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Wistar and Stanford Medicine to Begin Phase 2 Clinical Trial of VK-2019 in Patients with Epstein-Barr Virus (EBV)-Positive Advanced Nasopharyngeal Carcinoma

PHILADELPHIA — (Jan. 5, 2022) — The Wistar Institute announces the initiation of a Phase 2 clinical study of VK-2019 in patients with advanced Epstein-Barr Virus (EBV)-positive nasopharyngeal carcinoma (NPC) and lymphoma. The study, led by researchers at the Stanford University School of Medicine, will enroll patients who have recurred or progressed following standard therapy or have not responded to prior therapy.

The open-label, single-group Phase 2 clinical trial will assess the safety and efficacy of VK-2019. Enrollment is expected to begin later this year. The Phase 2 trial is supported by interim results from the Phase 1 study in advanced NPC patients that showed favorable safety, tolerability, pharmacokinetic (PK) and biomarker results as well as preclinical data that underscore the promising potential for the treatment of EBV-positive cancers.

The principal investigator of the study is A. Dimitrios Colevas, M.D., professor of Medicine at Stanford Cancer Institute.

NPC, a type of head and neck cancer that develops in the nasopharynx, is a serious global health problem, particularly prevalent in East and Southeast Asia. More than 90,000 people are diagnosed with NPC worldwide each year. Platinum-based chemotherapy is currently the first-line treatment after recurrence or metastasis. However, the duration of response is typically less than six months. One unique aspect of this type of cancer is that NPC tumor cells are infected with EBV.

VK-2019 is an oral, small molecule inhibitor of the latent form of EBV that drives the proliferation of infected cells. VK-2019 was invented by researchers at The Wistar Institute, an international biomedical research leader in cancer, immunology, infectious diseases, and vaccine development. According to Paul M. Lieberman, Ph.D., Hilary Koprowski, M.D., Endowed Professor and program leader, Gene Expression & Regulation Program, at The Wistar Institute, VK-2019 inhibits the viral protein Epstein-Barr Nuclear Antigen-1 (EBNA1), known as the master regulator of EBV replication and maintenance.

“EBNA1 is expressed consistently in all EBV-related cancer and is essential for the cancer to grow,” said Lieberman. “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.”

Lieberman heads Wistar’s Center for Chemical Biology and Translational Medicine, comprised of a team of researchers developing promising basic research findings into new therapeutics to fight disease, particularly cancer. He and the team worked for almost a decade to discover and develop VK-2019.

He added, “The good news is that if we can demonstrate that VK-2019 is safe and effective in patients with nasopharyngeal carcinoma, then it may be safe and effective for other EBV cancers including EBV-positive gastric carcinoma and lymphomas.”

“We are excited about the potential of VK-2019 in treating nasopharyngeal cancer,” said Troy Messick, Ph.D., senior staff scientist in the Lieberman lab. “VK-2019 is unique because it targets the underlying driver of growth of this head and neck cancer, namely EBV.”

Funding for this Phase 2 clinical trial is provided by a grant from the National Cancer Institute.


The Wistar Institute is an international leader in biomedical research with special expertise in cancer research and vaccine development. Founded in 1892 as the first independent nonprofit biomedical research institute in the United States, Wistar has held the prestigious Cancer Center designation from the National Cancer Institute since 1972. The Institute works actively to ensure that research advances move from the laboratory to the clinic as quickly as possible.

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