Helixmith has submitted a Phase 3-3 clinical trial protocol of Engensis (VM202) to the U.S. Food and Drug Administration to further evaluate the efficacy of the gene therapy product in patients with diabetic peripheral neuropathy (DPN).

The submission followed FDA guidelines that have recommended the South Korean biopharmaceutical firm to come up with multiple Phase 3 trial data, the company said on Wednesday.

VM202, a DNA plasmid that contains novel genomic cDNA hybrid human hepatocyte growth factor (HGF), targets a root cause of neuropathy through angiogenesis and nerve regeneration beyond simple pain control.

The FDA granted regenerative medicine advanced therapy designation to VM202 in 2018 based on the drug’s scientific and clinical data.

Under the protocol, the latest clinical trial has the same design as the Phase 3-2 trial, except a follow-up period that was set at one year to collect longer-term efficacy and safety data.

The primary endpoint is the pain reduction to be measured by a pain diary at baseline and month 6 and secondary endpoints includes the ratio of patients with an average pain reduction of 50 percent or more at month 6, 9, and 12, and the statistical significance of reduction in worst pain at month 6, 9, and 12 versus placebo.

Subjects are DPN patients who do not take pregabalin or gabapentin to control their current condition. The number of subjects will be between 152 and 250 pending interim analysis data for adjustment. The clinical trial will be conducted at 15 sites across the U.S. including Northwestern University in Chicago.

For the Phase 3-2 clinical trial, patient enrollment is still underway in the U.S. with the fist injection due next month.

Kosdaq-listed Helixmith shares finished Thursday 5.3 percent down at 52,500 won.

By Kim Si-gyun and Minu Kim

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