The Hereditary Neuropathy Foundation (HNF) is pleased to share that Helixmith, a gene therapy research company with more than 20 years of experience in the field, has launched a Phase I and Phase 2a clinical trial using VM202 (also known as Engensis) to treat CMT1A (the most common form of Charcot-Marie-Tooth disease). Helixmith is headquartered in Seoul, Korea, with clinical development and manufacturing activities based in San Diego, California.

About the trial

The trial is designed to evaluate the safety and tolerability of the treatment by administering it to the leg muscles of twelve CMT1A patients in Korea over a 270-day period. Preparations for CMT clinical trials in the United States were underway at the time of this announcement.

The primary efficacy endpoint will compare changes in average daily pain scores at regular intervals between participants receiving Engensis and those receiving a placebo.

Context and significance

VM202 had previously received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration for Diabetic Peripheral Neuropathy. Helixmith noted that this prior designation informed their expectation that the treatment may have a therapeutic effect in CMT.

“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.”Allison Moore, Founder and 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.”Kim Sun-young, CEO, Helixmith
“This is an exciting time for our patient community with several clinical trials in the pipeline for launch next year.”Joy Aldrich, GRIN Patient Registry Coordinator

How GRIN supports clinical trials

The Global Registry for Inherited Neuropathies (GRIN) continues to provide important patient data to industry partners to assist with clinical trial design, patient-focused research, and participant recruitment. Join GRIN to help advance understanding of how CMT affects daily life and to contribute to ongoing research efforts.