Chinese Biotechs Targeting ALS Secure Significant Funding and Advance Innovative Therapies

　　【Pharmaceutical Network Industry DynamicsIn recent years, ALS has gradually come into the public eye. ALS, medically known as Amyotrophic Lateral Sclerosis, is a neurodegenerative disease that affects motor neurons in the brain and spinal cord.
 

Currently, there are four drugs for treating ALS (Amyotrophic Lateral Sclerosis) approved by the U.S. FDA available globally: Rilutek (Riluzole tablets) developed by Sanofi (approved in 1995), Edaravone (approved in 2017), Relyvrio developed by Amylyx Pharmaceuticals (launched in 2022), and Qalsody, a therapy for hereditary ALS (launched in 2023).
 

It is reported that in the Chinese market, several companies developing innovative drugs for ALS have recently received capital investment.
 

SINEUGENE announced on August 1st that it has completed a Pre-A round of financing worth nearly 100 million yuan. This round was led by Zhongguancun Capital, with participation from ZB Julei, Chuanghua Capital, and Sanmei Investment. The funds will mainly be used for the IND (Investigational New Drug) application of SINEUGENE's first pipeline, SNUG01, and the advancement of other pipeline products.
 

Data shows that SINEUGENE started its entrepreneurial journey with ALS (Amyotrophic Lateral Sclerosis, commonly known as "Lou Gehrig's disease"). The company has built platforms for novel gene knock-in animal disease models, such as fruit flies and mice, along with a central nervous system Adeno-Associated Virus (AAV) screening platform. It is committed to developing treatments for neurological diseases like ALS, stroke, and Parkinson’s disease. It is reported that SINEUGENE has six R&D pipelines, five of which are related to ALS. Among them, patient recruitment for the IIT (Investigator-Initiated Trial) of SNUG01 (SG001 pipeline product) is currently underway at Peking University Third Hospital.
 

In July, ReviR Therapeutics completed a US$30 million Series A round of financing. This round was led by Lapam Capital, with continued investment from existing shareholders CDH Investments, Five Source Capital, and Yabai Capital. XtalPi and the CMT Research Foundation (CMTRF) also participated in the investment.
 

ReviR Therapeutics is a biotechnology company that combines AI technology to develop small-molecule drugs targeting RNA. This round of investment will be used to further build ReviR's self-developed AI drug discovery platform, VoyageR, and integrate AI technology with the extensive drug development experience of the ReviR team to continuously advance the preclinical and clinical development of existing pipelines for Huntington's disease (HD) as well as multiple neurological disease-related pipelines, including Charcot-Marie-Tooth disease (CMT) and amyotrophic lateral sclerosis (ALS).
 

It is reported that, through self-developed and collaborative approaches, ReviR Therapeutics has established a pipeline covering multiple neurological diseases such as Charcot-Marie-Tooth disease, amyotrophic lateral sclerosis (ALS), and cerebellar ataxia. Among them, the fastest progressing is the self-developed pipeline targeting Huntington's disease. Additionally, the company’s ALS pipeline is currently showing promising progress, with an anticipated IND submission and Phase I clinical trial initiation by early 2025.
 

In addition, Shenxi Bio, a technological innovation company focusing on nerve injury and degenerative diseases, also completed a Pre-A+ round of financing exceeding 100 million RMB in May 2023. Data shows that Shenxi Bio's product pipeline covers major neurological injuries and neurodegenerative diseases, such as stroke, glioblastoma, Huntington's disease, ALS, Alzheimer's disease, Parkinson's disease, traumatic brain injury, spinal cord injury, and ophthalmic diseases, among others. However, the advancement of Shenxi Bio’s ALS drug pipeline is still in the product optimization stage and has not yet reached the preclinical research phase.
 

Although a number of pharmaceutical companies are currently stepping up the development of drugs for ALS treatment and some have secured capital investment, industry analysts point out that achieving significant breakthroughs and bringing domestically-produced ALS drugs to market remains fraught with challenges. For instance, medical data on ALS patients cannot be fully integrated, and the samples available to doctors and research institutions are insufficient. Moreover, the main organs affected by ALS are the brain and spinal cord, which, unlike other parts of the body, cannot undergo biopsies while the patient is alive.
 

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