Transgene and BioInvent’s Armed Oncolytic Virus BT-001 Shows Positive Local, Abscopal, and Sustained Antitumoral Activity in Advanced Refractory Tumors

Core Insights - BT-001, in combination with pembrolizumab, demonstrates good tolerability and sustained antitumoral activity in both injected and non-injected lesions, supporting further development in solid tumors to enhance cancer immunotherapy responses [1][5][6] Clinical Results - Intra-tumoral injection of BT-001 combined with KEYTRUDA® (pembrolizumab) showed positive local, abscopal, and sustained antitumoral activity [2] - Significant tumor shrinkage (≥30% decrease in longest diameter) was observed in five of 16 injected lesions across three melanoma patients and one sarcoma patient, with four patients experiencing shrinkage in non-injected lesions [3] - Long-lasting partial responses were noted in a melanoma patient resistant to anti-PD-1/anti-CTLA-4 therapy and a heavily pre-treated, PD-L1 negative leiomyosarcoma patient [4] Mechanism of Action - The immune-mediated tumor shrinkages align with the hypothesis that BT-001, in combination with pembrolizumab, can convert "cold" tumors into immunologically active ones [5] - BT-001 is designed to induce a strong anti-tumor response by expressing GM-CSF and an anti-CTLA-4 antibody, potentially expanding treatment options with a favorable safety profile [6][11] Company Statements - Transgene and BioInvent are co-developing BT-001, which utilizes Transgene's Invir.IO® platform to enhance replication selectivity in tumor cells and express an anti-CTLA-4 antibody [6][12] - The companies aim to continue exploring the safety and efficacy of BT-001, with ongoing clinical trials evaluating its use alone and in combination with pembrolizumab [11][12]