Helixmith Announces Topline Results of Domestic Phase 1/2a Clinical Trial for Charcot-Marie-Tooth Disease with NGENSIS

[Asia Economy Reporter Jang Hyowon] Helixmith announced the topline data of the domestic Phase 1/2a clinical trial of the gene therapy drug Engensis (VM202) for Charcot-Marie-Tooth disease (CMT) on the 9th. This clinical trial is the world's first clinical study on gene therapy for CMT.

CMT is a genetic disorder characterized by progressive damage to motor and sensory nerves, leading to muscle atrophy in the arms and legs and gait disturbances. It has a prevalence of 1 in 2,500, making it one of the more common rare diseases. To date, there is no treatment approved by the U.S. FDA.

The Phase 1/2a clinical trial was conducted by Professor Choi Byung-ok, the leading expert on CMT in Korea at Samsung Seoul Hospital. The primary objective was to evaluate the safety of Engensis (VM202) in a total of 12 CMT patients. Additionally, various indicators to assess efficacy were measured.

Disability and neuropathy severity were assessed using FDS (Functional Disability Scale), ONLS (Overall Neuropathy Limitation Scale), and CMTNS-v2 (CMT Neuropathy Score v2). MRI was used to investigate the degree of muscle loss caused by neuropathy. The imaging analysis provided highly objective indicators.

Engensis (VM202) demonstrated excellent safety in this CMT clinical trial. Two adverse reactions suspected to be related to the drug occurred: one case of ankle edema and one case of injection site itching. Both were mild and recovered quickly.

Improvement trends were observed in the efficacy indicators FDS and ONLS. For FDS, 7 patients showed a 1-point improvement, while 5 showed no change. For ONLS-leg, 4 patients showed a 1-point improvement, and 8 showed no change. The average change was 0.33, which is higher than the 0.18 ONLS-leg improvement shown by PXT3003 from Pharnext, currently in Phase 3 clinical trials in the U.S., the leading CMT treatment.

In the CMTNS-v2 assessment, another efficacy indicator for disability improvement, the total score decreased by an average of 2.17 points over 9 months, showing a 14% improvement with statistical significance (p<0.01). Among the 9 items of CMTNS-v2, 3 items (sensory symptoms, pain sensitivity, vibration sense) showed significant symptom improvement compared to baseline. This suggests that Engensis (VM202) can improve neuropathy or halt disease progression.

Muscle loss due to neuropathy was also measured via MRI. Since muscle loss leads to increased fat, the fat ratio in the imaging of the affected area was calculated to assess muscle loss. Fat increased by 0.2 in the calf muscle and 1.9 in the lower leg muscle, similar to the known annual change of 1.8. When analyzing patients with a baseline fat percentage of over 10% at the start of the trial separately, the fat increase in the calf and lower leg muscles of patients injected with Engensis (VM202) ranged from -0.8% to 1.1% depending on the area. Considering that fat increase in patients with a baseline fat percentage over 10% is known to be about 3% annually, this suggests that Engensis (VM202) may prevent muscle loss.

Kim Sun-young, CEO of Helixmith, said, “We confirmed the excellent safety of Engensis in this CMT clinical trial and observed some improvement in sensory symptoms in FDS, ONLS, and CMTNS-v2, as well as less fat increase due to muscle loss in MRI. We expect that in the next clinical trial, by increasing the scale and selecting patients with more advanced symptoms, we will be able to measure the effects more clearly.”

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