Promising First-In-Human Phase 1B Clinical Study Data from VRON-0200, a Novel, First-in-Class Checkpoint Modifier Immunotherapy for Chronic Hepatitis B Virus Functional Cure, Presented as Late-Breaker at 2024 APASL Global Liver Meeting

Highlights from the Data Presentation

• Initial safety data presented on the first 10 chronically HBV-infected patients representing 674 patient days of treatment
• VRON-0200 was well tolerated, with no serious adverse events (SAEs) observed:
  • no patients discontinued treatment
  • one treatment related adverse event was reported (flu-like symptoms which resolved without treatment)
• No other clinically relevant abnormalities in laboratory tests, including liver function tests, electrocardiograms (ECG), or vital signs were reported
• Immunologic assessments are ongoing, data expected to be submitted to an upcoming meeting

PHILADELPHIA, March 27, 2024 /PRNewswire/ -- Virion Therapeutics, LLC, a clinical-stage biotechnology company, developing novel T cell-based immunotherapies, today announced promising safety results from the first-ever human data from its novel, first-in-class, checkpoint modifier immunotherapy, VRON-0200, for HBV functional cure, presented by Professor Grace Wong, M.D., from the Chinese University of Hong Kong, as a late breaker oral presentation at the 33^rd Annual Meeting of APASL (The Asian Pacific Association for the Study of the Liver) in Kyoto, Japan. 

These initial Phase 1B safety results, on the first 10 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 other clinically relevant abnormalities in laboratory tests, electrocardiograms (ECGs), or vital signs.

Professor Wong commented, "With almost 300 million persons living with chronic hepatitis B in the world, there is a high ? and critical - unmet medical need for a functional cure that is not only effective, but also safe. These first clinical data suggest that VRON-0200 is safe and well tolerated, after a single injection into the arm muscle. I look forward to additional safety and other clinical data being available soon."

"Finding a treatment for chronic HBV-infected patients will almost assuredly require an immune-based component to help control the infection. To date, the best responses have been observed with regimens that contain pegylated interferon, which requires weekly injections and is often accompanied by high rates of adverse events. These VRON-0200 data - the first ever for a checkpoint modifier containing T cell vaccine of any kind - represent a critical first step in Virion's mission to bring a safe, well tolerated, and easy to administer interferon-sparing functional cure immunotherapy to HBV-infected patients worldwide", said Dr. Sue Currie, COO of Virion, and one of the study authors. "We look forward to sharing additional safety, and the first-ever immunogenicity data, from this trial later this year."

Ocean Biomedical co-founder and Executive Chairman Dr. Chirinjeev Kathuria, commented: "We congratulate our Joint Venture Partner, Virion, for the first-ever clinical data presented for their checkpoint modifier immunotherapy and these initial results showing it can be administered simply, and safely, to chronically HBV-infected patients."

Summary of 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: Study consists of 2 cohorts: Cohort 1, low dose of VRON-0200; Cohort 2, receive a high dose
  • Prime or Prime-Boost: In each cohort, patients are randomized to receive an intramuscular injection as a prime vaccination only or as a prime and boost regimen

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. This includes almost 100 million persons in China who are affected by this disease. 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, 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

Contacts:
Virion Therapeutics, LLC Dr. Sue Currie
Chief Operating Officer [email protected]

SOURCE Virion Therapeutics, LLC