Highlights from the Data Presentation

  • Initial immunogenicity (n=9) and ongoing safety data (n=13) presented in chronically HBV-infected patients following a single, low dose, prime-only intramuscular injection of VRON-0200
  • VRON-0200 was well tolerated, with no serious adverse events (SAEs), study patient discontinuations, or laboratory abnormalities observed in 870 patient days on treatment
  • VRON-0200 induced CD8+ T cell responses in the majority (5 of 9) of patients
    • Responses were observed even in patients with limited pre-existing immunity
    • A 4-fold increase in T cell responses at Day 28 was observed in the 5 responders
    • At Day 91, several patients maintained T cell responses above their pre-treatment values

PHILADELPHIA, PA, USA I June 5, 2024 I Virion Therapeutics, LLC, a clinical-stage biotechnology company, developing novel T cell-based immunotherapies, today announced positive immunologic and continued safety and tolerability data, from the first-ever human study of its novel, first-in-class, checkpoint modifier immunotherapy, VRON-0200, for HBV functional cure, presented by Professor Ed Gane, M.D., from the University of Auckland, as a late breaker at the EASL Congress 2024 (The European Association for the Study of the Liver) in Milan, Italy. 

These Phase 1b safety data, on the first 13 chronically infected hepatitis B patients, on nucleos(t)ide antiviral therapy, all of whom received a single, low dose, intramuscular injection of VRON-0200, demonstrated that it was safe and well-tolerated, with no significant adverse events reported and no clinically relevant abnormalities in laboratory assessments, including liver function tests.  In 9 patients who had at least 28 days of immunologic follow up, the majority (n=5) had increased T cell responses, post vaccination, with several maintaining responses above their baseline values out to day 91. 

Professor Gane commented: “Patients with chronic HBV infection suffer from immune exhaustion. To date, immunomodulatory therapies, including therapeutic vaccination, have failed to stimulate HBV-specific T- and B-cell responses. These immunologic data are exciting because a single intramuscular injection of VRON-0200 was able to induce T cell responses in the majority of patients, most of whom had little, to no, documented immunity prior to vaccination. Being able to stimulate T cells in this patient population, with a good safety profile and ease of administration, are all critical components for potential therapies for HBV functional cure.  I look forward to seeing additional data from this ongoing study.”

“Immune-based treatments are now considered necessary for functional cure of Chronic HBV. These VRON-0200 data – the first-ever for a checkpoint modifier containing T cell vaccine of any kind – showed that VRON-0200 can induce immune responses in chronically HBV-infected patients, even after a single low dose vaccination.  Building upon these positive safety and immunogenicity data, we are now exploring ways to further evaluate VRON-0200, alone and in combination with other investigational agents, which we will be announcing very soon,” said Dr. Sue Currie, COO of Virion, and one of the study authors. “These data represent a critical first step in Virion’s mission to bring a safe, well tolerated, easy to administer, and immunogenic interferon-sparing functional cure immunotherapy to the almost 300 million patients living with chronic HBV.  We look forward to sharing more data later this year.”

Summary of VRON-0200 Phase 1b clinical trial design

  • VRON-0200 is a Phase 1b, multi-center, open-label, dose escalation, prime only, and prime plus boost, therapeutic vaccination study to evaluate the safety, tolerability, immunology, and other clinical measures:
    • Inclusion criteria: Non-cirrhotic, HBeAg positive or negative, chronic hepatitis B patients currently taking nucleos(t)ide antiviral therapy with HBV DNA < 40 IU/mL and HBsAg < 500 IU/mL
    • Dose escalation: Cohort 1 (low dose); Cohort 2 (high dose) of VRON-0200
    • Prime or Prime-Boost: In each cohort, patients are randomized to receive either a prime vaccination only or a prime and boost regimen
    • Cohort 3: VRON-0200 plus several investigational agents scheduled to begin later this year

More details of the study can be found at ClinicalTrials.gov (Identifier: NCT06070051).

About Chronic Hepatitis B
Despite a preventative vaccine, cases of chronic hepatitis B (HBV) continue to rise, with an estimated 296 million persons infected worldwide and 820,000 deaths per year from HBV-related liver complications. Chronic HBV remains a global health issue with a high unmet medical need since there is no cure available. The current standard of care requires lifelong antiviral therapy to maintain control of the virus.

About VRON-0200
VRON-0200 is a therapeutic immunotherapy, administered by intramuscular injection, designed with the goal of providing a functional cure for chronic HBV infection. While the virus itself stimulates HBV-specific CD8+ T cells, for those patients that can’t clear the initial infection, their T cells soon become exhausted, placing limits on their ability to proliferate and control the virus. Preclinical data support the hypothesis that VRON-0200, through checkpoint modification, can amplify, broaden, and enhance T cell responses that may include T cells that are not normally activated during a chronic HBV infection, which results in improved viral control.

About Virion Therapeutics (Virion)
Virion Therapeutics, LLC is a clinical-stage company developing novel T cell-based immunotherapies to cure cancer and chronic infectious diseases that utilize proprietary genetically encoded checkpoint modifiers to enhance and broaden CD8+ T cell responses to a tumor or chronic infection. Founded in early 2018 to advance technology licensed from The Wistar Institute, an international leader in biomedical research, Virion has since developed a robust pipeline, including its lead VRON-0200 clinical program, and several additional IND-enabling programs, including its VRON-0300 oncology program for advanced solid tumors, leveraging its proprietary platform technologies. In early Fall 2023, Virion and Ocean Biomedical entered a joint venture to accelerate and expand Virion’s pipeline of novel immunotherapies.

To learn more, visit www.VirionTx.com

SOURCE: Virion Therapeutics