Helixmith Completes Dosing for Enzygen CMT Phase 1 and 2a Clinical Trials in Korea

Disclosure of Phase 1 and 2a Clinical Trial Results After Q4

[Asia Economy Reporter Kim Ji-hee] Helixmith announced on the 5th that it has completed the final patient dosing in the domestic Phase 1/2a clinical trial of its gene therapy ‘Engensis’ for Charcot-Marie-Tooth disease (CMT). The company plans to disclose the results of the CMT Phase 1/2a trial through a topline announcement after the fourth quarter.

The Phase 1/2a trial was conducted at Samsung Medical Center with a total of 12 CMT patients. After the first patient dosing in September last year, the final dosing of the 12th patient was completed at the end of March. The clinical trial is led by Professor Choi Byung-ok, the top authority on CMT in Korea at Samsung Medical Center. 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 several types of CMT, among which CMT1A, the subject of this clinical trial, is the most common. It is known that there are about 8,000 patients in Korea and more than 1.2 million patients worldwide. Although CMT is classified as a rare disease, its prevalence is 1 in 2,500, making it the rare disease with the largest number of patients. However, there is currently no treatment approved by the U.S. Food and Drug Administration (FDA).

Helixmith expects that Engensis, administered via intramuscular injection, can slow or halt disease progression through mechanisms such as muscle regeneration, inhibition of apoptosis, anti-inflammation, anti-fibrosis, and neuroprotection.

Kim Sun-young, CEO of Helixmith, said, “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.