Chinese Biopharma Advances in ALS Therapeutics: Celixen, VITARIS, and WBD Pharma Report Key Milestones

　　【Pharmaceutical Network Industry DynamicsALS is an irreversible and fatal motor neuron disease. Due to its extremely complex pathogenic mechanisms and the lack of a clear therapeutic target, the failure rate for developing new ALS drugs has been high for a long time, and treatment options remain very limited. However, with the rapid advancement of biotechnology and the growing clinical demand, the number of clinical trials for ALS treatment drugs has been gradually increasing in recent years. Many new drugs, with their unique targeted mechanisms of action, are bringing new hope for patients.
 

Recently, it was reported that the NP001 project of Shanghai Saierxin Biomedical Technology Co., Ltd. (NOVABIOTX) has been accepted by the FDA. This drug is the world's first Treg cell therapy product for ALS, administered via intrathecal injection. The IND application has been completed in both China and the United States, and it is currently undergoing clinical research, with preliminary data showing effectiveness.
 

It is reported that on December 24, 2024, NOVABIOTX successfully performed the world's first intrathecal injection therapy using autologous polyclonal regulatory T cells (Treg) for patients with Amyotrophic Lateral Sclerosis (ALS, commonly known as Lou Gehrig's disease) at the First Affiliated Hospital of Zhengzhou University. The treatment utilized the NP001 product. At the time, all physiological indicators of the treated subject were in good condition, the treatment process went smoothly, the patient remained stable, and no adverse reactions were observed.
 

Notably, NOVABIOTX's NP001 project has recently seen a series of positive developments. In addition to being accepted by the FDA, the company also announced not long ago that it had successfully completed a Pre-A round of financing worth tens of millions of RMB. This round of financing will strongly support the advancement of clinical trials for the NP001 project as well as the research and development of other products in the pipeline, facilitating the clinical translation and commercial development of Treg therapy.
 

On February 19, according to CDE, SIR2501 tablets, a Class 1 new drug developed by Sironax (Beijing) Co., Ltd., have been approved for clinical trials for the treatment of ALS.
 

Public information shows that Sironax (Beijing) Co., Ltd. is committed to developing transformative therapies that fundamentally address aging-related degenerative diseases. The company adopts a pipeline strategy focused on key pathogenic signaling pathways for product development. Since its establishment, it has built a diversified R&D pipeline concentrating on the pathogenic mechanisms of aging-related degenerative diseases, including programmed cell death, neuroprotective pathways, and neuroinflammation. The recently approved clinical trial for SIR2501 tablets targets ALS as the indication, but the specific mechanism of action has not yet been disclosed.
 

On February 17, Wanbangde announced that its wholly-owned subsidiary, Wanbangde Pharmaceutical Group Co., Ltd., received a designation letter from the FDA on February 13, 2025. Mecobalamin for the treatment of Amyotrophic Lateral Sclerosis (ALS, also known as Lou Gehrig's disease) has been granted Orphan Drug Designation by the FDA.
 

The announcement pointed out that methylcobalamin possesses a dual neuroprotective mechanism and has shown potential value in the treatment of neurodegenerative diseases. Currently, the methylcobalamin lyophilized powder injection has completed clinical trials by Eisai, a leading international pharmaceutical company, and was approved for the treatment of ALS in Japan in September 2024.
 

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