Local pharma to begin phase 2 clinical trial for Lou Gehrig’s disease therapy

South Korea's drug regulator has granted approval for a Phase IIa trial of Helixmith's investigational gene therapy Engensis for treating amyotrophic lateral sclerosis (ALS), reported The Korea Herald.

Engensis utilizes a plasmid to provide the hepatocyte growth factor gene to patients' nerve and nerve-supporting cells, which could help the formation of new blood vessels, prevent muscle atrophy and aid the growth and survival of nerve cells.

The study in South Korea will be similar to one that Helixmith has been conducting at four clinical centers in the US since March this year. In South Korea, the trial will be carried out with 18 patients over a two-month period.

Previously, Engensis received orphan drug status and fast track designation for ALS by the FDA.

The clinical study in the US is expected to be completed in December 2022. Meanwhile, Helixmith plans to conduct an expanded Phase IIb trial to continue developing the gene therapy.