Helixmith Announces Phase 3 Study Results of Novel Gene Therapy Treatment for Diabetic Foot Ulcers at 2021 Annual Meeting of Diabetic Foot Ulcer Conference (DFCon)


Data indicate that gene therapy appears to be effective, particularly in neuroischemic ulcers

Published: Oct. 22, 2021 at 4:00 AM MDT|Updated: 1 hour ago

SAN DIEGO, Oct. 22, 2021 /PRNewswire/ — Helixmith, a gene therapy company based in Seoul, Korea and San Diego, CA, announced today the results of a Phase 3 study for the treatment of diabetic foot ulcers with their novel gene therapy VM202 (Engensis) at the 2021 annual meeting of the Diabetic Foot Conference (DFCon) held in San Francisco and virtually. The study, “Gene Therapy for Diabetic Foot Ulcers: Analysis of a Randomized, Placebo-Controlled Phase 3 Study of Engensis (VM202), a Plasmid DNA Expressing Two Isoforms of Human Hepatocyte Growth Factor (HGF),” demonstrated a positive trend toward wound closure, potential healing effects and an acceptable safety profile. This is the first study using gene therapy for the treatment of diabetic foot ulcers. DFCon is the premier international, interdisciplinary diabetic foot conference in North America.

The purpose of the un-prespecified interim analysis was to evaluate the status of a 7-month Phase 3 study conducted to test the effect of intramuscular injection of Engensis into the calf muscles of participants having chronic nonhealing diabetic foot ulcers (DFU) with concomitant peripheral artery disease. In the interim ITT population (n=44), there was a positive trend toward wound closure in the VM202 group from month 3 to month 7. Ulcer closure effects were prominent, particularly in neuroischemic ulcer. In 23 patients having this type of foot ulcers, the percentage of subjects reaching complete ulcer closure was significantly higher in the VM202 group at months 3, 4 and 5 (p = 0.0391, 0.0391, and 0.0361, respectively). Engensis seems to also improve hemodynamic features; a potentially clinically meaningful 0.15 increase in ABI was observed in the VM202 group at day 210 in ITT population (p=0.0776). The company believes that intramuscular injections of VM202 plasmid DNA to calf muscle may have promise in treatment of chronic neuroischemic DFUs.

“Diabetic foot ulcers are one of the most serious complications associated with diabetes, contributing to high levels of morbidity, mortality, and health-care costs in this population, and there have been limited treatment options to date,” said David G. Armstrong, DPM, MD, PhD Principal Investigator and Professor of Surgery, Co-Director, USC Limb Preservation Program, Keck School of Medicine of University of Southern California (USC). “Given the safety profile and potential healing effects we identified in this study, continuing a larger DFU study is warranted with iterations of the current protocol and expansion of the number of sites.”

About Diabetic Foot Ulcers

Diabetes mellitus affects more than 451 million people worldwide and is predicted to rise to nearly 700 million people by 2045. Persons with diabetes mellitus have a 19-34% chance of developing a diabetic foot ulcer (DFU) during their lifetime with a recurrence rate as high as 50-70% within 5 years.

Despite the serious nature of DFUs, current treatment methods are limited. The standard therapy (ST) for DFUs includes debridement, dressing, offloading, vascular assessment, and infection and glycemic control. However, complete healing rates are reported to be low – 24% and 31% at 12 and 20 weeks, respectively, for those receiving ST. Many patients progress to more serious stages, which may include gangrene, amputation, and when combined with peripheral artery problems, critical limb ischemia (CLI). Unmet medical need is indeed high for patients with DFUs. A series of adjuvant therapies have been investigated, including acellular matrix therapy, hyperbaric oxygen therapy, and shockwave therapy. However, the clinical effectiveness of these treatments has yet to be demonstrated.

About VM202 (Engensis)

Engensis (VM202) is an innovative gene therapy drug that provides fundamental treatment through tissue regeneration. Built on 20 years of experience in gene therapy, Engensis is designed to overcome the limitations of previous DNA plasmid candidates by expressing therapeutic levels of protein and inducing durable therapeutic effects. Helixmith’s non-viral plasmid DNA product, Engensis, is designed to express recombinant HGF protein in nerve and Schwann cells to promote nerve system regeneration and induce the formation of microvascular blood vessels. HGF has a short half-life (5 minutes or less) and is quickly removed from the body by the liver, creating an obstacle to effective treatment with previous injectable recombinant HGF protein products.

A single injection of Helixmith’s proprietary plasmid DNA product expresses the HGF gene at levels 30-40 times higher than conventional plasmid DNA and provides sustained gene expression in mouse models for 2 weeks, with peak protein expression at Day 7 and a gradual decrease over the next week To date, more than 500 patients have been treated with Engensis across ten clinical trials in six different diseases and conditions. Data from previous clinical studies suggest that Engensis is well tolerated and has the potential to provide durable analgesic and/or symptomatic relief in a variety of disease settings. Beyond potentially alleviating pain, Engensis is designed to target the underlying causes of neuropathy through its predicted angiogenic and neuroregenerative properties. The US FDA recognized the potential for Engensis to meet the unmet need for this condition in 2018 by designating it as a Regenerative Medicine Advanced Therapy (RMAT) for the treatment of painful diabetic peripheral neuropathy, making it the first RMAT-designated gene therapy for a prevalent disease with over one million patients. This designation grants all the benefits afforded by the fast track and breakthrough designations, including priority review, to Engensis.

About Helixmith

Helixmith is a gene therapy company headquartered in Seoul, Korea, developing new and innovative biopharmaceuticals to address previously untreated diseases, and is listed on the KOSDAQ. The company has an extensive gene therapy pipeline, including a CAR-T program targeting several different types of solid tumors and an AAV vector program targeting neuromuscular diseases. Engensis (VM202), the most advanced pipeline candidate, is a plasmid DNA therapy being studied for painful diabetic peripheral neuropathy, diabetic foot ulcers, claudication, amyotrophic lateral sclerosis, coronary artery disease, and Charcot-Marie-Tooth disease.

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SOURCE Helixmith USA Inc.

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