EsoBiotec Starts Clinical Trial of ESO-T01 BCMA CAR-T for Multiple Myeloma

On December 11, 2024, EsoBiotec SA, a biotechnology firm specializing in in vivo cell engineering for cancer treatment, announced the initiation of a clinical trial in China. The trial involves ESO-T01, an innovative immune-shielded lentiviral vector designed to reprogram T lymphocytes within the body into highly effective BCMA CAR-T cells, specifically targeting multiple myeloma.

ESO-T01 is a groundbreaking development in the field of cancer treatment, being the first in vivo BCMA CAR-T candidate to reach the clinical stage. Jean-Pierre Latere, Ph.D., the CEO of EsoBiotec, emphasized the significance of the company’s ENaBL technology platform, which allows for the efficient reprogramming of immune cells inside the patient’s body to combat cancer. He noted that existing treatments for multiple myeloma, including ex vivo CAR-T therapies, often come with severe side effects and accessibility issues due to manufacturing and logistical challenges. With this trial, EsoBiotec aims to assess the safety and efficacy of ESO-T01, potentially paving the way for its use in other conditions, such as autoimmune diseases.

Philippe Parone, Ph.D., the Chief Scientific Officer of EsoBiotec, elaborated on the advantages of ESO-T01, which harnesses the ENaBL platform to transform patient T cells into potent BCMA CAR-T cells. This method ensures high specificity and effective transduction in vivo, thanks to the unique design of the ENaBL technology. When paired with a scalable and reliable manufacturing process, ESO-T01 offers a promising opportunity to provide an affordable, ready-to-use therapy, thereby enhancing access to advanced treatments.

The first-in-human Investigator-Initiated Trial (IIT) is currently underway, with preliminary clinical results expected in the latter half of 2025. In preclinical trials, a single dose of ESO-T01 showed significant anti-tumor effects in humanized mice, demonstrating effective in vivo transduction and specific expression of the BCMA CAR transgene in T cells. This resulted in a substantial population of circulating BCMA CAR-T cells, whose presence was sustained throughout the study, indicating their potential long-term effectiveness.

Jean-Pierre Latere highlighted the company’s successful transition from operating discreetly to entering the clinical phase, supported by a €22 million funding round. This funding was secured from dedicated investors such as Thuja Capital, UCB Ventures, Invivo Partners, Wallonie Entreprendre, SambrInvest, and Investsud, even amidst challenging economic conditions. With the commencement of clinical trials, EsoBiotec is strategically positioned to introduce transformative cancer therapies worldwide, building on its unique scientific approach, experienced team, and established partnerships.

ESO-T01 is a third-generation, replication-deficient, self-inactivating lentiviral vector that encodes a BCMA-targeted CAR construct driven by a T cell-specific synthetic promoter. It is immune shielded and resistant to phagocytosis, offering a single-dose treatment that can be administered directly without lymphodepletion. The ENaBL platform used by EsoBiotec is crafted to selectively reprogram T cells, demonstrating remarkable CAR T cell potency in preclinical models. The company has maintained vector specificity and purity in large-scale clinical production, validating this novel approach with a clinically proven antigen.

EsoBiotec, a privately owned biotechnology company, focuses on the in vivo engineering of T-cells and other immune cells to create cost-effective, off-the-shelf therapies. The company, supported by investors such as Thuja Capital, UCB Ventures, Invivo Partners, Wallonie Entreprendre, SambrInvest, and Investsud, aims to enhance the accessibility of innovative cancer treatments through its ENaBL platform. The ongoing clinical trial evaluates the potential of ESO-T01 in treating multiple myeloma, while the company's pipeline includes two novel combination candidates, ESO-TX101 and ESO-TX102, intended for solid tumor treatment through the simultaneous engineering of T cells and monocytes to remodel the tumor microenvironment.