BioInvent and Transgene's BT-001 Virus Shows Promise in Phase 1/2a Trial for Solid Tumors

BioInvent International AB, a biotech firm headquartered in Lund, Sweden, and Transgene, based in Strasbourg, France, have announced promising initial data from their Phase 2/2a clinical trial for the oncolytic virus BT-001. This virus, designed to combat refractory solid tumors, has shown significant antitumor activity according to findings presented at the 2024 European Society for Medical Oncology (ESMO) Annual Meeting.

BT-001, which targets tumors unresponsive to previous anti-PD(L)-1 treatments, demonstrates its efficacy both as a standalone therapy and in combination with Merck’s anti-PD-1 therapy, KEYTRUDA® (pembrolizumab). The preliminary data suggest that BT-001 replicates within the tumor, leading to the expression of its therapeutic payloads while maintaining minimal systemic exposure. This minimizes side effects and enhances the treatment's safety profile. Notably, the trial reported positive clinical responses in two out of six patients when BT-001 was combined with pembrolizumab.

The treatment appears to convert "cold" tumors, which typically evade immune detection, into "hot" tumors that are more visible to the immune system. This is evidenced by increased T cell infiltration, a higher M1/M2 macrophage ratio, and a shift towards PD(L)-1 positivity within the tumor microenvironment.

Dr. S. Champiat, a Medical Oncologist at Institut Gustave Roussy, emphasized the significance of BT-001’s ability to replicate within the tumor and express its transgenes with limited systemic toxicity. Champiat looks forward to further results from this study, which could solidify BT-001’s potential as a new therapeutic option for patients with solid tumors.

BT-001 is a product of a collaboration between BioInvent and Transgene. It harnesses Transgene’s Invir.IO® platform, combined with BioInvent’s full-length anti-CTLA-4 monoclonal antibody, to trigger a robust anti-tumor response. Alessandro Riva, Chairman and CEO of Transgene, highlighted the clinical data's promise, noting that BT-001, when administered intratumorally, has shown initial signs of anti-tumor activity along with a favorable safety profile. Riva anticipates further exploration of BT-001’s safety and efficacy in collaboration with BioInvent.

BioInvent’s Chief Medical Officer, Andres McAllister, expressed optimism regarding the early clinical results. He noted that BT-001, which includes a Treg-depleting recombinant human anti-CTLA-4 antibody developed through BioInvent's proprietary platforms, has shown proof of concept in clinical settings. This validation confirms BioInvent's capability to develop antibodies targeting specific functions, paving the way for new drug candidates like BT-001.

The ongoing Phase 1/2a study of BT-001 is a multicenter, open-label trial aiming to evaluate the drug both as a monotherapy and in combination with pembrolizumab. The trial, which is taking place in Europe and has been authorized in the U.S., seeks to include patients with metastatic or advanced tumors. The study is divided into two parts: Phase 1 focuses on dose-escalation and safety, while Phase 2a will assess the combination regimen across various patient cohorts with selected tumor types, potentially expanding treatment options for other cancers.

BT-001's development leverages Transgene’s Invir.IO® platform, which creates oncolytic viruses capable of encoding therapeutic agents directly within tumors. By concentrating its action on the tumor microenvironment, BT-001 aims to provoke a stronger antitumor response while reducing systemic exposure and toxicity. This innovative approach is being co-developed by BioInvent and Transgene under a 50/50 partnership.

BioInvent International AB specializes in discovering and developing novel immunomodulatory antibodies for cancer therapy. The company currently has four drug candidates in various stages of clinical trials for treating both hematological cancers and solid tumors. BioInvent's proprietary technology platforms identify promising new drug candidates, fueling its clinical pipeline and creating opportunities for research collaborations and licensing.

Transgene focuses on targeted immunotherapies for cancer, utilizing viral vector technology to design treatments aimed at both direct and indirect cancer cell destruction. The company's clinical-stage programs include therapeutic vaccines and oncolytic viruses, with BT-001 being a prominent example of its innovative approach to cancer treatment.