professor and program leader, Gene Expression & Regulation Program, at The Wistar Institute, also explained that once researchers can determine VK-2019’s safety and efficacy in patients with NPC, then it may be possible to apply it for the treatment of other EBV cancers such as EBV-positive gastric carcinoma and lymphomas.

The trial received funding through a grant from the National Cancer Institute (NCI). Its planned completion date is February 2026.

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