Open label study to assess the safety of VM202 in subjects with amyotrophic lateral sclerosis

Abstract

Objective: To assess safety and define efficacy measures of hepatocyte growth factor (HGF) DNA plasmid, VM202, administered by intramuscular injections in patients with amyotrophic lateral sclerosis (ALS).

Methods: Eighteen participants were treated with VM202 administered in divided doses by injections alternating between the upper and lower limbs on d 0, 7, 14, and 21. Subjects were followed for nine months to evaluate possible adverse events. Functional outcome was assessed using the ALS Functional Rating Scale-Revised (ALSFRS-R) as well as by serially measuring muscle strength, muscle circumference, and forced vital capacity.

Results: Seventeen of 18 participants completed the study. All participants tolerated 64 mg of VM202 well with no serious adverse events (SAE) related to the drug. Twelve participants reported 26 mild or moderate injection site reactions. Three participants experienced five SAEs unrelated to VM202. One subject died from respiratory insufficiency secondary to ALS progression.

Conclusions: Multiple intramuscular injection of VM202 into the limbs appears safe in ALS subjects. Future trials with retreatment after three months will determine whether VM202 treatment alters the long-term course of ALS.

Keywords:

• Amyotrophic lateral sclerosis
• gene therapy
• hepatocyte growth factor
• VM202 DNA plasmid

Acknowledgements

We acknowledge the dedicated collaboration with Emile R. Mohler, University of Pennsylvania Health System; Robert C. Griggs University of Rochester School of Medicine; Tracie C, Collins, Kansas Public Health Department; J. Michael White, JM White Associates, who are DSMB members. This work was funded by ViroMed Co. Ltd. (Seoul, Korea) and Reyon Pharmaceuticals Co. Ltd (Seoul, Korea), and supported by grants from the Korea Health Industry Development Institute (Grant No. HI14C1043).

Declaration of interest

The authors report no conflicts of interest. The authors alone are responsible for the content and writing of this article.

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