ENCell Shares Phase 1 Results of EN001 Stem Cell Therapy for Charcot-Marie-Tooth at PNS Conference

SEOUL, South Korea--ENCell Co., Ltd., a biotechnology company based in South Korea, has announced encouraging results from their inaugural Phase 1 clinical trial of the innovative mesenchymal stem cell therapy (EN001) for Charcot-Marie-Tooth disease type 1A (CMT1A). The findings were presented at the Peripheral Nerve Society (PNS) annual conference, held in Montreal on June 23, 2024. This annual event is a significant congregation for professionals in the field, including neurologists, neurosurgeons, neuroscientists, and other healthcare specialists focusing on peripheral nerve conditions.

EN001 represents a groundbreaking allogeneic mesenchymal stem cell therapy created using ENCell's unique ENCell Technology. According to ENCell's non-clinical studies, EN001 possesses the ability to migrate to and repair damaged nerves and muscles. In June 2023, ENCell completed a Phase 1 clinical trial involving nine CMT1A patients at Samsung Medical Center (SMC), under the supervision of Professor Byung-Ok Choi from the Department of Neurology. The trial employed a dose-escalation design to assess the safety and exploratory efficacy of EN001 over a 16-week period.

The trial outcomes, selected for oral presentation at the PNS meeting, revealed no dose-limiting toxicity, serious adverse events, or infusion-related reactions among the participants, thereby confirming the safety profile of EN001. Additionally, exploratory efficacy assessments showed notable improvements. The Charcot-Marie-Tooth Neuropathy Score version 2 decreased by an average of 2.89 points (p=0.0039) after 16 weeks, with the high-dose group experiencing a more substantial reduction of 3.50 points (p=0.0313).

Further evaluations indicated that patients receiving the higher dose showed clinical improvements in disease severity, with some transitioning from severe to moderate or from moderate to mild categories. Specific sensory and motor function tests also demonstrated significant enhancements, particularly in the high-dose group. Improvements were noted in the 10 Meter Walking Test, Functional Disability Scale, Overall Neuropathy Limitation Score leg scale, and nerve conduction studies.

An ENCell representative underscored the importance of presenting these promising clinical results at a reputable conference, highlighting the company's commitment to developing new therapeutic options for CMT patients, who currently have no approved treatments. Professor Byung-Ok Choi, who led the clinical trial, expressed optimism, stating, "These results provide hope to CMT1A patients, a group affected by a rare disease without existing treatments." ENCell is planning to conduct a repeat-administration clinical trial at SMC.

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