Helixmith Administers First Patient Dose in Domestic Phase 2a Trial of Enzensus... Expectations for ALS Treatment

[Asia Economy Reporter Hyungsoo Park] Helixmith announced on the 10th that it has administered the first dose to a patient in the domestic Phase 2a clinical trial of the gene therapy drug 'Engensis (VM202)' for amyotrophic lateral sclerosis (ALS). Engensis has been designated as an orphan drug and granted fast track status for ALS by the U.S. FDA.

The clinical trial team led by Professor Seunghyun Kim of Hanyang University Hospital began administering Engensis to ALS clinical patients on the 9th. Helixmith's Phase 2a ALS clinical trials are being conducted in both the United States and Korea. The Phase 2a trial will enroll a total of 18 patients, with an expected 30-50% of the patients registered in Korea.

Helixmith plans to secure safety and efficacy data through the clinical trials. After completing the Phase 2a trials for ALS in the U.S. and Korea, the company will proceed with a Phase 2b trial of a scale sufficient to achieve statistical power based on the results.

Sunyoung Kim, CEO of Helixmith, stated, "Engensis represents a new approach to neuromuscular diseases," adding, "We hope to offer a new treatment option to ALS patients who currently have no available therapies."

The majority of ALS cases have unclear causes, and the drugs approved by the U.S. FDA have minimal therapeutic effects. To encourage the development of ALS treatments, the U.S. FDA has established separate guidelines to lower regulatory barriers.

Helixmith's Engensis is a drug composed of plasmid DNA expressing hepatocyte growth factor (HGF) protein. Over the past 15 years of research and clinical trials, it has been shown that a simple muscle injection can produce HGF protein in vivo, which is expected to promote nerve regeneration, angiogenesis, and prevent muscle atrophy.

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