EsoBiotec SA, in collaboration with Pregene Biopharma, has announced the dosing of the first patient in a Phase 1 investigator-initiated clinical trial of ESO-T01, an in vivo BCMA CAR-T therapy, for patients with relapsed/refractory multiple myeloma. This trial marks a significant step forward in the development of off-the-shelf CAR-T therapies that can be administered directly within the patient's body. The study, taking place in China, aims to evaluate the safety, tolerability, and preliminary efficacy of ESO-T01 in up to 24 patients.
Innovative In Vivo CAR-T Approach
ESO-T01 is designed to reprogram T lymphocytes in vivo into highly effective BCMA CAR-T cells using EsoBiotec's ENaBL platform, a third-generation immune-shielded lentiviral vector. This innovative approach aims to overcome the limitations of traditional ex vivo CAR-T therapies, which are often costly, logistically complex, and associated with significant side effects. ESO-T01 is administered as a single intravenous infusion and does not require prior lymphodepletion, potentially simplifying the patient journey and reducing treatment costs.
Early Clinical Observations
According to Principal Investigator Professor Heng MEI, initial clinical observations with ESO-T01 indicate a favorable safety profile and promising efficacy at the first dose level of 0.25E+09 transducing units per patient. Notably, the first patient treated with ESO-T01 showed no detectable cancer cells in the bone marrow by day 28, and elevated free light chain levels secreted by tumor cells had normalized. No significant adverse events were reported during the treatment of the first patient.
Trial Design and Endpoints
The multi-center, single-arm, open-label, dose-escalation clinical trial (NCT06691685) is designed to evaluate the safety, tolerability, and preliminary clinical activity, including in vivo reprogramming efficiency, of a single intravenous infusion of ESO-T01 across escalating doses. Primary endpoints for the study include the incidence and severity of cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS), dose-limiting toxicities (DLTs), and treatment-associated adverse effects.
Potential Benefits and Future Outlook
EsoBiotec CEO Jean-Pierre Latere, Ph.D., stated that ESO-T01 has the potential to offer a simplified patient journey with several benefits over current treatments for multiple myeloma. The treatment's ability to be administered without lymphodepletion, immediate availability, and single intravenous dose in under 10 minutes could significantly improve patient access and reduce healthcare costs. Initial clinical data from the trial are expected in the second half of 2025.
Dr. Li Hongjian, CEO of Pregene Biopharma, highlighted the potential of in vivo delivery to expand the benefits of CAR-T therapy to more multiple myeloma patients and potentially extend its application to autoimmune diseases. The collaboration between EsoBiotec and Pregene Biopharma aims to pioneer innovative scientific advancements that could redefine the future of cell therapy.
