VM BioPharma has begun late-stage testing for its diabetic peripheral neuropathy gene therapy VM202 as it looks to take on Pfizer’s ($PFE) Lyrica.
The biotech--a U.S. division of Korea’s ViroMed--said today that the first patient has been dosed in its new Phase III study of VM202 in those 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 in the footsteps of its midstage trial, which showed a signal for being a disease-modifying treatment.
The big players in pain and DPN come in the form of Pfizer’s blockbuster pill Lyrica (pregabalin) and Neurontin (gabapentin), which are both taken orally, whereas VM202 in injected.
VM BioPharma is aiming to treat the underlying causes of the disease, however, whereas Lyrica and Neurontin, which also have a host of other pain-related indications, are licensed to treat the symptoms of DPN.
DPN is a common complication of diabetes in which nerve damage results in sudden and severe pain. As the rates of diabetes have increased dramatically over the years (with 387 million now estimated to have the disease worldwide, and 26 million of those being in the U.S.), the rates of DPN have also grown, which effect around 5-20% of all diabetes patients.
The drug works by inducing angiogenesis and acts as a neurotrophic factor, leading to the formation of new microvasculature and induces regeneration of nerve cells.
The 9-month, double-blind placebo-controlled 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 or placebo and will be stratified by current use of Neurontin/Lyrica. The primary endpoint will be the change in average pain score from baseline to the 3-month follow-up visit, as well as a 50% responder rate.
"The initiation of a pivotal clinical trial for VM202 is incredibly exciting, because we observed in the Phase II trial a rapid and significant reduction in DPN pain, along with signals that VM202 may elicit a disease-modifying effect," said Dr. Jack Kessler, professor of neurology at Northwestern University's Feinberg School of Medicine and the principal investigator of the Phase III 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."
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 the 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," added Dr. Sunyoung Kim, chief scientific officer of ViroMed.
"This is the first Phase III 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."
Sigi Caron, regulatory and clinical adviser to VM BioPharma, Caron & Associates, told FierceBiotech that for this specific trial, “we are hoping to see the top line data in the latter part of 2018 and for a regulatory filing by 2020.”
Caron added that the biotech is not planning for a head-to-head trial with Lyrica.
VM202 has also recently completed a successful Phase II study for critical limb ischemia in the U.S., and has successfully completed Phase I/II study for amyotrophic lateral sclerosis (ALS) in the U.S. A midstage trial is also planned for coronary artery disease in Korea.
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