Helixmith said it has kicked off the phase 2a global clinical trial of its gene therapy, Engensis, also known as VM202, which treats amyotrophic lateral sclerosis (ALS) patients.

The clinical trial team, led by Professor Kim Seung-hyun team at Hanyang University Seoul Hospital, has administered the dose of the investigational drug to an ALS clinical patient on Thursday, the company said. ALS phase 2a clinical trial is going on in the U.S. and Korea.

There are 18 patients in the Phase 2a clinical trial. The company expects to enroll 30-50 percent of them in Korea, the company said, adding that it plans to secure the safety and efficacy data through this clinical trial. After completing phase 2a clinical trials, the company intends to proceed with phase 2b clinical trials on a scale that can secure statistical power according to the results.

"Engensis represents a novel approach to neuromuscular disease," Helixmith CEO Kim Sun-young said.

By proving the safety and efficacy of Engensis for ALS in the phase 2a clinical trial in the U.S. and Korea, the company hopes that it will provide the possibility of a new treatment option for ALS patients who have no treatment so far, Kim added.

ALS is a fatal progressive neuromuscular disease, also called Lou Gehrig's disease.

ALS destroys motor neurons that control muscle movement, making all kinds of voluntary motions impossible, and eventually, the muscle involved in breathing stops and leads the patient to death. About 30,000 ALS patients are in the U.S. ALS is a special disease of great social and political interest that the U.S. Food and Drug Administration had lowered the regulatory bar to facilitate the development of ALS treatments, according to Helixmith.

Helixmith's Engensis is a drug composed of plasmid DNA expressing hepatocyte growth factor (HGF) protein. The drug had also received orphan drug and fast track designation for ALS from the FDA in the past.