The HNF sent out a short notice yesterday about a new Phase 1 and 2a clinical trial for VM202 (Engensis) from Seoul-based Helixmith, previously known as ViroMed. This is a drug that Helixmith hopes will reduce nerve pain for people with CMT1a as an alternative to Lyrica (Pregabalin) and Neurontin (Gabapentin). (If it panned out, that would be a good thing — Pregabalin and Gabapentin have some unpleasant side-effects and potential for addiction.)
Update, October 2026: Results of the Korean phase 1/2a trial of Engensis (VM202) in CMT1A (NCT05361031) were published in March 2026. The trial treated 12 adults and had no placebo group. The drug was generally safe and well tolerated. Disease-severity scores (CMTNSv2 and CMTES) improved over about nine months, mainly on sensory items, but changes in walking and lower-limb function were not statistically significant. The researchers say larger, controlled trials are needed.
VM202’s promise appears to have faded a lot since its debut in clinical trials. It seems the new hope for VM202 as a CMT1a treatment is perhaps Helixmith’s latest attempt to find a market for an experimental product that has not panned out as hoped for other diseases.
It seems the new hope for VM202 as a CMT1a treatment is perhaps Helixmith’s latest attempt to find a market for an experimental product that has not panned out as hoped for other diseases.
What is VM202?
The drug itself is a DNA plasmid — a small loop of DNA that’s not part of any chromosomal DNA. VM202 doesn’t copy itself in the body; after it’s injected into muscle, local cells take it up and make the protein it encodes for a limited time. This could be described as “gene therapy.” The plasmid in VM202 encodes DNA for the human hepatocyte growth factor (HGF), which can help nerves and blood vessels regenerate by signalling to nearby cells at a short range (paracrine signalling).
Sorry, the placebo was laced with drugs…or maybe not?
PipelineReview.com summarizes the drug’s testing history: VM202 was developed to treat painful diabetic peripheral neuropathy (DPN), amyotrophic lateral sclerosis (ALS), coronary artery disease, and foot ulcers associated with diabetes.
CMT does not seem to have been in the original target market for VM202. Instead, DPN — likely a much bigger market — was the subject of the first trials.
The first Phase 3 trial in 500 DPN patients failed to show a reduced experience of pain after nine months that differed from a placebo group. 101 people in this test group were followed for another three months with no further treatment, which established the drug is at least safe enough for a year of follow-up without major adverse effects showing up. A significant analgesic (pain reduction) effect was reported as well — with the smaller, extended study group.
Helixmith hopes the reported pain reduction — as one point on the scale they used — indicates that VM202 regenerates damaged nerves. (HGF has neurotrophic and angiogenic effects in the lab, but these possible mechanisms remain unproved in humans.)
Correction, October 10, 2026: I’ve corrected three points above. I originally wrote that the plasmid can replicate itself; VM202 is taken up by local cells and expresses HGF only transiently. I wrote that the 101-person extension group kept getting the drug for three more months; it was followed up with no further treatment. And I called nerve regeneration presumably “the only way” an HGF plasmid could reduce pain; possible neurotrophic and angiogenic mechanisms remain unproved in humans. The main 500-person trial missed its efficacy endpoint, and results in diabetic neuropathy don’t establish that VM202 works in CMT.1
After the disappointing first test results, Helixmith alleged there had been a mixup where the drug was given to a placebo group. The company did not bring the study to a conclusion then as expected due to the data contamination they believed had occurred. Then, following an investigation, Helixmith said nothing was wrong, and their original data from the trial was valid!
During this fiasco, the company’s stock received a “Sell” rating from Goldman Sachs. The shares had already fallen by the Kosdaq’s 30% daily limit the day the trial problems were disclosed in September 2019, and they never recovered: from about 318,000 won in March 2019 to under 26,000 won by March 2021.
🤯 Amber Tong has a trenchant commentary about this slide over at Endpoints News.
Will VM202 help people with CMT1a? Maybe, maybe not. We can surely hope, but Helixmith’s performance in the trials so far is far from reassuring.
Update, October 2026: what happened to Helixmith. The US phase 3 trial of Engensis for diabetic foot ulcers was stopped early in June 2021 for slow recruitment. In January 2024 the remaining US phase 3 trials for diabetic nerve pain missed their primary endpoint: Engensis did no better than placebo, and the shares fell by the daily 30% limit again. By then control was passing to Bio Solution, which became the largest shareholder in a deal disclosed in December 2023 and restructured the company. Its net loss fell from 64.1 billion won in 2023 to about 300 million won in 2025. The one success came elsewhere: in May 2026 China’s drug regulator approved NL003, a product developed from Engensis and licensed to Northland Biotech back in 2004, for critical limb ischemia (poor blood flow to the legs). It is China’s first plasmid DNA gene therapy, and Helixmith collects royalties on its sales. So the drug that failed for nerve pain was finally approved for blood flow, not neuropathy. I follow the whole arc, and what NL003 might mean for cold CMT feet, in Three “Treatable CMT” Stories, and What the Market Taught Us.
Researching this topic motivated me to ask out loud a question I’ve had for a while: what condition is the Food and Drug Administration in these days? Some articles on that subject from 2019–20 (a period piece now) that I’d like to dig into later:
- Science is not amused: FDA enforcement actions plummet under Trump
“If the FDA isn’t doing enforcement, nobody’s doing enforcement.” - PwC (PricewaterhouseCoopers) is one of the Big Four accounting firms invested in risk assessment and used to mincing words a bit. They say the glass is half full: Continue to take as prescribed: Amid a steep slowdown in regulation, the FDA hit record highs for drug approvals while maintaining enforcement.
🧪 The CMT1A trial of Engensis has finished. If you have CMT and want to hear about future trials, you can head over to GRIN and bare your personal medical data. 👩💻
Notes
- Kessler et al., “Gene Therapy for Diabetic Peripheral Neuropathy”; open access at PMC8212761. Extension study: Helixmith, “Extension of Phase 3 Gene Therapy,” NCT04055090. ↩︎
Sources
- Helixmith. “Extension of Phase 3 Gene Therapy for Painful Diabetic Neuropathy.” ClinicalTrials.gov. Accessed October 14, 2026. https://clinicaltrials.gov/study/NCT04055090.
- Kessler, John A., Aziz Shaibani, Christine N. Sang, et al. “Gene Therapy for Diabetic Peripheral Neuropathy: A Randomized, Placebo-Controlled Phase III Study of VM202, a Plasmid DNA Encoding Human Hepatocyte Growth Factor.” Clinical and Translational Science 14, no. 3 (2021): 1176–1184. https://doi.org/10.1111/cts.12977.
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