First Pivotal Gene Therapy Trial to Target Underlying Cause of Diabetic Peripheral Neuropathy

ATLANTA, GA, USA I June 27, 2016 I VM BioPharma, the United States division of ViroMed Co., Ltd. in Seoul, Korea (084990.KQ), today announced the first patient was dosed in the recently initiated Phase 3 clinical study evaluating VM202, a proprietary DNA based biopharmaceutical, in patients with painful diabetic peripheral neuropathy (DPN). This is the first pivotal gene therapy trial specifically targeting the most common cause of severe neuropathy, and follows the successful completion of a Phase 2 trial conducted at Northwestern University and multinational sites in the U.S. and Korea.

“The initiation of a pivotal clinical trial for VM202 is incredibly exciting, because we observed in the Phase 2 trial a rapid and significant reduction in DPN pain, along with signals that VM202 may elicit a disease-modifying effect,” said Dr. Jack Kessler, M.D., professor of neurology at Northwestern University’s Feinberg School of Medicine and the principal investigator of the Phase 3 study. “Current treatments for DPN are aimed at providing symptom management, and along with a high rate of patient failure, do not modify the underlying pathology of the condition.”

DPN is a common complication of diabetes in which nerve damage results in sudden and severe pain. VM202 is a plasmid DNA that contains the human hepatocyte growth factor (HGF) gene, which in vivo produces two isoforms of HGF proteins that are naturally found in the human body. HGF is a growth factor that induces angiogenesis and acts as a neurotrophic factor to peripheral nervous system. After VM202 is injected into a patient’s muscle, it is taken up by a cell and produces the HGF proteins, which are then released from the cell and may induce new blood vessel formation by activating various signaling pathways. In this way, VM202 is believed to promote microvasculature and regenerate nerve cells, providing clinical benefit to patients with DPN.

“This milestone marks an exciting time for VM BioPharma, and we are pleased to be a part of such revolutionary research taking place in diabetic peripheral neuropathy, and other disease areas with high unmet needs,” said Dr. Sunyoung Kim, chief scientific officer of ViroMed Co., Ltd. “This is the first Phase 3 study to evaluate the use of gene therapy as a way to nourish and recover damaged nerve cells, and we are looking forward to providing pivotal data that will help us better understand the potential of this novel approach that could improve the quality of life for the millions of patients who suffer from this debilitating condition.”

Study Design

The nine month, Phase 3, double-blind, randomized, placebo-controlled, multicenter study is designed to assess the safety and efficacy of VM202 in 477 adult patients with painful diabetic peripheral neuropathy. Patients will be randomized in a 2:1 ratio to either VM202 (n=318) or placebo (n=159) and will be stratified by current use of gabapentin and/or pregabalin. The primary clinical efficacy outcome will be the change in average pain score from baseline to the 3 month follow-up visit, as well as a 50% responder rate. For more information on this study, please visit and reference Identifier NCT02427464.

About Diabetic Peripheral Neuropathy (DPN)

DPN is a neuronal disorder caused by abnormally high level of glucose in diabetes patients, mostly occurring in lower limbs. The high level of glucose can be toxic and damages microvasculature of the diabetes patients, which in turn damages nerve cells due to lack of supply of nutrients to these cells. These damaged nerve cells send out abnormal signals, causing the patients to feel abnormal pains that progresses with the disease. Patients with painful DPN, the most extreme form of the disease, feel extreme pain with a slight graze or touch. There are currently no fundamental treatments for the disease and maintaining glucose level in blood through strict diet is the only known method to diminish the disease incidence. The disease occurs in 5 ~ 20 % of diabetes patients (worldwide: 387 million, U.S.: 26 million). [International Diabetes Federation, 2014]

About VM202

VM202 is a proprietary gene therapy from VM BioPhrama targeting four different indications. When injected into patients, VM202 produces hepatocyte growth factor (HGF) protein, which induces angiogenesis and acts as a neurotrophic factor, leading to the formation of new microvasculature and induces regeneration of nerve cells. The results from a Phase 2 clinical study showed the possibility of VM202 as a new concept drug for the treatment of DPN (Annals of Clinical and Translational Neurology 2015, Volume 2, Issue 5,  465–478). [;jsessionid=5E9494D0A82AD459C41E3651101DF08F.f02t02]

VM202 also completed a successful Phase 2 study for of critical limb ischemia in the U.S. (Gene Therapy 2011, 18: 788–794) [], and has successfully completed phase 1/2 study for amyotrophic lateral sclerosis (ALS) in the U.S. A Phase 2 trial is also planned for coronary artery disease in Korea.

About VM BioPharma and ViroMed Co., Ltd.

VM BioPharma is a U.S. 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 U.S., Korea, and China.

ViroMed has assembled a diverse yet technologically- and conceptually-linked pipeline. Other research areas include antibody-based cancer therapeutics, immune disorders, recombinant protein-based thrombocytopenia treatment, and CAR-T technology. With constant track record of clinical efficacy and quality molecular biological research, ViroMed aims to become the trailblazer in the field of gene therapy.

SOURCE: VM BioPharma