ENCell Co., Ltd., a South Korean biotech leader specializing in Cell and Gene Therapy (CGT), has unveiled promising results from their first-in-human Phase 1 clinical trial of EN001, a novel mesenchymal stem cell therapy aimed at treating Charcot-Marie-Tooth disease type 1A (CMT1A). The findings were presented at the Peripheral Nerve Society (PNS) annual meeting held in Montreal on June 23, 2024, drawing attention from an international community of neurologists, neurosurgeons, and neuroscientists.

The trial, conducted at Samsung Medical Center under the supervision of Professor Byung-Ok Choi from the Department of Neurology, involved nine CMT1A patients. Utilizing a dose-escalation design over a 16-week period, the trial aimed to assess the safety and exploratory efficacy of EN001. Results from the trial, which were showcased during an oral presentation at the PNS meeting, revealed no dose-limiting toxicity, serious adverse events, or infusion-related responses, thus establishing the safety of the therapy.

More significantly, the exploratory efficacy evaluations demonstrated notable clinical improvements. The Charcot-Marie-Tooth Neuropathy Score version 2 showed an average decrease of 2.89 points (p=0.0039) post-treatment, with the high-dose group experiencing a more substantial reduction of 3.50 points (p=0.0313). Patients receiving the high dose also saw improvements in disease severity, with several moving from severe to moderate or moderate to mild categories.

Additional assessments, including the 10 Meter Walking Test, Functional Disability Scale, Overall Neuropathy Limitation Score leg scale, and nerve conduction studies, all indicated enhanced sensory and motor functions, particularly in the high-dose group.

Following these encouraging results, ENCell is planning a repeat-administration clinical trial at Samsung Medical Center. The company’s commitment to advancing treatment options was emphasized by an ENCell spokesperson who stated, “Presenting these promising clinical results at such a prestigious conference underscores our dedication to developing new therapies for CMT patients, who currently have no approved treatments.”

Professor Byung-Ok Choi also expressed optimism, noting, “These results provide hope to CMT1A patients, a group affected by a rare disease without existing treatments.” This sentiment reflects the potential impact of EN001 in addressing a significant unmet medical need within the neurology community.

The success of EN001 in early trials represents a significant stride in the field of regenerative medicine, particularly for treating neurological disorders like CMT1A. The therapy’s ability to target and regenerate damaged nerves could pave the way for broader applications in neuromuscular diseases, potentially offering new hope to patients with limited treatment options.

  Charcot-Marie-Tooth disease, clinical trial results, clinical trials, CMT1A, CMT1A treatment, ENCell, ENCell Co Ltd, neurology, Peripheral Nerve Society, PNS meeting, Regenerative Medicine, stem cell therapy