Sailxin Bio's NP001 (Treg) Receives FDA IND Clearance for ALS Treatment, Marking World’s First Treg Therapy for ALS

On February 19, 2025, the NP001 (Treg) project independently developed by Shanghai Saierxin Biomedical Technology Co., Ltd. (hereinafter referred to as "NOVABIOTX") was officially accepted by the U.S. Food and Drug Administration (FDA) for a new drug clinical trial application (IND) (Application Type/Number: IND 031401.0). The NP001 project is not onlyWorld's First Treg Cell Product Administered via Intrathecal Injection, and even moreWorld's First ALS (Amyotrophic Lateral Sclerosis) Treatment Completes IND Filings in China and the U.S.Cell therapy products. Currently, there are no Treg cell therapy products on the market globally, marking that China's cell therapy has entered the forefront of international research and development.

Previously, the NP001 project had received Orphan Drug Designation (ODD) from the FDA and also obtained IND acceptance from the Center for Drug Evaluation (CDE) of the National Medical Products Administration. The NP001 product is an autologous polyclonal regulatory T-cell (Treg) injection, with its IND application based on rigorous process development and non-clinical study results. NOVABIOTX has now become the first company to complete dual IND submissions in both China and the U.S. for ALS indication.

This product uses precise intrathecal injection to directly deliver autologous polyclonal Treg cells to the patient's affected area, more effectively modulating the immune environment, promoting tissue repair and regeneration, thereby slowing disease progression. Most clinical manifestations of ALS include speech and swallowing difficulties, as well as degenerative diseases of limb muscles, posing a severe threat to patients' quality of life. The survival period for patients is 2-5 years, with approximately 50% surviving within 3 years, and the overall mortality rate is extremely high. There are very limited treatments for ALS currently reported or on the market, mainly alleviating a specific pathophysiological process by inhibiting glutamate release (preventing neuronal overexcitation) or scavenging reactive oxygen species (preventing mitochondrial dysfunction). For most patients, multidimensional efficacy cannot be achieved.

In addition to the NP001 polyclonal Treg product, NOVABIOTX also has several products in development, including Car-Treg and off-the-shelf allogeneic Treg. NOVABIOTX will continue to leverage the unique advantages of Treg cells in therapeutic applications, advance the research and industrialization process of the NP001 product, and strive to bring the benefits of this innovative therapy to more patients with immune disorders and neurodegenerative diseases.

Latest Clinical Data and Future Prospects

Currently, the product is undergoing IIT clinical research at the First Affiliated Hospital of Zhengzhou University, with three ALS patients already in the clinical stage. Preliminary data shows that the first patient who completed the full dosing regimen has remained stable after being infused with the NP001 product. Additionally, the declining trend of the subject's ALSFRS-R score has been alleviated, and grip strength has also improved, demonstrating an encouraging trend of efficacy. Other data are still being continuously monitored and analyzed.