Iaso Bio's BCMA-Targeted CAR-T Therapy Shows Breakthrough Efficacy in Progressive Multiple Sclerosis, Published in Cell

Source: Xinhua Finance

Recently, IASO Bio, a company based in Nanjing Jiangbei New Area, published the results of its self-developed fully human BCMA-targeted CAR-T cell therapy, Ixcellen (generic name: elenctoclax), for the treatment of progressive multiple sclerosis, in the prestigious international journal *Cell*. While addressing whether CAR-T therapies can open new "battlefields" beyond areas like hematological tumors, this also showcases the innovative cellular therapy capabilities from Jiangbei New Area to the world.

The research成果 published in this issue of《Cell》confirms the safety and efficacy of CAR-T cell therapy in treating progressive multiple sclerosis. Multiple sclerosis is an autoimmune demyelinating disease of the central nervous system, known as the "killer of neurologic health in young and middle-aged adults." Common symptoms include sensory and visual disturbances, motor and coordination disorders, and in severe cases, patients may even experience difficulty walking or paralysis.

It is reported that this study was initiated by the research team led by Professor Wang Wei from the Department of Neurology at Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology. A total of five patients with progressive multiple sclerosis participated. In the study, all five patients received a single infusion of Idecabtagene Vicleucel Injection — a CAR-T product independently developed and fully produced by IASO Bio. This product was approved for marketing by China's National Medical Products Administration in June 2023 for the treatment of relapsed or refractory multiple myeloma. To date, it has benefited hundreds of multiple myeloma patients in China and attracted more than 40 overseas patients from Europe, Africa, Southeast Asia, and other regions to come to China for treatment.

After receiving the infusion, patients showed significant therapeutic effects: all patients experienced improvements in mobility, hand function, and walking speed. The "harmful elements" in the cerebrospinal fluid completely disappeared or were significantly reduced, and the disease progression halted. More importantly, the treatment demonstrated good safety; 80% of patients had only brief, mild reactions without serious side effects.

"This is an important step forward in the field of cell therapy," said Wang Wei. Progressive multiple sclerosis has always been a difficult problem in the medical community, with traditional drugs unable to halt its progressively worsening course. However, in this study, 83% of refractory patients achieved long-term clinical remission without the need for drug maintenance after receiving the treatment, indicating new possibilities for the treatment of multiple sclerosis.

Significant efficacy and good safety, such results are encouraging. It is worth mentioning that this is also the first time that "Cell" has published research results on the successful treatment of autoimmune diseases with targeted BCMA CAR-T cell therapy, marking an important scientific breakthrough in this field. In recent years, the application of CAR-T cell therapy in the field of malignant hematological tumor treatment has been widely accepted. At the same time, for the second largest drug market after tumors—autoimmune diseases—the academic and industrial communities are also exploring new possibilities. As a "leader" in the cell therapy track, IASO Bio is one of the earliest companies internationally to apply CAR-T therapy to the autoimmune field. As early as 2022, the team submitted the first clinical trial application for CAR-T cell therapy to treat autoimmune diseases in Jiangbei New Area.

After years of exploration, IASO Bio's Equecabtagene Autoleucel Injection has now been approved for multiple clinical trials in autoimmune indications in both China and the U.S., covering difficult-to-treat diseases such as multiple sclerosis, myasthenia gravis, and systemic lupus erythematosus. "The publication of these results in *Cell* demonstrates the therapeutic potential of Equecabtagene Autoleucel in the field of autoimmune diseases," said Zhang Jinhua, founder, chairman, and CEO of IASO Bio. "In the future, we will continue to accelerate our global presence, relying on a complete self-developed and production system to promote the clinical transformation of more innovative cell therapies, bringing fundamental breakthroughs in treatment to patients with autoimmune diseases."

From hematological tumors to autoimmune diseases, the exploration and expansion of IASO Bio are reshaping the treatment landscape for many refractory diseases and bringing hope to an increasing number of patients.New HopeAnchoring innovation, this team, which firmly grasps the capabilities of drug clinical development and production, is also set to advance onto a broader stage. (Shen Yangzi)

Editor: Mu Hao