News and Announcements
ddRNAi Drug for Hepatitis C Safe and Deliverable Reported in New Scientific Paper
- Published June 28, 2012 11:40PM UTC
- Publisher Wholesale Investor
- Categories Company Updates
Sydney June 28, 2012: Benitec Biopharma today announced that important peer-reviewed preclinical safety and delivery results from the hepatitis C program using Benitec Biopharma’s ddRNAi gene silencing technology licensed to Tacere Therapeutics have been published in the prestigious journal Molecular Therapy. This follows the previously announced publication of the first paper from this program in December last year that demonstrated the capability of the lead compound to efficiently inhibit the hepatitis C virus in model systems.
The authors of the current study included researchers from Tacere, Pfizer and the University of Pennsylvania. They reported that a single intravenous injection of TT-034, Tacere’s second generation anti-hepatitis C virus (HCV) ddRNAi-based drug, produced safe and sustained levels of shRNA in non-human primates (NHPs), the gold standard animal model for human toxicology testing. Furthermore, TT-034 successfully delivers its payload to nearly all of the hepatocytes within the liver. Importantly, none of the 18 animals dosed with the second generation vector demonstrated any signs of toxicity. The authors concluded that on this basis a clinical trial of the drug for treatment of HCV infection is warranted. Specifically, the authors state on the basis of these results: “…clinically feasible doses of TT-034 administered to patients should be sufficient to confer therapeutic benefit.”
The full text of the paper is attached and is available on-line at www.nature.com/mt/journal/vaop/ncurrent/pdf/mt2012119a.pdf
“Clearly, TT-034 is well positioned to move into the clinic. We believe that the unique therapeutic modality has the potential to be transformative in the way patients infected with HCV are treated,” said Dr David Suhy, Tacere’s Research Director and lead author on the paper.
Benitec Biopharma’s CEO, Dr Peter French, said, “These results indicate that TT-034 is likely to be a safe and effective single shot therapeutic for hepatitis C, a debilitating viral disease that affects 2% of the world’s population. The next step of the program is to enter a clinical trial, and we are in active discussion with Tacere’s management to determine the optimal path to the clinic for this key program.”