EnGeneIC, a Sydney-based clinical stage biopharmaceutical company focused on developing its proprietary EDV nanocell platform for targeted cyto-immunotherapy in cancer, has announced publication of clinical data from a 'first-in-man' trial of a novel microRNA-based cancer therapy enabled by the its EnGeneIC Dream Vector technology.
The results have been published in the The Lancet Oncology.
The article, co-authored by EnGeneIC, the Asbestos Diseases Research Institute (ADRI) and investigators from Northern Cancer Institute/Royal North Shore Hospital, Lifehouse/Royal Prince Alfred Hospital and Concord Repatriation General Hospital in Sydney, showcases the first time that tumor-targeted EDVs(TM) have been used to deliver a nucleic acid-based therapy to patients with cancer.
In the open-label, dose-finding Phase 1 study, EnGeneIC's EDVs loaded with a microRNA miR-16 mimic were administered intravenously and targeted with anti-epidermal growth factor receptor for delivery to tumour cells in an attempt to re-establish normal gene regulation and potentially inhibit tumor growth.
The results demonstrated that gradual escalation from 1x10(9) EDV nanocells/week up to 5x10(9) EDV nanocells/week was well tolerated and accompanied by encouraging early signs of anti-tumour activity.
According to the company, of 22 evaluable patients, one had a prolonged objective tumor response and 15 exhibited disease stabilisation.
Joint-CEOs of EnGeneIC, Jennifer MacDiarmid and Himanshu Brahmbhatt, said, "These results showcase the safety and versatility of our EDV platform. In previous clinical studies, we demonstrated the targeted delivery of small molecules at therapeutic levels, and we have now shown that EDVs can deliver miRNAs safely to patients' tumors, with promising outcomes.
"We are pleased that our technology was instrumental in this major advancement for miRNA therapeutics and for all patients whose tumors may respond to miRNA replacement therapy. Based on successful in vitro and animal studies, we would expect these encouraging human results to apply to other nucleic-acid based approaches, including the potential for our EDVs to deliver functional short interfering RNA (siRNA) to tumors. These studies have also shown that our EDV has the capacity to stimulate the immune system, which has clearly contributed to this encouraging result."