Phase 1, open-label, multicenter, dose escalation safety and tolerability study of oncolytic virus OVV-01 in advanced solid tumors

Background OVV-01 is a genetically engineered vesicular stomatitis virus oncolytic virus designed to selectively amplify in tumor cells and express tumor-associated antigen NY-ESO-1. This study was designed to evaluate the safety, tolerability, and efficacy of OVV-01 in patients with advanced solid...

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Main Authors: Yan Wang, Ning Li, Qiang Lin, Fan Li, Fan Zhang, Shuhang Wang, Yingqi Hua, Hui Wu, Liang Ma, Yinying Lu, Haitao Liu, Peng Yuan, Chongren Wang, Yanjie Han, Dongqing Zuo, Yu Lv, Mengxiong Sun, Ruirong Yuan, Guoqing Zhou
Format: Article
Language:English
Published: BMJ Publishing Group 2025-06-01
Series:Journal for ImmunoTherapy of Cancer
Online Access:https://jitc.bmj.com/content/13/6/e011517.full
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Summary:Background OVV-01 is a genetically engineered vesicular stomatitis virus oncolytic virus designed to selectively amplify in tumor cells and express tumor-associated antigen NY-ESO-1. This study was designed to evaluate the safety, tolerability, and efficacy of OVV-01 in patients with advanced solid tumors.Methods This is a phase 1, first-in-human, open-label, multicenter study of OVV-01 in patients with advanced solid tumors. OVV-01 was intratumorally injected biweekly (every two weeks, Q2W), 3 weeks after the first dose for a total of six doses. Dose escalation follows a 3+3 design at four doses of 6×107 Plaue-Forming Unit (PFU), 6×108 PFU, 6×109 PFU, and 1.2×1011 PFU. The primary endpoints were safety and tolerability. The second endpoints included overall response rate (ORR) and disease control rate (DCR) of OVV-01, by investigators per Response Evaluation Criteria in Solid Tumors V.1.1.Results 18 patients were enrolled into four dose groups, among whom 6 were soft tissue sarcoma (STS). No dose-limiting toxicities and treatment-related severe adverse events were observed. 11 patients were evaluated for efficacy, and the ORR was 27.3%, and the DCR was 63.6%. Among the four evaluable patients with advanced STS, the ORR was 75%. Two patients with STS achieved CR at doses above 6.0×109 PFU.Conclusions The intratumor injection of OVV-01 was safe and well-tolerated in patients with advanced solid tumors. A significant response was observed in patients with STS.Trial registration number NCT04787003.
ISSN:2051-1426