The Engensis CMT study, led by Professor Choi Byung-ok at Samsung Medical Center in Seoul, aims to evaluate the safety and tolerability of Engensis in 12 CMT patients. If it secures positive safety data from the current local study, Helixmith plans to conduct additional trials in the US and Europe.
CMT is a disease that gradually damages motor neurons and sensory neurons in arms and legs, causing muscle weakness and walking disorders. CMT1A, the most common type of the disease, occurs due to PMP22 gene duplication and accounts for about 40% of all CMT patients, with approximately 8,000 patients in Korea and more than 1.2 million worldwide. Despite such prevalence, no curative treatment has been approved worldwide, and only adjuvant therapies are currently 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 dystrophy prevention. In the ongoing CMT1A trial, Engensis is injected into the leg muscles of patients who suffer from muscle loss caused by peripheral nerve damage from CMT disease.
“Engensis represents a truly novel approach to treating neuromuscular diseases. We hope that it will provide a treatment option to patients who suffer from CMT," commented Sunyoung Kim, CEO of Helixmith.
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 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.
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.