Helixmith Announces First Patient Enrolled in Phase 2 Study of Engensis (VM202) for Amyotrophic Lateral Sclerosis

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Helixmith announced today enrollment of the first patient in a new Phase 2A clinical trial, REViVALS-1A, targeting Amyotrophic Lateral Sclerosis (ALS) using Helixmith’s flagship gene therapy product Engensis (VM202).

“Engensis represents a truly novel approach to treating neuromuscular diseases. We hope that it will provide a treatment option to patients who suffer from ALS," commented Sunyoung Kim, CEO of Helixmith.

REViVALS-1A has a target enrollment of 18 ALS patients at approximately 4 clinical sites across the US. The first patient was enrolled by Yessar Hussain, MD at Austin Neuromuscular Center. Patients and healthcare providers seeking more information about the REViVALS-1A clinical trial can visit: https://www.clinicaltrials.gov/ct2/show/NCT04632225?term=Helixmith&cond=ALS&draw=2&rank=2.

The purpose of the Engensis ALS study is to evaluate the safety and tolerability of Engensis in 18 ALS patients. In addition, Engensis’ effect on muscle function and strength will be assessed using the Amyotrophic Lateral Sclerosis Function Rating (ALSFRS-R) and Hand-Held Dynamometry (HHD). Helixmith will also be collecting muscle biopsies to identify biomarkers for future research. If positive data is secured from the current study, Helixmith plans to conduct an expanded Phase 2B trial.
ALS is an adult-onset neurodegenerative disorder characterized by the loss of upper and lower motor neurons. The disease is diverse in its presentation, cause, and progression, but, in general, ALS presents clinically as asymmetric muscle weakness, wasting, spasticity, weight loss, dysphagia, and paralysis. Estimates of the incidence of ALS worldwide range from 1 to 3 cases per 100,000 people annually. The ALS Association, the Centers for Disease Control and Prevention, and the National Institute of Neurological Disorders and Stroke estimate that as many as 30,000 Americans may have the disease at any given time, with approximately 5,600 newly diagnosed patients annually.

Engensis is a gene therapy that uses plasmid DNA to express hepatocyte growth factor (HGF). Over 15 years of research and clinical trials have demonstrated that simple intramuscular injections of the drug can produce HGF proteins in the body, which can potentially be effective in nerve regeneration, angiogenesis, and muscle dystrophy prevention. In the ongoing REViVALS-1A trial, Engensis is injected into the arm and leg muscles of patients who suffer from ALS.

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. In clinical trials for patients with diabetic peripheral neuropathy, Engensis is well tolerated and has the potential to provide durable response 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 in diabetic peripheral neuropathy 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. Engensis also has been given orphan designation in ALS.

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, coronary artery disease, and Charcot-Marie-Tooth disease.

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Jennifer Guzman
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