Helixmith "NGENSIS Mid-term Evaluation, Additional Data Requested... No Schedule Disruption"

Kim Sun-young "Originally planned to announce today... postponed to next month"
"Only occasional supplements... common occurrences"

Clinical trial scale expanded... no significant financial burden
Clinical trial suspension... even if suspended, analysis of '134 participants' possible

On the 28th, Kim Sun-young, CEO of Helixmith, is speaking at a press conference held at Helixmith headquarters in Magok, Gangseo-gu, Seoul. / Photo by Lee Chun-hee

[Asia Economy Reporter Lee Chun-hee] The announcement of the interim evaluation results for Helixmith's gene therapy 'Engensis (VM202)' Phase 3-2 clinical trial, initially expected to be released as early as this month, is likely to be somewhat delayed.

At a press conference held on the 28th at Helixmith headquarters in Magok, Gangseo-gu, Seoul, CEO Kim Sun-young stated, "We originally hoped to announce the interim results of the Engensis Phase 3-2 clinical trial (today)," but added, "We received a request for additional data from the Independent Data Monitoring Committee (IDMC) over the weekend," and "Therefore, the announcement has been temporarily postponed until next month."

Currently, Helixmith is conducting a Phase 3-2 clinical trial in the United States involving 152 patients with painful diabetic peripheral neuropathy (Painful DPN) related to Engensis. So far, 134 patients have been enrolled. Park Young-joo, Head of Clinical Development at Helixmith, explained, "The trial was designed using an adaptive design." This means that during the trial, interim results are reviewed to modify the trial design, scale, or objectives as needed.

Continue Trial vs. Expand Patient Enrollment vs. Stop Trial... 'Three Crossroads'

Regarding these interim results, Helixmith is currently awaiting the interim recommendations from the IDMC, which the U.S. Food and Drug Administration (FDA) recommends forming. The IDMC is an objective independent committee established to ensure safety and scientific validity during clinical trials. It consists of three members: one statistical expert and two physicians. They secure and analyze the data necessary for interim analysis, calculate the 'conditional power,' and recommend to the trial sponsor whether to 'continue the trial,' 'modify the trial design,' or 'stop the trial.'

This interim analysis is based on data observed for about six months after dosing 76 patients, which is half of the 152 patients planned for the Phase 3-2 trial. In this case, the IDMC's recommendation is expected to be one of three options: 'continue the trial with 152 patients,' 'adjust patient enrollment (up to 250 patients),' or 'stop the trial.' If the conditional power is 80% or higher, the trial will continue at the current scale; if it is between 39.6% and less than 80%, the IDMC will recalculate and notify the required number of patients. If it is below 39.6%, the IDMC will recommend stopping the trial.

Photo by Lee Chunhee

However, considering that CEO Kim, who recently visited San Diego, California, USA, to attend the '2022 BIO International Convention (BIO USA),' stated that "interim results will be available by mid-next month," some evaluations suggest that this is not a significant delay. The goal of "within this month" was somewhat tight, and it does not appear that the schedule for receiving the IDMC's recommendation has been significantly postponed.

On this day, CEO Kim also explained that, contrary to the perception among industry and investors that the 'request for additional data' indicates a major problem with the trial, this is a routine process related to Engensis. He said, "When analyzing data, there are always many back-and-forths," and "It is common to request additional data when something is not well understood because only numbers come out during the analysis process."

"Phase 3-3 Also Planned... Even If Stopping Is Recommended, a Reversal Story Could Emerge"

CEO Kim also disclosed the future clinical strategy for Engensis following the receipt of the IDMC's recommendation. Unless the IDMC decides to continue based on the current trial design, the planned Phase 3-3 trial and others will inevitably face setbacks.

Currently, Helixmith is conducting the Phase 3-2 trial with four doses administered at 90-day intervals (Day 0, Day 14, Day 90, and Day 104), and is also pursuing a Phase 3-3 trial in North America and Europe, which involves six doses (adding Day 180 and Day 194) for long-term administration. Concerns have been raised that if the Phase 3-2 patient enrollment expands and both Phase 3-2 and Phase 3-3 trials proceed simultaneously, they might compete for patients.

CEO Kim said, "Since they would compete with each other, there is some consideration about proceeding slowly," but added, "Since the Phase 3-3 trial is also actively planned in Europe, if there is a recommendation to expand patient enrollment, we will start the Phase 3-3 trial first in Europe." Regarding the cost burden related to patient enrollment expansion, he explained, "Clinical trials require significant setup costs at the start, but once underway, the ongoing costs are only slightly higher," and "Adding about 100 patients to an ongoing trial is not a significant burden."

CEO Kim also mentioned that even if the IDMC recommends stopping the trial, since a substantial number of patients have already been enrolled, the trial could effectively continue. He explained, "Only additional patient enrollment would stop, but we would not completely halt everything and leave," and "Currently, 134 patients are enrolled, close to the planned 152, and although it is rare, a reversal story with positive results could emerge from analyzing these patients."