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50% of Subjects Enrolled in Engensis Phase 1/2a Clinical Trial for Charcot-Marie-Tooth Disease

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  • 작성일 : 20-11-13 10:02
  • 조회 : 1,343회


November 9th, 2020 – Gene therapy developer Helixmith announced today that its Phase 1/2a study involving Korean patients with Charcot-Marie-Tooth disease (CMT) has passed 50% of its enrollment target. With seven patients enrolled, the company is projecting that all 12 subjects will be enrolled by the end of the year.

The trial aims to evaluate the safety and tolerability of Engensis (VM202) by injecting the treatment into the leg muscles of patients who suffer from muscle loss caused by peripheral nerve damage from CMT disease. The study is being conducted over 270 days and is supervised by Professor Choi Byung-ok, a leading expert in CMT at Samsung Medical Center. If positive results are obtained in this trial, the company plans to move forward with additional clinical trials in the US and Europe.

CMT is a hereditary peripheral neuropathy that gradually damages motor neurons and sensory neurons in arms and legs, causing muscle weakness and walking abnormalities. CMT1A, the most common type of the disease, affects approximately 8,000 patients in Korea and more than 1.2 million worldwide. CMT, despite being categorized as a rare disease, has a global prevalence of 1/2500. However, no curative treatment has been approved yet, with only adjuvant therapies available to patients.

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 atrophy prevention. 

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 type 1A version of CMT disease, or CMT1A, occurs due to problems in the myelin arising from an anomaly in the PMP22 gene. Preclinical studies have shown that Engensis acts on Schwann cells that make up myelin and is effective in remyelination, making it an ideal potential candidate for CMT treatment.

“There are still no FDA-approved treatments for CMT,” commented Sunyoung Kim, CEO of Helixmith. “We hope that Engensis will be able to provide relief to patients who suffer from this disease.”

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.

CONTACT: Jennifer Guzman
  Senior Director, Medical Affairs Strategy