Phase 3 study unblinded
pharmacokinetic results cloud interpretation
phase III studies needed for confirmation
meeting: 9/24, 8:30AM, Seoul
completed its first Phase 3 study of Engensis (VM202) vs. placebo in 500
subjects with painful diabetic peripheral neuropathy (DPN) and conducted an
unblinded data review meeting in Chicago with internal/external experts. Engensis is a
plasmid DNA product that encodes human hepatocyte growth factor (HGF). Engensis
was administered via intramuscular injection by medical personnel through 4
study visits during the 9 month clinical trial. Engensis has previously been
shown to alleviate neuropathic pain in DPN subjects (Phase 1 and Phase 2 clinical
trials). [WS1] The key findings from the meeting are as
Unexpected pharmacokinetic results: Engensis DNA was found in some placebo group
subjects. Also, in the Engensis group, there were some subjects with Engensis DNA
concentrations that were overly low compared to others in the Engensis group.
With the current information, it is difficult to determine the precise reason
for these results. Helixmith plans to share the details in the CSR (Nov) and
through a meeting (Dec) with the FDA.
Adverse event (AE) rates in the entire Intent-to-Treat (ITT) population were very
low and no serious adverse event (SAE) was determined to be related to the study
medication. Also, the injection site reactions (ISRs) were all Grade 1, except
for one case that was Grade 2. Therefore, the data is consistent with previous
results supporting the safety profile of Engensis.
3. Efficacy: In the ITT population, the primary endpoint
of 3 month pain change was not statistically meaningful. Although the adjusted
ITT group, the group that removes subjects with unexpected pharmacokinetic
results gave different results, it is impossible at the present time to derive accurate
conclusions with respect to the efficacy of Engensis.
1. Investigation team set up: Helixmith has set up a team led by
Leonard Fish, PhD, a Clinical QA expert, to[CF2] run a detailed investigation into the
pharmacokinetic results. The outcome of the investigation will be disclosed only
after the evidence is collected and data is analyzed.
2. Future Phase 3: Two or three phase 3 trials smaller than the
current phase 3 study are being planned to further evaluate the efficacy and
safety of Engensis vs. placebo in DPN subjects. The studies will start in the
next 6 months and are expected to be completed around early 2022.
※ Helixmith will hold a meeting on
September 24th, 8:30 AM at the 4th floor auditorium of the
NH Investment & Securities building (Yeouido-Dong 23-4) and explain the