Helixmith Announces First Patient Enrolled in Phase 3 Study of Engensis (VM202) for Diabetic Peripheral Neuropathy

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Helixmith announced today enrollment of the first patient in a second Phase 3 clinical trial targeting diabetic peripheral neuropathy (DPN), REGAiN-1A, using Helixmith’s flagship gene therapy product Engensis (VM202). REGAiN-1A has an initial target enrollment of 152 DPN patients at approximately 15 clinical sites across the US.

“Patients with DPN experience high levels of pain and discomfort, with no medicine available that treats the underlying disease,” says Miguel Trevino, MD, Medical Director, Innovative Research of West Florida. “REGAiN-1A will inform the ongoing clinical efforts to develop a new potentially regenerative medicine therapy.”

DPN is one of the most common complications of diabetes. Of approximately 30 million US adults with diabetes, up to 50% may develop DPN, and disabling neuropathic pain (painful DPN or PDPN) may affect approximately 25% of patients with diabetes (Shillo et al., 2019) . Painful DPN typically manifests as burning or stabbing pain in the lower extremities. Currently approved medications for PDPN are palliative, limited to managing existing pain, and have the potential for serious side effects.

The primary efficacy endpoint for the REGAiN-1A trial compares the change in average daily pain scores from the 7 days prior to the first injection on Day 0 to the 7 days prior to the six-month visit in participants treated with Engensis versus placebo. Secondary endpoints include the statistical significance of (1) reduction in worst pain at the end of a six-month period versus placebo, (2) the ratio of patients with an average pain reduction of 50% or more at the end of the six-month period versus placebo, and (3) safety of Engensis versus placebo.

“Engensis represents a truly novel approach to treating neuromuscular diseases, including DPN,” notes Dr. Sunyoung Kim, CEO and Head of Global Clinical Development at Helixmith. "This latest clinical study will add to the considerable body of knowledge and evidence we’ve gathered to support the optimal use of Engensis in patients needing treatment options. "

Patients and clinical investigators seeking more information about the REGAiN-1A Phase 3 Clinical Trial can visit

About Engensis

Helixmith's Engensis (VM202) is a gene therapy based on plasmid DNA. 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), 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 tackle previously untreated diseases, and is listed on 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 DPN, diabetic foot ulcers, claudication, amyotrophic lateral sclerosis (Phase 2 beginning in late 2020), coronary artery disease and Charcot-Marie-Tooth disease.

Helixmith clinical development and manufacturing activities are based in San Diego, California, where the company co-owns a cGMP-ready DNA production facility, Genopis, Inc., an affiliated CDMO also in San Diego. Genopis serves both Helixmith and external customers in need of plasmid DNA for medical purposes.

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Jennifer Guzman
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