ENCELL, 'EN001' Charcot-Marie-Tooth Disease Type 1A Clinical Phase 2a Initiated

[Financial News] ENCELL announced on the 18th that it has completed the application for a change to clinical phase 1b/2a to the Ministry of Food and Drug Safety for patients with Charcot-Marie-Tooth disease (CMT) type 1A, and has officially begun integrated clinical trials. With this integrated clinical strategy, ENCELL can now conduct clinical trials with more speed. In particular, the time required to transition from phase 1b to phase 2a can be significantly reduced, thereby increasing the overall speed of new drug development.
Additionally, the efficiency of the clinical trial is enhanced. By evaluating safety and efficacy simultaneously within a single clinical trial protocol, unnecessary administrative procedures and resource waste can be reduced, making it one of the advantageous integrated clinical strategies for developing treatments for rare diseases with high development cost burdens.
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 being a rare disease with a high incidence rate, there is currently no approved treatment, posing a significant challenge for both patients and medical professionals.
The EN001 clinical phase 1b was designed to evaluate the safety and efficacy of repeated administration of EN001 to CMT type 1A patients, with Professor Choi Byung-ok of the Neurology Department at Samsung Seoul Hospital as the principal investigator.
The first administration to the high-dose group patients began in December last year, and after administering to three patients in the low-dose group (1.25 × 10^6 cells/kg) without any occurrence of dose-limiting toxicity (DLT), the high-dose group administration proceeded after passing the safety review committee.
The high-dose group, with a dose twice that of the low-dose group (2.5 × 10^6 cells/kg), was also conducted on three patients, and after the recent completion of patient administration, positive results in terms of safety, tolerability, and exploratory efficacy were confirmed in the phase 1b stage after passing the safety review committee.
Through this integrated clinical trial, ENCELL plans to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) for the clinical phase 2a based on the safety and tolerability of repeated administration of EN001 in the phase 1b, and evaluate the efficacy of EN001 compared to placebo through the change in the CMT Neuropathy Score (CMTNSv2) at 24 weeks compared to baseline (visit 2) in the phase 2a.
EN001 is a mesenchymal stem cell therapy cultured with ENCELL's proprietary 'ENCT (ENCell Technology)' technology, characterized by inhibiting cell aging and secreting more therapeutic substances.
This therapy moves to the damaged nerves to secrete therapeutic substances and regenerates the nerve myelin. In February, it was also designated as an orphan drug (ODD) by the U.S. Food and Drug Administration (FDA).
In particular, CMT is classified as a rare disease, allowing for conditional product approval after the completion of phase 2, and recently, inquiries about the status and participation in EN001 clinical trials have been increasing from CMT patient associations worldwide, raising expectations for new drugs for rare diseases.
An ENCELL representative stated, "EN001 has already confirmed safety and exploratory therapeutic effects in both low-dose and high-dose group administrations, and we have completed the application for this integrated clinical trial change," adding, "As we enter clinical phase 1b/2a, we will conduct a more speedy and efficient integrated clinical trial to ensure it is provided to CMT patients in a timely manner."
Meanwhile, ENCELL successfully completed a clinical study conducted last year on CMT 1E patients and is currently preparing to initiate advanced regenerative therapy in collaboration with Samsung Seoul Hospital.

vrdw88@fnnews.com Kang Jung-mo Reporter