On the 18th, ENCell, a corporation specializing in contract development and manufacturing of advanced biopharmaceuticals (CDMO) and new drug development, announced that it would conduct clinical trials combining Phase 1b and 2a for patients with Charcot-Marie-Tooth disease (CMT) type 1A.
The corporation has completed a modification application to the Korea Food and Drug Administration to apply EN001 for clinical Phase 1b and 2a for patients with CMT type 1A.
CMT is a hereditary disease that causes deformations in hands and feet and muscle atrophy, and in severe cases, it can lead to loss of vision and hearing.
EN001 is a mesenchymal stem cell treatment developed using ENCell's proprietary technology. It plays a role in moving to damaged nerves to secrete therapeutic substances and regenerate nerve myelin; it has also been designated as an orphan drug by the U.S. Food and Drug Administration (FDA). The nerve myelin is a fatty layer structure that surrounds nerve fibers (axons) in peripheral and central nervous systems.
The corporation explained that combining Phase 1b and 2a in this way is a strategy to reduce administrative procedures and time while increasing efficiency. In this integrated clinical trial, they plan to determine the maximum tolerated dose (MTD) and recommended Phase 2a dose (RP2D), and evaluate efficacy based on changes in the CMT neuropathy score at 24 weeks.
An ENCell official said, "EN001 has confirmed safety and exploratory therapeutic effects in the assessment of low-dose and high-dose groups of patients, completing the application for this integrated clinical trial," and added, "We will do our best to conduct a more rapid and efficient integrated clinical trial so that treatment can be provided in a timely manner to CMT patients."