The bio corporation ENCell reported on the 13th 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). Tolerability refers to the degree to which a patient or clinical trial subject can endure side effects or discomfort when a drug is administered.
This clinical trial is the first stem cell therapy clinical trial in South Korea targeting DMD patients.
Duchenne muscular dystrophy, also known as DMD or Duchenne disease, is a genetic disorder that usually manifests symptoms between the ages of 2 and 5 and progressively weakens muscles as one ages. Individuals experience difficulty in running or climbing stairs, and by the age of 10 to 14, they may find walking difficult and begin to use a wheelchair, as well as struggle to lift their arms. The prevalence is approximately 4 per 100,000 people, with an incidence of 1 in 3,500 newborn males. It occurs much more frequently in males than in females. There are about 2,000 DMD patients in South Korea.
Professor Lee Ji-hoon of Samsung Seoul Hospital's Department of Pediatrics and Adolescent Medicine served as the principal investigator for the clinical trial, which was conducted from January 2022 to December 2022 involving six DMD patients.
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.
Further clinical trials will be conducted to statistically confirm significant clinical efficacy.
Professor Lee Ji-hoon, who led the clinical trial, noted regarding the results of the EN001 phase 1 trial, "It is encouraging to confirm safety and tolerability," and added, "If clinical efficacy is secured through additional clinical trials, EN001 could become one of the important treatment options for DMD patients." He also mentioned, "It is expected that it could be applicable to other muscular dystrophies that have been overlooked in drug development."
Currently, there is a gene therapy approved by the U.S. Food and Drug Administration (FDA) for treating DMD patients aged 4 and older, called Elevidys from Sarepta Therapeutics.
ENCell specializes in contract development and manufacturing of advanced bio-pharmaceuticals (CDMO) and new drug development. They are currently conducting a phase 1b trial to verify the safety and efficacy of repeated administration of EN001, having demonstrated the efficacy of EN001 in combination with insulin for Charcot-Marie-Tooth disease, which has the largest patient population among rare diseases.