Helixmith has reported topline data from a Phase IIA clinical trial of Engensis (VM202) in people with amyotrophic lateral sclerosis (ALS). 

The double-blind, placebo-controlled, multicentre trial was carried out at five sites, with four in the US and one in Korea.

It enrolled 18 participants, who were randomised into a 2:1 ratio to receive either Engensis or a placebo. 

In the trial, three treatments of Engensis or a placebo were administered in the upper and lower limbs on months zero, two, and four.

A treatment comprised two cycles of injections of 64mg Engensis or a placebo given at a gap of two weeks. 

According to the trial data, Engensis was found to be safe and well tolerated. 

No variations between the Engensis and the placebo arms in the frequency of treatment-emergent adverse events (TEAEs) were reported. 

50% of the subjects in the Engensis arm had injection site reactions versus 66.7% in the placebo group. 

These findings indicated that a high dose and repeated treatments with Engensis were safe and well tolerated in trial subjects, providing enhanced flexibility in developing dosing schemes for clinical trials in the future.

The company could not compare the efficacy between the Engensis and the placebo arms, as the size of the trial was small and four subjects dropped out early.

An advanced pipeline candidate of the company, Engensis is a plasmid deoxyribonucleic acid (DNA) therapy encoding human HGF.

This gene therapy is administered intramuscularly.

As part of the trial, muscle biopsy samples were obtained from trial subjects and will undergo histological and molecular biological assessments using RNA-Seq.

A fatal neurodegenerative ailment, ALS impacts upper and lower motor neurons. It leads to progressive paralysis and death eventually because of respiratory failure.

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