VM BioPharma, the US division of ViroMed Co., Ltd., has announced that the scientific journal, Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, has published clinical safety and tolerability data on its lead investigational drug, VM202, a novel, Phase I/II gene therapy for the potential treatment of amyotrophic lateral sclerosis, or ALS. The Phase I/IIopen-label, single center study was designed to assess the safety and tolerability of intramuscular (IM) injections of VM202 in patients with ALS. Measures of the ALS Functional Rating Scale-Revised (ALSFRS-r) and other efficacy parameters were evaluated as secondary endpoints. In this study, eighteen patients with ALS were enrolled and treated with a total of 64 mg of VM202 delivered in divided doses by bilateral IM injections. Safety and outcome measures were evaluated during the nine-month follow-up period. Functional outcome was assessed using ALSFRS-r and by serially measuring muscle strength, muscle circumference and forced vital capacity. The collective results reported that 17 of 18 patients completed the study, and VM202 was well tolerated by all patients with no reported serious adverse events (SAE) related to the drug. The most common treatment-related adverse events were injection site reactions. The FDA has granted Orphan and Fast Track designations for VM202 for the potential treatment of ALS. The company has recently obtained Investigational New Drug (IND) approval from the FDA for a Phase II clinical trial of VM202 for ALS. The trial will assess 84 study participants with ALS and the objective of the trial is to test the safety and efficacy of VM202 versus placebo. Upon the injection of VM202, hepatocyte growth factor (HGF) proteins, which are responsible for angiogenesis (formation of new blood vessels) and peripheral nerve cell regeneration are produced at the injection site. The produced HGF proteins stimulate the adjacent muscle, endothelial, and vascular smooth muscle cells, which in turn drives new blood vessel formation and nerve cell regeneration. HGF has shown nerve cell protection and regeneration effects of therapeutic use in various neuropathic animal models. In ALS, there have been reports showing HGF acts directly on motor neuron cells, or regulates the activity of the adjacent neuron cells, that show therapeutic effects as well. Based on various studies, it is expected that VM202 can stimulate the regeneration of compromised motor neurons, and angiogenesis within deteriorated muscles. Further, it stimulates the regeneration of motor neurons and may arrest the pace of disease progression. VM202 is a proprietary gene therapy from VM BioPharma targeting four different indications. When injected into patients, VM202 produces hepatocyte growth factor (HGF) protein, which induces angiogenesis (formation of new blood vessels) and acts as a neurotrophic factor to stimulate regeneration of damaged nerve cells. VM202 completed the Phase I/IIstudy for amyotrophic lateral sclerosis (ALS) in the US, and the FDA has granted the drug Orphan status, Fast Track designation and IND approval for a Phase II study. In addition to ALS, ViroMed is advancing the study of VM202 and enrolling patients in a pivotal Phase III trial for painful diabetic neuropathy. The Company also completed a Phase II clinical study of VM202 for the treatment of critical limb ischemia, and has received an IND approval from the FDA to initiate a pivotal Phase III study targeting chronic non-healing ischemic foot ulcer in diabetic patients. A Phase II trial of VM202 has been approved for coronary artery disease in Korea.