ENCell Completes High-Dose Cohort Dosing in Phase 1b Trial of EN001 for Charcot-Marie-Tooth Disease

Safety and Exploratory Therapeutic Effects of EN001 Confirmed in Low-Dose Cohort

ENCell, a company specializing in the development of advanced biopharmaceuticals, announced on May 19 that it has completed repeat dosing for the high-dose cohort in the Phase 1b clinical trial of EN001, targeting patients with Charcot-Marie-Tooth disease (CMT) type 1A.

Charcot-Marie-Tooth disease is a genetic disorder that causes deformities of the hands and feet and muscle atrophy, and in severe cases, can lead to loss of vision and hearing. Despite its relatively high prevalence among rare diseases, there are currently no approved treatments, making it a significant challenge for both patients and medical professionals.

The Phase 1b clinical trial of EN001 was designed to evaluate the safety and efficacy of repeated administration of EN001 in patients with CMT type 1A. The principal investigator is Professor Choi Byungok of the Department of Neurology at Samsung Medical Center. The first administration to the high-dose cohort began in December of last year. After administering the low-dose cohort (1.25 × 10⁶ cells/kg) to three patients and confirming that no dose limiting toxicity (DLT) occurred, the safety committee reviewed the results and proceeded with the high-dose cohort. The high-dose cohort, which is twice the dose of the low-dose group (2.5 × 10⁶ cells/kg), was also administered to three patients.

EN001 is a mesenchymal stem cell therapy developed using ENCell’s proprietary ENCT (ENCell Technology) platform. It is characterized by its ability to suppress cellular aging and secrete higher levels of therapeutic substances. This therapy migrates to damaged nerves, releases therapeutic substances, and promotes remyelination. In February, EN001 received Orphan Drug Designation from the U.S. FDA. Recently, patient associations for CMT around the world have increasingly inquired about the status of EN001 clinical trials, reflecting growing expectations for new treatments for rare diseases.

A representative from ENCell stated, "We have already confirmed the safety and exploratory therapeutic effects in the low-dose cohort, and with the completion of dosing in the high-dose cohort, we expect to observe similar effects. After confirming safety in the high-dose group, we will accelerate entry into the Phase 2a clinical trial so that we can provide timely treatment to CMT patients."

Meanwhile, in October of last year, ENCell announced the safety and exploratory therapeutic effects of the low-dose cohort in the repeat dosing clinical trial of EN001 for CMT type 1A patients. Three patients in the low-dose cohort received EN001 twice, and DLT was evaluated at eight weeks. As a result, no DLT was observed in any patient, and there were no serious adverse events or infusion-related reactions.

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