South Korea’s Helixmith said on Wednesday it has started a Phase 2 study of gene therapy Engensis (VM202) in the treatment of amyotrophic lateral sclerosis (ALS), often called Lou Gehrig`s disease, in the U.S.

Clinical study details were already registered with ClinicalTrials.gov.

Shares of Kosdaq-listed Helixmith gained 8.45 to close at 23,100 won ($20.87).

The investigational drug is a plasmid-based gene therapy to deliver the hepatocyte growth factor (HGF) directly to patient cells, capable of regenerating microvasculature and nerve cells and preventing muscular atrophy with an excellent safety profile, according to the company.

The latest clinical study divided into 2a and 2b stages aims to evaluate the safety and efficacy of the drug. The Phase 2a trial involving 18 patients with Lou Gehrig`s disease will be conducted for 180 days to measure the level of gene expression with a muscle tissue sample from the patients after Engensis injection. The Phase 2a will be followed by Phase 2b which will involve more than 100 patients.

In 2016, the FDA recognized the potential for Engensis to meet the unmet need for this condition by granting it orphan drug designation plus a fast-track regulatory review for marketing approval.

The cause of most ALS is still unknown, but there are only two drugs available whose effect remains limited, said Helixmith CEO Kim Sun-young, adding that Engensis, if proven effective in the ongoing trials, will represent one more step towards a new treatment option available in the market.

By Park Yoon-gyun and Minu Kim

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