High Interest in Gene Therapy for Painful Diabetic Neuropathy

A press release notes that results from a phase 3 study (NCT04087941) of gene therapy, donaperminogene seltoplasmid (VM202; Helixmith, San Diego, CA), for painful diabetic therapy is among the most downloaded article from Clinical and Translational Science (CTS) in the past year.

Donaperminogene seltoplasmid is a plasmid DNA encoding the human HGF (hepatocyte growth factor) gene that was administered to calf muscles on days 0 and 14, and on days 90 and 104. In the first part of the trial 336 were treated with gene therapy and 164 with placebo and followed for 9 months. In the extension period, 101 participants (65 treated with gene therapy and 36 with placebo, were followed for another 3 months without further intervention.

In the larger study, no statistically significant effect of gene therapy was observed. In the extension period, however, pain relief that occurred 8 months after the last injection in those treated with gene therapy vs placebo was statistically significant and clinically meaningful.
A second phase 3 trial, REGAiN-1A (NCT04469270) is ongoing, with top-line results expected by the end of 2022 and a third phase 3 trial is being planned and expected to be initiated in the second half of 2022.