VM202 is an investigational DNA-based therapy being developed by VM Biopharma (the U.S. branch of the Korean company ViroMed) for the treatment of amyotrophic lateral sclerosis (ALS). It is administered as an injection directly into the affected muscles.

VM202 is also being investigated for the treatment of coronary and peripheral artery disease, and is currently in a Phase 3 clinical trial for diabetic peripheral neuropathy.

How VM202 works

VM202 is a plasmid — a piece of circular DNA — that contains the instructions to produce more of a protein called hepatocyte growth factor (HGF). HGF has multiple roles in the body: it is involved in angiogenesis (the formation of new blood vessels) and is a neurotrophic factor or a chemical that improves the survival and growth of neurons (nerve cells).

In ALS patients, symptoms are caused by progressive damage to motor neurons, leading to a reduced ability to control muscles. Eventually, this can result in muscle paralysis that spreads through the body. As HGF can trigger the repair and growth of neurons, it is thought that by enhancing its production at the site of neurodegeneration (the muscles) it could slow or stop disease progression through motor neuron regeneration. Tests in mouse models of ALS showed that increasing HGF production in the nervous system can slow disease progression and increase life span.

VM202 in clinical trials

VM Biopharma carried out a Phase 1/2 clinical trial (NCT02039401) to assess the safety and tolerability of VM202 in 18 ALS patients. Trial participants received an intramuscular  injection of VM202 once weekly, over the course of four visits, and were monitored for adverse events for up to nine months. Patients were also assessed for factors such as how long VM202 remains in the blood, and the effect on HGF serum levels. Functional measures of ALS, such as the revised ALS Functional Rating Scale (ALSFRS-R), were used to assess whether VM202 could have an effect on the progression of ALS.

Study results were published in the scientific journal Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration in February 2017. Of the 18 patients, 17 completed the study. The treatment appeared to be well-tolerated, with no serious adverse events related to VM202 and the most common side effect being injection site reactions. The study also suggested that VM202 may have a clinical benefit, as the reduction in ALSFRS-R score was smaller than the reported average for ALS patients.

Other information

VM202 was granted orphan drug status in February 2014 and given fast track designation in May 2016 by the U.S. Food and Drug Administration (FDA). The FDA has approved an investigational new drug license for VM202, allowing for the start of Phase 2 clinical trials, where VM Biopharma intends to compare its safety and efficacy to a placebo in about 85 ALS patients.

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