VM202 CMT Phase I/IIA Trial Launched

작성자 helixmith 작성일 20-07-27 16:20


A phase I/IIA trial to treat patients with Charcot-Marie Tooth (CMT) disease has been launched in Korea using Engensis (VM202), Helixmith’s flagship gene therapy product.

In April, Helixmith’s clinical trial plan for a phase I/IIAstudy in CMT1A patients was approved by the Ministry of Food and Drug Safety (MFDS), the FDA of Korea. The study was officially initiated at the Samsung Medical Center with the recent approval from their Institutional Review Board (IRB).

The trial aims to evaluate Engensis’ safety and tolerability by administering the treatment to the leg of twelve CMT1A patients in Korea. The study will be conducted over 270 days and be supervised by a leading expert in CMT diseases, Professor Choi Byung-ok.

In the trial, Engensis will be administered to patients who suffer from muscle loss caused by peripheral nerve damage from CMT disease. The primary efficacy endpoint will evaluate the safety and tolerability of Engensis in patients.

CMT is a disease which 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 from an anomaly in the PMP22 gene 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 by the US FDA yet, and currently, only adjuvant therapies are available to patients.

Engensis is a gene therapy that uses plasmid DNA to express hepatocyte growth factor (HGF). Over the past 15 years of research and clinical trials, it has been proven 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.

“CMT is similar to diabetic peripheral neuropathy (DPN) in the sense that both diseases are caused by peripheral nerve damage,” commented Sunyoung Kim, CEO of Helixmith. “We hope that Engensis, which has been granted RMAT designation by the US FDA and recognized for its potential as a regenerative treatment for DPN, will also be able to provide relief to patients who suffer from this rare disease."