Helixmith's 'Enzencis' Completes First Treatment Cycle in Half of ALS Patients in Phase 2a Clinical Trial

[Asia Economy Reporter Jang Hyowon] Helixmith announced on the 5th that in the Phase 2a clinical trial for the gene therapy ‘Engensis (VM202)’ targeting amyotrophic lateral sclerosis (ALS, Lou Gehrig's disease), 9 patients, accounting for 50% of the total participants, have completed the first treatment cycle and are progressing smoothly.

Helixmith's ALS Phase 2a clinical trial is currently underway in the United States and Korea. It involves 18 ALS patients and is being conducted at four clinical trial centers in the U.S. and Hanyang University Hospital in Korea.

In the ALS Phase 2a trial, the safety and efficacy of Engensis (VM202) are being investigated through a total of three treatment cycles over 180 days. The primary endpoint is safety, focusing on the occurrence of serious adverse events during the clinical period. The efficacy endpoints being measured include ▲ changes from baseline in the ALS Functional Rating Scale-Revised (ALSFRS-r) assessing physical function of ALS patients ▲ changes in muscle function and strength ▲ disease-specific and patient-reported health status changes ▲ patients’ overall impression of change ▲ overall impression of change ▲ effects on respiratory function and survival ▲ changes in biomarkers of muscle atrophy.

Helixmith plans to proceed with a Phase 2b clinical trial with a statistically powered sample size based on the results after completing the ALS Phase 2a trials in the U.S. and Korea.

Sunyoung Kim, CEO of Helixmith, stated, “Among the indications discussed for licensing Engensis (VM202) with global pharmaceutical companies, ALS receives the most attention,” adding, “If safety and efficacy are demonstrated in this Phase 2a trial, the impact will be significant.”

Meanwhile, amyotrophic lateral sclerosis (ALS) is a fatal progressive neuromuscular disease also known as Lou Gehrig's disease. It destroys motor neurons (nerve cells) that control muscle movement, making all types of voluntary movement impossible, and eventually causes death as the diaphragm muscles responsible for breathing cease to function.

Approximately 1 in 20,000 people are diagnosed with ALS, and it is estimated that about 30,000 patients currently live in the United States. ALS attracts significant attention not only from global pharmaceutical companies but also socially and politically. The U.S. FDA has even issued separate guidelines to encourage the development of ALS treatments by lowering regulatory barriers. Most ALS cases still have unclear causes, and only two drugs approved by the U.S. FDA exist, both with limited efficacy.

Helixmith’s Engensis (VM202) is a drug composed of plasmid DNA expressing hepatocyte growth factor (HGF) protein. Through 15 years of research and clinical trials, it has been demonstrated that a simple muscle injection can produce HGF protein in vivo, promoting nerve regeneration, angiogenesis, and prevention of muscle atrophy.

This content was produced with the assistance of AI translation services.