EsoBiotec Begins First Patient Dosing in BCMA CAR-T Trial for Multiple Myeloma

EsoBiotec SA, in collaboration with Pregene Biopharma, has announced a significant advancement in cancer treatment with the dosing of the first patient in a clinical trial for ESO-T01, targeting relapsed/refractory multiple myeloma. This experimental therapy utilizes an immune-shielded lentiviral vector to reprogram T lymphocytes in vivo, transforming them into potent BCMA CAR-T cells.

The clinical trial, identified as NCT06691685, is a multi-center, single-arm, open-label study designed to evaluate the safety, tolerability, and early clinical effects of ESO-T01 through a single intravenous infusion. The study aims to enroll up to 24 multiple myeloma patients, focusing on determining the incidence and severity of side effects such as cytokine release syndrome and neurotoxicity, as well as identifying dose-limiting toxicities and other treatment-related adverse events.

In early observations, ESO-T01 has shown a promising safety profile and efficacy at an initial dose level of 0.25E+09 transducing units per patient. According to Professor Heng MEI, the Principal Investigator, the first patient received the treatment without prior lymphodepletion. By the 28th day post-treatment, no cancer cells were detected in the bone marrow, and abnormal light chain levels had returned to normal, without significant adverse events during the treatment.

The innovative approach of ESO-T01 offers a simplified treatment pathway, potentially providing several advantages over existing multiple myeloma therapies, which are often expensive and carry unfavorable side effects. EsoBiotec CEO Jean-Pierre Latere highlights that the treatment is available immediately to patients, administered in a single intravenous dose that takes less than 10 minutes, and does not require the lymphodepletion process.

Pregene Biopharma's CEO, Dr. Li Hongjian, emphasizes the potential of in vivo delivery to broaden patient access to effective CAR-T therapy, particularly for multiple myeloma and possibly extending to autoimmune diseases. This collaboration aims to make innovative, affordable treatments widely accessible, with the potential to redefine the future of cell therapy.

ESO-T01 is the first in vivo BCMA CAR-T candidate to be tested in humans and is developed using EsoBiotec's third-generation lentiviral vector platform, ENaBL. This platform reprograms immune cells within the body, combined with a transgene developed by Pregene Biopharma. This innovative approach offers an "off-the-shelf" treatment alternative, potentially reducing manufacturing costs significantly compared to traditional ex vivo CAR-T therapies.

EsoBiotec's ENaBL platform vectors are designed for efficient T cell reprogramming, demonstrating substantial CAR T potency in preclinical models. The company's advanced manufacturing processes maintain high vector specificity and purity, crucial for developing ESO-T01 as a viable treatment option. The ongoing clinical trial assesses ESO-T01's effectiveness for multiple myeloma, with plans to expand the application of their technology to other solid tumors through candidates like ESO-TX101 and ESO-TX102.

Pregene Biopharma, a leader in innovative cell and gene therapies, supports this clinical development with its advanced manufacturing technologies and a diverse pipeline of clinical-stage candidates. The collaboration between EsoBiotec and Pregene Biopharma represents a significant step forward in making state-of-the-art cancer care more accessible and cost-effective.