On November 29, 2021 Viracta Therapeutics, Inc. (Nasdaq: VIRX), a precision oncology company targeting virus-associated malignancies, reported that the U.S. Food and Drug Administration (FDA) has granted its all-oral combination product candidate, nanatinostat and valganciclovir (Nana-val), orphan drug designation (ODD) for the treatment of Epstein Barr virus-positive diffuse large B-cell lymphoma, not otherwise specified (EBV+ DLBCL, NOS) (Press release, Viracta Therapeutics, NOV 29, 2021, View Source [SID1234596184]). DLBCL is the most common subtype of non-Hodgkin lymphoma (NHL) in the U.S. and worldwide, accounting for approximately 25% of newly diagnosed cases of NHL in the U.S, of which a subset are EBV+. Viracta has previously received ODD from the FDA for the treatment of T-cell lymphoma, post-transplant lymphoproliferative disorder (PTLD) and plasmablastic lymphoma.
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"This latest orphan drug designation underscores the potential benefits of our all-oral kick and kill approach to targeting EBV-positive cancers," said Ivor Royston, M.D., President and Chief Executive Officer of Viracta. "Nana-val has shown promising preliminary efficacy across multiple subtypes of relapsed/refractory EBV-positive lymphoma, including DLBCL. We are dosing patients in the pivotal NAVAL-1 trial, which includes patients with EBV-positive DLBCL, and look forward to its continued momentum with sites now open for enrollment in the U.S., Europe, and Asia. Through NAVAL-1’s progress, we aim to further develop Nana-val as a new and actionable therapy in indications where many patients recur from the standard of care and have particularly poor prognoses."
The FDA grants orphan drug designations to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S. Orphan drug status is intended to facilitate drug development for rare diseases and may provide several benefits to drug developers, including tax credits toward qualified clinical trial costs, assistance with clinical study design and drug development, exemptions from certain FDA application fees, and seven years of market exclusivity upon regulatory approval for the disease or condition for which the drug has the orphan drug designation. In 2020, 31 of the 53 novel drug approvals, or 58%, in the FDA’s Center for Drug Evaluation and Research, were orphan designated products.
About NAVAL-1
NAVAL-1 (Nanatinostat in Combination with Valganciclovir) is a global, multicenter, open-label Phase 2 basket trial. The trial, which will include patients with multiple subtypes of relapsed/refractory EBV-positive (EBV+) lymphoma, is designed to evaluate the anti-tumor activity of Nana-val and enroll approximately 140 patients. The primary endpoint of the trial is objective tumor response rate as assessed by an independent review committee. If successful, Viracta believes this trial could potentially support multiple new drug application filings across various EBV+ lymphoma subtypes. The study employs a Simon two-stage design where a limited number of patients are enrolled into each cohort in Stage 1 and, if a pre-specified activity threshold is reached, additional patients will be enrolled in Stage 2. During Stage 2, Viracta anticipates discussing the preliminary results with the FDA and may amend the protocol to include additional patients as necessary to enable registration.
About Nanatinostat
Nanatinostat (VRx-3996) is an orally available histone deacetylase (HDAC) inhibitor being developed by Viracta. Nanatinostat is selective for specific isoforms of Class I HDACs, which is key to inducing viral genes that are epigenetically silenced in EBV-associated malignancies. Nana-val (nanatinostat and valganciclovir) is being investigated in multiple subtypes of relapsed/refractory EBV+ lymphoma and in advanced EBV+ solid tumors in three ongoing trials, one of which is a registration-enabling global, multicenter, open-label Phase 2 basket trial in relapsed/refractory EBV+ lymphoma (NAVAL-1).
About EBV-Associated Cancers
Approximately 95% of the world’s adult population is infected with Epstein-Barr Virus (EBV). Infections are commonly asymptomatic or associated with mononucleosis. Following infection, the virus remains latent in a small subset of lymphatic cells for the duration of the patients’ life. Cells containing latent virus are increasingly susceptible to malignant transformation. Patients who are immunocompromised are at an increased risk of developing EBV+ lymphomas. In addition, EBV is also associated with a variety of solid tumors, including nasopharyngeal carcinoma and gastric cancer.