Once touted as Lyrica rival for diabetic nerve pain, Korean biotech's plasmid product implodes in PhIII
The Korean biotech industry’s bad year just got worse as one of its highly touted members admitted an embarrassing late-stage flop.
Helixmith — known as ViroMed just a few months ago — had much going for it heading into the 507-patient Phase III: The FDA had granted VM202 orphan drug and fast track designations, and it even picked up an RMAT badge during the study. Sometimes classified as a gene therapy, VM202 is a plasmid product that encodes for the human hepatocyte growth factor (HGF). Due to its dual neurotrophic and angiogenic properties, the drug was believed to alleviate pain caused by diabetic neuropathy.
The results from the placebo-controlled trial though did not bear that hypothesis out. The primary endpoint around the 3-month change in average pain score was not statistically meaningful, the company reported.
Execs attempted to salvage some investor sympathy by disclosing that some patients in the placebo group was found to carry VM202 (also dubbed Engensis) DNA, while others in the treatment arm had suspiciously low concentrations of the plasmid. Helixmith claims that removing those subjects from the analysis gave different results, but details won’t be available until an investigation into those pharmacokinetic results — led by Vical exec Leonard Fish — is wrapped up.
The company plans to share more with the FDA in December, but post hoc analysis is unlikely to sway regulators until Helixmith can provide some solid data from the two other Phase III trials they are planning.
Those studies will start in the next six months and be completed in early 2020, it said in a statement.
Helixmith saw its shares drop 4.4% on the news, making a small dent in its $3 billion market cap.
Early in VM202’s development, the biotech had heralded its potential to beat Pfizer’s Lyrica, a blockbuster treatment for diabetic nerve pain.
“Existing drugs are mostly pain killers with reported side-effects such as headache, insomnia and depression experienced if taken repeatedly, not to mention the hassle of taking them twice a day for years, even for decades,” R&D director Jong-Mook Kim boasted at the time. “On the contrary, VM202-DPN showed to have excellent therapeutic effects even though it was taken at much lesser frequency – only two injections at 2-week intervals.”
The sore disappointment today echoes a larger trend for Korean biotechs that have built unicorn values on bold promises, only to tumble down on bad data. SillaJen’s recent failure with its oncolytic virus, which shaved off $800 million in market cap, underscored just how much could be at stake. And that followed some longstanding trouble that’s been brewing at bigger players like Hanmi and Samsung BioLogics.
Helixmith’s experimental drug is also being tested for amyotrophic lateral sclerosis, coronary artery disease and foot ulcers associated with diabetes.