Korean-made stem cell therapy EN001 enters phase 2a trial for Charcot–Marie–Tooth disease

A clinical trial of allogeneic umbilical cord-derived mesenchymal stem cell therapy is underway in Korea for Charcot–Marie–Tooth disease (CMT) type 1A.

ENCell said Tuesday it obtained approval from the Ministry of Food and Drug Safety (MFDS) for a phase 2a clinical trial. The trial will test allogeneic umbilical cord-derived mesenchymal stem cells EN001, a CMT type 1A therapeutic candidate.

The approval follows the completion of the review and final approval of the combined phase 1b/2a clinical trial amendment application submitted to the MFDS last August.

Following clinical approval, EN001 will begin a phase 2a trial. This trial will examine the dose-response among three groups: placebo, low dose (1.25×10⁶ cells/kg), and high dose (2.5×10⁶ cells/kg), and assess efficacy versus placebo.

The primary endpoint is the change in the CMT Neuropathy Scale from baseline at week 24, with safety and tolerability concurrently evaluated.

Based on the repeated-dose safety and exploratory efficacy results from the phase 1b trial, ENCell plans to determine the optimal dose of allogeneic umbilical cord-derived mesenchymal stem cells EN001 and demonstrate definitive therapeutic efficacy in this clinical trial.

EN001 is a WJ-MSC therapeutic agent derived from Wharton's Jelly. It uses ENCell's proprietary cell culture platform, ENCT (ENCell Technology), which suppresses cellular senescence and increases therapeutic factor secretion for nerve regeneration and inflammation regulation. It aids functional recovery of damaged peripheral nerves.

ENCell is preparing to submit a treatment plan for CMT1A therapy under the Advanced Regenerative Medicine Regulatory Sandbox project. After completing the phase 2a clinical trial, the company will apply for accelerated review status, seeking conditional approval to enable early commercialization of this rare disease treatment.

“This MFDS approval marks EN001's entry into the phase 2a trial,” an ENCell official said. “We will confirm efficacy by comparing placebo and two dose groups. We are committed to ensuring CMT patients receive treatment benefits as quickly as possible through accelerated review.”