Helixmith Files Patent Application for Enzencis CMT Treatment

[Asia Economy Reporter Jang Hyowon] Helixmith announced on the 10th that it has filed a patent for treating Charcot-Marie-Tooth disease (CMT) using the gene therapy ‘Engensis (VM202)’. This patent filed domestically is expected to be followed by overseas patent applications soon.

CMT is a congenital peripheral neuropathy caused by problems in Schwann cells, which play an important role in nerve conduction. Progressive damage to motor and sensory nerves leads to muscle atrophy in the arms and legs and causes gait disturbances. Although classified as a rare disease, its prevalence is 1 in 2,500, making it one of the most common rare diseases in terms of patient numbers.

Meanwhile, Helixmith announced the topline results of the CMT Phase 1/2a clinical trial on the 9th. In the clinical trial, Engensis (VM202) showed trends of improvement in safety and key efficacy indicators. Improvements were observed in analyses of FDS (Functional Disability Scale), ONLS (Overall Neuropathy Limitation Scale), and CMTNS-v2 (CMT Neuropathy Score v2), and MRI revealed a reduction in muscle loss caused by neuropathy.

There is still no treatment for CMT worldwide, resulting in a very high unmet medical need. This clinical trial is the world’s first gene therapy clinical trial for CMT and has attracted significant attention from U.S. media and patient organizations. The renowned U.S. hereditary neuropathy foundation, Hereditary Neuropathy Foundation (HNF), distributed the CMT clinical trial results to stakeholders via newsletter.

Dr. Park Youngjoo, head of clinical development at Helixmith, said, “Through the clinical trial, we once again realized that the bioactivity of Engensis (VM202) can be used to treat many types of diseases,” adding, “This patent application will serve as an opportunity to strengthen the market competitiveness and exclusivity of Engensis (VM202) in the CMT field.”

This content was produced with the assistance of AI translation services.