Helixmith said that it has kicked off its phase 1 and 2a clinical trial for using VM202 to treat Charcot-Marie-Tooth (CMT) 1A disease, a rare disorder that currently has no treatment.

The company received approval to start the clinical trials from the Ministry of Food and Drug Safety in April.

“CMT is a disease in which the muscles of the arms and legs atrophy and gait disorders occur due to the motor and sensory nerves' progressive damage. It is one of the most common ailments among rare diseases,” the company said. “Notably, CMT type 1A is a CMT that occurs due to an abnormality in the PMP22 gene and accounts for 40 percent of the total CMT.”

Professor Choi Byeong-ok at Samsung Medical Center will conduct the trial on 12 CMT1A patients for 270 days. The trial aims to evaluate the drug's safety and tolerability by administering the treatment to the leg muscles of CMT1A patients.

“CMT is a disease caused by damage to the peripheral nerve and is very similar to diabetic neuropathy (DPN),” Helixmith CEO Kim Sun-young said. “Since VM202 has already been designated by the U.S. Food and Drug Administration as regenerative medicine advanced therapy for DPN, the company expects the treatment will likely have a therapeutic effect on CMT treatment.”

According to the company, there are about 8,000 CMT1A patients in Korea and more than 1.2 million patients worldwide.

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