Paper Reporting Results from Helixmith's Phase 3 Gene Therapy Trial for Painful Diabetic Neuropathy was One of the Top-10 Most-Downloaded Articles in Clinical and Translational Science

SAN DIEGO, March 14, 2022 /PRNewswire/ -- Helixmith announced today that their publication, "Gene therapy for diabetic neuropathy: A randomized, placebo-controlled phase III study of VM202, a plasmid DNA encoding human hepatocyte growth factor," was one of the top-10 most-downloaded articles in Clinical and Translational Science (CTS) in 2021. CTS is the official publication of the American Society for Clinical Pharmacology & Therapeutics (ASCPT) and highlights original research that helps bridge laboratory discoveries with the diagnosis and treatment of human diseases. Dr. John Kessler, Professor of Neurology at Northwestern was the lead author on this study. This was the first gene therapy phase 3 that has ever been done for pain. According to Alethea Gerding, Managing Editor, ASCPT, "The article has been downloaded more than 3,000 times, meaning that it has reached a wide-ranging worldwide readership. The primary goal of CTS is to become the beacon of translational science, and articles such as yours clearly show the value of translational science."

In the paper, VM202 (donaperminogene seltoplasmid), a plasmid DNA encoding the human HGF (hepatocyte growth factor) gene, developed by Helixmith Co. Ltd, authors reported that in VMDN-003b, intramuscular injections of VM202 provided pain reduction for more than 8 months after the last cycle of treatment and that the safety and tolerability was highly favorable, consistent with previous studies. One of the most clinically important findings in both VMDN-003b and the phase II study is that VM202 was more effective in subjects not on pregabalin or gabapentin, two of the most prescribed medicine in the DPN field.

These results have important clinical implications as more than 4.2 million people in the US are known to suffer from painful DPN and nearly 1.3 million patients are considered to be refractory, meaning currently available medications do not work for them (Painful Diabetic Neuropathy, GlobalData 2018).

Helixmith launched a second phase 3 trial for DPN, REGAiN-1A (VMDN-003-2), in the US and are targeting release of top line results by the end of 2022. The company is planning to start a third phase 3 for DPN in the second half of 2022.

Key points of the CTS paper

• VM202 (donaperminogene seltoplasmid) is a first-in-class, proprietary, non-viral, potentially regenerative plasmid DNA gene therapy, from a non-clinical animal perspective.
• The Phase 3 study for painful DPN was conducted in two parts, one for 9 months (VMDN-003; five hundred subjects) and one with a 3-month extension to 12 months (VMDN-003b; 101 subjects).
• Safety and tolerability of VM202 continues to appear highly favorable, consistent with previous studies.
• These results have important clinical implications as more than 4.2 million people in the US are known to suffer from painful DPN and nearly 1.3 million patients to be refractory in that currently available medicines do not work for them.

About Diabetic Peripheral Neuropathy

Painful DPN is a common and debilitating complication of diabetes mellitus that has a profound negative impact on quality of life, sleep, and mood. Current therapies are palliative and do not target the mechanisms underlying painful DPN. Moreover, symptomatic relief is often limited, and many patients with painful DPN still use opioids.

About Helixmith Co., Ltd.

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

SOURCE Helixmith USA Inc.