Helixmith Receives Approval for 'Enzencis' Phase 2a Clinical Trial for ALS in South Korea

[Asia Economy Reporter Kim Ji-hee] Helixmith announced on the 17th that it has received approval from the Ministry of Food and Drug Safety for a domestic Phase 2a clinical trial plan to treat the rare genetic disease Amyotrophic Lateral Sclerosis (ALS) using its gene therapy candidate 'Engensis (VM202)'.

This trial is the same as the ongoing Phase 2a clinical trial in the United States. With this approval, clinical data from Korea will also be included in the U.S. approval process.

The ALS Phase 2a clinical trial will be conducted both in the U.S. and Korea. The first patient dosing in the U.S. took place in March, and the trial is ongoing at four clinical trial centers. In Korea, Hanyang University Hospital is participating in the trial. A total of 18 patients will be enrolled, with approximately 6 to 9 expected to be registered in Korea. Helixmith plans to proceed with a Phase 2b clinical trial of a scale sufficient to secure statistical power based on the results after completing the ALS Phase 2a trials in both the U.S. and Korea.

The ALS Phase 2 clinical trial for Engensis is divided into two stages, 2a and 2b, to investigate the drug's safety and efficacy. In this trial, the drug will be administered to patients three times at two-month intervals. For each administration, two injections will be given at two-week intervals. Specifically, injections will be administered on days 0 and 14, days 60 and 74, and days 120 and 134. According to the company, this represents the highest frequency and dosage of Engensis used to date.

The primary endpoint is the safety of the current method, investigating the occurrence of serious adverse events during the clinical trial period. Efficacy endpoints will also be measured.

Kim Sun-young, CEO of Helixmith, stated, “If the safety and efficacy of Engensis are proven in the ALS Phase 2a clinical trials conducted in the U.S. and Korea, it will have a significant impact not only on the ALS market but also on the company's value.”