ENCell Completes IND Application for EN001 Phase 2a Trial in Charcot-Marie-Tooth Disease Type 1A

Application for Integrated Phase 1b/2a Clinical Trial Amendment Completed

ENCell announced on August 18 that it has completed a request to the Ministry of Food and Drug Safety to amend its clinical trial protocol for a Phase 1b/2a study targeting patients with Charcot-Marie-Tooth disease (CMT) type 1A.

With this integrated clinical strategy, ENCell will be able to conduct its trials at a faster pace. In particular, the company expects to significantly reduce the time required to transition from Phase 1b to Phase 2a, thereby accelerating the overall pace of new drug development. The efficiency of the clinical trial process will also be enhanced. By evaluating both safety and efficacy within a single clinical trial protocol, ENCell can reduce unnecessary administrative procedures and resource waste, making this one of the most advantageous integrated clinical strategies for developing treatments for rare diseases, which typically involve high development costs.

Charcot-Marie-Tooth disease is a genetic disorder that causes deformities and muscle atrophy in the hands and feet, 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 healthcare professionals.

The Phase 1b study 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 from 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 observing no dose-limiting toxicity, the study proceeded to the high-dose cohort following approval from the safety review committee. The high-dose cohort, which received double the dose of the low-dose group (2.5 × 10⁶ cells/kg), also included three patients. Following the completion of dosing in this group and subsequent safety review committee approval, the Phase 1b stage confirmed positive results for safety, tolerability, and exploratory efficacy.

Through this integrated clinical trial, ENCell plans to determine the maximum tolerated dose (MTD) and the recommended dose for the Phase 2a trial based on the safety and tolerability of repeated EN001 administration in Phase 1b. In Phase 2a, the company will evaluate the efficacy of EN001 versus placebo by measuring changes in the CMT neuropathy score at 24 weeks compared to baseline.

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 helps regenerate nerve myelin. In February, EN001 was designated as an Orphan Drug by the U.S. FDA. Notably, since CMT is classified as a rare disease, ENCell can apply for conditional product approval after completing Phase 2 trials. Recently, patient advocacy groups for CMT around the world have shown increasing interest in the EN001 clinical trial and participation opportunities, reflecting growing expectations for new drugs targeting rare diseases.

An ENCell representative stated, "We have already confirmed the safety and exploratory therapeutic effects of EN001 in both low- and high-dose cohorts, which allowed us to complete the application to transition to this integrated clinical trial. As we move into Phase 1b/2a, we will do our utmost to conduct a faster and more efficient integrated trial, so that CMT patients can access this therapy in a timely manner."

Meanwhile, ENCell successfully completed a clinical study targeting CMT 1E patients last year and is now preparing to initiate advanced regenerative therapy in collaboration with Samsung Medical Center.

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