South Korea’s Helixmith said on Thursday it has entered into a master services agreement with Worldwide Clinical Trials (WTC) to support site recruitment and activation for the company’s ongoing Phase 2 study of gene therapy Engensis (VM202) in the treatment of amyotrophic lateral sclerosis (ALS) in the U.S.

Shares of Kosdaq-listed Helixmith closed the day 4.6 percent higher at 50,000 won ($42.19) on the news.

WTC, a global CRO providing full-service drug development services infrastructure and talent spanning 60 countries, focuses on central nervous system, cardiovascular and metabolic disorders, general medicine, oncology and rare diseases with high unmet medical needs.

ALS is a fatal neurological disease that affects nerve cells in the brain and spinal cord, causing loss of muscle control. ALS often called Lou Gehrig`s disease causes progressive physical disabilities and leads to eventual death due to respiratory muscle failure.

The incidence of ALS is 1 per 20,000 each year and it affects about 30,000 people in the U.S. alone. Currently, there is no cure for ALS or effective treatment to stop the progression of the disease.

Engensis 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 excellent safety profile, according to the company.

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

By Pulse

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