The bio corporation ENCell reported on February 13 that it confirmed the safety and tolerability of its developing next-generation mesenchymal stem cell therapy EN001 through a single intravenous administration in a phase 1 clinical trial for patients with Duchenne muscular dystrophy (DMD).
First in South Korea
This clinical trial is the first stem cell therapy clinical trial in South Korea targeting DMD patients.
ENCell DMD Stem Cell Therapy EN001
The primary objective of the phase 1 trial is to demonstrate the safety of a single intravenous administration of EN001 in DMD patients, and it was noted that safety and tolerability were confirmed in both the low-dose and high-dose administration groups.
Exploratory efficacy evaluation results to assess drug effectiveness showed that lung capacity, lower limb strength, and creatine kinase levels were maintained without deterioration or showed some improvement. The results of this phase 1 trial were published in the Journal of Clinical Neurology.
What is the Difference Between EN001 and CAP-1002 Studies?
The two investigational drugs (EN001 and CAP-1002) are similar in that both are allogeneic cell therapies and require pretreatment to inhibit allergic responses.
Both are allogeneic cell therapeutics but with different sources and cell types: EN001 are MSCs obtained from Wharton’s jelly, which is part of the umbilical cord, whereas CAP-1002 are CDC obtained from myocardial tissues. [Learn More: What is Deramiocel? What Does CAP-1002 Do?]
The expected mechanism of action is the same as for paracrine effects, where several proteins, exosomes, or miRNAs are secreted and indirectly promote immune modulation, anti-fibrosis, and muscle regeneration.
However, the efficacy endpoints are different: the outcome measures for EN001 include serum CK level, ambulatory assessment, and respiratory function, while those for CAP-1002 are PUL and cardiac MRI evaluations.
The intended therapeutic effects of EN001 therefore focused on lower-limb skeletal muscle and respiratory muscle and those of CAP-1002 focused on upper-limb skeletal muscle and cardiac muscle. These two types of studies are complementary, and there remains a need to confirm the efficacy of EN001 in nonambulatory patients.
