Building momentum from U.S. orphan drug designation to expand patient association connections in each country
[by Yu, Suin] ENCell disclosed on April 7 its phased global expansion strategy for 'EN001’ (development code), a treatment candidate currently under development.
The company is actively pursuing out-licensing (L/O) opportunities, leveraging the momentum gained from the U.S. Food and Drug Administration (FDA) granting EN001 Orphan Drug Designation for the treatment of Charcot-Marie-Tooth Disease (CMT) in February.
CMT is a hereditary neurological disorder characterized by hand and foot deformities, as well as progressive muscle atrophy. In severe cases, the condition can also lead to vision and hearing impairment. Although classified as a rare disease, CMT has a relatively high incidence. However, no approved treatments are currently available, posing a significant challenge for both patients and healthcare providers.
EN001 is a mesenchymal stem cell-based treatment candidate cultured using 에볼루션 바카라 사이트's proprietary technology platform, 'ENCT’ (에볼루션 바카라 사이트 Technology). This platform is designed to suppress cell aging and enhance the secretion of bioactive substances essential for treatment. EN001 exhibits targeted migration to damaged nerves, where it releases regenerative factors and contributes to the restoration of nerve myelin.
에볼루션 바카라 사이트 has identified a high level of understanding and acceptance of mesenchymal stem cell therapies in the Asian market and is currently prioritizing Japan as its primary target for licensing-out activities. In line with this strategy, the company is actively expanding its network and engagements with various Japanese firms. As part of these efforts, 에볼루션 바카라 사이트 recently signed a strategic memorandum of understanding (MOU) with Cell Resources, an affiliate of Alfresa Group, one of Japan’s largest pharmaceutical distribution companies, to secure a strategic foothold for market entry into Japan.
에볼루션 바카라 사이트’s secondary objective is to obtain clinical data from each country while simultaneously expanding its engagement with major patient advocacy organizations in key markets such as the U.S. and Europe. “The company is dedicating its full capabilities to the successful development of EN001. Building on the momentum of the FDA’s orphan drug designation, we have been maintaining close communication with patient associations worldwide,” an 에볼루션 바카라 사이트 official said. “We are committed to accelerating the ongoing clinical trials to offer EN001 as a new treatment option for CMT patients globally and to facilitate global L/O opportunities.”
On the other hand, in October 2024, 에볼루션 바카라 사이트 announced the safety and exploratory therapeutic effects observed in the low-dose cohort of its repeated-administration clinical trial of EN001 in CMT type 1A patients. In this study, three patients in the low-dose group received two administrations of EN001, with dose-limiting toxicity (DLT) evaluated at the 8-week point. The results showed no instances of DLT, no serious adverse events, or infusion-related reactions in any of the participants. Based on these findings, the company proceeded with the administration of EN001 to patients in the high-dose group as part of the Phase 1b clinical trial in December last year. 에볼루션 바카라 사이트 aims to successfully complete the Phase 1b clinical trial within the year.