The Phase 1/2 open-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 U.S. Food and Drug Administration (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 2 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.
"We are pleased to announce that these VM202 data have been published in a prestigious, peer-reviewed journal dedicated to reporting significant advances in the treatment of ALS, where significant unmet needs largely remain," said Dr. Sunyoung Kim, chief scientific officer at ViroMed Co., Ltd. "The results from this trial will help inform our future research efforts surrounding the potential clinical effectiveness of VM202 in the treatment of ALS. VM202 continues to show promise in a variety of disease states, including painful diabetic neuropathy, critical limb ischemia and coronary artery disease, with multiple Phase 2 and pivotal Phase 3 trials to further investigate the product underway."
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.
About Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's disease, is a rare neurodegenerative disease with unknown causes where the motor neurons necessary to move muscles in the body are destroyed, leading to paralysis of all muscles, such as those in the tongue, neck, and the limbs. The disease occurs mostly in adults and average life expectancy is 2-5 years from the onset of symptoms.i According to the National Institutes of Health (NIH), the prevalence of ALS in the US is 3.9 cases per 100,000 personsii, and there are currently an estimated 450,000 patients with ALS around the world.iii With roughly more than 20,000 - 30,000 patients in the US currently, approximately 6,000 new patients are diagnosed with the disease each year.iv,v
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 successfully completed the Phase 1/2 study 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 2 study.
In addition to ALS, ViroMed is advancing the study of VM202 and enrolling patients in a pivotal Phase 3 trial for painful diabetic neuropathy. The Company also completed a Phase 2 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 3 study targeting chronic non-healing ischemic foot ulcer in diabetic patients. A Phase 2 trial of VM202 has been approved for coronary artery disease in Korea.
About VM BioPharma and ViroMed Co., Ltd.
VM BioPharma is a US division of ViroMed Co., Ltd., an R&D focused biopharmaceutical company founded in 1996 and based in Seoul, Korea. ViroMed is developing new and innovative biopharmaceuticals for the treatment of currently untreatable diseases. The current development focus is on the proprietary plasmid DNA-based drug, VM202 in cardiovascular and neurological diseases at various clinical stages in US, Korea, and China.
ViroMed has assembled a diverse yet technologically and conceptually linked pipeline. Other research areas include therapeutic DNA vaccine for cancer, recombinant protein-based thrombocytopenia treatment, and CAR-T technology. With a constant track record of clinical efficacy and quality molecular biological research, ViroMed aims to become a trailblazer in the field of gene therapy.
VM BioPharma in Atlanta, USA
Sheila Yi, +1-404-355-5434
ViroMed Co., Ltd. (Headquarters) in Seoul, Korea
Seungshin Yu, +82-2-2102-7277
i ALS Association, Facts You Should Know; last accessed October 4, 2016: http://www.alsa.org/about-als/facts-you-should-know.html
ii Amyotrophic Lateral Sclerosis (ALS) "Fact Sheet", NINDS, Publication date June 2013; last accessed October 4, 2016: http://www.ninds.nih.gov/disorders/amyotrophiclateralsclerosis/detail_ALS.htm
iii ALS Therapy Development Institute; ALS FAQ; last accessed October 4, 2016: http://www.alstdi.org/about-als-tdi/als-faq/
iv ALS Association, Facts You Should Know; last accessed October 4, 2016: http://www.alsa.org/about-als/facts-you-should-know.html
v ALS Therapy Development Institute; ALS FAQ; last accessed October 4, 2016: http://www.alstdi.org/about-als-tdi/als-faq/
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SOURCE VM BioPharma