Transgene and BioInvent’s Oncolytic Virus BT-001 Shows Promising Antitumor Activity in Ongoing Phase I/IIa Trial in Solid Tumors that Failed Previous Treatments

Preliminary data presented at ESMO 2024 demonstrate that BT-001 induces tumor regression in patients who failed previous anti-PD(L)-1 treatment

In a patient with a heavily pretreated leiomyosarcoma, BT-001 was able to modulate the tumor microenvironment, turning a “cold” tumor to “hot”, enhancing the potential of T cell infiltration and a shift to PD(L)-1 positivity

Early signs of efficacy with clinical responses observed with BT-001 in combination with KEYTRUDA (pembrolizumab), in 2 of 6 patients who failed previous treatment

Strasbourg, France, and Lund, Sweden, September 14, 2024, 9:05 a.m. CET – Transgene (Euronext Paris: TNG), a biotech company that designs and develops virus-based immunotherapies for the treatment of cancer, and BioInvent International AB (“BioInvent”) (Nasdaq Stockholm: BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, today announce new initial data from their ongoing Phase I/IIa study on the multifunctional oncolytic virus BT-001, demonstrating antitumor activity in patients who failed previous treatments.

The data presented today at the 2024 European Society for Medical Oncology (ESMO) Annual Meeting, show that BT-001 induced tumor regression in patients unresponsive to prior anti PD(L)-1 treatment, both as a monotherapy and in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab).

Preliminary translational data suggest that BT-001 replicates in the tumor where the payloads are expressed with undetectable systemic exposure. BT-001 alone or in combination with pembrolizumab was well tolerated and showed first signs of efficacy with clinical responses in 2 of 6 patients who failed previous treatments, when given in combination with pembrolizumab. BT-001 treatment turned “cold” tumors to “hot” inducing T cell infiltration, a higher M1/M2 ratio, and a shift to PD(L)-1 positivity in the tumor microenvironment.

Dr. Stéphane Champiat, Medical Oncologist, Head of the Inpatient Unit, Drug Development Department (DITEP) at Institut Gustave Roussy, commented: “The immunological data generated by BT-001 suggest that, as hoped, BT-001 is replicating in the tumor and its payload of transgenes is expressed with very limited exposure outside of the tumor thereby limiting systemic toxicity. I look forward to additional results from this ongoing study which will provide further evidence of the safety and clinical activity of BT-001 and its potential role as a new therapy for cancer patients with solid tumors.”