Helixmith's 'Engensis' US Phase 3-2 Clinical Trial Completes Target Patient Enrollment

Sunyoung Kim, CEO of Helixmith

[Asia Economy Reporter Lee Chun-hee] Helixmith's core pipeline gene therapy 'Engensis (VM202)' for diabetic peripheral neuropathy (DPN) has completed the enrollment of 152 patients, the original target for its Phase 3-2 clinical trial.

Helixmith announced on the 24th that the 152nd patient enrolled in the Engensis Phase 3-2 trial received dosing on the 22nd (local time). This means that Helixmith has met the minimum patient size set in the original clinical protocol.

The DPN Phase 3-2 trial of Engensis (VM202) was designed based on an adaptive design. An independent data monitoring committee (IDMC) was expected to conduct an interim analysis based on data observed for about six months after dosing half of the 152 patients, i.e., 76 patients, and decide among three options: 'continue with 152 patients,' 'adjust patient size (up to 250),' or 'terminate the trial.'

However, on the 17th, the IDMC chose to defer judgment rather than select one of these three options. Since approximately 150 patients, close to the minimum number of 152, had already been dosed, it was analyzed that the interim analysis on half, 76 patients, was deemed meaningless. However, regarding safety, the Data Safety Monitoring Board (DSMB) judged the treatment to be safe as there was no difference in adverse events between the clinical and control groups.

Helixmith plans to enroll about 160 patients by deciding on the participation of patients who have already submitted informed consent forms for the clinical trial as of last week, in addition to the 152 patients. However, among the original 76 patients for interim analysis, 14 dropped out due to the COVID-19 pandemic, so the final number of patients available for data analysis is expected to be around 146.

Helixmith will decide in the second half of this year whether to conduct an additional interim analysis or replace it with a final analysis for these approximately 160 patients. The topline results are expected to be disclosed in the first half of next year.