BioLeaders Completes Phase 1 Clinical Trial of 'BLS-M22' for Pediatric Rare Disease Duchenne Muscular Dystrophy Treatment

[Asia Economy Reporter Eunmo Koo] Bioliders, a company specializing in new drug development, announced on the 29th that it has completed the Phase 1 clinical trial in South Korea for its new drug candidate ‘BLS-M22,’ which is being developed as a treatment for the pediatric rare disease Duchenne muscular dystrophy.

The Phase 1 clinical trial of BLS-M22, conducted at Seoul Samsung Hospital, was carried out to evaluate safety in healthy volunteers. No serious adverse reactions were reported following administration at various doses to each group, and no participants dropped out due to adverse reactions. Additionally, no dose-limiting toxicity was observed at the highest concentration set during repeated administration.

Duchenne muscular dystrophy (Duchenne type muscular dystrophy), a genetic disorder, is a pediatric rare disease caused by abnormalities in the X chromosome leading to the loss of a protein present in the muscle cell membrane. It primarily affects approximately 1 in 3,500 boys and is known to typically result in death around the age of 20.

BLS-M22 is an innovative new drug created based on Bioliders’ core MucoMax® platform technology. It utilizes a mechanism in which the antigen of myostatin, a protein that inhibits muscle formation, is displayed on the surface of probiotics, and antibodies are formed in the body through oral administration. It was selected for the Ministry of Health and Welfare’s advanced medical technology project and has successfully completed non-clinical efficacy studies and pharmaceutical manufacturing process research for conducting clinical trials for Duchenne muscular dystrophy.

The company received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the Duchenne muscular dystrophy treatment in 2017, followed by orphan drug designation at the development stage from the Korean Ministry of Food and Drug Safety last year. If the product is approved in the U.S., it will be eligible for various exclusive rights related to rare diseases as well as transferable priority review vouchers.

The head of Bioliders’ new drug development division stated, “This clinical trial is significant in confirming the safety and tolerability of single-dose administration at various doses and 14-day repeated administration of BLS-M22,” adding, “The efficacy such as muscle loss inhibition and improvement in motor function, confirmed in animal evaluations, has already been verified.”

A company official said, “We are actively pursuing overseas clinical trials alongside the domestic Phase 2 clinical trial,” and added, “Since this is a treatment for a rare disease, we also expect the possibility of conditional approval after the completion of Phase 2.” They emphasized, “As the likelihood of success for this new drug for Duchenne muscular dystrophy, which lacks adequate treatments, has increased, we will strive to expedite the commercialization of the product.”