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Subject Topline Results of DPN Phase III Study
Writer ViroMed
Date 2019/09/23

DPN Phase 3 study unblinded

Unexpected pharmacokinetic results cloud interpretation

Next phase III studies needed for confirmation

Investor meeting: 9/24, 8:30AM, Seoul


Helixmith recently 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 follows:

1. 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.

2. Safety: 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.

Next steps:

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 details.