The Ministry of Food and Drug Safety has granted approval for a phase 2a clinical trial of Engensis, an investigational gene therapy for treating amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, under development by South Korean biotechnology company Helixmith, the company said Thursday.
Engensis is a gene therapy that utilizes a plasmid, a small circular DNA molecule, 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 phase 2a clinical study here 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, according to the company. Six to nine patients will be selected in South Korea, while the remaining will be from the US. The company added that Hanyang University Hospital will cooperate in the clinical trial.
Helixmith expects the South Korean government’s approval will allow the company to acquire a wider range of clinical data. Helixmith said the company would send the clinical data collected here to the US Food and Drug Administration to speed up the authorization process.
Previously, Engensis received orphan drug status and fast track designation for ALS by the US FDA. The clinical study in the US is expected to be completed in December 2022. Helixmith plans to conduct an expanded phase 2b trial to continue developing the gene therapy.
In the US, there are around 30,000 people with ALS, a disease characterized by the progressive loss of motor neurons, the nerve cells that control voluntary muscles, making voluntary motion impossible. Eventually, the muscles involved in breathing stop working, leading to the patient’s death.
In South Korea, the number of patients with ALS is estimated at around 2,500-3,000, according to the Korean ALS Association.
By Shim Woo-hyun (email@example.com