Breakthrough Therapies Bring New Hope to ALS as Innovative Drug Developers Advance Toward IPO

　　【Pharmaceutical Network Industry Dynamics]"ALS" (Amyotrophic Lateral Sclerosis; ALS) is a type of motor neuron disease and progressive neurodegenerative disorder, with patients commonly referred to as "ALS patients." Clinical data shows that the average life expectancy for ALS patients is approximately 39 months. Currently, there are no effective clinical drugs or solutions that can substantially alleviate or reverse the progression of ALS. It is reported that in the field of ALS drug development, some pharmaceutical companies are actively advancing research and gradually achieving new progress.
 

On December 24, 2024, Ractigen announced that the first subject in the Phase I clinical trial of its innovative therapy RAG-17 for Amyotrophic Lateral Sclerosis (ALS) caused by Superoxide Dismutase 1 (SOD1) gene mutation was dosed at the Second Affiliated Hospital of Zhejiang University School of Medicine.
 

This study is a randomized, double-blind, placebo-controlled Phase I clinical trial designed to evaluate the safety/tolerability, pharmacokinetics, pharmacodynamics, and preliminary efficacy of RAG-17 in ALS patients carrying the SOD1 gene mutation. Relevant persons from Ractigen stated that the completion of dosing for the first subject in the RAG-17 trial marks an important step for the company in addressing amyotrophic lateral sclerosis (ALS), a neurodegenerative disease. This type of therapy is expected to bring life-changing hope to patients suffering from severe rare diseases like ALS and their families.
 

It is reported that RAG-17 obtained orphan drug designation from the U.S. FDA in March 2023, and subsequently received clinical trial approvals from both the FDA and China's National Medical Products Administration Center for Drug Evaluation (CDE) in 2024. In November 2024, RAG-17 demonstrated positive clinical outcomes in an investigator-initiated trial (IIT), further validating its potential.
 

In addition, on December 10, SPH announced that SRD4610, independently developed by its wholly-owned subsidiary Shanghai Shangyao Rui'er Pharmaceutical Co., Ltd. ("SPH Rui'er"), has been granted Orphan Drug Designation by the U.S. FDA for the treatment of Amyotrophic Lateral Sclerosis (ALS). It is reported that SRD4610 is an innovative compound traditional Chinese medicine, and the project has currently completed Phase II clinical trials in China targeting Amyotrophic Lateral Sclerosis.
 

On November 28, "Shizhe Bio" released an article stating that Shizhe Biopharmaceuticals (Suzhou) Co., Ltd., in collaboration with the Oriental Hospital of Shanghai (Oriental Hospital Affiliated to Tongji University) and the Liu Zhongmin team, successfully completed the world's first patient enrollment for "Clinical-grade iPSC-derived subtype neural progenitor cell therapy for ALS (Amyotrophic Lateral Sclerosis; ALS)."
 

It is reported that, after several rounds of rigorous review, this clinical research project has been officially approved by the two national commissions and bureaus as a national stem cell registered clinical research project to be formally carried out: This clinical research project is the first officially approved clinical study in China to use iPSC-derived cell therapy for ALS treatment, and also the world's first allogeneic universal-grade clinical iPSC-derived neural cell regenerative treatment for ALS.
 

Experts in the industry indicate that, with continuous advancements in innovative drugs, new hope is on the horizon for ALS, a significant challenge. According to statistics, there are currently three ALS drugs approved for use in China. Among them is Tofersen Injection (Tofersen), which was approved for marketing this October. The drug, produced by Biogen Biotechnology (Shanghai) Co., Ltd., is used to treat adult patients with amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease) who carry a mutation in the superoxide dismutase 1 (SOD1) gene. It is a causative treatment for SOD1-ALS.
 

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