Helixmith Accelerates Preparation for Enzygnosis DPN Phase 3-3 Trial

[Asia Economy Reporter Jang Hyowon] Helixmith has begun full-scale preparations for the Phase 3-3 clinical trial of its gene therapy ‘Engensis (VM202)’ targeting diabetic peripheral neuropathy (DPN).

On the 29th, Helixmith announced that it had selected ‘PRA Health Sciences (hereafter PRA)’ as the contract research organization (CRO) for the DPN Phase 3-3 clinical trial.

Helixmith is currently conducting its second Phase 3 trial (3-2, 3-2b) targeting DPN. As of July 20, a total of 45 participants have been randomly assigned and administered the drug. Helixmith judged that the progress of the DPN 3-2 trial is on track and expects recruitment of trial subjects to be completed in the second half of the year, making it possible to start the DPN 3-3 trial. In particular, the company explained that it gained confidence in the operation due to the high compliance level of subjects during the 3-2 trial, minimal variance in indicators among subjects, and high internal consistency.

Last September, following the US FDA guideline recommending multiple Phase 3 clinical results, Helixmith submitted the DPN 3-3 trial protocol. The DPN 3-3 protocol is very similar to the 3-2 trial, with the primary endpoint being pain reduction compared to placebo at six months after the first injection, and secondary endpoints including sensory function recovery. Additionally, a 3-3b trial will be conducted to collect 12-month safety data.

PRA is also managing the ongoing DPN 3-2 trial. PRA is a global company headquartered in the United States with a network spanning over 80 countries.

Kim Sun-young, CEO in charge of global clinical development, stated, “The Engensis DPN 3-2 trial is progressing smoothly, and the quality of various operational data produced is excellent, so we judged that further clinical trials are fully feasible,” adding, “Starting the DPN 3-3 trial brings us closer to our goals of licensing and market approval.”

Diabetic peripheral neuropathy (DPN) is one of the most common complications experienced by diabetic patients. Approximately 30 million adults in the United States have diabetes, and about 28.5% of them develop DPN, with 40-50% of DPN patients known to have painful DPN (PDPN). PDPN is characterized by burning or stabbing pain. Existing DPN treatments only alleviate pain and are not fundamental cures, often accompanied by severe side effects and high addiction potential.

Helixmith’s ‘Engensis (VM202)’ is a plasmid DNA-based gene therapy that targets the root cause of neuropathy through angiogenesis and nerve regeneration effects, rather than merely managing pain. The US FDA recognized the scientific and clinical results of Engensis and designated it as a Regenerative Medicine Advanced Therapy (RMAT) in 2018. This is the first gene therapy to receive RMAT designation in the US for a widespread disease affecting millions of patients.