According to Charcot-Marie-Tooth News, biotechnology company Helixmith recently began a Phase 1/2a clinical trial to test the efficacy of Engensis (VM202) for patients with Charcot-Marie-Tooth disease type 1A (CMT1A). The trial, which will explore the gene therapy solution, takes place in Korea. It is currently enrolling patients and was approved just a few months ago by the Ministry of Food and Drug Safety.

The clinical trial will treat 12 patients with CMT1A. During the trial, patients will receive Engensis directly in their leg muscles for 270 days (38.5 weeks). Researchers will track safety, tolerability, efficacy, and pain reduction. Engensis uses plasmids, a type of DNA molecule, to deliver hepatocyte growth factor (HGF) to cells. Ultimately, this could promote muscle tissue development and strength.

In addition to CMT1A, Engensis is a potential solution for heart disease, diabetic peripheral neuropathy (DPN), and amyotrophic lateral sclerosis (ALS). The drug received Regenerative Medicine Advanced Therapy designation from the FDA. According to the FDA, this status is given to drugs if:

the drug is a regenerative medicine therapy (cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination); the drug is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and preliminary clinical evidence indicates that the drug has the potential to address unmet medical needs.

Charcot-Marie-Tooth Disease (CMT1A)

Although it is considered a rare disease, Charcot-Marie-Tooth disease (CMT) is also one of the most common genetic neurological disorders. Also known as hereditary motor and sensory neuropathy, CMT results from CMT gene mutations.  These gene mutations cause peripheral nerve degeneration, preventing nerves and muscles from communicating. Different mutations cause particular subtypes. For example, CMT1A results from PMP22 gene duplication. Around two-thirds of all CMT cases are CMT1A. Over 1 million patients globally have CMT.

Symptom onset usually occurs in adolescence or early adulthood. However, for patients with CMT1A, symptoms may even appear in infancy. Symptoms include:

• Lower leg deformities
• Difficulty walking or running
• Hand atrophy and weakness
• Foot drop
• Foot and leg muscle weakness
• Decreased reflexes
• High foot arches
• Poor balance

Learn more about Charcot-Marie-Tooth disease.