ENCell Presents the Phase 1 Clinical Trial Results of their Mesenchymal Stem Cell Therapy, EN001, for Charcot-Marie-Tooth Disease at the PNS conference
26 Junio 2024 - 6:00AM
Business Wire
ENCell Co., Ltd., a biotech in South Korea specializing in Cell
and Gene therapy (CGT), presented promising results from their
First-in-Human Phase 1 clinical trial of the novel mesenchymal stem
cell therapy (EN001) for Charcot-Marie-Tooth disease type 1A
(CMT1A) at the Peripheral Nerve Society (PNS) annual meeting in
Montreal on June 23, 2024. This annual conference is a key
gathering for experts in the field, including neurologists,
neurosurgeons, neuroscientists, and other healthcare professionals
focused on peripheral nerve disorders.
EN001 is a next-generation allogeneic mesenchymal stem cell
therapy developed using ENCell's proprietary ENCell Technology.
ENCell reported EN001 can migrate to and regenerate damaged nerves
and muscles through non-clinical studies. In June 2023, ENCell
completed a Phase 1 clinical trial with nine CMT1A patients at
Samsung Medical Center (SMC) with Professor Byung-Ok Choi from the
Department of Neurology as the Principal Investigator. The trial
utilized a dose-escalation design to ensure the safety and
exploratory efficacy of EN001 over a 16-week period.
The trial results, which were selected for oral presentation at
the PNS meeting, showed no dose-limiting toxicity, serious adverse
events, or infusion-related responses among participants, thereby
confirming the safety of EN001. Additionally, exploratory efficacy
evaluations revealed significant 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 showing a more pronounced reduction of 3.50 points
(p=0.0313).
Further analysis indicated that patients receiving the high dose
experienced clinical improvements in disease severity, with some
moving from severe to moderate or from moderate to mild categories.
Specific sensory and motor function tests also showed marked
improvements, with the high-dose group. Enhancements in the 10
Meter Walking Test, Functional Disability Scale, Overall Neuropathy
Limitation Score leg scale, and nerve conduction studies were also
observed.
An ENCell spokesperson highlighted the significance of
presenting these promising clinical results at a prestigious
conference, emphasizing the company's dedication to developing new
treatment options for CMT patients, who currently lack approved
therapies. Professor Byung-Ok Choi, leading 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 repeat-administration
clinical trial at SMC.
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ENCell Co., Ltd. Choongseob Oh encell@encellinc.com