The Hereditary Neuropathy Foundation is excited to share that Helixmith, specializing in gene therapy research for over 20 years, has kicked off its phase I and 2a clinical trial for using VM202 (Engensis) to treat CMT1A. The company is currently headquartered in Seoul, Korea; with clinical development and manufacturing activities based in San Diego, California.

The trial aims to evaluate the drug’s safety and tolerability by administering the treatment to the leg muscles of twelve CMT1A patients in Korea for 270 days. Preparations for CMT clinical trials in the US are currently underway.

The primary efficacy endpoint will compare the change in average daily pain scores on regular intervals in participants treated with Engensis versus placebo. 

“HNF was the first to acknowledge and conduct research studies with funding support from Patient-Centered Outcomes Research Institute (PCORI), specifically addressing pain in CMT patients. We are thrilled to support Helixmith with our research findings and hope that this will be the first disease modifying treatment that addresses pain,” said Allison Moore, Founder/CEO HNF. 

“Since VM202 has already been designated by the U.S. Food and Drug Administration as regenerative medicine advanced therapy for Diabetic Peripheral Neuropathy, the company expects the treatment will likely have a therapeutic effect on CMT treatment” said Helixmith CEO Kim Sun-young. 

“This is an exciting time for our patient community with several clinical trials in the pipeline for launch next year,” says Joy Aldrich, Global Registry for Inherited Neuropathies (GRIN) patient Registry Coordinator. 

GRIN will continue to provide important patient data to industry to help with clinical trial design protocols, patient focused research data and for clinical trial recruitment. 

Help us advance knowledge of how CMT affects your life by joining GRIN.

Updates will be provided by HNF at www.hnf-cure.org