Israel-based clinical-stage biotechnology firm VBL Therapeutics has received fast-track designation from the US Food and Drug Administration (FDA) for its lead oncology drug VB-111, for prolonging survival of patients with recurrent glioblastoma multiforme (rGBM).
In the US and Europe, VB-111 was already granted orphan drug status for GBM, an aggressive form of brain tumour that carries a very poor prognosis with the existing therapy.
Given as a simple IV infusion, VB-111 is a novel gene-therapy drug that targets endothelial cells in the tumour vasculature, acting as a biological knife.
The company said that based on a non-replicating adenoviral vector, VB-111 harbours a proprietary promoter which regulates transcription of a Fas-Chimera transgene, leading to targeted cell-death of endothelial cells in tumour-feeding blood vessels, with no harm to normal vasculature and non-cancerous tissues in the body.
It is claimed to be the first agent based on transcriptional targeting of cancer endothelium to be assessed in a clinical trial.
In the recently released Phase I/II trial results, VB-111 showed safety and tolerability in patients with advanced metastatic cancer at a single administration.
In the trial, tumour response and superior overall survival were found in the 1×1013 VPs cohort compared to sub-therapeutic doses.
The company has also reported Phase II clinical trial results of VB-111 in 28 patients with recurrent glioblastomam, which showed that the drug is safe and well-tolerated with repeat doses of up to 1×1013 VPs.
In the Phase II trial, tumour responses and significant attenuation of tumour growth rate were also seen, while overall survival was one year, which is at least three months longer than historical data in rGBM with the standard of care with chemotherapy and anti-angiogenic agents.
VBL CEO Dror Harats said: "Building on the promising data from our Phase II trial, we plan ahead for a pivotal trial with VB-111 for rGBM under the ‘Fast-Track’ programme, which should expedite the development of VB-111."
As well as GBM, VB-111 is also assessed in multi-dose Phase II clinical trials for differentiated thyroid cancer and ovarian cancer.
Image: Histopathological image of cerebral glioblastoma. Photo: courtesy of KGH.