Helixmith "EngineSys Completes 6-Month Follow-Up Visits for All Patients"

[Asia Economy Reporter Hyunseok Yoo] Helixmith announced on the 28th that all patients in the domestic clinical Phase 1/2a trial of the gene therapy ‘Engensis (VM202)’ for Charcot-Marie-Tooth disease (CMT) have completed treatment and observation at 6 months. Based on this, the company plans to disclose the results of the CMT clinical Phase 1/2a trial through a topline announcement in the fourth quarter.

The CMT Phase 1/2a clinical trial was conducted at Samsung Medical Center with a total of 12 CMT patients. Clinical participants received a total of 4 doses of Engensis up to 3 months, followed by the first follow-up visit at 6 months and the second follow-up visit at 9 months. Helixmith completed the first patient dosing in September last year, the last dosing of the 12th patient at the end of March, and recently completed the 6-month follow-up visits for all clinical participants.

This CMT Phase 1/2a trial is led by Professor Choi Byung-ok of Samsung Medical Center, the top authority on CMT in Korea. If positive results are obtained in this domestic trial, the company plans to conduct global clinical trials in the United States, Europe, and other regions.

CMT is a disease characterized by progressive damage to motor and sensory nerves, leading to muscle atrophy in the arms and legs and gait disturbances. There are various types of CMT. The most common type, which is the subject of this clinical trial, is CMT1A, with approximately 8,000 patients in Korea and over 1.2 million patients worldwide. CMT is classified as a rare disease. With a prevalence of 1 in 2,500 people, it is the rare disease with the highest number of patients. However, there is currently no FDA-approved treatment.

Helixmith expects that Engensis, administered via intramuscular injection, will act to promote muscle regeneration, inhibit apoptosis, reduce inflammation and fibrosis, and provide neuroprotection, thereby slowing or halting disease progression.

Kim Sun-young, CEO of Helixmith, stated, “CMT is a disease for which there is no FDA-approved treatment worldwide. Since Engensis has been recognized by the U.S. FDA for safety and efficacy in diabetic peripheral neuropathy (DPN) and amyotrophic lateral sclerosis (ALS), we expect positive results in CMT patients as well.”

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